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Annals of Internal Medicine
Prioritization of Patient-Centered Comparative Effectiveness Research for Osteoarthritis Jennifer M. Gierisch, PhD, MPH; Evan R. Myers, MD, MPH; Kristine M. Schmit, MD, MPH; Douglas C. McCrory, MD, MHS; Remy R. Coeytaux, MD, PhD; Matthew J. Crowley, MD; Ranee Chatterjee, MD, MPH; Amy S. Kendrick, RN, MSN; and Gillian D. Sanders, PhD
Osteoarthritis is a leading cause of disability in the United States. This article describes a prioritized research agenda about osteoarthritis management developed for the Patient-Centered Outcomes Research Institute. Evidence gaps were identified by reviewing existing literature and engaging diverse stakeholders to expand and refine gaps. Stakeholders ranked evidence gaps by importance from their perspectives. Prioritized evidence gaps included the need to determine or evaluate key patient-centered outcomes; optimal duration, intensity, and frequency of nonsurgical interventions; whether the comparative effectiveness of nonsurgical interventions varies by socioeconomic factors; when and how to transition from nonsurgical to surgical interventions; effective ways to engage patients in self-
management and promote long-term behavior change; standardized screening tools that improve early diagnosis; biomechanical strategies that improve symptoms; mechanisms for promoting and delivering coordinated, longitudinal care; and comparative effectiveness of nonsurgical therapies. Searches of PubMed and ClinicalTrials .gov showed many recent and ongoing studies addressing comparative effectiveness of nonsurgical interventions; relatively few of these evaluated treatments across categories (for example, drug therapy vs. weight management) or combined categories of treatment. Few studies addressed other high-priority evidence gaps.
O
Health care costs of OA are high—and are increasing. Between 1997 and 2009, the number of hospitalizations with a primary diagnosis of OA more than doubled to an estimated 921 000 (5). Moreover, the health care costs of inpatient, outpatient, and pharmaceutical treatment have been estimated to be 50% greater than those for persons without the condition (9). Nonsurgical management options for OA include analgesics and anti-inflammatory drugs, weight management, exercise, and physical therapy or a combination of these strategies (such as medication plus exercise). When medication and exercise or physical therapy are not enough to decrease pain and improve quality of life, joint surgery is another option. Each management strategy has tradeoffs. For example, persons with OA are twice as likely as those without to develop peptic ulcers (relative risk, 2.49 [95% CI, 1.37 to 4.51]) and renal disease (relative risk, 2.10 [CI, 1.10 to 4.01]), which are probably associated with high use of anti-inflammatory medications (10). However, evidence is limited on the relative benefits and harms of therapies within each category (medication, physical therapy and exercise, weight loss, or surgery) and there are few direct comparisons of effectiveness, in terms of patient-centered outcomes, among categories or combinations of categories. Given the high prevalence of OA and the effect on functional status, productivity, and quality of life, optimizing treatments to stabilize symptoms, reduce pain, and maintain functional status should be a high priority. The aim of this project was to develop, in collaboration with diverse stakeholders, a prioritized research agenda about OA management that would reduce uncertainty about nonsurgical management options and optimize patientcentered outcomes.
steoarthritis (OA) is a leading form of disability in the United States and can affect weight-bearing and non–weight-bearing joints (1, 2). It can manifest as a generalized condition affecting multiple joint groups or a localized condition affecting only 1 joint. In OA, structural and then symptomatic failure of 1 or more synovial joints occurs with loss and erosion of articular cartilage, subchondral bone alterations, meniscal degeneration, limited synovial inflammatory response, and bone and cartilage overgrowth (3). Physical and psychological factors influence the symptoms of pain and stiffness in the joints affected by OA (4). Diagnosing OA can be complex, usually requiring a complete clinical history, physical examination, laboratory testing, and radiographic studies to help confirm the suspected diagnosis. Approximately 27 million adults in the United States are living with OA of 1 or more joints (5), and this number is projected to increase steadily over the coming decades because of an aging population and an increasing prevalence of obesity (6). In the Framingham Heart Study cohort, the prevalence of symptomatic knee OA has increased by 4.1% among women and 6.0% among men over 20 years (7). Osteoarthritis exacts a tremendous toll on patients and society; it ranks third (behind depression and alcohol misuse) in years lost to disability in the United States (8).
See also: Web-Only Supplement 836 © 2014 American College of Physicians
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Ann Intern Med. 2014;160:836-841. www.annals.org For author affiliations, see end of text. This article was published online first at www.annals.org on 13 May 2014.
Research for Osteoarthritis
METHODS The Appendix Figure (available at www.annals.org) shows our methods, which were previously detailed (11). We identified and appraised recently published systematic reviews; clinical practice guidelines; and documents on future research needs, including an Agency for Healthcare Research and Quality report identifying future research needs for physical therapy for knee OA (12). On the basis of recommended areas for future research from these sources, we developed a draft list of 31 possible evidence gaps. We then engaged a group of 13 stakeholders that included clinical experts and researchers in OA treatment strategies; representatives from federal and nongovernmental funding agencies, relevant professional societies, and related consumer and patient advocacy groups; and health care decision makers and policymakers. The stakeholders provided input on the draft list of evidence gaps. We modified certain gaps and added others on the basis of their feedback and then constructed a relevant analytic framework (Figure). Using a forced-ranking prioritization method (13), stakeholders prioritized the list of possible evidence gaps via Web-based surveys. We then performed 2 database searches to identify recently published and ongoing studies relevant to the future research questions that stakeholders ranked in the top tier. Database queries included a search of PubMed between 16 December 2008 and 16 December 2013 and ClinicalTrials.gov on 16 December 2013. We categorized topic areas and key characteristics of the identified studies. Finally, senior Evidence Synthesis Group investigators proposed appropriate study designs to address the stakeholders’ highest-ranked priority research areas. (Tables 1 and 2 of the Supplement [available at www.annals.org] show the stakeholder panel and search strategies.) Role of the Funding Source
The Patient-Centered Outcomes Research Institute (PCORI) provided funding for this work. The funding source stipulated the topic for prioritization but did not participate in the literature search, determination of study eligibility criteria, data analysis or interpretation, or preparation or approval of the manuscript for publication. Staff from PCORI did review a draft version of the manuscript and provided suggestions to clarify language describing the process in selecting this topic for prioritization.
RESULTS Expansion of Evidence Gaps Through Stakeholder Engagement
The stakeholder panel expanded the initial list of 31 evidence gaps to 48. The final list illustrated several common themes: Stakeholders stressed that future research should focus on engaging patients early in the disease process, helping patients navigate treatment options that optimize patient-centered outcomes (for example, negative www.annals.org
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Research and Reporting Methods
affect, sleep, pain, and employment), and developing strategies that promote successful long-term engagement in nonsurgical treatment options. New questions suggested by the stakeholders addressed a wide range of evidence gaps related to the unique needs of subpopulations and the comparative effectiveness of diverse interventions. Stakeholders acknowledged that several patient groups received less attention than others and that it would be important to assess the comparative effectiveness of nonsurgical management strategies by sex, socioeconomic status, and prevalence and severity of comorbid conditions among adults and patients who had previously had surgical treatment of OA. Stakeholders stressed the progressive and chronic nature of OA and the importance of early detection and patient engagement. They also emphasized the importance of setting up patients for success—that is, that treatments should be tailored to patients’ abilities, insurance coverage, culture, and preferences. Stakeholders acknowledged that many current and ongoing studies focus on short-term outcomes and do not necessarily provide the needed information about longterm outcomes reflecting the chronic nature of the disease. They highlighted the importance of shared decisionmaking tools for not only exploring nonsurgical options but also discussing the optimal timing for transition to surgical interventions. They also stressed the incorporation of patient preferences for specific outcomes and their personal characteristics (and the effect of those characteristics on potential efficacy of treatment options) in such shared decision-making tools. Stakeholder Ranking of Future Research Needs
The Appendix Table (available at www.annals.org) shows the 48 final potential research topics, the number of points each received, and the number of stakeholders who allotted points to each topic. The final ranking divides the possible evidence gaps into a top, middle, and lower tier on the basis of overall score. The top 12 future research needs (Table) prioritized by stakeholders were related to identifying key patient-centered outcomes; determining optimal duration, intensity, and frequency of interventions; examining the effect of socioeconomic differences on management strategies; determining the need for transition from nonsurgical to surgical interventions; promoting long-term behavior change; standardizing screening tools; evaluating biomechanical strategies; developing strategies to help patients engage in key self-management behaviors; promoting coordinated care in today’s new health care delivery system; comparing methods for identifying and engaging patients early in the disease process; comparing safety and effectiveness of usual-care nonsurgical therapies; and identifying ways for setting patients up to succeed with nonsurgical management. Although the stakeholder group expanded the initial list to 48 potential future research needs, 18 of the final questions received no votes (5 questions) or 1 to 2 votes by 17 June 2014 Annals of Internal Medicine Volume 160 • Number 12 837
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Figure. Analytic framework.
Effect of population factors (FRNs 1–6) Sociodemographic differences Differences in comorbid conditions Previous surgical intervention Prevalence/severity of comorbid conditions Comedications Disease aspects of OA (e.g., severity, functional limitations, location, multijoint)
Promotion of adherence and maintenance of effects (FRNs 30–38) Patient engagement tools/ prompts Follow-up treatments Financial incentives Self-management strategies Strategies for long-term behavior change Nonsurgical management strategies Cultural group differences Socioeconomic differences Supervised exercise program
Effect of outcome measurement factors (FRNs 45–48) Validity/reliability of patient-centered outcome measures Validity/reliability of intermediate outcome measures Defining intermediate outcomes Confounding variables
Decision-making interventions (FRNs 39–40) Decision-making tools Patients raising issue
Population: adults with OA
Identification and screening (FRNs 7–18) Patient management of OA Standardized screening tools Sensitivity/specificity of evaluations Strategies for engaging patients Optimal ways to track and identify risk factors for OA OA-specific barriers to physical activity Functional ability self-assessment tool Walkability audit/checklist Different models of health care Optimal timing for interventions Threshold level of symptoms Need for transition from nonsurgical to surgical interventions
Therapeutic interventions (FRNs 19–29) Nonsurgical therapies Physical therapy Orthotics Self-administered transcutaneous electrostimulation Biomechanical strategies Complementary and alternative approaches Individual treatment methods Method of delivery Duration/intensity/frequency Sequenced weight loss program Setting up patients to succeed
Defining patient-centered outcomes (FRN 44) Sleep Negative affect/depression/worry Delay to surgery Reduction in medications Pain/independence in ADLs/ instrumental ADLs Patient satisfaction Time to return to work/activities Quality of life Reliability of pain treatment
Adverse effects of therapeutic interventions
Health care, policy, and ecological approaches (FRNs 41–43) Promoting care available in today’s new health care delivery system Policy and environmental strategies to promote physical activity Insurance coverage
Italicized text indicates top-tier gaps. ADLs ⫽ activities of daily living; FRN ⫽ future research need; OA ⫽ osteoarthritis.
at most 2 stakeholders (13 questions). Of the 12 questions ranked as highest priority, 2 were new questions that stakeholders suggested (questions focusing on the transition from nonsurgical to surgical interventions and on methods for setting patients up to succeed with nonsurgical management). The other 10 high-priority ranked questions were part of the original list of suggested future research needs. Of the 18 questions ranked low priority, 9 were from our original list and 9 were suggested by stakeholders. 838 17 June 2014 Annals of Internal Medicine Volume 160 • Number 12
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Horizon Scan of Studies Potentially Relevant to Top-Tier Research Questions
The PubMed search identified 4692 articles. Of these, 427 met inclusion criteria, including 92 systematic reviews; 254 randomized, controlled trials; 41 cohort studies; 3 case– control studies; and 37 studies of other design types. Most studies (n ⫽ 339) pertained to the comparative effectiveness of available nonsurgical therapies (228 randomized, controlled trials and 79 systematic reviews). Within www.annals.org
Research for Osteoarthritis
this set of 339 studies, 119 explored the safety and effectiveness of oral medications (29 systematic reviews; 84 randomized, controlled trials; 3 cohort studies; and 3 other studies). All but 20 of these evaluated oral medications compared with another oral medication or placebo. Relatively few studies evaluated treatments across categories (for example, drug therapy compared with weight management) or combined categories of treatment (for example, drug therapy plus exercise compared with drug therapy only). There were few studies for most of the other high-priority evidence gaps, with only 3 studies targeting ways of determining the need to transition from nonsurgical to surgical interventions and only 7 studies investigating strategies for identifying and engaging patients early in the disease process. The ClinicalTrials.gov search yielded 153 open, potentially relevant protocols. Of these, 55 were categorized as potentially relevant to the top-tier research questions. Sample sizes of these studies ranged from 8 to 20 000 patients, and estimated completion dates of the studies ranged from currently overdue to June 2017. (Tables 3 to 14 of the Supplement show key characteristics of the re-
Research and Reporting Methods
cent and ongoing studies relevant to the 12 top-tier future research needs.) Suggested Research Design Considerations
The Table summarizes the Evidence Synthesis Group’s suggestions for appropriate study designs or suggested analyses to address the 12 top-tier research priorities. Proposed designs and analyses to address specific gaps included meta-analysis of individual-patient data, randomized trials, and observational studies requiring collection of new data. Pragmatic clinical trials (14, 15) may be particularly useful for several of these research priorities, as PCORI’s recent funding announcement emphasized (16). (Table 15 of the Supplement provides additional details about possible advantages and disadvantages of multiple study designs for each gap.)
DISCUSSION Osteoarthritis is a common chronic condition that leads to functional limitations, pain, disability, and lost earnings and is associated with high utilization of health care (17, 18). A key theme that emerged from our research
Table. Top-Ranked Research Priorities and Recommended Study Designs or Suggested Analyses to Address Them Rank
Prioritized Future Research Need
Recommended Study Design or Suggested Analyses
1
What are the most important patient-centered outcomes (e.g., sleep, negative affect/depression/worry, delay to surgery, reduction in medications, pain/independence in ADLs/instrumental ADLs, patient satisfaction, time to return to work/activities or other employment outcomes, quality of life, and reliability of pain treatment) for patients with foot, ankle, knee, or hip OA? What are the optimal duration, intensity, and frequency of examined nonsurgical interventions for OA to create sustained changes in patient-centered outcomes? Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression and disability from OA differ by sociodemographic differences (e.g., age, sex, race/ethnicity, SES, insurance status, access to health care, and having a physically demanding occupation)? How do these strategies differ in specific underrepresented patient populations (i.e., those with low literacy level, low SES, or less access to health care)? What are effective ways (e.g., checklist of functionality/symptoms) for patients or providers to determine the need for the transition from nonsurgical to surgical interventions for OA? What are the comparative safety and effectiveness of strategies promoting long-term behavior change (e.g., weight management and physical activity) in the context of chronic pain and functional limitations associated with OA? Are there potential standardized screening tools and indicators of OA that can improve early diagnosis of OA?
Observational study requiring new data collection
2 3
4 5
6
7 8
9 10
11
12
What are the comparative safety and effectiveness of biomechanical strategies to improve OA symptoms and slow progression of disease? What are the comparative safety and effectiveness of strategies to help patients engage in key self-management behaviors for managing OA (physical activity and weight management) in real-world settings (community and primary care)? What opportunities for promoting coordinated, proactive, longitudinal, long-term care for OA are now available in today’s new health care delivery system? What are the comparative safety and effectiveness of strategies for identifying and engaging patients early in the disease process, particularly fostering healthy behaviors (physical activity and weight management), to prevent progression of and disability from OA? What are the comparative safety and effectiveness of usual care nonsurgical therapies (pharmacotherapy, injections, physical therapy/exercise, and weight loss) or combinations of usual care nonsurgical therapies to prevent progression of and disability from OA? Are these effects maintained (i.e., long-term outcomes) over time? How do we best set up patients to succeed with nonsurgical management for OA, incorporating available options and their specific potential barriers for an individual patient?
RCT RCT and/or meta-analysis of individual-patient data analysis across RCTs
RCT or modeling study RCT
Observational study using existing data or involving new data collection RCT RCT
Observational study involving new data collection RCT
RCT and/or meta-analysis of individual-patient data analysis across RCTs RCT and/or meta-analysis of individual-patient data analysis across RCTs
ADLs ⫽ activities of daily living; OA ⫽ osteoarthritis; RCT ⫽ randomized, controlled trial; SES ⫽ socioeconomic status. www.annals.org
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prioritization process is that future research priorities should focus on attending to patients’ needs across the spectrum of the disease, from identification through treatment decisions on the transition from nonsurgical to surgical OA interventions. The most highly ranked research needs focused on identifying the most important patient-centered outcomes and optimizing attributes of nonsurgical intervention (that is, duration, intensity, and frequency) that create sustained changes in patient-centered outcomes. Each nonsurgical treatment method may present tradeoffs between effectiveness in terms of optimizing patient-centered outcomes and other important attributes of treatments (for example, time required for frequent clinician visits and sacrifices involved in dietary modification) that may affect patients’ willingness to adopt and maintain treatment options. Thus, direct measurement of patient preferences for different attributes of interventions (including effectiveness and duration, intensity, and frequency) would be highly useful in designing comparative effectiveness studies. The literature search and the review of ongoing research in ClinicalTrials.gov revealed that, although a substantial amount of ongoing research is focused on the comparative safety and effectiveness of usual-care nonsurgical therapies (with most focusing on pharmacotherapies), relatively little work is focused on identifying the most important patient-centered outcomes and how these may map to attributes of nonsurgical treatment strategies. Many of the other high-priority research needs focused on developing better approaches to help enhance longterm patient engagement in strategies that may prevent the progression of and disability from OA. This factor is reflected in the high ranking of future research needs that focus on promoting long-term behavior change, developing strategies to help patients engage in key selfmanagement behaviors for OA, promoting coordinated OA care in today’s new health care delivery system, comparing methods for identifying and engaging patients early in the disease process, and identifying methods for setting patients up to succeed with nonsurgical management. Osteoarthritis is a chronic illness, so focusing on strategies that promote continuing engagement is a practical approach to optimizing outcomes. Our search of the literature and ongoing studies identified few studies evaluating early engagement of patients, methods for setting patients up for success, and coordinating OA care in today’s new health care delivery system. Stakeholders stress that strategies tailored to patients’ abilities, insurance, culture, and preferences may enhance the success of OA interventions in the long term. The list of future research needs that we generated and expanded with stakeholder feedback may not reflect the total range of possible future research. Although we included a diverse group of stakeholders with particular interest in patient-centered research, the panel involved relatively few stakeholders. Another group of stakeholders 840 17 June 2014 Annals of Internal Medicine Volume 160 • Number 12
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might add other future research needs or rank the identified future research needs differently. We did not conduct comprehensive systematic reviews pertinent to the identified evidence gaps; therefore, we could not determine with certainty the degree to which prioritized future research needs have already been addressed. To advance a patient-centered research agenda, future research should focus on addressing patients’ needs across the disease spectrum and enhancing long-term engagement in strategies that stabilize the symptoms of OA. A panel of stakeholders representing diverse perspectives identified 12 research areas as the highest priorities for future research of patient-centered management strategies for persons with OA. If studied, these 12 research areas may enhance the patient experience of managing OA symptoms and enable patients to optimize their most valued health outcomes. From the Center for Health Services Research in Primary Care, Durham Veterans Affairs Medical Center, Duke Evidence Synthesis Group, Duke Clinical Research Institute, and Duke University School of Medicine, Durham, North Carolina. Acknowledgment: The authors thank Megan M. Chobot, MSLS, for project coordination, and Liz Wing, MA, for editorial assistance. They also thank the members of the stakeholder panel: Kelli Allen, PhD; Teresa Brady, PhD; G. Kelley Fitzgerald, PhD; Yvonne M. Golightly, PhD; Lyndon Joseph, PhD; Jeffrey Katz, MD; Gayle Lester, PhD; Dave Mekemson; Paul Rockar, DPT; Jyme Schafer, MD; Nancy Simington; Kim Templeton, MD; and Patience White, MD. Financial Support: By PCORI. Disclosures: Disclosures can be viewed at www.acponline.org/authors /icmje/ConflictOfInterestForms.do?msNum⫽M14-0318. Requests for Single Reprints: Jennifer M. Gierisch, PhD, MPH, De-
partment of Medicine, Duke University Medical Center, Campus Box 104427 DUMC, Durham, NC 27701; e-mail, [email protected]. Current author addresses and author contributions are available at www.annals.org.
References 1. Felson DT, Lawrence RC, Dieppe PA, Hirsch R, Helmick CG, Jordan JM, et al. Osteoarthritis: new insights. Part 1: the disease and its risk factors. Ann Intern Med. 2000;133:635-46. [PMID: 11033593] 2. Dillon CF, Rasch EK, Gu Q, Hirsch R. Prevalence of knee osteoarthritis in the United States: arthritis data from the Third National Health and Nutrition Examination Survey 1991-94. J Rheumatol. 2006;33:2271-9. [PMID: 17013996] 3. Nuki G. Osteoarthritis: a problem of joint failure. Z Rheumatol. 1999;58: 142-7. [PMID: 10441841] 4. Rosemann T, Laux G, Szecsenyi J, Wensing M, Grol R. Pain and osteoarthritis in primary care: factors associated with pain perception in a sample of 1,021 patients. Pain Med. 2008;9:903-10. [PMID: 18702636] 5. Murphy L, Helmick CG. The impact of osteoarthritis in the United States: a population-health perspective. Am J Nurs. 2012;112:S13-9. [PMID: 22373741] 6. Lawrence RC, Felson DT, Helmick CG, Arnold LM, Choi H, Deyo RA, et al; National Arthritis Data Workgroup. Estimates of the prevalence of arthritis www.annals.org
Research for Osteoarthritis and other rheumatic conditions in the United States. Part II. Arthritis Rheum. 2008;58:26-35. [PMID: 18163497] 7. Nguyen US, Zhang Y, Zhu Y, Niu J, Zhang B, Felson DT. Increasing prevalence of knee pain and symptomatic knee osteoarthritis: survey and cohort data. Ann Intern Med. 2011;155:725-32. [PMID: 22147711] 8. Michaud CM, McKenna MT, Begg S, Tomijima N, Majmudar M, Bulzacchelli MT, et al. The burden of disease and injury in the United States 1996. Popul Health Metr. 2006;4:11. [PMID: 17049081] 9. Lee DW, Meyer JW, Clouse J. Implications of controlling for comorbid conditions in cost-of-illness estimates: a case study of osteoarthritis from a managed care system perspective. Value Health. 2001;4:329-34. [PMID: 11705300] 10. Gabriel SE, Crowson CS, O’Fallon WM. Comorbidity in arthritis. J Rheumatol. 1999;26:2475-9. [PMID: 10555912] 11. Gierisch JM, Myers ER, Schmit KM, Crowley MJ, McCrory DC, Chatterjee R, et al. Prioritization of research addressing management strategies for ductal carcinoma in situ. Ann Intern Med. 2014;160:484-91. [PMID: 24567146] 12. Brasure M, Shamliyan TA, Olson-Kellogg B, Butler ME, Kane RL. AHRQ comparative effectiveness reviews. Physical therapy for knee pain secondary to osteoarthritis: future research needs: identification of future research needs from comparative effectiveness review no. 77. Report no. 13-EHC048-EF. Rockville, MD: Agency for Healthcare Research and Quality; 2013. [PMID: 23905194] 13. Chang SM, Carey TS, Kato EU, Guise JM, Sanders GD. Identifying research needs for improving health care. Ann Intern Med. 2012;157:439-45. [PMID: 22847017]
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14. Thorpe KE, Zwarenstein M, Oxman AD, Treweek S, Furberg CD, Altman DG, et al. A pragmatic-explanatory continuum indicator summary (PRECIS): a tool to help trial designers. CMAJ. 2009;180:E47-57. [PMID: 19372436] 15. Patsopoulos NA. A pragmatic view on pragmatic trials. Dialogues Clin Neurosci. 2011;13:217-24. [PMID: 21842619] 16. Patient-Centered Outcomes Research Institute. Pragmatic clinical studies and large simple trials to evaluate patient-centered outcomes. 2014. Accessed at www.pcori.org/funding-opportunities/funding-announcements/pragmatic -clinical-studies-and-large-simple-trials-to-evaluate-patient-centered-outcomes on 17 April 2014. 17. Yelin E, Murphy L, Cisternas MG, Foreman AJ, Pasta DJ, Helmick CG. Medical care expenditures and earnings losses among persons with arthritis and other rheumatic conditions in 2003, and comparisons with 1997. Arthritis Rheum. 2007;56:1397-407. [PMID: 17469096] 18. Centers for Disease Control and Prevention. NHIS arthritis surveillance: specific functional limitations among people with arthritis. Accessed at www.cdc .gov/arthritis/data_statistics/national_nhis.htm#functional on 5 February 2014. 19. Ip S, Raman G, Concannon TW, Ratichek SJ, Yu W, Chang LKW, et al. Intravascular Diagnostic Procedures and Imaging Techniques Versus Angiography Alone in Coronary Artery Stenting: Future Research Needs. Future Research Needs Paper no. 25. (Prepared by Tufts Evidence-based Practice Center under contract no. 290-2007-10055-I.) AHRQ publication no. 13-EHC016-EF. Rockville, MD: Agency for Healthcare Research and Quality; 2013. Accessed at www.effectivehealthcare.ahrq.gov/ehc/products/512/1413/coronary-artery -stenting-future-130604.pdf on 23 January 2014.
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Annals of Internal Medicine Current Author Addresses: Dr. Gierisch: Department of Medicine, Duke University Medical Center, Campus Box 104427 DUMC, Durham, NC 27701. Dr. Myers: Duke University Medical Center, Box 3279, 244 Baker House, Durham, NC 27710. Drs. Schmit, McCrory, Coeytaux, and Sanders and Ms. Kendrick: Duke Clinical Research Institute, PO Box 17969, Durham, NC 27715. Dr. Crowley: Durham Veterans Affairs Medical Center, Health Services Research and Development (152), 508 Fulton Street, Durham, NC 27705. Dr. Chatterjee: Sutton Station Internal Medicine, 5832 Fayetteville Road, Suite 113, Durham, NC 27713.
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Author Contributions: Conception and design: J.M. Gierisch, R.R.
Coeytaux, M.J. Crowley, G.D. Sanders. Analysis and interpretation of the data: J.M. Gierisch, E.R. Myers, D.C. McCrory, M.J. Crowley, R. Chatterjee, G.D. Sanders. Drafting of the article: J.M. Gierisch, R.R. Coeytaux, G.D. Sanders. Critical revision of the article for important intellectual content: J.M. Gierisch, E.R. Myers, M.J. Crowley, G.D. Sanders. Final approval of the article: J.M. Gierisch, E.R. Myers, D.C. McCrory, R.R. Coeytaux, M.J. Crowley, R. Chatterjee, A.S. Kendrick, G.D. Sanders. Provision of study materials or patients: A.S. Kendrick, G.D. Sanders. Statistical expertise: G.D. Sanders. Obtaining of funding: G.D. Sanders. Administrative, technical, or logistic support: J.M. Gierisch, A.S. Kendrick, G.D. Sanders. Collection and assembly of data: J.M. Gierisch, E.R. Myers, K.M. Schmit, D.C. McCrory, R.R. Coeytaux, M.J. Crowley, R. Chatterjee, A.S. Kendrick, G.D. Sanders.
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Appendix Figure. Overview of prioritization process. Steps
Output/Products
Identification of evidence gaps Review systematic reviews, clinical practice guidelines, and consensus statements for evidence gaps; structure evidence gaps into research questions Initial list of evidence gaps and draft analytic framework Selection of stakeholders Send stakeholders background materials and initial list of evidence gaps Establish stakeholder group Engage stakeholders via Webinar/ teleconference/e-mail Introduction to process; refine and expand initial evidence gaps Expanded list of evidence gaps and revised analytic framework Stakeholders participate in Web-based prioritization process Stakeholders participate in forced-ranking prioritization method used to facilitate prioritization of evidence gaps
Inform stakeholders of prioritized list and next steps
Conduct horizon scan of studies potentially relevant to top-tier research questions ESG investigators conduct PubMed and ClinicalTrials.gov searches for recent or ongoing relevant studies for the top-tier prioritized future research needs
Explore research question development and research design considerations ESG investigators consider advantages and disadvantages of various potential study designs
Produce future research needs report for PCORI ESG investigators integrate results of the prioritization process, horizon scan, and research design consideration into a report for PCORI leadership
Stakeholder-identified prioritized list of future research needs
Detailed list of recent published studies or ongoing research relevant to top-tier future research needs
List of suggested study designs to address top-tier future research needs
Future research needs report
Adapted from reference 19. ESG ⫽ Evidence Synthesis Group; PCORI ⫽ Patient-Centered Outcomes Research Institute.
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Appendix Table. Final Ranking of Future Research Needs for Alternative Strategies for OA* Question Top tier What are the most important patient-centered outcomes (e.g., sleep, negative affect/depression/worry, delay to surgery, reduction in medications, pain/independence in ADLs/instrumental ADLs, patient satisfaction, time to return to work/activities or other employment outcomes, quality of life, and reliability of pain treatment) for patients with foot, ankle, knee, or hip OA? What are the optimal duration, intensity, and frequency of examined nonsurgical interventions for OA to create sustained changes in patient-centered outcomes? Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression and disability from OA differ by sociodemographic differences (e.g., age, sex, race/ethnicity, SES, insurance status, access to health care, and having a physically demanding occupation)? How do these strategies differ in specific underrepresented patient populations (i.e., those with low literacy level, low SES, or less access to health care)? What are effective ways (e.g., checklist of functionality/symptoms) for patients or providers to determine the need for the transition from nonsurgical to surgical interventions for OA? What are the comparative safety and effectiveness of strategies promoting long-term behavior change (e.g., weight management and physical activity) in the context of chronic pain and functional limitations associated with OA? Are there potential standardized screening tools and indicators of OA that can improve early diagnosis of OA? What are the comparative safety and effectiveness of biomechanical strategies to improve OA symptoms and slow progression of disease? What are the comparative safety and effectiveness of strategies to help patients engage in key self-management behaviors for managing OA (physical activity and weight management) in real-world settings (community and primary care)? What opportunities for promoting coordinated, proactive, longitudinal, long-term care for OA are now available in today’s new health care delivery system? What are the comparative safety and effectiveness of strategies for identifying and engaging patients early in the disease process, particularly fostering healthy behaviors (physical activity and weight management), to prevent progression of and disability from OA? What are the comparative safety and effectiveness of usual care nonsurgical therapies (pharmacotherapy, injections, physical therapy/exercise, and weight loss) or combinations of usual care nonsurgical therapies to prevent progression of and disability from OA? Are these effects maintained (i.e., long-term outcomes) over time? How do we best set up patients to succeed with nonsurgical management for OA incorporating available options and their specific potential barriers for an individual patient? Middle tier Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression and disability from OA differ by disease aspects of OA (e.g., OA symptom severity, functional limitations, location of OA, and multijoint OA)? Because OA is a progressive disease, what is the optimal timing for nonsurgical (e.g., physical therapy, injections, NSAIDs, acetaminophen, and opioids) or surgical interventions to enhance outcomes? What are the comparative safety and effectiveness of models of organizing health care that lead to identifying and engaging patients early in the disease process? What are the comparative safety and effectiveness of comprehensive multimodal physical therapy treatments for OA on patient-centered outcomes when compared with exercise alone? Are these effects maintained (i.e., long-term outcomes) over time? What are the comparative safety and effectiveness of strategies to increase patient engagement in nonsurgical management strategies to prevent progression of and disability from OA? What are valid and reliable instruments used to measure the following patient-centered outcomes for persons with OA: pain/independence in ADLs/instrumental ADLs, patient satisfaction, time to return to work/activities, quality of life, community integration, psychological disability, and self-perceived health? What is the minimum clinically important difference for each of these instruments? What cut points should be used to describe clinically meaningful categories in the scale scores created by these instruments? What are effective ways to track and identify OA risk factors and OA progression/outcomes (e.g., electronic health records)? What are effective ways for health care professionals to assess OA-specific barriers to physical activity (as a routine component of “vital signs”) when screening their adult patients? Which intermediate outcomes meet the criteria for surrogate patient-centered outcomes for persons with OA? What are the comparative sensitivity and specificity of biomedical evaluations (genetic markers and imaging evaluations) for detecting OA among adult patients at risk? Do differences in accuracy lead to clinically relevant differences in choice of therapeutic interventions? Do the comparative safety and effectiveness of physical therapy/exercise management strategies for OA on patient-centered outcomes differ by method of delivery (e.g., the involvement of a physical therapist or physical therapist assistant, group vs. individual exercise, self-administered vs. supervised exercises, electronic vs. in-person delivery)? In patients with OA, what individual characteristics can predict who first needs to receive a professionally supervised exercise program vs. who does not before engaging in an independent physical activity program to maximize benefits and long-term participation in physical activity programs? What are the comparative safety and effectiveness of patient-engagement tools and prompts that promote learning about and management of OA (and pre-OA)? What is the effect of policy, human resources, and environmental strategies on physical activity behaviors among adults with OA? Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression of and disability from OA differ by comorbid conditions (including obesity)? What are the prevalence and severity of comorbid conditions among adults with OA?
Score
Stakeholders, n
13
8
12
9
9
7
8
7
8
6
7 7
6 6
7
6
7
6
7
5
7
5
7
5
5
5
5
5
5
4
5
4
5
4
5
4
4 4
4 4
4 4
4 3
4
3
4
3
4
2
4 3
2 3
3
3
Continued on following page
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Appendix Table—Continued Question What are the valid and reliable instruments used to measure the following intermediate outcomes when evaluating the comparative safety and effectiveness of physical therapy interventions for foot, ankle, knee, or hip pain secondary to OA: joint function, swelling, inflammation, gait function, strength, and transfers? At what point along the continuum of symptoms, functionality, and risk for OA do patients become actively engaged in managing their OA/reducing their risk for progression? Lower tier Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression of and disability from OA differ for patients who have previously had a surgical intervention for OA? What are effective functional ability self-assessment tools or programmatic approaches for adults with OA that will allow patients to tailor physical activity accordingly? How do the comparative safety and effectiveness of current adherence and maintenance strategies for nonsurgical management of OA differ by cultural groups? What are the effects of decision-making tools compared with usual care to assist patients with shared decision-making processes with the primary care provider about OA treatments (e.g., medication use, joint injections, physical therapy, and joint replacement surgery), with a focus on persons with low health literacy and limited health access compared with usual care? What are the optimal format, content, and timing for these? Do periodic follow-up treatments beyond the initial physical therapy treatments of OA enhance effectiveness of these treatments? How do we empower patients to raise the issues of their joint pain and functional limitations with their health care professional? What are effective walkability audits/checklists that are specifically relevant for adults with OA? Should there be some threshold level of symptoms or functionality that justifies the transition from nonsurgical to surgical interventions for OA? What are the comparative safety and effectiveness of orthotics in patients with (foot, ankle, knee, and hip) OA to prevent progression of and disability from lower-body OA? What are the comparative safety and effectiveness of a sequenced weight loss and physical activity approach (weight loss program administered first, followed by initiation of physical activity when minimal weight loss criteria have been achieved) vs. the simultaneous administration of weight loss and physical activity in reducing pain and disability and promoting long-term physical activity behavior in patients with OA and obesity? What is the role of financial incentives to enhance effectiveness, adherence, and maintenance of nonsurgical management strategies for OA? How do the comparative safety and effectiveness of current adherence and maintenance strategies for nonsurgical management of OA differ by socioeconomic groups? What confounding variables (e.g., adherence, weight loss, and activity levels) and effect modifiers, including concomitant therapies, should be controlled for when examining patient-centered outcomes for persons with OA? Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression of and disability from OA differ by comedications (e.g., anticoagulants) in the population of interest? What are the comparative safety and effectiveness of self-administered transcutaneous electrostimulation compared with other usual care nonpharmacologic or pharmacologic approaches for patients with (foot, ankle, knee, and hip) OA to prevent progression of and disability from OA? What are the comparative safety and effectiveness of complementary/alternative medicine strategies (e.g., plant and herbal therapies, electroacupuncture, needle acupuncture, and balneotherapy) compared with other usual care nonpharmacologic approaches for patients with (foot, ankle, knee, and hip) OA to prevent progression of and disability from OA? Are these effects maintained (i.e., long-term outcomes) over time? What is the benefit from individual treatment methods (e.g., heat and ice) delivered for OA? How does coverage by insurance of nonsurgical management strategies to prevent progression of and disability from OA affect the comparative effectiveness and/or adherence of available strategies?
Score
Stakeholders, n
3
3
3
2
2
2
2
2
2
2
2
2
2 2
1 1
1 1
1 1
1
1
1
1
1
1
1
1
1
1
0
0
0
0
0
0
0 0
0 0
ADLs ⫽ activities of daily living; OA ⫽ osteoarthritis; NSAID ⫽ nonsteroidal anti-inflammatory drug; SES ⫽ socioeconomic status. * Italicized text indicates new questions based on feedback from the stakeholder group.
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Annals of Internal Medicine
Prioritization of Patient-Centered Comparative Effectiveness Research for Osteoarthritis Jennifer M. Gierisch, PhD, MPH; Evan R. Myers, MD, MPH; Kristine M. Schmit, MD, MPH; Douglas C. McCrory, MD, MHS; Remy R. Coeytaux, MD, PhD; Matthew J. Crowley, MD; Ranee Chatterjee, MD, MPH; Amy S. Kendrick, RN, MSN; and Gillian D. Sanders, PhD
Osteoarthritis is a leading cause of disability in the United States. This article describes a prioritized research agenda about osteoarthritis management developed for the Patient-Centered Outcomes Research Institute. Evidence gaps were identified by reviewing existing literature and engaging diverse stakeholders to expand and refine gaps. Stakeholders ranked evidence gaps by importance from their perspectives. Prioritized evidence gaps included the need to determine or evaluate key patient-centered outcomes; optimal duration, intensity, and frequency of nonsurgical interventions; whether the comparative effectiveness of nonsurgical interventions varies by socioeconomic factors; when and how to transition from nonsurgical to surgical interventions; effective ways to engage patients in self-
management and promote long-term behavior change; standardized screening tools that improve early diagnosis; biomechanical strategies that improve symptoms; mechanisms for promoting and delivering coordinated, longitudinal care; and comparative effectiveness of nonsurgical therapies. Searches of PubMed and ClinicalTrials .gov showed many recent and ongoing studies addressing comparative effectiveness of nonsurgical interventions; relatively few of these evaluated treatments across categories (for example, drug therapy vs. weight management) or combined categories of treatment. Few studies addressed other high-priority evidence gaps.
O
Health care costs of OA are high—and are increasing. Between 1997 and 2009, the number of hospitalizations with a primary diagnosis of OA more than doubled to an estimated 921 000 (5). Moreover, the health care costs of inpatient, outpatient, and pharmaceutical treatment have been estimated to be 50% greater than those for persons without the condition (9). Nonsurgical management options for OA include analgesics and anti-inflammatory drugs, weight management, exercise, and physical therapy or a combination of these strategies (such as medication plus exercise). When medication and exercise or physical therapy are not enough to decrease pain and improve quality of life, joint surgery is another option. Each management strategy has tradeoffs. For example, persons with OA are twice as likely as those without to develop peptic ulcers (relative risk, 2.49 [95% CI, 1.37 to 4.51]) and renal disease (relative risk, 2.10 [CI, 1.10 to 4.01]), which are probably associated with high use of anti-inflammatory medications (10). However, evidence is limited on the relative benefits and harms of therapies within each category (medication, physical therapy and exercise, weight loss, or surgery) and there are few direct comparisons of effectiveness, in terms of patient-centered outcomes, among categories or combinations of categories. Given the high prevalence of OA and the effect on functional status, productivity, and quality of life, optimizing treatments to stabilize symptoms, reduce pain, and maintain functional status should be a high priority. The aim of this project was to develop, in collaboration with diverse stakeholders, a prioritized research agenda about OA management that would reduce uncertainty about nonsurgical management options and optimize patientcentered outcomes.
steoarthritis (OA) is a leading form of disability in the United States and can affect weight-bearing and non–weight-bearing joints (1, 2). It can manifest as a generalized condition affecting multiple joint groups or a localized condition affecting only 1 joint. In OA, structural and then symptomatic failure of 1 or more synovial joints occurs with loss and erosion of articular cartilage, subchondral bone alterations, meniscal degeneration, limited synovial inflammatory response, and bone and cartilage overgrowth (3). Physical and psychological factors influence the symptoms of pain and stiffness in the joints affected by OA (4). Diagnosing OA can be complex, usually requiring a complete clinical history, physical examination, laboratory testing, and radiographic studies to help confirm the suspected diagnosis. Approximately 27 million adults in the United States are living with OA of 1 or more joints (5), and this number is projected to increase steadily over the coming decades because of an aging population and an increasing prevalence of obesity (6). In the Framingham Heart Study cohort, the prevalence of symptomatic knee OA has increased by 4.1% among women and 6.0% among men over 20 years (7). Osteoarthritis exacts a tremendous toll on patients and society; it ranks third (behind depression and alcohol misuse) in years lost to disability in the United States (8).
See also: Web-Only Supplement 836 © 2014 American College of Physicians
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Ann Intern Med. 2014;160:836-841. www.annals.org For author affiliations, see end of text. This article was published online first at www.annals.org on 13 May 2014.
Research for Osteoarthritis
METHODS The Appendix Figure (available at www.annals.org) shows our methods, which were previously detailed (11). We identified and appraised recently published systematic reviews; clinical practice guidelines; and documents on future research needs, including an Agency for Healthcare Research and Quality report identifying future research needs for physical therapy for knee OA (12). On the basis of recommended areas for future research from these sources, we developed a draft list of 31 possible evidence gaps. We then engaged a group of 13 stakeholders that included clinical experts and researchers in OA treatment strategies; representatives from federal and nongovernmental funding agencies, relevant professional societies, and related consumer and patient advocacy groups; and health care decision makers and policymakers. The stakeholders provided input on the draft list of evidence gaps. We modified certain gaps and added others on the basis of their feedback and then constructed a relevant analytic framework (Figure). Using a forced-ranking prioritization method (13), stakeholders prioritized the list of possible evidence gaps via Web-based surveys. We then performed 2 database searches to identify recently published and ongoing studies relevant to the future research questions that stakeholders ranked in the top tier. Database queries included a search of PubMed between 16 December 2008 and 16 December 2013 and ClinicalTrials.gov on 16 December 2013. We categorized topic areas and key characteristics of the identified studies. Finally, senior Evidence Synthesis Group investigators proposed appropriate study designs to address the stakeholders’ highest-ranked priority research areas. (Tables 1 and 2 of the Supplement [available at www.annals.org] show the stakeholder panel and search strategies.) Role of the Funding Source
The Patient-Centered Outcomes Research Institute (PCORI) provided funding for this work. The funding source stipulated the topic for prioritization but did not participate in the literature search, determination of study eligibility criteria, data analysis or interpretation, or preparation or approval of the manuscript for publication. Staff from PCORI did review a draft version of the manuscript and provided suggestions to clarify language describing the process in selecting this topic for prioritization.
RESULTS Expansion of Evidence Gaps Through Stakeholder Engagement
The stakeholder panel expanded the initial list of 31 evidence gaps to 48. The final list illustrated several common themes: Stakeholders stressed that future research should focus on engaging patients early in the disease process, helping patients navigate treatment options that optimize patient-centered outcomes (for example, negative www.annals.org
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Research and Reporting Methods
affect, sleep, pain, and employment), and developing strategies that promote successful long-term engagement in nonsurgical treatment options. New questions suggested by the stakeholders addressed a wide range of evidence gaps related to the unique needs of subpopulations and the comparative effectiveness of diverse interventions. Stakeholders acknowledged that several patient groups received less attention than others and that it would be important to assess the comparative effectiveness of nonsurgical management strategies by sex, socioeconomic status, and prevalence and severity of comorbid conditions among adults and patients who had previously had surgical treatment of OA. Stakeholders stressed the progressive and chronic nature of OA and the importance of early detection and patient engagement. They also emphasized the importance of setting up patients for success—that is, that treatments should be tailored to patients’ abilities, insurance coverage, culture, and preferences. Stakeholders acknowledged that many current and ongoing studies focus on short-term outcomes and do not necessarily provide the needed information about longterm outcomes reflecting the chronic nature of the disease. They highlighted the importance of shared decisionmaking tools for not only exploring nonsurgical options but also discussing the optimal timing for transition to surgical interventions. They also stressed the incorporation of patient preferences for specific outcomes and their personal characteristics (and the effect of those characteristics on potential efficacy of treatment options) in such shared decision-making tools. Stakeholder Ranking of Future Research Needs
The Appendix Table (available at www.annals.org) shows the 48 final potential research topics, the number of points each received, and the number of stakeholders who allotted points to each topic. The final ranking divides the possible evidence gaps into a top, middle, and lower tier on the basis of overall score. The top 12 future research needs (Table) prioritized by stakeholders were related to identifying key patient-centered outcomes; determining optimal duration, intensity, and frequency of interventions; examining the effect of socioeconomic differences on management strategies; determining the need for transition from nonsurgical to surgical interventions; promoting long-term behavior change; standardizing screening tools; evaluating biomechanical strategies; developing strategies to help patients engage in key self-management behaviors; promoting coordinated care in today’s new health care delivery system; comparing methods for identifying and engaging patients early in the disease process; comparing safety and effectiveness of usual-care nonsurgical therapies; and identifying ways for setting patients up to succeed with nonsurgical management. Although the stakeholder group expanded the initial list to 48 potential future research needs, 18 of the final questions received no votes (5 questions) or 1 to 2 votes by 17 June 2014 Annals of Internal Medicine Volume 160 • Number 12 837
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Figure. Analytic framework.
Effect of population factors (FRNs 1–6) Sociodemographic differences Differences in comorbid conditions Previous surgical intervention Prevalence/severity of comorbid conditions Comedications Disease aspects of OA (e.g., severity, functional limitations, location, multijoint)
Promotion of adherence and maintenance of effects (FRNs 30–38) Patient engagement tools/ prompts Follow-up treatments Financial incentives Self-management strategies Strategies for long-term behavior change Nonsurgical management strategies Cultural group differences Socioeconomic differences Supervised exercise program
Effect of outcome measurement factors (FRNs 45–48) Validity/reliability of patient-centered outcome measures Validity/reliability of intermediate outcome measures Defining intermediate outcomes Confounding variables
Decision-making interventions (FRNs 39–40) Decision-making tools Patients raising issue
Population: adults with OA
Identification and screening (FRNs 7–18) Patient management of OA Standardized screening tools Sensitivity/specificity of evaluations Strategies for engaging patients Optimal ways to track and identify risk factors for OA OA-specific barriers to physical activity Functional ability self-assessment tool Walkability audit/checklist Different models of health care Optimal timing for interventions Threshold level of symptoms Need for transition from nonsurgical to surgical interventions
Therapeutic interventions (FRNs 19–29) Nonsurgical therapies Physical therapy Orthotics Self-administered transcutaneous electrostimulation Biomechanical strategies Complementary and alternative approaches Individual treatment methods Method of delivery Duration/intensity/frequency Sequenced weight loss program Setting up patients to succeed
Defining patient-centered outcomes (FRN 44) Sleep Negative affect/depression/worry Delay to surgery Reduction in medications Pain/independence in ADLs/ instrumental ADLs Patient satisfaction Time to return to work/activities Quality of life Reliability of pain treatment
Adverse effects of therapeutic interventions
Health care, policy, and ecological approaches (FRNs 41–43) Promoting care available in today’s new health care delivery system Policy and environmental strategies to promote physical activity Insurance coverage
Italicized text indicates top-tier gaps. ADLs ⫽ activities of daily living; FRN ⫽ future research need; OA ⫽ osteoarthritis.
at most 2 stakeholders (13 questions). Of the 12 questions ranked as highest priority, 2 were new questions that stakeholders suggested (questions focusing on the transition from nonsurgical to surgical interventions and on methods for setting patients up to succeed with nonsurgical management). The other 10 high-priority ranked questions were part of the original list of suggested future research needs. Of the 18 questions ranked low priority, 9 were from our original list and 9 were suggested by stakeholders. 838 17 June 2014 Annals of Internal Medicine Volume 160 • Number 12
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Horizon Scan of Studies Potentially Relevant to Top-Tier Research Questions
The PubMed search identified 4692 articles. Of these, 427 met inclusion criteria, including 92 systematic reviews; 254 randomized, controlled trials; 41 cohort studies; 3 case– control studies; and 37 studies of other design types. Most studies (n ⫽ 339) pertained to the comparative effectiveness of available nonsurgical therapies (228 randomized, controlled trials and 79 systematic reviews). Within www.annals.org
Research for Osteoarthritis
this set of 339 studies, 119 explored the safety and effectiveness of oral medications (29 systematic reviews; 84 randomized, controlled trials; 3 cohort studies; and 3 other studies). All but 20 of these evaluated oral medications compared with another oral medication or placebo. Relatively few studies evaluated treatments across categories (for example, drug therapy compared with weight management) or combined categories of treatment (for example, drug therapy plus exercise compared with drug therapy only). There were few studies for most of the other high-priority evidence gaps, with only 3 studies targeting ways of determining the need to transition from nonsurgical to surgical interventions and only 7 studies investigating strategies for identifying and engaging patients early in the disease process. The ClinicalTrials.gov search yielded 153 open, potentially relevant protocols. Of these, 55 were categorized as potentially relevant to the top-tier research questions. Sample sizes of these studies ranged from 8 to 20 000 patients, and estimated completion dates of the studies ranged from currently overdue to June 2017. (Tables 3 to 14 of the Supplement show key characteristics of the re-
Research and Reporting Methods
cent and ongoing studies relevant to the 12 top-tier future research needs.) Suggested Research Design Considerations
The Table summarizes the Evidence Synthesis Group’s suggestions for appropriate study designs or suggested analyses to address the 12 top-tier research priorities. Proposed designs and analyses to address specific gaps included meta-analysis of individual-patient data, randomized trials, and observational studies requiring collection of new data. Pragmatic clinical trials (14, 15) may be particularly useful for several of these research priorities, as PCORI’s recent funding announcement emphasized (16). (Table 15 of the Supplement provides additional details about possible advantages and disadvantages of multiple study designs for each gap.)
DISCUSSION Osteoarthritis is a common chronic condition that leads to functional limitations, pain, disability, and lost earnings and is associated with high utilization of health care (17, 18). A key theme that emerged from our research
Table. Top-Ranked Research Priorities and Recommended Study Designs or Suggested Analyses to Address Them Rank
Prioritized Future Research Need
Recommended Study Design or Suggested Analyses
1
What are the most important patient-centered outcomes (e.g., sleep, negative affect/depression/worry, delay to surgery, reduction in medications, pain/independence in ADLs/instrumental ADLs, patient satisfaction, time to return to work/activities or other employment outcomes, quality of life, and reliability of pain treatment) for patients with foot, ankle, knee, or hip OA? What are the optimal duration, intensity, and frequency of examined nonsurgical interventions for OA to create sustained changes in patient-centered outcomes? Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression and disability from OA differ by sociodemographic differences (e.g., age, sex, race/ethnicity, SES, insurance status, access to health care, and having a physically demanding occupation)? How do these strategies differ in specific underrepresented patient populations (i.e., those with low literacy level, low SES, or less access to health care)? What are effective ways (e.g., checklist of functionality/symptoms) for patients or providers to determine the need for the transition from nonsurgical to surgical interventions for OA? What are the comparative safety and effectiveness of strategies promoting long-term behavior change (e.g., weight management and physical activity) in the context of chronic pain and functional limitations associated with OA? Are there potential standardized screening tools and indicators of OA that can improve early diagnosis of OA?
Observational study requiring new data collection
2 3
4 5
6
7 8
9 10
11
12
What are the comparative safety and effectiveness of biomechanical strategies to improve OA symptoms and slow progression of disease? What are the comparative safety and effectiveness of strategies to help patients engage in key self-management behaviors for managing OA (physical activity and weight management) in real-world settings (community and primary care)? What opportunities for promoting coordinated, proactive, longitudinal, long-term care for OA are now available in today’s new health care delivery system? What are the comparative safety and effectiveness of strategies for identifying and engaging patients early in the disease process, particularly fostering healthy behaviors (physical activity and weight management), to prevent progression of and disability from OA? What are the comparative safety and effectiveness of usual care nonsurgical therapies (pharmacotherapy, injections, physical therapy/exercise, and weight loss) or combinations of usual care nonsurgical therapies to prevent progression of and disability from OA? Are these effects maintained (i.e., long-term outcomes) over time? How do we best set up patients to succeed with nonsurgical management for OA, incorporating available options and their specific potential barriers for an individual patient?
RCT RCT and/or meta-analysis of individual-patient data analysis across RCTs
RCT or modeling study RCT
Observational study using existing data or involving new data collection RCT RCT
Observational study involving new data collection RCT
RCT and/or meta-analysis of individual-patient data analysis across RCTs RCT and/or meta-analysis of individual-patient data analysis across RCTs
ADLs ⫽ activities of daily living; OA ⫽ osteoarthritis; RCT ⫽ randomized, controlled trial; SES ⫽ socioeconomic status. www.annals.org
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Research for Osteoarthritis
prioritization process is that future research priorities should focus on attending to patients’ needs across the spectrum of the disease, from identification through treatment decisions on the transition from nonsurgical to surgical OA interventions. The most highly ranked research needs focused on identifying the most important patient-centered outcomes and optimizing attributes of nonsurgical intervention (that is, duration, intensity, and frequency) that create sustained changes in patient-centered outcomes. Each nonsurgical treatment method may present tradeoffs between effectiveness in terms of optimizing patient-centered outcomes and other important attributes of treatments (for example, time required for frequent clinician visits and sacrifices involved in dietary modification) that may affect patients’ willingness to adopt and maintain treatment options. Thus, direct measurement of patient preferences for different attributes of interventions (including effectiveness and duration, intensity, and frequency) would be highly useful in designing comparative effectiveness studies. The literature search and the review of ongoing research in ClinicalTrials.gov revealed that, although a substantial amount of ongoing research is focused on the comparative safety and effectiveness of usual-care nonsurgical therapies (with most focusing on pharmacotherapies), relatively little work is focused on identifying the most important patient-centered outcomes and how these may map to attributes of nonsurgical treatment strategies. Many of the other high-priority research needs focused on developing better approaches to help enhance longterm patient engagement in strategies that may prevent the progression of and disability from OA. This factor is reflected in the high ranking of future research needs that focus on promoting long-term behavior change, developing strategies to help patients engage in key selfmanagement behaviors for OA, promoting coordinated OA care in today’s new health care delivery system, comparing methods for identifying and engaging patients early in the disease process, and identifying methods for setting patients up to succeed with nonsurgical management. Osteoarthritis is a chronic illness, so focusing on strategies that promote continuing engagement is a practical approach to optimizing outcomes. Our search of the literature and ongoing studies identified few studies evaluating early engagement of patients, methods for setting patients up for success, and coordinating OA care in today’s new health care delivery system. Stakeholders stress that strategies tailored to patients’ abilities, insurance, culture, and preferences may enhance the success of OA interventions in the long term. The list of future research needs that we generated and expanded with stakeholder feedback may not reflect the total range of possible future research. Although we included a diverse group of stakeholders with particular interest in patient-centered research, the panel involved relatively few stakeholders. Another group of stakeholders 840 17 June 2014 Annals of Internal Medicine Volume 160 • Number 12
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might add other future research needs or rank the identified future research needs differently. We did not conduct comprehensive systematic reviews pertinent to the identified evidence gaps; therefore, we could not determine with certainty the degree to which prioritized future research needs have already been addressed. To advance a patient-centered research agenda, future research should focus on addressing patients’ needs across the disease spectrum and enhancing long-term engagement in strategies that stabilize the symptoms of OA. A panel of stakeholders representing diverse perspectives identified 12 research areas as the highest priorities for future research of patient-centered management strategies for persons with OA. If studied, these 12 research areas may enhance the patient experience of managing OA symptoms and enable patients to optimize their most valued health outcomes. From the Center for Health Services Research in Primary Care, Durham Veterans Affairs Medical Center, Duke Evidence Synthesis Group, Duke Clinical Research Institute, and Duke University School of Medicine, Durham, North Carolina. Acknowledgment: The authors thank Megan M. Chobot, MSLS, for project coordination, and Liz Wing, MA, for editorial assistance. They also thank the members of the stakeholder panel: Kelli Allen, PhD; Teresa Brady, PhD; G. Kelley Fitzgerald, PhD; Yvonne M. Golightly, PhD; Lyndon Joseph, PhD; Jeffrey Katz, MD; Gayle Lester, PhD; Dave Mekemson; Paul Rockar, DPT; Jyme Schafer, MD; Nancy Simington; Kim Templeton, MD; and Patience White, MD. Financial Support: By PCORI. Disclosures: Disclosures can be viewed at www.acponline.org/authors /icmje/ConflictOfInterestForms.do?msNum⫽M14-0318. Requests for Single Reprints: Jennifer M. Gierisch, PhD, MPH, De-
partment of Medicine, Duke University Medical Center, Campus Box 104427 DUMC, Durham, NC 27701; e-mail, [email protected]. Current author addresses and author contributions are available at www.annals.org.
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Research for Osteoarthritis and other rheumatic conditions in the United States. Part II. Arthritis Rheum. 2008;58:26-35. [PMID: 18163497] 7. Nguyen US, Zhang Y, Zhu Y, Niu J, Zhang B, Felson DT. Increasing prevalence of knee pain and symptomatic knee osteoarthritis: survey and cohort data. Ann Intern Med. 2011;155:725-32. [PMID: 22147711] 8. Michaud CM, McKenna MT, Begg S, Tomijima N, Majmudar M, Bulzacchelli MT, et al. The burden of disease and injury in the United States 1996. Popul Health Metr. 2006;4:11. [PMID: 17049081] 9. Lee DW, Meyer JW, Clouse J. Implications of controlling for comorbid conditions in cost-of-illness estimates: a case study of osteoarthritis from a managed care system perspective. Value Health. 2001;4:329-34. [PMID: 11705300] 10. Gabriel SE, Crowson CS, O’Fallon WM. Comorbidity in arthritis. J Rheumatol. 1999;26:2475-9. [PMID: 10555912] 11. Gierisch JM, Myers ER, Schmit KM, Crowley MJ, McCrory DC, Chatterjee R, et al. Prioritization of research addressing management strategies for ductal carcinoma in situ. Ann Intern Med. 2014;160:484-91. [PMID: 24567146] 12. Brasure M, Shamliyan TA, Olson-Kellogg B, Butler ME, Kane RL. AHRQ comparative effectiveness reviews. Physical therapy for knee pain secondary to osteoarthritis: future research needs: identification of future research needs from comparative effectiveness review no. 77. Report no. 13-EHC048-EF. Rockville, MD: Agency for Healthcare Research and Quality; 2013. [PMID: 23905194] 13. Chang SM, Carey TS, Kato EU, Guise JM, Sanders GD. Identifying research needs for improving health care. Ann Intern Med. 2012;157:439-45. [PMID: 22847017]
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MOC POINTS
Research and Reporting Methods
14. Thorpe KE, Zwarenstein M, Oxman AD, Treweek S, Furberg CD, Altman DG, et al. A pragmatic-explanatory continuum indicator summary (PRECIS): a tool to help trial designers. CMAJ. 2009;180:E47-57. [PMID: 19372436] 15. Patsopoulos NA. A pragmatic view on pragmatic trials. Dialogues Clin Neurosci. 2011;13:217-24. [PMID: 21842619] 16. Patient-Centered Outcomes Research Institute. Pragmatic clinical studies and large simple trials to evaluate patient-centered outcomes. 2014. Accessed at www.pcori.org/funding-opportunities/funding-announcements/pragmatic -clinical-studies-and-large-simple-trials-to-evaluate-patient-centered-outcomes on 17 April 2014. 17. Yelin E, Murphy L, Cisternas MG, Foreman AJ, Pasta DJ, Helmick CG. Medical care expenditures and earnings losses among persons with arthritis and other rheumatic conditions in 2003, and comparisons with 1997. Arthritis Rheum. 2007;56:1397-407. [PMID: 17469096] 18. Centers for Disease Control and Prevention. NHIS arthritis surveillance: specific functional limitations among people with arthritis. Accessed at www.cdc .gov/arthritis/data_statistics/national_nhis.htm#functional on 5 February 2014. 19. Ip S, Raman G, Concannon TW, Ratichek SJ, Yu W, Chang LKW, et al. Intravascular Diagnostic Procedures and Imaging Techniques Versus Angiography Alone in Coronary Artery Stenting: Future Research Needs. Future Research Needs Paper no. 25. (Prepared by Tufts Evidence-based Practice Center under contract no. 290-2007-10055-I.) AHRQ publication no. 13-EHC016-EF. Rockville, MD: Agency for Healthcare Research and Quality; 2013. Accessed at www.effectivehealthcare.ahrq.gov/ehc/products/512/1413/coronary-artery -stenting-future-130604.pdf on 23 January 2014.
FOR
PATIENT SAFETY MODULE
Physicians enrolled in the American Board of Internal Medicine’s (ABIM) Maintenance of Certification (MOC) program can earn 8 medical knowledge self-assessment points for successful completeing the 25-question module based on the Annals of Internal Medicine: Patient Safety Learning Module. These CME and MOC activities are free to ACP members and individual subscribers to Annals of Internal Medicine. Others who are interested in completing this MOC activity can learn more about ACP membership and individual subscriptions to Annals of Internal Medicine at www.acponline.org. To earn MOC points: 1. Read the Annals supplement: “Making Health Care Safer: A Critical Review of Evidence Supporting Strategies to improve Patient Safety” (5 March 2013; vol. 158, no. 5), available at annals.org. 2. Take the Patient Safety Learning Module MOC quiz (available at www.acponline.org/patientsafetymoc) to submit your answers to the 25 questions and transfer the information to the ABIM. Important: Even if you have submitted answers to the CME quiz on annals.org, you will need to reenter your answers into the online MOC submission forms. 3. Succesful completion of the module (score of 100%) qualifies for 8 ABIM MOC points. Please visit www.acponline.org/patientsafetymoc to learn more and take the MOC quiz.
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17 June 2014 Annals of Internal Medicine Volume 160 • Number 12 841
Annals of Internal Medicine Current Author Addresses: Dr. Gierisch: Department of Medicine, Duke University Medical Center, Campus Box 104427 DUMC, Durham, NC 27701. Dr. Myers: Duke University Medical Center, Box 3279, 244 Baker House, Durham, NC 27710. Drs. Schmit, McCrory, Coeytaux, and Sanders and Ms. Kendrick: Duke Clinical Research Institute, PO Box 17969, Durham, NC 27715. Dr. Crowley: Durham Veterans Affairs Medical Center, Health Services Research and Development (152), 508 Fulton Street, Durham, NC 27705. Dr. Chatterjee: Sutton Station Internal Medicine, 5832 Fayetteville Road, Suite 113, Durham, NC 27713.
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Author Contributions: Conception and design: J.M. Gierisch, R.R.
Coeytaux, M.J. Crowley, G.D. Sanders. Analysis and interpretation of the data: J.M. Gierisch, E.R. Myers, D.C. McCrory, M.J. Crowley, R. Chatterjee, G.D. Sanders. Drafting of the article: J.M. Gierisch, R.R. Coeytaux, G.D. Sanders. Critical revision of the article for important intellectual content: J.M. Gierisch, E.R. Myers, M.J. Crowley, G.D. Sanders. Final approval of the article: J.M. Gierisch, E.R. Myers, D.C. McCrory, R.R. Coeytaux, M.J. Crowley, R. Chatterjee, A.S. Kendrick, G.D. Sanders. Provision of study materials or patients: A.S. Kendrick, G.D. Sanders. Statistical expertise: G.D. Sanders. Obtaining of funding: G.D. Sanders. Administrative, technical, or logistic support: J.M. Gierisch, A.S. Kendrick, G.D. Sanders. Collection and assembly of data: J.M. Gierisch, E.R. Myers, K.M. Schmit, D.C. McCrory, R.R. Coeytaux, M.J. Crowley, R. Chatterjee, A.S. Kendrick, G.D. Sanders.
17 June 2014 Annals of Internal Medicine Volume 160 • Number 12
Appendix Figure. Overview of prioritization process. Steps
Output/Products
Identification of evidence gaps Review systematic reviews, clinical practice guidelines, and consensus statements for evidence gaps; structure evidence gaps into research questions Initial list of evidence gaps and draft analytic framework Selection of stakeholders Send stakeholders background materials and initial list of evidence gaps Establish stakeholder group Engage stakeholders via Webinar/ teleconference/e-mail Introduction to process; refine and expand initial evidence gaps Expanded list of evidence gaps and revised analytic framework Stakeholders participate in Web-based prioritization process Stakeholders participate in forced-ranking prioritization method used to facilitate prioritization of evidence gaps
Inform stakeholders of prioritized list and next steps
Conduct horizon scan of studies potentially relevant to top-tier research questions ESG investigators conduct PubMed and ClinicalTrials.gov searches for recent or ongoing relevant studies for the top-tier prioritized future research needs
Explore research question development and research design considerations ESG investigators consider advantages and disadvantages of various potential study designs
Produce future research needs report for PCORI ESG investigators integrate results of the prioritization process, horizon scan, and research design consideration into a report for PCORI leadership
Stakeholder-identified prioritized list of future research needs
Detailed list of recent published studies or ongoing research relevant to top-tier future research needs
List of suggested study designs to address top-tier future research needs
Future research needs report
Adapted from reference 19. ESG ⫽ Evidence Synthesis Group; PCORI ⫽ Patient-Centered Outcomes Research Institute.
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Appendix Table. Final Ranking of Future Research Needs for Alternative Strategies for OA* Question Top tier What are the most important patient-centered outcomes (e.g., sleep, negative affect/depression/worry, delay to surgery, reduction in medications, pain/independence in ADLs/instrumental ADLs, patient satisfaction, time to return to work/activities or other employment outcomes, quality of life, and reliability of pain treatment) for patients with foot, ankle, knee, or hip OA? What are the optimal duration, intensity, and frequency of examined nonsurgical interventions for OA to create sustained changes in patient-centered outcomes? Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression and disability from OA differ by sociodemographic differences (e.g., age, sex, race/ethnicity, SES, insurance status, access to health care, and having a physically demanding occupation)? How do these strategies differ in specific underrepresented patient populations (i.e., those with low literacy level, low SES, or less access to health care)? What are effective ways (e.g., checklist of functionality/symptoms) for patients or providers to determine the need for the transition from nonsurgical to surgical interventions for OA? What are the comparative safety and effectiveness of strategies promoting long-term behavior change (e.g., weight management and physical activity) in the context of chronic pain and functional limitations associated with OA? Are there potential standardized screening tools and indicators of OA that can improve early diagnosis of OA? What are the comparative safety and effectiveness of biomechanical strategies to improve OA symptoms and slow progression of disease? What are the comparative safety and effectiveness of strategies to help patients engage in key self-management behaviors for managing OA (physical activity and weight management) in real-world settings (community and primary care)? What opportunities for promoting coordinated, proactive, longitudinal, long-term care for OA are now available in today’s new health care delivery system? What are the comparative safety and effectiveness of strategies for identifying and engaging patients early in the disease process, particularly fostering healthy behaviors (physical activity and weight management), to prevent progression of and disability from OA? What are the comparative safety and effectiveness of usual care nonsurgical therapies (pharmacotherapy, injections, physical therapy/exercise, and weight loss) or combinations of usual care nonsurgical therapies to prevent progression of and disability from OA? Are these effects maintained (i.e., long-term outcomes) over time? How do we best set up patients to succeed with nonsurgical management for OA incorporating available options and their specific potential barriers for an individual patient? Middle tier Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression and disability from OA differ by disease aspects of OA (e.g., OA symptom severity, functional limitations, location of OA, and multijoint OA)? Because OA is a progressive disease, what is the optimal timing for nonsurgical (e.g., physical therapy, injections, NSAIDs, acetaminophen, and opioids) or surgical interventions to enhance outcomes? What are the comparative safety and effectiveness of models of organizing health care that lead to identifying and engaging patients early in the disease process? What are the comparative safety and effectiveness of comprehensive multimodal physical therapy treatments for OA on patient-centered outcomes when compared with exercise alone? Are these effects maintained (i.e., long-term outcomes) over time? What are the comparative safety and effectiveness of strategies to increase patient engagement in nonsurgical management strategies to prevent progression of and disability from OA? What are valid and reliable instruments used to measure the following patient-centered outcomes for persons with OA: pain/independence in ADLs/instrumental ADLs, patient satisfaction, time to return to work/activities, quality of life, community integration, psychological disability, and self-perceived health? What is the minimum clinically important difference for each of these instruments? What cut points should be used to describe clinically meaningful categories in the scale scores created by these instruments? What are effective ways to track and identify OA risk factors and OA progression/outcomes (e.g., electronic health records)? What are effective ways for health care professionals to assess OA-specific barriers to physical activity (as a routine component of “vital signs”) when screening their adult patients? Which intermediate outcomes meet the criteria for surrogate patient-centered outcomes for persons with OA? What are the comparative sensitivity and specificity of biomedical evaluations (genetic markers and imaging evaluations) for detecting OA among adult patients at risk? Do differences in accuracy lead to clinically relevant differences in choice of therapeutic interventions? Do the comparative safety and effectiveness of physical therapy/exercise management strategies for OA on patient-centered outcomes differ by method of delivery (e.g., the involvement of a physical therapist or physical therapist assistant, group vs. individual exercise, self-administered vs. supervised exercises, electronic vs. in-person delivery)? In patients with OA, what individual characteristics can predict who first needs to receive a professionally supervised exercise program vs. who does not before engaging in an independent physical activity program to maximize benefits and long-term participation in physical activity programs? What are the comparative safety and effectiveness of patient-engagement tools and prompts that promote learning about and management of OA (and pre-OA)? What is the effect of policy, human resources, and environmental strategies on physical activity behaviors among adults with OA? Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression of and disability from OA differ by comorbid conditions (including obesity)? What are the prevalence and severity of comorbid conditions among adults with OA?
Score
Stakeholders, n
13
8
12
9
9
7
8
7
8
6
7 7
6 6
7
6
7
6
7
5
7
5
7
5
5
5
5
5
5
4
5
4
5
4
5
4
4 4
4 4
4 4
4 3
4
3
4
3
4
2
4 3
2 3
3
3
Continued on following page
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Appendix Table—Continued Question What are the valid and reliable instruments used to measure the following intermediate outcomes when evaluating the comparative safety and effectiveness of physical therapy interventions for foot, ankle, knee, or hip pain secondary to OA: joint function, swelling, inflammation, gait function, strength, and transfers? At what point along the continuum of symptoms, functionality, and risk for OA do patients become actively engaged in managing their OA/reducing their risk for progression? Lower tier Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression of and disability from OA differ for patients who have previously had a surgical intervention for OA? What are effective functional ability self-assessment tools or programmatic approaches for adults with OA that will allow patients to tailor physical activity accordingly? How do the comparative safety and effectiveness of current adherence and maintenance strategies for nonsurgical management of OA differ by cultural groups? What are the effects of decision-making tools compared with usual care to assist patients with shared decision-making processes with the primary care provider about OA treatments (e.g., medication use, joint injections, physical therapy, and joint replacement surgery), with a focus on persons with low health literacy and limited health access compared with usual care? What are the optimal format, content, and timing for these? Do periodic follow-up treatments beyond the initial physical therapy treatments of OA enhance effectiveness of these treatments? How do we empower patients to raise the issues of their joint pain and functional limitations with their health care professional? What are effective walkability audits/checklists that are specifically relevant for adults with OA? Should there be some threshold level of symptoms or functionality that justifies the transition from nonsurgical to surgical interventions for OA? What are the comparative safety and effectiveness of orthotics in patients with (foot, ankle, knee, and hip) OA to prevent progression of and disability from lower-body OA? What are the comparative safety and effectiveness of a sequenced weight loss and physical activity approach (weight loss program administered first, followed by initiation of physical activity when minimal weight loss criteria have been achieved) vs. the simultaneous administration of weight loss and physical activity in reducing pain and disability and promoting long-term physical activity behavior in patients with OA and obesity? What is the role of financial incentives to enhance effectiveness, adherence, and maintenance of nonsurgical management strategies for OA? How do the comparative safety and effectiveness of current adherence and maintenance strategies for nonsurgical management of OA differ by socioeconomic groups? What confounding variables (e.g., adherence, weight loss, and activity levels) and effect modifiers, including concomitant therapies, should be controlled for when examining patient-centered outcomes for persons with OA? Do the comparative safety and effectiveness of nonsurgical management strategies to prevent progression of and disability from OA differ by comedications (e.g., anticoagulants) in the population of interest? What are the comparative safety and effectiveness of self-administered transcutaneous electrostimulation compared with other usual care nonpharmacologic or pharmacologic approaches for patients with (foot, ankle, knee, and hip) OA to prevent progression of and disability from OA? What are the comparative safety and effectiveness of complementary/alternative medicine strategies (e.g., plant and herbal therapies, electroacupuncture, needle acupuncture, and balneotherapy) compared with other usual care nonpharmacologic approaches for patients with (foot, ankle, knee, and hip) OA to prevent progression of and disability from OA? Are these effects maintained (i.e., long-term outcomes) over time? What is the benefit from individual treatment methods (e.g., heat and ice) delivered for OA? How does coverage by insurance of nonsurgical management strategies to prevent progression of and disability from OA affect the comparative effectiveness and/or adherence of available strategies?
Score
Stakeholders, n
3
3
3
2
2
2
2
2
2
2
2
2
2 2
1 1
1 1
1 1
1
1
1
1
1
1
1
1
1
1
0
0
0
0
0
0
0 0
0 0
ADLs ⫽ activities of daily living; OA ⫽ osteoarthritis; NSAID ⫽ nonsteroidal anti-inflammatory drug; SES ⫽ socioeconomic status. * Italicized text indicates new questions based on feedback from the stakeholder group.
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