FROM
THE ACADEMY
The International Dermatology Outcome Measures Group: Formation of patient-centered outcome measures in dermatology Alice B. Gottlieb, MD, PhD,a,b Adriane A. Levin, BA,a,c April W. Armstrong, MD, MPH,d April Abernethy, MD,e Kristina Callis Duffin, MD, MS,f Reva Bhushan, MA, PhD,g Amit Garg, MD,h Joseph F. Merola, MD, MMSc,i Mara Maccarone,j and Robin Christensen, MSc, PhDk Boston, Massachusetts; Denver, Colorado; Portland, Oregon; Salt Lake City, Utah; Schaumburg, Illinois; Manhasset, New York; Rome, Italy; and Copenhagen, Denmark As quality standards are increasingly in demand throughout medicine, dermatology needs to establish outcome measures to quantify the effectiveness of treatments and providers. The International Dermatology Outcome Measures Group was established to address this need. Beginning with psoriasis, the group aims to create a tool considerate of patients and providers using the input of all relevant stakeholders in assessment of disease severity and response to treatment. Herein, we delineate the procedures through which consensus is being reached and the future directions of the project. ( J Am Acad Dermatol 2015;72:345-8.) Key words: body surface area; Delphi exercises; Dermatology Life Quality Index; disease severity; International Dermatology Outcome Measures; National Psoriasis Foundation; Outcome Measures in Rheumatology; patient-centered outcome measures; Physician Global Assessment; psoriasis; Psoriasis Area and Severity Index; psoriatic arthritis; quality of life.
T
he International Dermatology Outcome Measures (IDEOM) initiative was established to address the need for standardized, patientcentered clinical outcome measures to assess disease course and response to treatments, and ultimately to improve patient outcomes and access to high-quality
From the Department of Dermatology, Tufts Medical Center, Bostona; Tufts University School of Medicine, Bostonb; Boston University School of Medicinec; University of Colorado School of Medicined; National Psoriasis Foundation, Portlande; Department of Dermatology, University of Utahf; American Academy of Dermatology, Schaumburgg; Department of Dermatology, Hofstra North Shore Long Island Jewish School of Medicine, Manhasseth; Brigham and Women’s Hospital, Harvard Medical School, Bostoni; Associazione per la Difesa delgi Proriasici, Romej; and Department of Rheumatology, Michigan State University, Parker Institute, Copenhagen.k Funding sources: None. Disclosure: Dr Gottlieb maintains current consulting/advisory board agreements with Amgen Inc, Astellas, Centocor (Janssen), Celgene Corp, Bristol Meyers Squibb Co, Beiersdorf Inc, Abbott Labs (Abbvie), TEVA, Actelion, UCB, Novo Nordisk, Novartis, Dermipsor Ltd, Incyte, Pfizer, Canfite, Eli Lilly, Coronado, Vertex, Karyopharm, CSL Behring Biotherapies for Life, Glaxo Smith Kline, Xenoport, Catabasis, and Sanofi Aventis. Dr Armstrong served as a consultant and was investigator for Abbvie, Amgen Inc, Janssen, Eli Lilly, Pfizer, and Merck. Dr Abernethy served on the advisory board of Abbvie and as speaker for Pfizer receiving honoraria and is an employee for National Psoriasis Foundation receiving salary. Dr Duffin served on the advisory board and was consultant and investigator for Amgen Inc, Janssen, Eli Lilly, and Pfizer;
Abbreviations used: IDEOM:
International Dermatology Outcome Measures OMERACT: Outcome Measures in Rheumatology PASI: Psoriasis Area and Severity Index
served as consultant and investigator for VBL Therapeutics receiving honoraria and salary; was investigator for Abbvie receiving salary; and served as consultant for Bristol Myers Squibb Co receiving honoraria. Dr Garg served on the advisory board of Eli Lilly, Amgen Inc, Abbvie, Genentech, and Pfizer receiving honoraria. Dr Merola served on the advisory board and was investigator for Amgen Inc; was on the advisory board on Eli Lilly and Novartis; was an investigator for Pfizer and Biogen; was speaker for Abbvie; and had an ‘‘other’’ relationship with Abbvie and Biogen receiving honoraria. Ms Maccarone is the founder of Associazione per la Difesa degli PsoriasicieItalian Association for Psoriasis receiving no compensation. Dr Christensen receives no compensation from industry. Tufts Medical Center received research/educational grants from Centocor (Janssen), Amgen Inc. Abbott Labs (Abbvie), Novartis, Celgene, Pfizer, Eli Lilly, Coronado, Levia, and Merck. Ms Levin and Dr Bhushan have no conflicts of interest to declare. Accepted for publication November 3, 2014. Reprint requests: Reva Bhushan, MA, PhD, American Academy of Dermatology, 930 E Woodfield Road. Schaumburg, IL 60173. E-mail: [email protected]. Published online December 6, 2014. 0190-9622/$36.00 Ó 2014 by the American Academy of Dermatology, Inc. http://dx.doi.org/10.1016/j.jaad.2014.11.002
345
346 Gottlieb et al
dermatologic care1; this goal is represented in the mission statement, ‘‘Establish patient-centered measurements to enhance research and treatment for those with dermatologic disease.’’1 It is IDEOM’s practice that the perspectives of patients, health economists, and payers are represented along with those of physicians and regulatory agencies in outcome measures for dermatologic disease. Alice B. Gottlieb, MD, PhD, first proposed the group’s formation in recognition of a lack of comprehensive outcome measures that satisfy the needs of all stakeholders and that can be used in clinical practice. IDEOM’s goal is to establish validated and standardized outcome measures that can be applied both in clinical trials and clinical practice. Unlike the evaluation of blood pressure or diabetes, for example, quantifying the severity of psoriasis in clinical records is not straightforward. As a result, we lack adequate tools to compare the quality of care for psoriasis among clinical practices, thus cost becomes the default measure to compare dermatologists. Payers limit access to dermatologists who provide the full spectrum of psoriasis care based on higher cost and not quality of care. This manifests as higher copays for physicians caring for the sickest patients; access is thus limited by economic disincentives. US payers are increasingly demanding disease-specific outcome measures generated by both patients and physicians that can be easily used in the clinical setting. Most of the measures in existence do nt fully address patients’ concerns. Some are not practical for use in clinic whereas others are overly reductive in their simplicity. IDEOM has included patients and physicians from its beginnings to develop clinically meaningful end points reflective of measures primary to patients, including how a patient feels, functions, and performs activities of daily living. At the inaugural IDEOM meeting in January 2013, in Boston, MA, 35 members selected psoriasis as the prototype disease given its burden on patients, the relative absence of patient input for existing measures, and the significant advancement in therapy over the past 2 decades. Patients with psoriasis are frequently undertreated; 1 National Psoriasis Foundation survey found that up to 50% of patients with severe psoriasis are treated exclusively with topical medications.2 Participants agreed that current outcome measures lack truth, discrimination, and feasibility. Psoriasis Area and Severity Index (PASI), for example, is not efficiently applied to the clinical setting because of cumbersome calculations and a lengthy process; this demonstrates lack of feasibility.3 Moreover, it is not sensitive to change in patients with low body surface
J AM ACAD DERMATOL
FEBRUARY 2015
Fig 1. Flowsheet delineating the Delphi process.
Fig 2. Onion model. Core set: Pcombined $ .70 AND a lower limit of the 95% confidence interval $ 0.50, outer core: either patients or health care providers report P $ .70, research agenda: Pcombined $ .50. Pcombined is the proportion of votes for an item being important across both patients and health care providers. (P $ .70 was adopted from the Outcome Measures in Rheumatology model. Modified with permission from Mease et al.13)
area and it does not accurately represent disease impact on nonskin integument, such as nails: limitations in its truth and discrimination.4 The PASI scale is not on a normal distribution curve and the upper end is infrequently used.5 Although the PASI has good intraobserver variability, it lacks significant interobserver variability.6 It also lacks a patient-reported portion, it does not measure quality of life, and it fails to account for the impact of involvement of cosmetically or functionally sensitive areas. Nevertheless, PASI remains the most common outcome measure in use, and has in fact increased in prevalence from use in 30.6% of studies in 1977 through 2000 to 57.7% in 2001 through 2006.7 Physician Global Assessment is also problematic, with various numerical scales coexisting and no
J AM ACAD DERMATOL VOLUME 72, NUMBER 2
Gottlieb et al 347
Fig 3. Results of the second Delphi exercise. The median percent importance of each of the 21 domains is represented. The dispersion around the median corresponds to the 10th through 90th percentile of all responders; each line captures 80% of stakeholder opinions, with the median representing the 50th percentile. The domains of psoriatic arthritis signs and symptoms, location and area of involvement, and psoriasis primary morphology were statistically most important.
included measure of body surface area.6,8 Neither of these tools include quality-of-life measures, although strong correlation has been found between Dermatology Life Quality Index and PASI.7 Moreover, associated risk factors of cardiovascular disease9 and depression10 are not measured. None of the established outcome measures in psoriasis were created with any patient input.
METHODS Based on the model put forth by Outcome Measures in Rheumatology (OMERACT), IDEOM’s first meeting consisted of group discussions to determine candidate domains and items essential to inclusion in psoriasis outcome measures (Fig 1). These were the concepts deemed on initial evaluation central to the diagnosis, treatment, and management of psoriatic disease. Together, the group composed a preliminary list of items and domains for psoriasis. The group consisted of dermatologists and rheumatologist mentors from the OMERACT team along with patients, pharmaceutical scientists, and
payers. The establishment of domains was followed by the first of several Delphi exercises aimed at achieving consensus among stakeholders as to the relative importance of these concepts.11 The Delphi survey is a multistep technique used to form consensus among a group of experts. Several rounds of structured questionnaires are distributed, statistically analyzed, and evaluated by the group.12 IDEOM participants prepared the Delphi using OMERACT’s ‘‘onion model,’’ represented by a central core of essential components, a middle ring of factors of undetermined significance, and an outer ring for those domains requiring further investigation13 (Fig 2). The first meeting was made possible by the generous donation from a nonmember and the National Psoriasis Foundation. Presently, funding is similar to that of OMERACT, with pharmaceutical companies paying for membership. The nonprofit organization, Advancing Innovations in Dermatology, was also instrumental in funding IDEOM.
J AM ACAD DERMATOL
348 Gottlieb et al
RESULTS The first Delphi was administered by anonymous World Wide Webebased survey in May 2013 to 155 participants, including 138 experts (dermatologists, rheumatologists, payers, regulators, and industry partners) and 17 patients, with the goal of stratifying items into the onion model. Participants were asked to rank each item as ‘‘very important,’’ ‘‘maybe important,’’ or ‘‘not important.’’ The results from 80 responders (51.6% response rate) were discussed at the second IDEOM conference in Toronto, Ontario, Canada, in July 2013. In all, 61 attendees determined which items that would have been eliminated because of a low Delphi score were important enough to remain in the item set.4 Following this procedure, IDEOM members including patients preliminarily identified 21 element groupings; these consisted of categories into which individual items fit together. In a second Delphi exercise, 208 participants were invited to weight the importance of the 21 elements. Among the 102 responders representing 8 countries, there were 79 health care providers, 12 patients, 8 pharmaceutical scientists, 1 researcher, 1 payer, and 1 professional association. The results of this second Delphi (Fig 3) were discussed among 51 attendees at the third IDEOM conference in April 2014 in Rome, Italy. The most heavily weighted (highest-scoring) domains identified by the survey were psoriasis morphology, location and area, and psoriatic arthritis. At the meeting, participants agreed that we currently lack adequate measures for the morphological extent of psoriatic disease, including characterization of such presentations as erosions, pustules, and fissures. In addition, the group found nail involvement and inverse or intertriginous area involvement to be underrecognized by current disease tools. The salience of location of plaques, for example in visible areas of the body, was discussed at great length, with patients suggesting that location is more important even than total body surface area. Plaques on the face, distal arms and legs, and hands, it was thought, should be more heavily weighted, corroborating findings from the literature.7 Participants expressed the need for sensitive and reliable measures to adequately capture disease severity that are feasible in both trial and practice settings. Patients and physicians agreed that psoriatic arthritis assessment was an essential component of outcome measurement and that loss of independence from functional disability was rarely addressed in psoriasis trials. In fact, many dermatologists do not screen for psoriatic arthritis in the psoriasis visits.14 Discussion also revealed that the impact of psoriasis-specific
FEBRUARY 2015
symptoms such as pruritus, inflexibility from thick plaques, and skin pain are not adequately captured.
FUTURE DIRECTIONS IDEOM members structured plans for the next iteration of outcome development in psoriatic disease. Before administering the next Delphi, a team of dermatologists and patients regrouped elements into the following core areas: pathophysiological manifestations, adverse events, life impact, death, resource use/economic impact, and contextual factors. IDEOM will recruit more patients through outreach to international patient groups, with the goal of having equal patient, provider, and other stakeholder contributions in preparation for the next Delphi planned for 2015. REFERENCES 1. Gottlieb AB, Swerlick RIDEOM. International Dermatology Outcomes Measures; a call to action. Psoriasis Forum. 2013; 19:107-109. 2. Horn EJ, Fox KM, Patel V, Chiou CF, Dann F, Lebwohl M. Are patients with psoriasis undertreated? Results of National Psoriasis Foundation survey. J Am Acad Dermatol. 2007;57:957-962. 3. Feldman SR, Krueger GG. Psoriasis assessment tools in clinical trials. Ann Rheum Dis. 2005;64(Suppl):ii65-ii73. 4. Gottlieb AB, Armstrong AW, Christensen R, et al. The International Dermatology Outcome Measures initiative as applied to psoriatic disease outcomes: a report from the GRAPPA 2013 meeting. J Rheumatol. 2014;41:1227-1229. 5. Armstrong AW, Parsi K, Schupp CW, Mease PJ, Duffin KC. Standardizing training for psoriasis measures: effectiveness of an online training video on Psoriasis Area and Severity Index assessment by physician and patient raters. JAMA Dermatol. 2013;149:577-582. 6. Gottlieb AB, Armstrong AW. Psoriasis outcome measures: a report from the GRAPPA 2012 annual meeting. J Rheumatol. 2013;40:1428-1433. 7. Heredi E, Rencz F, Balogh O, et al. Exploring the relationship between EQ-5D, DLQI and PASI, and mapping EQ-5D utilities: a cross-sectional study in psoriasis from Hungary. Eur J Health Econ. 2014;15(Suppl):S111-S119. 8. Callis Duffin K, Gottlieb AB. Outcome measures for psoriasis severity: a report from the GRAPPA 2012 annual meeting. J Rheumatol. 2013;40:1423-1424. 9. Gelfand JM, Neimann AL, Shin DB, Wang X, Margolis DJ, Troxel AB. Risk of myocardial infarction in patients with psoriasis. JAMA. 2006;296:1735-1741. 10. Gupta MA, Gupta AK. Depression and suicidal ideation in dermatology patients with acne, alopecia areata, atopic dermatitis and psoriasis. Br J Dermatol. 1998;139:846-850. 11. Boers M, Brooks P, Strand CV, Tugwell P. The OMERACT filter for outcome measures in rheumatology. J Rheumatol. 1998;25:198-199. 12. Hasson F, Keeney S, McKenna H. Research guidelines for the Delphi survey technique. J Adv Nurs. 2000;32:1008-1015. 13. Mease PJ, Clauw DJ, Christensen R, et al. Toward development of a fibromyalgia responder index and disease activity score: OMERACT module update. J Rheumatol. 2011;38:1487-1495. 14. Kimball AB, Guerin A, Tsaneva M, et al. Economic burden of comorbidities in patients with psoriasis is substantial. J Eur Acad Dermatol Venereol. 2011;25:157-163.
THE ACADEMY
The International Dermatology Outcome Measures Group: Formation of patient-centered outcome measures in dermatology Alice B. Gottlieb, MD, PhD,a,b Adriane A. Levin, BA,a,c April W. Armstrong, MD, MPH,d April Abernethy, MD,e Kristina Callis Duffin, MD, MS,f Reva Bhushan, MA, PhD,g Amit Garg, MD,h Joseph F. Merola, MD, MMSc,i Mara Maccarone,j and Robin Christensen, MSc, PhDk Boston, Massachusetts; Denver, Colorado; Portland, Oregon; Salt Lake City, Utah; Schaumburg, Illinois; Manhasset, New York; Rome, Italy; and Copenhagen, Denmark As quality standards are increasingly in demand throughout medicine, dermatology needs to establish outcome measures to quantify the effectiveness of treatments and providers. The International Dermatology Outcome Measures Group was established to address this need. Beginning with psoriasis, the group aims to create a tool considerate of patients and providers using the input of all relevant stakeholders in assessment of disease severity and response to treatment. Herein, we delineate the procedures through which consensus is being reached and the future directions of the project. ( J Am Acad Dermatol 2015;72:345-8.) Key words: body surface area; Delphi exercises; Dermatology Life Quality Index; disease severity; International Dermatology Outcome Measures; National Psoriasis Foundation; Outcome Measures in Rheumatology; patient-centered outcome measures; Physician Global Assessment; psoriasis; Psoriasis Area and Severity Index; psoriatic arthritis; quality of life.
T
he International Dermatology Outcome Measures (IDEOM) initiative was established to address the need for standardized, patientcentered clinical outcome measures to assess disease course and response to treatments, and ultimately to improve patient outcomes and access to high-quality
From the Department of Dermatology, Tufts Medical Center, Bostona; Tufts University School of Medicine, Bostonb; Boston University School of Medicinec; University of Colorado School of Medicined; National Psoriasis Foundation, Portlande; Department of Dermatology, University of Utahf; American Academy of Dermatology, Schaumburgg; Department of Dermatology, Hofstra North Shore Long Island Jewish School of Medicine, Manhasseth; Brigham and Women’s Hospital, Harvard Medical School, Bostoni; Associazione per la Difesa delgi Proriasici, Romej; and Department of Rheumatology, Michigan State University, Parker Institute, Copenhagen.k Funding sources: None. Disclosure: Dr Gottlieb maintains current consulting/advisory board agreements with Amgen Inc, Astellas, Centocor (Janssen), Celgene Corp, Bristol Meyers Squibb Co, Beiersdorf Inc, Abbott Labs (Abbvie), TEVA, Actelion, UCB, Novo Nordisk, Novartis, Dermipsor Ltd, Incyte, Pfizer, Canfite, Eli Lilly, Coronado, Vertex, Karyopharm, CSL Behring Biotherapies for Life, Glaxo Smith Kline, Xenoport, Catabasis, and Sanofi Aventis. Dr Armstrong served as a consultant and was investigator for Abbvie, Amgen Inc, Janssen, Eli Lilly, Pfizer, and Merck. Dr Abernethy served on the advisory board of Abbvie and as speaker for Pfizer receiving honoraria and is an employee for National Psoriasis Foundation receiving salary. Dr Duffin served on the advisory board and was consultant and investigator for Amgen Inc, Janssen, Eli Lilly, and Pfizer;
Abbreviations used: IDEOM:
International Dermatology Outcome Measures OMERACT: Outcome Measures in Rheumatology PASI: Psoriasis Area and Severity Index
served as consultant and investigator for VBL Therapeutics receiving honoraria and salary; was investigator for Abbvie receiving salary; and served as consultant for Bristol Myers Squibb Co receiving honoraria. Dr Garg served on the advisory board of Eli Lilly, Amgen Inc, Abbvie, Genentech, and Pfizer receiving honoraria. Dr Merola served on the advisory board and was investigator for Amgen Inc; was on the advisory board on Eli Lilly and Novartis; was an investigator for Pfizer and Biogen; was speaker for Abbvie; and had an ‘‘other’’ relationship with Abbvie and Biogen receiving honoraria. Ms Maccarone is the founder of Associazione per la Difesa degli PsoriasicieItalian Association for Psoriasis receiving no compensation. Dr Christensen receives no compensation from industry. Tufts Medical Center received research/educational grants from Centocor (Janssen), Amgen Inc. Abbott Labs (Abbvie), Novartis, Celgene, Pfizer, Eli Lilly, Coronado, Levia, and Merck. Ms Levin and Dr Bhushan have no conflicts of interest to declare. Accepted for publication November 3, 2014. Reprint requests: Reva Bhushan, MA, PhD, American Academy of Dermatology, 930 E Woodfield Road. Schaumburg, IL 60173. E-mail: [email protected]. Published online December 6, 2014. 0190-9622/$36.00 Ó 2014 by the American Academy of Dermatology, Inc. http://dx.doi.org/10.1016/j.jaad.2014.11.002
345
346 Gottlieb et al
dermatologic care1; this goal is represented in the mission statement, ‘‘Establish patient-centered measurements to enhance research and treatment for those with dermatologic disease.’’1 It is IDEOM’s practice that the perspectives of patients, health economists, and payers are represented along with those of physicians and regulatory agencies in outcome measures for dermatologic disease. Alice B. Gottlieb, MD, PhD, first proposed the group’s formation in recognition of a lack of comprehensive outcome measures that satisfy the needs of all stakeholders and that can be used in clinical practice. IDEOM’s goal is to establish validated and standardized outcome measures that can be applied both in clinical trials and clinical practice. Unlike the evaluation of blood pressure or diabetes, for example, quantifying the severity of psoriasis in clinical records is not straightforward. As a result, we lack adequate tools to compare the quality of care for psoriasis among clinical practices, thus cost becomes the default measure to compare dermatologists. Payers limit access to dermatologists who provide the full spectrum of psoriasis care based on higher cost and not quality of care. This manifests as higher copays for physicians caring for the sickest patients; access is thus limited by economic disincentives. US payers are increasingly demanding disease-specific outcome measures generated by both patients and physicians that can be easily used in the clinical setting. Most of the measures in existence do nt fully address patients’ concerns. Some are not practical for use in clinic whereas others are overly reductive in their simplicity. IDEOM has included patients and physicians from its beginnings to develop clinically meaningful end points reflective of measures primary to patients, including how a patient feels, functions, and performs activities of daily living. At the inaugural IDEOM meeting in January 2013, in Boston, MA, 35 members selected psoriasis as the prototype disease given its burden on patients, the relative absence of patient input for existing measures, and the significant advancement in therapy over the past 2 decades. Patients with psoriasis are frequently undertreated; 1 National Psoriasis Foundation survey found that up to 50% of patients with severe psoriasis are treated exclusively with topical medications.2 Participants agreed that current outcome measures lack truth, discrimination, and feasibility. Psoriasis Area and Severity Index (PASI), for example, is not efficiently applied to the clinical setting because of cumbersome calculations and a lengthy process; this demonstrates lack of feasibility.3 Moreover, it is not sensitive to change in patients with low body surface
J AM ACAD DERMATOL
FEBRUARY 2015
Fig 1. Flowsheet delineating the Delphi process.
Fig 2. Onion model. Core set: Pcombined $ .70 AND a lower limit of the 95% confidence interval $ 0.50, outer core: either patients or health care providers report P $ .70, research agenda: Pcombined $ .50. Pcombined is the proportion of votes for an item being important across both patients and health care providers. (P $ .70 was adopted from the Outcome Measures in Rheumatology model. Modified with permission from Mease et al.13)
area and it does not accurately represent disease impact on nonskin integument, such as nails: limitations in its truth and discrimination.4 The PASI scale is not on a normal distribution curve and the upper end is infrequently used.5 Although the PASI has good intraobserver variability, it lacks significant interobserver variability.6 It also lacks a patient-reported portion, it does not measure quality of life, and it fails to account for the impact of involvement of cosmetically or functionally sensitive areas. Nevertheless, PASI remains the most common outcome measure in use, and has in fact increased in prevalence from use in 30.6% of studies in 1977 through 2000 to 57.7% in 2001 through 2006.7 Physician Global Assessment is also problematic, with various numerical scales coexisting and no
J AM ACAD DERMATOL VOLUME 72, NUMBER 2
Gottlieb et al 347
Fig 3. Results of the second Delphi exercise. The median percent importance of each of the 21 domains is represented. The dispersion around the median corresponds to the 10th through 90th percentile of all responders; each line captures 80% of stakeholder opinions, with the median representing the 50th percentile. The domains of psoriatic arthritis signs and symptoms, location and area of involvement, and psoriasis primary morphology were statistically most important.
included measure of body surface area.6,8 Neither of these tools include quality-of-life measures, although strong correlation has been found between Dermatology Life Quality Index and PASI.7 Moreover, associated risk factors of cardiovascular disease9 and depression10 are not measured. None of the established outcome measures in psoriasis were created with any patient input.
METHODS Based on the model put forth by Outcome Measures in Rheumatology (OMERACT), IDEOM’s first meeting consisted of group discussions to determine candidate domains and items essential to inclusion in psoriasis outcome measures (Fig 1). These were the concepts deemed on initial evaluation central to the diagnosis, treatment, and management of psoriatic disease. Together, the group composed a preliminary list of items and domains for psoriasis. The group consisted of dermatologists and rheumatologist mentors from the OMERACT team along with patients, pharmaceutical scientists, and
payers. The establishment of domains was followed by the first of several Delphi exercises aimed at achieving consensus among stakeholders as to the relative importance of these concepts.11 The Delphi survey is a multistep technique used to form consensus among a group of experts. Several rounds of structured questionnaires are distributed, statistically analyzed, and evaluated by the group.12 IDEOM participants prepared the Delphi using OMERACT’s ‘‘onion model,’’ represented by a central core of essential components, a middle ring of factors of undetermined significance, and an outer ring for those domains requiring further investigation13 (Fig 2). The first meeting was made possible by the generous donation from a nonmember and the National Psoriasis Foundation. Presently, funding is similar to that of OMERACT, with pharmaceutical companies paying for membership. The nonprofit organization, Advancing Innovations in Dermatology, was also instrumental in funding IDEOM.
J AM ACAD DERMATOL
348 Gottlieb et al
RESULTS The first Delphi was administered by anonymous World Wide Webebased survey in May 2013 to 155 participants, including 138 experts (dermatologists, rheumatologists, payers, regulators, and industry partners) and 17 patients, with the goal of stratifying items into the onion model. Participants were asked to rank each item as ‘‘very important,’’ ‘‘maybe important,’’ or ‘‘not important.’’ The results from 80 responders (51.6% response rate) were discussed at the second IDEOM conference in Toronto, Ontario, Canada, in July 2013. In all, 61 attendees determined which items that would have been eliminated because of a low Delphi score were important enough to remain in the item set.4 Following this procedure, IDEOM members including patients preliminarily identified 21 element groupings; these consisted of categories into which individual items fit together. In a second Delphi exercise, 208 participants were invited to weight the importance of the 21 elements. Among the 102 responders representing 8 countries, there were 79 health care providers, 12 patients, 8 pharmaceutical scientists, 1 researcher, 1 payer, and 1 professional association. The results of this second Delphi (Fig 3) were discussed among 51 attendees at the third IDEOM conference in April 2014 in Rome, Italy. The most heavily weighted (highest-scoring) domains identified by the survey were psoriasis morphology, location and area, and psoriatic arthritis. At the meeting, participants agreed that we currently lack adequate measures for the morphological extent of psoriatic disease, including characterization of such presentations as erosions, pustules, and fissures. In addition, the group found nail involvement and inverse or intertriginous area involvement to be underrecognized by current disease tools. The salience of location of plaques, for example in visible areas of the body, was discussed at great length, with patients suggesting that location is more important even than total body surface area. Plaques on the face, distal arms and legs, and hands, it was thought, should be more heavily weighted, corroborating findings from the literature.7 Participants expressed the need for sensitive and reliable measures to adequately capture disease severity that are feasible in both trial and practice settings. Patients and physicians agreed that psoriatic arthritis assessment was an essential component of outcome measurement and that loss of independence from functional disability was rarely addressed in psoriasis trials. In fact, many dermatologists do not screen for psoriatic arthritis in the psoriasis visits.14 Discussion also revealed that the impact of psoriasis-specific
FEBRUARY 2015
symptoms such as pruritus, inflexibility from thick plaques, and skin pain are not adequately captured.
FUTURE DIRECTIONS IDEOM members structured plans for the next iteration of outcome development in psoriatic disease. Before administering the next Delphi, a team of dermatologists and patients regrouped elements into the following core areas: pathophysiological manifestations, adverse events, life impact, death, resource use/economic impact, and contextual factors. IDEOM will recruit more patients through outreach to international patient groups, with the goal of having equal patient, provider, and other stakeholder contributions in preparation for the next Delphi planned for 2015. REFERENCES 1. Gottlieb AB, Swerlick RIDEOM. International Dermatology Outcomes Measures; a call to action. Psoriasis Forum. 2013; 19:107-109. 2. Horn EJ, Fox KM, Patel V, Chiou CF, Dann F, Lebwohl M. Are patients with psoriasis undertreated? Results of National Psoriasis Foundation survey. J Am Acad Dermatol. 2007;57:957-962. 3. Feldman SR, Krueger GG. Psoriasis assessment tools in clinical trials. Ann Rheum Dis. 2005;64(Suppl):ii65-ii73. 4. Gottlieb AB, Armstrong AW, Christensen R, et al. The International Dermatology Outcome Measures initiative as applied to psoriatic disease outcomes: a report from the GRAPPA 2013 meeting. J Rheumatol. 2014;41:1227-1229. 5. Armstrong AW, Parsi K, Schupp CW, Mease PJ, Duffin KC. Standardizing training for psoriasis measures: effectiveness of an online training video on Psoriasis Area and Severity Index assessment by physician and patient raters. JAMA Dermatol. 2013;149:577-582. 6. Gottlieb AB, Armstrong AW. Psoriasis outcome measures: a report from the GRAPPA 2012 annual meeting. J Rheumatol. 2013;40:1428-1433. 7. Heredi E, Rencz F, Balogh O, et al. Exploring the relationship between EQ-5D, DLQI and PASI, and mapping EQ-5D utilities: a cross-sectional study in psoriasis from Hungary. Eur J Health Econ. 2014;15(Suppl):S111-S119. 8. Callis Duffin K, Gottlieb AB. Outcome measures for psoriasis severity: a report from the GRAPPA 2012 annual meeting. J Rheumatol. 2013;40:1423-1424. 9. Gelfand JM, Neimann AL, Shin DB, Wang X, Margolis DJ, Troxel AB. Risk of myocardial infarction in patients with psoriasis. JAMA. 2006;296:1735-1741. 10. Gupta MA, Gupta AK. Depression and suicidal ideation in dermatology patients with acne, alopecia areata, atopic dermatitis and psoriasis. Br J Dermatol. 1998;139:846-850. 11. Boers M, Brooks P, Strand CV, Tugwell P. The OMERACT filter for outcome measures in rheumatology. J Rheumatol. 1998;25:198-199. 12. Hasson F, Keeney S, McKenna H. Research guidelines for the Delphi survey technique. J Adv Nurs. 2000;32:1008-1015. 13. Mease PJ, Clauw DJ, Christensen R, et al. Toward development of a fibromyalgia responder index and disease activity score: OMERACT module update. J Rheumatol. 2011;38:1487-1495. 14. Kimball AB, Guerin A, Tsaneva M, et al. Economic burden of comorbidities in patients with psoriasis is substantial. J Eur Acad Dermatol Venereol. 2011;25:157-163.
