EDITORIAL For reprint orders, please contact: [email protected]
Comparing treatments for idiopathic sudden sensorineural hearing loss Keywords: comparative effectiveness n corticosteroids n financial incentive n intratympanic n sudden deafness
In 2008, the US Institute of Medicine issued ‘Knowing What Works in Health Care: A Roadmap for the Nation’ and stated that patient care should be based on “the conscientious, explicit, and judicious use of current best evidence” [101] . The challenges for this endeavor are legion. They must include overcoming hur‑ dles to performing comparative effectiveness research (CER) and encouraging healthcare providers to adopt practices in accordance with the findings of CER. In May 2011, Rauch et al. published results of a multicenter, randomized, prospective, comparative effectiveness trial of oral corticosteroids versus intra‑ tympanic steroid injections for sudden sensorineural hearing loss (SSNHL) [1] , an idiopathic condition that strikes 1:5000 people annually. Oral treatment has been standard practice since a landmark paper in 1980 demonstrated efficacy [2] . Intratympanic therapy has gained wide popularity in the last 10–15 years, largely based upon the assumption that intratympanic injection could achieve higher drug concentrations at the target inner ear with less risk of the systemic side effects of oral steroid therapy. Using a noninferiority design, no difference in effectiveness was found between the two treatments. In cases with the most severe hearing loss there was actually a slight trend for better outcome in the oral treatment group. It was also found that both treatment groups exhibited similar rates of the expected and easily managed side effects associated with corticosteroids. From these data, it was concluded that oral treatment should remain the standard of care, but that intratympanic administration was a suit‑ able alternative if, in the judgment of the physician and patient, there were other reasons to opt for intratympanic treatment. While the objective of the SSNHL study was to compare two drug treatments, several other issues were illuminated, issues that have greater general relevance than just treating SSNHL. First, it is notable that a prescription for oral prednisone costs approximately US$6 while, during the years of the SSNHL study, physician reimbursement for a series of intratympanic steroid injections was US$2700 per patient. In January 2011, the US Center for Medicare and Medicaid Services (CMS) pub‑ lished a revised policy for reimbursement of intratympanic drug therapy. The new policy eliminated the 90‑day ‘global’ reimbursement and implemented a fee of US$172 per injection. Most patients receive a series of four injections, at a total cost of US$688. The SSNHL study intended to develop evidence to support best clinical practices. However, we must consider the possibility that financial incentives may sometimes be stronger than evidence as a driver of practice patterns. Because CMS reduced the financial incentive to inject corti‑ costeroids into the ear at about the same time that evidence of equivalence with oral steroid treatment was published, we will never know if the expected future decline in intratympanic therapy was driven by one or the other. Concordance or
10.2217/CER.11.8 © 2012 Future Medicine Ltd
1(1), 5–7 (2012)
Steven D Rauch*
Lisa N Geller Harvard Medical School, Mass. Eye & Ear Infirmary, Boston, MA, USA *Author for correspondence: Tel: +1 617 573 3644 Fax: +1 617 573 3939 [email protected]
part of
ISSN 2042-6305
5
EDITORIAL
Rauch & Geller
discordance of financial and evidentiary incen‑ tives remains a topic for future investigation and public policy. A second set of issues arose around the logistics of conducting CER. In the SSNHL study, despite conservative estimates for sub‑ ject accrual, two additional years (and no-cost funding extensions) were required to achieve our targeted accrual. The study began with eight clinical sites and was expanded to 16 sites to achieve the target. Planned site budgets were changed to capitated reimbursement to afford continuation of the study, and to incentivize enrollment at participating sites. The SSNHL study took over 9 years from the initial plan‑ ning meeting to publication. As with any clini‑ cal research, the research time frame can be quite lengthy. Meanwhile, the practice land‑ scape may change over time due to factors other than comparative effectiveness evidence (e.g., clinical innovation or financial incentives). By the time a lengthy comparative effectiveness study is completed, it may be too late to influ‑ ence practice patterns. Timely accomplishment of studies should be an ongoing consideration for CER. “Mentoring young clinical investigators to inspire them along the path of CER will necessitate frank discussions of the barriers to success and the need to cultivate parallel research and publication activities with more frequent output than can be expected from a multicenter comparative effectiveness trial.”
When the SSNHL study was originally con‑ ceived, and again when the number of clini‑ cal sites was expanded, special emphasis was placed on identifying young collaborators with the interest and motivation to join the study and thereby gain clinical trials experience. Our health education system is training an increas‑ ing number of clinicians who can design and execute clinical trials. These clinicians develop expertise in research methodology and biosta‑ tistics that is necessary, but not sufficient, to meet the needs of CER. A host of additional questions and issues – economic, sociologic, geographic, ethical and political – must be considered and addressed in order to reap the benefits of hard-won comparative effective‑ ness evidence. Such questions and issues must be included in the education of comparative
6
J. Compar. Effect. Res. (2012) 1(1)
effectiveness researchers. Likewise, as we seek to grow the cadre of investigators qualified to perform CER, we must consider their academic success and viability. It took 9 years to accom‑ plish the SSNHL study. One major publication every decade will not make for a successful aca‑ demic career. Mentoring young clinical inves‑ tigators to inspire them along the path of CER will necessitate frank discussions of the barriers to success and the need to cultivate parallel research and publication activities with more frequent output than can be expected from a multicenter comparative effectiveness trial. “The Golden Rule – “he who has the gold makes the rules” – predicts that the same research question might have different answers depending upon who sponsors the study. The clamor for CER is coming from many quarters – third party payors, patients, governments and physician groups.” Finally, who will pay for CER? The SSNHL study, sponsored by the NIH Institute of Deafness and Other Communication Disorders, cost US$5.8 million, about the same as five R01 basic or translational science grants. Where is the political will and intel‑ lectual integrity to fund such research, par‑ ticularly if it comes at the expense of reducing funding for basic and translational research? The answer to this question could have pro‑ found consequences. The Golden Rule – “he who has the gold makes the rules” – predicts that the same research question might have dif‑ ferent answers depending upon who sponsors the study. The clamor for CER is coming from many quarters – third party payors, patients, governments and physician groups. Presumably payors, including health maintenance organi‑ zations and governments, have an incentive to pursue such research for both quality of care and cost–effectiveness reasons. Pharmaceutical companies are unlikely to be interested in funding such research unless there is a strong probability that their drug will outperform other products marketed for the same indica‑ tion. Because of differing agendas of each of these groups, the prioritization of study topics, and the choice of study hypotheses and study designs may vary widely. How do we align these needs to pull in the same direction, toward bet‑ ter patient care, more available and equitable
future science group
Comparing treatments for idiopathic sudden sensorineural hearing loss
patient care, and wiser, more economical uti‑ lization of limited healthcare resources? In the months and years to come, we hope that read‑ ers will avail themselves of this new Journal of Comparative Effectiveness Research to create a forum for these discussions and debates. Financial & competing interests disclosure The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. No writing assistance was utilized in the production of this manuscript.
future science group
EDITORIAL
References 1
Rauch SD, Halpin CF, Antonelli PJ et al. Oral vs intratympanic corticosteroid therapy for idiopathic sudden sensorineural hearing loss. JAMA 305(20), 2071–2079 (2011).
2
Wilson WR, Byl FM, Laird N. The efficacy of steroids in the treatment of idiopathic sudden sensorineural hearing loss: a double-blind clinical study. Arch. Otolaryngol. 106(12), 772–776 (1980).
■■ Website 101 Committee on reviewing evidence to identify highly
effective clinical services, Institute of Medicine. Knowing What Works in Health Care: A Roadmap for the Nation. Eden J, Wheatley B, McNeil B, Sox H (Eds). National Academy Press, Washington, DC, USA (2008). www.nap.edu/catalog.php?record_id= 12038
www.futuremedicine.com
7
Comparing treatments for idiopathic sudden sensorineural hearing loss Keywords: comparative effectiveness n corticosteroids n financial incentive n intratympanic n sudden deafness
In 2008, the US Institute of Medicine issued ‘Knowing What Works in Health Care: A Roadmap for the Nation’ and stated that patient care should be based on “the conscientious, explicit, and judicious use of current best evidence” [101] . The challenges for this endeavor are legion. They must include overcoming hur‑ dles to performing comparative effectiveness research (CER) and encouraging healthcare providers to adopt practices in accordance with the findings of CER. In May 2011, Rauch et al. published results of a multicenter, randomized, prospective, comparative effectiveness trial of oral corticosteroids versus intra‑ tympanic steroid injections for sudden sensorineural hearing loss (SSNHL) [1] , an idiopathic condition that strikes 1:5000 people annually. Oral treatment has been standard practice since a landmark paper in 1980 demonstrated efficacy [2] . Intratympanic therapy has gained wide popularity in the last 10–15 years, largely based upon the assumption that intratympanic injection could achieve higher drug concentrations at the target inner ear with less risk of the systemic side effects of oral steroid therapy. Using a noninferiority design, no difference in effectiveness was found between the two treatments. In cases with the most severe hearing loss there was actually a slight trend for better outcome in the oral treatment group. It was also found that both treatment groups exhibited similar rates of the expected and easily managed side effects associated with corticosteroids. From these data, it was concluded that oral treatment should remain the standard of care, but that intratympanic administration was a suit‑ able alternative if, in the judgment of the physician and patient, there were other reasons to opt for intratympanic treatment. While the objective of the SSNHL study was to compare two drug treatments, several other issues were illuminated, issues that have greater general relevance than just treating SSNHL. First, it is notable that a prescription for oral prednisone costs approximately US$6 while, during the years of the SSNHL study, physician reimbursement for a series of intratympanic steroid injections was US$2700 per patient. In January 2011, the US Center for Medicare and Medicaid Services (CMS) pub‑ lished a revised policy for reimbursement of intratympanic drug therapy. The new policy eliminated the 90‑day ‘global’ reimbursement and implemented a fee of US$172 per injection. Most patients receive a series of four injections, at a total cost of US$688. The SSNHL study intended to develop evidence to support best clinical practices. However, we must consider the possibility that financial incentives may sometimes be stronger than evidence as a driver of practice patterns. Because CMS reduced the financial incentive to inject corti‑ costeroids into the ear at about the same time that evidence of equivalence with oral steroid treatment was published, we will never know if the expected future decline in intratympanic therapy was driven by one or the other. Concordance or
10.2217/CER.11.8 © 2012 Future Medicine Ltd
1(1), 5–7 (2012)
Steven D Rauch*
Lisa N Geller Harvard Medical School, Mass. Eye & Ear Infirmary, Boston, MA, USA *Author for correspondence: Tel: +1 617 573 3644 Fax: +1 617 573 3939 [email protected]
part of
ISSN 2042-6305
5
EDITORIAL
Rauch & Geller
discordance of financial and evidentiary incen‑ tives remains a topic for future investigation and public policy. A second set of issues arose around the logistics of conducting CER. In the SSNHL study, despite conservative estimates for sub‑ ject accrual, two additional years (and no-cost funding extensions) were required to achieve our targeted accrual. The study began with eight clinical sites and was expanded to 16 sites to achieve the target. Planned site budgets were changed to capitated reimbursement to afford continuation of the study, and to incentivize enrollment at participating sites. The SSNHL study took over 9 years from the initial plan‑ ning meeting to publication. As with any clini‑ cal research, the research time frame can be quite lengthy. Meanwhile, the practice land‑ scape may change over time due to factors other than comparative effectiveness evidence (e.g., clinical innovation or financial incentives). By the time a lengthy comparative effectiveness study is completed, it may be too late to influ‑ ence practice patterns. Timely accomplishment of studies should be an ongoing consideration for CER. “Mentoring young clinical investigators to inspire them along the path of CER will necessitate frank discussions of the barriers to success and the need to cultivate parallel research and publication activities with more frequent output than can be expected from a multicenter comparative effectiveness trial.”
When the SSNHL study was originally con‑ ceived, and again when the number of clini‑ cal sites was expanded, special emphasis was placed on identifying young collaborators with the interest and motivation to join the study and thereby gain clinical trials experience. Our health education system is training an increas‑ ing number of clinicians who can design and execute clinical trials. These clinicians develop expertise in research methodology and biosta‑ tistics that is necessary, but not sufficient, to meet the needs of CER. A host of additional questions and issues – economic, sociologic, geographic, ethical and political – must be considered and addressed in order to reap the benefits of hard-won comparative effective‑ ness evidence. Such questions and issues must be included in the education of comparative
6
J. Compar. Effect. Res. (2012) 1(1)
effectiveness researchers. Likewise, as we seek to grow the cadre of investigators qualified to perform CER, we must consider their academic success and viability. It took 9 years to accom‑ plish the SSNHL study. One major publication every decade will not make for a successful aca‑ demic career. Mentoring young clinical inves‑ tigators to inspire them along the path of CER will necessitate frank discussions of the barriers to success and the need to cultivate parallel research and publication activities with more frequent output than can be expected from a multicenter comparative effectiveness trial. “The Golden Rule – “he who has the gold makes the rules” – predicts that the same research question might have different answers depending upon who sponsors the study. The clamor for CER is coming from many quarters – third party payors, patients, governments and physician groups.” Finally, who will pay for CER? The SSNHL study, sponsored by the NIH Institute of Deafness and Other Communication Disorders, cost US$5.8 million, about the same as five R01 basic or translational science grants. Where is the political will and intel‑ lectual integrity to fund such research, par‑ ticularly if it comes at the expense of reducing funding for basic and translational research? The answer to this question could have pro‑ found consequences. The Golden Rule – “he who has the gold makes the rules” – predicts that the same research question might have dif‑ ferent answers depending upon who sponsors the study. The clamor for CER is coming from many quarters – third party payors, patients, governments and physician groups. Presumably payors, including health maintenance organi‑ zations and governments, have an incentive to pursue such research for both quality of care and cost–effectiveness reasons. Pharmaceutical companies are unlikely to be interested in funding such research unless there is a strong probability that their drug will outperform other products marketed for the same indica‑ tion. Because of differing agendas of each of these groups, the prioritization of study topics, and the choice of study hypotheses and study designs may vary widely. How do we align these needs to pull in the same direction, toward bet‑ ter patient care, more available and equitable
future science group
Comparing treatments for idiopathic sudden sensorineural hearing loss
patient care, and wiser, more economical uti‑ lization of limited healthcare resources? In the months and years to come, we hope that read‑ ers will avail themselves of this new Journal of Comparative Effectiveness Research to create a forum for these discussions and debates. Financial & competing interests disclosure The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. No writing assistance was utilized in the production of this manuscript.
future science group
EDITORIAL
References 1
Rauch SD, Halpin CF, Antonelli PJ et al. Oral vs intratympanic corticosteroid therapy for idiopathic sudden sensorineural hearing loss. JAMA 305(20), 2071–2079 (2011).
2
Wilson WR, Byl FM, Laird N. The efficacy of steroids in the treatment of idiopathic sudden sensorineural hearing loss: a double-blind clinical study. Arch. Otolaryngol. 106(12), 772–776 (1980).
■■ Website 101 Committee on reviewing evidence to identify highly
effective clinical services, Institute of Medicine. Knowing What Works in Health Care: A Roadmap for the Nation. Eden J, Wheatley B, McNeil B, Sox H (Eds). National Academy Press, Washington, DC, USA (2008). www.nap.edu/catalog.php?record_id= 12038
www.futuremedicine.com
7
