The Institute for Clinical and Economic Review (ICER) has set up a draft document to review evidence of the effectiveness of treatments for Duchenne muscular dystrophy (DMD).
The review, “Deflazacort, Eteplirsen, and Golodirsen for Duchenne Muscular Dystrophy: Effectiveness and Value,” focuses on Sarepta Therapeutics’ Exondys 51 (eteplirsen) and Golodirsen (SRP-4053), and on PTC Therapeutics’ Emflaza (deflazacort), and will include input from diverse stakeholders, including patients and their families, researchers, and the therapies’ developers.
The document is open for comments from the public and suggestions for improvements. Comments may be submitted by email to [email protected] until 5 p.m. Thursday (Feb. 1). ICER’s Patient Participation Guide and Manufacturer Engagement Guide provide guidance for submitting public comments, including suggestions for what types of information may be most useful.
Additionally, ICER welcomes stakeholders’ assessments on examples of poor-value of care for DMD that will be used to draft strategies for high-value treatment and to reduce waste.
“ICER looks forward to continued engagement with stakeholders throughout its review and encourages comments to refine our understanding of the clinical effectiveness and value of preventive treatments,” the document states.
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Specifically, the draft will include evidence from randomized and non-randomized clinical trials, as well as high-quality systematic reviews and comparative group studies, with an emphasis on patients’ long-term outcomes and uncommon adverse side effects.
The draft will focus on clinical and patient-centered outcomes, as well as on the therapies’ safety. The key outcomes being analyzed are: mortality; mobility; cardiac issues (such as, cardiomyopathy, arrhythmias, and heart failure); respiratory complications; health-related quality of life; caregiver burden, and; education and employment-related outcomes (e.g., ability to attend work or school).
Safety outcomes include adverse and serious adverse events; serious adverse events leading to discontinuation of drug; weight gain; incidence of cataracts; hirsutism (unwanted hair growth); bone fractures, and; deaths.
Specifically, the review will compare Emflaza to prednisone (a corticosteroid sold under the name Sterapred), and Exondys 51 and Emflaza plus corticosteroids to corticosteroids alone.
Corticosteroids are the mainstay of therapy for DMD, and previous trials showed that treatment with the anti-inflammatory glucocorticoid prednisone or prednisolone improved muscle strength and function, and delayed loss of walking ability.
Following the public comment period, a revised scoping document will be available about Feb. 11.
In July, ICER’s report will be the discussed in a meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), one of ICER’s three independent evidence appraisal committees.
Exondys 51 (eteplirsen) is an exon-skipping treatment approach that for specific genetic mutations can restore production of the dystrophin protein.
The FDA approved it in 2016, making it the first approved treatment designed to target the underlying cause of DMD.
Golodirsen (SRP-4053) also is an exon-skipping therapy for DMD patients whose disease stems from a deleted part of the DMD gene known as exon 53. Sarepta Therapeutics is seeking FDA approval for Golodirsen. The FDA’s decision is expected by June.
Emflaza (deflazacort) is a corticosteroid to treat people with DMD age 5 and older, regardless of disease-causing genetic mutation.