ICER blasts FDA, PTC and Sarepta for high prices on DMD drugs Emflaza, Exondys 51
ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.
The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.
As corticosteroids and supportive care currently form the bedrock of treatment for the 6,000 young DMD patients in the US, ICER (more formally known as Institute for Clinical and Economic Review) set out to make two main comparisons with costs in mind: Is deflazacort that much more effective than prednisone, a much cheaper corticosteroid? Does eteplirsen bring a big enough improvement to warrant adding it to the standard regimen given its high price?
The answer to both questions, according to ICER, is no.
To be sure, 10 out of 17 members on its advisory council agreed that deflazacort confers a better health benefit than prednisone, as patients taking the drug appears to have better motor function and experience less weight gain. The latter point is also important to caregivers as it reduces their burden on a day-to-day basis, they acknowledged.
However, they argue that the drug’s price tag is unjustified, with the majority agreeing the longtime value for money is low.
While US patients once imported the drug for as little as $1,000 per year, Marathon jacked the price up to $89,000 after acquiring it and winning an FDA approval based on old data. PTC has subsequently acquired the controversial biotech and pledged to reduce the price.
“The events around and following the FDA approval of deflazacort is a model of how not to promote innovation,” read the report.
The panelists were less impressed by eteplirsen’s results, voting 16 to 1 that there is not adequate evidence to claim treatment with the drug is superior to corticosteroid and supportive care alone. They were also unanimously skeptical of Sarepta’s experimental treatment golodirsen, which was also included for review but not a substantial analysis due to the lack of clinical data and pricing information.
Given that eteplirsen’s efficacy has yet to be demonstrated — Sarepta has just recently submitted a plan for a confirmatory trial, with data expected in 2024 — ICER suggested it should be priced closer to the marginal cost.
The main takeaway:
Acclaim is rightly given to innovative treatments that can offer benefits for patients and families grappling with serious conditions that lead to early death. However, when treatments like eteplirsen are introduced with evidence that is wholly inadequate to demonstrate clinical benefit, at an extremely high price, and without an adequate commitment from the manufacturer to generate and share further evidence within a rapid timeline, patients, families, other patients in the health system, and even future innovators suffer significant losses.
Patient advocates at the public meeting, however, questioned ICER’s methodology. Fleur Chandler, a health economist with Duchenne UK, called their assumptions “huge, multi-layered and not tested.”
“The ICER process simply did not allow enough time to adequately review a disease as complex as DMD; for systematic literature reviews, evidence collection or robust modelling,” she said at the meeting, according to a summary.
A representative of the influential Parent Project Muscular Dystrophy further urged ICER to consider delaying their review of products gaining accelerated approval as “there is no value in conducting an assessment at a time point in which it is known in advance that ‘insufficient evidence’ will be available to conduct analyses.”
She also cautioned against ICER’s suggestion to payers about prior authorization, in which insurers set up criteria to determine who would qualify for coverage. Frequent prior authorizations could result in gaps or delays in care and undermine effectiveness, she said.
Social image: The key pathologic change of the Duchenne muscular dystrophy is the myonecrosis. At an early phase, necrotic fibers appear swollen, homogeneous and deeply eosinophilic. Shutterstock