Rare disease drugmakers to Congress: Don't gut the accelerated approval pathway
The controversy over the FDA’s accelerated approval pathway is heating up.
Last week, the FDA’s top oncology official Rick Pazdur said the pathway is “under attack,” largely due to the agency’s recent accelerated approval of Biogen’s controversial Alzheimer’s drug and the surrogate endpoint used in that decision. In the meantime, three accelerated approval indications have been pulled since July 1 (two from Bristol Myers Squibb and one from Merck in recent weeks), even as Pazdur called on critics of the pathway to not miss the more positive, big picture, with some cancer drugs proving to be enormously helpful and approved years before their confirmatory trials were completed.
Now, a coalition of rare disease drugmakers is seeking to defend the use of the pathway, raising concerns with a recent call from the nonpartisan MACPAC to increase rebates around drugs approved under the FDA’s accelerated pathway. The rare disease firms claim such a move on rebates would disincentivize drug developers from pursuing therapies in otherwise intractable disease areas, the coalition said in a recent letter to top congressional health committees.
While MACPAC did not recommend a specific increase in rebates for those treatments approved under the accelerated pathway, the agency, which advises Congress and HHS, notes that the amount needs to be significant enough to “provide a meaningful reduction in spending and provide a strong incentive to encourage completion of the confirmatory trial, but not so large as to discourage development of drugs for conditions that disproportionately affect Medicaid beneficiaries.”
Providing that encouragement to complete the confirmatory trial could be helpful in some ways. For instance, with Sarepta, its confirmatory trial for the controversial accelerated approval of its DMD drug Exondys 51 may end up taking a decade to complete.
But the Rare Disease Company Coalition — composed of drugmakers including Alnylam, Sarepta, Agios, and others, some of which have won approvals via the accelerated pathway in recent years — disagree with the MACPAC recommendations, maintaining that higher rebates on accelerated approval drugs for rare diseases may actually do the reverse and delay those post-approval confirmatory trials even further.
“Targeting a distinct, and higher rebate for accelerated approval therapies, as MACPAC’s proposal does, could deprive patients who suffer from certain conditions the important, safe, and effective therapies they need. In many cases, these therapies are the only effective course of treatment for their disease. It is a striking omission that the MACPAC proposal did not include an analysis of the impact on patient access to treatments nor consider the voice of impacted communities,” the coalition wrote.
While only 7% of rare diseases have an FDA-approved treatment, the coalition explains how members of Congress interested in further strengthening the accelerated approval pathway should “recognize the harmful consequences of undercutting reimbursement, coverage and patient access to accelerated approved therapies and instead focus on meaningful opportunities to optimize FDA’s governance and implementation of this pathway in a way that accounts for evolving science and data generation in the post-market setting and continues to keep patients front and center.”
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