For years, opponents of “right to try” warned that loosening the rules for rare-disease therapy approvals would turn drug regulation into an anything-goes environment, where inferior drugs would be pushed on unsuspecting patients. The irony is that they got it backward: The far greater threat is standards that seem to change by the week and risk aversion that seems more about politics than patients’ lives. 

Capricor has spent years in a bureaucratic ordeal with FDA officials, spending huge amounts of time and money jumping through hoops so that Deramiocel could reach patients. Just in the past year, the FDA declined to approve Deramiocel, reopened its application after receiving additional data, and then convened the panel that concluded that the company had to go even further with its evidence. 

That’s not protecting patients or stopping Big Pharma. It’s simply making sure that patients suffer while officials protect their own salaries. 

Capricor is not alone. Families relying on newly approved cell and gene therapies have already watched evidence once judged adequate get second-guessed after the fact. Huntington’s disease advocates watched promising therapies face shifting expectations. Across rare diseases, companies that invested years in following FDA guidance have increasingly found themselves confronting new standards at the end of the approval process instead of predictable ones established from the beginning.

Scientists will always disagree about data, and no one expects the FDA to guarantee positive outcomes for the drugs it reviews. What we need is a coherent process in which potential roadblocks are identified as early as possible and evidentiary standards are transparently and consistently applied. 

Rare-disease families and their advocates feel this institutional disaster most keenly and have become some of the loudest voices demanding FDA accountability. They were particularly dissatisfied with Commissioner Marty Makary and biologics chief Vinay Prasad, both of whom stepped down after sustained criticism, including fierce opposition from patient communities.

Those departures did not resolve the uncertainty. We have now seen another vote leave patients waiting for answers they simply cannot afford to wait for.

The FDA owes the public an answer: Why should patients believe this is simply science rather than institutional retaliation against the very advocates who demanded accountability? 

Until that question is answered, the FDA should halt the use of advisory committees, or adcomms, for drug applications until the system is reformed. These panels of outside experts are convened to assess evidence and advise the agency on difficult applications. Before they resume, the FDA must ensure that committee members have relevant rare-disease expertise, disclose the precise questions panels will consider, and clearly define the evidentiary standards they will apply — all ordered toward putting patients first.

Sponsors should also have a meaningful opportunity to address disagreements before a public vote. Patient experience should inform deliberations without replacing evidence.

DELAYED TO DEATH: HOW HEALTHCARE MIDDLEMEN RUN OUT THE CLOCK ON SICK AMERICANS

Adcomms should strengthen decisions through independent expertise. They must not introduce an unpredictable second standard at the end of an already lengthy process.

Why did Capricor fail the test? Until we know the answer, it’s just another piece of evidence that the FDA has become a brick wall for the people it’s most supposed to help. 

Tricia Flanagan is the founder and CEO of Azuza Laboratories, a bioscientist, immuno-oncology specialist, and rare disease advocate whose career spans biotechnology, medicine, and public policy.