President Trump has signaled—often and recently—that he believes drug approval moves too slowly. Meeting with drug manufacturers at the White House on Jan. 31, Trump promised quick yes-or-no approval decisions on new drugs (and stressed the need for lower prices and U.S.-based manufacturing).
But the FDA moves fast already—fast enough, say some experts who worry about the standards used to move drugs through review pathways like Priority Review, Breakthrough Therapy, Accelerated Approval, and Fast Track. And when the president pushes for even less rigor, these questions become even more pressing. More frictionless approval could mean sacrificing proof of drugs’ effectiveness, leading to approval of glorified (and expensive) placebos. Even when approved, insurers might not cover them.
A case study for this debate emerged last September when the FDA approved a drug under the accelerated approval pathway. Marketed as Exondys 51 and made by Sarepta Therapeutics, the drug is intended to treat a subset of young boys with a mutation that causes rare and fatal disease, Duchenne muscular dystrophy. It’s a horrible disease that previously had no approved treatment, and it generated a fierce clamor among patient advocacy groups for FDA approval. The subject caught the attention of members of Congress, who wrote letters to FDA advocating for the drug. There was also concern that Sarepta—the company closest to putting a Duchenne treatment on the market—would fold if approval were denied.
These strong incentives tipping the scales toward approval led to an FDA review that generated a media outpour. By the time of approval, a top official had overridden several review-board recommendations not to approve and the FDA Commissioner had called to retract publication of one of the two clinical trials used for approval. But despite questions surrounding a drug described by a reviewing scientist as an “elegant placebo,” doctors are set to prescribe and patients are set to begin the $300,000 treatment.
To gain insight into the situation, the Media Freedom and Information Access Clinic partnered with a science journalist to request records and correspondence under the federal Freedom of Information Act. Knowing the standards applied in this case could help interested parties understand how the system works and how various Trump-administration policies would alter it.
The request is pending, but regardless of its result, a galvanized movement for healthcare transparency will only become more important. An FDA that is publicly accountable and operating at full capacity is in the best interests of all Americans. As the agency integrates with the Trump team, it is vital that public attention be paid to all links in the chain between pharmaceutical companies, FDA, and consumers. A tool like FOIA can be useful in ensuring healthcare integrity.