Remember in high school when a friend told you a secret and you guarded it assiduously, only to find out that most of your classmates were already in on it? That’s a fair description of the secrecy surrounding elements of the Food and Drug Administration’s drug approval process.
As several of my former FDA colleagues and I describe in a report published Monday in JAMA Internal Medicine, it’s an open secret when a company files a new drug application (NDA), despite the FDA’s decades-long practice of treating those filings as “confidential commercial information.” That secrecy, a crucial element in the FDA’s approach to transparency, results in its withholding reams of documents under the Freedom of Information Act. (Some of this withholding — of trade secret information, for example — is eminently reasonable.)
Our study demonstrated that between 2010 and 2016, 89% of all NDA filings were announced by sponsoring companies in press releases, Securities and Exchange Commission filings, or both. And those announcements are becoming more common: In 2016, 98% of filings were announced publicly.
If the fact that an NDA has been filed is public knowledge, why does the FDA’s nondisclosure matter? Because the agency’s stubborn insistence on not acknowledging the filing ties its hands behind its back. When the agency is called by patients wishing to know if there might be new treatments for their disease, FDA staffers can’t mention NDAs for drugs that may be on the cusp of approval. And if the public knew when an NDA had been filed, the agency would be more accountable for complying with the drug review time targets set by Congress.
In principle, the FDA can disclose information that sponsoring companies have already made public. But determining whether something has been made public takes time and resources, so in practice the agency often simply resorts to nondisclosure.
But, you say, the FDA can speak without constraint when it renders a decision on the NDA, typically less than a year after it is filed. Actually, that’s not the case. Pursuing the logic behind NDA secrecy to its illogical conclusion, the FDA publicizes only its decisions to approve drugs, not those that deny approval. (If the public knew that an NDA had been rejected, it would know that an NDA had been filed … you get the picture.)
In previous research, my FDA colleagues and I demonstrated that only 15% of the material information in FDA documents denying the approval of an NDA — “complete response letters,” in FDA parlance — see the light of day in press releases or SEC filings.
This could leave unsuspecting investors sinking funds, and researchers sinking time, into chemical cousins of the unsuccessful products, instead of focusing on more promising drugs. And it deprives clinicians and patients of useful information, particularly on safety, that could affect the course of clinical care.
For the FDA to acknowledge the filing of NDAs and release complete response letters may well require regulatory changes. But the European Medicines Agency publishes monthly lists of drugs being evaluated for marketing and releases its equivalent of complete response letters.
In January 2018, then-FDA Commissioner Scott Gottlieb indicated that the FDA was considering whether to release significant portions of the letters. This approach would have the added benefit of allowing the FDA to explain in detail why it did not permit a drug to be marketed, countering frequent allegations of agency nitpicking. But our analysis showed that 87% of complete response letters included at least one safety or efficacy concern standing between the product and FDA approval.
Sixteen months after Gottlieb’s announcement, there is no outward sign of any effort to release the letters.
The pharmaceutical industry has claimed that releasing complete response letters would provide an advantage to competitors. But announcing the filing of NDAs or the release of complete response letters would not result in an uneven playing field; it would simply create a different but still level field in which the benefits to the public and industry as a whole took precedence over the narrow interests of the companies affected by the disclosure.
Peter G. Lurie, M.D., is the president of the Center for Science in the Public Interest and a former associate commissioner for public health strategy and analysis at the FDA.