|
Do We Need an Independent Body to Oversee FDA Decisions?
(Thursday, June 27, 2024) If you think that the FDA is a “Black Box” where critical decisions are made in a subjective and opaque manner by a few powerful executives, you are not alone. Last week’s decision by the Director of the Center for Biologics at the FDA, Dr. Peter Marks, to expand the approval of Sarepta's treatment for Duchenne Muscular Dystrophy (DMD) for practically all DMD patients, despite the product failing in all clinical trials and strong opposition from all his reviewers, is a prime example of that. FDA’s senior officials are literally able to make these decisions that impact the lives of patients and make billions for the industry, with impunity. It raises the question, why don’t we have an independent body with the ability to oversee and, if needed, overturn, FDA’s such decisions that are arguably neither based on science, nor public interest? Sarepta’s DMD drugs, and gene therapy treatments have an interesting story. The company got its first approval for a DMD drug, Exondys 51, in 2016 also based on a similar decision by another FDA executive, Janet Woodcock, who at that time overruled FDA’s internal reviewers and the Advisory Committee to approve, citing “giving hope to patients”. Although the company promised a post-market study to confirm the efficacy of Exondys 51, eight years hence, the study is still not completed, while the company has secured approval for additional DMD drugs based on similarly deficient data by exploiting this loophole in the approval process, namely that the FDA executives don’t need to care about the opinion of its own reviewers. The company openly claims to allocate hundreds of millions of dollars each year to its public relations campaigns while its budget for the post-market clinical trials is a fraction of that. The latest approval is for a gene therapy treatment which is priced at $3.2 million per patient. This treatment failed the clinical trials but was still approved by the FDA under similar circumstances for a limited number of patients. Now it has been expanded for practically all patients even after failing the primary endpoints of its clinical trials based on weak effectiveness seen for secondary endpoints. This expansion of approval is expected to exponentially increase the company’s revenue irrespective of its benefit to the patients. The company stock jumped 35% immediately upon approval. By all public accounts, while Sarepta’s DMD drugs have not fared well in the clinic or clinical trials, Sarepta’s annual revenue has steadily increased year after year and currently stands at about $1.5 Billion. The company has made more than $7 billion in revenue since its first approval while we have no numbers on how many patients it actually helped. But this is not about Sarepta, but about the FDA’s black-box approach to reviews and approval of therapies. There are many examples where the FDA has cited similar evidence to reject other treatments. This kind of subjectiveness has no place in the regulatory process. Regulatory decisions should be based on scientific evidence and justifiable rationale. Dr. Marks did not cite either when approving the latest application. And in the process hurt the morale of all those developers who played by the rules. There is a need for an independent review of the FDA’s decisions. So far, the supporters of the FDA’s decisions have argued that the regulators need to be able to make decisions without fear of reprisal but would you allow a government official who knowingly approved a defective bridge or a poorly designed building? Then why should you allow another government official who knowingly approved a defective drug? 
AUTHOR
Dr. Mukesh Kumar Founder & CEO, FDAMap Email: [email protected] Linkedin: Mukesh Kumar, PhD, RAC Instagram: mukeshkumarrac Twitter: @FDA_MAP Youtube: MukeshKumarFDAMap |
|
|
|
