Dr. Vinay Prasad: Balancing Innovation and Evidence in Rare Disease Treatment

When Dr. Martin Makary took his seat as FDA Commissioner earlier this year, his early remarks signaled a fresh era for rare disease and gene therapy developers. From promising to “customize the approval process to the condition” to voicing support for faster reviews in ultra-rare populations, Makary struck an optimistic tone that resonated with RareMoon’s clients.

Just a few weeks later, the appointment of Dr. Vinay Prasad as Director of the Center for Biologics Evaluation and Research (CBER) sparked concern across the biotech space, particularly among developers of cell and gene therapies, as reported by news outlets and analysts.

Prasad has not shied away from controversy, hosted a podcast called “Plenary Session,” covering topics including medicine, oncology, and health policy, frequently participated in media discussions that challenged mainstream public health narratives, and questioned the industry’s influence on regulatory decisions. Prasad has been recognized as a vocal critic of the pharmaceutical industry and FDA regulatory practices, particularly regarding accelerated approval pathways, surrogate endpoints in oncology, and the 2023 ELEVIDYS gene therapy approval.

Today, Prasad makes his first formal appearance before the industry at the NORD 2025 Inaugural Rare Disease Scientific Symposium. The three main topics he discussed included therapeutic approaches to lessening “years-of-life-lost,” how the FDA will make judgments in the context of novel methodologies, and how he and the FDA Commissioner plan to engage with the patient community.

Therapeutic Approaches to Lessening “Years-of-Life-Lost”

Prasad indicated that the FDA will pay attention to what he called “years of life lost” in patients diagnosed with a life-threatening disease (life expectancy of affected patients versus normal life expectancy in the United States), in the context of what the FDA will favor. For example, Prasad noted advancements like Novartis’ breakthrough, imatinib (Gleevec), which transformed the treatment of chronic myeloid leukemia (CML). While noting this “heroic story of medicine” and position that every rare disease patient deserves this, Prasad also acknowledged that not every medicine will be curative or transformative; some will provide patients with “stepwise advancements”, indicating that the FDA will “approve anything that is an incremental advancement.” This topic was concluded with the acknowledgement that “there is a lot of opportunity for the FDA to advance more therapies in this space.”

FDA Judgment in the Context of Evidence of Effectiveness

Prasad jumped right into this topic with the message “not every disease needs a randomized controlled trial, and not every randomized controlled trial is informative,” and understanding and having compassion for these patients who do not want to enroll in such a trial. He noted that novel trial designs are not new to him pointing out he has been long-investigating novel trial methodologies going back over 5 years with examples used to drive his talking points, including his 2015 book titled “Ending Medical Reversal: Improving Outcomes, Saving Lives” (co-authored with Adam Cifu), which examined the idea of abandoning established medical practices after being found ineffective or harmful. The example used was dostarlimab, a PD-1 inhibitor immunotherapy that has shown unprecedented results in a subset of newly diagnosed rectal cancer. In this trial, 100% of (42) patients demonstrated a clinical complete response, meaning there was no detectable tumor. Prasad pointed out that in this trial, none of the patients required “old-fashioned drugs” that don’t offer benefit.

What if studies were designed where every participant was randomized to newly developed drugs rather than randomized to “old-fashioned,” or “barbaric” drugs that don’t offer benefit, sparing patients outdated treatment options? He then moved to bespoke therapeutics, tailoring therapies to patient diagnosis, using the example of a personalized CRISPR–Cas9–based gene-editing treatment to correct a CPS1 deficiency in an infant, detailed in a study published by NEJM and presented at the 2025 American Society of Gene & Cell Therapy Annual Meeting. This case marked a historic milestone in the application of bespoke gene editing for rare, life-threatening genetic diseases. FDA is looking at these as more than one-off therapies; instead, at ways we can use these technologies to offer tailored solutions for groups of patients, such as in metabolic disease. Prasad indicated FDA is “committed to moving these therapies as quickly as possible to patients that need them,” noting that the “devil is in the details.”

Surrogate Versus Overall Survival? “It’s Both!”

While Prasad did not spend much time discussing surrogate endpoints, he did make some bold statements. He noted that the FDA will accelerate therapies by taking “action at the first sign of  promise for rare diseases,” at the “earliest sign of statistical evidence,” but “will monitor to ensure people live longer, stronger.” The FDA will hold Sponsors accountable for their post-marketing commitments.

And lastly, on the topic of real-world evidence, he reported, “Yes, we will use real-world data in every way possible to draw more conclusions about what these products are achieving.”

Commitment to Engagement

In his last few minutes on stage, Prasad noted that the FDA will “think critically, but also remain flexible”, that he and Makary are committed to engagements, including YouTube, an FDA Commissioner podcast, and peer-reviewed published literature. While the FDA will still use black boxes, “they will not be a black box themselves,” and they will make their positions as transparent as possible.

RareMoon’s takeaways:

Prasad indicated that his views have been consistent across books and papers since he was 22 years old, and he brings those views and commitments to the FDA. He maintains that FDA under his leadership, will advance all types of therapies, from curative and transformative therapies to those that offer incremental advancements to patients that need them, that the FDA is supportive of novel clinical designs rather than randomized controlled studies, will approve bespoke therapies and are committed to looking at these in the context of broad applicability and “platform technologies”, and will make their positions as transparent as possible. Ultimately, he appeared committed to cures, promising treatments, speed, and continued engagement.

RareMoon was hopeful following Makary’s early comments and then a little wary following Prasad’s appointment, not based on personal experience, but on public perception. Today’s remarks from Prasad at NORD’s Scientific Symposium confirmed our optimism, raised valid questions, and showed signs of collaboration, possibly setting the tone for the coming months.

Stay tuned. We’ll keep you posted.