A New Era for Drug Development: Vinay Prasad Takes the Helm at FDA’s Biologics Division

A long-standing contributor to research and conversations around drug policy, medical evidence, and government regulation in oncology, Vinayak (Vinay) Prasad, MD, MPH, has been appointed director of the Food and Drug Administration (FDA) Center for Biologic Evaluation and Research (CBER).

In a statement to the public, the 27th Commissioner of Food and Drugs, Martin Makary, MD, MPH said, “I’m proud to welcome Dr. Vinay Prasad as the new head of FDA’s Center for Biologics Evaluation & Research. With 500+ peer-reviewed publications and two books, Dr. Prasad brings the kind of scientific rigor, independence, and transparency we need at CBER—a significant step forward.”

Historically, Dr. Prasad’s laboratory at the University of California, San Francisco (UCSF) has examined the practices of biopharmaceutical companies and government agencies, particularly about clinical trials. His work has raised questions about the potential risks and benefits of certain trials, including whether some may offer limited clinical value or involve unknown risks to patients. Additionally, the FDA’s review process for such trials has been noted for its complexity and duration, which can sometimes delay outcomes or lead to results that differ from initial expectations.

Today, Dr. Prasad is hoping to make an impact from the inside out. In an FDA Direct conversation with the commissioner, he said, “At CBER, we govern a huge portfolio of medical products, including vaccines, gene therapy, cellular therapy, a lot of the most innovative things you read about in the news are things coming from CBER. Not to say the other departments here don’t do innovative products. They do, but I think I’m particularly excited about what’s been coming out in the last decade through [the] CBER division, and we hope to always strike the balance between data, evidence, innovation, and [the] entrepreneurial spirit of America.”

Vaccines, gene therapies, and cellular therapies all have a place in treating hematologic malignancies today. Some exist mainly in the clinical trial setting, but many have reached the clinic. A common public belief about Dr. Prasad has been that he is against vaccinations.

During the FDA Direct conservation, Dr. Prasad denied being “anti-vax.” He explained that instead, he is careful about what to vaccinate and when, and if there is enough clinical evidence to support it. As an example, he discussed criticisms he raised during the COVID-19 pandemic.

“I think vaccines are like drugs—when given at the right time, in the right moment [for] the right person, they’re lifesaving. But just like drugs, they need to be evaluated on a case-by-case basis and always taken to the context that you’re giving. Marty and I, throughout the pandemic, were proponents of vaccines for the people in whom it had a huge benefit, but we were always a bit skeptical, from a scientific standpoint, about perhaps overdoing it in some low-risk populations.”

CBER has played a role in Vividencel receiving FDA Fast Track Designation for the treatment of acute myeloid leukemia (AML). As demonstrated in the phase 2 ADVANCE trial, this cell-based vaccine has shown potential to boost the production of T-cells that target and kill cancer cells, reducing immune-suppressive T-cells in patients with AML. Additionally, the FDA has granted two investigational new drug (IND) applications for cancer vaccines being studied in blood cancers. One is WGC-043, an mRNA-based vaccine targeting Epstein-Barr virus–associated cancers, which has shown preliminary efficacy and tolerability. The other is DSP-7888, a peptide vaccine under investigation for hematologic malignancies and solid tumors, which has also been granted FDA Orphan Drug Designation for treating myelodysplastic syndromes.

Overall, Dr. Prasad’s stance is that vaccines are important for the practice of medicine in general, but that there needs to be a better balance of moving at a reasonable pace and getting the necessary evidence to move forward. He explained his plan for vaccines at the FDA in detail, again using the pandemic as a backdrop.

“I think ultimately, we get excited about innovative medical products, just as the FDA always has. We all continue to believe in a flexible regulatory standard, taking into account the context of a disease—whether it’s rare, if it’s dire—we will continue to be flexible. There’s not going to be a light switch change here. But around the edges, I think there were a number of missteps along the way,” Dr. Prasad said.

The same plan Dr. Prasad and his team have to bring to the development and review of vaccines will carry over into the development of therapeutics.  Specifically, Dr. Prasad wants to lead on new requirements for surrogate endpoints in clinical research. Also, under the commissioner’s new implementations, FDA reviews going forward will be guided by AI, and less paperwork will be needed from industry.

“We have to make our submissions easier. We have to make it so sponsors don’t have to fill out information over and over again,” Dr. Prasad stated. “[We] just can’t be the same. [We] have to always be changing and adapting, and too often in big institution[s], that doesn’t happen.”

About his appointment, Dr. Prasad expressed excitement about getting started and being in the FDA facility during his first week. “I do feel sort of an energy being here, and I’m coming from a college campus. It’s a very different energy—a productive energy.”