Former FDA Oncology Reviewer Paz Vellanki on Novel Endpoints and Early FDA Engagement Under CDER's ARC Program

In an interview with BioPharm International®, Paz Vellanki, MD, PhD, vice president of clinical development at Precision for Medicine, discusses what the Center for Drug Evaluation and Research's (CDER's) Accelerating Rare disease Cures (ARC) program means for sponsors developing therapies for rare diseases and rare tumors. Vellanki spent 7 years in FDA's Division of Oncology 2, including 2 years as an associate director.

How could ctDNA help rare tumor programs that lack validated end points?

Vellanki explains how circulating tumor DNA (ctDNA) could support drug development in rare cancers. "Oftentimes you don't have clinically validated endpoints for rare diseases. Is there a way to get an early look at tumor response using circulating tumor DNA?" she said. "That would be a great way to utilize ctDNA if we can help support drug development there."

The challenge, she notes, is generating enough evidence in small patient populations. One open question is whether data from more common tumor types could be extrapolated to rare diseases. She points to the Rare Disease Endpoint Advancement Pilot Program as a forum where sponsors can discuss novel end points with FDA.

Vellanki sees ARC's updated 5-year roadmap as building on the program's earlier pilots rather than changing course. "I wouldn't say that this is necessarily different than goals that FDA always has. I think they always encourage sponsors to come to FDA, talk with them about their drug development programs," she said. What the roadmap adds, she explains, is more ways to engage, including pilot programs with more informal sponsor meetings, patient listening sessions, and public workshops.

Her main advice to sponsors is to engage FDA before the end point strategy is locked in. "I think the biggest misstep I was seeing is that the sponsors aren't reaching out to FDA," Vellanki said. She distinguished between an early-phase protocol that FDA deems "safe to proceed" and a trial whose data are meant "to support an approval of a drug or a biologic."

"I really think sponsors need to be engaging early on, beginning with the end in mind," Vellanki said.

About the Speaker

Paz Vellanki, MD, PhD, vice president of clinical development, oncology, Precision for Medicine

Paz Vellanki partners with life sciences companies on clinical development strategy and regulatory planning. Before joining Precision for Medicine, she served as associate director of the Division of Oncology 2 at the FDA where she oversaw drug development and regulatory decisions for therapeutics in lung cancer, head and neck cancer and rare tumors and molecular subsets of cancers.