FDA’s Real-Time Review of Trial Data Is a Step Forward, Industry Says

February 25, 2019

An FDA pilot program that offers real-time review of oncology trial data is a positive move toward speeding drug development, sponsor participants say.

“We think what the agency is doing is a net positive for patients and industry,” says Michael Meo, director of global media relations for Novartis Oncology Communications & Advocacy. Novartis was the first participant to win approval for its drug, Kisqali, under the accelerated program.

The agency’s Center for Oncology Excellence conceived the pilot last summer as a way to reduce regulatory hurdles and get cancer treatments to market as soon after they have demonstrated safety and efficacy as possible. Under the program, which currently applies only to supplemental applications, a sponsor can begin sharing bottom-line trial data with the agency once it makes the decision to file for FDA approval, submitting findings as soon as two to four weeks after the trial’s database is locked.

The initial submission includes key raw datasets such as safety and efficacy tables and a draft of the package insert, at which point the agency will immediately begin evaluating for data integrity and adequacy.

The program has enabled the FDA “to approve several product applications within a few weeks of the application’s complete submission,” officials say. The FDA would not provide information on how many trials are in the program’s pipeline.

“We were confident in our data even before beginning talks with the FDA,” Meo says. “We were eager to bring a treatment option to premenopausal patients as quickly as possible (as well as providing greater flexibility in prescribing hormonal therapies). The [program] helped us do that.”

Three other drugs have won approval under OCE’s pilot: Merck’s Keytruda, Seattle Genetics’ Adcetris and Amgen’s Kyprolis.

To be eligible for the pilot, a drug must be likely to demonstrate substantial improvements over available therapy and have both a straightforward study design and easily interpreted endpoints. Supplements with chemistry, manufacturing or control formulation changes will be excluded, and submissions with greater complexity may be as well, according to the FDA.

In early February, Roche’s breast cancer drug, Kadcyla, became the latest drug to qualify for the program, and the company hopes to have approval by the end of the first quarter. Kadcyla is already indicated for previously treated HER2+ metastatic breast cancer and will be reviewed under the program for an additional indication for HER2-positive early breast cancer patients who don’t fully respond to drug therapy before surgery (CenterWatch Weekly, February 11, 2019).

Novartis says the experience was positive. “The process was very collaborative and transparent,” according to Meo. “Throughout the review, there was a continuous and open dialogue between Novartis and the FDA about the data, with discussions happening almost on a daily basis. Instead of scheduling formal appointments and waiting for feedback, we collaborated with each other to clarify answers in real-time.”

Although the FDA has not announced plans to replicate the program in other centers, Meo is optimistic. “We look forward to seeing how these programs evolve over time.”


-By Zack Budryk