FDA and industry assert that user fee pilot programs have been successful in expediting product approvals

The multidisciplinary approach and interactive product reviews under two user fee pilots conducted authorized by the Prescription Drug User Fee Act has benefitted industry, fostering more efficient product reviews and cost savings compared to traditional review programs, according to a panel of US Food and Drug Administration officials and pharmaceutical industry officials speaking at an 8 June workshop sponsored by the Duke-Margolis Center for Health Policy and FDA.

Panelists also discussed how the experiences of past user fee programs authorized under PDUFA such as model-informed drug development (MIDD) and Complex Innovative Trial Designs (CID) can be leveraged to support the recently launched rare-disease development (RDEA) program.

Rajanikanth Madabushi, associate director of the Office of Clinical Pharmacology at the Center for Drug Evaluation and Research (CDER), discussed learnings from the MIDD shared pilot program. “Clearly there was an appetite for this program right from the beginning and it kept increasing,” he said, adding that through the program, “we were able to provide lots of insights on developing these models and their applications.”

The program was launched in 2018 under the Prescription Drug User Fee Act (PDUFA VI). (RELATED: Model-Informed Drug Development: Detials on FDA Pilot Project, Regulatory Focus, 16 April 2018).

The goal is to have early meeting discussions and to impart advice on how proposed MIDD approaches can be used in a specific drug development program. FDA committed to accepting two to four meeting requests quarterly each fiscal year.

Madabushi said that since the program’s inception, the agency has received 66 meeting requests and has granted most of them. Requests have been granted for drugs in therapeutic areas including oncology, cardiology, dermatology, and infectious diseases.

Some approved products that have been modified through these early meetings include a shortened time for ramucirumab in patients with gastrointestinal cancer from 60 minutes to 20 minutes and a new dosing strategy for sotalol hydrocholoride injection that reduces hospital stays from 3 days to 1.

Industry demonstrated its support for the pilot in a paper published last year in Clinical Pharmacology Therapy. The paper found that companies that participated in the pilot were able to save between $30 million and $70 million in development costs and were able to get products to market faster.

Madabushi said it is important to understand that there is substantial work involved in conducting multidisciplinary reviews.

“You cannot underestimate the amount of time and resources that will be required…. bringing multidisciplinary stakeholders and to have the shared understanding, it will not happen immediately, it takes time, and it takes different avenues of engagements.”

Learnings from the CID program

Dionne Price, deputy director of the Office of Biostatistics in the Office of Translational Sciences in CDER said that FDA Complex Innovative Trials Designs (CID) program also could not have been successful “without the participation of all the disciplines that were needed for the submissions.” She added that “similar to the RDEA program, the multidisciplinary approach will be similar.”

The CID pilot was launched in 2018, and fulfilled commitments under the sixth iteration of the Prescription Drug User Fee Act (RELATED: FDA Launches Pilot Program in Support of Complex Trial Designs for Drugs and Biologics, Regulatory Focus, 29 August 2018).

CIDs include trials designs that have rarely or never been used to demonstrate evidence of effectiveness in new drug applications (NDAs) or biologics license applications (BLAs).

CIDs can include complex adaptive designs, formal incorporation of prior information, and master protocols.

Eli Lilly participated in the CID program in 2019 and 2020. For the program, Lilly submitted a master protocol for chronic pain; the master protocol is now an ongoing phase 2 study that evaluates multiple investigational agents as they come in.

Eli Lilly’s Susan Warner said the company benefitted from having different FDA experts review the protocol, such as the statistical experts and the review division. “This was very beneficial to us before we had the protocol finalized. Also, we were doing something very novel. Lilly had not really implemented the master protocol in pain with different investigational agents being run at the same time, there is added operational complexity when you do something new.” She added that “having FDA come alone for the ride” was helpful.

She added that there is a "huge unmet need" for new pain treatments for patients suffering from chronic pain.

Lat year, FDA shared the results of some successful case studies showing how sponsors can incorporate innovative study’s designs of their clinical trials using the CID pilot last year (RELATED: FDA offers examples of innovative study designs accepted into CID pilot, Regulatory Focus, 20 January 2022).

Duke-FDA meeting

FDA and industry assert that user fee pilot programs have been successful in expediting product approvals

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