Industry officials report mixed experiences with recent FDA pilot programs

San Diego – Pharmaceutical industry officials generally gave high marks to the US Food and Drug Administration’s (FDA) Model-Informed Drug Development Paired Meeting Program (MIDD) and lower marks to the Advancing Real-World Evidence (RWE) pilot during a panel at the DIA 2024 Global Annual Meeting on 18 June.

On industry representative called the MIDD program a “runaway success,” yet complained that FDA has not translated the learnings from these pilots into its standard review practice.

Danielle Friend, senior director and US head of regulatory policy at Johnson & Johnson, reported the results of a survey conducted by Greenleaf Health to gauge industry’s views on four pilot programs agreed to under various iterations of the Prescription Drug User Fee Act (PDUFA).

The MIDD, RWE and the Rare Disease Endpoint Advancement (RDEA) pilots were agreed to as a performance goal under PDUFA VII. The Complex Innovative Trials Desing meeting Program (CID) was esbalhed under the PDUFA VI commitment letter.

The panel participants included Donna Boyce, head and senior vice prenent of global regulatory sciences for Pfizer, Rasika Kalamegham, head of the US regulatory policy for Genentech, Mary Jo Salerno, a science policy analyst with FDA’s Office of New Drugs (OND) in the Center for Drug Evaluation and Research (CDER), and Yueqin Zhao, a lead mathematical statistician for the Office of Bioequivalence (OB) in CDER.

Survey results

Friend said the survey polled 13 companies, including small-, medium-, and large-size firms, to gauge their experiences with the pilots. Of the 13 companies, only five participated in one of the pilot programs. Four of the five participated in both the MDD and the CID pilot programs.

The survey showed most companies that participated in a pilot program had a positive experience, said Friend. The MIDD program garnered the most positive comments, followed by CID as the next most popular program.

Friend noted that both CID and MIDD are not pilots anymore but are permanent programs at the agency.

Pilots are a lot of work

Boyce and Kalamegham reported their experiences with the pilots and said they generally mirrored the results of the survey.

Boyce said that “overall we are immensely supportive of the pilots, and I will continue to push my team to participate. The interactions and the learnings that you get on each specific product and the ability to apply and share those learning is really helpful.”

She added a caveat that participation in pilot programs is “a lot of work.”

Boyce said that the RWE program was more challenging than they anticipated. “The RWE [pilot] we have been working on quite a bit. It is not as easy as perhaps we thought it could be.” She said that regulators are tending to be conservative in this pilot and are “falling back on the need for [randomized controlled trials] RCTs.”

Kalamegham said that Genentech’s experiences with the pilots generally mirror those of Pfizer. “I just want to say ditto that everything that Donna just said. I will say that our experience is that when we took the survey internally you can take the number and the graphs and that is exactly what we saw. MIDD from our experience has been a runaway success.’

However, one disappointing aspect of these pilots is that they have not yet been incorporated into FDA’s standard review process, Kalamegham said.

“I think there is a little bit of a gap between what the industry wanted and what the commitment letter said and what the FDA agreed to. The aspirational ls KPIs [key performance indicators] that we all wanted on the industry side was for the pilots to be sustained practice and I think that is the gap that we need to address now.”

“I am very glad to hear that the feedback is positive” on the MIDD and the CID pilots, Zhao said. Referring to the RDEA pilot, both Zhao and Salerno noted that rare disease drug development is especially challenging.

While not necessarily addressing the pilot, Salerno she said that rare diseases often have a “poorly understood natural history” and that these are serious and life-threatening diseases where 95% of the population does not have access to treatment. In addition, she said that most rare diseases occur in pediatric population or small population which makes designing clinical trials “very difficult.” Underlying all of this is the “urgent need for results.”

Of the 10 applications that have been submitted into the RDEA program, two have been accepted.

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Industry officials report mixed experiences with recent FDA pilot programs

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