While waiting for the Senate to schedule a vote on his nomination to be the next FDA commissioner, Robert Califf is busy articulating what his priorities will be once he’s confirmed.

Industry experts are also debating what he will focus on, with some expecting that he will make reforming clinical trials his top priority.

Califf last week provided two senators a deeper look into the agenda he would set at the helm of the agency, including using real-world data (RWD) and real-world evidence (RWE) in trials, how trials should be conducted for software as a medical device (SaMD) and innovative trial designs.

In letters to Sens. Mike Braun (R-Ind.) and Robert Marshall (R-Kan.), Califf expressed interest in developing an improved system for generating postmarket trial data and pointed to several RWD/RWE initiatives at the agency that have already produced positive results, including a March 2021 report detailing 90 instances of RWE being used to inform regulatory decisions for a range of medical devices. He also noted the four draft guidances the agency recently issued on using RWE in trials.

Califf also intends to ensure that the agency keeps pace with the advances being seen in the digital health technology and SaMD spaces, particularly how trials can be done effectively for them and noting the importance of the FDA’s Digital Health Center of Excellence in these efforts and his eagerness to further them.

And speaking during an FDAnews webinar last week, panelist Nancy Myers, CEO of Catalyst Healthcare Consulting and moderator/panelist Wayne Pines, president of healthcare at APCO Worldwide, agreed that Califf will push for a greater use of RWE and the expansion of clinical trial size.

While the pair believes that he would immediately focus on clinical trial reform should he get confirmed, Myers also noted the challenges he would be up against as commissioner that he would have to navigate.

In particular, the new agency head will have to deal with increased scrutiny of the FDA drug approval process while contending with employee burnout after two years of relentless work during the public health emergency.

She referenced controversial agency decisions, such as the approval of Biogen’s Alzheimer’s disease therapy Aduhelm (aducanumab) in June 2021 and Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen) in 2016, which occurred under Califf’s first stint as FDA commissioner from 2016 to 2017.

Another presenter, David Fox, partner at law firm Hogan Lovells, also touched on the perception that FDA approvals are not always based on adequate data.

“When you issue 50 approvals a year, how do you walk a straight line?” Fox asked, noting the challenges of having a one-size-fits-all approach to approving dozens of new drugs. He said the agency “has to become better” at articulating what type of data are actually used to support approvals in order to build public confidence.

Sens. Braun and Marshall were among a bipartisan group of eight lawmakers who did not vote for Califf during a Senate committee’s consideration of his nomination.

Read the responses to Braun here: https://bit.ly/3FNk7sY.

Read the responses to Marshall here: https://bit.ly/3IltqCj.