Califf Testifies on Accelerated Approvals
FDA commissioner Robert Califf told a Senate appropriations hearing 4/19 that FDA and CMS are working to make sure clinical trials for products that are seeking accelerated approval are designed to address FDA approval requirements as well as include data to support a CMS ”reasonable and necessary” determination for reimbursement purposes. Califf was asked to address a concern raised by Agriculture Subcommittee chair Martin Heinrich (D-NM) over accelerated approval drugs gaining FDA approval but not clearing the subsequent hurdles to obtain a CMS coverage determination.
The most likely public instance of this that Heinrich was probably referring to was the 2022 CMS National Coverage Determination (NCD) on Medicare coverage that limited payments to only FDA traditionally approved monoclonal antibodies that target amyloid (or plaque) for treating Alzheimer’s disease, a significant blow to Biogen’s controversial accelerated approval of Aduhelm (aducanumab). Additionally, CMS said that as part of its NCD it would provide “enhanced access and coverage” for people with Medicare who participated in CMS-approved studies, such as a data collection through routine clinical practice or registries.
Califf compared the dilemma to a familiar sports analogy, saying the previous process was equivalent to a relay race “where we run the first lap and then we hand off a baton to the payers, private payers and CMS. What we’re doing now is trying to make sure the information is much more seamless so that we have clinical trials that are relevant to what they need too.”
Califf also said that it’s “abundantly critical that CMS does not influence our decisions about safety and effectiveness and we don't have any authority to influence CMS decisions about the reasonable and necessary criterion. What we want to do is to make sure people have the right information so that CMS can make the best decisions it can so that it understands what we were looking at when we made our decisions. I think this is an area in American medicine where there's a lot more work to be done.”
Additionally, the hearing heard from Appropriations Committee vice chair Susan Collins (R-ME), who raised concerns about FDA placing too many clinical holds without explanation on some promising cell therapy development programs, and she asked Califf whether there is a problem with these product reviews. Califf responded by acknowledging that there are “some issues there” that need to be worked out.
“This was recognized in the recent user fee agreement,” Califf continued. “We're going to be hiring 150 to 200 people in this area, under the leadership of Dr. Marks who I think you'd agree has done a remarkable job with vaccines and now this is going to be a big focus of attention. I would emphasize when it comes to clinical holds, it’s not just that something is going wrong with a patient. Often it has to do with the integration of manufacturing and a lot of the companies are startups without robust manufacturing facilities. There's a lot of work to go on, but we agree that this is an area we’ve got to move along more quickly.”