January 20, 2021

The Niche

Trusted stem cell blog & resources

Congress pushes FDA on stem cell clinic mess in new letter

It can be kind of a big deal when politicians, especially at the federal level, write the FDA about stem cells.Congress letter to FDA stem cell clinics 2019

Is this going to be good news or bad? Some combination?

I tend to worry that some senators or representatives might pressure the FDA to weaken oversight. However, lately it seems like the opposite is happening. Perhaps pressure the other direction, for less oversight, doesn’t see the light of day.

Last April Senator Grassley wrote the FDA about problematic stem cell clinics and related issues such as the problems at Clinicaltrials.gov.

My sense was that he wanted more action and quickly.

Maybe it was coincidence, but only weeks after that the FDA moved forward via the DOJ on lawsuits in federal district court seeking permanent injunctions on some firms.

Perhaps Grassley was an ally behind the scenes to move things forward at DOJ? Instead, a skeptic might say that he knew the FDA suits were coming and when they’d be announced, so he sent his letter before that to get some credit? Is it bad to think that?

Now in late July 2019 the U.S. Congress Committee on Energy and Commerce has sent a stern letter to new FDA Commissioner Ned Sharpless basically asking why more hasn’t been done about the stem cell clinic problem. It’s an intriguing letter to read and I largely agree with its sentiments. David Roza over at Inside Health Policy broke the story on the new letter (paywall).

The letter is signed by U.S. Reps Frank Pallone, Jr., Diana DeGette, Greg Walden, and Fred Upton.

Two Democrats and two Republicans. Nice.

Interestingly, the Committee seems to suggest that if the FDA needs more resources to tackle this serious problem, it might get them if it asks. I wonder — has it asked in the past or just struggled with too few resources?

The letter asks six questions that are quoted below (to the best of OCR text recognition via Acrobat):

  1. “How is the agency’s current work on regenerative medicine therapies, including stem cells resourced? Approximately how many requests has FDA received for INTERACT (Initial Targeted Engagement for Regulatory Advice on CBER products) meetings prior to development of a stem cell therapy? How many of the potential sponsors that requested a meeting subsequently submitted an application? Of those applications that resulted from such an informational meeting, how many have requested an RMA T designation?
  2. An article in the New England Journal of Medicine authored by Drs. Marks and Gottlieb acknowledged the importance of providing tools that will encourage individuals or small groups of physicians to develop these products, such as a common manufacturing and clinical trial protocols. How many products is FDA aware of in the pipeline that are utilizing this approach to development? Have any such applications received RMAT designation? Have any applications using this approach received approval? Will the agency provide resources to explain how these tools could be best utilized by developers or to account for the number of products being developed under this approach?
  3. Given the current development environment for stem cell therapies, are there strategies other government entities should be considering in order to engage with legitimate researchers early on in their process and spur the use of the traditional development pathways? Is FDA considering other strategies to encourage developers to engage with the agency early on?
  4. How many agency officials are working to monitor adverse event reporting and respond to safety signals related to stem cell therapies? What level of staffing and funding has the FDA determined is needed to adequately monitor adverse event reporting and respond to safety signals related to stem cell therapies? Would additional resources help the agency to conduct a more rigorous survey of the market? Will additional resources be required when the period of enforcement discretion ends in November 2020?
  5. As you prepare for November 2020, and the planned end to FDA’s policy of enforcement discretion, how will you communicate the transition to enforcement to non-compliant stem cell clinics? How will you announce planned changes to enforcement policies?
  6. How is FDA coordinating with other state and federal agencies? Is the agency coordinating with state medical boards? If not, would the agency consider coordinating its enforcement work with state medical boards or, at the very least, providing its compliance letters to such medical boards, which could revoke the licenses of physicians that perform these procedures?”

I’d be curious of the answers to these questions too.

I also wonder if this letter might spark another round of additional high-level FDA action well before the 3-year “grace” period for compliance with FDA guidance expires? FDA has the discretion to take action during this period on stem cell clinics that it views as risky.