Bipartisan Energy and Commerce Committee leaders sent a letter to Food and Drug Administration (FDA) Acting Commissioner Norman E. “Ned" Sharpless, M.D., today raising concerns over the slower than expected progress of manufacturers and providers of stem cell products to come into compliance with FDA’s guidance and premarket approval requirements, including those established by the 21st Century Cures Act. The request comes after the FDA recently announced that it has sent letters to 20 manufacturers and providers who may be offering unapproved stem cell products.
The letter was signed by Chairman Frank Pallone, Jr. (D-NJ), Ranking Member Greg Walden (R-OR), Rep. Diana DeGette (D-CO), and Rep. Fred Upton (R-MI). Each of the four members that signed the letter to FDA played key roles in the passage of the 21st Century Cures Act, which was signed into law in December 2016.
In an effort to provide greater clarity around the regulatory requirements for regenerative therapies, including stem cell products, the 21st Century Cures Act, directed FDA to establish a regulatory program for the development and approval of regenerative therapies. The agency also released guidance in 2017 further clarifying when cell and tissue-based products would be subject to FDA regulation and provided a 36-month period of enforcement discretion. Despite these actions, manufacturers and providers have been slow to come into compliance ahead of the November 2020 deadline, while reports of adverse events and misleading advertising and marketing to patients continue to rise.
“With this added clarity from FDA about the premarket approval process and its quick work to clarify its expedited review program, it is difficult to understand why the industry has made only ‘modest progress’ coming into compliance during the first half of this three-year period of enforcement discretion," the four bipartisan Committee leaders wrote to Acting Commissioner Sharpless. “The slow pace of compliance is particularly concerning given the high stakes for patients. For example, many of these unproven therapies are being provided directly to patients outside of controlled clinical studies resulting in numerous serious adverse events, including infection, hospitalization, and permanent blindness."
Besides FDA’s letters to manufacturers and providers who may be offering unapproved stem cell products, two lawsuits have also been filed against clinics in California and Florida over the last year. These clinics treated patients with unapproved stem cell products that caused patients to go blind and resulted in the hospitalization of patients following the contraction of infections in their bloodstream or joints.
“We want to register our concerns about the proliferation of unproven stem cell therapies on the market and our desires to ensure that the agency is doing everything in its power to protect patients," the four Committee leaders continued in their letter. “We would like to better understand the FDA’s current strategy for monitoring the market and bringing these therapies into compliance."
As part of their inquiry, the Committee leaders are requesting answers to a series of questions by Aug. 15, 2019, including:
* How is the agency’s current work on regenerative medicine therapies, including stem cells, resourced?
* Is FDA considering other strategies to encourage developers to engage with the agency early on?
* How many agency officials are working to 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?
* 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 is FDA coordinating with other state and federal agencies?
