Regenerative medicine technologies, including CAR-T cell and stem cell therapies, are highly promising medical advancements but are being held back by the lack of widely accepted standards and guidelines to govern their use, development and manufacture, finds a report by the U.S. Government Accountability Office released Thursday.
Unlike typical therapies that treat symptoms, regenerative medicines restore or repair the function of cells, tissues and organs afflicted by diseases. This approach enables highly personalized treatment regimens and may eventually lead to cures for conditions that are currently considered terminal, chronic or untreatable.
However, regenerative medicines also present significant standardization, regulation and manufacturing challenges that may affect the development and use of these technologies, according to the GAO.
The watchdog group found that developing standards is challenging “because these technologies are complex and rapidly evolving” and due to “the need to reach consensus across a range of stakeholders and the need for accurate, well-developed measurement science in the field.”
Thursday’s report noted that, unlike regulations, standards can be voluntary and are not typically developed by government agencies. As a result, “broad buy-in is important for them to be accepted and used” but “even if stakeholders agree that a particular standard should exist, it can be difficult to reach agreement on the details.”
When it comes to the regulatory environment, the GAO found it can be challenging for companies to “navigate the complex regulatory framework for regenerative medicine products, which may span multiple FDA centers and pathways to approval.” What’s needed, the report said, are “clear and predictable regulations” to “ensure that product developers are able to understand the data and other requirements needed for approval without unnecessary delays or uncertainty.”
Specifically, the GAO’s report found that current regulatory pathways may be insufficient for regenerative medicine technologies and therapies that “blur the lines” between drugs, biologics, and devices, making their paths to approval or licensure more uncertain.
In the area of manufacturing, the GAO cited three challenges related to the widespread and efficient manufacture of regenerative medicines: the lack of infrastructure, the difficulties in ensuring quality, and workforce shortages.
“The cell, tissue, and organ products being developed for regenerative medicine will require more complex manufacturing facilities than are currently used to produce small-molecule drugs,” according to the GAO. In addition, the report noted that “stakeholders often lack consensus on how to measure quality for regenerative medicine products” with “few standardized reference materials that can be used to evaluate a finished product.”
Workforce shortages of skilled technical personnel, who work on regenerative medicine manufacturing lines, are another major challenge. “As demand for regenerative medicine products grows, workforce needs will also continue to grow,” the report said, while increasing the need for technical workers in multiple locations and not just cities that already have a large biomedical workforce.
To address these barriers and help maximize the potential of regenerative medicine, the GAO report made several policy recommendations. They included increasing investment in the development of industry guidelines and facilitating greater interaction between regulatory experts and smaller biotech companies, particularly those that are still early in the development process.
On the manufacturing front, the GAO report proposed increasing oversight and providing better feedback to suppliers to improve their consistency, especially regarding starting materials. The watchdog group also recommended that federal agencies could help by supporting public-private partnerships that can share costs for manufacturing facilities.
The Alliance for Regenerative Medicine, the advocacy organization which represents the industry, has previously warned that manufacturing continues to be a major challenge for the sector due to a lack of standardization and that manufacturing scale-up has often become an obstacle to regulatory approval and commercialization.
“The GAO report illustrates the transformative potential of regenerative medicine, but also the challenges that stakeholders across government, industry, and academia must collaborate on to ensure these technologies benefit as many patients as possible,” Stephen Majors, global head of communications at the Alliance for Regenerative Medicine (ARM), said in an email to BioSpace.
Majors said that the report “reflects contributions from ARM experts on topics including standards development, workforce challenges in both industry and at the FDA, the need for clear regulatory guidance, and the importance of increased interactions among regulatory experts, especially early in the development process.”
