March 09, 2018
By A. Rahman Ford, Columnist

The Food and Drug Administration is finally getting the message.

In a special report published in The New England Journal of Medicine, the FDA makes a clear and positive shift in its stem cell policy – conceding that the old paradigm of drug approval just doesn’t work for stem cells.

The report, entitled “Balancing Safety and Innovation for Cell-Based Regenerative Medicine” is authored by FDA Commissioner Scott Gottlieb, MD, and Peter Marks, MD, Director of the FDA’s Center for Biologics Evaluation and Research.

Although short on specifics, Gottlieb and Marks declare their openness to creating alternative paths toward FDA approval of stem cell products – a policy change that could help stem cell therapies get to market faster and help patients sooner. This is a welcome move by the FDA.

The tone used by the authors signals that the FDA is listening to the voice of the people and stem cell developers. Gottlieb and Marks wrote the FDA must take “an original policy approach to the regulation of a highly innovative field, one in which [the FDA’s] traditional approach to regulation may not be as efficient or effective as in more mature fields.”

They maintain further that by working “within the existing regulatory framework” and by adopting “new principles,” the FDA’s premarket evaluation of stem-cell therapies will become more efficient.

It seems the agency could no longer ignore the fact that patients – such as those who suffer from chronic pain – cannot wait for the rusty gears of the antiquated clinical trial process to churn out the treatments they need to save their lives.

FDA Breaks with Past

The tone set by Marks and Gottlieb differs significantly from that of Gottlieb’s predecessor, Robert Caliiff, MD, who co-authored a NEJM article last year entitled “Clarifying Stem Cell Therapy’s Benefits and Risks.” As I’ve previously discussed, the arguments made by Califf were seriously problematic, specifically with regard to autologous therapies, which use stem cells made from a patient’s own blood or body tissue

Although Califf and his co-authors acknowledged the “unique challenges of stem cell clinical research,” their overall posture was decisively rigid in regard to new approaches to FDA approval. As an indication of just how low of a priority outreach was to them, they made no mention of working with stem cell investigators and sponsors until the final sentence of the article. For the previous regime, outreach was an afterthought.

Marks and Gottlieb seem to be taking a more conciliatory approach by extending a regulatory olive branch to stem cell physicians and small clinics. Unlike previous FDA statements, they spent less time on the spurious issue of safety and instead pivot toward the effectiveness of treatment and moving forward with commercialization. In doing so, the they acknowledge that the novelty of stem cell technologies require a more flexible path toward approval.

To accommodate this move and to facilitate the expedited availability of stem cells to patients, the FDA will use the expanded authorities granted it by the 21st Century Cures Act. The Cures Act allows the FDA to use non-traditional types of data – such as clinical data – to receive FDA approval. Notably, the FDA will be incorporating some “new concepts for how small investigators and firms can seek and meet the approval standard for products through efficient expedited pathways.” This is a step in the right direction.

How exactly will this work in practice? No one really knows. Marks and Gottlieb only provide one theoretical example: the FDA will provide “tools” to allow small firms to work collaboratively to obtain a biologics license for physicians, researchers and clinics.

Any outreach by the FDA should be welcomed and any attempt to expedite the availability of stem cell therapies to patients who need them should be encouraged. However, given the dearth of detail offered by Gottlieb and Marks, precisely how these alternatives will work in practice remains nebulous. Thus, the overtures made by the FDA are – at this point -- best met with skepticism and a cautious optimism

If the FDA is truly open to novel approaches to stem cell regulation, it should devise separate rules for autologous therapies – which former Commissioner Califf acknowledged “raise fewer safety concerns than allogenic cells.” Regulation of autologous therapies by the FDA should be minimal, with the majority of oversight left to state governments and their agencies.

These new policy changes by the FDA are forward-thinking and should proceed further. However, as promising as those options appear, they should in no way be construed as delegitimizing or nullifying legislative advances made in Texas or those that will be made when Congress enacts “Right to Try” legislation.

All of these options can function cooperatively to ensure that the patients – often the most overlooked quantity in the medical policy equation – can receive the life-saving and curative treatments they need as soon as possible.

The FDA may offer many paths, but it need not be the only path.

A. Rahman Ford, PhD, is a lawyer and research professional. He is a graduate of Rutgers University and the Howard University School of Law, where he served as Editor-in-Chief of the Howard Law Journal. He earned his PhD at the University of Pennsylvania.

Rahman lives with chronic inflammation in his digestive tract and is unable to eat solid food. He has received stem cell treatment in China.