Will Right-To-Try Morph into Right-To-Profit for Stem Cell Clinics?

FDA RMAT
Screenshot from FDA Voice page on RMAT going live

On one level a reasonable argument can be made for letting terminally ill patients have a right to try experimental as yet unproven therapies, but in the real world Right-To-Try laws have many downsides. On the whole, they are likely to be negative for patients as a group.  Raising the stakes is a push for a federal Right-To-Try law in the U.S. A bill passed the U.S. Senate so a law is a real possibility in coming months. What are the problems and risks?

One of the main red flags with Right-To-Try in the stem cell arena is that commercial businesses selling unproven stem cell therapies are some of its biggest proponents. It’s no coincidence that these stem cell clinics are also some of the biggest critics of the FDA and lobby against careful oversight of stem cells. Clinics view Right-To-Try as a potential boost to their financial bottom line. For these reasons, I’ve argued that from a clinic perspective such laws can be seen as “Right-To-Profit”. The sad irony here is that some of the clinics say their main goal and the reason they support Right-To-Try is just to help patients, but I’ve never seen a clinic willing to provide stem cells to patients “at cost” without profit.

In fact, in Texas the big legislative battle in 2017 over stem cells raised concerns over proposed language that specifically would give a seal of approval to clinics charging patients large sums for unproven stem cells. More broadly across the globe, Right-To-Try type of laws can encourage predatory behavior by businesses that siphon money off patients for risky “shot-in-the-dark” therapies. In theory one way this issue could be resolved is prohibiting charges for therapies administered under the auspices of Right-To-Try, but the fact that many Right-To-Try fans don’t like this idea of uncoupling it from profits is a clear signal that for some folks Right-To-Try is not all about patient rights. It’s about money.

FDA RMAT
Screenshot from FDA Voice page on RMAT going live

Another problem with Right-To-Try is that some proponents including businesses are not satisfied with its limitation to terminally ill patients. They want to expand Right-To-Try to include a host of other less severe health problems. The less severe their illness the more patients stand to lose by trying experimental offerings that have little data behind them. However, the more loosely eligibility for Right-To-Try is defined, the larger the potential customer base for clinics.

It’s not even clear that Right-To-Try is needed. The current FDA Expanded Access (a.k.a “Compassionate Use”) program is very responsive to terminal patient needs and requests. Approval of expanded access requests is common, while rejection is rare at as low as only a few percent of requests. Some complain of what they see as onerous paperwork for doctors purportedly leading some to not even bother to make Compassionate Use requests, but FDA has streamlined the process dramatically in recent years. Further weakening the argument for a national Right-To-Try law in the U.S. is the fact that the state Right-To-Try laws haven’t led to a stream of documented success stories for American patients by taking a short cut around the FDA.

The new FDA program of Regenerative Medicine Advanced Therapy (RMAT) designation, whereby promising investigational stem cell-based drugs can be advanced more quickly through the approval evaluation and approval process, has started with a bang with the FDA seeming to fully embrace the spirit of the 21st Century Cures Act that established RMATs and already approving 4 RMATs in just a few months. This makes Right-To-Try on the stem cell front seem less necessary as well, particularly if Right-To-Try gets expanded beyond terminally ill patients.

In the end, is Right-To-Try sometimes more about profits than patients’ rights from the business perspective? This isn’t always the case and it should be noted that many patients support Right-To-Try, but in practical terms it is likely to achieve very little on the positive side while risking a great deal. Our energies as patient advocates would be better spent on working to further improve the FDA Compassionate Use program, see how the RMAT program develops, and work on stem cell and regenerative medicine standards.

This post is just one of many covering Right-To-Try as part of Signal’s second annual blog carnival about the theme Right to Try. Click here to read what other bloggers think.

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