√ Senate Passes Federal Right-To-Try: Poll On Your Opinion

The U.S. Senate just passed a bill that if it becomes law would codify Right-To-Try as a federal law, meaning terminally patients across the U.S. could ask their physicians and drug manufacturers to be given still investigational therapies such as still unproven stem cell therapies in development. The bill’s official name is

“The Trickett Wendler Frank Mongiello Jordan McLinn,and Matthew Bellina Right to Try Act of 2017.” Its goal is summarized this way, “To authorize the use of unapproved medical products by patients diagnosed with a terminal illness in accordance with State law, and for other purposes.”

It has 7 key bullet points according to the Senate:

It is the sense of the Senate that section 561B of the Federal Food, Drug, and Cosmetic Act, as added by section 2—

(1) does not establish a new entitlement or modify an existing entitlement, or otherwise establish a positive right to any party or individual;

(2) does not establish any new mandates, directives, or additional regulations;

(3) only expands the scope of individual liberty and agency among patients, in limited circumstances;

(4) is consistent with, and will act as an alternative pathway alongside, existing expanded access policies of the Food and Drug Administration;

(5) will not, and cannot, create a cure or effective therapy where none exists;

(6) recognizes that the eligible terminally ill patient population often consists of those patients with the highest risk of mortality, and use of experimental treatments under the criteria and procedure described in such section 561A involves an informed assumption of risk; and

(7) establishes national standards and rules by which investigational drugs may be provided to terminally ill patients.

Proponents say it makes good sense and is ethical that such extremely ill patients should be given the right to try a wide range of options including unproven therapies, while opponents say it is too risky and that the FDA’s current compassionate use/expanded access agenda already works for such patients. It is likely that this bill will become law soon.

For two strong opinions either way on Right-To-Try see this debate from this week involving Beth Roxland of NYU and Christina Sandefur of the Goldwater Institute.

What do you think of the idea of a federal Right-To-Try law? Take our poll above.

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√ Will Right-To-Try Morph Into Right-To-Profit For Stem Cell Clinics?

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.

Screenshot from FDA Voice page on RMAT going live

Another masalah 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”) kegiatan 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 kegiatan 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 21^st 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 kegiatan 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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