On May 30th 2018, the President signed the recently passed Right to Try (RTT) legislation into law. This new law allows patients to receive unapproved medications without FDA notification for up to one year. It also removes the FDA from prohibiting access to an experimental therapy and removes the FDA from advising on dosing, schedule, method of administration or other important safety measures. In short, you’re able to side step the FDA and go directly to the drug manufacturer. You’re basically on your own without the oversight of the FDA. So, Caveat Emptor (buyer beware) and proceed with caution.
Nevertheless, this opens up the door to new possibilities that were simply not available before.
Here’re some of the Pros and Cons as we see them.
PRO’s:
- Finally, some options. The RTT provides a level of freedom…and hope, that would otherwise be denied patients of terminal diseases.
- Some of the therapies may prove beneficial and/or point the investigators and drug companies in a better direction.
- It’s the right of every individual to choose their own destiny.
- Side stepping Governmental bureaucracy and perhaps accelerating therapies that show promise.
- Patients can try other drugs, supplements and therapies and report their results to their physicians and drug companies. Now information can flow in more directions.
- The ALS Association both welcomed and voiced reservations about RTT. The association is aware of and is tracking this new law and will communicate with the ALS community.
- Since the drugs are experimental and not FDA approved, this MIGHT lower treatment costs, as pharmaceutical companies cannot make a profit on these drugs until they’re FDA approved.
- Possible access to other treatments and drugs, available outside the US, that have shown promise in other countries.
CON’s:
- Some medications may not be safe, although they must have passed Phase 1 of clinical trials before patients of RTT can try the drugs.
- Patients must look out for “bad actors” stepping in now to take advantage of patients vulnerabilities. Check history and reliability of manufacturer.
- Pharmaceutical companies have very little incentive to provide access or broadly distribute their meds since they cannot make money until the drugs are FDA approved.
- The RTT law could weaken current patient protection laws enforced by the FDA.
- The reality is that many experimental drugs have limited access and availability anyway.
- The RTT law may work as a pressure release valve and may reduce pressure on the FDA to speed up approval of Phase 1, 2 and 3 drugs in the pipeline.
- Patient has little to no legal safeguards in case therapy produces more harm than good.
All in all, we think that the net effect of the new RTT law will be a positive one. This law opens up the conversation about the FDA’s unjustifiably slow and expensive process and provides the patient with some control over their destiny. That being said, patients need to be careful not to make a bad situation worse and should continue to consult with their physicians before venturing into unexplored territories.
Sources:
- ALS News Today: US “Right to Try” Law meets with mix of Praise and Criticism, Including Among Those with Rare Diseases.” June 4th, 2018 by Larry Luxner
- “Easier Access to Experimental Drugs” vitaeducation.org/wp-content/uploads/easier-access-to-experimental-drugs.pdf
- What You don’t Know about “Right to Try” Laws can Hurt You. https://medshow.org/fda-watch/right-to-try/
