When you’re out of options, you’ll need Right to Try
A recent column in The Hill made several points in opposition to S. 2912 and the broader Right to Try concept that are either the result of misinformation or an incomplete understanding of how the laws will actually work. I am writing to correct these (presumably unintentional) misrepresentations.
S. 2912 and the underlying state laws that it helps enable pharmaceutical and biotech companies to provide medicines still in the FDA clinical trial process to doctors for their terminally ill patients.
In his column, Aaron Shapiro opposes Right to Try because it does not allow all patients to try all medicines and it does not mandate that companies provide investigational drugs to patients. By themselves these arguments seem silly; we don’t say laws against crimes are useless because they don’t stop all crime?
Right to Try laws aren’t meant to provide access to all drugs for all patients. The laws aim to help the very sickest patients by giving them access to medicines that have successfully navigated an FDA safety trial, and are still in the FDA ongoing trial and review process. The point of the law is to help people who have run out of options. Patients who haven’t yet run out of options, still have potential avenues to be helped.
Right to Try laws provide a vehicle for companies that want to participate, to be able to participate. Many will decide that it doesn’t make sense for them, and many will decide that it does. Wherever that line is drawn, it will give hope to many patients and their families who would not otherwise have it; and for many add a great deal of meaning to their lives as they choose to be active participants in the fight against their illness.
Shapiro further argues Right to Try “does little to help terminally ill patients” because the FDA has a process permitting early access and the agency approves 99% of requests. The FDA’s various early access and compassionate use vehicles are very useful tools; but they are also very limited tools. The agency can handle about 1,000 cases a year, which is roughly where they are now. They are not equipped to handle a demand even 100 times greater, never mind the actual demand.
The thorniest problem with Right to Try is dealing with adverse events. The FDA cannot “ignore” adverse safety data brought to its attention as it is considering the safety and efficacy of a drug. Like provisions in all laws, the provision protecting drug developers who participate by providing patients with investigational drugs will need fleshing out in the real world. There are ways to deal with this issue that neither increase safety risks to patients, nor slow down the traditional trial-enabled approval of drugs.
No one, not patients or drug developers, is asking the FDA to ignore scientific data; but the FDA is not the arbiter of how that information is used. Congress charges the FDA with its responsibilities. If the agency was charged with working out a new system for analyzing this data, they will. Just because something is nuanced and difficult does not mean that it cannot be done; and certainly does not mean that it should not be done.
Finally, Shapiro makes the argument that if public servants want to “really” help patients, they should focus on trying to make major policy changes to the overall health care system that would help these patients. Really? Perhaps he has not been watching the same gridlocked Congress that we all have been watching for the past six years.
Put aside for a moment the fact that Right to Try applies only to terminally ill patients and the time constraints that implies. There is nothing that keeps Congress from doing BOTH Right to Try in the short term and a bigger fix in the long term.
As President & CEO of a biopharmaceutical company developing treatments for currently incurable diseases; as a member of the advisory board that helped craft the model Right to Try act; and as the father of a son diagnosed with a brain tumor at age 4, I have been involved in the scientific, FDA regulatory, business, legislative and patient advocacy arenas for over two decades. Speaking from experience, I can assure you that S. 2912 is a good bill that will provide hope and comfort to many patients diagnosed with fatal diseases; and it will accelerate the effort to find cures for currently incurable diseases.
Mr. I. Richard Garr, JD, served as Chief Executive Officer and President of Neuralstem Inc. from 1996 to February 15, 2016. Neuralstem is developing a stem cell therapy to treat ALS or Lou Gehrig’s Disease.