The EveryLife Foundation for Rare Diseases hosted a panel discussion on Right to Try Proposals, as part of their 2018 Legislative Program for Rare Disease Week on Capitol Hill. What makes this video worth watching is the first hand accounts and the reasoning of patients who have a major stake in it: their lives and the lives of those whom they love.
EveryLife even had a rare, stage IV cancer patient participate who gave his precious time to try to explain why he felt such legislation was potentially not only unhelpful, but might actually hurt the very people it was written to help:
“I’m the ideal person to support right to try. But I can’t — it’s a disaster in the making” -Michael D. Becker Author of “A Walk with Purpose: Memoir of a Bioentrepreneur.” and stage IV Oropharyngeal Cancer patient.
The University of Michigan, Michigan Health Lab comes down also on side of no new legislation, stating that the existing procedures both already give patients a “right to try” and also protects them from the unscrupulous.
Currently, a physician may ask the FDA and get a timely response on the use of an experimental drug, called “Single Patient Access,” and at times some additional guidance about the drug in terms of administration, and side effects. More importantly, patients do not have to have exhausted all conventional treatments to be eligible. The requesting physician must only believe that the unproven medication might be beneficial for their single patient.
Many of the new versions of legislation have a “need to exhaust” conventional therapies clause, as a requirement which might instead exhaust and weaken a fragile patient as well as delay the administration of the experimental medicine.
But Right to Try is more complicated than this. In the 90’s “compassionate use” and “expanded access” became possibilities for those patients who did not qualify for clinical trials. However, there are not always sufficient supplies of experimental drugs, and manufacturers are not always keen about granting access: an adverse outcome in a patient or a lack of efficacy may hinder the approval process for others, and many doctors lack the knowledge, experience and time to administer and monitor a medication with which they are totally unfamiliar.
So in real life, compassionate use does not always happen. On the other hand, single patient access has been approved in emergency situations in less than 24 hours. Is this widely available? No. You need a promising drug, a patient who is not too far gone, a first class physician aware of promising new therapies, and a willingness to take the risks involved, including insurance coverage risks of adverse effects of an experimental drug, and the action needed.
The FDA has made great strides to bring more drugs to market faster by streamlining the approval process and designating Orphan Drug status for promising new therapies. NCAT’s is also involved in using “tissue chips” to identify promising therapies without the need to use humans to do it. For the majority of patients this remains the safest and surest route.
