The Bargain For Patients in “Right To Try” Unapproved Drugs
By Paul Pender MD
Published on Linked-In May 23, 2018
The US House recently passed a measure that cleared the Senate allowing patients access to potentially promising drugs to treat life-threatening conditions. Yet the American Cancer Society Action Network opposes the bill. Why would there be resistance to offering patients the chance to use investigational drugs for conditions in which approved drugs either don’t work or don’t extend life? The answer is complicated, but it involves the concept of risk.
Supporters of the bill believe that patients who have a life-threatening disease should not have to wait for the traditional FDA drug approval process, which can take years. Even in situations where compassionate use provisions are allowed, the timetable works against the patient who may have already tried traditional treatments. The time lapse between FDA Phase II of an investigational drug or device and the Pre-Market Approval for distribution can take years, time that terminal patients don’t have. Opponents of the proposed law worry about charlatans pushing snake oil on unsuspecting patients, or the chance that useful, proven therapies will be bypassed. Really? If you have a terminal condition, wouldn’t you be informed of your options and wouldn’t you have already exhausted traditional treatment? Besides, shouldn’t you have the choice, rather than have Big Brother tell you that you can’t try something that might help you?
The bargain for patients is that, for the right to try an unapproved drug, the drug company and the physician administering the drug should be held harmless. This requirement would protect those offering the treatment from legal repercussions and would put the risk of side effects squarely on the shoulders of the patient. Most FDA Phase II drugs have already passed safety requirements in Phase I and are awaiting efficacy and dosing studies in Phase III. For terminal patients, signing such a waiver to hold harmless drug companies and physicians is a risk they should be willing to take. After all, if you are dying, what do you have to lose?