Steven Joffe, MD, MPH, Emanuel and Robert Hart Professor, Medical Ethics and Health Policy; Holly Fernandez Lynch, JD, MBE, John Russell Dickson, MD Presidential Assistant Professor, Medical Ethics and Health Policy, University of Pennsylvania

In August 2017, the US Senate passed The Right to Try Act, which would limit the Food and Drug Administration’s (FDA’s) oversight of the use of unapproved drugs in life-threatening situations. It now awaits a vote in the US House of Representatives. In a New England Journal of Medicine Perspective, LDI Senior Fellows Steven Joffe and Holly Fernandez Lynch argue that the benefits of the proposal are more symbolic than real, while the potential long-term harms to the FDA’s public health mission are great.

This bill enters a long history of debate about the role of the FDA in assuring the safety and efficacy of drugs before they are available on the market. Due to public demand to make investigational drugs available outside of clinical trials for patients in life-threatening situations, the FDA has already created pathways to approve patient requests for early use, called expanded-access.

The Right to Try Act would change this expanded-access through three major provisions. First, it would allow expanded-access solely with a physician’s statement that the patient has exhausted all other approved treatment options and is not a candidate for the drug’s clinical trial. Previously, FDA approval was required as well. Second, it would prohibit the FDA from using clinical outcomes of the patients who get early access to these drugs to adversely affect the drug’s evaluation, a concern of drug manufacturers, unless the agency believed that the data were “critical to determining the safety of the eligible investigational drug.” Finally, it would prohibit patients and families from taking legal action against drug manufacturers for any negative outcomes due to expanded-access use, and would prohibit legal action against providers or dispensers  unless there was “reckless or willful misconduct, gross negligence, or an intentional tort.”

Joffe and Lynch argue that the Act would not actually increase patients’ ability to obtain unapproved drugs because it does not target obstacles to early access. A study of all expanded-access requests between 2010-2014 found that the FDA gave its “stamp of approval” 98% of the time. The same study found no evidence that expanded access had adversely affected the drug’s likelihood of FDA marketing approval, nor could the authors find any product liability cases arising from the use of drugs through expanded access.

Given that the bill does not add much, it’s important to look at what it takes away. The first provision that removes FDA sign-off would mean the end of required safety modifications that the FDA attaches to its approval in 10% of cases. Further, passage would be a symbolic advancement in a larger movement towards deregulation of medical products. While the “right to try” might seem like a boon for desperate patients, Joffe and Lynch point out that the lack of an FDA safety check may actually lead to more harm to patients taking unsafe drugs. They warn of the dangers of continuing down this path:

Are we prepared to abandon the FDA’s gatekeeping role in favor of unfettered patient autonomy and market forces, risking precisely the problems that prompted Congress to grant the FDA its present authority? The agency has made substantial progress in balancing the needs of desperate patients and the principle that all patients deserve evidence that the benefits of medical products justify their risks. We upset that balance, and diminish the FDA’s public health mission, at our peril.

Read their full analysis here.

Katharine (Kara) Freeman is a medical student at the University of Pennsylvania.