Federal “right-to-try” legislation will restrict the US Food and Drug Administration’s (FDA) oversight of access to investigational drugs for patients with life-threatening illnesses and diminish the FDA’s public health mission, according to an article published in The New England Journal of Medicine.
Steven Joffe, MD, MPH, and Holly Fernandez Lynch, JD, MBE, of the Perelman School of Medicine, and the Leonard Davis Institute of Health Economics, University of Pennsylvania in Philadelphia, argue that the legislation will restrict the FDA’s oversight and allow unfettered patient autonomy, which could create the very problems that prompted Congress to grant the FDA its present authority.
Recently, the Senate passed the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act, which enables a patient with a life-threatening disease to access an unapproved drug after phase 1 testing, as long as a physician certifies that the patient has exhausted approved options and is unable to participate in a clinical trial of the drug in question.
A second provision of the bill prevents the FDA from using data on negative clinical outcomes from patients who obtain early access to a drug to “delay or adversely affect the review or approval of such drug,” unless those outcomes are critical to determining the safety of the drug.
A third provision protects manufacturers of investigational drugs, as well as prescribers and dispensers, from liability unless they engage in “reckless or willful misconduct, gross negligence, or an intentional tort.”
Critics of the proposed new law are concerned that frail, desperate patients, especially patients with advanced disease, may be at increased risk for worsened quality of life or accelerated death. Furthermore, if the requirement that patients must be unable to participate in a clinical trial is not strictly enforced, data needed to make evidence-based decisions about approval may be delayed.
While the investigators noted that these concerns may be overstated, the legislation under consideration reflects a broader effort to weaken medical product regulation. They also noted that expanded access may be extended to patients with serious chronic illness in the future and perhaps eventually to all patients who are not satisfied with their current treatment options.
Joffe S, Fernandez Lynch H. Federal Right-to-Try legislation—threatening the FDA’s public health mission [published online January 10, 2018]. N Engl J Med. doi:10.1056/NEJMp1714054