HJNO Sep/Oct 2020

THE RIGHT TO TRY Can you please explain the difference between Right to Try, expanded access, and compassionate use? Dr. Marc Matrana Patients with life- threatening diseases often exhaust approved treatments, and many turn to clinical trials in order to access additional therapy options. When clinical trials are not an option (if patient is not eligible for example), patients still may have avenues to obtain experimental drugs. Expanded access, also known as compassionate use, is a path by which the U.S. Food and Drug Administration (FDA) may allow a drug that has successfully completed testing in initial phase 1 trials, and is going through later phase trials, to be made available to patients who are not eligible for active trials. Right to Try refers to laws at the state and federal levels, which allow patients access to experimental therapies in a similar manner as expanded access, but do not require 24 SEP / OCT 2020 I  HEALTHCARE JOURNAL OF NEW ORLEANS FDA oversight. Currently, pharmaceutical companies are under no obligation to provide drugs, under these laws, and use of this program has been relatively limited. Right to Try sounds like a gift to terminal patients who do not qualify for a clinical trial, and in some cases, might be better for an individual patient than a trial. Do you agree? Matrana We strongly believe that clinical trials are best, but not all patients meet the often stringent eligibility criteria to enroll in trials. For these patients who have no other options, the expanded access program and right to trymay allow for access to potential- ly live saving therapies. What are the pros and cons of Right to Try? Matrana Advocates for Right to Try tout the time it can potentially save, especially for terminally ill patients who have little time to deal with red tape to get access to poten- tially life-lengthening therapies. Critics see FDA oversight as a critical step to maintain patient safety, and helpful because of the support and knowledge the FDAcan provide about these experimental drugs. Expanded access has been around a lot longer. What are the pros and cons of this option? Matrana Expanded access programs pro- vide the ability for patients to access certain experimental drugs through an FDA sanc- tioned process, without having to enroll in a clinical trial. Unfortunately, this process can be time consuming, and generally requires a burdensome amount of regulatory and ad- ministrative paperwork and red tape, and may not always be approved.The treatments haven’t been completely studied, may not actually work, and in some cases, may even cause more harm than good.