“Right-to-Try” Legislation: Progress or Peril?

Since 2014, state legislators have increased their efforts to find a way for terminally ill patients to gain legal access to experimental drugs not yet approvedbytheU.S.FoodandDrugAdministration(FDA).AsofJune1, 2015,40statesandtheDistrictofColumbiahadexaminedorwillexamine this burgeoning issue with bills that have already been filed and 18 signed so-called “Right-to-Try” laws (Table 1). Patients who are terminally ill and who are facing less than 1 year of life expectancy are willing to take greater risks and endure a higher danger of potential adverse effects than other patients, including taking experimental drugs with unknown risk-benefit ratios. For patients who have exhausted existing pharmacotherapy options, there are two conventional ways to access potentially life-prolonging investigational drugs: join a clinical trial if they are well enough to meet rigorous entry criteria or apply for FDA’s “compassionate use” exception. Although the FDA accepted 5,816 of the 5,849 expanded access applications it received in the last 4 years, some argue that the FDA approval process for allowing expanded access to experimental drugs has been slow and cumbersome. Besides, many of the delays in the process occur before submission to FDA when the drug sponsor is deciding whether or not to provide the drug in question. Although the compassionate use of investigational drugs has been a contentious issue, public debate has centered on whether the gain in providing the terminally ill with a slim chance at prolonging life is worth endangering a process designed to ensure the public health and the development of safe, effective medicines.