FDA Touts Benefits of Compassionate Use
​[Thursday, April 04, 2019]

A joint blog by the FDA Commissioner and the Center Directors for Drug and Biologics this week highlighted the important role expanded access (also known as Compassionate INDs) plays in the treatment of patients with no available options, and the benefits it may provide the manufacturers. Besides being potentially helpful to needy patients, the FDA noted that participating in an expanded access program has rarely led to negative outcome regarding a manufacturer’s market approval application. FDA has never made a negative decision about a market approval application based on outcomes of the expanded access data. On the contrary, data from expanded access has provided additional supportive data for market approval in several cases. Expanded access was always strongly supported by the FDA with more than 99% of the expanded access applications approved, simplified process, and faster decisions. However, the expanded access program has recently been challenged by the Right to Try law that provides all the same benefits as the expanded access program while eliminating FDA and IRB approvals. There is no legal or regulatory reason for the FDA to reject supporting data from Right to Try use, while FDA is explicitly prohibited from using unfavorable data from Right to Try-related use for negative decisions. Unlike the expanded access program that has more than three decades of history, the Federal Right to Try is less than one year old so data about its utility is not available. Because expanded access involves filing an IND with FDA and IRB approval, there is a financial and logistical cost for this program, which is eliminated with Right to Try. The biggest differentiator between Right to Try and Expanded Access is the ability to charge for the drug provided under each program. While expanded access is still considered an IND and the IND rules for charging for investigational drugs apply, the Right to Try is silent about this aspects, leading some legal experts to conclude that manufacturers can charge a patient for providing drugs under Right to Try without justifying the cost to regulators. Although so far there are no reports of manufacturers charging for drugs under Right to Try, since there is no public or regulatory reporting requirement, it is not possible to rule out that manufacturers have not charged for drug supplied under Right to Try. The debate between compassionate use or Right to Try is just beginning. FDA obviously prefers the expanded access pathway as it gives FDA control over the use of investigational drugs outside of clinical trials. Only time will tell if industry or patients prefer to keep FDA out.