Last week, more than 500 people from industry, academia, government, and patient advocacy groups convened at the National Press Club in Washington to discuss, debate, and develop what comes next for expanded access, also known as compassionate use. The first voice to be heard was Dr. Janet Woodcock, director of the Food and Drug Administration’s Center for Drug Evaluation and Research, in a “fireside chat” I had the honor to moderate. Woodcock shared her belief that expanded access programs are only an iterative step towards more regular and robust use of platform trials.
Platform trials, which involve simultaneously studying multiple therapies for a single disease, provide an innovative pathway for broader access to experimental rare disease treatments. Platform trials can target a variety of subtypes of a disease and enroll “just about anybody who is willing to enter the trial,” Woodcock noted. Her hope is that drug developers will someday be more willing to pursue master protocols (clinical trials intended to simultaneously evaluate more than one investigational drug or more than one cancer type in an overall trial structure) if there were more successful examples of them. Step right up, ladies and gentlemen of the drug development industry.
But the issue of expanded access also exists in the present tense, and many thorny issues persist. One of the most significant issues is the silent elephant in the room: Who should pay for access to experimental medicines, and how much should they pay? Right to try doesn’t mean right to try for free.
Current FDA guidance offers only the ability to recover direct costs such as how much it costs per unit to manufacture the drug — raw materials, labor, and nonreusable supplies and equipment needed to make the quantity of drug required for the patient’s use — or costs to acquire the drug from another manufacturing source plus shipping and handling. In short, drugs offered under an expanded access protocol cannot be priced for profit.
In the wake of the imbroglio over BrainStorm Cell Therapeutics’ considering embracing a right-to-try strategy for expanded access to its experimental ALS compound that included charging a “semi-commercial” price significantly higher than its direct costs, the debate over expanded access reimbursement has come front and center. Insurance doesn’t generally cover treatments that haven’t been approved by regulators. But should it?