A provocative piece recently published in the New England Journal of Medicine focused on the cost of new drugs that are approved under the FDA’s accelerated approval program. The program allows drugs to be approved based on surrogate endpoints that are “reasonably likely to predict clinical benefit,” and now represents about 10% of all new drug approvals. The surrogate endpoints often are a biomarker or functional endpoint, but approval comes with the requirement that the manufacturer conduct postapproval trials to confirm the drug’s efficacy for the approved indication. But once approved, manufacturers are free to charge consumers and insurers whatever they believe the market will bear. In the case of eteplirsen, approved for the treatment of muscular dystrophy, the cost is $300,000 per year. That price comes without evidence that the drug affects disease progression.
The authors suggested several remedies. Manufacturers could be required to offer additional price concessions to public insurance programs until confirmatory trials are completed. They also suggested that the FDA do more to ensure that confirmatory trials are conducted in a timely fashion after accelerated approval. Lastly, the authors recommended that drugs approved under the accelerated approval program with a cost above a certain threshold (such as $100,000 per year) be the subject of formal economic impact analyses after one to two years on the market (N Engl J Med 2017;376:2001-2004). The FDA’s new commissioner, Scott Gottlieb, MD, has promised to ease regulations to make sure that drugs reach the market more quickly. Dr. Gottlieb has worked at the FDA in other capacities and also has served on the boards of several pharmaceutical companies.
Once a drug receives accelerated approved, manufacturers are free to charge whatever they believe the market will bear without evidence that the drug affects disease progression.
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