Accelerated Approval and Expensive Drugs — A Challenging Combination

• Published in: The New England journal of medicine Vol. 376; no. 21; pp. 2001 - 2004
• Main Authors: Gellad, Walid F, Kesselheim, Aaron S
• Format: Journal Article
• Language: English
• Published: United States Massachusetts Medical Society 25.05.2017
• Subjects: Drug Approval; Drug Costs; FDA approval; Humans; Insurance Coverage; Insurance, Pharmaceutical Services; Medicaid; Medicare; Pharmaceutical industry; Pharmaceuticals; Prices; Time Factors; United States; United States Food and Drug Administration
• ISSN: 0028-4793; 1533-4406
• DOI: 10.1056/NEJMp1700446

Accelerated FDA approval can mean that private payers choose not to cover a drug because of high cost and lack of evidence of efficacy, thwarting patient access, while major government payers must cover the product, directing substantial tax dollars to unproven drugs. For serious or life-threatening disease, the Food and Drug Administration (FDA) can approve drugs on the basis of surrogate end points that are “reasonably likely to predict clinical benefit,” through its accelerated approval review track. This pathway, which dates back to the early 1990s, was designed as a response to the demand for faster drug development in the context of the HIV/AIDS crisis. 1 Since then, the accelerated-approval program has expanded to include oncology products and drugs for other diseases, now accounting for about 10% of new drug approvals. 2 The accelerated pathway commits a drug manufacturer to conducting postapproval trials to . . .