User fees and beyond--the FDA Safety and Innovation Act of 2012.

n engl j med 367;14 nejm.org october 4, 2012 1277 legislators, and patient advocates. The driving force behind the legislation was the need to reauthorize statutorily defined user fees that pharmaceutical manufacturers (since 1992) and medical-device manufacturers (since 2002) have paid when they submitted applications to the FDA for the evaluation of new products. The bill sailed through a closely divided Congress for two main reasons. First, most policymakers understand that because of chronic underfunding of the FDA by the federal government, these fees have become essential components of the agency’s budget — particularly on the drug side, where they have amounted to more than $500 million annually in recent years (see graph). User fees have become required for the continued daily operation of the agency as it pursues its mission of protecting the public health. Second, the FDA and industry had reached agreement on important elements of the legislation and presented Congress with their plans to guide the law’s development. Apart from user fees, a major focus of FDASIA is streamlining the process of premarketing review of drugs and devices. For example, the legislation broadens the scope of the fast-track designation and the accelerated approval process for drugs for “serious or life-threatening” conditions. It requires the FDA to consider as clinical end points in the study of these drugs “epidemiological, pathophysiological, therapeutic, pharmacologic, or other evidence developed using biomarkers, for example, or other scientific methods or tools.” The FDA is also given the authority to designate a new drug candidate as a “breakthrough therapy” if “preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies [for serious or life-threatening conditions] on 1 or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.” When a drug is classified as a breakthrough therapy, the FDA will work closely with its User Fees and Beyond — The FDA Safety and Innovation Act of 2012