FDA's Expedited Approval Mechanisms for New Drug Products

Discovery is seeing what everybody else has seen, and thinking what nobody else has thought. ''-Albert Szent-Gyorgi M odern medicine and science have made in- credible strides in improving and extending lives. Nonetheless, many diseases and conditions still lack adequate therapies. According to a report issued by the President's Council of Advisors on Science and Technology (PCAST) in September 2012 on Propelling Innovation in Drug Discovery, Development, and Evaluation, roughly 30 million Americans suffer from 7, 000 rare diseases, but only 350 therapies are approved as treatments. 1 Indeed, ''96 percent of orphan diseases, including rare cancers, lack effective therapies. '' 2Other conditions demand improved therapies as well. For example, ''heart disease and stroke remain leading causes of mortality. '' 3The public health need for continued research and development of new drug and biologic products for significant diseases is clear and compelling. But beyond these important health reasons for stimulating research and development of new compounds, there are ancillary and supportive economic considerations for propelling innovative research and development. According to the Pharmaceutical Research and Manufacturers of America (PhRMA), 4 the United States' biopharmaceutical industry contributes substantially to the U. S. economy. PhRMA reports that the industry directly employs over 800, 000 workers in well-paid jobs and diverse fields, and supports an additional 2. 5 million jobs across the country. 5Moreover, PhRMA asserts that it supports over 789 billion in total economic output. 6 For several years, though, the increased time and money necessary to develop a new compound, the failure rate of prospective products, and a decrease in venture capital investments, among other strains on the industry, have propelled concerns that innovative research in the U. S. might wither, stop, or move to other nations or regions, decreasing the potential short term access for U. S. patients to some new products, potentially leaving others unexplored entirely, and hurting a significant segment of the U. S. economy. As a result, Congress, the Food and Drug Administration (FDA), and the pharmaceutical industry have sought to nurture an ''ecosystem'' conducive to the development of innovative, safe, and effective new compounds in the U. S. Among the mechanisms developed are four expedited approval mechanisms, the most recent of which-the Breakthrough Therapy designation-Congress created in 2012 through