title: Existing FDA pathways have potential to ensure early access to, and appropriate use of, specialty drugs.
abstract: Specialty drugs are notable among prescription drugs in that they offer the possibility of substantial clinical improvement, come with important risks of adverse events and mortality, can be complex to manufacture or administer, and are usually extremely costly. The Food and Drug Administration (FDA) plays a critical role in ensuring that patients who could benefit from specialty drugs have access to them in a timely fashion. In this article we review the different strategies that the FDA can use to approve and influence the post-approval prescribing of specialty drugs. When specialty drugs show promise in early clinical trials, the FDA can expedite the drugs' availability to patients through expanded access programs and expedited approval pathways that speed regulatory authorization. After approval, to ensure that specialty drugs are directed to the patients who are most likely to benefit from them, the FDA can limit the scope of the drugs' indications, encourage the development of companion diagnostic tests to indicate which patients should receive the drugs, or require that manufacturers subject them to Risk Evaluation and Mitigation Strategies to ensure that their use is appropriately limited to a restricted population that is aware of the drugs' risks and benefits. Implementing these existing regulatory approaches can promote timely patient access to specialty drugs while preventing expensive and potentially inappropriate overuse.
authors.affiliation: Aaron S. Kesselheim (akesselheim@partners.org) is an associate professor of medicine at Harvard Medical School/Brigham and Women's Hospital, in Boston, Massachusetts.
authors.forename: Aaron S
authors.initials: AS
authors.lastname: Kesselheim
authors.affiliation: Yongtian Tina Tan is a medical student at Harvard Medical School, in Boston.
authors.forename: Yongtian Tina
authors.initials: YT
authors.lastname: Tan
authors.affiliation: Jonathan J. Darrow was a postdoctoral research fellow at Brigham and Women's Hospital at the time of this research.
authors.forename: Jonathan J
authors.initials: JJ
authors.lastname: Darrow
authors.affiliation: Jerry Avorn is a professor of medicine at Harvard Medical School/Brigham and Women's Hospital.
authors.forename: Jerry
authors.initials: J
authors.lastname: Avorn
mesh_terms: D017277:Drug Approval / Q000379:methods* / Q000458:organization & administration; D006801:Humans; D055553:Prescription Drugs / Q000600:supply & distribution* / Q000627:therapeutic use; D013997:Time Factors; D014481:United States; D014486:United States Food and Drug Administration / Q000458:organization & administration*
chemical_list: D055553:Prescription Drugs
keywords: Cost of Health Care; Legal/Regulatory Issues; Pharmaceuticals