Expanded access programs can create high demand for a treatment that’s in short supply, straining a company’s resources and potentially jeopardizing the treatment’s full approval and long-term availability. Be sure to read The FDA Approval Process: Can We Have This Drug Now? for a Q&A about the FDA and its process for approving new drugs.
The U.S. Food and Drug Administration (FDA) decides whether a company has permission to provide a drug or other treatment to patients prior to completion of the full FDA approval process. But actually supplying the drug is left to its developer.
All the experts we interviewed said that supplying a drug early — either through expanded access or accelerated approval — can jeopardize a company’s ability to take the drug through the full approval process and ultimately to provide it on a long-term basis. Bioethicist Arthur Caplan suggests that an independent advisory board could help companies with these complex decisions, and that funding from outside the company could help offset the high costs and risks of supplying a drug earlier than usual.
See the four articles included in this Web-exclusive package:
Abby Bronson: A Drug Developer's Perspective — Bronson is the Duchenne muscular dystrophy program manager at Children's National Medical Center in Washington, D.C., where she works with various stakeholders to further therapeutic development for this disease.
Arthur Caplan: A Bioethicist's Perspective — Caplan is the William F. and Virginia Connolly Mitty Professor and founding head of the Division of Bioethics at New York University Langone Medical Center in New York City. He has served on national and international committees as an adviser on ethical issues related to biology and medicine.
Timothy Miller: A Doctor's Perspective — Miller has been a university-associated neurologist specializing in neuromuscular disorders who has recently moved to the biotechnology industry. He has been the director of the MDA-supported pediatric neuromuscular disorders clinic at Children's Clinics for Rehabilitative Services in Tucson, Ariz.; an assistant professor of neurology, pathology and pediatrics at the University of Arizona in Tucson; and medical director for U.S. medical programs at the Cambridge, Mass., biotechnology company Genzyme. He is now senior director and head of medical affairs at Cytokinetics, a biopharmaceutical company in South San Francisco, Calif.