title: Compassionate use: a story of ethics and science in the development of a new drug.
abstract: This history chronicles the unusual development of the antiviral drug ganciclovir. The first compound with activity against human cytomegalovirus (CMV), ganciclovir was so clearly efficacious that a placebo-controlled clinical trial could not ethically be done, and the FDA rejected the first application to market the drug. Used to treat a blinding eye infection in patients with AIDS, the story of ganciclovir paralleled the spread of the AIDS epidemic. Both ganciclovir and AIDS caught the federal government off guard. Caught in a Catch-22 situation, the pharmaceutical company developing ganciclovir gave the drug away free for five years under compassionate use guidelines. The problems encountered in the development of ganciclovir provide guidance on how future drugs to treat life-threatening diseases can be developed.
authors.affiliation: Clinical Science Consulting, 612 Oak Avenue, Davis, CA 95616, USA. wbuhles@msn.com
authors.forename: William C
authors.initials: WC
authors.lastname: Buhles
mesh_terms: D000163:Acquired Immunodeficiency Syndrome / Q000150:complications / Q000453:epidemiology / Q000821:virology; D000998:Antiviral Agents / Q000266:history / Q000627:therapeutic use; D057176:Compassionate Use Trials / Q000941:ethics*; D003587:Cytomegalovirus / Q000187:drug effects / Q000472:pathogenicity; D003586:Cytomegalovirus Infections / Q000150:complications / Q000188:drug therapy* / Q000821:virology; D017277:Drug Approval / Q000331:legislation & jurisprudence*; D054796:Drug Dosage Calculations; D004345:Drug Industry; D015774:Ganciclovir / Q000266:history* / Q000627:therapeutic use*; D006678:HIV / Q000472:pathogenicity; D049673:History, 20th Century; D006801:Humans; D012173:Retinitis / Q000150:complications / Q000188:drug therapy / Q000821:virology; D014481:United States
chemical_list: D000998:Antiviral Agents; D015774:Ganciclovir