When Dying Patients Want Unproven Drugs

June 21, 2023

(The New Yorker) – AIDS advocacy—which drafted on previous movements—helped adapt the health-care system to the desires of patients. Today, they are consulted at every stage of the drug-development and approval process: they help shape funding strategies at the National Institutes of Health and contribute to technical debates over trial design, study criteria, and the relevance of particular metrics to their own experiences. As an F.D.A. representative put it, patients come to the table with their own Ph.D.—“personal history of disease.” Patient-advocacy organizations have flourished, and some have been extremely fortunate in their strategic decisions: the Cystic Fibrosis Foundation funded research into drugs that have proved nothing short of magical for some patients, transforming a death sentence into a manageable condition.

Investments in basic research, however, are generally long-term bets, and people with terminal diagnoses are understandably impatient. They have come to see drug-approval decisions as their moments of maximum leverage. (Read More)