COMPASSIONATE USE: Physician Calls for Extended Programs
In the current issue of The New Republic, Harvard Medical School and Beth Israel Deaconess Medical Center's Dr. Jerome Groopman explores the need to regulate so-called "compassionate" or "expanded access" programs, which aim to bring drugs not yet approved by the FDA to those who desperately need them. As "[o]ne of the most important components of the experimental-drug development process," Groopman argues it is "imperative that attention and structure be placed" on such programs, which exist "in a gray area of federal regulation, drug company prerogatives, and patient advocacy." He notes that one of the programs' greatest shortcomings is the failure to supply an adequate amount of medication to the population of patients, necessitating lotteries, quotas and other distribution schemes. Groopman describes a number of factors that conspire to keep drugs like anti-breast cancer treatment Herceptin and protease inhibitor Saquinavir from patients who might benefit. For drug companies required to provide the drug for free while closely monitoring usage, "the process seems fraught with financial and strategic risk." Further, the FDA has no codified protocol for compassionate use programs, and reacts instead on an inefficient, case-by-case basis. Groopman observes, however, that a greater good can come of expanded access programs, such as when AIDS activists and political pressure forced a "shotgun wedding" of drug companies and the FDA at the "expanded-access alter." He calls for the FDA to "generate a set of criteria designed to encompass the largest number of patients who might benefit from the treatment," and for tax write-offs or extended patents to go to drug companies who provide their drugs in compassionate use programs" (4/26-5/3 issue).This is part of the California Healthline Daily Edition, a summary of health policy coverage from major news organizations. Sign up for an email subscription.