Wall Street Journal Examines ‘Compassionate Use’ for Prescription Drugs Waiting for FDA Approval
The Wall Street Journal today examines a "surge in patient activism" that has prompted some pharmaceutical companies to provide experimental treatments to patients with serious illnesses. In the past, many pharmaceutical companies did not distribute treatments before they received FDA approval, "viewing it as a diversion from getting their products to market," the Journal reports. Today, however, some large pharmaceutical companies, such as Sanofi-Synthelabo, Eli Lilly and AstraZeneca, have made medications available for "compassionate use," a move that promotes treatments in development and "can generate goodwill for an industry that has been under fire for high prices and profit margins," the Journal reports. The new trend has allowed more patients who cannot participate in clinical trials or who suffer from diseases with limited treatment options to take medications that the FDA has not approved, but "getting drugs this way remains quite difficult and involves navigating a sometimes complicated bureaucracy," the Journal reports. Some companies -- such as ImClone and Bristol-Myers Squibb -- have established expanded-access programs, but the programs require FDA approval. Physicians also can obtain experimental treatments for patients on a case-by-case basis, but the FDA must agree that the medication will help the patient and that the patient does not qualify for a formal clinical trial (Fuhrmans, Wall Street Journal, 12/5).
The Journal today also examines the increased number of not-for-profit health care organizations that have launched accreditation programs for clinical trials in response to patient concerns about the safety of the trials, a lack of volunteers for the trials and a decision by HHS to disband a federal trial oversight committee. The National Committee for Quality Assurance on Tuesday launched the Human Research Protection Accreditation Program, and a coalition of medical schools, universities and patient advocacy groups formed the Association for the Accreditation of Human Research Protection Programs last year. In addition, the Pharmaceutical Research and Manufacturers of America earlier this year established a set of "voluntary principles" for clinical trials. However, a report issued in October by the Institute of Medicine found that accreditation of clinical trials has "considerable potential to systematize and accelerate quality-improvement processes" but added that Congress should require researchers to conduct the trials under the authority of a federal program. HHS recently disbanded the National Human Research Protections Advisory Committee, established in 2000 under the Clinton administration, but an HHS spokesperson said that the department plans to establish a new Advisory Committee on Human Subjects Protection (Landro, Wall Street Journal, 12/5).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.