Compassionate Use of Unapproved Drugs Raises Difficult Ethical Issues

Patients with terminal illnesses, including late-stage cancer patients, are understandably eager to try still-unapproved experimental drugs once other options have been exhausted. The U.S. Food and Drug Administration (FDA) recently created a program to make it easier for drug companies to grant ‘compassionate use,’ which allows patients in extraordinary need access to otherwise unauthorized treatments. However, the decision to dispense experimental drugs is left to the companies, which are often reluctant to take this risky step. Experimental drugs are just that–experimental; they may have catastrophic side effects or simply not work. When these drugs are given outside the framework of a well-designed clinical trial, it becomes impossible to decisively judge whether they actually work. And if all patients could bypass clinical trials, there would be no incentive for anyone to enroll in these clinical trials, meaning that new drugs would never be properly studied and approved. Compassionate use therefore needs to be approved by experienced medical professionals on a case-by-case basis, and remain an option of last resort only.