Pressure for new US FDA experimental drug regulations could create problems

The US Food and Drug Administration has proposed new regulations it hopes will ensure greater access to experimental drugs for patients with severe conditions who have no further approved treatment options. Pressure to introduce such measures comes, in part, from the combination of legislative and judicial activism by patients and their relatives, although some health care professionals are concerned about the effects on the availability of new drugs.

The FDA's Deputy Commissioner for Operations, Janet Woodcock, told a group of reporters that the main areas currently affected by experimental drug access programs are cancers, cardiovascular diseases and HIV/AIDS. The agency recognizes that, for patients who are not enrolled in clinical trials, the regulatory system is "confusing."

The FDA is therefore looking to provide clarity for patients and drugmakers so that the latter can recoup some of their costs of providing drugs on an individual basis. Larger drugmakers offer patient access programs which do not charge individuals, but smaller firms cannot afford to do so. There are also concerns about the potential liability of firms in cases of adverse reactions.