Federal and state lawmakers are questioning whether the FDA’s Expanded Access program, which allows terminally ill patients to use experimental drugs in certain cases, adequately addresses the current need, with several states considering legislation that would let patients bypass the agency in acquiring investigational therapies.

Under federal law, patients and physicians wishing to try experimental drugs as a last-ditch effort at prolonging life must first get FDA approval. However, four recently introduced bills — in Colorado, Louisiana, Arizona and Missouri — would grant terminally ill patients access to post-Phase-I experimental drugs without having to go through the agency.

The state actions have caught the attention of lawmakers on Capitol Hill. A June 18 letter from GOP senators Lamar Alexander (Tenn.), Tom Coburn (Okla.) and Richard Burr (N.C.) asks FDA Commissioner Margaret Hamburg how many requests for expanded access have been submitted in the last five years, how many were approved, what were the reasons behind the FDA’s decisions, and how much time did it take to grant approval.

There definitely is some level of need, says Arthur Caplan, director of medical ethics at New York University. “There are people who still do encounter trouble getting through the FDA [with their requests]. Sometimes the paperwork can seem onerous and sometimes the doctor isn’t sure what to do.” Still, the FDA may not be as much of a barrier as these lawmakers claim, he added.

Even if patients make it through the application process, “the drug company is under no obligation to release the experimental drug,” says bioethicist Yoram Unguru in the Johns Hopkins Bioethics Bulletin. Drugmakers may simply balk at the cost of producing extra supplies of the drug, or they may worry that extending compassionate use can make trial results more difficult to interpret.

Unguru also worries about potential unintended consequences, such as drugmakers offering physicians “incentives” to refer patients who may not be ideal candidates for the drug in question.

All these points may be moot, however, as state laws are still superseded by federal laws.

The FDA has not taken an official position on right-to-try laws, CDER’s Stephen King said, though it believes that the drug approval process still represents the best way to assure development of safe and effective medicines. — Lena Freund

Originally appeared in Drug Industry Daily, the pharmaceutical industry’s number one source for regulatory news and information. Click here for more information.