FDA seeks to expand access to experimental drugs

A new agency proposal would also clarify when companies can charge for an investigational product.

By Susan J. Landers, AMNews staff. Jan. 1/8, 2007.

Washington -- Gaining access to a promising but still experimental treatment can present a bewildering path to physicians and their very sick, often desperate patients. The Food and Drug Administration proposed to shine light on that trail with new clarifying rules issued last month.

The agency intends to make experimental drugs more easily available to physicians and to seriously ill patients who have exhausted all other treatment options. The proposals also clarify how manufacturers can recoup the expense of providing the still-unmarketed products to those in need, said Janet Woodcock, MD, the FDA's deputy commissioner for operations.

"There have been barriers to patients seeking access, because the provisions were somewhat confusing," said Dr. Woodcock. "We felt there was unequal access."

Patients have been able to obtain experimental treatments for decades. For instance, in 1987 the FDA created a regulatory mechanism to broaden access to drugs beyond the scope of clinical trials. Treatment INDs -- investigational new drugs -- allow those with serious and life-threatening illnesses to use such products. As a result, thousands of people with HIV/AIDS, cancer and cardiovascular disease have taken advantage of promising therapies.

Participating in a trial is the surest route to investigational pharmaceuticals, but sometimes a patient doesn't qualify for a trial or can't travel to a distant site, said Dr. Woodcock. In such cases, treatment INDs could allow access. Many large drug companies also run compassionate-use programs to provide investigational drugs to such patients.