An Rx for the System: Gaining Access to Investigational Drugs

Expanded access programs are run in conjunction with late-stage clinical trials so that promising investigational drugs can get into the hands of patients who stand a good chance of benefiting from them, and who have exhausted their other treatment options, including clinical trials.
BY TONY HAGEN @oncobiz
R. Ruth Linden is an independent health advocate and bioethicist who prides herself on publicly avowing her opinions. On the issue of expanded access programs (EAP) for investigational drugs, she comes down squarely on the side of “not good enough.”

EAPs are run in conjunction with late-stage clinical trials so that promising investigational drugs can get into the hands of patients who stand a good chance of benefiting from them, and who have exhausted their other treatment options, including clinical trials. EAPs can include either individuals or groups of patients. To open, they must be requested by a drug developer or a treating physician, and then approved by the U.S. Food and Drug Administration.

But the programs come too late in the game for many cancer patients, and many more are simply unable to participate, says Linden, who holds a PhD in sociology and is president of Tree of Life Health Advocacy of San Francisco, California.

As a result, she and other patient advocates are calling for dramatic changes to FDA policy designed to get experimental drugs much more quickly into the hands of patients who may benefit from them.


Linden has been a part of the battle to widen access to experimental drugs since the mid-1990s.

In those days, HIV/AIDS patients were dying in droves and had nothing to lose by seeking every means possible to compel drug companies to grant them broad access to promising new drugs. Civil disobedience and dramatic protests were key to their modus operandi, but their efforts also could make it difficult to impossible for drug companies to recruit patients for clinical studies.

 “The HIV/AIDS activists had tremendous power because they could shut down a trial,” Linden says.

Seeing how successful they were, a coalition of breast cancer activists took a leaf out of the same playbook and in December 1994 marched on Genentech’s campus in South San Francisco in an attempt to force wider access to an experimental formulation they thought could save lives.

“A small cadre of breast cancer activists worked with HIV/AIDS activists to adapt their methodologies to a very promising biological that Genentech was developing—Herceptin (trastuzumab). They did civil disobedience on the Genentech campus, handcuffing themselves to a building and driving onto the campus honking horns and shouting through bull horns and carrying portable sirens to make a great spectacle—because women were dying who had a chance at life extension if they had access to this investigational therapy,” Linden says.

It worked by bringing Genentech to the bargaining table, and an expanded access arm of the clinical trial was created using a lottery, becoming the first EAP for any cancer drug, according to Linden.

Though EAPs have come into broader use, they remain a poor solution to the problem of getting promising treatments into the bodies of those who need them in a timely manner, says Linden. Many patients who gain access to such medications do so when their bodies are already severely ravaged by disease or by highly toxic chemotherapy.

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