When Compassion Isn't Enough

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  • 04/01/2014

A very important series of stories in this week’s issue of BioCentury (authored by Steve Usdin) addressing the thorny, urgent (and too frequently ignored) topic of expanded access to experimental medicines. The articles generally refer to “compassionate use” and “compassionate access.” That’s the wrong terminology – and a good place to begin the conversation.

In January 2008, the U.S. Supreme Court, without comment, opted not to accept an appeal of Abigail Alliance v. von Eschenbach. In other words, the federal appeals court ruling that patients do not have a constitutional right to experimental drugs stands.

This is a tough, emotional issue and, with such heated rhetoric on both sides, it's easy to lose sight of the fact that everyone wants the same thing -- expanded access to drugs under clinical investigation.

For me this issue began (when I was Associate Commissioner for External Affairs at the FDA) during a meeting with Theresa Toigo (then the Director of the agency’s Office of Special Health Issues) and Frank Burroughs (founder of the Abigail Alliance for Better Access to Developmental Drugs). The meeting centered on Frank’s assertion that the FDA could do more to expand its parameters for patient access. We all agreed more could be done. Frank left. Terry stayed – and I asked her why we (the FDA) used the term “compassionate use.” Her answer was simple – “It’s what we’ve always called it.”

I said, “Let’s change what we call it." Allowing desperately ill patients into clinical trial programs shouldn't be an act of noblesse oblige it should be an act of civil society. We decided to work towards changing the term to “expanded access.” And we did. It’s one of the things I am most proud of accomplishing during my time at the FDA.

But it has to be more than rhetoric. The words have to mean something – and what they have to mean is that more patients get more access to more experimental medicines more expeditiously. In many cases it is, literally, a life-or-death situation.

When it comes to pharmaceutical safety, pure libertarianism isn't in the best interests of the public health. Expanded access to experimental drugs simply can't and shouldn't morph into total, unfettered access.

That doesn't mean the status quo is working. What it means is that the FDA needs to figure out a way to dramatically broaden and facilitate expanded access to experimental drugs under its review. And the Abigail Alliance and its supporters need to keep up the pressure to improve the current system.

This is best done in a spirit of collegiality rather than a confrontational courtroom or in Congress. Should expanded access design and development planning discussions be binary conversations between a sponsor and the FDA? What about the patient community? Perhaps, as part of the FDA’s current initiatives to enhance both the timeliness and weight of the patient voice, expanded access plan development and execution should involve patient organizations.  

In the BioCentury articles, Usdin speaks to the phenomenon of social media and the pressures it can (and does) bring to bear on sponsors. Maybe it’s time to harness that power to make the process both more inclusive and better.

It’s time.

The BioCentury articles can be found here, here, and here.


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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