The compassionate use conundrum

Every so often the mainstream press runs a heart-wrenching story [New York Times 05/18/09] about a person with terminal illness-often cancer-who is trying to gain access to an "experimental therapy," hoping for a last chance at life. Some of the more vocal advocates argue that terminal cancer patients should be allowed to use an experimental treatment prior to FDA approval.

On a purely emotional level, it makes sense. However, there are sound reasons to deny patients, even those with no other option, use of untested medications.

In short, compassionate use is the program that gives patients who have no other treatment options access to drugs that have not yet been approved by FDA. Drug companies can give patients access to experimental drugs by either the expanded access program or single access program.

The expanded access program usually involves drugs in phase III trials. In cases in which the drug has only phase I or animal data, the company can use a single access approval request to FDA. Paperwork for these emergency requests can be done in as little as 24 hours.

But there is one caveat that is overlooked by certain patient advocates and the mainstream press: FDA must have evidence that the drug will be effective in the cancer it is being approved for. FDA does not assert a prerogative in denying patients potentially life-saving treatments; the agency uses rigorous scientific evaluation to determine the efficacy of every drug it approves. That is its charter with the US government.

Approving a drug that does not meet FDA efficacy requirements simply out of a false sense of compassion would, in effect, be like approving a toxic placebo, which would do more harm than good for a terminally ill person's quality of life.

The issue of unfettered access to experimental drugs has seen its share of courtroom battles. One group, the Abigail Alliance for Better Access to Developmental Drugs, lost a bitterly fought lawsuit in 2006 against FDA in which it sought essentially unlimited access to experimental drugs. The Alliance is bent on radically changing the drug approval process, one that it deems archaic and broken.

There is currently compassionate access legislation on the Hill [The ACCESS Act].  Proponents are hopeful that this initiative will finally get more traction under the new administration. However, the push for greater access to experimental drugs has its opponents within the cancer community.

The National Coalition for Cancer Survivorship and the American Society of Clinical Oncology are concerned that bills like ACCESS, will obfuscate the more important challenges facing the cancer community. One concern is that as only about 6% of cancer drugs make it through the trial system to FDA approval, allowing open access to untested drugs would undermine an already struggling clinical trial system. They contend, among other things, that we should be working in concert to persuade companies to sponsor more large, randomized trials that would give desperately ill patients access to drugs in a controlled environment.

At this year's annual ASCO meeting in Orlando, Rick Pazdur, head of FDA's Oncologic Drug Products will be chairing an ODAC meeting, giving us a bird's eye view of how an important part of the drug approval process works. It will be worth attending.

In recent years, public sentiment on the benefits of experimental cancer drugs has been guided by unrealistic hope.  As we move toward health care reform, we need to place science over emotion.