Abigail Alliance and patient safety

In the April issue of the ASCO News & Forum (note: currently only in print form; the Web site is not yet updated to April so I will include some passages from the article), Dr. Emil J. Freireich, MD, writes a letter to the editor in response to Dr. Allen S. Lichter, MD’s article explaining ASCO’s support of the FDA before the U.S. Court of Appeals for the District of Columbia Circuit in the case of Abigail Alliance for Better Access to Developmental Drugs v. Andrew C. von Eschenbach.

In his letter, Dr. Freireich — a professor at the University of Texas M.D. Anderson Cancer Center, an ASCO founding member and ASCO past president — argues that the oncology community should support expanded access or compassionate use access to experimental agents for patients. He states that, “Our first priority as physicians, as scientists, and as members of ASCO should be to provide the best possible care for our patients. Secondly, the Society should support its members as they provide such care to their patients.” Dr. Lichter argued that patient safety would be jeopardized if unselected patients were given access to unproven agents. Dr. Freireich responds:

I have difficulty understanding how such a position ensures patient safety. The patients on whose behalf the Abigail Alliance filed its brief are in the same circumstance as patients who have participated in phase-1 clinical trials to evaluate investigational drugs with one notable exception: they have the advantage of knowing the outcomes of such trials. That is, they have critical information about appropriate dosage schedules and dose-limiting toxicities. So, for these patients, the situation is actually “safer” than for patients enrolled in the original phase-1 clinical trials. One balances the risks of administering a drug that has completed phase-1 clinical trial testing against the risk of mortality for patients with advanced cancer without significant treatment options. It is hard for me to understand who is being protected from what.

I agree with Dr. Freireich in that our first priority should be to provide excellent patient care and that the Society should support such endeavors. I disagree that providing unproven agents to unselected patients is in the patient’s best interest. I believe that his premise of promise of a phase-1 drug is flawed. We know response rates in phase-1 trials are roughly 3% give or take 2%. Why would one motivated to provide patient-centered quality health care expose a patient to a drug with minimal to no chance of a meaningful result? At least on study one meaningful result is the knowledge learned from that patient’s experience.

The motivation, I think, is a misinterpretation of hope. As oncologists, our most obvious hammer is anti-tumor therapy. Given we have this chemotherapy "hammer," and a little bit of information about an emerging agent, we want to use it to treat the cancer. This is a far easier approach than talking with the patient, exploring their goals, taking a life history, or simply being present in the weight of bad news and impending death. It is the oncologist’s great gift that we are positioned to care for people at the most important of times: the end of their lives. It is a flawed premise that hope is tied to survival. Hope is pervasive emotion, not some ephemeral passion, and hope has much more to do with connection, family, meaning, spirit, integrity.