Returning to the subject of expanded access to developmental medicines (When Compassion Isn’t Enough), I want to be clear that it wasn’t me who coined the term “expanded access.” As one of my former FDA colleagues commented, “In March 1990 the IOM Roundtable for the Development of Drugs and Vaccines Against AIDS held a workshop “Expanding Access to Investigational Therapies for HIV Infection and AIDS.” FDA staff, including me, participated in the Keystone national policy dialogue (Expanded access to promising therapeutic drugs for HIV infection and AIDS with implications for other life-threatening diseases) in the early 1990s. Also in the early 90s, FDA used the term expanded access at advisory committee meetings and at meetings of the National Task Force on AIDS Drug Development.”
Naming issues aside, this remains a highly contentious issue – and for all the wrong reasons. A new paper from the Goldwater Institute, “Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of their Treatment,” points the finger at the FDA as a roadblock to access, “Sadly, over half a million cancer patients and thousands of patients with other terminal illnesses die each year as the bureaucratic wheels at the FDA slowly turn.”
Nothing could be further from the truth.
What the paper presents a libertarian platform, “The burdens imposed on a terminal patient who fights to save his or her own life are a violation of personal liberty.” Maybe so, but the Supreme Court has ruled otherwise. 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
The paper continues, “Such people should have the option of accessing investigational drugs which have passed basic safety tests, provided there is a doctor’s recommendation, informed consent, and the willingness of the manufacturer of the medication to make such drugs available.”
I don’t think that anyone of those constituencies has any argument on that point. But should the FDA be cut out of the process? According to the paper, “… bureaucratic impediments violate an individual’s fundamental right to try to save his own life.”
But that’s consistent with the author’s libertarian philosophy. She believes that the “vast new granting of power (of the Kefauver-Harris Amendments) “was unwarranted.” So, consider the source of the argument.
Alacrity is important, certainly. But process is important too, as is collecting data on expanded access use.
The paper does raise important questions, such as at what point in the drug development process should an investigational product be available to patients? The author argues for Phase I. That’s an aggressive position, but one worth debating.
One item that paper ignores is that for the FDA to address single patient INDs with both more careful attention and speed is funding. That’s more than the 800-pound gorilla sitting in the room – it’s the 800-pound gorilla sitting on the chests of desperately ill patients who want access to investigational medicines.
The author quotes Patty Delaney. Patty (who passed away in 2008) was the FDA’s main liaison to the cancer community and a tireless soldier for “doing the right thing.” She was a pit bull on behalf of patients.
According to the paper, “As Patty Delaney, the former director of the FDA’s cancer liaison program explained in 2007, “the patient has a right to be heard, but in the end, it’s the data that matters. FDA opinions about safety and efficacy are always based on data.”
I’ll side with Patty.