PharmExec Blog

Evaluating Expanded Access Programs

Pharmaceutical Executive recently spoke with David Vance, Senior Director of the Compliance Counsel at Noven Pharmaceuticals, about the birth of Expanded Access Programs and the role patient advocacy groups can play in shaping those programs.

PE: How did patient advocacy groups first take on a role in shaping Expanded Access Programs (EAPs)?

DV: I was working at Burroughs Wellcome back in 1986. In fact, September 19 was the 25th anniversary of the first AIDS drug, and I was there at Wellcome when we did that. It was the FDA’s recognition that people were dying, sometimes in only six months, of AIDS. And when we announced that we had a drug that could extend lives, FDA knew we had to figure out a way of getting the drug to people before it was approved, because those patients just didn’t have that much time to wait. So the whole idea of expanded access or early access actually began in large part with AZT. Burroughs Wellcome began working a lot with patients, particularly the AIDS activists who were so persistent and were pushing for FDA to do things quickly, and for companies to do things quicker and cheaper.

PE: What is the relationship today between patients groups and pharma when it comes to setting up these programs?

I think it’s wise for the companies to work with the activists to make sure that there is a proper expectation of what the drug may or may not be able to do, make sure that the company is aware of the needs of the patients, and make sure that the materials that are being developed for the EAP are developed with the activists groups—so that if anyone questions whether the materials were clear enough, the pharma company would be in a far better product liability position. Although generally I don’t think that there’s been a great product liability risk with these programs, if they’re done responsibly. We’ve all got to be careful, because of the distrust of the pharmaceutical industry, to make sure that we are transparent.

Another area where the patients groups have played a key role at times is when a pharma company has a life-saving drug and just can’t make enough of it. And sometimes getting patient groups involved, if they’re willing to be the people who help run the lottery, is a big help. They can explain to their membership—the good news is that we’ve got something here, but the bad news is that it can’t be ramped up fast enough to treat everybody.

PE: What stipulations govern which patients will gain access to these EAP medications?

EAPs are for serious or life-threatening diseases. Serious is defined as a disease that, without treatment, would result in a major, irreversible adverse event or condition that would lead to hospitalization or significant impairment of life.

Generally you’re going to start off asking, what were the original criteria for taking patients into the clinical trial? It may be, in the clinical trial—as so often happens—perhaps you’re excluding the sickest of the patients, because they’re so unlikely to make it that their deaths could destroy the drug for everybody, when you could possibly help people who are earlier in the disease state. Depending upon the toxicity of the drug itself, FDA often will allow you to treat a broader range of people in the EAP than were treated in the clinical study if it looks like it’s medically reasonable. And it is a standard that there is no other reasonable alternative for these patients.

PE: Is liability a concern for pharma when setting up EAPs?

DV: EAPs are still considered clinical studies. So anybody that wants to administer a drug through an EAP has to register as a clinical investigator. They get the full clinical investigator’s brochure of all the background and the information that is known to date on the drug. And the patients who enter have to sign an informed consent as a patient in a clinical study. So again it is very important to let patients know what legitimate expectations are, because the drug still is not approved. They’ve always had to have the informed consents and to make very clear that this drug is promising, but it has not been approved and things can still go wrong.

At Burroughs Wellcome I think we felt, to a degree, that with EAPs—particularly for life-threatening diseases or those that are causing severe handicaps—that people were just so grateful to get help, that there’s less of a liability risk in reality.

PE: What kinds of incentive does pharma have to participate in these EAPs?

I think it’s kind of a win-win, if you’re saving lives. And you’re also hopefully getting the drug out early, and getting the data back in a hopefully a little bit more controlled fashion. It also allows you to get a little bit more safety issues data. I’ve often said it might be an opportunity to add other kinds of patients in, in a sub-study. Potentially, if the EAP program goes well, you’re going to actually pull your whole adoption curve forward, you’re going to save some patients, and hopefully give them a free drug. And by the time the drug gets approved, the day it’s approved, you’re going to already have a certain number of patients on the drug.

PE: What work is still left to be done to leverage relationships between pharma and patient groups on these issues?

I think it’s making sure that we continue to build and maximize credibility together and that we recognize the minefields. We need to use the opportunities of transparency, of good clinical trial reporting, to make sure that we can hopefully reestablish the vanished credibility of the pharmaceutical industry with patient groups. We want to help remind people that there are companies out there that are trying. We win when we help patients. That is our goal. We’re not looking to sell drugs for indications that don’t work, because that hurts us in the long run.

David Vance will be part of a panel discussion moderated by William Looney on “The Challenges and Importance of Expanded Access Programs in the Context of the Current Industry Landscape” at the upcoming Center for Business Intelligence (CBI) conference: Expanded Access Programs—Develop Compliant Strategies to Manage Global Patient Access to Investigational Drugs, October 5-6 in Philadelphia. For more information about the conference and to register, visit

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