How HIV/AIDS Activism Led to Broader Patient Activism in Clinical Trials

Supporters of AIDS Project Los Angeles protest against budget cuts in HIV/AIDS prevention and care programs by Sacramento on June 5th, 2009 in Hollywood, CA. Credit: Jose Gil / Shutterstock.com

History shows just how slow public health officials, the federal government, regulators and pharmaceutical companies were to respond to the emergence of HIV in the 1980s. The reasons are numerous—it was a puzzling viral disease, it first appeared in what was then a largely stigmatized patient population, gay men, and there weren’t really any existing treatments for viral diseases.

One of the outcomes was the emergence of a strong patient advocacy program. That’s what you would call it in 2019, although in the mid-1980s it looks far more like political protests. Over the course of about a decade, thousands of gay men and their supporters formed the AIDS Coalition to Unleash Power (ACT UP). Initially, the organization protested. Ultimately that probably led to greater awareness of the disease, but it didn’t immediately turn into changes in treatment and research for HIV.

NPR writes, “The gay and lesbian community had created a dynamic network of self-help groups in response to the crisis. But their focus was on providing comfort to the sick: buddies to take you to hospital, lawyers to help you write your will.”

Mike Petrelis, who was diagnosed with AIDS in 1985 and given two years to live, recently told NPR of yelling at one of them, “I don’t want to write my will! I want a cure!”

Slowly, the organizers of ACT UP became more strategic. It wasn’t just demonstrations on Wall Street or protests at city hall, but specific rallies and attacks on the FDA, government offices and pharmaceutical companies.

David France, documentarian and author of “How to Survive a Plague,” remembers, “They would storm people’s offices with fake blood and cover people’s computers with [it.] They locked themselves to politicians’ desks. At one point, they barged into a meeting of a pharmaceutical company and turned over the shrimp cocktail tables.”

That led to an event at FDA headquarters. ACT UP wanted the agency to provide AIDS patients access to an experimental drug. Hundreds of activists arrived at the FDA headquarters, one group wearing lab coats stained with bloody hands, others bringing tombstones they made and lying down in front of the building. The tombstones read “Dead from FDA red tape.”

The FDA agreed to meet and within months created a policy to provide access to experimental drugs.

Although not quite to the level of the HIV epidemic, participation in FDA approval processes by patients and patient advocacy groups continue to have an influence on FDA policy.

A more recent example was the 2016 approval of Sarepta Therapeutics’ Exondys 51 to treat Duchenne muscular dystrophy (DMD). The disease affects mostly boys, and it is almost always fatal, with deaths ranging from the early 20s to 40s.

The approval was a year-long drama that included 109 members of Congress sending a letter to the FDA urging it to accelerate approval of a DMD drug, any DMG drug. In March 2016, 36 DMD experts signed a letter to the FDA urging it to approve the drug. And in an April 2016 advisory committee meeting that lasted 12 hours, there were 52 public commentators, including families and DMD children.

Internally, there was quite a bit of division, with several FDA leaders opposed to the drug, saying the company’s research was inadequate. Eventually, Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research (CDER), pushed it through, overruling her staff. The final decision went to Robert Califf, then the FDA’s Commissioner. Califf has similar reservations to the opposition groups, but ultimately sided with Woodcock.

Although nobody really knows exactly what was going on in Woodcock’s head, there were many who thought she was taking into consideration Sarepta’s financial position, that if the drug wasn’t approved, its stock would crash, and it wouldn’t be able to continue working on promising DMD research. And it’s likely that Congressional pressure, the pressure of the research community and patient advocacy played a role in her decision-making.

In a STAT article at the time, Ed Silverman wrote, “The fate of the Sarepta Drug has been closely watched as a litmus test for an intensifying struggle between the FDA and patient groups that want the agency to take a more expansive view toward approving medicines for unmet medical needs. In this instance, patient advocates hoped the FDA would use the accelerated approval process to endorse eteplirsen. This approach relies on a substitute outcome in a clinical trial to suggest a drug may have, but does not guarantee, a benefit.”

The relative flexibility on the part of the FDA to take social factors, and potentially even financial factors—for better or worse—into account can find its roots in ACT Up and the HIV epidemic.

France told NPR, “ACT Up created a model for patient advocacy within the research system that never existed before.”