By: Richard M. Klein
The first patient-focused office at FDA – and very possibly the first patient engagement office in the federal government – was the Office of AIDS and Special Health Issues, established in 1994 at the height of the AIDS epidemic.
Richard M. Klein, a former entomologist in the agency’s New York Laboratory who had been working on human research protection policy at FDA’s suburban Washington, D.C., headquarters, was instrumental in setting up the new office. He recently sat down with FDA Voice to talk about his work with patient engagement during the AIDS epidemic.
Could you set the scene for us? What was it like as the AIDS epidemic grew in the years and months leading up to the creation of the Office of AIDS and Special Health Issues?
It was a desperate time. People lived 6 to 18 months after diagnosis. There was a pervasive sense that the government was not doing anything. One day in October 1988, the advocacy group ACTUP (AIDS Coalition to Unleash Power) functionally closed down the FDA. They surrounded the building at our Parklawn Headquarters in Rockville, Md., preventing employees from getting into the building. FDA saw other forms of protest as well. The director of the first antiviral division, created to address AIDS, received death threats. Individuals continually called up various offices of FDA complaining that no one at FDA was paying attention to AIDs. There were “fax attacks” – repeated attempts to send faxes clogging the phone lines. It was designed to disrupt “business as usual,” because they thought AIDS shouldn’t be business as usual. I was working in the New York lab at the time, and the community in New York was overwhelmed by this epidemic. People were getting sick left and right. I worked with a guy in the lab who contracted and later died from AIDS. That’s when I decided I wanted to take a more active role with AIDS and when the opportunity came up, I knew it was something I needed to do.
What did FDA do?
I mentioned the creation of the antiviral division to focus on AIDS drug development. There was also a National Commission on Aids with which we were involved. And FDA was integral to the National Task Force on AIDS Drug Development, a public/private effort brought together government, industry, academia, and patients to identify and remove barriers to successful drug development. Initially, FDA’s AIDS activities were coordinated by CBER since so many issues early on involved the blood supply, but then responsibility was shifted to the Office of the Commissioner. That’s when the new Office of AIDS and Special Health Issues was established to better communicate with the public and coordinate AIDS activities. The idea was to insulate the review division so they could do their work – and we would take the phone calls and we would get the faxes. The phone calls in the beginning were really terrible. People would say, “Murderer. Nazi. FDA is not doing anything, you are killing people,” and I wondered, what did I open myself up to?
We were the insulator, but also the conduit to the review divisions.
Tell us how you interacted with the AIDS community in those early days.
After the first meeting of the Task Force, I went down to Connecticut Avenue and had a couple beers with one of the major activists, Moises Agosto. I told him, I’m starting in this new job. What do you guys need and want? It was a really great conversation that I think opened up relationships. Later, when AIDS drug applications were filed with FDA, I would go to the advisory committee meetings and I would sit with the AIDS activists, rather than FDA staff. It gave me the opportunity to really know the community issues as we talked together about the proceedings.
Do you think that education was one of your principal roles?
Yes. I thought, if I could rationally explain to people how things work, how drugs get developed and why you can’t rush it, that you can’t expect it to turn out well if you don’t have the data, people would listen and open up to the idea that regulation has a real purpose. I would go to scientific meetings and companies with their marketing people would be there. They would hold meetings with activists and give them what I would describe as sales pitches on results involving maybe one or two patients and everybody would get very excited. But the companies would talk to the activists and say FDA is holding things up. The community would come to me and ask why is FDA is doing that. While I couldn’t talk about specific applications, I would tell them “This is how it works, any action FDA takes they send a letter to the company describing the reason(s).” I always told the activists to ask the company for the letter. They would get the letter and it would be redacted, or missing pages. “What does it say to you?” I asked them. We can’t talk about the drug and they don’t want to provide the specifics. The activists would say “Oh.”
Ultimately, patients played an important role in AIDS drug development didn’t they?
They did. People really need to understand how involved the activists were in both the policy and the science. They learned and they got into the fabric of what was going on. They were thinking outside of the box and doing a really good job at that. They would meet with NIH to learn about the basic viral and cellular research, and write White Papers about drugs in development. They pushed for the use of accelerated approval based on a surrogate endpoint – viral load. And they also pushed for expanded access, which was already on the books but wasn’t being used as much as it could have been. What’s important though, is that they recognized that policy had to move in tandem with science.
And these advocates really pioneered the patient representative program?
It all started with these guys coming in from San Francisco, all jet-lagged, to speak at the advisory committee’s open public hearing scheduled at 8:30 a.m. – before FDA and the company gave their scientific presentations. Mind you, this was before the internet and before advisory committee documents were made available 48 hours in advance. They told me “We’re coming in blind, deaf, and dumb. How can we meaningfully comment?” Some were so frustrated they would stand up during meetings and blow whistles and shout “this is an outrage.” So Lee Zwanziger, the executive director of the advisory committee, to her credit, moved the open public hearing to later in the day, after the sponsor and FDA presentations about the data. The activists also wanted to sit at the table and be part of the conversation that could so strongly affect their survival. So we worked with the review staff and with Lee to make them consultants to the committee, but they still couldn’t see the confidential background documents. So Lee said if we pay them, they can sign a contract that binds them to confidentiality. That was brilliant. This was the beginning of the patient representative program that today includes about 300 patient representatives covering more than 200 different diseases, giving patients a voice at the table.
In 1996, when protease inhibitors were approved, everything turned around. As the age of the drug cocktail evolved, AIDS patients were living longer, better lives. They could worry less about having to go to the doctor and fearing a diagnosis of new opportunistic infections and, instead, could worry about such mundane things as going to work, or paying their gas bill. This was such a success.
Interestingly, much later, when FDA was coming under fire because of its regulatory role and activities, it was AIDS activists who went to Congress and argued that FDA added necessary and important value to drug development.
Editor’s Note: The Office of AIDS and Special Health Issues took on other patient engagement responsibilities over the years and its name and its place in FDA’s organizational structure evolved. Today, it is known as the Office of Health and Constituent Affairs.
Richard M. Klein, is the Director of FDA’s Patient Liaison Program