The Power of Advocacy
A First-Hand Account of How Testimony Helped Win an FDA Panel's Approval of a New Drug
Lipohypertrophy, caused by excess visceral adipose (fat) tissue (VAT), has been estimated to affect as many as one-third of people living with HIV, and is associated with the use of antiretroviral therapy, as seen from studies in which treatment-naive people began therapy and accumulated fat in the abdominal area. VAT is different from subcutaneous fat, as it surrounds the vital organs and causes a hard, distended belly. It can be quite painful and extremely debilitating, and adversely affects quality of life and body image for those who have the condition. It can also put you at higher risk for cardiovascular disease (CVD).
The efficacy of the drug is modest, but studies of the drug so far have shown up to a 17% reduction in VAT for those using the drug for one year, which must be injected once daily under the skin. There is some evidence that it may lead to increased glucose intolerance and risk for diabetes in certain patients. However, the number of those developing increased glucose intolerance was only around 2-4%. And the question as to whether using the drug could lead to reduced risk for CVD, which was an initial hope, remains unanswered.
Only two weeks earlier, I had figured I would be home in Chicago that day, getting ready for a busy weekend of barbecues and some beach time for the Memorial Day holiday. But following a flurry of e-mails, phone calls, and communications between a core group of advocates that I had been working with closely for several years on development of the drug, I found myself instead at a Marriott Inn at the University of Maryland University College in Adelphi, Maryland.
Initially, when asked by some if I was coming to the meeting, I had said no, for a couple of reasons, not the least of which was the cost of the trip, but also the fact that I had some serious doubts about whether my being there could have any real effect on the panel's decision. This was the first ever Advisory Committee meeting I'd attended, so I didn't really know what to expect. When I asked my friend and colleague Tim Horn, of AIDSmeds.com, what it would be like, his reply was "fascinating, but exhausting."
As usual, he was right.
"Years of studies and millions of dollars invested in research and development all boiled down to an eight-hour meeting and 16 votes -- with a treatment that had the potential to benefit thousands of people living with HIV hanging in the balance."
As the meeting got underway, and I listened to the presentations by the drug's sponsor and by the FDA representative about the studies that had been conducted so far, and the safety and efficacy data compiled to date, it quickly became clear that this was exactly where I needed to be.
I had come to testify during the Open Public Hearing portion of the meeting on behalf of the Drug Development Committee of the AIDS Treatment Activists Coalition (ATAC), in support of a letter we had submitted urging the panel to recommend approval of the drug.
I was in awe of the processes of the meeting as they unfolded that day, with all the rules, procedures, and protocols. I was also struck by how years of studies and millions of dollars invested in research and development all boiled down to an eight-hour meeting and 16 votes -- with a treatment that had the potential to benefit thousands of people living with HIV hanging in the balance.
I arrived at the meeting location shortly before it started at 8 a.m., signed in, and received a badge designating me only as OPH #3, the third speaker during the Open Public Hearing. When I asked a very personable and helpful federal officer named Rheese why my name was not on the badge, she explained to me that it was to ensure confidentiality as a speaker. She then took the time to tell me exactly how the meeting would play out over the course of the entire day, and the point at which I would be speaking. She helped me put my luggage in a secure area, as I had already checked out of the hotel, and led me to my seat.
There were three public speakers for the day, and we each had been instructed prior to the meeting that we would only be allowed seven minutes to speak. A two-minute warning light would flash at five minutes, and the microphone would be cut off if we went beyond our allotted time.
The day before, I had rehearsed aloud what I was going to say several times and had timed myself -- making sure to enunciate clearly and speak at an even pace, providing proper emphasis -- and it came in at just under six minutes, so I felt comfortable with the length. But as I practiced, each time that I got to the part of my speech where I talked about the stigmatizing and disfiguring effects of lipodystrophy, I would start to get emotional, so I made a mental note to myself to make sure that it didn't happen on the day of the meeting.
After the morning's presentations and following a lunch break, the Open Public Hearing began promptly at 1 p.m. Debbie, a registered nurse from Florida, went first and spoke eloquently about her life living with HIV for nearly 20 years, the advocacy work she has done, and how the drug had helped her while she was in the study. In a moment of high drama, and as she neared the end of her talk, Debbie removed her pretty lime green jacket and revealed to the committee members and audience the effects that HIV and years of antiretroviral treatment had wreaked upon her body. Unfortunately, once you stop treatment with Egrifta, the visceral fat returns, so you have to remain on it indefinitely to maintain its positive effects. When the microphone suddenly cut off, the committee chair asked her to please continue, and she was allowed to speak until she was finished.
The second speaker, Lisa, talked about how painful it was for her to even just bend over, and how the treatment had helped her as well. She held up pictures for all to see how trim and fit she looked while in the study. I was amazed at both Lisa's and Debbie's strength, and by the great amount of courage it must have taken them to stand up and tell their very personal stories to a room full of complete strangers.
As I sat there listening to both of their stories, I remembered how, when I was deciding on whether or not to come to the meeting, I had wondered to myself, "How much difference can seven minutes really make?" At that instant I knew the answer to my question.
It was then my turn, and I stood up to speak. Sure enough, when I came to the part about the stigmatizing and disfiguring effects from lipodystrophy, my voice began to crack, but I quickly gathered my thoughts, composed myself, and continued on. I spoke about the need for additional post-marketing studies, and what exactly those studies might look like. I ended with the recommendation to the panel that the drug be approved as a medical and reconstructive modality, not as a cosmetic treatment, and that it was no different than breast reconstruction surgery after a mastectomy.
The committee chair and several panel members thanked the speakers for their "moving," "powerful," and "important" testimony. The panel went on with their thoughtful discussion, responding to questions from the chair, and continued to debate the pros and cons of Egrifta throughout the afternoon.
Finally, at 4 p.m., it came time to put it to a vote -- the members would be given 20 seconds, at which point their vote would be locked in. You could literally hear a pin drop in the carpeted room. The results flashed on the screen: 16 "yes," zero "no," with no abstentions -- unanimous. An audible gasp arose from the room. I choked up once again, and turned and smiled at Debbie and Lisa. Their chance at living a fulfilling life had just been handed back to them and thousands of others -- just like that.
After the meeting, representatives from the company, the agency, researchers, and even complete strangers walked up to thank us for our words, and expressed to us how important our testimonies had been. And even though I've thought of myself as an advocate for many years, I learned a very valuable lesson that day about the power and importance of advocacy, what it means, why it's important, and the sacrifices we sometimes have to make to truly be advocates.
It had made a difference. It was worth it.
Today was a great day for people with HIV.
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This article was provided by Test Positive Aware Network. It is a part of the publication Positively Aware. Visit TPAN's website to find out more about their activities, publications and services.
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