Bending the Rules of Science Can Save Lives
Says Fauci
Marcia Frellick
|November 20, 2015
The top HIV/AIDS expert in the United States took to the TEDMED stage to tell an audience of mostly scientists that although core scientific principles must remain sacrosanct, there are moments when conventional wisdom should be turned on its ear.
Anthony Fauci, MD, director of the National Institute for Allergy and Infectious Disease, said he used his ears to listen to patients and their caregivers to help inform the decision making that led him to the top of his field.
The beginning of the AIDS pandemic, in the early-1980s, were dark days for Dr Fauci, the director of the institute behind the design, implementation, and funding of infectious disease research, including HIV/AIDS. But he was also a physician taking care of hundreds of desperately ill AIDS patients.
"I was trained as a healer and I was healing no one," Dr Fauci said at TEDMED 2015 in Palm Springs, California.
In the early days, an activist community of mostly gay men was becoming increasingly frustrated by the lack of inclusion in decisions about drugs and treatment.
There was a rush to develop drugs and get them into clinical trials, but the system to get them approved was controlled almost exclusively by the federal government, and the process took years. Patients presenting with advanced disease had just months to live, and they demanded input.
Dr Anthony Fauci (Source: TEDMED 2015)
Dr Fauci acknowledged he was among the majority of scientists who were not inclined to listen to a group of nonscientists, at first.
"They came onto our campuses and our facilities with dramatic theatrical costumes, blowing whistles, setting off smoke bombs, disrupting our function. They wanted to be heard, but no one was listening to them," Dr Fauci said.
"It was clear that something had to give. And at that moment, that something had to be me," he said.
Over a period of months, Dr Fauci said he started looking beyond the theatrics and saw "real underlying fear and terror for their lives." He listened at protests. He went to Greenwich Village in New York City to participate in town hall meetings. And then he accompanied an activist to the Castro District in San Francisco, a hotbed for the disease, and talked with a man who had HIV and cytomegalovirus, which is one of the complicating infections that attacks the retina and can lead to blindness.
I was trained as a healer and I was healing no one.
At the time, the National Institutes of Health and the US Food and Drug Administration (FDA) were putting together a protocol to determine the safety and efficacy of an experimental and potentially toxic drug against cytomegalovirus, called ganciclovir. But protocol mandated that if you take ganciclovir, you couldn't be on any other drug. Most of the people who needed the drug, however, were HIV-positive and were taking azidothymidine, the only HIV drug available at the time.
The man with HIV and cytomegalovirus, who was being cared for by his partner in his apartment, told Dr Fauci that the protocols left him with two choices: blindness or death.
After the realization that an inflexible system was leading patients to make impossible choices, Dr Fauci, with the help of activists in New York and San Francisco, proposed the "parallel track." In that system, researchers adhered to a strict protocol, according to the rules, but in parallel provided access to a drug on an experimental basis to patients with informed consent.
A Parallel Track
"Activists were pushing for the concept and they wanted me to come out publicly to endorse it," Dr Fauci told Medscape Medical News. "But the FDA was fundamentally against any changes."
When he endorsed the parallel track publicly anyway, it "created a bit of a brouhaha," he said. But the public response to his endorsement was so favorable that the government turned around and endorsed it as well.
Dr Fauci acknowledged there are still certain core scientific principles "that must remain sacrosanct and inviolate." However, he said, he has come to know when conventional wisdom needs to be challenged.
Another example of that came in the 1990s when a triple cocktail of drugs became available that increased the life expectancy of a 20-year-old infected with HIV from a few months to another 50 years.
Once again, conventional wisdom got it wrong and flexibility got it right.
Although that was great for the developed world, the drugs weren't getting to sub-Saharan Africa, which had 67% of the global cases of HIV, said Dr Fauci. Conventional wisdom said that with the state of the health delivery system there, doctors wouldn't be able to get people to take drugs every day for the rest of their lives.
Africans disagreed. In 2002, President George W. Bush sent Dr Fauci and a team on a fact-finding mission to determine the feasibility of a plan to effectively bring treatment and prevention to southern Africa.
There, Dr Fauci heard the same refrain: "Give us the opportunity and we will make it work."
When he returned, he and his colleagues presented what came to be called the President's Emergency Plan for AIDS Relief, or PEPFAR, which, over the past 13 years, "has prevented millions of infections and saved millions of lives," he reported.
The PEPFAR commitment is thought to be the largest by any nation to combat a single disease, and now provides treatment for 7.7 million people around the world.
"Once again, conventional wisdom got it wrong and flexibility got it right," Dr Fauci said.
He explained that his story is a message to others in medicine, science, and public health who will be questioned or confronted by people who don't agree with them. This will likely happen even more in the age of the internet, he warned.
Without violating scientific principles, Dr Fauci said, flexibility can alleviate suffering and save lives.
Dr Fauci has disclosed no relevant financial relationships.
TEDMED 2015. Presented November 19, 2015.
Marcia Frellick
|November 20, 2015
The top HIV/AIDS expert in the United States took to the TEDMED stage to tell an audience of mostly scientists that although core scientific principles must remain sacrosanct, there are moments when conventional wisdom should be turned on its ear.
Anthony Fauci, MD, director of the National Institute for Allergy and Infectious Disease, said he used his ears to listen to patients and their caregivers to help inform the decision making that led him to the top of his field.
The beginning of the AIDS pandemic, in the early-1980s, were dark days for Dr Fauci, the director of the institute behind the design, implementation, and funding of infectious disease research, including HIV/AIDS. But he was also a physician taking care of hundreds of desperately ill AIDS patients.
"I was trained as a healer and I was healing no one," Dr Fauci said at TEDMED 2015 in Palm Springs, California.
In the early days, an activist community of mostly gay men was becoming increasingly frustrated by the lack of inclusion in decisions about drugs and treatment.
There was a rush to develop drugs and get them into clinical trials, but the system to get them approved was controlled almost exclusively by the federal government, and the process took years. Patients presenting with advanced disease had just months to live, and they demanded input.
Dr Anthony Fauci (Source: TEDMED 2015)
Dr Fauci acknowledged he was among the majority of scientists who were not inclined to listen to a group of nonscientists, at first.
"They came onto our campuses and our facilities with dramatic theatrical costumes, blowing whistles, setting off smoke bombs, disrupting our function. They wanted to be heard, but no one was listening to them," Dr Fauci said.
"It was clear that something had to give. And at that moment, that something had to be me," he said.
Over a period of months, Dr Fauci said he started looking beyond the theatrics and saw "real underlying fear and terror for their lives." He listened at protests. He went to Greenwich Village in New York City to participate in town hall meetings. And then he accompanied an activist to the Castro District in San Francisco, a hotbed for the disease, and talked with a man who had HIV and cytomegalovirus, which is one of the complicating infections that attacks the retina and can lead to blindness.
I was trained as a healer and I was healing no one.
At the time, the National Institutes of Health and the US Food and Drug Administration (FDA) were putting together a protocol to determine the safety and efficacy of an experimental and potentially toxic drug against cytomegalovirus, called ganciclovir. But protocol mandated that if you take ganciclovir, you couldn't be on any other drug. Most of the people who needed the drug, however, were HIV-positive and were taking azidothymidine, the only HIV drug available at the time.
The man with HIV and cytomegalovirus, who was being cared for by his partner in his apartment, told Dr Fauci that the protocols left him with two choices: blindness or death.
After the realization that an inflexible system was leading patients to make impossible choices, Dr Fauci, with the help of activists in New York and San Francisco, proposed the "parallel track." In that system, researchers adhered to a strict protocol, according to the rules, but in parallel provided access to a drug on an experimental basis to patients with informed consent.
A Parallel Track
"Activists were pushing for the concept and they wanted me to come out publicly to endorse it," Dr Fauci told Medscape Medical News. "But the FDA was fundamentally against any changes."
When he endorsed the parallel track publicly anyway, it "created a bit of a brouhaha," he said. But the public response to his endorsement was so favorable that the government turned around and endorsed it as well.
Dr Fauci acknowledged there are still certain core scientific principles "that must remain sacrosanct and inviolate." However, he said, he has come to know when conventional wisdom needs to be challenged.
Another example of that came in the 1990s when a triple cocktail of drugs became available that increased the life expectancy of a 20-year-old infected with HIV from a few months to another 50 years.
Once again, conventional wisdom got it wrong and flexibility got it right.
Although that was great for the developed world, the drugs weren't getting to sub-Saharan Africa, which had 67% of the global cases of HIV, said Dr Fauci. Conventional wisdom said that with the state of the health delivery system there, doctors wouldn't be able to get people to take drugs every day for the rest of their lives.
Africans disagreed. In 2002, President George W. Bush sent Dr Fauci and a team on a fact-finding mission to determine the feasibility of a plan to effectively bring treatment and prevention to southern Africa.
There, Dr Fauci heard the same refrain: "Give us the opportunity and we will make it work."
When he returned, he and his colleagues presented what came to be called the President's Emergency Plan for AIDS Relief, or PEPFAR, which, over the past 13 years, "has prevented millions of infections and saved millions of lives," he reported.
The PEPFAR commitment is thought to be the largest by any nation to combat a single disease, and now provides treatment for 7.7 million people around the world.
"Once again, conventional wisdom got it wrong and flexibility got it right," Dr Fauci said.
He explained that his story is a message to others in medicine, science, and public health who will be questioned or confronted by people who don't agree with them. This will likely happen even more in the age of the internet, he warned.
Without violating scientific principles, Dr Fauci said, flexibility can alleviate suffering and save lives.
Dr Fauci has disclosed no relevant financial relationships.
TEDMED 2015. Presented November 19, 2015.
