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In an interview with The Washington Post, U.S. Food and Drug Administration Commissioner Scott Gottlieb said the medical and scientific community had reached an "inflection" point in the development of innovative, new treatments to fight cancer, including the FDA's approval of CAR T-cell gene therapy last month.
"This was the first approval in the United States and it was the first approval of a product that looks to be highly effective. We've been working with gene therapy as a concept for many years, for decades and we've had a lot of false starts and some tragedies along the way and a lot of challenges," Gottlieb said in an interview with The Washington Post's Laurie McGinley. "I think we've finally reached an inflection point in science where we have the techniques sufficiently perfected to actually allow these products to be translated into therapies for humans."
The CAR T-cell therapy, a first-of-its-kind treatment that uses patients' own immune cells to fight cancer, comes at a high cost: $475,000. The drugmaker, Novartis, has issued a money-back guarantee if the patient does not respond within the first month of treatment.
"I think we're going to see more outcomes-based pricing for a lot of these treatments," he said. "Especially treatments where the response is very binary, where you can get a dramatic result or no result at all."
Gottlieb also acknowledged the high costs of new drug development.
"We can all agree it's really expensive," he said. "I think if it continues to remain very expensive and it continues to grow at the rate that it is - at a rate that by many measures is an excess of even the significant rates of increases in the prices - we're going to see fewer drugs come into these categories and less competition. And that certainly will impact prices."
Gottlieb, who was appointed by President Donald Trump, is a physician and a cancer survivor who has long pushed for deregulation of the FDA to speed up the review and approval of new therapies.
"I think where we can have an impact is on a development timeline," he said, adding "There's different ways we can incorporate new technology, new methodologies to make that process more efficient."
During the interview, which was part of The Washington Post's "Chasing Cancer" summit, Gottlieb also discussed the FDA's new initiative to reduce the amount of nicotine in cigarettes to minimally addictive or non-addictive levels.
The goal: "So we don't hook a whole new generation of smokers onto cigarettes, and so that people who want to get access to nicotine look to other, potentially less harmful products than tobacco, than the combustible cigarettes."
Gottlieb said his approach to the top job at the FDA grew out of successfully beating Hodgkin Lymphoma at age 33. Gottlieb has been a supporter of adaptive clinical trials, drug trials that can be modified based on patient response, and the 21st Century Cures Act, which speeds the drug approval process.
"I think it's affected how I think about my job in so far as, for me, I was a patient who was told you have a 90 percent chance of a cure and I was trying to figure out, how do I get 90 percent to 92 percent and 93 percent?" he said. "The way to do that was to scour the literature and look for the most decisive studies I could find about how to structure my regimen."
For patients with tougher odds, he said: "What that patient is looking for is something new, they're looking for a silver bullet, they're looking for something experimental."
Other highlights from The Washington Post's "Chasing Cancer" summit:
• Tom Whitehead of the Emily Whitehead Foundation shared the emotional story of his daughter, Emily's, cancer diagnosis. Emily Whitehead joined the panel, which included Children's Hospital of Philadelphia Attending Physician Shannon Maude and National Cancer Institute Associate Research Physician Nirali Shah, to talk about what it was like to be the first pediatric recipient of experimental gene therapy for cancer.
• National Cancer Institute Acting Director Douglas Lowy talked about the Cancer Moonshot, including how long-term, bipartisan political support is important for greater advances in clinical trials.
• Top medical experts in the field, including American Cancer Society Chief Medical Officer Otis Brawley, MIT Professor Regina Barzilay and Color Genomics' Chief Medical Officer Jill Hagenkord, discussed what can be achieved in the year ahead in cancer detection and treatment.