FDA head calls approval of the first U.S. cancer gene therapy a significant milestone

In an interview with The Washington Post on Monday, 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 moneyback guarantee if the patient does not respond within the first month of treatment.

Gottlieb 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.”