https://www.wsj.com/articles/when-medical-innovation-meets-politics-1535147861
An Iowa teenager last summer found himself conscripted into a national debate over health care. The state’s largest insurer, Wellmark Blue Cross and Blue Shield, was threatening to pull out of the ObamaCare market. One reason, a Wellmark executive said, was a single patient whose care cost $1 million a month. The young man (never publicly named) has hemophilia, which prevents his blood from clotting. The standard treatment, infusions of the missing clotting factor, is expensive. In severe cases, a patient can require several infusions a day.
What if there were a cure? Researchers are developing therapies that could permanently alter a patient’s genes, allowing his body to produce the clotting element. The early results are promising. Patients would live longer, better lives. And with thousands of hemophilia cases nationwide, the potential savings for insurers—and for Medicaid—are enormous. But when?
Part of the answer depends on the Food and Drug Administration, where hundreds of applications involving gene therapy for various diseases are pending. At the helm is Commissioner Scott Gottlieb, who operates without much publicity but may have the most consequential job in Washington given the furious pace of scientific change and his potential for long-term impact. For years Dr. Gottlieb, 46, wrote forcefully, often in these pages, about how to reform the FDA, including by overcoming an “excessive desire for certainty” that delays treatments.
That’s a countercultural sentiment at the FDA, where Dr. Gottlieb, who has also practiced internal medicine, was a deputy commissioner from 2005-07. Now he’s running the agency during a historic time for medical innovation.