America faces explosive growth in chronic disease, but our drug system is set up to fail in fighting it. What needs to happen?
From my article in December 2017 in Politico
If there’s a dream of what a new drug is supposed to do, it might look something like Kalydeco. In 2012, the new light-blue pill from Vertex Pharmaceuticals rocked the world of cystic fibrosis, a fatal disease that affects 30,000 people in the United State. It’s best known for its attack on the lungs, slowly suffocating its victims while attacking other organs—but when patients got the drug in its experimental phase, some started reporting such enormous improvement in their breathing and energy they were able to take up running, even marathoning.
Kalydeco is emblematic of the promise of new approaches in drug development. Built on a new understanding of how a particular defect in a gene can disrupt the workings of the body, the drug zeroes in on critical proteins inside cells to keep them functioning. “The drug was so good it broke the blind,” says Bernard Munos, a senior fellow at FasterCures, a think tank based at the Milken Institute—meaning the positive trial results were so clear that patients and doctors could easily tell who was receiving the drug and who got a placebo.
At the same time, Kalydeco serves as a cautionary tale. “The results for the patients it helps are absolutely spectacular, but those patients are only a sliver of the population with the disease,” says Munos. If you haven’t heard of Kalydeco—and you probably haven’t—that’s because this drug, which costs the U.S. health care system nearly half a billion dollars per year, currently helps fewer than 2,000 American patients.
The drug-development system that produced Kalydeco is one of proudest achievements of American medicine, and one of our biggest investments as a society. When American leaders talk about “innovation” in health care, they’re largely talking about the development of new pharmaceuticals. But as America looks squarely at the biggest health challenges of the future, there’s reason to worry that the system we’ve built may not be adequate to what’s in front of us. When it comes to the two diseases likely to become our biggest killers— Alzheimer’s and diabetes—death rates are relentlessly ticking up, with few solutions on the horizon. Read more