By David H. Freedman
When it comes to some of the biggest diseases America faces in the future, our drug system is set up to fail. What needs to happen?
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.