Another week, another U.S. medical breakthrough. The latest in the annals of American innovation is a new cystic-fibrosis gene therapy that will help patients with the debilitating disease live comfortably for decades longer. Remember this the next time politicians, on the left or right, complain about greedy drug companies.
Cystic fibrosis is caused by a genetic defect that results in excess mucus production in the lungs, digestive tract and other parts of the body. The disease affects about 30,000 Americans and can lead to respiratory infections and many other complications. Decades ago most sufferers were lucky to live into their 20s.
a Boston-based biotech company, has spent the last decade developing treatments that target the disease’s genetic culprits. Its first cystic-fibrosis drug in 2012 helped improve symptoms for a small number of patients. Vertex developed two follow-on therapies that brought more benefits to more patients.
Its fourth therapy called Trikafta, which was approved by the Food and Drug Administration last week, has the potential to greatly improve the lives for 90% of patients. The three-drug cocktail works by fixing the defective proteins that result from a genetic mutation. This week the New England Journal of Medicine published results from a Trikafta trial, which have led doctors to speculate that younger patients given the drug may be able to live more or less normally into old age.
The drug won’t be cheap. It will initially cost $311,000 per year, which is about the same as Vertex’s earlier therapies when they launched. Gene therapies can cost more to produce but often don’t treat as many people as blockbuster drugs so prices will naturally be higher. Life-saving innovations are never free, and patients will be getting literally years of extra life for the dollar.
Sometimes the prices of these therapies will come down with competition, as we’ve seen with Hepatitis C drugs. But in other cases drug makers, insurers and government will have to make accommodations that allow access to these life-savers without depressing the incentive to develop them.
The worst idea, but the popular one of the moment, is to impose government price controls. House Speaker
the Senate Finance Committee and
all have variations on this theme. But the House left may save us for now by demanding more than Republicans will concede. Some liberals, including
have also proposed stripping patents from expensive drugs—a form of ex post facto government confiscation of property rights.
This ignores that drug makers have to finance new treatments with the profits they make on current drugs. But as with Trikafta, the benefits in new discovery are incalculable. Consider the new Ebola treatment (REGN-EB3) by
that helped control a viral outbreak this summer in the Democratic Republic of Congo.
U.S. drug investment and innovation encouraged by market incentives are saving lives around the world, and the prospects are even more promising as biotech discoveries multiply. The rule for politicians, as for doctors, should be, first, do no harm.
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