RACHEL MARTIN, HOST:
New, astronomically expensive drugs are expected on the market in the next few years. And they could drive up the cost of medicine and health insurance for everyone. NPR's Richard Harris reports.
RICHARD HARRIS, BYLINE: These new treatments are gene therapies, which target certain cancers and rare diseases. Take, for example, hemophilia, an inherited disorder that prevents a person's blood from clotting properly. Mark Skinner is one of about 20,000 Americans with the condition.
MARK SKINNER: So when I was born, treatment did not exist for hemophilia. So within my lifetime - I'm 58 years old - there have been remarkable advances.
HARRIS: He now gives himself daily injections of expensive medication to prevent the painful and potentially dangerous bleeding episodes. Skinner says eight or nine companies are now working on treatments that could actually cure his underlying condition.
SKINNER: I think we could see a gene therapy in the clinics and available to patients within a couple years.
HARRIS: One infusion might be enough to correct the genetic flaw and give him many years - maybe even a lifetime - free from bleeding episodes and daily medication - drugs that often cost hundreds of thousands of dollars per year.
SKINNER: And so a gene therapy that costs a million dollars or even $2 million, you could see it becoming economically viable over a couple years in terms of return of investment for a health system and certainly significant savings over a lifetime.
HARRIS: But it could also be a big shock to the system. Gene therapy for every American with hemophilia could cost tens of billions of dollars. Mark Trusheim at MIT says a few years ago, insurance companies balked at the new, high-priced drugs to cure hepatitis C and ended up rationing treatment. Would that happen for hemophilia?
MARK TRUSHEIM: If a gene therapy came through and was reasonably expensive, it would be terrible if we could only treat a thousand patients a year and it took us 10 or 15 years to treat all those people.
HARRIS: Trusheim's effort at MIT, called the NEWDIGS program, is trying to dream up better ways to pay for expensive, new, long-term treatments. We're used to paying as we go for drugs, with monthly bills for monthly pills. Trusheim says it's like paying rent on an apartment.
TRUSHEIM: But now, with these new gene therapies, we get to take the treatment once. And it can last for years - perhaps a lifetime, perhaps 10 years, but certainly for many years.
HARRIS: He says it makes more sense, then, to think of these drugs in terms of paying a mortgage over time for a condo rather than paying monthly rent.
TRUSHEIM: Plus, we have the option here not just to turn it into a mortgage payment, but make that mortgage payment contingent on whether the apartment's really a good apartment or not. If the roof begins to leak, maybe we won't pay so much on the future payments.
HARRIS: This strategy has already been put to use for one gene therapy which treats an inherited form of blindness at a cost of $850,000. The company that produces that has agreed to give health insurers some money back if a patient's vision doesn't improve enough.
Trusheim is eager to get funding schemes like this instituted now because he expects to see another three dozen new expensive therapies coming online in just the next four years. Those include treatments called CAR-T for cancer and gene therapies for diseases that, up till now, have had no treatment at all.
TRUSHEIM: It could be really exciting. But it could also be really expensive as we're treating conditions we never used to be able to treat.
HARRIS: The technology seems poised to take off, with hundreds of potential treatments in the pipeline - good for health, tough on the nation's health care budget. At a recent conference in Washington, D.C., Joe Grogan from the White House Office of Management and Budget warned that it could push health care spending over the edge.
JOE GROGAN: If we get a cure for Alzheimer's priced at a hundred thousand dollars or a million dollars a pop, we're toast.
HARRIS: That could add trillions to the cost of health care. Even if politicians step in to control drug prices, these products cost a lot to develop. So they will still be expensive. Mark Skinner says we will need creative ways to manage that.
SKINNER: At the pace at which gene therapy's coming, I think we have no choice. I don't think the public would tolerate cures sitting on the shelf that people don't have access to.
HARRIS: A solution will require not only new ways of thinking but new rules and regulations that govern how insurance companies and government programs pay for medical care. Richard Harris, NPR News.
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