GOV. CHARLIE BAKER’S proposal to rein in the cost of high-priced drug treatments got a cool reception on Wednesday at a MassBio policy breakfast, but most industry officials didn’t appear to have their own ready-made alternative.
In his fiscal 2020 budget proposal, Baker sought legislative permission to demand additional rebates from the manufacturers of very high-priced drugs – and if that effort fails to reach a satisfactory result to refer the manufacturers to the Health Policy Commission for hearings and possibly to Attorney General Maura Healey for prosecution under state consumer protection laws.
Patrick Plues, the vice president for state government affairs at BIO (the Biotechnology Innovation Organization), said during a panel discussion at the policy breakfast that Baker’s proposal was underwhelming.
“It needs some work, quite frankly,” he said. “It’s early in the budget process and we hope we can work with our allies in the Legislature to have a constructive conversation with the governor.”
About 30 lawmakers attended the jam-packed breakfast, signaling the broad support in the Legislature for an industry that is thriving in Cambridge’s Kendall Square and also in Worcester.
Kristin Wolff, director of global government affairs and public policy at bluebird bio of Cambridge, offered a possible solution to the pricing problem, at least for her company’s products. Bluebird is developing a gene therapy called LentiGlobin, which attempts to address an inherited blood disorder caused by a mutated gene. Patients with the disorder require regular transfusions to maintain their hemoglobin levels and stay alive. In clinical trials, LentiGlobin has shown success in eliminating the need for the transfusions.
Company officials have suggested the “intrinsic value” of LentiGlobin if it comes to market would be in the neighborhood of $2.1 million per treatment.
Wolff said the current health care system requires payment for treatments at the time they are delivered. She said bluebird is pushing the idea that payments be made when the benefits of the treatment accrue, perhaps over the course of five years.
“Why don’t we actually put our money where our mouth is, and say you don’t pay for therapy all at once,” Wolff said. “Maybe you only pay 20 percent in the beginning and then wait to see what the outcomes are. Are you transfusion independent at year one? If you are, great. Then there’s another installment payment. At year two, are you still transfusion independent? Then maybe there’s another installment payment. If you’re not, then maybe there is no installment payment.”
Tamar Thompson, executive director for state government affairs at Bristol-Myers Squibb, expressed interest in value-based payments, but she stressed that not all drugs lend themselves to the approach being pushed by bluebird.
“People should know an amortized or mortgage-type payment won’t work for all products because not everything is a curative one-time therapy,” she said.
Walter Whitt, a Harvard University student, said he was diagnosed with cystic fibrosis at birth and learned early on that he was not expected to live beyond 25 or 30. During middle school, he was constantly in and out of the hospital and began to come to grips with what it would be like living with a degenerative disease. “It was kind of a terrifying reality,” he said.
But new medications began to come on the market when he was in high school. He tried Orkambi initially and then shifted in college to Symdeco. Both drugs are manufactured by Vertex Pharmaceuticals of Boston. Orkambi costs $272,000 a year and Symdeko has a list price of $292,000 a year.
Some states are balking at the high cost, but Whitt said the therapies have changed his life. He coughs a lot less. He’s in the hospital infrequently.
“It was the first time I saw a life beyond 30 or 35 years,” he said. “I actually saw myself becoming an adult and being able to do things, kind of growing up, having a job, and almost being a normal person.”
