Just one week after Vermont Sen. Bernie Sanders sent a scathing letter to Catalyst Pharmaceuticals CEO questioning his plans to charge $375,000 for its rare-disease drug Firdapse, the company said it's sticking to that number.
That will indeed be the drug’s list price, Catalyst confirmed at the BIO CEO & Investor Conference in New York yesterday, and it should work out to a net price of at least $300,000 for the drug, approved by the FDA in November.
Sanders’ outrage about the list price of Firdapse, used to treat the neuromuscular disorder Lambert-Eaton myasthenic syndrome (LEMS), is that until now, patients have had access to an unapproved version of the drug free of charge through the FDA’s compassionate use program. After a two-year battle for approval, Catalyst got the nod for Firdapse in November and shortly thereafter set the list price of $375,000.
In his letter (PDF), Sanders said that price “is not only a blatant fleecing of American taxpayers, but is also an immoral exploitation of patients who need this medication. Simply put, it is corporate greed.”
Catalyst said in a statement emailed to FiercePharma that it would respond to Sanders’ letter “in a timely manner,” and that it will provide information about its plans to facilitate access to Firdapse. “Catalyst’s top priority is improving patient care in the LEMS community and potentially elsewhere within the neuromuscular community.”
During its BIO presentation on Monday, Catalyst said it would offer copay support and patient assistance programs for uninsured patients, and that independent charities provide additional support for LEMS patients. Catalyst also has a bridge program that provides the drug at no charge for patients transitioning from the unapproved drug or waiting for their health plans to verify coverage.
But the company’s largesse is falling on deaf ears, and not just those of Sanders and other legislators who have been threatening to pass legislation to control drug prices. Pharmacy benefits manager Express Scripts is also speaking out, even though it has already put the drug on its formulary for patients who qualify.
“Our first priority is to ensure access to medicines for people who need them, even drugs that may be egregiously priced as this one is,” an Express Scripts spokeswoman said in a statement emailed to FiercePharma. “The pricing strategy for this medication takes advantage of vulnerable patients who need this medication and the plans who help pay for it. This is an example of how some companies misuse the Orphan Drug Act, and we have been vocal in our support of amending this legislation to prevent such situations.”
Amending the Orphan Drug Act is one strategy that federal legislators have proposed. In 2017, three Republican senators asked the U.S. Government Accountability Office to review orphan drug laws after Marathon Pharmaceuticals slapped an $89,000 price tag on the old Duchenne muscular dystrophy drug Emflaza.
More recently, federal lawmakers have laid out several other strategies for lowering drug prices. President Donald Trump proposed a rule that would force pharma companies to disclose drug prices in direct-to-consumer TV ads, for example. Just last week, Johnson & Johnson decided to get ahead of that proposal before it passes, becoming the first drug company to announce a plan to put list prices in TV ads.
But many of the proposals out of Washington go well beyond demanding transparency and instead get to the nitty gritty of how drugs are priced and reimbursed. In late January, for example, Senators Bill Cassidy, M.D., R-La., and Mark Warner, D-Va., introduced a bill that would allow payers to link reimbursement of pricey treatments to how patients respond to them. Meanwhile, HHS Secretary Alex Azar is on a tear to overhaul the rebates that drug makers award to pharmacy benefit managers. And in January, Sanders and more than two dozen of his colleagues in Congress introduced three bills that they believe would slash drug prices in half.
That was only a few weeks before Sanders dashed off his note to Catalyst CEO McEnany demanding that the company justify Firdapse’s price. Sanders listed 16 questions for the CEO, requesting details about how much Catalyst is paying for the drug’s active ingredients, the price it plans to charge for it overseas, and more. The company has until Feb. 18 to respond to Sanders.