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Regulatory Focus™ > News Articles > 2019 > 2 > Sanders Questions $375,000 Price Tag for Previously Free Drug

Sanders Questions $375,000 Price Tag for Previously Free Drug

Posted 04 February 2019 | By Michael Mezher 

Sanders Questions $375,000 Price Tag for Previously Free Drug

Amid hearings on prescription drug pricing in both the US Senate and House of Representatives, Sen. Bernie Sanders (I-VT) on Monday sent a letter to Catalyst Pharmaceuticals requesting the company explain the $375,000 per year list price for its recently approved drug Firdapse (amifampridine).
Firdapse is the first drug approved to treat Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder that affects patients' muscles and nerves.
"Catalyst's decision to set the annual list price at $375,000 is not only a blatant fleecing of American taxpayers, but is also an immoral exploitation of patients who need this medication," Sanders writes, noting that the estimated cost to manufacture the drug is $1,600-6,000 per year.
In 2012 Catalyst licensed the North American rights to Firdapse from BioMarin for an undisclosed amount in a deal to commercialize the drug.
Catalyst first sought US Food and Drug Administration (FDA) approval for the drug in 2015 but received a refuse to file (RTF) letter from the agency, after conducting additional clinical studies, Catalyst resubmitted the application in March 2018.
In November, FDA announced it had approved Firdapse based on data from two small placebo-controlled clinical trials that included a total of 64 adult patients. The drug also received a priority review and was granted breakthrough and orphan designation.
At the time, Billy Dunn, director of the Division of Neurology Products at FDA's Center for Drug Evaluation and Research said, "there has been a long-standing need for a treatment for this rare disorder," underscoring the fact that Firdapse was the first drug approved by FDA to treat LEMS.
Aside from the sticker shock over the six-figure price tag, much of the controversy over the price of Firdapse stems from the fact that the drug had been available to patients for free through expanded access programs for the last two decades.
But now that the drug has been approved, Catalyst said the expanded access programs will shut down by the end of March.
Catalyst CEO Pat McEnany explained the company's approach to pricing Firdapse during a December 2018 conference call with investors, saying that "our pricing strategy reflects our focus on ensuring broad and sustainable coverage from both private and public payers and assistance for patients in need. We have also factored in continued clinical investment in the product to evaluate it for other neuromuscular diseases as well as future development of a new sustained-release formulation."
However, Sanders is requesting details from the company about how it came to its $375,000 per year list price as well as details about how much public payers are anticipated to spend on the drug. Additionally, Sanders is asking the company for more information on how the drug will be covered by private insurers as well as how the company's patient access programs will work.
Catalyst currently has a dedicated website detailing its patient assistance offerings for Firdapse, which include providing resources for dealing with healthcare providers and insurers, as well as offering financial assistance via a copay card program for eligible patients.
The company has also said that patients that do not have health coverage and do not qualify for charity assistance programs will be provided the drug for free.
Sanders Statement, Letter


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