A new group of activist doctors has joined the drug pricing protests. This time, it's neuromuscular specialists, and they're targeting a forthcoming drug, Firdapse, that's likely to turn treatment of a rare disease from a no-cost endeavor to an expensive affair.
Catalyst Pharmaceuticals asked the FDA to approve the med earlier this year, and it's counting on Firdapse to pump up its sales by $300 million to $900 million--a revenue haul that implies a price of up to $100,000 per patient, The New York Times reports.
Thing is, Firdapse is a slightly tweaked version of an old drug--3,4-DAP--that one U.S. drugmaker Jacobus Pharmaceutical is giving away for free. The drug has been used to treat Lambert-Eaton myasthenic syndrome and congenital myasthenic syndromes for 30 years. But the FDA gave Firdapse orphan drug status, which means it's entitled to 7 years of market exclusivity if approved.
Firdapse's development and Catalyst's plans for premium pricing have been stirring controversy in biotech circles for months. But now, in an article published in Muscle & Nerve, a group of 80 physicians say they're worried about "a potentially harmful price increase of 3,4-DAP" if and when Catalyst wins its FDA approval.
As the doctors point out, Catalyst is using a "loophole" in the orphan drug law that allows older, existing drugs to win the designation and its exclusivity benefits. Catalyst snapped up U.S. rights to Firdapse--sold by BioMarin Pharmaceuticals ($BMRN) in Europe--took over a clinical trial that was already underway and unveiled positive data from that trial last fall.
"We recognize that pricing of some FDA-approved drugs under the ODA may be justified by the substantial investor risk and research and development costs incurred by pharmaceutical companies in preclinical and clinical development," the doctors note in their article. "However, 3,4-DAP is not a newly discovered molecule, nor is it expensive to manufacture; thus, it differs dramatically from some innovative drugs now in or coming to the market. It has been reported to be effective and safe since 1983."
So, the doctors say, Congress should close that loophole in the Orphan Drug Act and thwart companies like Catalyst that plan to charge premium prices for long-used meds, after spending minimal time, money and effort on readying them for approval.
At least one lawmaker closely involved in the law sees the need for changes. "The Orphan Drug Act has been turned on its head in recent years," Henry A. Waxman, the former Democratic congressman who sponsored the ODA, told the Times. "It has created a special status for orphan diseases that offer large potentials for making generous profits."
In the absence of legislative changes, "we publicly implore ethical and just pricing of 3,4-DAP," the doctors write--and urge the FDA to consider approving both Firdapse and Jacobus' version of the drug when it comes up for review.
As the NYT reports, Catalyst says it hasn't yet chosen a price for the drug. It defends its development work on the med, saying its formulation doesn't need refrigeration, whereas the older version does.
But doctors and scientists who've studied Firdapse say it's "still basically the same drug," and the raw material for it "was a simple chemical."
The doctors who wrote the Muscle & Nerve article want an FDA-approved drug. Obtaining Jacobus' product under a special FDA program requires reams of paperwork for each patient, and that can be a deterrent to treatment. But they also want their patients to be able to afford an FDA-approved med--and with co-insurance on drugs like Firdapse running at 20%, patients could be on the hook for $20,000 a year.
These doctors aren't the only ones up in arms over high prices for drugs. One oncologist at MD Anderson Cancer Clinic in Houston took to social media to protest 6-digit price tags for blood cancer meds, and Memorial Sloan Kettering physicians have written op-ed articles, formulated drug-cost calculators online--and even blocked use of certain pricey meds at their hospital.
And Catalyst is facing a very different pricing climate now than it might have months ago when it unveiled its sales estimates for investors. Since then, we've seen the Turing Pharmaceutical price-hike debacle and an ensuing outcry over big prices on older meds. Under the public eye, Catalyst may not be as bold with its Firdapse price tag.
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