Last year, Marathon Pharmaceuticals took an inexpensive steroid available for sale in the EU and Canada and brought it through the US FDA approval process before trumpeting an $89,000 annual price.
The move, criticized by lawmakers and others, offered a test case for how a company can use FDA’s approval system and the US pricing system to reap outsized rewards without doing any of the development work.
Now, according to a new preprint from pharmaceutical consultants at the firm Pharmagellan, another company called Acer Therapeutics, chaired by the current CEO of Martin Shkreli’s old company Retrophin, acquired celiprolol, a cheap beta blocker long available outside the US but never submitted for FDA approval. Acer has said it will seek FDA approval for the drug this year.
“If Acer launches Edsivo [celiprolol] at a high US price (a logical bet), this will be a reprise of last year’s Emflaza to-do,” Pharmagellan managing director Frank David said. “Acer bought the data rights and obtained orphan designation, but has not conducted further trials as far as we can tell.”
An Acer executive told Focus via email: “No comment.”
What Can be Done?
Looking to advance the discussion on how to pre-emptively identify and block such behavior, David and Richa Dixit said “alterations to FDA’s existing rules on personal importation” may be one solution.
“Refining these criteria to allow Americans to import generics in certain situations where a drug with the same active ingredient is already available in the US – for example, if the generic was launched abroad before the first FDA approval – would permit continued importation of drugs like deflazacort and celiprolol, and thus eliminate the incentive for future companies to take a similar approach,” they write.
And though FDA has been resistant in the past to altering importation or re-importation policies out of supply chain safety concerns, David told Focus he thinks these are unique situations.
“We already allow patients to import drugs unavailable in the US for personal use (although I reckon this is cumbersome, and it’s obviously not covered by payers),” David said via email. “So in principle, FDA already acknowledges it’s appropriate to have a pathway for patients to access drugs from abroad that aren’t approved here, and accepts the tradeoffs. The issue with celiprolol and deflazacort is that under our current policies, the ability to personally import halts when/if the drug is approved by FDA. That’s certainly appropriate in the case of on-patent meds, but here we’re talking about drugs that have already gone generic.”
In addition, drug pricing scandals tend to get lumped together. But the Emflaza example should not be misconstrued as the same as say, Shkreli’s old company Turing Pharmaceuticals acquiring Daraprim (pyrimethamine) and jacking up the price over night.
“Here, we focused on a narrow subset, epitomized by Emflaza: old, cheap drugs only available in the EU that are then brought to the US by third parties and get the pricing and patent benefits of ‘innovator’ drugs. These aren’t going to be common – we only found one looming example in our work! – but they’re unique compared with daraprim, etc., and solutions to those latter situations aren’t going to prevent future Emflaza-like scenarios,” David said.
First published here. Regulatory Focus is the flagship online publication of the Regulatory Affairs Professionals Society (RAPS), the largest global organization of and for those involved with the regulation of healthcare and related products, including medical devices, pharmaceuticals, biologics and nutritional products. Email email@example.com for more information.
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