Several prominent biotech and pharmaceutical executives are openly criticizing a huge price hike for a decades-old medicine and argue that the unexpectedly higher cost is due to the misuse of a federal law designed to encourage drug development for rare diseases. And the executives are using this to bolster a social compact they issued last month to address the debate over rising drug costs.
At issue is dehydrated alcohol, which hospitals and clinics have used for years to treat chronic pain or to prevent infections in patients who must receive nutrients intravenously. But after winning orphan designation and a seven-year monopoly to sell its injectable version for use with a specific heart procedure, Belcher Pharmaceutical began charging nearly $10,000 for a pack of 10 vials, up from $1,300.
As we recently reported, older versions sold by other manufacturers were never actually approved by the Food and Drug Administration, but had been available anyway on a grandfathered basis because they predated stricter requirements. The agency began requiring those companies to remove their versions by this year, since they did not seek regulatory approval.
This article erroneously describes the scope of exclusivity under the ODA. The ODA confers market exclusivity for a particular drug only for the rare disease indication. The exclusivity conferred to Belcher would not prevent other manufacturers from bringing dehydrated alcohol to market for other indications.
The problem is that the cost to submit an NDA is high even if clinical trials are unnecessary – this is an unfortunate byproduct of FDA’s ill-considered Unapproved Drugs Initiative.
For more, see here: https://www.ncbi.nlm.nih.gov/pubmed/28944731
Thanks for the note. Yes, you’re correct that the FDA unapproved drugs initiative is what prompted the agency to direct manufacturers of dehydrated alcohol to discontinue production, and that running new trials may be an undesirable undertaking.
It’s also true that another company could win FDA approval to sell a version of dehydrated alcohol before the 2025 exclusivity awarded to Belcher expires. However, that could only occur if another company wins approval for a different, but also very specific indication and is granted orphan designation. In such a case, this would require studies which, as you note, come with a high cost. In any event, I’ve updated the story to clarify and reflect that point.
Meanwhile, though, unless another company takes such a step, Belcher has its monopoly.
ed at pharmalot
Other companies could seek FDA approval for dehydrated alcohol for any indication other than the indication covered by Belcher’s orphan drug exclusivity. The other companies would not need to be granted orphan designation. The real culprit here is the Unapproved Drugs Initiative, which unnecessarily requires manufacturers to take their unapproved drugs off the market, even in cases such as this one where the product has an established record of safety and efficacy demonstrated by decades of use in medical practice.
Thanks for the give and take.
I’ve gone back to FDA to clarify and update previous conversations and yes, there are two issues here as noted. The combined one-two punch – the unapproved drug initiative and the orphan exclusivity – have created hurdles to competition and left Belcher with a wide open field to dominate, at least for now.
Anyway, I appreciate you reaching out and the conversation.
ed at pharmalot
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