Would-be makers of rare disease meds, rejoice: A 6-month exclusivity extension, proposed last year in Congress, has returned. The 21st Century Cures Act, now under consideration in the House of Representatives, once again includes a measure that would prolong competition-free marketing for some meds repurposed to treat rare diseases.
As Regulatory Focus reports, the provision would operate similarly to the pediatric exclusivity bonus drugmakers can now win. When pharma companies conduct studies specifically in children and adolescents, they can net an extra 6 months of exclusive access to their markets.
Under the new proposal, the FDA could grant that same boon to companies that turn meds approved for broader populations into treatments for rare diseases. To win the extra 6 months, a company would have to a., study one of its nonorphan meds for a rare disorder, and b., win agency approval in that indication.
Of course, 6 extra months of brand exclusivity can be a lucrative proposition. It can be particularly lucrative if a drug carries a high price tag to begin with, of course. Orphan meds come with sky-high prices--the highest in the market--because they treat such small numbers of patients, relatively speaking. As RF notes, the orphan designation applies to indications covering 200,000 or fewer patients.
Question is whether a company could command orphan-level pricing for a med that's already on the market for a more typical use, and at a lower price. Companies have managed to switch lower-priced drugs to new indications at higher prices before; Sanofi's ($SNY) Genzyme unit, for instance, yanked the leukemia drug Campath off the market as the company prepared to launch a new formula for multiple sclerosis. The new iteration, Lemtrada, would be dosed at much lower levels in MS than in cancer, but Sanofi wanted to continue charging a close-to-$60,000 price.
- see the RF article
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