Having been around the block more than a few times, the position of the FDA regarding pricing is clear. The Agency is not supposed to take pricing considerations into drug approval or enforcement decisions. However, there is controversy swirling around the FDA’s recent decision on the drug shortage for GPL-1 obesity medications and the practice of compounding during drug shortages. The FDA made a bit of a flip-flop as described in two previous posts, here and here.
Scott Gottlieb, M.D., former FDA commissioner, penned an opinion piece in Stat (here) describing the dangers of some compounding of injectable products from bulk pharmaceutical active ingredients, such as the New England compounding debacle “where contaminated compounded drugs caused a meningitis outbreak, killing more than 100 patients and sickening 800.”
However, he also noted that the FDA’s recent flip-flop regarding its decision to declare that the shortage of GLP-1 products is over appears to be clearly related to and based on the cost savings realized by patients using products compounded by pharmacies and outsourcing facilities. He discussed his opinion that the FDA’s policy on compounding, which is driven by regulations and the statute regarding compounded drug products that are duplicates of already approved products, has been clouded by the decision to permit continued compounding in certain situations. As he pointed out, in the Makena case, “[b]y permitting compounders to compete with Makena rather than enforcing its exclusivity, the FDA sent a clear signal: The agency was opening the door to competition by compounding pharmacies as a rebuke to a branded drug’s steep price.” We posted blogs detailing and explaining the issues with Makena here, here, here, and here.
Shortage or not, this same cost issue has surfaced over and over again with the FDA, especially when the Agency has delayed taking enforcement action on unapproved, marketed drug products after the first NDA is approved for a drug. Other examples include colchicine, where the FDA delayed enforcement action for over two years until after NDA approval of the previously unapproved versions of the “grandfathered” drug. The first FDA-approved colchicine drug, marketed as Colcrys, became significantly more expensive for patients than older, unapproved, marketed generic versions once it gained exclusive market access, leading to concerns about affordability for many gout sufferers; this price increase was attributed to the company conducting clinical trials and gaining market exclusivity for its newly approved formulation of colchicine. Colcrys was sold for approximately $5 per tablet, a substantial increase over the average of $0.09 per tablet for which the unapproved version of prescription colchicine was sold by numerous manufacturers. Price complaints by consumers of the new product put the FDA in the crosshairs and resulted in delayed enforcement action on the marketers of the unapproved drug. Enforcement did not occur until the firm that had received the first approval developed a plan to ensure that anyone who needed the product could afford it through a patient assistance program. Once that occurred, the FDA demanded that other, unapproved versions of the drug product were removed from the market.
Another example of delayed enforcement action occurred after the first NDA approval of quinine sulfate. As in the colchicine example, the FDA action to remove unapproved versions of the drug was significantly delayed, primarily because patients complained that the drug, which previously cost pennies, was unaffordable. See here for the Federal Register notice announcing enforcement action against unapproved quinine marketers. In this case, the first product approval was for malaria and it was subject to a seven-year period of orphan drug exclusivity.
So, while according to the FDA, the Agency does not use cost as a basis for its approval or enforcement decisions, the actions that the FDA takes are not always consistent with its stated position; rather, it clearly takes into consideration how its actions may create inaccessibility to lifesaving drugs.
