A Food and Drug Administration advisory panel's recommendation to pull an early birth drug from the market is only the latest controversy surrounding a popular program aimed at getting promising new treatments to patients faster.
Why it matters: Critics — including the FDA itself — say the program lacks the appropriate guardrails, putting expensive, unproven treatments in consumers' hands while drugmakers often delay proving the drugs' effectiveness.
Driving the news: Wednesday's recommendation that the FDA pull Makena, a drug owned by Covis Pharmaceuticals that is marketed as preventing pre-term births, came after years of tension between the company, the agency, consumer protection groups and medical associations.
- Makena received what's called an accelerated approval from the agency in 2011, allowing patients to access it and opening the door to insurance coverage. The news was particularly welcomed by Black patients, who are more likely to have pre-term births.
- Accelerated approvals are granted on early data that suggest a product is effective, but require drugmakers to perform a confirmatory trial.
- In 2019, Makena's confirmatory trial found that the drug has no clinical benefit. The FDA in 2020 proposed removing the drug from the market, but the manufacturer resisted. Covis argued the confirmatory trial was poorly designed and didn't capture the drug's benefit.
- "All of these issues are not unique to Makena, but the manufacturer here has taken them to greater lengths than other manufacturers," said Rachel Sachs, a Washington University law professor.
The big picture: Accelerated approval began drawing policymakers' attention after it was used last year to approve Aduhelm, a controversial Alzheimer's drug that an FDA advisory panel had recommended the agency not approve. The manufacturer Biogen later largely gave up marketing the compound.
- The more routine issue with the program is that drugmakers frequently miss their deadlines for completing confirmatory trials, yet their products remain on the market.
- Both the House and the Senate have since proposed reforms to the program, and the FDA has asked specifically for more authority to ensure confirmatory studies "progress in a timely manner."
- However, lawmakers punted on any legislative reforms when they debated a must-pass FDA bill last month. The issue could resurface during a post-election lame duck session.
By the numbers: A recent report by HHS's internal watchdog found that nearly 40% of the 278 drug applications granted accelerated approvals from 1992 to the end of 2021 haven't completed their confirmatory trials.
- Many of those trials are ongoing and haven't missed any deadlines. But about a third of drug applications with incomplete confirmatory trials are past their planned completion dates.
- Medicare and Medicaid spent more than $18 billion on those drugs from 2018 to 2021, according to the report. It estimated that Medicaid spent nearly $700 million on Makena alone.
Between the lines: The recent congressional debate largely ignored questions around whether payers — particularly Medicare and Medicaid — should have to pay full price for unproven drugs.
- MACPAC, a Medicaid advisory board, has recommended that manufacturers of drugs that receive accelerated approval should pay higher rebates to the program until they are fully approved. The National Association of Medicaid Directors supports this recommendation.
- MedPAC, its Medicare equivalent, has also discussed capping a drug's payment rate until effectiveness is confirmed.
What we're watching: The FDA will now officially decide whether to pull Makena from the market, likely ending this particular saga.
- The question then becomes whether Congress will enact any reforms to the program after the midterm elections, and how far those reforms will go.
