UPDATED October 4, 2022 // Editor's note: This article has been updated with additional comments.
The Medicare and Medicaid programs spent more than $18 billion over 3 years on medications for which there was no proof of a significant clinical benefit, a new federal report shows.
On Tuesday, the Office of the Inspector General (OIG) for the Department of Health and Human Services released an initial take on its ongoing evaluation of medicines cleared through the US Food and Drug Administration's (FDA's) accelerated approval process.
As reported by Medscape Medical News, last year, amid controversy generated by the FDA's clearance of the controversial Alzheimer's drug aducanumab (Aduhelm), the OIG announced its plan to take an in-depth look at how the administration manages its accelerated approval pathway. The goal of this process is to speed the approval of promising medications for serious and fatal diseases even though evidence of efficacy is limited.
The FDA's expectation is that drug companies will continue research in order to definitively prove the efficacy of medications approved via this process.
"However, for a variety of reasons, sponsors do not always complete trials promptly. This can result in drugs staying on the market ― and being administered to patients ― for years without the predicted clinical benefit being verified and insurers ― including Medicare and Medicaid ― paying billions for treatments that are not verified to have clinical benefit," the OIG said in its report.
In granting accelerated approvals, the FDA sets timelines for drugmakers to carry out confirmatory trials. But these target dates are often missed. OIG staff evaluated 104 cases of accelerated approvals for which confirmatory trials have not been completed and found that as of May 5, 2022, 35 drug studies had missed their original target completion dates.
In its report, the OIG staff offered a deep look at the four drugs in which confirmatory trials were furthest past their original completion deadlines:
Mafenide acetate (Sulfamylon), a topical antimicrobial treatment for burns that was approved in 1998. The trial is 140 months past its original deadline.
Midodrine hydrochloride (Proamatine), a treatment for postural hypotension that was approved in 1996. The trial is 85 months past its original deadline.
Makena cost Medicaid programs almost $700 million from 2018 to 2021, the OIG said.
In 2020, the FDA started the process of withdrawing the application for Makena's accelerated approval after a trial failed to demonstrate a clinical benefit. Covis Pharma, the maker of Makena, has been contesting this demand. A public review of the data on Makena is slated for October.
At a hearing, the FDA's Obstetrics, Reproductive and Urologic Drugs Advisory Committee will review the data while an appropriate confirmatory study is designed and conducted.
The OIG released its report as members of Congress dropped plans to address the accelerated approval process in FDA legislation.
In June, the House passed a bill to reauthorize the Prescription Drug User Fee Act (PDUFA), which included new authority for the FDA to better ensure swift completion of confirmatory trials.
However, these provisions were dropped amid protest by Sen. Richard Burr (R-NC). Congress is expected to clear a PDUFA reauthorization that strips the FDA's authority to ensure completion of trials for drugs cleared via the accelerated approval process.
This PDUFA reauthorization will be bundled with a stopgap budget bill, known as a continuing resolution. On Thursday, the Senate voted 72-25 on this package. The House is expected to clear it quickly.
Reshma Ramachandran, MD, MPP, MHS, of Yale University in New Haven Connecticut, who serves as the chair of the FDA Task Force of the group, Doctors for America, tweeted that it was "Truly ironic that the same day when @OIGatHHS releases report on concerns raised from delays in confirmatory trials for @US_FDA accelerated approval drugs is the same day when Congress votes on a user fee package stripped of accelerated approval reforms."
In a statement, the drugmaker's trade group, the Pharmaceutical Research and Manufacturers of America (PhRMA), said its members try to hold up their end of agreements for further studies of drugs granted accelerated approvals.
Confirmatory trials sometimes take longer than expected owing to several factors, including the inability to enroll patients as quickly as anticipated because of patients enrolling in other studies aimed at the same population, patients being less willing to volunteer for studies of FDA-approved medicines, or small patient populations, Andrew Powaleny, a PhRMA spokesman, told Medscape Medical News.
"Our industry is committed to meeting the requirements for converting accelerated approval to traditional approval and the FDA already has procedures in place to remove products should confirmatory trials not show that the drug provides clinical benefit," Powaleny wrote.
Kerry Dooley Young is a freelance journalist based in Miami Beach. She earlier covered health policy and the federal budget for Congressional Quarterly/CQ Roll Call and the pharmaceutical industry and the US Food and Drug Administration for Bloomberg. Follow her on Twitter @kdooleyyoung.
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Cite this: CMS Spent $18 Billion on Drugs Without Confirmed Benefits, OIG Report Finds - Medscape - Sep 30, 2022.