The Medicare program spent $ 1.8 billion in 2019 on drugs without proven clinical benefits

In 2019, the U.S. federal government spent about $ 1.8 billion on Medicare funds for drugs whose clinical benefits have not yet been confirmed by the Food and Drug Administration, according to a new study led by researchers from the Johns Hopkins Bloomberg School of Public Health.

The FDA approves about 50 new drugs each year thereafter clinical trials and data analysis demonstrates the safety and efficacy of the drug. To expedite the receipt of potentially beneficial new drugs for critically ill patients, some of these new drugs have been approved through accelerated FDA approval. The program provides drug approval based on what is known as a surrogate endpoint. This usually includes a biological endpoint, such as tumor size, but no clinical benefit, such as improved survival. As part of the program, drug companies then the clinical benefit of the drug is required to be demonstrated through a validated study.

Research has shown that these confirmatory trials can take years, and some drugs have been on the market for more than five years with no proven clinical benefit.

The findings were published online May 24 Annals of internal disease.

“Some patients have been taking drugs for years that are not yet proven, if they can take more effective alternative drugs,” says Gerard Anderson, Ph.D., a professor in Bloomberg School of Health Policy and Management. International Health. “Patients and the government are paying a lot for drugs that may not work.”

Medicare is a federal program that insures older Americans ages 65 and older, as well as people under the age of 65 with disabilities. In their analysis, the researchers identified 45 drugs with 69 indications – use for certain diseases or conditions – in an accelerated FDA-approved program in 2019. Using anonymous data on Medicare claims, the researchers found that the Medicare service payment program spent $ 1.2 billion on 36 of these drugs on 55 indications in 2019. The majority – 45 indications or 82 percent – were used for cancer-related indications. This accounted for 72 percent of Medicare’s spending on accelerated approved drugs in 2019. In total, Medicare recipients incurred $ 209 million in their own spending on these drugs in 2019. Extrapolating to Medicare Advantage – an alternative Medicare plan offered by private insurers – Medicare’s total spending on these drugs in 2019 was $ 1.8 billion.

The study found that seven drugs for 11 indications were withdrawn from the FDA’s accelerated pathway in 2020 and 2021. These seven drugs on 11 indications accounted for approximately $ 187 million in Medicare costs for services in 2019. The authors note that additional testimony may be taken this year and in the future.

In the current system, which began in 1992, pharmaceutical companies do not have much financial incentive to complete additional clinical trials to confirm all the benefits of the drug, as drugs can be fully sold under an accelerated approval program. The study authors propose to impose a limit on how long accelerated-approval drugs can be marketed before confirming clinical benefit. Another possible incentive would be for Medicare to provide lower payments for drugs that did not benefit clinically.

The authors recommend that Congress reconsider the FDA accelerated approval the way the FDA’s Prescription Medicines Fees Act will be re-approved later this year. The law requires drug manufacturers pay fees that facilitate agency regulation and revision of prescription drugs, funds that currently make up about half of the FDA’s $ 6 billion budget.

“We want to make sure people are taking drugs that work,” Anderson adds. “The government should not spend money on drugs that do not have such a guarantee for such a long period of time.”