FDA to Streamline Orphan Drug Designation Following Scathing Government Report

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A government investigation claims that the U.S. Food and Drug Administration (FDA) has failed to live up to the legal standards required for Orphan Drug designation.

A year-long report from the Government Accountability Office said the FDA has not ensured that drugs currently approved under the rare disease designation are meeting the intent of a 35-year-old law, Kaiser Health News reported. The program was created 35 years ago to spur the development of therapies for people struggling with diseases that affect fewer than 200,000 people in the United States. According to the KHN report, drugmakers have been manipulating the program to “maximize profits and to protect niche markets for medicines being taken by millions.”

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The report examined 148 applications to the FDA submitted by drugmakers for orphan drug approval last year. Reviewers were supposed to apply to criteria -- how many patients would be served and whether there is scientific evidence the drug will treat their disease, KHN said. In about 60 percent of all cases, the FDA failed to capture any regulatory history information from other agencies, including adverse actions taken, the GAO said, according to KHN. In approximately 15 percent of cases, the FDA failed to verify the number of patients that drugmakers said the medication will serve, according to the report. Out of 148 applications, 26 that received approval for the Orphan Drug designation had missing information, KHN noted. Additionally, the report showed that more than 80 drugs given the orphan drug status earned regulatory approval for more than one disease. That came with “its own bundle of rich incentives,” the report said.

In its investigation, the GAO discovered “inconsistent and often incomplete reviews” during the process of designating orphan drug status by the FDA. The GAO said that in some cases, the FDA reviewers of the orphan drug status “failed to show they had checked how many patients could be treated by a drug being considered for orphan drug status; instead, they appeared to trust what drugmakers told them,” KHN said. In its report, the GAO recommended that the FDA take “executive action” to fix the system to ensure that drug companies are not manipulating the process to protect those niche markets.

John Dicken, head of the GAO’s healthcare team, told KHN that the focus of its investigation into the FDA’s use of the Orphan Drug Designation was to ensure that the intent of the law was being met.

In a statement, the FDA agreed with the report recommendations regarding the need for greater documentation. KHN said the regulatory agency will streamline its process. The Department of Health and Human Services also agreed with the GAO assessment, KHN said.

The Orphan Drug program at the FDA was launched in 1983 after Congress passed the Orphan Drug Act. The new law was passed to “motivate” pharmaceutical companies to develop medications for these conditions for which there were few treatment options. Treatments for rare diseases were typically ignored by the companies due to a belief they were not profitable.

In its report, KHN said that with the abuse of the rare disease program, about one-third of all pharmaceutical spending in the United States had shifted to orphan drug research.

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