FDA Loosens Approval Process for Drugs Targeting Rare Diseases

The drugs eligible for the looser standards are for diseases that affect fewer than 1,000 people in the United States, regulators said.

The U.S. Food and Drug Administration building in White Oak, Md., on June 5, 2023. Madalina Vasiliu/The Epoch Times

Food and Drug Administration officials on Sept. 3 said they will approve some drugs that target rare diseases with no traditional randomized controlled trial data, citing challenges with testing investigational products for diseases that affect low numbers of Americans.

Manufacturers can apply to the FDA under the new process for drugs against rare diseases driven by genetic defects. The new requirements are that the drug targets a rare disease that impacts fewer than 1,000 people in the United States, the drug is aimed at treating a genetic defect that is a major driver of the disease, people with the disease deteriorate rapidly, and there are no cleared products for the disease.

Drugs that meet those criteria can be cleared with one trial, which could consist of a single arm, the FDA’s Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research said in a joint statement.

Applicants were told to apply before launching pivotal trials. If the FDA accepts the application, the manufacturer and the FDA will meet and go over what data will be used to establish safety and efficacy.

“Americans with rare diseases deserve a faster, more predictable process for access to promising therapies. Period,” Dr. Marty Makary, the FDA’s commissioner, said on X.

The proposed process would be implemented jointly by the two centers, the FDA said in a statement.

Developing drugs for rare diseases comes with challenges, including difficulty generating evidence of safety and efficacy in multiple trials, according to the agency.

The new process “addresses the inherent uncertainties of rare disease drug development by assuring sponsors that reviews will encompass additional supportive data,” it stated.

The Center for Drug Evaluation and Research is led by Dr. George Tidmarsh, while Dr. Vinay Prasad leads the Center for Biologics Evaluation and Research.

Prasad’s division in August approved a therapy for a rare disease based on data from a single-arm trial. Prasad said at the time that “randomized trials are not always needed to approve medical products and this approval is proof of that philosophy.”

Some other approvals for rare disease drugs have come after more substantial data. The FDA’s approval of a drug for a blood disorder called immune thrombocytopenia, for example, was based on data from a double-blind study.

Prasad has also said that for issues affecting more people, such as COVID-19, the FDA is requiring randomized, controlled trials to determine if they are efficacious.