The U.S. Food and Drug Administration (FDA) is working to expedite a process for new drug approval. Last week the FDA announced the federal agency would no longer require multiple drug trials before drug approvals. In the future FDA drug reviewers will accept one well-planned trial as evidence on a drug’s efficacy. Today the FDA also proposed a faster way to approve drugs for rare conditions and hard-to-treat conditions.
The process for drug approval is bureaucratic and overly rigid. After a new drug molecule is identified, Stage I and Stage II trials are completed to test for toxicity and safety. Once a drug’s safety is evaluated, it can take years longer to finalize the Stage III clinical trials on drug efficacy. The new FDA Commissioner plans to speed the process. The following was reported by Medical Press:
The Food and Drug Administration plans to drop its longtime standard of requiring two rigorous studies to win approval for new drugs, the latest change from Trump administration officials vowing to speed up the availability of certain medical products.
Going forward, the FDA’s “default position” will be to require one study for new drugs and other novel health products, FDA Commissioner Dr. Marty Makary and a top deputy, Dr. Vinay Prasad, wrote in a New England Journal of Medicine piece published Wednesday.
The FDA Commissioner, Marty Makary, has shown a willingness to change long standing standards and procedures that slowed drug approvals. He has mandated use of artificial intelligence (AI), and implemented deadlines for medications in the “national interest.”
The 2-study requirement dates to the 1960s when Congress passed a law requiring the FDA to only approve new drugs after “adequate and well-controlled investigations.” The agency decided to require at least two clinical trial studies. The second study was to determine the first study’s results were not an anomaly. There was never a Congressional mandate to have at least two clinical studies. The move away from two rigid clinical studies did not come as a surprise to many. Former FDA Center for Drug Evaluation and Research director Janet Woodcock said the FDA had already moved to single study trials for some rare conditions. Clinical trials for rare conditions are tedious because recruiting enough participants is often a struggle.
Indeed, the agency is also seeking to fast-track bespoke drug therapies and drugs to treat rare diseases agency officials announced on Monday. Researchers struggle to meet the requirements due to the small population of people who suffer from some rare, genetic conditions. Historically, double-blind clinical trials required evaluating a control group using a placebo and comparing the results to the treatment group. For rare disease that requirement made it double difficult to recruit enough participants. The following is from the Associated Press News:
Senior FDA officials said the recent changes, including the pathway proposed Monday, don’t constitute new FDA standards. The FDA will take comments on its draft guidance for 60 days, before beginning to finalize it.
In recent years, academic researchers have shown they can use emerging technology to correct individual defects in a patient’s genetic code. Last year, a team at Children’s Hospital of Philadelphia and the University of Pennsylvania designed a therapy using CRISPR, the Nobel Prize-winning gene editing tool, to treat a baby born with a rare disease that causes ammonia to build up in the blood.
The FDA reports the newer procedures will be reserved for conditions where the mechanisms are well understood, while continuing to require data to show treatments successfully targeted patients genetic or biological abnormality.
Read more at: Rare diseases: FDA proposes new system to therapy approvals.
FDA will drop two-study requirement for new drug approvals, aiming to speed access.