The Food and Drug Administration is proposing a system for approving customized drugs and medical treatments for patients with rare or hard-to-treat diseases.
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 The FDA will take comments on its draft guidance for 60 days, before beginning to finalize it.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 Pennsylvaniausing 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. Traditionally, the FDA requires drugmakers to demonstrate the safety and effectiveness of their experimental treatments in clinical studies that compare a set of patients getting the therapy with others taking a sham treatment or an alternative intervention. The more patients enrolled, the stronger the evidence. But for conditions that can affect a tiny fraction of people worldwide, drug companies often have little incentive to invest millions of dollars needed to complete a study and bring it through the FDA approval process, which can take a decade or longer. The pathway announced Monday would create a standardized process for authorizing experimental treatments and, importantly, offering companies the possibility of commercializing them. The FDA already authorizes the use of experimental drugs under what’s called “compassionate use,” for people with no other medical options. But the process is cumbersome to navigate and strictly prohibits companies or researchers from profiting from treatments that haven’t been vetted by the FDA. The new pathway’s name — plausible mechanism — is a reference to criteria FDA regulators will require before greenlighting any experimental therapies. FDA officials say the approach will be reserved for conditions that are well understood and where there is a plausible reason to think that the therapy will act on the underlying genetic or cellular biology of the disease. Researchers must also confirm that the therapy successfully targeted the patient's genetic or biological abnormality.The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content. Copyright 2026 The Associated Press. All rights reserved. This material may not be published, broadcast, rewritten or redistributed without permission.Here's how much you can expect to pay for a World Cup ticketSan Antonio man killed by HSI agent last March in undisclosed shooting, records showCommunity members pack meeting as Judson ISD weighs closing 3 elementary schoolsFamily seeks answers, city action after deadly shooting on Rigsby AvenueMother released on bond after arrest for telling child to walk to school 19 miles awayJudge rules Paxton lacked authority to sue Bexar County over legal aid for immigrants30 students disciplined at East Central High School after anti-ICE walkout‘It was intentional’: San Antonio owner demands answers after his dog was shot in neighborhoodProposed high-voltage power line sparks pushback across Texas Hill Country0:36How messaging, emergency preparedness have changed 5 years after 2021 winter stormJefferson HS teacher looks to introduce fencing to engage students, prevent troubleAfter son’s hit-and-run death, San Antonio mother struggles with homelessness and burial costsSuspect arrested in connection with scheme that cost Converse woman her life’s savingsSakai, Nirenberg tout experience in faceoff for Bexar County JudgeWhat we know after two people killed at NW Side hookah barSan Antonio police launch investigation after dog left behind during owner’s arrestWATCH: Truck flies into Oregon home‘It looked like he got hit by a car’: Helotes dog returns home with mysterious injuriesJudson ISD board votes to close Judson Middle School amid $37 million deficitVIA employee union ‘not on board’ with free bus fare pushVerbal altercation between Judson ISD trustee and board president over Open Meetings ActJudson ISD student shares frustration over potential Franz Elem. closureHeadstone unveiled for Savanah Soto and her unborn babyFIVE YEARS AGO: A look back at the historic February 2021 winter storm in San AntonioPrevious photo
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