New FDA Proposal Aims to Help Patients With Hard-to-Treat Diseases

Key TakeawaysThe U.S. Food and Drug Administration wants to make it easier to create custom treatments for super rare diseasesThe plan could help patients with conditions that are too rare for large studiesPublic comments will be accepted for 60 days before changes are finalized.TUESDAY, Feb. 24, 2026 (HealthDay News) — U.S. health officials are proposing a new way to develop and approve custom-made treatments for people with rare and hard-to-treat conditions.The U.S. Food and Drug Administration (FDA) just released a draft of guidelines that would create a special pathway for therapies designed for just a small number of people. Drug companies often avoid these, because they are considered unprofitable.Under the proposed guidelines, these treatments might only be tested in a handful of patients, instead large group studies.The FDA said the approach could apply to gene-editing treatments, as well as other types of drugs and therapies.“It is our priority to remove barriers and exercise regulatory flexibility to encourage scientific advances and deliver more cures and meaningful treatments for patients suffering from rare diseases,” FDA Commissioner Dr. Marty Makary said.Many rare diseases affect only a few people worldwide. Because of that, drug companies are reluctant to invest the time and money needed to run large studies and bring a drug through the typical approval process, which can take 10 years or more.Traditionally, the FDA requires strong evidence from large clinical trials. For super rare conditions, that kind of study may not be possible.Researchers and advocacy groups have pushed for years for a different system that better fits these rare diseases, The Associated Press reported.Under the proposal, the FDA would allow certain experimental, custom treatments to move forward if scientists can show a clear reason the therapy should work, something the agency calls a “plausible mechanism.”That means researchers would need to prove:The disease is well understood.The treatment targets the exact genetic or biological problem.The therapy successfully reached and changed that patient's issue.If those standards are met, companies may be allowed not only to use the treatment, but also commercialize it.Right now, the FDA sometimes allows experimental drugs through a process known as "compassionate use." This lets very sick patients try unapproved treatments when no other options exist.But the process is complex, slow and does not allow companies to profit from the treatment.The new method is intended to create a clearer, more practical process — one that could make custom treatments more available to patients who desperately need them.FDA officials stressed that these guidelines are not final rules. The agency will accept comments for 60 days before deciding whether to finalize the proposal.The announcement follows other recent FDA changes, including Makary’s decision last week to move away from the long-standing requirement that drugs must pass two major clinical trials before approval.More informationThe U.S. Food and Drug Administration explains its longstanding drug development and approval process.SOURCES: The Associated Press, Feb. 23, 2026.What This Means For YouFor patients with a rare disease, this new proposal could make it easier to access custom treatments when standard drugs do not exist..Sign up for our weekly HealthDay newsletter

New FDA Proposal Aims to Help Patients With Hard-to-Treat Diseases

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