The U.S. Food and Drug Administration proposed on Monday a new framework to speed approvals of personalized treatments for rare and life-threatening genetic diseases, allowing drugmakers to rely on small, well-controlled studies when traditional trials are not possible. WASHINGTON, Feb 23 (Reuters) - The U.S. Food and Drug Administration proposed on Monday a new framework to speed approvals of personalized treatments for rare and life-threatening genetic diseases, allowing drugmakers to rely on small, well-controlled studies when traditional trials are not possible. The plan aims to update approval standards for individualized genetic medicines, creating a pathway for therapies aimed at patient groups too small for traditional trials. For treatments targeting multiple mutations within the same gene, it recommended observational studies and "master protocol" designs to allow evidence-sharing across related products. "We anticipate that we're going to get a flood of applications for treatments of rare diseases," a senior FDA official told reporters.

Original title: “US FDA proposes framework to speed rare disease gene therapy approvals | Reuters”

The U.S. Food and Drug Administration today issued draft guidance for sponsors seeking approval for targeted individualized therapies by generating substantial evidence of effectiveness and safety when randomized controlled trials are not feasible due to small patient populations.

Original title: “FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases ”

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Original title: “Leadership”

The FDA has introduced a new approval process for targeted individualized therapies, including genome editing and RNA-based therapies, intended to treat rare and ultra rare diseases in which randomized controlled trials are not feasible.HHS Secretary Robert F. Kennedy Jr. and FDA Commissioner ... The FDA has introduced a new approval process for targeted individualized therapies, including genome editing and RNA-based therapies, intended to treat rare and ultra rare diseases in which randomized controlled trials are not feasible.HHS Secretary Robert F. Kennedy Jr. and FDA Commissioner Martin Makary, MD, MPH, announced the new process, called the “plausible mechanism pathway” The pathway aims to streamline approval for individualized treatments for rare and ultra-rare diseases. An infant with a rare genetic disorder was treated with a therapy that serves as a model for the protocol. The FDA has introduced a new approval process for targeted individualized therapies, including genome editing and RNA-based therapies, intended to treat rare and ultra rare diseases in which randomized controlled trials are not feasible. Under the framework we are announcing today, one well-controlled clinical investigation, supported by confirmatory evidence, can support approval.” · The press conference centered on the case of an infant in the Philadelphia area born with a rare disease called carbamoyl-phosphate synthase 1 deficiency (CPS1), which is caused by a deficiency in the CPS1 gene. ... Rebecca Ahrens-Nicklas, MD, PhD, director of the Children’s Hospital of Philadelphia (CHOP) Gene Therapy for Inherited Metabolic Disorders Program, and Kiran Musunuru, MD, PhD, MPH, a cardiologist, geneticist and gene editor at the University of Pennsylvania, and co-director of the Orphan Disease Center, a collaborative center between CHOP and Penn, had conducted research into the use of gene editing to create personalized interventions for patients with heart disease. According to an HHS press release, the proposed pathway will include five key elements — identifying molecular or cellular abnormalities, targeting the underlying alteration, use of natural history data, evidence of successful application of the technology, and demonstration of clinical benefit. Stipulations for FDA approval and post-marketing analyses are also part of the protocol. According to Makary, approximately 30 million Americans have rare diseases. He added that with the plausible mechanism protocol, the FDA hopes to ensure the safety of new treatments like the one developed by the team at CHOP.

Original title: “FDA provides new approval process for rare disease therapies without large clinical trials”

The FDA has introduced a new approval process for targeted individualized therapies, including genome editing and RNA-based therapies, intended to treat rare and ultra rare diseases in which randomized controlled trials are not feasible.HHS Secretary Robert F. Kennedy Jr. and FDA Commissioner ... The FDA has introduced a new approval process for targeted individualized therapies, including genome editing and RNA-based therapies, intended to treat rare and ultra rare diseases in which randomized controlled trials are not feasible.HHS Secretary Robert F. Kennedy Jr. and FDA Commissioner Martin Makary, MD, MPH, announced the new process, called the “plausible mechanism pathway” The pathway aims to streamline approval for individualized treatments for rare and ultra-rare diseases. An infant with a rare genetic disorder was treated with a therapy that serves as a model for the protocol. The FDA has introduced a new approval process for targeted individualized therapies, including genome editing and RNA-based therapies, intended to treat rare and ultra rare diseases in which randomized controlled trials are not feasible. Under the framework we are announcing today, one well-controlled clinical investigation, supported by confirmatory evidence, can support approval.” · The press conference centered on the case of an infant in the Philadelphia area born with a rare disease called carbamoyl-phosphate synthase 1 deficiency (CPS1), which is caused by a deficiency in the CPS1 gene. ... Rebecca Ahrens-Nicklas, MD, PhD, director of the Children’s Hospital of Philadelphia (CHOP) Gene Therapy for Inherited Metabolic Disorders Program, and Kiran Musunuru, MD, PhD, MPH, a cardiologist, geneticist and gene editor at the University of Pennsylvania, and co-director of the Orphan Disease Center, a collaborative center between CHOP and Penn, had conducted research into the use of gene editing to create personalized interventions for patients with heart disease. According to an HHS press release, the proposed pathway will include five key elements — identifying molecular or cellular abnormalities, targeting the underlying alteration, use of natural history data, evidence of successful application of the technology, and demonstration of clinical benefit. Stipulations for FDA approval and post-marketing analyses are also part of the protocol. According to Makary, approximately 30 million Americans have rare diseases. He added that with the plausible mechanism protocol, the FDA hopes to ensure the safety of new treatments like the one developed by the team at CHOP.

Original title: “FDA provides new approval process for rare disease therapies without large clinical trials”

Will consider data that includes 'the experience of individuals,' FDA commissioner says "We'll be continuing our work with the FDA using the plausible mechanism framework that's being published today, with the goal of getting to an approval of this platform as efficiently as possible, and getting individualized treatments for [these types of] disorders to all the patients in the U.S. who could benefit from them," he added. "But that's just the first step ... We'll share our learnings with everyone in the hope that when scientists develop technologies to get this kind of therapy to many organs besides the liver, it will allow doctors to treat many, many rare diseases by getting at their root causes. It will be a whole new medical subspecialty: interventional genetics." According to an FDA press release, the draft guidance "focuses on therapies that target a specific genetic, cellular, or molecular abnormality and are designed to correct or modify the underlying cause of disease." Criteria for approval under this pathway would include: Kiran Musunuru, MD, PhD, director of Penn Cardiovascular Institute's Genetic and Epigenetic Origins of Disease Program in Philadelphia, explained how the draft guidance might apply to his team's research into genetic treatments for cardiovascular disease. "My team created a genetic therapy to turn off a cholesterol gene in the liver, permanently reduce cholesterol levels, and give lifelong protection against heart disease," he said. It's common sense," FDA Commissioner Marty Makary, MD, MPH, said at a press conference at HHS headquarters. "For the first time, the FDA is issuing guidance giving drug developers of ultra-rare disease therapies a path to accelerated or traditional approval based on the experience of individuals."

Original title: “FDA Proposes New Approval Pathway for Rare Disease Gene Therapy | MedPage Today”

Andrew Russell, co-owner of Elko New Market Family Pharmacy in Elko, Minnesota, writes about mounting pressures from pharmacy benefit managers, or PBMs. Strengthening oversight of PBM reimbursement practices, ensuring transparency in drug pricing, supporting a sustainable payment model for independent pharmacies, and acknowledging Pharmacy Services Administrative Organizations for what they are — support partners, not decision-makers — are essential steps in protecting Minnesota patients.

Original title: “Letter: Protect patient access by confronting pharmacy challenges - InForum | Fargo, Moorhead and West Fargo news, weather and sports”

Referred to the House Committee on Energy and Commerce.

Original title: “HR 7602: To direct the Secretary of Health and Human Services to study and report on the state of men's health in the United States and to establish an Office of Men's Health within the Department of Health and Human Services.”

Referred to the Committee on Financial Services, and in addition to the Committee on House Administration, for a period to be subsequently determined by the Speaker, in each case for consideration of such provisions as fall within the jurisdiction of the committee concerned.

Original title: “HR 7623: To award a Congressional Gold Medal to the Freedom House Ambulance Service, in recognition of its dedicated service to the Pittsburgh community and contributions to the field of emergency medical services.”

This quarterly notice lists Centers for Medicare & Medicaid Services (CMS) manual instructions, substantive and interpretive regulations, and other Federal Register notices that were published in the 3-month period, relating to the Medicare and Medicaid programs and other programs administered by CMS.

Original title: “Medicare and Medicaid Programs; Quarterly Listing of Program Issuances-October Through December 2025”

The Administration for Children and Families (ACF) is requesting a 3-year extension of the information collection Case Plan Requirement, Title IV-E of the Social Security Act, (Office of Management and Budget (OMB) #0970-0428, expiration September 30, 2026). There are no changes to the requirements, but burden estimates have been updated to reflect a reduction in average time to complete a case plan and the current numbers of children in foster care.

Original title: “Proposed Information Collection Activity; Case Plan Requirement, Title IV-E of the Social Security Act”

The Food and Drug Administration (FDA) is announcing the issuance of a priority review voucher to the sponsor of a rare pediatric disease product application. The Federal Food, Drug, and Cosmetic Act (FD&C Act) authorizes FDA to award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA is required to publish notice of the award of the priority review voucher. FDA has determined that WASKYRA (etuvetidigene autotemcel), appro

Original title: “Issuance of Priority Review Voucher; Rare Pediatric Disease Product; WASKYRA (etuvetidigene autotemcel)”

The Food and Drug Administration (FDA or Agency) has determined that KLONOPIN (clonazepam) tablets, 0.125 milligrams (mg) and 0.25 mg, were not withdrawn from sale for reasons of safety or effectiveness. This determination will allow FDA to approve abbreviated new drug applications (ANDAs) for clonazepam tablets, 0.125 mg and 0.25 mg, if all other legal and regulatory requirements are met.

Original title: “Determination That Klonopin (Clonazepam) Tablets, 0.125 Milligrams and 0.25 Milligrams, Were Not Withdrawn From Sale for Reasons of Safety or Effectiveness”

This document corrects two outdated references that should have been deleted and one outdated reference in the final rule that appeared in the February 2, 2024 Federal Register titled "Medications for the Treatment of Opioid Use Disorder," specifying final modified and updated certain provisions related to Opioid Treatment Program (OTP) accreditation, certification, and standards for the treatment of Opioid Use Disorder with Medications for Opioid Use Disorders in OTPs. The effective date of the

Original title: “Medications for the Treatment of Opioid Use Disorder; Correction”

This document corrects technical errors in the final rule with comment period that appeared in the November 25, 2025 issue of the Federal Register titled "Medicare Program: Hospital Outpatient Prospective Payment and Ambulatory Surgical Center Payment Systems; Quality Reporting Programs; Overall Hospital Quality Star Rating; Hospital Price Transparency; and Notice of Closure of a Teaching Hospital and Opportunity To Apply for Available Slots" (hereinafter referred to as the "CY 2026 OPPS/ASC fin

Original title: “Medicare Program; Hospital Outpatient Prospective Payment and Ambulatory Surgical Center Payment Systems; Quality Reporting Programs; Overall Hospital Quality Star Rating; Hospital Price Transparency; and Notice of Closure of a Teaching Hospital and Opportunity To Apply for Available Slots; Correction”

Read twice and referred to the Committee on Veterans' Affairs.

Original title: “S 3771: Department of Veterans Affairs Therapeutic Medical Physicist Pay Cap Relief Act of 2026”

Read twice and referred to the Committee on Indian Affairs.

Original title: “S 3767: Stronger Engagement for Indian Health Needs Act of 2026”

Read twice and referred to the Committee on Health, Education, Labor, and Pensions.

Original title: “S 3869: Healthy Families Act”

Referred to the Committee on Energy and Commerce, and in addition to the Committee on Ways and Means, for a period to be subsequently determined by the Speaker, in each case for consideration of such provisions as fall within the jurisdiction of the committee concerned.

Original title: “HR 6494: Territories Health Equity Act of 2025”

Referred to the Committee on Energy and Commerce, and in addition to the Committee on Ways and Means, for a period to be subsequently determined by the Speaker, in each case for consideration of such provisions as fall within the jurisdiction of the committee concerned.

Original title: “HR 7277: Emergency Medical Services Reimbursement for On-Scene and Support Act”