Vital guidelines for diagnosis, treatment, and prevention of dengue for healthcare providers.

Original title: “Dengue for Health Care Providers”

Lower your risk of pneumonia with vaccines and other healthy living practices.

Original title: “Pneumonia Prevention and Control”

CDC offers resources about cancer in a variety of formats.

Original title: “Resource Library”

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft document entitled "Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause." The draft guidance document describes considerations for generating substantial evidence of effectiveness and evidence of safety of individualized therapies based on a plausible mechanism framework.

Original title: “Considerations for the Use of the Plausible Mechanism Framework To Develop Individualized Therapies That Target Specific Genetic Conditions With Known Biological Cause; Draft Guidance for Industry; Availability”

This document extends the comment period for the proposed rule that appeared in the Federal Register on December 23, 2025, titled "Transparency in Coverage". The comment period for the proposed rule, which would end on February 23, 2026, is extended until March 2, 2026.

Original title: “Private Health Insurance; Transparency in Coverage; Extension of Comment Period”

The Office of Early Childhood Development (ECD) is requesting a 3-year extension with revisions to annual reporting instruments under Office of Management and Budget (OMB) Control #: 0970-0614. This includes proposed revisions to the Demographic and Service Utilization Data Report (DSUR), an extension of the Performance Measurement Data Report (PMR) with no changes, and announces the discontinuation of the Quarterly Performance Report (QPR).

Original title: “Proposed Information Collection Activity; Tribal Maternal, Infant, and Early Childhood Home Visiting Program: Demographic and Service Utilization Data Report and Performance Measurement Data Report”

Original title: “Managed Care”

In compliance with the requirement of the Paperwork Reduction Act of 1995 to provide an opportunity for public comment on proposed data collection projects, the National Institutes of Health, National Cancer Institute (NCI) will publish periodic summaries of proposed projects to be submitted to the Office of Management and Budget (OMB) for review and approval.

Original title: “Proposed Collection: 30-Day Comment Request; The Clinical Trials Reporting Program (CTRP) Database (NCI)”

The Administration for Children and Families (ACF) Office of Planning, Research, and Evaluation (OPRE) is requesting a one-year extension to one of the data collection activities conducted for the Next Generation of Enhanced Employment Strategies (NextGen) Project, the second follow-up survey (OMB #: 0970-0545, expiration April 30, 2026).

Original title: “Proposed Information Collection Activity; Next Generation of Enhanced Employment Strategies Project”

Referred to the House Committee on Agriculture.

Original title: “HR 7010: To amend the Agriculture, Rural Development, Food and Drug Administration, and Related Agency Appropriations Act, 2026, to delay the implementation of amendments made by such Act to the hemp production provisions of the Agricultural Marketing Act of 1946.”

On Monday, the FDA released more details about the Plausible Mechanism Framework, first announced by FDA Commissioner Makary and Director Prasad in the New England Journal of Medicine in November 2025. In an […] The post FDA Takes Important Step Toward Individualized Therapies appeared first on EveryLife Foundation for Rare Diseases.

Original title: “FDA Takes Important Step Toward Individualized Therapies”

The proposed system would create a standardized process for authorizing cutting-edge treatments where there is a plausible reason to think they might work, the AP reported. AP: Rare Diseases: FDA Proposes New System To Therapy Approvals Federal health officials on Monday laid out a proposal to spur development of customized treatments for patients with hard-to-treat diseases, including for rare genetic conditions that the pharmaceutical industry has long considered unprofitable. Stat: Epstein Blood Samples Ignited A 2013 Furor In Harvard Genome Lab Among several fridges inside the Harvard Medical School lab of renowned geneticist George Church is one devoted to housing tubes of human blood and spit destined to have their cellular contents cracked open, the DNA letters read out and posted to the wilds of the open internet. The preliminary Food and Drug Administration guidelines, if implemented, would create a new pathway for bespoke therapies that have only been tested in a handful of patients due to the challenges of conducting larger studies. The FDA announcement specifically mentions gene editing, although agency officials said the new approach could also be used by other drugs and therapies.

Original title: “FDA Unveils Framework To Fast-Track Rare Disease Gene Therapy Approvals - KFF Health News”

Dr Tracy Beth Høeg, Acting Director ... therapies, thereby driving innovation, improving safety, lowering costs and offering more patients with ultra-rare diseases a unique shot at a life-saving treatment.” · Public comment on the draft guidance can be submitted to regulation.gov. FDA also recently shared details of a new drug approval policy with criteria for clinical trial ... Dr Tracy Beth Høeg, Acting Director for FDA’s Center for Drug Evaluation and Research, added: “We anticipate our Plausible Mechanism draft guidance will inspire industry to place increased focus on individualised therapies, thereby driving innovation, improving safety, lowering costs and offering more patients with ultra-rare diseases a unique shot at a life-saving treatment.” · Public comment on the draft guidance can be submitted to regulation.gov. FDA also recently shared details of a new drug approval policy with criteria for clinical trial requirements. Agency’s planned framework is the first to support the approval process for new ultra-rare disease treatments. For traditional approval, therapies should demonstrate improvement in clinical outcomes, disease course or biomarkers if they are established to predict clinical benefit. FDA noted that a product targeting different mutations in a single gene is appropriate for inclusion in a single product application. It would then be potentially assessed via master protocols that evaluate these product variations in a single trial. business news, Clinical Development, Clinical Trials, Data Analysis, Drug Development, Drug Markets, Drug Safety, Industry Insight, Rare diseases, RNA, Therapeutics ... business news, Clinical Development, Clinical Trials, Data Analysis, Drug Development, Drug Markets, Drug Safety, Industry Insight, Rare diseases, RNA, Therapeutics

Original title: “FDA drafts new ultra-rare disease therapy guidance”

Dr Tracy Beth Høeg, Acting Director ... therapies, thereby driving innovation, improving safety, lowering costs and offering more patients with ultra-rare diseases a unique shot at a life-saving treatment.” · Public comment on the draft guidance can be submitted to regulation.gov. FDA also recently shared details of a new drug approval policy with criteria for clinical trial ... Dr Tracy Beth Høeg, Acting Director for FDA’s Center for Drug Evaluation and Research, added: “We anticipate our Plausible Mechanism draft guidance will inspire industry to place increased focus on individualised therapies, thereby driving innovation, improving safety, lowering costs and offering more patients with ultra-rare diseases a unique shot at a life-saving treatment.” · Public comment on the draft guidance can be submitted to regulation.gov. FDA also recently shared details of a new drug approval policy with criteria for clinical trial requirements. Agency’s planned framework is the first to support the approval process for new ultra-rare disease treatments. For traditional approval, therapies should demonstrate improvement in clinical outcomes, disease course or biomarkers if they are established to predict clinical benefit. FDA noted that a product targeting different mutations in a single gene is appropriate for inclusion in a single product application. It would then be potentially assessed via master protocols that evaluate these product variations in a single trial. business news, Clinical Development, Clinical Trials, Data Analysis, Drug Development, Drug Markets, Drug Safety, Industry Insight, Rare diseases, RNA, Therapeutics ... business news, Clinical Development, Clinical Trials, Data Analysis, Drug Development, Drug Markets, Drug Safety, Industry Insight, Rare diseases, RNA, Therapeutics

Original title: “FDA drafts new ultra-rare disease therapy guidance”

Referred to the House Committee on the Judiciary.

Original title: “HR 7651: To provide a private right of action for children and the parents of children whose healthy body parts have been damaged by medical professionals practicing or participating in certain interventions.”

Placed on Senate Legislative Calendar under General Orders. Calendar No. 340.

Original title: “S 233: A bill to amend the Office of National Drug Control Policy Reauthorization Act of 2006 to modify the authority of the Office of National Drug Control Policy with respect to the World Anti-Doping Agency, and for other purposes.”

Health and Human Services (HHS) ... treatments to reach the patients who need them most.” ... Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence · The main feature of the pathway is reducing the reliance on clinical trial data, which is harder to obtain for personalised therapies drugging rare diseases with small ... Health and Human Services (HHS) Secretary Robert F. Kennedy Jr (RFK Jr) said: “We are cutting unnecessary red tape, aligning regulation with modern biology, and clearing a path for breakthrough treatments to reach the patients who need them most.” ... Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence · The main feature of the pathway is reducing the reliance on clinical trial data, which is harder to obtain for personalised therapies drugging rare diseases with small patient population sizes. In June 2025, in front of a cell and gene therapy panel, RFK Jr said he was “going to continue to figure out new ways of accelerating approvals for rare disease drugs and treatments”. Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva. Given that many genetic editing technologies are considered modular in their approach, a highly supported “plausible” mechanism of action may used to approve therapy variants that target a different mutation. This effectively means that patients not included in the clinical trial used to support the original approval can still be included in the approved label. For example, a single disease with 150 different genetic mutations would be well suited to the bespoke pathway. Makary stated: “This guidance is a critical step the FDA is taking to tailor our regulatory approach to patients with ultra-rare conditions. The aim of the pathway is to remove red tape for bespoke therapies designed for patients with rare diseases.

Original title: “Gene editing takes centre stage in FDA’s new rare disease approval pathway - Clinical Trials Arena”

Driving the news: The agency is ... clinical trials because the patient populations are too small. Treatments with a similar mechanism could be grouped together to allow for a single approval that can be adapted based on different genetic mutations, for example. The big picture: The move was inspired by "Baby KJ," an infant born with a rare metabolic disease who became first person to be successfully treated with a personalized CRISPR therapy last year ... Driving the news: The agency is establishing a way to evaluate rare disease treatments that can't be put through traditional clinical trials because the patient populations are too small. Treatments with a similar mechanism could be grouped together to allow for a single approval that can be adapted based on different genetic mutations, for example. The big picture: The move was inspired by "Baby KJ," an infant born with a rare metabolic disease who became first person to be successfully treated with a personalized CRISPR therapy last year at Children's Hospital of Philadelphia. The FDA would evaluate evaluate rare disease treatments that can't be put through traditional clinical trials. He said 30 million Americans have a rare disease. "For too long they have been under-appreciated, under-recognized, the research has been underfunded, because our system is built for common diseases," he added. Tracy Beth Høeg, acting head of the FDA's drug center, said the draft guidance is "going to inspire industry to start developing these treatments in a way that we haven't seen before." The Food and Drug Administration on Monday laid out a new pathway aimed at spurring novel individualized treatments targeting rare diseases.

Original title: “FDA smooths reviews of rare disease treatments”

Billions of dollars’ worth of cancer drugs are discarded each year. Manufacturers must refund Medicare for some of this waste. A data-driven approach offers a practical path to greater efficiency.

Original title: “Cut Down on Oncology Drug Waste With Better Forecasting”

Clinical information on testing for, preventing, and managing pneumococcal disease.

Original title: “Clinical Guidance for Pneumococcal Disease”