Original title: “Agency Forms Undergoing Paperwork Reduction Act Review”

Last Friday, April 2, the White House released the President’s proposed Budget for Fiscal Year 2027. While there is still much we will learn and distill over the coming days and […] The post The President’s Budget Proposal: What Does It Mean for our Rare Disease Community? appeared first on EveryLife Foundation for Rare Diseases.

Original title: “The President’s Budget Proposal: What Does It Mean for our Rare Disease Community?”

In its budget request, the agency proposed a way to speed up early drug testing and help smaller biotech firms. Elsewhere, two startups raised megarounds.

Original title: “FDA floats new clinical trial reforms; Takeda punts Denali brain drug”

The US Food and Drug Administration has pursued many new initiatives since 1977. The agency began accepting abbreviated applications for generic versions of drugs, collected user fees to support drug review, launched the Critical Path Initiative to encourage innovation, and worked to harmonize ... The US Food and Drug Administration has pursued many new initiatives since 1977. The agency began accepting abbreviated applications for generic versions of drugs, collected user fees to support drug review, launched the Critical Path Initiative to encourage innovation, and worked to harmonize its regulatory standards with those of Europe and Japan. Orphan-drug development. Congress passed the Orphan Drug Act in 1983, launching an innovative program that encourages industry to develop treatments for rare diseases, i.e., conditions affecting fewer than 200,000 people in the United States. The law provides extended market exclusivity for such drugs and medical products and awards grants to support orphan-drug clinical trials. The Project BioShield Act of 2004 encouraged the development and regulation of drugs and vaccines to prevent or defend against bioterrorist attacks, a policy reinforced by additional legislation in 2006. FDA has played a lead role in spurring the development of new vaccines and updating production methods to address the need for more preventives and treatments for infectious diseases and biological attacks. AIDS-influenced regulation. The first reports of AIDS expanded FDA collaboration with researchers and scientists to approve initial immunoassay tests. Several rule changes in the mid-1980s broadened access to experimental drugs for patients with serious diseases and no alternative treatment options.

Original title: “Three Decades of Drug Regulation and Legislation | Pharmaceutical Technology”

While recent FDA guidance speaks to the agency’s support of innovative trial designs—including the use of external controls—the application of this flexibility appears to be inconsistent. One former regulator says the situation is more nuanced.

Original title: “FDA Reversals in Rare Disease Space Highlight Confusion Around External Controls”

This final rule revises the Medicare Advantage (Part C), Medicare Prescription Drug Benefit (Part D), and Medicare cost plan regulations to implement changes related to Star Ratings, marketing and communications, drug coverage, enrollment processes, special needs plans, and other programmatic areas.

Original title: “Medicare Program; Contract Year 2027 and Certain Contract Year 2026 Policy and Technical Changes to the Medicare Advantage Program, Medicare Prescription Drug Benefit Program, and Medicare Cost Plan Program”

This proposed rule would update the hospice wage index, payment rates, and aggregate cap amount for Fiscal Year (FY) 2027. This proposed rule also includes an analysis of Medicare non-hospice spending, including details regarding a hospice service and spending variation index (SSVI), and proposes to require that hospices provide the hospice election statement addendum to all Medicare beneficiaries at the time of hospice election. Additionally, this rule proposes conforming regulation text change

Original title: “Medicare Program; FY 2027 Hospice Wage Index and Payment Rate Update and Hospice Quality Reporting Program Requirements”

This proposed rule would update the prospective payment rates for inpatient rehabilitation facilities (IRFs) for Federal fiscal year (FY) 2027. As required by statute, this proposed rule includes the classification and weighting factors for the IRF prospective payment system's case-mix groups and a description of the methodologies and data used in computing the prospective payment rates for FY 2027. It also continues the third year of the 3-year phaseout of the rural adjustment, which began in F

Original title: “Medicare Program; Inpatient Rehabilitation Facility Prospective Payment System for Federal Fiscal Year 2027 and Updates to the IRF Quality Reporting Program”

ACF proposes to make changes to the Temporary Assistance for Needy Families (TANF) program regulations to reset the base year of the caseload reduction credit from fiscal year (FY) 2005 to the new year established by Congress, which is currently FY 2015, and to exclude from the TANF work participation rate calculations certain cases that receive assistance payments benefits of less than $35 for a month. These changes are required by the Fiscal Responsibility Act (FRA) of 2023. The docket on http

Original title: “Work Participation Rate Calculation Changes: Recalibration of the Caseload Reduction Credit and Prohibition of Small Checks in Work Participation Rate Calculation”

The Department of Health and Human Services, Administration for Children and Families, proposes to remove duplicative or unnecessary Sections from the Family Violence Prevention and Services program regulations (45 CFR part 1370). These amendments will streamline the Family Violence Prevention and Services regulations and make them more accessible to the public. The docket on https:// www.regulations.gov will include a plain language summary of the NPRM.

Original title: “Reducing Bureaucracy and Burden in Family Violence and Prevention Services”

The Food and Drug Administration (FDA or we) is announcing an updated data standard requirement for the submission of postmarketing individual case safety report (ICSR) submissions for human drug products, biological products, and drug- or biologic-led combination products to the FDA Adverse Event Monitoring System (AEMS) database (formerly FDA Adverse Event Reporting System (FAERS)) via the Electronic Submissions Gateway Next Generation (ESG NextGen). Starting October 1, 2026, postmarketing ICS

Original title: “Electronic Submission of Postmarketing Individual Case Safety Reports to the Food and Drug Administration Adverse Event Monitoring System Using International Council of Harmonisation E2B(R3) Data Standards; Regional Data Elements and Implementation Schedule”

This notice announces a delay in the implementation of two services from the list of Wasteful and Inappropriate Services Reduction (WISeR) model items and services.

Original title: “Medicare Program; Delayed Implementation of Certain Prior Authorization for Select Services for the Wasteful and Inappropriate Services Reduction (WISeR) Model”

The Department of Health and Human Services, Administration for Children and Families proposes to remove duplicative or unnecessary sections from the Runaway and Homeless Youth Program regulations (45 CFR part 1351). These amendments will streamline the Runaway and Homeless Youth Program regulations to make them more accessible to the public. The docket on https://www.regulations.gov will include a plain language summary of the NPRM.

Original title: “Reducing Bureaucracy and Burden for Children, Youth, and Family Programs”

The Food and Drug Administration (FDA or Agency) has determined that STRATTERA (atomoxetine hydrochloride) capsules 5 milligrams, 10 milligrams, 18 Milligrams, 25 Milligrams, 40 Milligrams, 60 Milligrams, 80 Milligrams, and 100 Milligrams, were not withdrawn from sale for reasons of safety or effectiveness. This determination means that FDA will not begin procedures to withdraw approval of abbreviated new drug applications (ANDAs) that refer to this drug product, and it will allow FDA to continu

Original title: “Determination That STRATTERA (Atomoxetine Hydrochloride) Capsules 5 Milligrams, 10 Milligrams, 18 Milligrams, 25 Milligrams, 40 Milligrams, 60 Milligrams, 80 Milligrams, and 100 Milligrams, Were Not Withdrawn From Sale for Reasons of Safety or Effectiveness”

In accordance with Title II of the Foundations for Evidence- Based Policymaking Act of 2018, known as the Open, Public, Electronic, and Necessary (OPEN) Government Data Act, CMS announces the forthcoming release of public data assets in open, machine-readable formats under an open license. These data are intended to support public engagement in identifying and preventing fraud, waste, and abuse, and to promote transparency and accountability. CMS has taken steps to ensure that the release of the

Original title: “Notice of Public Data Asset Release Under the Open, Public, Electronic, and Necessary (OPEN) Government Data Act”

This document corrects typographical and technical errors in the notice that appeared in the July 1, 2025, Federal Register titled "Medicare Program; Implementation of Prior Authorization for Select Services for the Wasteful and Inappropriate Services Reduction (WISeR) Model".

Original title: “Medicare Program; Implementation of Prior Authorization for Select Services for the Wasteful and Inappropriate Services Reduction (WISeR) Model; Correction”

Notice is hereby given, in accordance with the Federal Advisory Committee Act of October 6, 1972, that the Advisory Committee on Immunization Practices (ACIP), Centers for Disease Control and Prevention, Department of Health and Human Services, has been renewed for a 2-year period through April 1, 2028.

Original title: “Advisory Committee on Immunization Practices (ACIP); Notice of Charter Renewal”

Original title: “Submission for OMB Review; 30-Day Comment Request; Hazardous Waste Worker Training-42 CFR Part 65, National Institute of Environmental Health Sciences (NIEHS)”

The Trump administration is requesting about $111 billion in discretionary funding for the HHS, nearly $16 billion less than its budget in 2026.

Original title: “White House seeks 12% cut to HHS in 2027”

"Section 232" national security levies won't apply to a wide array of drugs, meaning the "overall threat to the sector should be low," according to one analyst.

Original title: “Trump revives pharma tariffs with 100% charges, but leaves loopholes”