The recent High-Level Meeting on rare diseases in Brussels has laid the groundwork for a comprehensive action plan to enhance Europe's response to rare diseases. This conference highlighted the need for collaboration among stakeholders to improve patient outcomes and access to treatments.

A recent NIH-supported survey reveals that drug use among U.S. teens remains at historic lows, indicating a significant trend in abstention. This finding may influence future public health strategies and interventions targeting adolescent substance use.

The Office of Management and Budget is reviewing a temporary extension for the Sexual Risk Avoidance Education Program Performance Analysis Study, with revisions planned for 2026. This study aims to evaluate the effectiveness of sexual risk avoidance education initiatives.

The HHS has issued corrections to the FY 2026 Inpatient Rehabilitation Facility Prospective Payment System final rule, addressing typographical and technical errors. The effective date for these updates remains October 1, 2025.

NORD commends HHS for including metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD) in the Recommended Uniform Screening Panel, enhancing early detection for these rare diseases. This decision supports better patient outcomes through timely diagnosis.

The U.S. Department of Health and Human Services has officially added Duchenne muscular dystrophy and Metachromatic Leukodystrophy to the Federal Recommended Uniform Screening Panel. This decision enhances early detection and intervention for these rare diseases, impacting newborn screening practices nationwide.

EURORDIS applauds the European Commission's introduction of the Biotech Act I, which aims to enhance Europe's innovation capacity in rare disease medicine. This initiative is seen as a significant step forward for the rare disease community.

The CDC has implemented individual-based decision-making for hepatitis B immunization in infants born to mothers who test negative for the virus. This policy shift aims to enhance personalized healthcare approaches in immunization practices.

The FDA has released final guidance on safety reporting requirements for investigational new drug applications and bioavailability studies. This guidance aims to assist sponsors in meeting compliance standards for safety assessments.

The FDA has released final guidance on safety reporting responsibilities for clinical investigators involved in investigational drug and device studies. This guidance aims to enhance compliance with safety reporting requirements for IND and IDE studies.

The FDA has issued a reminder to the food industry about its legal obligations for food recalls, particularly in light of a recent infant botulism outbreak linked to ByHeart infant formula. The agency is urging companies to adopt best practices to enhance recall effectiveness.

The FDA has lifted a significant restriction on the use of real-world evidence (RWE) in drug and device application reviews, potentially streamlining the approval process. This change could enhance the incorporation of patient data into regulatory decisions, impacting future drug development.

FDA has awarded a national priority voucher for teclistamab in combination with daratumumab, following strong Phase 3 study results for relapsed/refractory multiple myeloma. This designation may expedite the development and review process for this treatment.

The 41st Workshop of the EURORDIS Round Table of Companies will focus on aligning biotechnology innovation with the needs of rare disease patients in Europe. Scheduled for February 25, 2026, in Brussels, the workshop aims to discuss the upcoming Biotech Act as a key legislative tool for advancing rare disease priorities.

Ralph Abraham, M.D., has been sworn in as the Principal Deputy Director of the CDC, a key position that may influence public health policy and initiatives. His experience as a Louisiana Surgeon General could bring valuable insights to the CDC's efforts.