NATIONAL INSTITUTES OF HEALTH

Senators Marsha Blackburn (R-TN) and Mark Kelly (D-AZ) introduced a bill to allow state Medicaid programs to develop direct primary care models. CDC, FDA, NIH, and HRSA released various notices and guidance documents. House and Senate leadership negotiate an end-of-year funding package. President-elect Donald Trump announced nominations for CDC Director, NIH Director, FDA Commissioner, and Surgeon General.

Epilepsy care advancements, including new antiepileptic drugs like fenfluramine for Dravet and LGS, were highlighted at the 2024 AES Annual Meeting. UCB presented extensive data on fenfluramine's efficacy and safety, focusing on real-world evidence and medication adjustments. Brad Chapman emphasized UCB's commitment to addressing unmet needs, particularly for women of childbearing age, through initiatives like CONCEPTION and partnerships with NIH. The conference underscored ongoing efforts to improve epilepsy care and support for those with rare syndromes.

Feinberg's implementation science initiatives, led by Sara Becker, aim to bridge research and practice, with rapid growth seen since 2022. Key achievements include the establishment of the Center for Dissemination and Implementation Science (CDIS), new grants, and collaborations across Northwestern, focusing on improving public health and health equity.

Ally Schott's early endometriosis diagnosis at 12 led to limited treatment options and self-taught research. Despite its prevalence affecting 10% of women globally, endometriosis remains underfunded and poorly understood, with only two new drugs approved in the U.S. for pain management in the past decade. Women's health research generally suffers from insufficient funding and information deficits, hindering drug development. Recent federal efforts, including a $100 million investment by the Biden administration, aim to address these gaps, but future priorities under a new administration remain uncertain.

AbbVie announced positive Phase 3 trial results for tavapadon, a Parkinson's disease treatment, meeting primary and secondary endpoints. The company plans to seek FDA approval in 2025.

Healthcare faces significant diversity issues due to limited evidence from clinical trials, with 70% of the U.S. population excluded. Women and minorities often lack representation, leading to disparities in treatment outcomes. AI advancements can bridge these gaps by generating real-world evidence, enhancing data quality, and personalizing care, ultimately transforming evidence-based healthcare.

The article advocates for a comprehensive framework to incentivize the discovery, validation, and regulatory approval of biomarkers to accelerate clinical trials and increase the availability of health-extending drugs. It highlights the socio-economic costs of biological aging and the need for new solutions to target diseases before they become life-threatening. The proposed framework includes establishing a shared repository for biomarker data, creating a streamlined regulatory pathway within the FDA, and implementing data-sharing incentives such as Priority Review Vouchers (PRVs). The article emphasizes the urgent need for federal action to address market failures and misaligned incentives, which currently hinder the extension of healthy lifespans.

Coauthors from UC San Diego, Columbia University, GSK, Scripps Research, Harvard, and other institutions collaborated on a study funded by the Bill and Melinda Gates Foundation, NIH, and National Institute for General Medical Sciences. The authors declare no competing interests.

Global Nanopharmaceuticals Market expected to grow from USD 7.5 Billion in 2023 to USD 259.2 Billion by 2032, driven by advantages like enhanced bioavailability, targeted delivery, and reduced side effects. North America leads with USD 25.3 Billion in 2023. Nanopharmaceuticals revolutionize drug delivery, addressing complex diseases and overcoming traditional therapy limitations.

Sen. Patty Murray opposes Robert F. Kennedy Jr. as Health and Human Services secretary, citing his anti-vaccine stance. Kennedy, chosen by President-elect Trump, has questioned vaccine safety and promoted debunked claims. Murray highlights the bipartisan support for vaccines and public health research, noting the politicization of COVID vaccines has increased vaccine hesitancy. Washington state has seen a rise in whooping cough cases and a decline in childhood vaccination rates, raising concerns about herd immunity.