National Health Council

The United States Inflation Reduction Act of 2022 established the Medicare Drug Price Negotiation Program, directing the Centers for Medicare and Medicaid Services (CMS) to establish "maximum fair prices" for select drugs. In arriving at maximum fair prices, CMS is required to consider several criteria, including the extent to which a selected drug addresses unmet medical need (UMN). Through a targeted literature review of 48 original research studies, we identified and categorized 40 potential elements of UMN as they relate to pharmaceuticals, as well as treatment-related barriers to addressing this UMN, from the patient, caregiver, and societal perspectives. We synthesized these elements of UMN into seven domains: (1) traditional clinical effectiveness, (2) impacts on patient, caregiver, or family quality of life, (3) economic burden on the patient, caregiver, or family, (4) economic burden on society, (5) societal perspective elements, (6) elements of treatment administration, and (7) availability of other treatments. Comparing these elements with the United States Food and Drug Administration guidance regarding UMN reveals potential key gaps in the current CMS approach to assessing UMN in the Medicare Drug Price Negotiation Program, particularly regarding domains 2 through 6, including quality of life, economic burdens, societal elements, and treatment administration.

Metabolic dysfunction-associated steatotic liver disease (MASLD) and steatohepatitis (MASH) are associated with adverse clinical outcomes, impaired health-related quality of life, and significant economic burden. The growing burden of MASLD and MASH has led to the publication of a large number of MASLD/MASH guidelines by national and international societies. However, important differences among the recommendations have created confusion, contributing to a low implementation rate and suboptimal management of MASLD and MASH. Creating a consensus recommendation has become more important since the approval of a selective agonist of thyroid hormone β receptor (resmetirom) for MASH treatment in the United States. We built a consensus among the most recently published recommendations for MASLD/MASH. A comprehensive search for MASLD and MASH guidelines, guidance documents, or similar publications from January 2018 to January 2025 using PubMed, Embase, Web of Science, and society websites was conducted. Each selected document was assessed across 8 specific domains with 145 variables. Variables with <50% concordance were used for the Delphi statement development. A supermajority threshold of 67% was set for statement acceptance. There were 61 documents published from 2018 through January 2025. Four rounds of Delphi were conducted: 46 statements were generated for Round 1, 32 statements for Round 2, 16 statements for Round 3, and 8 statements for Round 4, whereby 100% of statements achieved a greater than 90% agreement. All final consensus recommendations were summarized in tables and algorithms. Our study provides an extensive set of recommendations generated based on a comprehensive review of the most recent MASLD/MASH guidelines and a consensus-building process.

Achieving equitable access to medicines requires understanding of how pharmaceutical use and spending vary by race and ethnicity across the US. To quantify variation in prescription drug utilization and spending per capita and per prevalent case by race, ethnicity, health condition, payer, and US state. In this cross-sectional study, the US Disease Expenditure project was extended to incorporate disaggregation by race and ethnicity for state-level retail prescription drug utilization and spending-in addition to 143 health conditions, 38 age and sex groups, and 4 payers (Medicare, Medicaid, private insurance, and out of pocket)-across the 2019 population in all 50 states and Washington, DC. Data were analyzed from October 2023 to April 2025. Four mutually exclusive racial and ethnic groups (Asian or Pacific Islander, Black, Hispanic, and White). Outcomes include prescriptions dispensed and spending for retail pharmaceuticals. Estimates were standardized by population size, population age, and-where data permitted-by disease burden (52 conditions). Das Gupta decomposition was used to estimate the relative contribution of 3 factors (disease prevalence, prescriptions per prevalent case, and spending per prescription) on observed disparities in age-standardized per capita pharmaceutical spending. In 2019, age-standardized pharmaceutical utilization and spending per person with a given disease was substantially lower than the all-population mean for Black populations, close to the mean for Hispanic populations, and often higher than the mean for Asian or Pacific Islander and White populations. These trends-particularly those for the Black population-were generally consistent across 52 health conditions but varied widely across payers and US states. The decomposition analysis for these 52 conditions showed that differences in per capita pharmaceutical spending across race and ethnicity groups were primarily explained by disease prevalence for Black populations (associated with increased per capita spending) and by utilization rates per prevalent case for Hispanic populations (also associated with increased spending). In contrast, differences in drug price or product type (spending per prescription) contributed less to observed spending disparities. In this cross-sectional study, racial and ethnic disparities in medication use persisted, most notably the underutilization of medicines relative to disease burden among Black populations. These patterns varied by state, highlighting the need for local- and condition-specific approaches to advancing pharmacoequity in the US.

CRISPR-Cas systems have revolutionized molecular diagnostics through their specificity and programmability, yet their broad adoption is hindered by the reliance on expensive and complex instrumentation. Here, we present an optimized quantum dot (QD) molecular beacon (QD-MB) platform that integrates Förster resonance energy transfer (FRET)-based detection with CRISPR-Cas functionality, achieving sub-picomolar sensitivity without the need for target amplification. By systematically tuning components, including His-tag modifications for improved QD conjugation, nucleic acid hairpin structures for enhanced enzyme interaction, and QD surface passivation strategies, we demonstrate a two-order-of-magnitude improvement in detection sensitivity. Using LwaCas13a and RNA targets, the limit of detection (LOD) decreased to under 1 pM with plate-reader-based fluorescence measurements and below 10 pM with a lamp-and-smartphone setup, establishing the feasibility of portable, field-ready applications. This work highlights the transformative potential of QD-MBs in biosensing and sets a foundation for further advances in CRISPR-based diagnostics and nanotechnology-enabled sensing platforms.

Utilization management criteria influence patients' access to specialty drugs, yet the processes used by health plans to establish these criteria are not well understood. This study examined the alignment between clinical practice guidelines and plans' utilization management criteria. Using the Tufts Medicine Specialty Drug Evidence and Coverage Database (December 2023), we reviewed US-based guidelines for 389 drug-indication pairs, excluding oncology and biosimilar treatments. We categorized guidelines as recommending or not recommending utilization management, based on alignment with each drug's Food and Drug Administration label-specifically, line of therapy and clinical requirements (for example, disease severity). We analyzed 5,699 coverage policies from eighteen large commercial health plans. When guidelines recommended utilization management, 67 percent of plans' coverage decisions aligned with the recommendation; when guidelines did not recommend utilization management, only 37 percent of decisions were consistent. Most plans imposed utilization management criteria (61 percent of all decisions), and plans were more likely to be consistent with guideline recommendations when utilization management was recommended, indicating a weak alignment with guideline recommendations.

[This corrects the article DOI: 10.3389/fpubh.2024.1493331.].

BackgroundThe Inflation Reduction Act’s (IRA) Drug Price Negotiation Program (DPNP) may reduce incentives for industry investments in post-approval clinical development. We aimed to explore the IRA’s impact on the initiation of industry-sponsored, post-approval clinical trials.MethodsUsing Citeline’s Trialtrove database (7/2014–8/2024), we conducted an interrupted time series analysis (ITSA) to estimate the IRA’s impact on the initiation of industry-sponsored Phase I-III trials in previously approved drugs, excluding all vaccines and COVID-19 treatments. We conducted an additional ITSA to examine post-IRA changes in government-funded trials, hypothesized to be unaffected by the IRA, and sensitivity analyses to explore potential exogenous confounding factors. Finally, we explored differences in the IRA’s impact on post-approval industry-sponsored clinical trial initiation in small versus large molecule drugs.ResultsFollowing the IRA’s passage, the average monthly number of industry-sponsored trials on post-approval drugs decreased by 38.4%. The ITSA indicated that the IRA’s passage was associated with an immediate drop of 11.1 industry-sponsored trials (p-value < 0.05) and an additional decrease by 0.9 trials per month (p-value < 0.01). The IRA’s passage was not statistically associated with changes in government-funded trial initiation. Sensitivity analyses supported ITSA findings. Initiation of post-approval industry-sponsored trials decreased by 47.3% and 32.9% for small and large molecule drugs, respectively.ConclusionsThe IRA’s passage was associated with reductions in industry-sponsored, but not government-funded, post-approval trials, with larger reductions for small molecule drugs. These findings provide early evidence supporting concerns around IRA-related reductions in incentives for post-approval clinical development.