Clinical Meaningfulness in Alzheimer's Disease Clinical Trials. A Report from the EU-US CTAD Task Force.

Recent positive results of three phase III anti-amyloid monoclonal antibody trials are transforming the landscape of disease-modifying therapeutics for Alzheimer's disease, following several decades of failures. Indeed, all three trials have met their primary endpoints. However, the absolute size of the benefit measured in these trials has generated a debate on whether the change scores observed on clinical outcome assessments represent a clinically meaningful benefit to patients. An evidence-based conclusion is urgently required to inform decision-making related to the approval, reimbursement, and ultimately, the management of emerging therapies in clinical practice. The EU-US CTAD Task Force met in Boston to address this important question. The current state-of-the-art knowledge for interpreting clinical meaningfulness of AD clinical trial results, including the point of view of patients and study partners on what is clinically meaningful, was discussed and is summarized here. A combination of methodologies to address the challenges emerged. There remain gaps in the understanding of clinical meaningfulness that only long-term longitudinal studies will be able to address.

Temporary FDA Advisory Committee Members, Recommendations, and Agency Actions, 2017-2021.

This cross-sectional study examines the recommendations and agency actions of temporary and permanent US Food and Drug Administration advisory committee members from 2017 to 2021.

Hospital Diversity, Equity, and Inclusion Efforts: Perspectives of Patient and Family Advisors.

BACKGROUND AND OBJECTIVES: Patient and family advisory councils are common within children's hospitals. However, lack of diversity among patient and family advisors (PFAs) may result in exclusion of crucial perspectives and perpetuate inequities. We sought to understand PFA perspectives on how children's hospitals should approach: (1) recruitment and support of PFAs from groups at greater risk of health inequities; and (2) development of meaningful partnerships with PFAs or patient and family advisory councils on institutional diversity, equity, and inclusion (DEI) efforts. METHODS: We conducted a qualitative study of PFAs of children's hospitals from communities at greater risk for health inequities based on self-identified race, ethnicity, gender, socioeconomic status, disability, language, or other factors. Focus groups were virtual and group discussions were recorded, transcribed, and analyzed using inductive qualitative analysis. RESULTS: In total, 17 PFAs participated across 5 focus groups (4 in English, 1 in Spanish). We identified 6 themes: (1) PFA diversity is necessary to understand existing health inequities; (2) diversity needs to be considered broadly; (3) recruiting for diverse PFAs requires intentionality, visibility of PFACs within and outside of the hospital, and deliberate connections with families and communities; (4) efforts to increase PFAC diversity must be accompanied by work to develop inclusive environments; (5) diversity efforts require meaningful engagement and equity; and (6) diverse PFACs can enrich DEI efforts but require organizational commitment and follow-through. CONCLUSIONS: Insights from our qualitative study of PFAs can be used by healthcare systems to foster diversity and inclusion in PFACs and advance hospital DEI efforts.

Bridging the Gap: A Community Advisory Board Promoting Community Engagement in Cancer Research for Ethnically Diverse Populations.

Prostate cancer disproportionately affects Black men in the United States, leading to higher mortality rates and health disparities. In addition, based on historical mistreatment and discrimination and the resulting distrust of the medical system, Black populations are consistently underrepresented in health care-related research. Addressing these challenges requires community-driven approaches integrating diverse perspectives and fostering equitable health outcomes. This article describes the formation and impact of The Multidisciplinary Health Outcomes Research and Economics (MORE) Lab Community Advisory Board (CAB) at The University of Oklahoma Health Sciences. We purposefully recruited Black men with CaP and Black health care professionals to serve on a CAB and advise on ongoing research to address quality of life (QoL) issues in ethnically diverse Black CaP survivors. The CAB seeks to mitigate CaP disparities and improve health equity by empowering Black voices and promoting collaborative research practices. The MORE Lab CAB has successfully provided a venue for community members to contribute to designing a culturally relevant research program to improve the QoL in ethnically diverse Black men with CaP. The CAB has been instrumental in developing research goals and tools, implementing a series of town hall meetings to educate and support Black CaP survivors, and disseminating research findings. In conclusion, CABs are potentially critical in guiding research, enhancing community engagement, and advocating for culturally responsive health interventions.

Navigating high-cost medicines: summary of the Guiding Principles for the governance of high-cost medicines in Australian hospitals.

High-cost medicines (HCMs) can be clinically impactful for individual patients but are also subject to variable funding mechanisms. Public hospitals and health services are often asked to fund HCMs, but inconsistent processes frequently create large variations in care. CATAG (Council of Australian Therapeutic Advisory Groups) is the Australian peak national advisory body for the quality use of medicines in hospitals and health services, with all states and territories collaborating to support Drug and Therapeutics Committees (DTCs). CATAG has developed national Guiding Principles to assist DTCs to effectively govern HCMs. An established process for the development of CATAG Guiding Principles was undertaken to develop these Guiding Principles. This includes the formation of an Expert Advisory Group (EAG) comprising individuals with recognised expertise, development of draft principles and stakeholder consultation from within the CATAG membership and externally. All feedback was discussed, and changes were agreed upon. The final version was approved by the EAG and CATAG members. This document represents a summary of the seven Guiding Principles developed, covering the areas of governance, application and assessment, communication, training and resourcing. Although many outstanding priorities still exist, including the development of national coordination regarding HCM assessment, these Guiding Principles offer a basis to navigate this complex area.

Use of 21-Valent Pneumococcal Conjugate Vaccine Among U.S. Adults: Recommendations of the Advisory Committee on Immunization Practices - United States, 2024.

On June 17, 2024, the Food and Drug Administration approved 21-valent pneumococcal conjugate vaccine (PCV) (PCV21; CAPVAXIVE; Merck Sharp & Dohme, LLC) for adults aged ≥18 years. PCV21 does not contain certain serotypes that are included in other licensed pneumococcal vaccines but adds eight new serotypes. The Advisory Committee on Immunization Practices (ACIP) recommends use of a PCV for all adults aged ≥65 years, as well as adults aged 19-64 years with certain risk conditions for pneumococcal disease if they have not received a PCV or whose vaccination history is unknown. Previously, options included either 20-valent PCV (PCV20; Prevnar20; Wyeth Pharmaceuticals, Inc.) alone or a 15-valent PCV (PCV15; VAXNEUVANCE; Merck Sharp & Dohme, LLC) in series with 23-valent pneumococcal polysaccharide vaccine (PPSV23; Pneumovax23; Merck Sharp & Dohme, LLC). Additional recommendations for use of PCV20 exist for adults who started their pneumococcal vaccination series with 13-valent PCV (PCV13; Prevnar13; Wyeth Pharmaceuticals, Inc.). The ACIP Pneumococcal Vaccines Work Group employed the Evidence to Recommendations framework to guide its deliberations on PCV21 vaccination among U.S. adults. On June 27, 2024, ACIP recommended a single dose of PCV21 as an option for adults aged ≥19 years for whom PCV is currently recommended. Indications for PCV have not changed from previous recommendations. This report summarizes evidence considered for these recommendations and provides clinical guidance for use of PCV21.

Use of Haemophilus influenzae Type b-Containing Vaccines Among American Indian and Alaska Native Infants: Updated Recommendations of the Advisory Committee on Immunization Practices - United States, 2024.

Invasive Haemophilus influenzae type b (Hib) disease is a serious bacterial infection that disproportionally affects American Indian and Alaska Native (AI/AN) populations. Hib vaccination with a monovalent Hib conjugate vaccine consisting of Hib capsular polysaccharide (polyribosylribitol phosphate [PRP]) conjugated to outer membrane protein complex of Neisseria meningitidis serogroup B, PRP-OMP (PedvaxHIB, Merck and Co., Inc.) has historically been preferred for AI/AN infants, who are at increased risk for invasive Hib disease, because it provides substantial protection after the first dose. On June 26, 2024, CDC's Advisory Committee on Immunization Practices (ACIP) recommended that a hexavalent, combined diphtheria and tetanus toxoids and acellular pertussis (DTaP), inactivated poliovirus (IPV), Hib conjugate, and hepatitis B (HepB) vaccine, DTaP-IPV-Hib-HepB (Vaxelis, MSP Vaccine Company) should be included with monovalent PRP-OMP in the preferential recommendation for AI/AN infants because of the PRP-OMP Hib component. A primary Hib vaccination series consisting of either 1) monovalent PRP-OMP (2-dose series at ages 2 and 4 months) or 2) DTaP-IPV-Hib-HepB (3-dose series at ages 2, 4, and 6 months) is preferred for AI/AN infants. DTaP-IPV-Hib-HepB is only indicated for use in infants at ages 2, 4, and 6 months and should not be used for the booster doses of Hib, DTaP, or IPV vaccines. For the booster dose of Hib vaccine, no vaccine formulation is preferred for AI/AN children; any Hib vaccine (except DTaP-IPV-Hib-HepB) should be used. This report summarizes evidence considered for these recommendations and provides clinical guidance for the use of Hib-containing vaccines among AI/AN infants and children.