Introduction: Value-based contracts (VBCs) that link drug payments to disease-related performance metrics aim to increase the value and lower the cost of medications by aligning incentives and sharing risk between payers and pharmaceutical manufacturers. This study sought to identify outcome measures that are meaningful to key stakeholders to inform VBCs for coronary artery disease (CAD) medications.
Methods: We administered a modified Delphi survey to gather expert opinion from a diverse panel of patients (n = 9), cardiologists (n = 4), primary care physicians (n = 5), payers (n = 2), pharmacy benefits managers (n = 3), and pharmaceutical company representatives (n = 2). A list of 16 CAD-associated clinical indicators was generated from the literature and expert consultation. Delphi participants rated the importance of each outcome on a five-point Likert scale, and selected the three most meaningful outcomes. We defined consensus as ≥ 75% agreement on the importance of an outcome (Likert scores 4 or 5 or selection of an outcome as most meaningful).
Results: Eleven of 13 outcomes reached consensus for importance on the Likert scale. "Preventing heart attacks" was selected as the most meaningful outcome (80%) while "preventing death" ranked second (76%).
Conclusions: Our study results verify the utility of a widely used clinical CAD outcome measure, myocardial infarction events, for the purpose of pharmaceutical value-based contracting.
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http://dx.doi.org/10.1007/s40119-019-0132-7 | DOI Listing |
Sociol Health Illn
January 2025
Center for Public Policy and Health, Kent State University, Kent, Ohio, USA.
Fundamental Cause Theory (FCT) offers a unique middle range and longitudinal understanding of the lasting social causational relationships between certain social conditions and disease/death. In this research note, I argue that FCT should extend its outcome measures beyond physical disease and death into holistic health. I briefly review how FCT is evaluated, explore the proposed extension and discuss some operational and conceptual challenges using mental illness and positive mental health outcomes as exemplars.
View Article and Find Full Text PDFNutr Rev
December 2024
Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford OX2 6GG, United Kingdom.
Context: The use of prebiotics and probiotics as a treatment for psychiatric conditions has gained interest due to their potential to modulate the gut-brain axis. This review aims to assess the effectiveness of these interventions in reducing symptoms of depression and anxiety in psychiatric populations.
Objective: The aim was to comprehensively review and appraise the effectiveness of prebiotic, probiotic, and synbiotic interventions in reducing clinical depression and anxiety symptoms.
Ann Med
December 2025
Department of Hematology, The First Affiliated Hospital of Zhejiang Chinese Medical University (Zhejiang Provincial Hospital of Chinese Medicine), Hangzhou, China.
Objectives: Platelet transfusion refractoriness (PTR) is a frustrating clinical problem, and primary and persistent (P/P) PTR who experienced persistent PTR since the first transfusion was failed to be well recognized. This study aims to investigate the incidence and risk factors for P/P PTR.
Methods: Patients with hematologic disorders who underwent HLA high-resolution genotyping and donor-specific HLA antibody or panel reactive antibody (PRA) testing between January 2019 and March 2023 were reviewed.
Genet Med
December 2024
Movement Disorders Program, Department of Neurology and F.M. Kirby Neurobiology Center, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA. Electronic address:
Objectives: Biallelic HPDL variants have been identified as the cause of a progressive childhood-onset movement disorder, with a broad clinical spectrum from severe neurodevelopmental disorder to juvenile-onset pure hereditary spastic paraplegia type 83. This study aims at delineating the geno- and phenotypic spectra of patients with HPDL-related disease, quantitatively modelling the natural history, and uncovering genotype-phenotype associations.
Methods: A cross-sectional analysis of 90 published and one novel case was performed, employing a Human Phenotype Ontology-based approach.
Ann Med
December 2025
Department of Hematology, Affiliated Hangzhou First People's Hospital, Westlake University, School of Medicine, Hangzhou, China.
Background: The management of high-risk acute myeloid leukaemia (AML) remains challenging, highlighting the need for innovative conditioning strategies beyond current regimens.
Methods: In the present single-arm study, a FACT regimen comprised of low-dose total body irradiation (TBI) with fludarabine, cytarabine and cyclophosphamide was employed to treat cytogenetically high-risk AML patients exhibiting pre-transplant active disease. This clinical trial is registered in the Chinese Clinical Trial Registry with the registration number ChiCTR2000035111.
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