Drug outcomes research and policies – trends and challenges
Résumé
outcomes research, pharmaceutical policies, effectiveness trials, health technology assessment, patient-reported outcomes, healthcare delivery, equityOutcomes research quantifies the results of medical treatments and/or interventions and their benefits and risks in patient populations to optimize patient outcomes and healthcare delivery and to assist all stakeholders (clinicians, patients, healthcare providers and managers, funders, policymakers and government) to make informed decisions.Outcomes research can be economic (pharmacoeconomics), clinical (comparative clinical effectiveness research), or humanistic (health-related quality of life) (Krumholz et al., 2005; Academy of Managed Care Pharmacy, 2024).It provides real-world data to support the concepts of fairness and equity (Universal Health Coverage, UHC), evidencebased healthcare, personalised medicine, and health technology assessments/costeffectiveness.By addressing individual patient care and global health, outcomes research is aligned with the United Nations Sustainable Development Goals (SDGs) adopted in 2015, most importantly with SDG 3 (Good Health and Wellbeing), but also with societal and economic ones (SDG 5 (Gender equality), SDG 9 (Industry, Innovation and Infrastructure), SDG 10 (Reduced Inequalities), and SDG 12 (Responsible Consumption and Production).This Editorial focusses on current trends and challenges in outcomes research and policy development.Medicines development programmes are designed to demonstrate quality, safety and efficacy of a novel medicinal product to obtain marketing authorisation approval.Traditionally, at least two adequate and well controlled efficacy trials in selected patient populations under strictly controlled conditions are required (Clancy and Eisenberg, 1998;Naci and Forrest, 2023).However, what is considered adequate for regulatory approval does not provide all information necessary for treatment of a diversity of patients in different regions in the world.Research efforts after marketing authorisation assess safety and effectiveness of medicines as they are disseminated beyond the clinical trial environment.In this way, phase 3 clinical efficacy trials are complemented by outcomes research to develop health policies that are safe, effective, patient-focused and cost-effective, and will often lead to re-evaluation of prior evidence and adjustments in practice, treatment guidelines and policy (Krumholz, 2009).Although the understanding of "outcomes research" has evolved over time and there is no unambiguous definition of this term (Jefford et al., 2003), there is consensus that it is defined by its aims and objectives, effectiveness of public-health interventions (including medicines) and health services, related to the individual patient perspective or to effectiveness and economic considerations of healthcare delivery (Kolte, 2017).The appropriate research methodologies to achieve these goals are applied, such as observational studies, randomized clinical trials, cost-effectiveness measures, metaanalysis, pharmacometrics and modelling, surveys, patient registries, assessment of health status or burden of disease studies.Clinical treatment outcomes are assessed in drug effectiveness trials, which -in contrast to trials designed for marketing approval -test
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