BACKGROUND: Medicines that are based on known molecules and are further developed to address healthcare needs and deliver relevant improvement for patients, healthcare professionals and/or payers are called value-added medicines (VAMs). The evaluation process of VAMs is heterogeneous across countries, and it has been primarily designed for originator pharmaceuticals with confirmatory evidence collected alongside pivotal clinical trials. There is a mismatch between evidence requirements by public decision-makers and evidence generated by manufacturers of VAMs. Our objective was to develop a core evaluation framework for VAMs. METHODS: Potential benefits offered by VAMs were collected through a systematic literature review and allocated to separate domains in an iterative process. The draft list of domains and their applicability were validated during two consecutive virtual workshops by health policy experts representing countries with different economic statuses, geographical and decision-making contexts. RESULTS: Based on 158 extracted studies, the final consensus on the evaluation framework resulted in 11 value domains in 5 main clusters, including unmet medical needs, health gain (measured by health care professionals), patient-reported outcomes, burden on households, and burden on the health care system. CONCLUSIONS: The proposed framework could reduce the heterogeneity in value assessment processes across countries and create incentives for manufacturers to invest in incremental innovation. However, some domains may not be equally relevant or accepted in all countries, therefore the core framework needs thorough adaptation in specific jurisdictions.

Andalusian School of Public Health Granada Spain

Association of Health Insurance Companies Prague Czech Republic

Center for Health Technology Assessment Semmelweis University Üllői rd 25 Budapest 1085 Hungary

Department of Organisation and Management of Pharmacy Faculty of Pharmacy Comenius University in Bratislava Bratislava Slovakia

Department of Public Health Policies Sector of Health Systems and Policy School of Public Health University of West Attica Athens Greece

Syreon Research Institute Mexikói str 65 A Budapest 1142 Hungary

Technische Hochschule Nürnberg Nürnberg Germany

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Development of a core evaluation framework of value-added medicines: report 2 on pharmaceutical policy perspectives