International experiences in multicriteria decision analysis (MCDA) for evaluating orphan drugs: a scoping review.

Introduction: Orphan diseases are low-prevalence conditions with chronically debilitating or life-threatening consequences. Their treatments are generally called orphan drugs (OD). Health-technology assessment processes have traditionally considered cost-effectiveness analysis (CEA), when making reimbursement and pricing decisions for health-care plans. Valuing OD with standard CEA raises important issues due to uncertain evidence, inability to meet cost-effectiveness thresholds for reimbursement and high budget impact, among others. Multi-criteria decision analysis (MCDA) allows to overcome these issues and improve the technical and ethical quality of decisions regarding prioritization, coverage, and reimbursement of OD. Areas covered: A scoping review was conducted in order to characterize MCDA frameworks for assessing OD and implementation experiences. We reviewed electronic databases (Medline, Embase, Cochrane Library, EBSCO, CINAHL, EconLit, Web of Science, LILACS, Google Scholar) key journals (Orphanet Journal of Rare Diseases and Value in Health) and organization repositories. Expert opinion: The theoretical framework for MCDA considers areas related to characteristics of orphan diseases and their technologies' clinical and economic impact. Participation processes are critical in incorporating societal values in weighting different dimensions and constructing decision rules. Local implementation pilots considering different stakeholders are necessary in order to pinpoint specific barriers and opportunities.

Conceptual framework of equity-focused implementation research for health programs (EquIR).

BACKGROUND: Implementation research is increasingly used to identify common implementation problems and key barriers and facilitators influencing efficient access to health interventions. OBJECTIVE: To develop and propose an equity-based framework for Implementation Research (EquIR) of health programs, policies and systems. METHODS: A systematic search of models and conceptual frameworks involving equity in the implementation of health programs, policies and systems was conducted in Medline (PubMed), Embase, LILACS, Scopus and grey literature. Key characteristics of models and conceptual frameworks were summarized. We identified key aspects of equity in the context of seven Latin American countries-focused health programs We gathered information related to the awareness of inequalities in health policy, systems and programs, the potential negative impact of increasing inequalities in disadvantaged populations, and the strategies used to reduce them. RESULTS: A conceptual framework of EquIR was developed. It includes elements of equity-focused implementation research, but it also links the population health status before and after the implementation, including relevant aspects of health equity before, during and after the implementation. Additionally, health sectors were included, linked with social determinants of health through the "health in all policies" proposal affecting universal health and the potential impact of the public health and public policies. CONCLUSION: EquIR is a conceptual framework that is proposed for use by decision makers and researchers during the implementation of programs, policies or health interventions, with a focus on equity, which aims to reduce or prevent the increase of existing inequalities during implementation.