Tangible and intangible benefits were identified with respect to the interests of the parties involved. This has led to the incorporation of innovation for patients under acceptable conditions.

Objectives: Publications assessing health and economic outcomes of risk-sharing arrangements (RSAs) are limited. Better knowledge of these outcomes would shed light on the pertinence of such arrangements, informing design improvements for the future. The aim of the study is to describe the different types of RSAs implemented in Catalonia and their health and economic outcomes.Methods: Retrospective descriptive analysis of RSAs implemented from January 2016 to December 2019 in the Catalan Health Service, CatSalut. Individual RSAs were reviewed and categorized according to standard RSA guidelines. Relevant health and economic outcomes pertaining to the RSAs were analyzed using aggregate data recorded in Catalan central registries.Results: A total of 15 RSAs were implemented over the study period (10 of which are still ongoing). A total of 8 consisted of performance-linked reimbursements (PLRs) and 7 of cost-sharing arrangements (CSAs). The arrangements were implemented in the oncohematology (n = 11), rare disease (n = 3), and neurology (n = 1) areas. A total of 951 patients were included in PLR and 73% achieved the target health outcomes. Total medication costs were V9 295 755 of which 11% were refunded to CatSalut. CSAs involved 2066 patients and resulted in overall refunds of V1 349 564 (2.61%) for CatSalut.Conclusions: Both PLRs and CSAs were used to manage the different uncertainties related to accessing innovative medicines in Catalonia. The data generated provide relevant information to inform decision-making, allowing an adaptation of the initial recommendation for use and access. Additional efforts are required to increase the RSA assessments and their publication.

Early access to medicines allows the prescription of a medicine before it is available in the public formulary to patients with severe or rare diseases with high unmet needs who have no authorised therapeutic alternatives available. In this context, consistent decision making is difficult, and a systematic assessment procedure could be useful to tackle complex situations and guarantee the equity of medicines’ access. A multidisciplinary panel (MP) conducted four workshops to develop an early access framework based on a reflective multiple criteria decision analysis (MCDA). A set of 12 criteria was agreed: eight quantitative (severity of disease, urgency, efficacy, safety, internal and external validity, therapeutic benefit and plausibility) and four qualitative (therapeutic alternative, existence of precedents, management impact and costs). Quantitative criteria were weighted using a five-point scale. The relative importance of quantitative criteria had mean weights from 4.7 to 3.6, showing its relevance in the decisions. The framework was tested using two case studies, and reliability was assessed by re-test. The re-test revealed no statistical differences, indicating the consistency and replicability of the framework developed. MCDA may help to structure discussions for heterogeneous treatment requests, providing predictability and robustness in decision making involving sensitive and complex situations.

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