Implementing Risk-Sharing Arrangements for Innovative Medicines: The Experience in Catalonia (Spain)

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 euro9 295 755 of which 11% were refunded to CatSalut. CSAs involved 2066 patients and resulted in overall refunds of euro1349564 (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: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain)

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

Changes in treatment patterns and costs for lung cancer have not resulted in relevant improvements in survival: a population-based observational study in Catalonia

Objective: Few published studies have described multidisciplinary therapeutic strategies for lung cancer. This study aims to describe the different approaches used for treating lung cancer in Catalonia in 2014 and 2018 and to assess the associated cost and impact on patient survival. Methods: A retrospective observational cohort study using data of patients with lung cancer from health care registries in Catalonia was carried out. We analyzed change in treatment patterns, costs and survival according to the year of treatment initiation (2014 vs. 2018). The Kaplan-Meier method was used to estimate survival, with the follow-up until 2021. Results: From 2014 to 2018, the proportion of patients undergoing surgery increased and treatments for unresectable tumors decreased, mainly in younger patients. Immunotherapy increased by up to 9% by 2018. No differences in patient survival were observed within treatment patterns. The mean cost per patient in the first year of treatment increased from EUR 14,123 (standard deviation [SD] 4327) to EUR 14,550 (SD 3880) in surgical patients, from EUR 4655 (SD 3540) to EUR 5873 (SD 6455) in patients receiving curative radiotherapy and from EUR 4723 (SD 7003) to EUR 6458 (SD 10,116) in those treated for unresectable disease. Conclusions: From 2014 to 2018, surgical approaches increased in younger patients. The mean cost of treating patients increased, especially in pharmaceutical expenditure, mainly related to the use of several biomarker-targeted treatments. While no differences in overall patient survival were observed, it seems reasonable to expect improvements in this outcome in upcoming years as more patients receive innovative treatments

Implementing reflective multicriteria decision analysis (MCDA) to assess orphan drugs value in the Catalan Health Service (CatSalut)

Catalan healthcare; Decision-making; Multi-criteria decision analysis; Orphan drugsSanitat catalana; Presa de decisions; Anàlisi de decisions multicriteri; Medicaments orfesSanidad catalana; Toma de decisiones; Análisis de decisiones multicriteria; Medicamentos huérfanosBACKGROUND: Orphan medicines show some characteristics that hinder the evaluation of their clinical added value. The often low level of evidence available for orphan drugs, together with a high budget impact and an incremental cost-effectiveness ratio many times higher than drugs used for non-orphan diseases, represent challenges in their appraisal and effective access to clinical use. In order to explore how to handle these hurdles, the Catalan Health Service (CatSalut) began an initiative on a multidimensional assessment of drugs value during the appraisal process. Reflective multicriteria decision analysis (MCDA) using analytical methods was chosen, since it may help to standardise and contextualize all the relevant data related with the drug that could contribute to a decision. The aim of the study was to determine whether the implementation of reflective MCDA methodology could support the decision-making process about orphan medicines in the context of CatSalut. METHODS: The assessment and decision-making process for orphan drugs in the Programa d'Harmonització Farmacoterapeutica (PHF) of CatSalut was prioritized to test the implementation of the reflective MCDA both a qualitative and quantitatively. A staged approach was used with the following main steps: selection and structuration of quantitative criteria (Core Model) and qualitative criteria (Contextual Tool), framework scoring and assessment of three orphan drug case studies. This proof-of-concept would grant a continued refinement of the methodology and, if and when validated, its potential integration to other therapeutic areas of the PHF. RESULTS: The final framework was composed by 10 quantitative criteria (Core Model) and 4 qualitative criteria (Contextual Tool) according to the PHF goals being the most important criteria "disease severity", "unmet need", "comparative effectiveness" and "comparative safety /tolerability". The matrix developed for the case studies served as a guide for the selection of the essential information that the decision-makers were expected to include in a framework. The reflective discussion was considered the most relevant phase of the approach to support inputs for health decision-making processes reflecting both drug value and place in therapy. CONCLUSIONS: The study showed that reflective MCDA methodology could be implemented to complement the decision-making process in CatSalut, as an aid to determine the clinical added value for orphan medicines. MCDA provided transparency and a structured discussion during the committee meetings, thus increasing transparency and predictability of the relevant items supporting the agreements adopted on orphan drugs access

Characterization of the Pharmaceutical Risk-Sharing Arrangement Process in Catalonia

Pharmaceutical risk-sharing arrangements have emerged as a reasonable tool to promote sustainable access to innovative medicines with uncertain clinical evidence and/or economic impact from the payer perspective. These funding mechanisms pose an alternative option to the traditional fixed-price methods and are intended to align the price of medication with the value delivered in treating patients, balancing clinical need with affordability in the face of increasing therapeutic innovation and ever-tight budgets. The Catalan Health Service (CatSalut) has set up a systematic, traceable, and transparent methodology for the design and implementation of risk-sharing arrangements and 15 of such access schemes have been successfully implemented until December 2019. Our experience has acknowledged the need for a robust study design, appropriate financial, technical, and administrative resources, and strong stakeholder commitment and communication as critical to the success of risk-sharing arrangements. While the experience in Catalonia has been positive and has served to highlight the potential of such schemes in tackling public health policy concerns, this exchange can often be undermined by the lack of transparency surrounding risk-sharing arrangements and the fact that the literature related to their methodology, implementation, and impact is scarce. Further studies should be conducted and shared to address this obstacle

Trends in lung cancer incidence by age, sex and histology from 2012 to 2025 in Catalonia (Spain)

Lung cancer remains one the most common cancers in Europe and ranks frst in terms of cancer mortality in both sexes. Incidence rates vary by region and depend above all on the prevalence of tobacco consumption. In this study we describe recent trends in lung cancer incidence by sex, age and histological type in Catalonia and project changes according to histology by 2025. Bayesian age period-cohort models were used to predict trends in lung cancer incidence according to histological type from 2012 to 2025, using data from the population-based Catalan cancer registries. Data suggest a decrease in the absolute number of new cases in men under the age of 70 years and an increase in women aged 60 years or older. Adenocarcinoma was the most common type in both sexes, while squamous cell carcinoma and small cell carcinoma were decreasing signifcantly among men. In both sexes, the incident cases increased by 16% for patients over 70 years. Increases in adenocarcinoma and rising incidence in elderly patients suggest the need to prioritize strategies based on multidisciplinary teams, which should include geriatric specialist

Changes in Treatment Patterns and Costs for Lung Cancer Have Not Resulted in Relevant Improvements in Survival : A Population-Based Observational Study in Catalonia

Real-world data collect clinical and economic information from daily clinical practice and can support decisions in the context of health evaluation and management. The aim of our retrospective cohort study was to describe the different approaches used for treating lung cancer in Catalonia in 2014 and 2018 and to assess the associated cost and impact on patient survival until December 2021. Treatment patterns for lung cancer changed in younger patients, and all costs of treatment increased significantly. These changes, mainly related to the use of several novel drugs, such as immunotherapy and targeted therapy, were not associated with an increase in the overall patient survival in the period of time under evaluation. Objective: Few published studies have described multidisciplinary therapeutic strategies for lung cancer. This study aims to describe the different approaches used for treating lung cancer in Catalonia in 2014 and 2018 and to assess the associated cost and impact on patient survival. A retrospective observational cohort study using data of patients with lung cancer from health care registries in Catalonia was carried out. We analyzed change in treatment patterns, costs and survival according to the year of treatment initiation (2014 vs. 2018). The Kaplan-Meier method was used to estimate survival, with the follow-up until 2021. From 2014 to 2018, the proportion of patients undergoing surgery increased and treatments for unresectable tumors decreased, mainly in younger patients. Immunotherapy increased by up to 9% by 2018. No differences in patient survival were observed within treatment patterns. The mean cost per patient in the first year of treatment increased from EUR 14,123 (standard deviation [SD] 4327) to EUR 14,550 (SD 3880) in surgical patients, from EUR 4655 (SD 3540) to EUR 5873 (SD 6455) in patients receiving curative radiotherapy and from EUR 4723 (SD 7003) to EUR 6458 (SD 10,116) in those treated for unresectable disease. From 2014 to 2018, surgical approaches increased in younger patients. The mean cost of treating patients increased, especially in pharmaceutical expenditure, mainly related to the use of several biomarker-targeted treatments. While no differences in overall patient survival were observed, it seems reasonable to expect improvements in this outcome in upcoming years as more patients receive innovative treatments

Implementing reflective multicriteria decision analysis (MCDA) to assess orphan drugs value in the Catalan Health Service (CatSalut)

Background: orphan medicines show some characteristics that hinder the evaluation of their clinical added value. The often low level of evidence available for orphan drugs, together with a high budget impact and an incremental cost-effectiveness ratio many times higher than drugs used for non-orphan diseases, represent challenges in their appraisal and effective access to clinical use. In order to explore how to handle these hurdles, the Catalan Health Service (CatSalut) began an initiative on a multidimensional assessment of drugs value during the appraisal process. Reflective multicriteria decision analysis (MCDA) using analytical methods was chosen, since it may help to standardise and contextualize all the relevant data related with the drug that could contribute to a decision. The aim of the study was to determine whether the implementation of reflective MCDA methodology could support the decision-making process about orphan medicines in the context of CatSalut. Methods: the assessment and decision-making process for orphan drugs in the Programa d'Harmonització Farmacoterapeutica (PHF) of CatSalut was prioritized to test the implementation of the reflective MCDA both a qualitative and quantitatively. A staged approach was used with the following main steps: selection and structuration of quantitative criteria (Core Model) and qualitative criteria (Contextual Tool), framework scoring and assessment of three orphan drug case studies. This proof-of-concept would grant a continued refinement of the methodology and, if and when validated, its potential integration to other therapeutic areas of the PHF. Results: the final framework was composed by 10 quantitative criteria (Core Model) and 4 qualitative criteria (Contextual Tool) according to the PHF goals being the most important criteria "disease severity", "unmet need", "comparative effectiveness" and "comparative safety /tolerability". The matrix developed for the case studies served as a guide for the selection of the essential information that the decision-makers were expected to include in a framework. The reflective discussion was considered the most relevant phase of the approach to support inputs for health decision-making processes reflecting both drug value and place in therapy. Conclusions: the study showed that reflective MCDA methodology could be implemented to complement the decision-making process in CatSalut, as an aid to determine the clinical added value for orphan medicines. MCDA provided transparency and a structured discussion during the committee meetings, thus increasing transparency and predictability of the relevant items supporting the agreements adopted on orphan drugs access

Unitats del dolor al SISCAT: cartera de serveis i nivells de complexitat

Unitats del dolor; Hospitals; Cartera de serveis; SISCATUnidades del dolor; Hospitales; Cartera de servicios; SISCATPain units; Hospitals; Portfolio of services; SISCATLes unitats del dolor són unitats de tipus funcional pròpies de l’àmbit hospitalari, on diferents professionals d’un mateix centre sanitari treballen conjuntament per a l’abordatge del pacient amb dolor. Aquestes presten l’atenció en l’àmbit hospitalari i, dins d’aquest, majoritàriament, en l’entorn de les consultes externes hospitalàries o de l’hospital de dia o àrees de tècniques especials. A més, els procediments més complexos s’han de realitzar en entorns de tipus quiròfan. Així, es fa palesa la necessitat de disposar d’una major concreció en la cartera de serveis de les unitats del dolor i recomanacions per al maneig del dolor a Catalunya, garantint en tot moment la qualitat i seguretat d’aquesta prestació, amb l’objectiu de millorar la qualitat de vida de les persones que pateixen dolor i garantir l’equitat d’accés per a la població, tot ordenant els fluxos quan sigui necessari. Per assolir aquests objectius, cal establir una codificació correcta de les diferents tècniques i/o procediment

Implementation of access initiatives for pharmaceutical innovations in CatSalut: towards an interated concept of health and care services model: value driven transformations

Innovacions farmacèutiques; Preus; Implementació d'iniciatives d'accés; Serveis sanitaris i assistencialsInnovaciones farmacéuticas; Precios; Implementación de iniciativas de acceso; Servicios sanitarios y asistencialesPharmaceutical innovations; Prices; Implementation of access initiatives; Health and healthcare servicesLes innovacions farmacèutiques són essencials per millorar la salut pública, aportant noves oportunitats per tractar determinades malalties. No obstant això, els medicaments recentment aprovats sovint estan relacionats amb preus elevats que representen els components que creixen més ràpidament de la despesa sanitària. L'accés a les innovacions farmacèutiques és un repte, de manera que els sistemes sanitaris públics introdueixen cada cop més mecanismes d'accés no tradicionals.Las innovaciones farmacéuticas son esenciales para mejorar la salud pública y brindan nuevas oportunidades para tratar determinadas enfermedades. No obstante, los medicamentos recientemente aprobados suelen estar vinculados a precios elevados, lo que representa los componentes del gasto sanitario que aumentan con mayor rapidez. El acceso a las innovaciones farmacéuticas es un desafío, por lo que los sistemas de salud pública están introduciendo cada vez más mecanismos de acceso no tradicionales.Pharmaceutical innovations are essential for improving public health, bringing new opportunities to treat certain diseases. Nervertheless newly approved medicines are often linked to high prices representing the most rapidy increasing components of health care spending. Access to pharmaceutical innovations is challenging, so public healthcare systems are increasingly introducing non-traditional access mechanism