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CAR-T cell therapy represents a therapeutic revolution in the prognosis and treatment of patients with certain types of hematological cancer. However, they also pose new challenges in the healthcare, regulatory and financial fields. The aim of the RET-A project was to undertake a strategic reflection on the management of CAR-T therapies within the Spanish National Health System, to agree on recommendations that will help to better deal with the new context introduced by these cell therapies in the present and in the future. This think tank involved 40 key agents and opinion leaders. The experts identified three great challenges for implementing advanced therapies in Spain: therapeutic individualisation, with a multidisciplinary approach; acceleration of access times, by minimizing bureaucracy; and increase in the number of centers qualified to manage the CAR-T therapies in the NHS. The experts agreed on the ideal criteria for designating those qualified centers. They also agreed on a comprehensive CAR-T care pathway with the timings and roles which would ideally be involved in each part of the process.

Introduction: Hereditary angioedema (HAE) is a rare genetic disease that impairs quality of life and could be life-threatening. The aim of this study was to apply a multicriteria decision analysis to assess the value of three long-term prophylactic (LTP) therapies for HAE in Spain. Methods: A multidisciplinary committee of 10 experts assessed the value of lanadelumab (subcutaneous use), C1-inhibitor (C1-INH; intravenous), and danazol (orally), using placebo as comparator. We followed the EVIDEM methodology that considers a set of 13 quantitative criteria. The overall estimated value of each intervention was obtained combining the weighting of each criterion with the scoring of each intervention in each criterion. We used two alternative weighting methods: hierarchical point allocation (HPA) and direct rating scale (DRS). A reevaluation of weightings and scores was performed. Results: Lanadelumab obtained higher mean scores than C1-INH and danazol in all criteria, except for the cost of the intervention and clinical practice guidelines. Under the HPA method, the estimated values were 0.51 (95% confidence interval [CI]: 0.44-0.58) for lanadelumab, 0.47 (95%CI: 0.41-0.53) for C1-INH, and 0.31 (95%CI: 0.24-0.39) for danazol. Similar results were obtained with the DRS method: 0.51 (95%CI: 0.42-0.60), 0.47 (95%CI: 0.40-0.54), and 0.27 (95%CI: 0.18-0.37), respectively. The comparative cost of the intervention was the only criterion that contributed negatively to the values of lanadelumab and C1-INH. For danazol, four criteria contributed negatively, mainly comparative safety. Conclusion: Lanadelumab was assessed as a high-value intervention, better than C1-INH and substantially better than danazol for LTP treatment of HAE.

Introduction: In March 2020, due to the pandemic caused by COVID-19, a pilot experience of pharmaceutical continuity of care received by hospital outpatients was launched in Cantabria, a Spanish autonomous community. Thanks to this experience, the medication was dispensed by the hospital pharmacy through the community pharmacy of the patient’s choice. Our objective was to estimate the costs avoided by this practice during the months of April and May 2020. Methods: The analysis of the economic impact was carried out in terms of saved costs, from the hospital and from the social perspective. Savings associated with the avoided home delivery of medication and avoided labor productivity losses were calculated, as well as the time associated with avoided trips in monetary terms. Results: A total of 2,249 hospital-dispensed drugs were delivered through the community pharmacy in the analyzed period, with an average of 57.7 daily deliveries. The experience, which involved one hospital and 262 pharmacies of Cantabria, saved patients 93,305 km in trips to the hospital, associated with an average time saving of 1,374 hours. In terms of costs, the hospital saved on shipments by courier, estimated at €30,205, since it was the community pharmacy and the distribution warehouses that delivered the drugs. From a social perspective, this initiative saved €23,309 due to the trips (€8,907) and productivity losses (€14,402) that were avoided. Conclusions: This exceptional situation may be a good opportunity to improve the coordination between hospital pharmacies and community pharmacies in Spain, not only during the pandemic but also in the post-COVID-19 era.

The approval of new non-insulin treatments has broadened the therapeutic arsenal, but it has also increased the complexity of choice for the treatment of type 2 diabetes mellitus (DM2). The objective of this study was to systematically review the literature on economic evaluations associated with non-insulin antidiabetic drugs (NIADs) for DM2. We searched in Medline, IBECS, Doyma and SciELO databases for full economic evaluations of NIADs in adults with DM2 applied after the failure of the first line of pharmacological treatment, published between 2010 and 2017, focusing on studies that incorporated quality-adjusted life years (QALYs). The review included a total of 57 studies, in which 134 comparisons were made between NIADs. Under an acceptability threshold of 25,000 euros per QALY gained, iSLGT-2 were preferable to iDPP-4 and sulfonylureas in terms of incremental cost-utility. By contrast, there were no conclusive comparative results for the other two new NIAD groups (GLP-1 and iDPP-4). The heterogeneity of the studies’ methodologies and results hindered our ability to determine under what specific clinical assumptions some NIADs would be more cost-effective than others. Economic evaluations of healthcare should be used as part of the decision-making process, so multifactorial therapeutic management strategies should be established based on the patients’ clinical characteristics and preferences as principal criteria.

It is well known that pharmaceutical innovation has improved the health and quality of life of patients. It is however sometimes forgotten that new drugs also have the potential of improving the efficiency and the sustainability of the healthcare system. The objective of this review is to shed light on the magnitude of the offset effect that drugs may have in the realm of the healthcare system and for society as a whole. A narrative literature review was carried out. This review demonstrated that a growing body of literature has tried to measure the magnitude of the offset effect associated with pharmaceutical innovation, both at the aggregate level and for different diseases. There is evidence that the aggregate use of new drugs can generate net savings to the healthcare system and to society, as they may release both healthcare and non-healthcare resources for alternative uses. A high degree of heterogeneity in the magnitude of the effect has been found across different pathologies and different types of drugs.