BackgroundPulmonary Arterial Hypertension (PAH) is a chronic rare disease that can lead to serious cardiovascular problems and death. Additional treatments that increase effectiveness, that are safe and with a convenient administration that improve outcomes and quality of life for patients are needed. The aim of this study was to assess the value contribution of the new, oral prostacyclin receptor agonist, selexipag, for PAH treatment in Spain through reflective Multicriteria Decision Analysis (MCDA) methodology.MethodsA comprehensive literature review was performed to develop an evidence matrix, composed of twelve quantitative criteria and four contextual criteria, based on an EVIDEM MCDA framework adapted to orphan drugs evaluation by the Spanish region of Catalonia. Quantitative performance scores, qualitative impact of contextual criteria and individual reflections from stakeholders were collected for each MCDA framework criteria. The value contribution of selexipag to PAH treatment compared to inhaled iloprost was calculated.ResultsOral selexipag for PAH treatment was considered as a treatment which adds value, compared to iloprost, in the following MCDA quantitative criteria: comparative efficacy, patient reported outcomes, preventive benefit, therapeutic benefit, other medical costs and other non-medical costs, without significant differences in safety profile but with a higher acquisition cost than inhaled iloprost.ConclusionsSelexipag was considered to provide value to PAH treatment. It was perceived as an intervention indicated for a severe rare disease with high unmet needs, supported by high quality clinical evidence. When compared to inhaled iloprost, oral selexipag has demonstrated improvements in efficacy and patient reported outcomes, with a similar safety profile and some additional costs.Reflective MCDA provided a standardised, transparent approach to evaluate multiple criteria relating to the overall value contribution of selexipag to PAH treatment facilitating decision-making.

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Juvenile idiopathic arthritis (JIA) is a chronic inflammatory disease that often requires biological therapy to control its activity. Medication persistence and adherence are important aspects on which we have scarce information. We performed a longitudinal, retrospective, and observational study based on data from the daily clinical management of JIA patients. We recorded clinical remission at 6 and 12 months. Persistence of biological therapy was evaluated using Kaplan–Meier curves, and adherence was assessed using the medication possession ratio (MPR). We included 68 patients who received biological therapy. Of these, 11 (16.2%) and 5 (7.4%) required a second and third drug, respectively. The persistence rate for biological therapy at 5 years was 64%, with no differences between the first and second lines. Adherence was high during the first year of treatment (MPR80: 96.3%) and also in the second and third years (MPR80: 85.2% and 91.8%, respectively). Persistence and adherence to biological therapy were remarkably high in our JIA cohort. Adherence to biological treatments could be related to a higher probability of fulfilling the Wallace remission criteria at 6 months, although this was not confirmed at 12 months.

BackgroundJuvenile Idiopathic Arthritis (JIA) is a chronic inflammatory disease that often requires the use of biological therapies to control disease activity. Persistence and adherence to treatment are important aspects on which we have scarce information.MethodsWe performed a longitudinal, retrospective, and observational study based on daily clinical management of JIA patients. We calculated the clinical remission status at 6 and 12 months. Persistence of biological therapy was evaluated by Kaplan-Meier curves and adherence by the Medication Possession Ratio (MPR).ResultsWe included 68 patients who received biological therapy. Of these, 11 (16.2%) and 5 (7.4%) required a second and third biological, respectively. Persistence rate of biological therapies at 5 years was 64%, with no differences between the first and second biological line. Adherence was high the first year of treatment (MPR80: 96.3%) as well as the second and third years (MPR80: 85.2% and 91.8% respectively).ConclusionsPersistence and adherence to biological therapies were remarkably high in our JIA cohort. Adherence to biological treatments could be related to a higher probability of accomplishing Wallace remission criteria at 6 months, but it was not confirmed at 12 months.

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