Strategic discussion on funding and access to therapies targeting rare diseases in Spain: an expert consensus paper.

BACKGROUND: In recent years, significant advances have been made in the field of rare diseases (RDs). However, there is a large number of RDs without specific treatment and half of these treatments have public funding in Spain. The aim of the FINEERR project was to carry out a multidisciplinary strategic discussion on the challenge of funding and access to RD-targeted drugs in Spain, in order to agree on specific proposals for medium-term improvement and hence support decision-making in the Spanish National Healthcare System (SNHS). RESULTS: The FINEERR Project was organized around a CORE Advisory Committee, which provided an overview, agreed on the design and scope of the project, and selected the members within each of four working groups (WG). Overall, 40 experts discussed and reached a consensus on different relevant aspects, such as conditioning factors for initial funding and access, evaluation and access to RD-targeted therapies, funding of these therapies, and implementation of a new funding and access model. From these meetings, 50 proposals were defined and classified by their level of relevance according to the experts. A descriptive analysis of responses was performed for each proposal. Thereafter, experts completed another questionnaire where they ranked the 25 most relevant proposals according to their level of feasibility of being implemented in the SNHS. The most relevant and feasible proposals were to improve: process of referral of patients with RDs, control over monitoring mechanisms, and communication between healthcare professionals and patients. CONCLUSIONS: The FINEERR project may provide a starting point for stakeholders involved in the process of funding and access to RD-targeted therapies in Spain to provide the necessary resources and implement measures to improve both the quality of life and life expectancy of patients with RDs.

Changes over the last 15 years in the psychopharmacological management of persons with borderline personality disorder.

OBJECTIVE: To describe the pharmacological management of borderline personality disorder (BPD) in Spain from 2001 through 2016, the factors associated with prescriptions, and changes in pharmacotherapy over this time period. METHODS: Retrospective, cross-sectional, observational study conducted in a sample of 457 patients with BPD consecutively admitted to a specialist BPD Program between January 2001 and November 2016. Data on sociodemographic and clinical variables, as well as pharmacological treatment upon the admission to the programme, were used to describe pharmacological prescriptions, the factors associated with these medications, and changes in prescription over the last 15 years. RESULTS: Most (88.4%) patients were on pharmacological treatment, with 53.8% of persons taking ≥3 medications. No significant changes in these percentages were observed over the study period. The use of tricyclic antidepressants and benzodiazepines decreased, while the use of atypical antipsychotics increased. Axis I comorbidity was the main factor associated with pharmacological treatment and polypharmacy. CONCLUSIONS: This study provides further evidence confirming the worldwide overuse of prescription medications for BPD and shows that there has been a shift in the prescription pattern in the last 15 years. These results suggest that real clinical practice only partially adheres to clinical treatment guidelines.