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

Poliangeitis amb Granulomatosis eosinofílica i asma al·lèrgica; dues monedes amb una mateixa cara?

La síndrome de Churg-Strauss o Poliangeitis amb Granulomatosis eosinofílica (EGPA), clàssicament s'ha vinculat a un substrat asmàtic d'origen al·lèrgic. No obstant, l'evidència en aquest sentit és equívoca. L'objectiu d'aquest treball és identificar diferències clíniques i serològiques entre pacients amb EGPA i pacients asmàtics al·lèrgics, i determinar el perfil al·lèrgic d'un grup de pacients diagnosticats d'EGPA. Es comparen dos grups de pacients: un grup de diagnosticats d'EGPA, i un altre amb asma al·lèrgic. Malgrat les similituds clíniques entre els dos grups, les reaccions immunoal·lèrgiques no semblen intervenir en la fisiopatologia de la majoria dels casos d'EGPA

Outcomes of off-label drug uses in hospitals: a multicentric prospective study

Purpose: The study aims to assess the clinical evidence, outcome and cost of off-label use of medicines in the hospital setting. Methods: A multicentric prospective cohort study of patients treated with off-label medicines was carried out in five tertiary hospitals from May 2011 to May 2012. Information on clinical characteristics of patients, drugs, outcomes and costs was collected. Patients were followed up to 6 months, and information was assessed by reviewing clinical records and interviewing physicians. Results: A total of 226 patients were included. The median (interquartile range (IQR)) age of patients was 46 (3362) years; 59 % were women. Patients had received a median of three previous treatments, and a lack of response (or suboptimal) was the main reason for off-label use (72.1 %). A total of 232 off-label medicines were administered for 102 different indications. The most frequent medicines were rituximab (49; 21.1 %), botulinum toxin (25; 10.7 %) and omalizumab (14; 6.0 %). In 117 (51.8 %) cases, the level of clinical evidence for their use was low. A partial clinical response was observed in 82 patients (36.3 %), complete response in 71 (31.4 %) and stabilization in 11 (4.9 %). A total of 58 (26.5 %) patients had adverse effects, which in 11 (4.9 %) were severe. The median (IQR) cost per patient was 2,943.07 (541.95,872.54). Conclusions: There was a high variability of off-label medicines and indications. Although the clinical evidence of off-label medicines was often low, clinical response was observed in many patients with previous multiple treatment failure, but at the expense of some adverse effects and a high cost. Registers of patients would be helpful for clinical decisions, although clinical trials are needed

Poliangeitis amb Granulomatosis eosinofílica i asma al·lèrgica; dues monedes amb una mateixa cara?

La síndrome de Churg-Strauss o Poliangeitis amb Granulomatosis eosinofílica (EGPA), clàssicament s'ha vinculat a un substrat asmàtic d'origen al·lèrgic. No obstant, l'evidència en aquest sentit és equívoca. L'objectiu d'aquest treball és identificar diferències clíniques i serològiques entre pacients amb EGPA i pacients asmàtics al·lèrgics, i determinar el perfil al·lèrgic d'un grup de pacients diagnosticats d'EGPA. Es comparen dos grups de pacients: un grup de diagnosticats d'EGPA, i un altre amb asma al·lèrgic. Malgrat les similituds clíniques entre els dos grups, les reaccions immunoal·lèrgiques no semblen intervenir en la fisiopatologia de la majoria dels casos d'EGPA