Identifying key unmet needs and value drivers in the treatment of focal-onset seizures (FOS) in patients with drug-resistant epilepsy (DRE) in Spain through Multi-Criteria Decision Analysis (MCDA)

Headaches are among the most prevalent and disabling neurologic disorders and there are several unmet needs as current pharmacological options are inadequate in treating patients with chronic headache, and a growing interest focuses on nutritional approaches as non-pharmacological treatments. Among these, the largest body of evidence supports the use of the ketogenic diet (KD). Exactly 100 years ago, KD was first used to treat drug-resistant epilepsy, but subsequent applications of this diet also involved other neurological disorders. Evidence of KD effectiveness in migraine emerged in 1928, but in the last several year’s different groups of researchers and clinicians began utilizing this therapeutic option to treat patients with drug-resistant migraine, cluster headache, and/or headache comorbid with metabolic syndrome. Here we describe the existing evidence supporting the potential benefits of KDs in the management of headaches, explore the potential mechanisms of action involved in the efficacy in-depth, and synthesize results of working meetings of an Italian panel of experts on this topic. The aim of the working group was to create a clinical recommendation on indications and optimal clinical practice to treat patients with headaches using KDs. The results we present here are designed to advance the knowledge and application of KDs in the treatment of headaches.

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DRUG EVALUATION AND DECISION MAKING IN CATALONIA: DEVELOPMENT AND VALIDATION OF A METHODOLOGICAL FRAMEWORK BASED ON MULTI-CRITERIA DECISION ANALYSIS (MCDA) FOR ORPHAN DRUGS

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462317000149 ◽

2017 ◽

Vol 33 (1) ◽

pp. 111-120 ◽

Cited By ~ 19

Author(s):

Antoni Gilabert-Perramon ◽

Josep Torrent-Farnell ◽

Arancha Catalan ◽

Alba Prat ◽

Manel Fontanet ◽

...

Keyword(s):

Decision Making ◽

Decision Analysis ◽

Unmet Needs ◽

Drug Evaluation ◽

Evaluation Criteria ◽

Orphan Drugs ◽

Methodological Framework ◽

Multi Criteria Decision Analysis ◽

Development And Validation ◽

Weighting Technique

Objectives:The aim of this study was to adapt and assess the value of a Multi-Criteria Decision Analysis (MCDA) framework (EVIDEM) for the evaluation of Orphan drugs in Catalonia (Catalan Health Service).Methods:The standard evaluation and decision-making procedures of CatSalut were compared with the EVIDEM methodology and contents. The EVIDEM framework was adapted to the Catalan context, focusing on the evaluation of Orphan drugs (PASFTAC program), during a Workshop with sixteen PASFTAC members. The criteria weighting was done using two different techniques (nonhierarchical and hierarchical). Reliability was assessed by re-test.Results:The EVIDEM framework and methodology was found useful and feasible for Orphan drugs evaluation and decision making in Catalonia. All the criteria considered for the development of the CatSalut Technical Reports and decision making were considered in the framework. Nevertheless, the framework could improve the reporting of some of these criteria (i.e., “unmet needs” or “nonmedical costs”). Some Contextual criteria were removed (i.e., “Mandate and scope of healthcare system”, “Environmental impact”) or adapted (“population priorities and access”) for CatSalut purposes. Independently of the weighting technique considered, the most important evaluation criteria identified for orphan drugs were: “disease severity”, “unmet needs” and “comparative effectiveness”, while the “size of the population” had the lowest relevance for decision making. Test–retest analysis showed weight consistency among techniques, supporting reliability overtime.Conclusions:MCDA (EVIDEM framework) could be a useful tool to complement the current evaluation methods of CatSalut, contributing to standardization and pragmatism, providing a method to tackle ethical dilemmas and facilitating discussions related to decision making.

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