Aim: Criteria weighting is a key element of multicriteria decision analysis that is becoming extensively used in healthcare decision-making. In our narrative review we describe the advantages and disadvantages of various weighting methods. Methods: An assessment of the eight identified primary criteria weighting methods was compiled on domains including their resource requirements, and potential for bias. Results: In general, we found more complex methods to have less potential for bias; however, resource intensity and general participant burden is greater for these methods. Conclusion: The selection of the most appropriate method depends on the decision-making context. The simple multiattribute rating technique (SMART) method combined with swing-weighting technique and the analytic hierarchy process methods may be the most feasible approaches for low- and middle-income countries.

There is a growing need for economic evaluations describing the disease course, as well as the costs and clinical outcomes related to the treatment of schizophrenia. A systematic review on studies describing health economic models in schizophrenia and a targeted literature review on utility mapping algorithms in schizophrenia were carried out. Models found in the review were collated and assessed in detail according to their type and various other attributes. Fifty-nine studies were included in the review. Modeling techniques varied from simple decision trees to complex simulation models. The models used various clinical endpoints as value drivers, 47% of the models used quality-adjusted life years, and eight percent used disability-adjusted life years to measure benefits, while others applied various clinical outcomes. Most models considered patients switching between therapies, and therapeutic adherence, compliance or persistence. The targeted literature review identified four main approaches to map PANSS scores to utility values. Health economic models developed for schizophrenia showed great variability, with simulation models becoming more frequently used in the last decade. Using PANSS scores as the basis of utility estimations is justifiable.

Schizophrenia is a chronic disease associated with significant and long-lasting effects on health, and it is also a social and financial burden, not only for patients but also for families, other caregivers, and the wider society. It is essential to conduct the assessment of indirect costs, to understand all the effects of the disease on society. Our aim is to gain a better understanding of the indirect costs of schizophrenia in Europe. We conducted a comprehensive systematic literature review covering EMBASE, Medline, and PsycINFO as well as reviewing Health Technology Assessment databases from different countries. We used a qualitative research synthesis for presenting information, as most of the studies were methodologically diverse, a quantitative analysis would have been impractical. Indirect cost adjusted to inflation ranged vastly between studies included in the review from 119 Euros to 62, 034 Euros annually. The average proportion of indirect costs of total costs was 44%. Studies highlighted important cost drivers as age, gender, and disease severity, explaining the variation in costs between treatment and patient groups. Regardless of the methodological heterogeneity of the reviewed studies, there was an agreement about the significance of indirect costs of schizophrenia on the society. Considering the relatively high prevalence of schizophrenia in Europe, a need for more cost of illness studies especially from Central Eastern and Southern Europe is suggested.

Aim: Our study aimed at estimating differences in quality-adjusted life year (QALY) gain for patients with predominant negative symptoms of schizophrenia treated with cariprazine compared with risperidone. Materials & methods: A Markov model was built, based on the Mohr–Lenert approach and data derived from clinical trials, to estimate potential QALY gains of patients. Results: Patients had higher probability of reaching better health states treated with cariprazine compared with risperidone. In the model, this resulted in an estimated QALY gain of 0.029 per patient, after 1 year of treatment. Conclusion: Cariprazine, which showed clinically meaningful improvement in the symptoms, and personal and social performance, can also provide significant QALY gain in the treatment of patients with predominant negative symptoms of schizophrenia compared with risperidone.

Introduction: Multiple Criteria Decision Analysis (MCDA) is increasingly used in health care mainly because it moves decision-making from ad hoc to an evidence-based and comprehensive process. Developing countries with more restricted financial and human research capacities, however, should consider their own methods of MCDA development and implementation. Areas covered: An MCDA framework to improve procurement decisions of off-patent pharmaceuticals was developed for developing countries and adapted to Indonesia, Kazakhstan and Vietnam during three policy workshops. Based on the experience of these workshops and one joint workshop with international experts and decision makers from multiple developing countries, general recommendations were formulated on how to implement MCDA specifically in developing countries. We provide 17 practical MCDA implementation recommendations in four major areas, including (1) MCDA objectives; (2) technical considerations of MCDA tool; (3) development and customization of MCDA tool and (4) policy implementation of MCDA in decision-making. Expert commentary: These practical MCDA recommendations for developing countries contribute to feasible, transparent, stepwise, iterative and standardized decision-making in health care.

Background Traditionally, the economic value of health technologies is assessed with cost-effectiveness (CE) and budget impact (BI) analyses. However, the evaluation of rare disease therapies often considers novel value criteria. Multi-criteria decision analysis (MCDA) is a promising tool in the assessment of value criteria that typically cannot be captured with traditional approaches. Objectives The objective of this research was to investigate the criteria and scoring functions applied in value frameworks and MCDA tools relevant to the evaluation of rare disease therapies. The aim was to gain a better understanding of the domains and measurement of commonly referenced novel value criteria. Methods A systematic literature review was performed covering the period from 2013 to 2019. MCDA or value framework articles and structured review papers on orphan-drug-specific MCDA articles were reviewed. Information sources included MEDLINE, Embase, Scopus, and 26 other gray literature sources. A descriptive review of identified criteria and scoring functions was performed, with special focus on “novel” value criteria that are traditionally not considered in CE or BI analyses. Results In total, 15 relevant value frameworks and MCDA tools were identified. These studies included a large number (n = 56) of individual value criteria. The most commonly included novel criteria were unmet medical need, severity of disease, and reduction in uncertainty. The identified scoring functions (measurement methods) for novel criteria were highly heterogeneous and tailored. Standardized scoring functions were not observed. Additionally, the studies did not provide their rationale for choosing a specific scoring function for a criterion. Conclusions MCDA is a promising tool to include novel value criteria into the health technology assessment of therapies for rare diseases. To support the development of a transparent and justified evaluation process, scoring functions should be further investigated.