Implementing reflective multicriteria decision analysis (MCDA) to assess orphan drugs value in the Catalan Health Service (CatSalut)

Abstract

Background Orphan medicines show some characteristics that hinder the evaluation of their clinical added value. The often low level of evidence available for orphan drugs, together with a high budget impact and an incremental cost-effectiveness ratio many times higher than drugs used for non-orphan diseases, represent challenges in their appraisal and effective access to clinical use. In order to explore how to handle these hurdles, the Catalan Health Service (CatSalut) began an initiative on a multidimensional assessment of drugs value during the appraisal process. Reflective multicriteria decision analysis (MCDA) using analytical methods was chosen, since it may help to standardise and contextualize all the relevant data related with the drug that could contribute to a decision. The aim of the study was to determine whether the implementation of reflective MCDA methodology could support the decision-making process about orphan medicines in the context of CatSalut. Methods The assessment and decision-making process for orphan drugs in the Programa d’Harmonització Farmacoterapeutica (PHF) of CatSalut was prioritized to test the implementation of the reflective MCDA both a qualitative and quantitatively. A staged approach was used with the following main steps: selection and structuration of quantitative criteria (Core Model) and qualitative criteria (Contextual Tool), framework scoring and assessment of three orphan drug case studies. This proof-of-concept would grant a continued refinement of the methodology and, if and when validated, its potential integration to other therapeutic areas of the PHF. Results The final framework was composed by 10 quantitative criteria (Core Model) and 4 qualitative criteria (Contextual Tool) according to the PHF goals being the most important criteria “disease severity”, “unmet need”, “comparative effectiveness” and “comparative safety /tolerability”. The matrix developed for the case studies served as a guide for the selection of the essential information that the decision-makers were expected to include in a framework. The reflective discussion was considered the most relevant phase of the approach to support inputs for health decision-making processes reflecting both drug value and place in therapy. Conclusions The study showed that reflective MCDA methodology could be implemented to complement the decision-making process in CatSalut, as an aid to determine the clinical added value for orphan medicines. MCDA provided transparency and a structured discussion during the committee meetings, thus increasing transparency and predictability of the relevant items supporting the agreements adopted on orphan drugs access.

Value-based decision-making for orphan drugs with multiple criteria decision analysis: burosumab for the treatment of X-linked hypophosphatemia

“…The pros and cons of each weighting method have been extensively discussed elsewhere and should be judged not only on their level of precision but also on their theoretical foundations 31,36,37 . Although not comparable, the criteria used in our research have some resemblances with the research by Guarga et al (2019) 30 . Our approach allows for more granularity at criteria hierarchy and the results we obtained showed substantial differences in the ranking of the criteria relative to Guarga et al (2019) 30 .…”

“…(2016) organized a twelve criteria MCDA framework into three categories: health technology characteristics (five criteria), indicated disorder characteristics (two criteria), and public health aspects (five criteria) 29 . More recently, Guarga et al (2019) suggested a reflective MCDA to assess the value OMPs in the Catalan Health Service (CatSalut) composed by 10 quantitative and 4 qualitative criteria, respectively; disease severity, unmet needs, improvement of efficacy/effectiveness, improvement of safety/tolerability, improvement of patient perceived health/PRO, type of therapeutic benefit, annual patient cost of treatment, other medical costs, quality of evidence, expert consensus/clinical practice guidelines; population priorities and access (principle of equity), common goal and specific interests, system capacity and appropriate use of OMPs, opportunity costs and affordability (budget impact) 30 .…”

“…Guarga et al (2019) followed a simpler approach based on a direct rating scale (five-point weighting technique), while the adapted EVIDEM MCDA framework by Wagner et al (2016) used hierarchical point allocation. Both fall under the structured-weighting methods 30,31,34 .…”

Assessing the Preferences for Criteria in Multi-Criteria Decision Analysis in Treatments for Rare Diseases

“…AGNSS was subsequently taken over by the Highly Specialized Technologies (HST) group for NICE, who also use the MCDA approach (48). In Spain, the Spanish Agency for Medicines and Health Products developed a MCDA framework based on the EVIDEM framework (49) and similarly in Catalonia (50). The region of Lombardia in Italy has adopted a MCDA approach to regulate the introduction of new health technologies.…”

“…While ample research has been performed in recent years on the suitable criteria for inclusion in a MCDA model for orphan drugs (42,50,53), less research seems to have been done on the weights or preferences that should be applied to the different criteria. The weighting scales used in research range from 5-point scales (50,54), to 10-point scales (55) and several studies mention using a 100-point scale (34,41,56). A pilot study by Reddy et al included a group of eight participants (plus the facilitator) to select the criteria that should be considered for a MCDA framework for several public health preventative programmes.…”