Presentation (CTI)

OBJECTIVES: The evaluation of rare disease medicines is poorly suited to decisions based on a single criterion, such as the cost-effectiveness ratio, which overlooks the multidimensional nature of their value. This study aimed to develop a multi-criteria decision analysis (MCDA) framework specific to the Algerian context for the evaluation of orphan drugs, simultaneously integrating the clinical, economic, social, and organizational dimensions of the decision rather than relying on a single evaluative axis.
METHODS: A literature review and an analysis of the EVIDEM model were used to construct a preliminary list of 29 criteria. A consensus questionnaire was sent to 103 key opinion leaders (KOLs) across five stakeholder categories, pharmacists, physicians, health and regulatory authorities, the pharmaceutical industry, and patient representatives, to assess the relevance of each criterion and its feasibility in the local context, in line with the conditions defined by the ISPOR MCDA Task Force. Content validity was quantified using the item-level content validity index (I-CVI), with a retention threshold set at 0.78.
RESULTS: The analysis demonstrated high overall content validity (S-CVI/Ave = 0.80), reflecting a solid consensus across an otherwise heterogeneous panel. The three criteria with the highest I-CVI (0.961) were disease severity, clinical efficacy of the innovation under consideration, and patient quality of life. These criteria span both the clinical and humanistic dimensions of therapeutic value, confirming the relevance of a multidimensional evaluation in this context.
CONCLUSIONS: The MCDA approach proves applicable and relevant in the Algerian context and represents, to our knowledge, the first decision-support framework of its kind dedicated to the evaluation of rare disease medicines in this country.