OBJECTIVES: MCDA is a methodological tool that explicitly considers multiple factors relevant to decision-making, being particularly useful for rare diseases. cTTP is an ultra-rare prothrombotic disorder, characterized by a severe congenital deficiency of ADAMTS13, and is associated with life-threatening acute episodes and long-term chronic symptoms. Recombinant ADAMTS13 (rADAMTS13) is a new orphan treatment for cTTP and the only specifically developed for this disease. The aim of this project was to apply a MCDA methodology to assess the value of rADAMTS13 in cTTP in the Spanish context, compared to standard of care.

METHODS: After a literature review, an evidence matrix was elaborated and validated by a multidisciplinary committee of 9 experts, following the MCDA EVIDEM framework (1,2). According to the type of criteria, different qualitative and quantitative scoring scales were used, with higher scores representing higher value.

RESULTS: Disease related criteria were rated high for rADAMTS13 in both unmet needs [mean: 4.3/5 SD (0.5)] and severity of the disease [4.2/5 (0.7)]. rADAMTS13 obtained higher mean scores than the standard of care in all quantitative criteria. This included among others efficacy [3.8 (1.2)] and safety [3.6 (1.3)] on a scale of -5 to 5; and quality of the evidence [3.7/5 (1.1)]. Higher scores were mainly given considering rADAMTS13’s mechanism of action, administration convenience, lower incidence of treatment-related adverse events and improved tolerability to patients. The overall quantitative rating of rADAMTS13 was 0.7/1. Furthermore, the overall qualitative criteria showed a positive impact, except for the neutral impact of opportunity and affordability costs criterion.

CONCLUSIONS: cTTP is recognized as a severe disease for which there are important unmet needs. Under this methodology, rADAMTS13 is perceived as a more effective, safer and more convenient therapeutic option than standard of care.