Zolgensma (onasemnogene abeparvovec-xioi) - Novartis
pirfenidone - Generic mfg.
Iclusig (ponatinib) - Takeda
Myalept (metreleptin) - Novelion
Making trade-offs explicit: development and pilot of a Belgian MCDA framework for orphan drug appraisal. (PubMed, Int J Technol Assess Health Care) - Oct 20, 2026 - "This study presents the first MCDA framework for orphan drug reimbursement in Belgium and illustrates how criterion weights and scores can structure committee deliberation, complemented by contextual appraisal, to support more transparent, consistent, and accountable decision making." 
Journal • Orphan drug • Rare Diseases
http://www.ncbi.nlm.nih.gov/pubmed/42894980
 
Alessandra Blonda; Daniel A Ollendorf; Isabelle Huys; Steven Simoens; Yvonne Denier
 
Oct 20, 2026
 
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