[Clinical experience with orphan drugs for rare metabolic diseases].

CONCLUSIONS: Orphan drugs used in inborn errors of metabolism had an easy to manage toxicity profile, with many disparities in effectiveness. These drugs have a high economic impact. The cost-effectiveness ratio for orphan drugs is a controversial issue due to their high cost and the inconclusive clinical evidence.
PMID: 33158793 [PubMed - as supplied by publisher]

Source: Anales de Pediatria - November 3, 2020 Category: Pediatrics Authors: Caso-González A, Núñez-Rodríguez J, Nebot-Villacampa MJ, González-Pérez Y, Marín-Gorricho R, Leralta-González C, Obaldia-Alaña C Tags: An Pediatr (Barc) Source Type: research