Evaluating a Stone of Hope: ICER's 2019 Review of Treatments for Duchenne Muscular Dystrophy

J Manag Care Spec Pharm. 2020 Apr;26(4):366-368. doi: 10.18553/jmcp.2020.26.4.366.

Abstract

No funding contributed to the writing of this commentary. Brandsema reports consulting for Alexion, Audentes, AveXis, Biogen, Cytokinetics, PTC Therapeutics, Sarepta, and WaVe and has received research funding as a site investigator from Alexion, AveXis, Biogen, CSL Behring, Cytokinetics, Fibrogen, Pfizer, PTC Therapeutics, Sarepta, Summit, and WaVe.

Publication types

  • Review

MeSH terms

  • Cost-Benefit Analysis
  • Humans
  • Insurance Coverage / economics*
  • Insurance, Pharmaceutical Services / economics*
  • Insurance, Pharmaceutical Services / legislation & jurisprudence
  • Morpholinos / economics
  • Morpholinos / therapeutic use*
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Muscular Dystrophy, Duchenne / economics
  • Oligonucleotides / economics
  • Oligonucleotides / therapeutic use*
  • Pregnenediones / economics
  • Pregnenediones / therapeutic use*
  • United States
  • United States Food and Drug Administration / legislation & jurisprudence

Substances

  • Morpholinos
  • Oligonucleotides
  • Pregnenediones
  • golodirsen
  • eteplirsen
  • deflazacort