The clinical effectiveness and cost-effectiveness of enzyme replacement therapy for Gaucher's disease: a systematic review

Abstract

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Generating Health Technology Assessment Evidence for Rare Diseases

“…This leads to a lack of consensus about the most important outcomes to study and the size of benefit that is important. This is exemplified by a recent HTA of therapies for Gaucher's disease, which described a range of "potentially beneficial effects" and improvements across a wide range of outcomes including hematological markers and skeletal improvement but it was unclear how these effects translated into patient wellbeing (12).…”

The cost-effectiveness of enzyme replacement therapy (ERT) for the infantile form of Pompe disease: comparing a high-income country's approach (England) to that of a middle-income one (Colombia)

“…It was also assumed that since Pompe disorder is progressive and once diagnosed it is always symptomatic, no alive-asymptomatic transition state was considered. Costs from diagnostic phase and out-patient care were seen as irrelevant due to coinciding with previous economic studies concerning lysosomal storage disorders (LSD) (21). Additional limitations were related to infl ation rates and trends for both countries, and the possible impact on currency exchange rates that the ongoing economic crisis may have had on international and pharmaceutical markets.…”

Myoclonic Epilepsy in Lysosomal Storage Disorders

“…Twenty years ago the availability of enzyme replacement therapy (ERT) for GD opened a new era for the treatment of LSDs, giving to the patients a concrete hope for recovering (Brady, 2006;Connock et al 2006). However, clinical history of GD demonstrated the limited effect of ERT on neurological phenotypes.…”