[Migalastat for Fabry disease]
Klappenbach R, Pichon-Riviere A, Alfie V, Rojas-Roque C, Augustovski F, García Martí S, Ciapponi A, Bardach A, Alcaraz A.
Record ID 32018001593
Spanish
Original Title:
Migalastat para enfermedad de Fabry
Authors' recommendations:
Low-quality evidence suggests that migalastat for Fabry disease might have a similar efficacy to that of enzyme replacement therapy, which would mean a greater net benefit, mainly due to a decrease in lysosomal inclusions and an improvement in kidney function and cardiac mass. In addition, migalastat shows an additional benefit because it is easy to administer and has the convenience of an oral treatment when compared with enzyme replacement therapy, which is administered as an intravenous infusion biweekly. However, there is uncertainty since the evidence is inaccurate, there is a risk of bias and a short follow-up period although it should be noted the difficulties in generating evidence on rare diseases that require prolonged follow-up such as Fabry disease.
The only updated clinical practice guideline from Canada, considers the use of migalastat as an alternative to enzyme replacement therapy for specific cases, taking into account age, disease severity, compliance and complications of intravenous treatment. Most coverage policies from high-income countries cover this technology as a treatment alternative to enzyme replacement therapy, although in some cases just under the condition that its cost should be equal to or less than the cheapest enzyme replacement therapy alternative for the patient; or if there is an agreement in place to reduce the price to guarantee its cost-effectiveness in the local setting. Because of this and because the list price of migalastat is more expensive than that of its active comparators, it was considered as non favorable in the economic assessment, although this might vary if the cost of migalastat were equal to or less than the cheapest enzyme replacement therapy or if they showed poor response to them, provided that the physician and the patients consider it appropriate.
Details
Project Status:
Completed
Year Published:
2020
URL for published report:
https://www.iecs.org.ar/home-ets/
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
Argentina
MeSH Terms
- Fabry Disease
- 1-Deoxynojirimycin
- Enzyme Replacement Therapy
- alpha-Galactosidase
Contact
Organisation Name:
Institute for Clinical Effectiveness and Health Policy
Contact Address:
Dr. Emilio Ravignani 2024, Buenos Aires - Argentina, C1414 CABA
Contact Name:
info@iecs.org.ar
Contact Email:
info@iecs.org.ar
Copyright:
<p>Institute for Clinical Effectiveness and Health Policy (IECS)</p>
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.