Pharmacoeconomic review report : Sebelipase Alfa (Kanuma) (Alexion pharmaceuticals, Inc.) / Canadian Agency for Drugs and Technologies in Health.

Format:

Book

Author/Creator:

Canadian Agency for Drugs and Technologies in Health, author, issuing body.

Language:

English

Subjects (All):

Medicine--Research.

Medicine.

Cost effectiveness.

Physical Description:

1 online resource

Other Title:

Pharmacoeconomic review report Sebelipase Alfa

Place of Publication:

Ottawa (ON) : CADTH, 2018.

Summary:

Sebelipase alfa (Kanuma) is a recombinant form of the human lysosomal acid lipase (LAL) enzyme, and is indicated for the treatment of infants, pediatric, and adult patients diagnosed with LAL deficiency. The recommended starting dose in infants (less than six months of age) presenting with rapidly progressive LAL deficiency is 1 mg/kg administered as an intravenous infusion once weekly, with dose escalation to 3 mg/kg once weekly, based on clinical response. In pediatric and adult patients the recommended dose is 1 mg/kg administered as an intravenous infusion once every other week. The manufacturer submitted a price of

Contents:

Abbreviations

Executive Summary

Background

Summary of Identified Limitations and Key Results

Conclusions

Information on the Pharmacoeconomic Submission

Summary of the Manufacturer's Pharmacoeconomic Submission

Manufacturer's Base Case

Summary of Manufacturer's Sensitivity Analyses

Limitations of Manufacturer's Submission

CADTH Common Drug Review Reanalyses

Patient Input

Appendix 1. Cost Comparison

Appendix 2. Summary of Key Outcomes

Appendix 3. Additional Information

Appendix 4. Summary of Other HTA Reviews of Drug

Appendix 5. Reviewer Worksheets

References.

Notes:

Description based on publisher supplied metadata and other sources.