The University of Southampton
University of Southampton Institutional Repository

Azacitidine for treating acute myeloid leukaemia: a nice single technology appraisal

Azacitidine for treating acute myeloid leukaemia: a nice single technology appraisal
Azacitidine for treating acute myeloid leukaemia: a nice single technology appraisal
Objectives To review the evidence, submitted to the National Institute of Health and Care Excellence (NICE) by Celgene, on the clinical and cost-effectiveness of azacitidine for the treatment of acute myeloid leukaemia in adults with >30% bone marrow blasts who are not eligible for hematopoietic stem cell transplantation, as part of the NICE Single Technology Appraisal process. Methods The company’s analysis was based on data from a randomized controlled trial, AZA-AML-001. The trial was conducted to determine the efficacy and safety of azacitidine against a conventional care regimen (CCR) comprised of three individual comparators: intensive chemotherapy followed by best supportive care (BSC) upon disease relapse or progression, non-intensive chemotherapy followed by BSC, and BSC alone. The primary trial end point, overall survival, was affected by treatment switching in both arms. The company’s economic evaluation was based on a partitioned survival model with four states: Remission, Non-remission, Relapse/Progressive disease and Death. We critically appraised the company’s submission; assessed the replicability and internal validity of their analysis using individual-patient data from AZA-AML-001; and examined the effect on incremental cost-effectiveness ratio (ICER) of crossover, and of altering model assumptions to reflect current UK practice. Results In the company’s analysis, the base-case ICER of azacitidine vs. CCR was £20,648 per quality-adjusted life-year (QALY) gained; in their probabilistic sensitivity analysis, the ICER was £17,423. After our amendments to Celgene’s model, the respective ICERs were £273,308 and £277,123 per QALY. In all our exploratory analyses, the ICER exceeded NICE’s threshold range of £20,000 to £30,000 per QALY gained. The major drivers of the increase in the ICER were model corrections related to healthcare resource use, and calibration of the number of treatment cycles (for consistency with AZA-AML-001). Conclusions After considering these analyses and statements from patient and clinical experts, the NICE Appraisal Committee did not recommend azacitidine for this indication.
1098-3015
A731-A731
Tikhonova, I.
577ccbcc-6643-4190-bb6d-19e13b44bcd1
Hoyle, M.
ebc06a93-bd41-4c23-8c96-1d9e74512c38
Snowsill, T.
33dbf192-8a97-43ed-9317-538d41cf498f
Cooper, C.
e05f5612-b493-4273-9b71-9e0ce32bdad6
Varley-Campbell, J.
0952839e-b1f4-4e45-b538-6dfc7936f259
Rudin, C.
9041e62b-450d-4455-95aa-302f98d11c5c
Mota, Mujica R.
8c906e16-c414-4051-a890-17dd82111d08
Tikhonova, I.
577ccbcc-6643-4190-bb6d-19e13b44bcd1
Hoyle, M.
ebc06a93-bd41-4c23-8c96-1d9e74512c38
Snowsill, T.
33dbf192-8a97-43ed-9317-538d41cf498f
Cooper, C.
e05f5612-b493-4273-9b71-9e0ce32bdad6
Varley-Campbell, J.
0952839e-b1f4-4e45-b538-6dfc7936f259
Rudin, C.
9041e62b-450d-4455-95aa-302f98d11c5c
Mota, Mujica R.
8c906e16-c414-4051-a890-17dd82111d08

Tikhonova, I., Hoyle, M., Snowsill, T., Cooper, C., Varley-Campbell, J., Rudin, C. and Mota, Mujica R. (2016) Azacitidine for treating acute myeloid leukaemia: a nice single technology appraisal. Value in Health, 19 (7), A731-A731. (doi:10.1016/j.jval.2016.09.2201).

Record type: Meeting abstract

Abstract

Objectives To review the evidence, submitted to the National Institute of Health and Care Excellence (NICE) by Celgene, on the clinical and cost-effectiveness of azacitidine for the treatment of acute myeloid leukaemia in adults with >30% bone marrow blasts who are not eligible for hematopoietic stem cell transplantation, as part of the NICE Single Technology Appraisal process. Methods The company’s analysis was based on data from a randomized controlled trial, AZA-AML-001. The trial was conducted to determine the efficacy and safety of azacitidine against a conventional care regimen (CCR) comprised of three individual comparators: intensive chemotherapy followed by best supportive care (BSC) upon disease relapse or progression, non-intensive chemotherapy followed by BSC, and BSC alone. The primary trial end point, overall survival, was affected by treatment switching in both arms. The company’s economic evaluation was based on a partitioned survival model with four states: Remission, Non-remission, Relapse/Progressive disease and Death. We critically appraised the company’s submission; assessed the replicability and internal validity of their analysis using individual-patient data from AZA-AML-001; and examined the effect on incremental cost-effectiveness ratio (ICER) of crossover, and of altering model assumptions to reflect current UK practice. Results In the company’s analysis, the base-case ICER of azacitidine vs. CCR was £20,648 per quality-adjusted life-year (QALY) gained; in their probabilistic sensitivity analysis, the ICER was £17,423. After our amendments to Celgene’s model, the respective ICERs were £273,308 and £277,123 per QALY. In all our exploratory analyses, the ICER exceeded NICE’s threshold range of £20,000 to £30,000 per QALY gained. The major drivers of the increase in the ICER were model corrections related to healthcare resource use, and calibration of the number of treatment cycles (for consistency with AZA-AML-001). Conclusions After considering these analyses and statements from patient and clinical experts, the NICE Appraisal Committee did not recommend azacitidine for this indication.

Text
1-s2.0-S1098301516335677-main - Version of Record
Available under License Other.
Download (68kB)

More information

e-pub ahead of print date: 31 October 2016
Published date: November 2016

Identifiers

Local EPrints ID: 437832
URI: http://eprints.soton.ac.uk/id/eprint/437832
ISSN: 1098-3015
PURE UUID: 7b82d67c-2d26-475b-a800-8ceacabe8d63
ORCID for I. Tikhonova: ORCID iD orcid.org/0000-0003-2723-0802
ORCID for C. Cooper: ORCID iD orcid.org/0000-0003-3510-0709

Catalogue record

Date deposited: 19 Feb 2020 17:32
Last modified: 18 Mar 2024 02:46

Export record

Altmetrics

Contributors

Author: I. Tikhonova ORCID iD
Author: M. Hoyle
Author: T. Snowsill
Author: C. Cooper ORCID iD
Author: J. Varley-Campbell
Author: C. Rudin
Author: Mujica R. Mota

Download statistics

Downloads from ePrints over the past year. Other digital versions may also be available to download e.g. from the publisher's website.

View more statistics

Atom RSS 1.0 RSS 2.0

Contact ePrints Soton: eprints@soton.ac.uk

ePrints Soton supports OAI 2.0 with a base URL of http://eprints.soton.ac.uk/cgi/oai2

This repository has been built using EPrints software, developed at the University of Southampton, but available to everyone to use.

We use cookies to ensure that we give you the best experience on our website. If you continue without changing your settings, we will assume that you are happy to receive cookies on the University of Southampton website.

×