Cargando…

“Don’t Think Twice, It’s All Right”: Using Additional Data to Reduce Uncertainty Regarding Oncologic Drugs Provided Through Managed Access Agreements in England

OBJECTIVES: The Cancer Drugs Fund (CDF) in England uses managed access agreements to facilitate additional data collection to address uncertainties identified in the appraisals of new drugs. This study reviews the uncertainties highlighted in the original appraisals where recommendations “to use wit...

Descripción completa

Detalles Bibliográficos
Autores principales: Kang, Jiyeon, Cairns, John
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9929033/
https://www.ncbi.nlm.nih.gov/pubmed/36123583
http://dx.doi.org/10.1007/s41669-022-00369-9
_version_ 1784888758741499904
author Kang, Jiyeon
Cairns, John
author_facet Kang, Jiyeon
Cairns, John
author_sort Kang, Jiyeon
collection PubMed
description OBJECTIVES: The Cancer Drugs Fund (CDF) in England uses managed access agreements to facilitate additional data collection to address uncertainties identified in the appraisals of new drugs. This study reviews the uncertainties highlighted in the original appraisals where recommendations “to use within the CDF” were made and how additional data were used to address these uncertainties in the CDF review appraisals where final decisions on routine commissioning were made. METHODS: The first 24 drugs exiting the 2016 CDF were included in this review. The information about uncertainty and the use of newly collected data were extracted from the original appraisals and the CDF review appraisals. The additional data used in the CDF review appraisals, distinguishing between clinical trial data and real-world data (RWD), were reviewed to assess the extent to which the additional data were able to reduce the original uncertainties. RESULTS: The recommendation that the drug be routinely commissioned was made in 87.5% of re-appraisals. Uncertainty stemming from immaturity of the survival data in clinical trials was frequently found in appraisals. Later follow-up of clinical trials was used to address this uncertainty, whereas limited use was made of RWD. The Systemic Anti-Cancer Therapy (SACT) dataset is the most frequently used source of RWD. SACT data were mostly used in review appraisals to support the clinical outcomes based on later follow-up of trial participants and to inform modelling of subsequent treatments or treatment duration. CONCLUSIONS: While additionally collected RWD attracted attention when the 2016 CDF was introduced, RWD have not been widely used in CDF review appraisals and (to date) have done little to reduce uncertainty. Experience with these appraisals has highlighted the importance of longer follow-up of clinical trials and the relatively limited role of RWD, in general, and of SACT data in particular. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s41669-022-00369-9.
format Online
Article
Text
id pubmed-9929033
institution National Center for Biotechnology Information
language English
publishDate 2022
publisher Springer International Publishing
record_format MEDLINE/PubMed
spelling pubmed-99290332023-02-16 “Don’t Think Twice, It’s All Right”: Using Additional Data to Reduce Uncertainty Regarding Oncologic Drugs Provided Through Managed Access Agreements in England Kang, Jiyeon Cairns, John Pharmacoecon Open Original Research Article OBJECTIVES: The Cancer Drugs Fund (CDF) in England uses managed access agreements to facilitate additional data collection to address uncertainties identified in the appraisals of new drugs. This study reviews the uncertainties highlighted in the original appraisals where recommendations “to use within the CDF” were made and how additional data were used to address these uncertainties in the CDF review appraisals where final decisions on routine commissioning were made. METHODS: The first 24 drugs exiting the 2016 CDF were included in this review. The information about uncertainty and the use of newly collected data were extracted from the original appraisals and the CDF review appraisals. The additional data used in the CDF review appraisals, distinguishing between clinical trial data and real-world data (RWD), were reviewed to assess the extent to which the additional data were able to reduce the original uncertainties. RESULTS: The recommendation that the drug be routinely commissioned was made in 87.5% of re-appraisals. Uncertainty stemming from immaturity of the survival data in clinical trials was frequently found in appraisals. Later follow-up of clinical trials was used to address this uncertainty, whereas limited use was made of RWD. The Systemic Anti-Cancer Therapy (SACT) dataset is the most frequently used source of RWD. SACT data were mostly used in review appraisals to support the clinical outcomes based on later follow-up of trial participants and to inform modelling of subsequent treatments or treatment duration. CONCLUSIONS: While additionally collected RWD attracted attention when the 2016 CDF was introduced, RWD have not been widely used in CDF review appraisals and (to date) have done little to reduce uncertainty. Experience with these appraisals has highlighted the importance of longer follow-up of clinical trials and the relatively limited role of RWD, in general, and of SACT data in particular. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s41669-022-00369-9. Springer International Publishing 2022-09-20 /pmc/articles/PMC9929033/ /pubmed/36123583 http://dx.doi.org/10.1007/s41669-022-00369-9 Text en © The Author(s) 2022 https://creativecommons.org/licenses/by-nc/4.0/Open AccessThis article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) .
spellingShingle Original Research Article
Kang, Jiyeon
Cairns, John
“Don’t Think Twice, It’s All Right”: Using Additional Data to Reduce Uncertainty Regarding Oncologic Drugs Provided Through Managed Access Agreements in England
title “Don’t Think Twice, It’s All Right”: Using Additional Data to Reduce Uncertainty Regarding Oncologic Drugs Provided Through Managed Access Agreements in England
title_full “Don’t Think Twice, It’s All Right”: Using Additional Data to Reduce Uncertainty Regarding Oncologic Drugs Provided Through Managed Access Agreements in England
title_fullStr “Don’t Think Twice, It’s All Right”: Using Additional Data to Reduce Uncertainty Regarding Oncologic Drugs Provided Through Managed Access Agreements in England
title_full_unstemmed “Don’t Think Twice, It’s All Right”: Using Additional Data to Reduce Uncertainty Regarding Oncologic Drugs Provided Through Managed Access Agreements in England
title_short “Don’t Think Twice, It’s All Right”: Using Additional Data to Reduce Uncertainty Regarding Oncologic Drugs Provided Through Managed Access Agreements in England
title_sort “don’t think twice, it’s all right”: using additional data to reduce uncertainty regarding oncologic drugs provided through managed access agreements in england
topic Original Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9929033/
https://www.ncbi.nlm.nih.gov/pubmed/36123583
http://dx.doi.org/10.1007/s41669-022-00369-9
work_keys_str_mv AT kangjiyeon dontthinktwiceitsallrightusingadditionaldatatoreduceuncertaintyregardingoncologicdrugsprovidedthroughmanagedaccessagreementsinengland
AT cairnsjohn dontthinktwiceitsallrightusingadditionaldatatoreduceuncertaintyregardingoncologicdrugsprovidedthroughmanagedaccessagreementsinengland