PMID- 33435846
OWN - NLM
STAT- MEDLINE
DCOM- 20211125
LR  - 20211125
IS  - 1552-681X (Electronic)
IS  - 0272-989X (Print)
IS  - 0272-989X (Linking)
VI  - 41
IP  - 2
DP  - 2021 Feb
TI  - Exploring Heterogeneity in Histology-Independent Technologies and the 
      Implications for Cost-Effectiveness.
PG  - 165-178
LID - 10.1177/0272989X20980327 [doi]
AB  - BACKGROUND: The National Institute for Health and Care Excellence and a number of 
      international health technology assessment agencies have recently undertaken 
      appraisals of histology-independent technologies (HITs). A strong and untested 
      assumption inherent in the submissions included identical clinical response 
      across all tumour histologies, including new histologies unrepresented in the 
      trial. Challenging this assumption and exploring the potential for heterogeneity 
      has the potential to impact upon cost-effectiveness. METHOD: Using published 
      response data for a HIT, a Bayesian hierarchical model (BHM) was used to identify 
      heterogeneity in response and to estimate the probability of response for each 
      histology included in single-arm studies, which informed the submission for the 
      HIT, larotrectinib. The probability of response for a new histology was 
      estimated. Results were inputted into a simplified response-based economic model 
      using hypothetical parameters. Histology-independent and histology-specific 
      incremental cost-effectiveness ratios accounting for heterogeneity were 
      generated. RESULTS: The results of the BHM show considerable heterogeneity in 
      response rates across histologies. The predicted probability of response 
      estimated by the BHM is 60.9% (95% credible interval 16.0; 91.8%), lower than the 
      naively pooled probability of 74.5%. A mean response probability of 56.9% (0.2; 
      99.9%) is predicted for an unrepresented histology. Based on the economic 
      analysis, the probability of the hypothetical HIT being cost-effective under the 
      assumption of identical response is 78%. Allowing for heterogeneity, the 
      probability of various approval decisions being cost-effective ranges from 93% to 
      11%. CONCLUSIONS: Central to the challenge of reimbursement of HITs is the 
      potential for heterogeneity. This study illustrates how heterogeneity in clinical 
      effectiveness can result in highly variable and uncertain estimates of 
      cost-effectiveness. This analysis can help improve understanding of the 
      consequences of histology-independent versus histology-specific decisions.
FAU - Murphy, Peter
AU  - Murphy P
AUID- ORCID: 0000-0001-8864-1416
AD  - Centre for Reviews and Dissemination, University of York, York, Yorkshire, UK.
FAU - Claxton, Lindsay
AU  - Claxton L
AD  - Centre for Reviews and Dissemination, University of York, York, Yorkshire, UK.
FAU - Hodgson, Robert
AU  - Hodgson R
AD  - Centre for Reviews and Dissemination, University of York, York, Yorkshire, UK.
FAU - Glynn, David
AU  - Glynn D
AD  - Centre for Health Economics, University of York, York, Yorkshire, UK.
FAU - Beresford, Lucy
AU  - Beresford L
AD  - Centre for Reviews and Dissemination, University of York, York, Yorkshire, UK.
FAU - Walton, Matthew
AU  - Walton M
AUID- ORCID: 0000-0003-1932-3689
AD  - Centre for Reviews and Dissemination, University of York, York, Yorkshire, UK.
FAU - Llewellyn, Alexis
AU  - Llewellyn A
AD  - Centre for Reviews and Dissemination, University of York, York, Yorkshire, UK.
FAU - Palmer, Stephen
AU  - Palmer S
AD  - Centre for Health Economics, University of York, York, Yorkshire, UK.
FAU - Dias, Sofia
AU  - Dias S
AUID- ORCID: 0000-0002-2172-0221
AD  - Centre for Reviews and Dissemination, University of York, York, Yorkshire, UK.
LA  - eng
GR  - NIHR127852/DH_/Department of Health/United Kingdom
GR  - 127582/DH_/Department of Health/United Kingdom
GR  - 12/87/63/DH_/Department of Health/United Kingdom
GR  - 12/78/95/DH_/Department of Health/United Kingdom
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20210113
PL  - United States
TA  - Med Decis Making
JT  - Medical decision making : an international journal of the Society for Medical 
      Decision Making
JID - 8109073
SB  - IM
MH  - Bayes Theorem
MH  - Cost-Benefit Analysis
MH  - Humans
MH  - Models, Economic
MH  - *Neoplasms
MH  - *Technology Assessment, Biomedical
PMC - PMC7879234
OTO - NOTNLM
OT  - bayesian hierarchical model
OT  - economic evaluation
OT  - heterogeneity
OT  - histology-independent
COIS- The authors declared no potential conflicts of interest with respect to the 
      research, authorship, and/or publication of this article.
EDAT- 2021/01/14 06:00
MHDA- 2021/11/26 06:00
PMCR- 2021/02/12
CRDT- 2021/01/13 05:42
PHST- 2021/01/14 06:00 [pubmed]
PHST- 2021/11/26 06:00 [medline]
PHST- 2021/01/13 05:42 [entrez]
PHST- 2021/02/12 00:00 [pmc-release]
AID - 10.1177_0272989X20980327 [pii]
AID - 10.1177/0272989X20980327 [doi]
PST - ppublish
SO  - Med Decis Making. 2021 Feb;41(2):165-178. doi: 10.1177/0272989X20980327. Epub 
      2021 Jan 13.