TY - JOUR
T1 - Registry-based trials
T2 - A potential model for cost savings?
AU - Anderson, Brett R.
AU - Gotlieb, Evelyn G.
AU - Hill, Kevin
AU - McHugh, Kimberly E.
AU - Scheurer, Mark A.
AU - Mery, Carlos M.
AU - Pelletier, Glenn J.
AU - Kaltman, Jonathan R.
AU - White, Owen J.
AU - Trachtenberg, Felicia L.
AU - Hollenbeck-Pringle, Danielle
AU - McCrindle, Brian W.
AU - Sylvester, Donna M.
AU - Eckhauser, Aaron W.
AU - Pasquali, Sara K.
AU - Anderson, Jeffery B.
AU - Schamberger, Marcus S.
AU - Shashidharan, Subhadra
AU - Jacobs, Jeffrey P.
AU - Jacobs, Marshall L.
AU - Boskovski, Marko
AU - Newburger, Jane W.
AU - Nathan, Meena
N1 - Funding Information:
We thank the Collaborators listed in Appendix 1 for contributing to our data collection in semi-structured interviews. This work was supported by the US National Institutes of Health.
Funding Information:
This study was supported by grants (U24HL135691, U10HL068270, HL109818, HL109778, HL109816, HL109743, HL109741, HL109673, HL068270, HL109781, HL135665, HL135680) from the National Heart, Lung, and Blood Institute, National Institutes of Health (NHLBI, NIH). Dr. Brett Anderson receives support from K23 HL133454 from NHLBI, NIH. Dr. Meena Nathan receives support from K23 HL119600 from NHLBI, NIH. The authors have no other financial relationships relevant to this article to disclose.
Publisher Copyright:
© 2020 Cambridge University Press. All rights reserved.
PY - 2020/6/1
Y1 - 2020/6/1
N2 - Background/Aims: Registry-based trials have emerged as a potentially cost-saving study methodology. Early estimates of cost savings, however, conflated the benefits associated with registry utilisation and those associated with other aspects of pragmatic trial designs, which might not all be as broadly applicable. In this study, we sought to build a practical tool that investigators could use across disciplines to estimate the ranges of potential cost differences associated with implementing registry-based trials versus standard clinical trials.Methods: We built simulation Markov models to compare unique costs associated with data acquisition, cleaning, and linkage under a registry-based trial design versus a standard clinical trial. We conducted one-way, two-way, and probabilistic sensitivity analyses, varying study characteristics over broad ranges, to determine thresholds at which investigators might optimally select each trial design.Results: Registry-based trials were more cost effective than standard clinical trials 98.6% of the time. Data-related cost savings ranged from 4300 to 600,000 with variation in study characteristics. Cost differences were most reactive to the number of patients in a study, the number of data elements per patient available in a registry, and the speed with which research coordinators could manually abstract data. Registry incorporation resulted in cost savings when as few as 3768 independent data elements were available and when manual data abstraction took as little as 3.4 seconds per data field.Conclusions: Registries offer important resources for investigators. When available, their broad incorporation may help the scientific community reduce the costs of clinical investigation. We offer here a practical tool for investigators to assess potential costs savings.
AB - Background/Aims: Registry-based trials have emerged as a potentially cost-saving study methodology. Early estimates of cost savings, however, conflated the benefits associated with registry utilisation and those associated with other aspects of pragmatic trial designs, which might not all be as broadly applicable. In this study, we sought to build a practical tool that investigators could use across disciplines to estimate the ranges of potential cost differences associated with implementing registry-based trials versus standard clinical trials.Methods: We built simulation Markov models to compare unique costs associated with data acquisition, cleaning, and linkage under a registry-based trial design versus a standard clinical trial. We conducted one-way, two-way, and probabilistic sensitivity analyses, varying study characteristics over broad ranges, to determine thresholds at which investigators might optimally select each trial design.Results: Registry-based trials were more cost effective than standard clinical trials 98.6% of the time. Data-related cost savings ranged from 4300 to 600,000 with variation in study characteristics. Cost differences were most reactive to the number of patients in a study, the number of data elements per patient available in a registry, and the speed with which research coordinators could manually abstract data. Registry incorporation resulted in cost savings when as few as 3768 independent data elements were available and when manual data abstraction took as little as 3.4 seconds per data field.Conclusions: Registries offer important resources for investigators. When available, their broad incorporation may help the scientific community reduce the costs of clinical investigation. We offer here a practical tool for investigators to assess potential costs savings.
KW - Registry-based trial
KW - cost savings
KW - pragmatic trial
KW - trial design
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U2 - 10.1017/S1047951120001018
DO - 10.1017/S1047951120001018
M3 - Article
C2 - 32605679
AN - SCOPUS:85087396771
VL - 30
SP - 807
EP - 817
JO - Cardiology in the Young
JF - Cardiology in the Young
SN - 1047-9511
IS - 6
ER -