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Registry-based trials: a potential model for cost savings?

Part of: Pediatric Heart Network The International Society for Nomenclature of Paediatric and Congenital Heart Disease (ISNPCHD)

Published online by Cambridge University Press:  08 May 2020

Brett R. Anderson ,
Evelyn G. Gotlieb Open the ORCID record for Evelyn G. Gotlieb [Opens in a new window] ,
Kevin Hill ,
Kimberly E. McHugh ,
Mark A. Scheurer ,
Carlos M. Mery ,
Glenn J. Pelletier ,
Jonathan R. Kaltman ,
Owen J. White  and
Felicia L. Trachtenberg
...Show all authors
Brett R. Anderson*
Affiliation:
Division of Pediatric Cardiology, NewYork-Presbyterian/Morgan Stanley Children’s Hospital, Columbia University Irving Medical Center, New York, NY, USA
Evelyn G. Gotlieb
Affiliation:
The Wharton School, University of Pennsylvania, Philadelphia, PA, USA
Kevin Hill
Affiliation:
Department of Pediatrics, Duke Clinical Research Institute, Duke University Medical Center, Durham, NC, USA
Kimberly E. McHugh
Affiliation:
Department of Pediatrics, Medical University of South Carolina, Charleston, SC, USA
Mark A. Scheurer
Affiliation:
Department of Pediatrics, Medical University of South Carolina, Charleston, SC, USA
Carlos M. Mery
Affiliation:
Texas Center for Pediatric and Congenital Heart Disease, Dell Children’s Medical Center, University of Texas Dell Medical School, Austin, TX, USA
Glenn J. Pelletier
Affiliation:
Division of Cardiac Surgery, Nemours Cardiac Center, Alfred I duPont Hospital for Children, Wilmington, DE, USA
Jonathan R. Kaltman
Affiliation:
National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA
Owen J. White
Affiliation:
CardioAccess, Fort Lauderdale, FL, USA
Felicia L. Trachtenberg
Affiliation:
New England Research Institutes, Watertown, MA, USA
Danielle Hollenbeck-Pringle
Affiliation:
New England Research Institutes, Watertown, MA, USA
Brian W. McCrindle
Affiliation:
Department of Cardiology at SickKids, Labatt Family Heart Centre, University of Toronto, The Hospital for Sick Children, Toronto, Canada
Donna M. Sylvester
Affiliation:
Department of Cardiology, The Children’s Hospital of Philadelphia, Philadelphia, PA, USA
Aaron W. Eckhauser
Affiliation:
Section of Pediatric Cardiothoracic Surgery, Division of Cardiothoracic Surgery, Department of Surgery, University of Utah, Salt Lake City, UT, USA
Sara K. Pasquali
Affiliation:
Division of Pediatric Cardiology, C.S. Mott Children’s Hospital, Ann Arbor, MI, USA
Jeffery B. Anderson
Affiliation:
Division of Pediatric Cardiology, Cincinnati Children’s Hospital and Medical Center, Cincinnati, OH, USA
Marcus S. Schamberger
Affiliation:
Division of Pediatric Cardiology, Riley Children’s Hospital, Indianapolis, IN, USA
Subhadra Shashidharan
Affiliation:
Division of Cardiovascular Surgery, Children’s Healthcare of Atlanta, Atlanta, GA, USA
Jeffrey P. Jacobs
Affiliation:
Division of Cardiovascular Surgery, Department of Surgery, Johns Hopkins All Children’s Heart Institute, St. Petersburg, FL, USA
Marshall L. Jacobs
Affiliation:
Division of Cardiac Surgery, Department of Surgery, Johns Hopkins School of Medicine, Baltimore, MD, USA
Marko Boskovski
Affiliation:
Division of Cardiac Surgery, Harvard Medical School, The Brigham and Women’s Hospital, Boston, MA, USA
Jane W. Newburger
Affiliation:
Department of Cardiology, Boston Children’s Hospital, Boston, MA, USA Department of Pediatrics, Harvard Medical School, Boston, MA, USA
Meena Nathan
Affiliation:
Department of Pediatrics, Harvard Medical School, Boston, MA, USA Department of Cardiac Surgery, Boston Children’s Hospital, Boston, MA, USA
*
Author for correspondence: B. Anderson, MD, MBA, MS, NewYork-Presbyterian/Morgan Stanley Children’s Hospital, 3959 Broadway, CH-2 North, New York, NY10032-3784, USA. Tel: +1 (212) 305 8509; Fax: +1 (212) 305 4429. E-mail: [email protected]
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Abstract

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.

Keywords

Registry-based trialcost savingspragmatic trialtrial design
Type
Original Article
Information
Cardiology in the Young , Volume 30 , Issue 6 , June 2020 , pp. 807 - 817
Copyright
© The Author(s), 2020. Published by Cambridge University Press

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Footnotes

*

A complete list of non-author contributors appears in Appendix 1.

References

Califf, RM, Sanderson, I, Miranda, ML.The future of cardiovascular clinical research: informatics, clinical investigators, and community engagement. JAMA 2012; 308: 1747–1748. doi: 10.1001/jama.2012.28745.CrossRefGoogle ScholarPubMed
Tricoci, P, Allen, JM, Kramer, JM, et al.Scientific evidence underlying the ACC/AHA clinical practice guidelines. JAMA 2009; 301: 831–841. doi: 10.1001/jama.2009.205.CrossRefGoogle ScholarPubMed
Frobert, O, Lagerqvist, B, Olivecrona, GK, et al.Thrombus aspiration during ST-segment elevation myocardial infarction. NEJM 2013; 369: 1587–1597. doi: 10.1056/NEJMoa1308789.CrossRefGoogle ScholarPubMed
Hess, CN, Rao, SV, Kong, DF, et al.Embedding a randomized clinical trial into an ongoing registry infrastructure: unique opportunities for efficiency in design of the Study of Access site For Enhancement of Percutaneous Coronary Intervention for Women (SAFE-PCI for Women). Am Heart J 2013; 166: 421–428. doi: 10.1016/j.ahj.2013.06.013.CrossRefGoogle Scholar
Huang, SS, Septimus, E, Kleinman, K, et al.Targeted versus universal decolonization to prevent ICU infection. NEJM 2013; 368: 2255–2265. doi: 10.1056/NEJMoa1207290.CrossRefGoogle ScholarPubMed
Thabane, L, Kaczorowski, J, Dolovich, L, et al.Reducing the confusion and controversies around pragmatic trials: using the Cardiovascular Health Awareness Program (CHAP) trial as an illustrative example. Trials 2015; 16: 387. doi: 10.1186/s13063-015-0919-3.CrossRefGoogle ScholarPubMed
Detsky, AS.Using cost-effectiveness analysis to improve the efficiency of allocating funds to clinical trials. Stat Med 1990; 9: 173–184.CrossRefGoogle ScholarPubMed
Eisenstein, EL, Lemons, PW, 2nd, Tardiff, BE, et al.Reducing the costs of phase III cardiovascular clinical trials. Am heart J 2005; 149: 482–488. doi: 10.1016/j.ahj.2004.04.049.CrossRefGoogle ScholarPubMed
Sertkaya, A, Wong, HH, Jessup, A, et al.Key cost drivers of pharmaceutical clinical trials in the United States. Clin Trials 2016; 13: 117–126. doi: 10.1177/1740774515625964.CrossRefGoogle ScholarPubMed
Lauer, MS, D’Agostino, RB Sr.The randomized registry trial--the next disruptive technology in clinical research? NEJM 2013; 369: 1579–1581. doi: 10.1056/NEJMp1310102.CrossRefGoogle ScholarPubMed
Ramsberg, J, Neovius, M.Register or electronic health records enriched randomized pragmatic trials: the future of clinical effectiveness and cost-effectiveness trials? Nordic J Health Econ 2015; 5: 62. clinical trials, pragmatic trials, disease registries, electronic health records, costs. doi: 10.5617/njhe.1386.CrossRefGoogle Scholar
Ford, I, Norrie, J.Pragmatic trials. NEJM 2016; 375: 454–463. doi: 10.1056/NEJMra1510059.CrossRefGoogle ScholarPubMed
Li, G, Sajobi, TT, Menon, BK, et al.Registry-based randomized controlled trials- what are the advantages, challenges, and areas for future research? J Clin Epi 2016; 80: 16–24. doi: 10.1016/j.jclinepi.2016.08.003.CrossRefGoogle ScholarPubMed
Loudon, K, Treweek, S, Sullivan, F, et al.The PRECIS-2 tool: designing trials that are fit for purpose. BMJ 2015; 350: h2147. doi: 10.1136/bmj.h2147.CrossRefGoogle ScholarPubMed
Nathan, M, Trachtenberg, FL, Van Rompay, MI, et al.Design and objectives of the pediatric heart network’s residual lesion score study. J Thorac Cardiovasc Surg 2019. doi: 10.1016/j.jtcvs.2019.10.146.Google Scholar
Anderson, BR, Kumar, SR, Gottlieb-Sen, D, et al.The congenital heart technical skill study: rationale and design. World J Pediatr Congenit Heart Surg 2019; 10: 137–144. doi: 10.1177/2150135118822689.CrossRefGoogle ScholarPubMed
Hill, K, Baldwin, H, Bichel, D, et al.Rationale and design of the steroids to reduce systemic inflammation after infant heart surgery (stress) trial. Am Heart J 2019.Google ScholarPubMed
Boskovski, M. The Brigham and Women’s Hospital, Harvard Medical School, 2019.Google Scholar
ResearchMatch. https://www.researchmatch.org/.Google Scholar
Pediatric Health Information System Database. https://www.childrenshospitals.org/programs-and-services/data-analytics-and-research/pediatric-analytic-solutions/pediatric-health-information-system.Google Scholar
Healthcare Cost and Utilization Project. https://www.hcup-us.ahrq.gov/databases.jsp.Google Scholar
Surveillance, Epidemiology, and End Results. https://seer.cancer.gov/registries/.Google Scholar
Society of Thoracic Surgeons. https://www.sts.org/registries-research-center/sts-national-database.Google Scholar
American College of Cardiology. https://cvquality.acc.org/NCDR-Home/registries.Google Scholar
Anderson, BR, Dragan, K, Glied, S, et al.Building capacity to assess longitudinal outcomes and health expenditures for children with congenital heart disease. J Am Coll Card 2019; 73: 649. doi: 10.1016/s0735-1097(19)31257-4.CrossRefGoogle Scholar
Boscoe, FP, Schrag, D, Chen, K, et al.Building capacity to assess cancer care in the Medicaid population in New York State. Health Serv Res 2011; 46: 805–820. doi: 10.1111/j.1475-6773.2010.01221.x.CrossRefGoogle ScholarPubMed
Thorpe, KE, Zwarenstein, M, Oxman, AD, et al.A pragmatic-explanatory continuum indicator summary (PRECIS): a tool to help trial designers. J Clin Epidemiol 2009; 62: 464–475. doi: 10.1016/j.jclinepi.2008.12.011.CrossRefGoogle ScholarPubMed
Schwartz, D, Lellouch, J.Explanatory and pragmatic attitudes in therapeutical trials. J Chronic Dis 1967; 20: 637–648. doi: 10.1016/0021-9681(67)90041-0.CrossRefGoogle ScholarPubMed
McDonald, AM, Knight, RC, Campbell, MK, et al.What influences recruitment to randomised controlled trials? A review of trials funded by two UK funding agencies. Trials 2006; 7: 9. doi: 10.1186/1745-6215-7-9.CrossRefGoogle ScholarPubMed
Carlisle, B, Kimmelman, J, Ramsay, T, et al.Unsuccessful trial accrual and human subjects protections: an empirical analysis of recently closed trials. Clin Trials 2015; 12: 77–83. doi: 10.1177/1740774514558307.CrossRefGoogle ScholarPubMed
Tan, MH, Thomas, M, MacEachern, MP.Using registries to recruit subjects for clinical trials. Contemp Clin Trials 2015; 41: 31–38. doi: 10.1016/j.cct.2014.12.012.Google ScholarPubMed
Strasser, JE, Cola, PA, Rosenblum, D.Evaluating various areas of process improvement in an effort to improve clinical research: discussions from the 2012 Clinical Translational Science Award (CTSA) Clinical Research Management workshop. Clin Transl Sci 2013; 6: 317–320. doi: 10.1111/cts.12051.CrossRefGoogle Scholar
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