BACKGROUND: Over the past decade, annual funding for biomedical research has more than doubled while new molecular entity approvals have declined by one third. OBJECTIVE: To assess the value of practices commonly employed in the conduct of large-scale clinical trials, and to identify areas where costs could be reduced without compromising scientific validity. METHODS: In the qualitative phase of the study, an expert panel recommended potential modifications of mega-trial designs and operations in order to maximize their value (cost versus scientific benefit tradeoff). In the quantitative phase, a mega-trial economic model was used to assess the financial implications of these recommendations. Our initial chronic disease trial design included 20,000 patients randomized at 1000 sites. Each site was assigned 24 monitoring visits and a $10,000 per patient site payment. The case report form (CRF) was 60 pages long, and trial duration was assumed to be 48 months. RESULTS: The total costs of the initial trial design were $421 million ($US 2007). Following the expert panel's recommendations, we varied study duration, CRF length, number of sites, electronic data capture (EDC), and site management components to determine their individual and combined effects upon total trial costs. The use of EDC and modified site management practices were associated with significant reductions in total trial costs. When reductions in all five trial components were combined in a streamlined pharmaceutical industry design, a 59% reduction in total trial costs resulted. When we assumed an even more streamlined trial design than has typically been considered for regulatory submissions in the past, there was a 90% reduction in total trial costs. CONCLUSION: Our results suggest that it is possible to reduce substantially the cost of large-scale clinical trials without compromising the scientific validity of their results. If implemented, our recommendations could free billions of dollars annually for additional clinical studies. Research in the setting of clinical trials should be conducted to refine these findings.
RCT Entities:
BACKGROUND: Over the past decade, annual funding for biomedical research has more than doubled while new molecular entity approvals have declined by one third. OBJECTIVE: To assess the value of practices commonly employed in the conduct of large-scale clinical trials, and to identify areas where costs could be reduced without compromising scientific validity. METHODS: In the qualitative phase of the study, an expert panel recommended potential modifications of mega-trial designs and operations in order to maximize their value (cost versus scientific benefit tradeoff). In the quantitative phase, a mega-trial economic model was used to assess the financial implications of these recommendations. Our initial chronic disease trial design included 20,000 patients randomized at 1000 sites. Each site was assigned 24 monitoring visits and a $10,000 per patient site payment. The case report form (CRF) was 60 pages long, and trial duration was assumed to be 48 months. RESULTS: The total costs of the initial trial design were $421 million ($US 2007). Following the expert panel's recommendations, we varied study duration, CRF length, number of sites, electronic data capture (EDC), and site management components to determine their individual and combined effects upon total trial costs. The use of EDC and modified site management practices were associated with significant reductions in total trial costs. When reductions in all five trial components were combined in a streamlined pharmaceutical industry design, a 59% reduction in total trial costs resulted. When we assumed an even more streamlined trial design than has typically been considered for regulatory submissions in the past, there was a 90% reduction in total trial costs. CONCLUSION: Our results suggest that it is possible to reduce substantially the cost of large-scale clinical trials without compromising the scientific validity of their results. If implemented, our recommendations could free billions of dollars annually for additional clinical studies. Research in the setting of clinical trials should be conducted to refine these findings.
Authors: Jules T Mitchel; Yong Joong Kim; Joonhyuk Choi; Glen Park; Silvana Cappi; David Horn; Morgan Kist; Ralph B D Agostino Journal: Drug Inf J Date: 2011-07
Authors: Kathryn Blake; Janet T Holbrook; Holly Antal; David Shade; H Timothy Bunnell; Suzanne M McCahan; Robert A Wise; Chris Pennington; Paul Garfinkel; Tim Wysocki Journal: Contemp Clin Trials Date: 2015-04-03 Impact factor: 2.226
Authors: Maryam Y Garza; Michael Rutherford; Sahiti Myneni; Susan Fenton; Anita Walden; Umit Topaloglu; Eric Eisenstein; Karan R Kumar; Kanecia O Zimmerman; Mitra Rocca; Gideon Scott Gordon; Sam Hume; Zhan Wang; Meredith Zozus Journal: AMIA Annu Symp Proc Date: 2021-01-25
Authors: David F Lobach; Kensaku Kawamoto; Kevin J Anstrom; Garry M Silvey; Janese M Willis; Fred S Johnson; Rex Edwards; Jessica Simo; Pam Phillips; David R Crosslin; Eric L Eisenstein Journal: J Med Syst Date: 2013-01-13 Impact factor: 4.460