Value-Based Clinical Trials: Selecting Recruitment Rates and Trial Lengths in Different Regulatory Contexts
References
- (2010) How to optimize patient recruitment. Canadian J. Surgery 53(3):205–210.Google Scholar
- (2016) Response-adaptive designs for clinical trials: Simultaneous learning from multiple patients. Eur. J. Oper. Res. 248(2):619–633.Crossref, Google Scholar
- (2018) Extending a Bayesian decision-theoretic approach to value-based sequential clinical trial design. Rabe M, Juan A, Mustafee N, Skoogh A, Jain S, Johansson B, eds. Proc. 2018 Winter Simulation Conf. (IEEE, Inc., Piscataway, NJ), 2459–2470.Google Scholar
- (2022) Adaptive clinical trial designs with surrogates: When should we bother? Management Sci. 68(3):1982–2002.Google Scholar
- (2001) Quality-adjusted survival in the first year after the acute respiratory distress syndrome. Amer. J. Respiratory Critical Care Medicine 163(6):1389–1394.Crossref, Google Scholar
- (1995) Adaptive assignment vs. balanced randomization in clinical trials: A decision analysis. Statist. Medicine 14(3):231–246.Crossref, Google Scholar
- (1988) One-sided sequential stopping boundaries for clinical trials: A decision-theoretic approach. Biometrics 44(1):219–227.Crossref, Google Scholar
- (2016) The increasing trend in ceasarean section rates: Global, regional and national estimates: 1990–2014. PLoS One 11(2):e0148343.Crossref, Google Scholar
- (2014) Interventions to improve recruitment and retention in clinical trials: A survey and workshop to assess current practice and future priorities. Trials 15(399).Google Scholar
- (2007) Selecting a selection procedure. Management Sci. 53(12):1916–1932.Link, Google Scholar
- (2022) Flexible drug approval policies. Manufacturing Service Oper. Management 24(1):542–560.Google Scholar
- (2012) Regret analysis of stochastic and nonstochastic multi-armed bandit problems. Foundations and Trends in Machine Learning 5(1):1–122.Google Scholar
- (2014) Asia’s ascent—Global trends in biomedical R&D expenditures. New England J. Medicine 370(1):3–6.Crossref, Google Scholar
- (2005) Selection procedures with frequentist expected opportunity cost bounds. Oper. Res. 53(5):867–878.Link, Google Scholar
- (2017) A Bayesian decision-theoretic model of sequential experimentation with delayed response. J. Roy. Statist. Soc. B 79(5):1439–1462.Crossref, Google Scholar
- (2021) Bayesian sequential learning for clinical trials of multiple correlated medical interventions. Management Sci. 68(7):4919–4938.Link, Google Scholar
- (1999) The irrelevance of inference: A decision-making approach to the stochastic evaluation of healthcare technologies. J. Health Econom. 18(3):341–364.Crossref, Google Scholar
- (1996) An economic approach to clinical trial design and research priority-setting. Health Econom. 5(6):513–524.Crossref, Google Scholar
- (2000) Selecting treatments: A decision theoretic approach. J. Roy. Statist. Soc. Ser. A 163(2):211–215.Crossref, Google Scholar
- (2016) Cost effectiveness of surgical vs. non-surgical treatment of adults with displaced fractures of the proximal humerus. Bone Joint J. 92-B(2):152–159.Crossref, Google Scholar
- , DRAFFT Study Group (2014) Percutaneous fixation with Kirschner wires vs. volar locking plate fixation in adults with dorsally displaced fracture of distal radius: Randomised controlled trial. BMJ 349:4807.Crossref, Google Scholar
- (1970) Optimal Statistical Decisions, 1st ed. (McGraw-Hill, New York).Google Scholar
- (2016) Innovation in the pharmaceutical industry: New estimates of R&D costs. J. Health Econom. 47:20–33.Crossref, Google Scholar
- (2013) Discussion of the paper by Hampson and Jennison. J. Roy. Statist. Soc. B 75(1):40–54.Google Scholar
- (2015) Methods for the Economic Evaluation of Healthcare Programmes, 4th ed. (Oxford, New York).Google Scholar
- (2007) Expected value of information and decision making in HTA. Health Econom. 16(2):195–209.Crossref, Google Scholar
- , (2020) Drug reimbursement regulation in 6 peer countries. JAMA Internal Medicine 180(11):1510–1517.Crossref, Google Scholar
- (2021) Clinical trial regulation EU no. 536/2014. European Medicines Agency, Update April 21, 2021. Accessed June 28, 2022, https://www.ema.europa.eu/en/human-regulatory/research-development/clinical-trials/clinical-trial-regulation.Google Scholar
- (2020) Value of information analysis for research decisions—An introduction: Report 1 of the ISPOR value of information analysis emerging good practices task force. Value Health 23(2):139–150.Crossref, Google Scholar
- (2013) Integrating mortality and morbidity outcomes using quality-adjusted life years in critical care trials. Amer. J. Respiratory Critical Care Medicine 187(3):256–261.Crossref, Google Scholar
- (2019) A review of clinical trials with an adaptive design and health economic analysis. Value Health 22(4):391–398.Crossref, Google Scholar
- (2021) Cost-effective clinical trial design: Application of a Bayesian sequential stopping rule to the ProFHER pragmatic trial. Clinical Trials 18(6):647–656.Crossref, Google Scholar
- (2008) A knowledge-gradient policy for sequential information collection. SIAM J. Control Optim. 47(5):2410–2439.Crossref, Google Scholar
- (2000) A behavioural Bayes method for determining the size of a clinical trial. Drug Inform. J. 34(2):355–363.Crossref, Google Scholar
- (2010) Exploring the research decision space: The expected value of information for sequential research designs. Medical Decision Making 30(2):155–162.Crossref, Google Scholar
- (2013) Group sequential tests for delayed responses. J. Roy. Statist. Soc. B 75(1):3–54.Crossref, Google Scholar
- (2015) The ProFHER (proximal fracture of the humerus: Evaluation by randomisation) trial—A pragmatic multicentre randomised controlled trial evaluating the clinical effectiveness and cost-effectiveness of surgical compared with non-surgical treatment for proximal fracture of the humerus in adults. Health Tech. Assessment 19(24):1–280.Crossref, Google Scholar
- (2016) Toward a new era of trust and transparency in clinical trials. JAMA 316(13):1353–1354.Crossref, Google Scholar
- (1998) Comparison of Bayesian and frequentist assessments of uncertainty for selecting the best system. Medeiros DJ, Watson EJ, Manivannan M, Carson J, eds. Proc. 1998 Winter Simulation Conf. (IEEE, Inc., Piscataway, NJ), 727–734.Google Scholar
- (1989) Interim analyses: The repeated confidence interval approach. J. Roy. Statist. Soc. B 51(3):305–361.Google Scholar
- (2016) Late-stage pharmaceutical R&D and pricing policies under two-stage regulation. J. Health Econom. 50:298–311.Crossref, Google Scholar
- Kaplan EH, Brandeau ML, eds. (1994) Modeling the AIDS Epidemic: Planning, Policy, and Prediction (Raven Press, New York).Google Scholar
- (2017) Clinical trials for new drug development: Optimal investment and application. Manufacturing Service Oper. Management 19(3):437–452.Link, Google Scholar
- (2019) A value of information analysis of research on the 21-gene assay for breast cancer management. Value Health 22(10):1102–1110.Crossref, Google Scholar
- (1981) Introduction to sample size determination and power analysis for clinical trials. Controlled Clinical Trials 2(2):93–113.Crossref, Google Scholar
- (2021) Time to marketing of generic drugs after patent expiration in Canada. JAMA Network Open 4(3):e211143–e211143.Crossref, Google Scholar
- (2019) Is this study feasible? Facilitating management of pragmatic trial planning milestones under a phased award funding mechanism. Trials 20(2):241–251.Google Scholar
- (2019) Matching clinical trials with unmet clinical need. BioScience Today 16:24–25. https://www.biosciencetoday.co.uk/matching-clinical-trials-with-unmet-clinical-need/.Google Scholar
- (2008) Controlling co-epidemics: Analysis of HIV and tuberculosis infection dynamics. Oper. Res. 56(6):1366–1381.Link, Google Scholar
- (2020) The inclusion of economic endpoints as outcomes in clinical trials reported to clinicaltrials. gov. J. Managed Care Specialty Pharmacy 26(4):386–393.Crossref, Google Scholar
- (2017) Use of Bayesian decision analysis to minimize harm in patient-centered randomized clinical trials in oncology. JAMA Oncology 3(9):e170123.Crossref, Google Scholar
- National Institute for Health and Care Excellence (2013) Guide to the methods of technology appraisal. Accessed June 28, 2022, https://www.nice.org.uk/process/pmg9/chapter/foreword.Google Scholar
- National Institute for Health and Care Excellence (2014) Developing NICE guidelines: The manual. Accessed June 28, 2022, https://www.nice.org.uk/process/pmg20/chapter/incorporating-economic-evaluation.Google Scholar
- National Institute for Health and Care Excellence (2018) Guide to the processes of technology appraisal. Accessed June 28, 2022, https://www.nice.org.uk/Media/Default/About/what-we-do/NICE-guidance/NICE-technology-appraisals/technology-appraisal-processes-guide-apr-2018.pdf.Google Scholar
- National Institute for Health and Care Research (2018) NIHR Efficacy and mechanism evaluation. Accessed June 28, 2022, https://www.nihr.ac.uk/explore-nihr/funding-programmes/efficacy-and-mechanism-evaluation.htm.Google Scholar
- National Institute for Health and Care Research (2019) NIHR Clinical Research Network: Impact and value assessment. Accessed June 28, 2022, https://www.nihr.ac.uk/documents/impact-and-value-report/21427#Executive_summary.Google Scholar
- National Institute for Health and Care Research (2020) Annual efficient studies funding calls for CTU projects. Accessed June 28, 2022, https://www.nihr.ac.uk/documents/ad-hoc-funding-calls-for-ctu-projects/20141.Google Scholar
- (2018) Adaptive designs in clinical trials: Why use them, and how to run and report them. BMC Medicine 16:29.Crossref, Google Scholar
- (2015) From market access to patient access: Overview of evidence-based approaches for the reimbursement and pricing of pharmaceuticals in 36 European countries. Health Res. Policy Systems 13(39).Crossref, Google Scholar
- (2014) How to improve R&D productivity: The pharmaceutical industry’s grand challenge. Nature Rev. Drug Discovery 9:203–214.Crossref, Google Scholar
- (2014) Optimal Bayesian sequential sampling rules for the economic evaluation of health technologies. J. Roy. Statist. Soc. Ser. A 177(2):419–438.Crossref, Google Scholar
- (1961) Applied Statistical Decision Theory, 1st ed. (Harvard University Graduate School of Business Administration, Boston).Google Scholar
- (2015) Surgical vs nonsurgical treatment of adults with displaced fractures of the proximal humerus: The PROFHER randomized clinical trial. JAMA 313(10):1037–1047.Crossref, Google Scholar
- (2007) The Bayesian Choice: From Decision-Theoretic Foundations to Computational Implementation, 2nd ed. (Springer, New York).Google Scholar
- (2018) Optimal patient enrollment in sequential adaptive clinical trials with binary response. Preprint, submitted August 28, https://dx.doi.org/10.2139/ssrn.3234590.Google Scholar
- (2021) Market exclusivity length for drugs with new generic or biosimilar competition, 2012–2018. Clinical Pharmacology Therapeutics 109(2):367–371.Crossref, Google Scholar
- (2020) Value of information analytical methods: Report 2 of the ISPOR value of information analysis emerging good practices task force. Value Health 23(3):277–286.Crossref, Google Scholar
- (2012) The knowledge gradient algorithm for a general class of online learning problems. Oper. Res. 60(1):180–195.Link, Google Scholar
- (2021) National healthcare economic evaluation guidelines: A cross-country comparison. PharmacoEconom. Open 5(3):349–364.Crossref, Google Scholar
- U.S. Food and Drug Administration (2015) Patents and exclusivity (U.S. Food and Drug Administration, Silver Spring, MD). Accessed June 28, 2022, https://www.fda.gov/downloads/drugs/developmentapprovalprocess/smallbusinessassistance/ucm447307.pdf.Google Scholar
- U.S. Food and Drug Administration (2019) Interacting with the FDA on complex innovative trial designs for drugs and biological products (U.S. Food and Drug Administration, Silver Spring, MD). Accessed June 28, 2022, https://www.fda.gov/regulatory-information/search-fda-guidance-documents/interacting-fda-complex-innovative-trial-designs-drugs-and-biological-products.Google Scholar
- U.S. Department of Health and Human Services (2014) Examination of Clinical Trial Costs and Barriers for Drug Development (U.S. Department of Health and Human Services, Office of the Assistant Secretary for Planning and Evaluation). Accessed June 28, 2022, https://aspe.hhs.gov/reports/examination-clinical-trial-costs-barriers-drug-development-0.Google Scholar
- (2018) Covariate-adjusted response-adaptive randomization for multi-arm clinical trials using a modified forward looking Gittins index rule. Biometrics 74(1):49–57.Crossref, Google Scholar
- (2018) Response-adaptive designs for binary responses: How to offer patient benefit while being robust to time trends? Pharmaceutical Statist. 17(2):182–197.Crossref, Google Scholar
- (2017) Recruitment and retention of participants in randomised controlled trials: A review of trials funded and published by the United Kingdom Health Technology Assessment programme. BMJ Open 7(3):e015276.Crossref, Google Scholar
- (2008) Optimal sample size determination from an industry perspective based on the expected value of information. Clinical Trials 5(6):587–594.Crossref, Google Scholar
- (2017) A Bayesian adaptive design for clinical trials in rare diseases. Comput. Statist. Data Anal. 113:136–153.Crossref, Google Scholar
- World Trade Organization (2006) Pharmaceutical patents and the TRIPS agreement. Accessed June 28, 2022, https://www.wto.org/english/tratop_e/trips_e/pharma_ato186_e.htm.Google Scholar
- (2016) Bayesian optimization via simulation with pairwise sampling and correlated prior beliefs. Oper. Res. 64(2):542–559.Link, Google Scholar

