Woodcock, J, LaVange, LM. Master protocols to study multiple therapies, multiple diseases, or both. N Eng J Med. 2017;377(1):62–70.Google Scholar

Park, JJH, Siden, E, Zoratti, MJ, et al. Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols. Trials. 2019;20(1):572.CrossRefGoogle ScholarPubMed

Siden, EG, Park, JJ, Zoratti, MJ, et al. Reporting of master protocols towards a standardized approach: a systematic review. Contemp Clin Trials Commun. 2019;15:100406.CrossRefGoogle ScholarPubMed

Park, JJH, Harari, O, Dron, L, et al. An overview of platform trials with a checklist for clinical readers. J Clin Epidemiol. 2020;125:1–8.CrossRefGoogle ScholarPubMed

Mills, EJ, Thorlund, K, Ioannidis, JP. Demystifying trial networks and network meta-analysis. BMJ. 2013;346:f2914.Google Scholar

Redman, MW, Allegra, CJ. The master protocol concept. Semin Oncol. 2015;42(5):724–30.CrossRefGoogle ScholarPubMed

Hirakawa, A, Asano, J, Sato, H, Teramukai, S. Master protocol trials in oncology: review and new trial designs. Contemp Clin Trials Commun. 2018;12:1–8.CrossRefGoogle ScholarPubMed

Park, JJH, Hsu, G, Siden, EG, Thorlund, K, Mills, EJ. An overview of precision oncology basket and umbrella trials for clinicians. CA Cancer J Clin. 2020;70(2):125–37.CrossRefGoogle ScholarPubMed

United States Department of Health and Human Services, Food and Drug Administration. Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics Guidance for Industry. United States Department of Health and Human Services; 2022. www.fda.gov/media/120721/download.Google Scholar

United States Department of Health and Human Services, Food and Drug Administration. Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics Guidance for Industry (Draft Guidance). United States Department of Health and Human Services; 2018. www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM621817.pdf.Google Scholar

Zhou, X, Lei, L, Liu, J, et al. A systems approach to refine disease taxonomy by integrating phenotypic and molecular networks. EBioMedicine. 2018;31:79–91.CrossRefGoogle ScholarPubMed

Mullauer, L. Milestones in pathology-from histology to molecular biology. Memo. 2017;10(1):42–5.Google Scholar

Zhang, H, Zeng, Z, Mukherjee, A, Shen, B. Molecular diagnosis and classification of inflammatory bowel disease. Expert Rev Mol Diagn. 2018;18(10):867–86.CrossRefGoogle ScholarPubMed

Abrams, J, Conley, B, Mooney, M, et al. National Cancer Institute’s Precision Medicine Initiatives for the new National Clinical Trials Network. Am Soc Clin Oncol Educ Book. 2014:71–6.CrossRefGoogle Scholar

Heckman-Stoddard, BM, Smith, JJ. Precision medicine clinical trials: defining new treatment strategies. Semin Oncol Nurs. 2014;30(2):109–16.Google Scholar

Kumar-Sinha, C, Chinnaiyan, AM. Precision oncology in the age of integrative genomics. Nat Biotechnol. 2018;36(1):46–60.CrossRefGoogle ScholarPubMed

National Institutes of Health All of US Research Program. About the All of Us Research Program. Cited 2019. https://allofus.nih.gov/about/about-all-us-research-programGoogle Scholar

Clinical Trials Transformation Initiative. Master Protocol Studies; 2021 https://ctti-clinicaltrials.org/our-work/novel-clinical-trial-designs/master-protocol-studies/Google Scholar

Berry, SM, Connor, JT, Lewis, RJ. The platform trial: an efficient strategy for evaluating multiple treatments. JAMA. 2015;313(16):1619–20.Google Scholar

Adaptive Platform, Trials C. Adaptive platform trials: definition, design, conduct and reporting considerations. Nat Rev Drug Discov. 2019;18(10):797–807.CrossRefGoogle Scholar

Park, JJH, Dron, L, Mills, EJ. Moving forward in clinical research with master protocols. Contemp Clin Trials. 2021;106:106438.CrossRefGoogle ScholarPubMed

Vanderbeek, AM, Bliss, JM, Yin, Z, Yap, C. Implementation of platform trials in the COVID-19 pandemic: a rapid review. Contemp Clin Trials. 2022;112:106625.Google Scholar

Dean, NE, Gsell, PS, Brookmeyer, R, et al. Creating a framework for conducting randomized clinical trials during disease outbreaks. N Engl J Med. 2020;382(14):1366–9.CrossRefGoogle ScholarPubMed

Schiavone, F, Bathia, R, Letchemanan, K, et al. This is a platform alteration: a trial management perspective on the operational aspects of adaptive and platform and umbrella protocols. Trials. 2019;20(1):264.CrossRefGoogle ScholarPubMed

Fogel, DB. Factors associated with clinical trials that fail and opportunities for improving the likelihood of success: a review. Contemp Clin Trials Commun. 2018;11:156–64.CrossRefGoogle ScholarPubMed

Antoniou, M, Kolamunnage-Dona, R, Wason, J, et al. Biomarker-guided trials: challenges in practice. Contemp Clin Trials Commun. 2019;16:100493.Google Scholar

Preston, J, VanZeeland, A, Peiffer, D. Innovation at Illumina: the road to the $600 human genome. Nature (Illumia). 2021.Google Scholar

Lam, VK, Papadimitrakopoulou, V. Master protocols in lung cancer: experience from Lung Master Protocol. Curr Opin Oncol. 2018;30(2):92–7.CrossRefGoogle ScholarPubMed

Ferrarotto, R, Redman, MW, Gandara, DR, Herbst, RS, Papadimitrakopoulou, VA. Lung-MAP – framework, overview, and design principles. Chin Clin Oncol. 2015;4(3):36.Google ScholarPubMed

Houston, L, Yu, P, Martin, A, Probst, Y. Heterogeneity in clinical research data quality monitoring: a national survey. J Biomed Inform. 2020;108:103491.CrossRefGoogle ScholarPubMed

Kuchinke, W, Ohmann, C, Yang, Q, et al. Heterogeneity prevails: the state of clinical trial data management in Europe – results of a survey of ECRIN centres. Trials. 2010;11:79.Google Scholar

Remap-Cap Investigators, ACTIV-4a Investigators, Attacc Investigators, Goligher, EC, Bradbury, CA, McVerry, BJ, et al. Therapeutic anticoagulation with heparin in critically ill patients with Covid-19. N Engl J Med. 2021;385(9):777–89.Google Scholar

Thorlund, K, Dron, L, Park, J, et al. A real-time dashboard of clinical trials for COVID-19. Lancet Digit Health. 2020;2(6):e286–e7.Google Scholar

Dillman, A, Park, JJH, Zoratti, MJ, et al. Reporting and design of randomized controlled trials for COVID-19: a systematic review. Contemp Clin Trials. 2021;101:106239.Google Scholar

Park, JJ, Mogg, R, Smith, GE, et al. How COVID-19 has fundamentally changed clinical research in global health. Lancet Glob Health. 2021;9(5):e711–e20.Google Scholar

NIHR. Recruiting patients for clinical trials for COVID-19 therapeutics. 2020. www.nihr.ac.uk/news/uks-chief-medical-officers-urge-hospitals-to-recruit-60-of-eligible-covid-19-patients-into-recovery-trial/25515Google Scholar

Recovery Collaborative Group, Horby, P, Lim, WS, Emberson, JR, et al. Dexamethasone in hospitalized patients with Covid-19. N Engl J Med. 2021;384(8):693–704.Google Scholar

Berry, DA, Graves, T, Connor, J, et al. Adaptively randomized seamless-phase multiarm platform trial: Glioblastoma Multiforme Adaptive Global Innovative Learning Environment (GBM AGILE). Cancer Research. 2017;77(13 Supplement 1).CrossRefGoogle Scholar

Rugo, HS, Olopade, OI, DeMichele, A, et al. Adaptive randomization of veliparib-carboplatin treatment in breast cancer. N Eng J Med. 2016;375(1):23–34.Google Scholar

Barker, AD, Sigman, CC, Kelloff, GJ, et al. I-SPY 2: an adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin Pharmacol Ther. 2009;86(1):97–100.Google Scholar

Papadimitrakopoulou, V, Lee, JJ, Wistuba, II, et al. The BATTLE-2 study: a biomarker-integrated targeted therapy study in previously treated patients with advanced non-small-cell lung cancer. J Clin Oncol. 2016;34(30):3638–47.Google Scholar

Tam, AL, Kim, ES, Lee, JJ, et al. Feasibility of image-guided transthoracic core-needle biopsy in the BATTLE lung trial. J Thorac Oncol. 2013;8(4):436–42.Google Scholar

Kim, ES, Herbst, RS, Wistuba, II, et al. The BATTLE trial: personalizing therapy for lung cancer. Cancer Discov. 2011;1(1):44–53.Google Scholar

Wang, H, Yee, D. I-SPY 2: a neoadjuvant adaptive clinical trial designed to improve outcomes in high-risk breast cancer. Curr Breast Cancer Rep. 2019;11(4):303–10.CrossRefGoogle ScholarPubMed

Bogin, V. Master protocols: new directions in drug discovery. Contemp Clin Trials Commun. 2020;18:100568.Google Scholar

Ali, A, Hoyle, A, Haran, AM, et al. Association of bone metastatic burden with survival benefit from prostate radiotherapy in patients with newly diagnosed metastatic prostate cancer: a secondary analysis of a randomized clinical trial. JAMA Oncol. 2021;7(4):555–63.Google Scholar

Cortazar, P, Geyer, CE. Pathological complete response in neoadjuvant treatment of breast cancer. Ann Surg Oncol. 2015;22(5):1441–6.Google Scholar

Siden, EG, Park, JJ, Zoratti, MJ, et al. Reporting of master protocols towards a standardized approach: a systematic review. Contemp Clin Trials Commun. 2019;15:100406.CrossRefGoogle ScholarPubMed

Park, JJH, Siden, E, Zoratti, MJ, et al. Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols. Trials. 2019;20(1):572.CrossRefGoogle ScholarPubMed

Vanderbeek, AM, Bliss, JM, Yin, Z, Yap, C. Implementation of platform trials in the COVID-19 pandemic: a rapid review. Contemp Clin Trials. 2022;112:106625.Google Scholar

Park, JJ, Mogg, R, Smith, GE, et al. How COVID-19 has fundamentally changed clinical research in global health. Lancet Glob Health. 2021;9(5):e711–e20.Google Scholar

Woodcock, J, LaVange, LM. Master protocols to study multiple therapies, multiple diseases, or both. N Eng J Medi. 2017;377(1):62–70.Google Scholar

Berry, SM, Connor, JT, Lewis, RJ. The platform trial: an efficient strategy for evaluating multiple treatments. JAMA. 2015;313(16):1619–20.Google Scholar

Angus, DC, Alexander, BM, Berry, S, et al. Adaptive platform trials: definition, design, conduct and reporting considerations. Nat Rev Drug Discov. 2019;18(10):797–807.Google Scholar

Park, JJH, Detry, MA, Murthy, S, Guyatt, G, Mills, EJ. How to use and interpret the results of a platform trial: users’ guide to the medical literature. JAMA. 2022;327(1):67–74.Google Scholar

Thorlund, K, Dron, L, Park, J, Hsu, G, Forrest, JI, Mills, EJ. A real-time dashboard of clinical trials for COVID-19. Lancet Digit Health. 2020;2(6):e286–e7.Google ScholarPubMed

Kanters, S, Mills, EJ, Thorlund, K, Bucher, HC, Ioannidis, JP. Antiretroviral therapy for initial human immunodeficiency virus/AIDS treatment: critical appraisal of the evidence from over 100 randomized trials and 400 systematic reviews and meta-analyses. Clin Microbiol Infect. 2014;20(2):114–22.CrossRefGoogle ScholarPubMed

Parmar, MK, Carpenter, J, Sydes, MR. More multiarm randomised trials of superiority are needed. Lancet. 2014;384(9940):283–4.CrossRefGoogle ScholarPubMed

Ventz, S, Alexander, BM, Parmigiani, G, Gelber, RD, Trippa, L. Designing clinical trials that accept new arms: an example in metastatic breast cancer. J Clin Oncolo. 2017;35(27):3160–8.Google Scholar

Parmar, MK, Barthel, FM, Sydes, M, et al. Speeding up the evaluation of new agents in cancer. J Natl Cancer Inst. 2008;100(17):1204–14.CrossRefGoogle ScholarPubMed

Sydes, MR, Parmar, MKB, James, ND, et al. Issues in applying multi-arm multi-stage methodology to a clinical trial in prostate cancer: the MRC STAMPEDE trial. Trials. 2009;10:39.Google Scholar

Parmar, MK, Sydes, MR, Cafferty, FH, et al. Testing many treatments within a single protocol over 10 years at MRC Clinical Trials Unit at UCL: multi-arm, multi-stage platform, umbrella and basket protocols. Clin Trials. 2017;14(5):451–61.Google Scholar

Mills, EJ, Thorlund, K, Ioannidis, JP. Demystifying trial networks and network meta-analysis. BMJ. 2013;346:f2914.Google Scholar

Park, JJH, Harari, O, Dron, L, et al. An overview of platform trials with a checklist for clinical readers. J Clin Epidemiol. 2020;125:1–8.Google Scholar

Angus, DC, Derde, L, Al-Beidh, F, et al. Effect of hydrocortisone on mortality and organ support in patients with severe COVID-19: the REMAP-CAP COVID-19 Corticosteroid Domain Randomized Clinical Trial. JAMA. 2020;324(13):1317–29.Google ScholarPubMed

Recovery Collaborative Group, Horby, P, Lim, WS, Emberson, JR, et al. Dexamethasone in hospitalized patients with Covid-19. N Engl J Med. 2021;384(8):693–704.Google ScholarPubMed

Park, JJH, Mogg, R, Smith, GE, et al. How COVID-19 has fundamentally changed clinical research in global health. Lancet Glob Health. 2021;9(5):e711–e20.CrossRefGoogle ScholarPubMed

Bugin, K, Woodcock, J. Trends in COVID-19 therapeutic clinical trials. Nat Rev Drug Discov. 2021;20(4):254–5.Google Scholar

Recovery Collaborative Group, Horby, P, Lim, WS, Emberson, JR, et al. Dexamethasone in hospitalized patients with Covid-19 – preliminary report. N Engl J Med. 2020;384(8):693–704.Google Scholar

Beigel, JH, Tomashek, KM, Dodd, LE, et al. Remdesivir for the treatment of Covid-19 – final report. N Engl J Med. 2020;383:1813–26.CrossRefGoogle ScholarPubMed

Writing Committee for the Remap-CAP Investigators, Angus, DC, Derde, L, Al-Beidh, F, et al. Effect of hydrocortisone on mortality and organ support in patients with severe COVID-19: the REMAP-CAP COVID-19 Corticosteroid Domain Randomized Clinical Trial. JAMA. 2020;324(13):1317–29.Google Scholar

Angus, DC, Berry, S, Lewis, RJ, et al. The Randomized Embedded Multifactorial Adaptive Platform for Community-acquired Pneumonia (REMAP-CAP) study: rationale and design. Ann Am Thorac Soc. 2020;17(7):879–91.Google Scholar

Reis, G, Moreira Silva, E, Medeiros Silva, DC, et al. Effect of early treatment with hydroxychloroquine or lopinavir and ritonavir on risk of hospitalization among patients with COVID-19: the TOGETHER Randomized Clinical Trial. JAMA Netw Open. 2021;4(4):e216468.Google Scholar

Group PTC. Azithromycin for community treatment of suspected COVID-19 in people at increased risk of an adverse clinical course in the UK (PRINCIPLE): a randomised, controlled, open-label, adaptive platform trial. Lancet. 2021;397(10279):1063–74.Google Scholar

Park, JJH, Dron, L, Mills, EJ. Moving forward in clinical research with master protocols. Contemp Clin Trials. 2021;106:106438.Google Scholar

Saville, BR, Berry, SM. Efficiencies of platform clinical trials: a vision of the future. Clin Trials. 2016;13(3):358–66.Google Scholar

Adaptive Platform Trials C. Adaptive platform trials: definition, design, conduct and reporting considerations. Nat Rev Drug Discov. 2019;18(10):797–807.CrossRefGoogle Scholar

Hummel, J, Wang, S, Kirkpatrick, J. Using simulation to optimize adaptive trial designs: applications in learning and confirmatory phase trials. Clin Invest. 2015;5(4):401–13.Google Scholar

Viele, K, McGlothlin, A, Broglio, K. Interpretation of clinical trials that stopped early. Jama. 2016;315(15):1646–7.Google Scholar

Park, JJ, Thorlund, K, Mills, EJ. Critical concepts in adaptive clinical trials. Clin Epidemiol. 2018;10:343–51.Google Scholar

Biswas, A, Bhattacharya, R. Response-adaptive designs for continuous treatment responses in phase III clinical trials: a review. Stat Methods Med Res. 2016;25(1):81–100.CrossRefGoogle ScholarPubMed

U.S. Department of Health and Human Services, Food and Drug Administration. Adaptive Designs for Clinical Trials of Drugs and Biologics Guidance for Industry. U.S. Department of Health and Human Services; 2019.Google Scholar

Thorlund, K, Haggstrom, J, Park, JJ, Mills, EJ. Key design considerations for adaptive clinical trials: a primer for clinicians. BMJ. 2018;360:k698.Google Scholar

Park, JJH, Hsu, G, Siden, EG, Thorlund, K, Mills, EJ. An overview of precision oncology basket and umbrella trials for clinicians. CA Cancer J Clin. 2020;70(2):125–37.Google Scholar

Little, RJ, Lewis, RJ. Estimands, estimators, and estimates. JAMA. 2021;326(10):967–8.Google Scholar

Collignon, O, Schiel, A, Burman, CF, et al. Estimands and complex innovative designs. Clin Pharmacol Ther. 2022. Online ahead of print.CrossRefGoogle Scholar

Prentice, RL. Surrogate endpoints in clinical trials: definition and operational criteria. Stat Med. 1989;8(4):431–40.Google Scholar

Haslam, A, Hey, SP, Gill, J, Prasad, V. A systematic review of trial-level meta-analyses measuring the strength of association between surrogate end-points and overall survival in oncology. Eur J Cancer. 2019;106:196–211.Google Scholar

Prasad, V, Kim, C, Burotto, M, Vandross, A. The strength of association between surrogate end points and survival in oncology: a systematic review of trial-level meta-analyses. JAMA Intern Med. 2015;175(8):1389–98.Google Scholar

Beauchemin, C, Johnston, JB, Lapierre, ME, Aissa, F, Lachaine, J. Relationship between progression-free survival and overall survival in chronic lymphocytic leukemia: a literature-based analysis. Curr Oncol (Toronto, Ont). 2015;22(3):e148–56.Google Scholar

Cortazar, P, Zhang, JJ, Sridhara, R, Justice, RL, Pazdur, R. Relationship between OS and PFS in metastatic breast cancer (MBC): review of FDA submission data. J Clin Oncol. 2011;29(15_suppl):1035.CrossRefGoogle Scholar

Gyawali, B, Hey, SP, Kesselheim, AS. A Comparison of response patterns for progression-free survival and overall survival following treatment for cancer with PD-1 inhibitors: a meta-analysis of correlation and differences in effect sizes. JAMA Netw Open. 2018;1(2):e180416–e.CrossRefGoogle ScholarPubMed

Berry, DA. Bayesian clinical trials. Nat Rev Drug Discov. 2006;5(1):27–36.Google Scholar

Saville, BR, Connor, JT, Ayers, GD, Alvarez, J. The utility of Bayesian predictive probabilities for interim monitoring of clinical trials. Clin Trials. 2014;11(4):485–93.Google Scholar

Lachin, JM. A review of methods for futility stopping based on conditional power. Stat Med. 2005;24(18):2747–64.Google Scholar

Thorlund, K, Golchi, S, Haggstrom, J, Mills, E. Highly Efficient Clinical Trials Simulator (HECT): software application for planning and simulating platform adaptive trials. Gates Open Res. 2019;3:780.Google Scholar

Thorlund, K, Golchi, S, Mills, E. Bayesian adaptive clinical trials of combination treatments. Contemp Clin Trials Commun. 2017;8:227–33.CrossRefGoogle ScholarPubMed

Schiavone, F, Bathia, R, Letchemanan, K, et al. This is a platform alteration: a trial management perspective on the operational aspects of adaptive and platform and umbrella protocols. Trials. 2019;20(1):264.Google Scholar

Hague, D, Townsend, S, Masters, L, et al. Changing platforms without stopping the train: experiences of data management and data management systems when adapting platform protocols by adding and closing comparisons. Trials. 2019;20(1):294.Google Scholar

Dunnett, CW. A multiple comparison procedure for comparing several treatments with a control. JASA. 1955;50(272):1096–121.Google Scholar

Berry, SM, Petzold, EA, Dull, P, et al. A response adaptive randomization platform trial for efficient evaluation of Ebola virus treatments: a model for pandemic response. Clin Trials. 2016;13(1):22–30.Google Scholar

Sydes, MR, Parmar, MKB, Mason, MD, et al. Flexible trial design in practice – stopping arms for lack-of-benefit and adding research arms mid-trial in STAMPEDE: a multi-arm multi-stage randomized controlled trial. Trials. 2012;13:168.Google Scholar

Barker, AD, Sigman, CC, Kelloff, GJ, et al. I-SPY 2: an adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin Pharmacol Ther. 2009;86(1):97–100.Google Scholar

Committee for Proprietary Medicinal Products. ICH Topic E 10: Choice of Control Group in Clinical Trials., p. 30. European Medicines Agency (EMEA); 2001;Google Scholar

Thorlund, K, Dron, L, Park, JJH, Mills, EJ. Synthetic and external controls in clinical trials – a primer for researchers. Clin Epidemiol. 2020;12:457–67.Google Scholar

Lee, KM, Wason, J. Including non-concurrent control patients in the analysis of platform trials: is it worth it? BMC Med Res Methodol. 2020;20(1):165.Google Scholar

Berry, SM, Reese, CS, Larkey, PD. Bridging different eras in sports. JASA 1999;94(447):661–76.Google Scholar

Viele, K, Berry, S, Neuenschwander, B, et al. Use of historical control data for assessing treatment effects in clinical trials. Pharm Stat. 2014;13(1):41–54.Google Scholar

Dron, L, Golchi, S, Hsu, G, Thorlund, K. Minimizing control group allocation in randomized trials using dynamic borrowing of external control data – an application to second line therapy for non-small cell lung cancer. Contemp Clin Trials Commun. 2019;16:100446.Google Scholar

Yu, LM, Bafadhel, M, Dorward, J, et al. Inhaled budesonide for COVID-19 in people at high risk of complications in the community in the UK (PRINCIPLE): a randomised, controlled, open-label, adaptive platform trial. Lancet. 2021;398(10303):843–55.Google Scholar

Heckman-Stoddard, BM, Smith, JJ. Precision medicine clinical trials: defining new treatment strategies. Semin Oncol Nurs. 2014;30(2):109–16.Google Scholar

Berry, DA. The brave new world of clinical cancer research: adaptive biomarker-driven trials integrating clinical practice with clinical research. Mol Oncol. 2015;9(5):951–9.Google Scholar

Antoniou, M, Jorgensen, AL, Kolamunnage-Dona, R. Biomarker-guided adaptive trial designs in phase II and phase III: a methodological review. PloS ONE. 2016;11(2):e0149803.CrossRefGoogle Scholar

Antoniou, M, Kolamunnage-Dona, R, Jorgensen, AL. Biomarker-guided non-adaptive trial designs in phase II and phase III: a methodological review. J Pers Med. 2017;7(1).Google Scholar

Kumar-Sinha, C, Chinnaiyan, AM. Precision oncology in the age of integrative genomics. Nat Biotechnol. 2018;36(1):46–60.Google Scholar

Abrams, J, Conley, B, Mooney, M, et al. National Cancer Institute’s Precision Medicine Initiatives for the new National Clinical Trials Network. Am Soc Clin Oncol Educ Book. 2014:71–6.Google Scholar

Collins, FS, Varmus, H. A new initiative on precision medicine. N Engl J Med. 2015;372(9):793–5.Google Scholar

Ashley, EA. The precision medicine initiative: a new national effort. JAMA. 2015;313(21):2119–20.Google Scholar

Ashley, EA. Towards precision medicine. Nat Rev Genet. 2016;17(9):507–22.Google Scholar

Woodcock, J, LaVange, LM. Master protocols to study multiple therapies, multiple diseases, or both. N Eng J Med 2017;377(1):62–70.Google Scholar

U.S. Department of Health and Human Services, Food and Drug Administration. Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics Guidance for Industry (Draft Guidance). U.S. Department of Health and Human Services; 2018. www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM621817.pdf.Google Scholar

Park, JJH, Siden, E, Zoratti, MJ, et al. Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols. Trials. 2019;20(1):572.Google Scholar

Siden, EG, Park, JJ, Zoratti, MJ, et al. Reporting of master protocols towards a standardized approach: a systematic review. Contemp Clin Trials Commun. 2019;15:100406.Google Scholar

Hirakawa, A, Asano, J, Sato, H, Teramukai, S. Master protocol trials in oncology: review and new trial designs. Contemp Clin Trials Commun. 2018;12:1–8.Google Scholar

Lam, VK, Papadimitrakopoulou, V. Master protocols in lung cancer: experience from Lung Master Protocol. Curr Opin Oncol. 2018;30(2):92–7.Google Scholar

Ledford, H. ‘Master protocol’ aims to revamp cancer trials: pilot project will bring drug companies together to test targeted lung-cancer therapies. Nature. 2013;498(7453):146–8.Google Scholar

Redman, MW, Allegra, CJ. The master protocol concept. Semin Oncol. 2015;42(5):724–30.Google Scholar

Renfro, LA, Sargent, DJ. Statistical controversies in clinical research: basket trials, umbrella trials, and other master protocols: a review and examples. Ann Oncol. 2017;28(1):34–43.Google Scholar

Parmar, MK, Sydes, MR, Cafferty, FH, et al. Testing many treatments within a single protocol over 10 years at MRC Clinical Trials Unit at UCL: multi-arm, multi-stage platform, umbrella and basket protocols. Clin Trials. 2017;14(5):451–61.CrossRefGoogle ScholarPubMed

De Benedetti, F, Gattorno, M, Anton, J, et al. Canakinumab for the treatment of autoinflammatory recurrent fever syndromes. N Engl J Med. 2018;378(20):1908–19.Google Scholar

Muhlbacher, J, Jilma, B, Wahrmann, M, et al. Blockade of HLA antibody-triggered classical complement activation in sera from subjects dosed with the anti-C1s monoclonal antibody TNT009 – results from a randomized first-in-human phase 1 trial. Transplantation. 2017;101(10):2410–18.Google Scholar

The ASCO Post. 2018 ASCO: IMPACT Trial matches treatment to genetic changes in the tumor to improve survival across multiple cancer types. 2018. www.ascopost.com/News/58897Google Scholar

Le Tourneau, C, Delord, JP, Goncalves, A, et al. Molecularly targeted therapy based on tumour molecular profiling versus conventional therapy for advanced cancer (SHIVA): a multicentre, open-label, proof-of-concept, randomised, controlled phase 2 trial. Lancet Oncol. 2015;16(13):1324–34.Google Scholar

Pon, JR, Marra, MA. Driver and passenger mutations in cancer. Annu Rev Pathol. 2015;10:25–50.Google Scholar

Brown, AL, Li, M, Goncearenco, A, Panchenko, AR. Finding driver mutations in cancer: elucidating the role of background mutational processes. PLoS Comput Biol. 2019;15(4):e1006981.Google Scholar

Park, JJ, Harari, O, Dron, L, Mills, EJ, Thorlund, K. Effects of biomarker diagnostic accuracy on biomarker-guided phase 2 trials. Contemp Clin Trials Commun. 2019;15:100396.Google Scholar

Bubendorf, L, Lantuejoul, S, de Langen, AJ, Thunnissen, E. Nonsmall cell lung carcinoma: diagnostic difficulties in small biopsies and cytological specimens: number 2 in the series ‘Pathology for the Clinician’ edited by Peter Dorfmuller and Alberto Cavazza. Eur Respir Rev. 2017;26(144).Google Scholar

Iding, JS, Krimsky, W, Browning, R. Tissue requirements in lung cancer diagnosis for tumor heterogeneity, mutational analysis and targeted therapies: initial experience with intra-operative Frozen Section Evaluation (FROSE) in bronchoscopic biopsies. J Thorac Dis. 2016;8(Suppl 6):S488–S93.Google Scholar

Arneth, B. Update on the types and usage of liquid biopsies in the clinical setting: a systematic review. BMC Cancer. 2018 Dec;18(1):1–2.Google Scholar

Heitzer, E, Ulz, P, Geigl, JB. Circulating tumor DNA as a liquid biopsy for cancer. Clin Chem. 2015;61(1):112–23.Google Scholar

Huang, GD, Bull, J, Johnston McKee, K, et al. Clinical trials recruitment planning: a proposed framework from the Clinical Trials Transformation Initiative. Contemp Clin Trials. 2018;66:74–9.Google Scholar

Brown, SR, Gregory, WM, Twelves, CJ, et al. Designing phase II trials in cancer: a systematic review and guidance. Br J Cancer. 2011;105(2):194–9.Google Scholar

Simon, R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials. 1989;10(1):1–10.Google Scholar

Clark, GM, Zborowski, DM, Culbertson, JL, et al. Clinical utility of epidermal growth factor receptor expression for selecting patients with advanced non-small cell lung cancer for treatment with erlotinib. J Thorac Oncol. 2006;1(8):837–46.Google Scholar

Clark, GM. Prognostic factors versus predictive factors: examples from a clinical trial of erlotinib. Mol Oncol. 2008;1(4):406–12.Google Scholar

Friedman, LM, Furberg, C, DeMets, DL, Reboussin, D, Granger, CB. Fundamentals of Clinical Trials: Springer; 2015.Google Scholar

Cartwright, N. What are randomised controlled trials good for? Phil Stud. 2010;147(1):59.Google Scholar

Roberts, C, Torgerson, DJ. Understanding controlled trials: baseline imbalance in randomised controlled trials. BMJ. 1999;319(7203):185.Google Scholar