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The Era of Alternative Designs to Connect Randomized Clinical Trials

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The Era of Alternative Designs to Connect Randomized Clinical Trials CORRESPONDENCE Jeroen W. G. Derksen 1,2*, Anne M. May2 and Miriam Koopman1 1Department of Medical Oncology, University Medical The era of alternative designs to Center Utrecht, Utrecht University, Utrecht, Netherlands. connect randomized clinical trials 2Department of Epidemiology, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht University, and real-world data Utrecht, Netherlands. *e-mail: [email protected] Jeroen W. G. Derksen , Anne M. May and Miriam Koopman https://doi.org/10.1038/s41571-019-0250-0 1. Booth, C. M., Karim, S. & Mackillop, W. J. Real- 4,5 world data: towards achieving the achievable in In their Review published in the May 2019 is another innovative trial design of interest . cancer care. Nat. Rev. Clin. Oncol. 16, 312–325 issue of this journal (Real-world data: towards In oncology RCTs, this design enables the (2019). 2. James, S., Rao, S. V. & Granger, C. B. Registry- achieving the achievable in cancer care. incorporation of methodo logical advantages, based randomized clinical trials — a new clinical Nat. Rev. Clin. Oncol. 16, 312–325 (2019))1, such as high recruitment rates, avoiding dis- trial paradigm. Nat. Rev. Cardiol. 12, 312–316 (2015). Booth et al. address challenges and future appointment bias, impro ved generalizability of 3. Li, G. W. et al. Registry-based randomized controlled pers pectives relating to the use of real-world trial results and improved ethical acceptability trials- what are the advantages, challenges, and areas for future research? J. Clin. Epidemiol. 80, 16–24 data (RWD) in oncology. They describe the when a two-stage infor med consent procedure (2016). 6 quality, and current and future applications is applied . The implementation of TwiCs in 4. Relton, C. et al. Rethinking pragmatic randomised controlled trials: introducing the “cohort multiple of RWD, and the pitfalls of studies of nationwide cohorts of patients with cancer randomised controlled trial” design. BMJ 340, c1066 comp ara tive effectiveness using RWD. We brings researchers one step closer to achiev- (2010). acknow ledge the authors’ efforts to provide ing the achievable by using prospective RCT 5. van der Velden, J. M. et al. The cohort multiple randomized controlled trial design: a valid and this comp rehensive overview and appreciate designs together with real-world follow-up data efficient alternative to pragmatic trials? the future perspectives they present. In light from cancer registries. Recently, several ongo- Int. J. Epidemiol. 46, 96–102 (2017). 6. Young-Afat, D. A. et al. Brief report: staged- 7–10 of the authors’ appeal for increasing focus to be ing or completed TwiCs have been described . informed consent in the cohort multiple randomized placed in the implementation of randomized Owing to the brevity of this correspondence controlled trial design. Epidemiology 27, 389–392 (2016). clinical trials (RCTs) in real-world settings, piece, we highlight only the Prospective Dutch 7. Dickerson, J. et al. Born in Bradford’s Better Start: herein we would like to address overlooked Colorectal Cancer (PLCRC) cohort, which an experimental birth cohort study to evaluate the impact of early life interventions. BMC Public Health. opportunities for using alternative trial designs serves as an infrastructure for a broad body of 16, 711 (2016). to enhance the real-world nature of the data cancer registry-based research including (but 8. Gal, R. et al. The Trials within Cohorts (TwiCs) design faced methodological advantages and disadvantages within RCTs. Owing to the length limitations not limited to) aetiological, biomarker, basic in the exercise-oncology setting. J. Clin. Epidemiol. of this Correspondence, we cannot define each and (epi)genetic, interventional (TwiCs), and 113, 137–146 (2019). 9. Burbach, J. P. et al. RandomizEd controlled trial for modality but provide supporting references. health-care policy and cost-effectiveness stud- pre-operAtive dose-escaLation BOOST in locally Pragmatic trial designs, such as registry- ies11. Addition ally, tumour biospecimens and advanced rectal cancer (RECTAL BOOST study): study protocol for a randomized controlled trial. Trials based RCTs (R-RCTs), have become of incre- repeated population-based patient-reported 16, 58 (2015). asing interest globally as efficient and cost- outcomes (PROs) are collected within the 10. Kwakkenbos, L. et al. The Scleroderma Patient- centered Intervention Network (SPIN) Cohort: effective tools that combine the advan tages of PLCRC. PROs are enriched follow-up RWD protocol for a cohort multiple randomised a prospective RCT with the stren gths of large- available for TwiCs, as well as an important controlled trial (cmRCT) design to support trials of psychosocial and rehabilitation interventions scale clinical registries. R-RCTs are charac- element that enables the implementation of in a rare disease context. BMJ Open 3, e003563 terized by low cost, enhanced generalizability learning health-care systems12. (2013). 11. Burbach, J. P. M. et al. Prospective Dutch colorectal of findings, rapid consecutive enrolment and Future logistic developments (such as cancer cohort: an infrastructure for long-term the high potential for completeness in the obtaining the patient’s informed consent at observational, prognostic, predictive and (randomized) intervention research. Acta Oncol. 55, 1273–1280 follow-up management of participants, espe- hospital entry) will enable a closer integra- (2016). cially hard end points2,3. Although R-RCTs tion of medical services into clinical research. 12. LeBlanc, T. W. & Abernethy, A. P. Patient-reported outcomes in cancer care — hearing the patient voice retain methodological limitations (for example, Finally, innovations in clinical oncology at greater volume. Nat. Rev. Clin. Oncol. 14, a dependence on the quality of the registry) practice by standardizing nation-wide entry 763–772 (2017). and could face ethical challenges given the of clinical data in electronic health records Acknowledgements 3 differences in national ethical guidelines , (EHRs) and subsequent implementation of The work of J.W.G.D. is fully supported by the Province of such studies can be designed to efficiently electronic data-capture systems, which enable Utrecht, Netherlands. address research questions about comparative real-time data transfer from EHRs to national Competing interests effectiveness in real-world settings. cancer registries, will support further improve- M. K. is the principle investigator of the Prospective Dutch Colorectal Cancer (PLCRC) cohort. The PLCRC is sponsored The trials within cohorts (TwiCs) design, also ments in quality and efficiency of RWD and by the Dutch Colorectal Cancer Group (DCCG). J.W.G.D. and referred to as cohort multiple RCT (cmRCT), cohort-embedded RCTs. A.M.M. declare no competing interests. NATURE REVIEWS | CLINICAL ONCOLOGY VOLUME 16 | SEPTEMBER 2019 | 589.
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