Kidney disease trials for the 21st century: innovations in design and conduct.
Journal
Nature reviews. Nephrology
ISSN: 1759-507X
Titre abrégé: Nat Rev Nephrol
Pays: England
ID NLM: 101500081
Informations de publication
Date de publication:
03 2020
03 2020
Historique:
accepted:
18
09
2019
pubmed:
2
11
2019
medline:
21
4
2020
entrez:
2
11
2019
Statut:
ppublish
Résumé
Compared to other specialties, nephrology has reported relatively few clinical trials, and most of these are too small to detect moderate treatment effects. Consequently, interventions that are commonly used by nephrologists have not been adequately tested and some may be ineffective or harmful. More randomized trials are urgently needed to address important clinical questions in patients with kidney disease. The use of robust surrogate markers may accelerate early-phase drug development. However, scientific innovations in trial conduct developed by other specialties should also be adopted to improve trial quality and enable more, larger trials in kidney disease to be completed in the current era of burdensome regulation and escalating research costs. Examples of such innovations include utilizing routinely collected health-care data and disease-specific registries to identify and invite potential trial participants, and for long-term follow-up; use of prescreening to facilitate rapid recruitment of participants; use of pre-randomization run-in periods to improve participant adherence and assess responses to study interventions prior to randomization; and appropriate use of statistics to monitor studies and analyse their results. Nephrology is well positioned to harness such innovations due to its advanced use of electronic health-care records and the development of disease-specific registries. Adopting a population approach and efficient trial conduct along with challenging unscientific regulation may increase the number of definitive clinical trials in nephrology and improve the care of current and future patients.
Identifiants
pubmed: 31673162
doi: 10.1038/s41581-019-0212-x
pii: 10.1038/s41581-019-0212-x
pmc: PMC7030944
mid: EMS84811
doi:
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
173-185Subventions
Organisme : Medical Research Council
ID : MC_UU_00017/3
Pays : United Kingdom
Organisme : Medical Research Council
ID : MR/R007764/1
Pays : United Kingdom
Références
Collins, R. Back to the future: the urgent need to re-introduce streamlined trials. Eur. Heart J. Suppl. 20, C14–C17 (2018).
doi: 10.1093/eurheartj/suy001
European Society of Cardiology. Clinical Practice Guidelines https://www.escardio.org/Guidelines/Clinical-Practice-Guidelines (2019).
Nabel, E. G. & Braunwald, E. A tale of coronary artery disease and myocardial infarction. N. Engl. J. Med. 366, 54–63 (2012).
pubmed: 22216842
doi: 10.1056/NEJMra1112570
Herrington, W., Lacey, B., Sherliker, P., Armitage, J. & Lewington, S. Epidemiology of atherosclerosis and the potential to reduce the global burden of atherothrombotic disease. Circ. Res. 118, 535–546 (2016).
pubmed: 26892956
doi: 10.1161/CIRCRESAHA.115.307611
Wanner, C. et al. Empagliflozin and progression of kidney disease in type 2 diabetes. N. Engl. J. Med. 375, 323–334 (2016).
pubmed: 27299675
doi: 10.1056/NEJMoa1515920
Perkovic, V. et al. Canagliflozin and renal outcomes in type 2 diabetes: results from the CANVAS program randomised clinical trials. Lancet Diabetes Endocrinol. 6, 691–704 (2018).
pubmed: 29937267
doi: 10.1016/S2213-8587(18)30141-4
Zelniker, T. A. et al. Comparison of the effects of glucagon-like peptide receptor agonists and sodium-glucose co-transporter 2 inhibitors for prevention of major adverse cardiovascular and renal outcomes in type 2 diabetes mellitus. Circulation 139, 2022–2031 (2019).
pubmed: 30786725
doi: 10.1161/CIRCULATIONAHA.118.038868
Strippoli, G. F., Craig, J. C. & Schena, F. P. The number, quality, and coverage of randomized controlled trials in nephrology. J. Am. Soc. Nephrol. 15, 411–419 (2004).
pubmed: 14747388
doi: 10.1097/01.ASN.0000100125.21491.46
Inrig, J. K. et al. The landscape of clinical trials in nephrology: a systematic review of Clinicaltrials.gov. Am. J. Kidney Dis. 63, 771–780 (2014).
pubmed: 24315119
doi: 10.1053/j.ajkd.2013.10.043
Tonelli, M. et al. Comparison of the complexity of patients seen by different medical subspecialists in a universal health care system. JAMA Netw. Open 1, e184852 (2018).
pubmed: 30646392
pmcid: 6324421
doi: 10.1001/jamanetworkopen.2018.4852
Kerr, M., Bray, B., Medcalf, J., O'Donoghue, D. J. & Matthews, B. Estimating the financial cost of chronic kidney disease to the NHS in England. Nephrol. Dial Transpl. 27, iii73–iii80 (2012).
doi: 10.1093/ndt/gfs269
Deeks, J. J. et al. Evaluating non-randomised intervention studies. Health Technol. Assess. 7, 1–179 (2003).
doi: 10.3310/hta7270
Gerstein, H. C., McMurray, J. & Holman, R. R. Real-world studies no substitute for RCTs in establishing efficacy. Lancet 393, 210–211 (2019).
pubmed: 30663582
doi: 10.1016/S0140-6736(18)32840-X
Grady, D. et al. Hormone therapy to prevent disease and prolong life in postmenopausal women. Ann. Intern. Med. 117, 1016–1037 (1992).
pubmed: 1443971
doi: 10.7326/0003-4819-117-12-1016
Rossouw, J. E. et al. Risks and benefits of estrogen plus progestin in healthy postmenopausal women: principal results from the women's health initiative randomized controlled trial. JAMA 288, 321–333 (2002).
pubmed: 12117397
doi: 10.1001/jama.288.3.321
Collins, R. et al. Interpretation of the evidence for the efficacy and safety of statin therapy. Lancet 388, 2532–2561 (2016).
pubmed: 27616593
doi: 10.1016/S0140-6736(16)31357-5
Bennett, D. A. & Holmes, M. V. Mendelian randomisation in cardiovascular research: an introduction for clinicians. Heart 103, 1400–1407 (2017).
pubmed: 28596306
doi: 10.1136/heartjnl-2016-310605
Sun, L. et al. Causal associations of blood lipids with risk of ischemic stroke and intracerebral hemorrhage in Chinese adults. Nat. Med. 25, 569–574 (2019).
pubmed: 30858617
pmcid: 6795549
doi: 10.1038/s41591-019-0366-x
Holmes, M. V. Human genetics and drug development. N. Engl. J. Med. 380, 1076–1079 (2019).
pubmed: 30865805
doi: 10.1056/NEJMe1901565
Collins, R. & MacMahon, S. Reliable assessment of the effects of treatment on mortality and major morbidity. I: Clinical trials. Lancet 357, 373–380 (2001).
pubmed: 11211013
doi: 10.1016/S0140-6736(00)03651-5
Pitt, B. et al. The effect of spironolactone on morbidity and mortality in patients with severe heart failure. Randomized Aldactone Evaluation Study Investigators. N. Engl. J. Med. 341, 709–717 (1999).
pubmed: 10471456
doi: 10.1056/NEJM199909023411001
Perkovic, V. et al. Canagliflozin and renal outcomes in type 2 diabetes and nephropathy. N. Engl. J. Med. 380, 2295–2306 (2019).
pubmed: 30990260
doi: 10.1056/NEJMoa1811744
Ettehad, D. et al. Blood pressure lowering for prevention of cardiovascular disease and death: a systematic review and meta-analysis. Lancet 387, 957–967 (2016).
pubmed: 26724178
doi: 10.1016/S0140-6736(15)01225-8
Xie, X. et al. Effects of intensive blood pressure lowering on cardiovascular and renal outcomes: updated systematic review and meta-analysis. Lancet 387, 435–443 (2016).
pubmed: 26559744
doi: 10.1016/S0140-6736(15)00805-3
Herrington, W. et al. How are the effects of reducing LDL cholesterol with a statin-based regime influenced by renal function? Meta-analysis of individual data from 28 randomised trials. Lancet Diabetes Endocrinol. 4, 829–839 (2016).
pubmed: 27477773
doi: 10.1016/S2213-8587(16)30266-2
Jafar, T. H. et al. Angiotensin-converting enzyme inhibitors and progression of nondiabetic renal disease. A meta-analysis of patient-level data. Ann. Intern. Med. 135, 73–87 (2001).
pubmed: 11453706
doi: 10.7326/0003-4819-135-2-200107170-00007
Webster, A. C., Playford, E. G., Higgins, G., Chapman, J. R. & Craig, J. C. Interleukin 2 receptor antagonists for renal transplant recipients: a meta-analysis of randomized trials. Transplantation 77, 166–176 (2004).
pubmed: 14742976
doi: 10.1097/01.TP.0000109643.32659.C4
Egger, M. & Smith, G. D. Misleading meta-analysis. BMJ 310, 752–754 (1995).
pubmed: 7711568
pmcid: 2549158
doi: 10.1136/bmj.310.6982.752
Chertow, G. M. et al. Effect of cinacalcet on cardiovascular disease in patients undergoing dialysis. N. Engl. J. Med. 367, 2482–2494 (2012).
pubmed: 23121374
doi: 10.1056/NEJMoa1205624
Parfrey, P. S. et al. Lessons learned from EVOLVE for planning of future randomized trials in patients on dialysis. Clin J. Am. Soc. Nephrol. 11, 539–546 (2016).
pubmed: 26614406
doi: 10.2215/CJN.06370615
Haynes, R., Landray, M. J., Herrington, W. G. & Baigent, C. in Oxford Textbook of Clinical Nephrology 4th edn (eds Turner, N. et al.) 161–170 (Oxford Uni. Press, 2015).
Baigent, C. et al. Challenges in conducting clinical trials in nephrology: conclusions from a kidney disease-improving global outcomes (KDIGO) controversies conference. Kidney Int. 92, 297–305 (2017).
pubmed: 28709600
pmcid: 6326036
doi: 10.1016/j.kint.2017.04.019
Pfeffer, M. A. et al. A trial of darbepoetin alfa in type 2 diabetes and chronic kidney disease. N. Engl. J. Med. 361, 2019–2032 (2009).
pubmed: 19880844
doi: 10.1056/NEJMoa0907845
Phrommintikul, A., Haas, S. J., Elsik, M. & Krum, H. Mortality and target haemoglobin concentrations in anaemic patients with chronic kidney disease treated with erythropoietin: a meta-analysis. Lancet 369, 381–388 (2007).
pubmed: 17276778
doi: 10.1016/S0140-6736(07)60194-9
European Renal Association − European Dialysis and Transplant Association. ERA−EDTA registry annual report 2016 (ERA, 2016).
UK Renal Registry. The National Registry of Rare Kidney Disease (RaDaR) https://www.renalreg.org/projects/the-national-registry-of-rare-kidney-disease-radar/ (2019).
Moxey-Mims, M. M. et al. Glomerular diseases: registries and clinical trials. Clin J. Am. Soc. Nephrol. 11, 2234–2243 (2016).
pubmed: 27672219
pmcid: 5142054
doi: 10.2215/CJN.00540116
Miner, J. H. et al. The 2014 International workshop on Alport syndrome. Kidney Int. 86, 679–684 (2014).
pubmed: 24988067
pmcid: 4182137
doi: 10.1038/ki.2014.229
Bowman, L. et al. ASCEND: a study of cardiovascular events in diabetes: characteristics of a randomized trial of aspirin and of omega-3 fatty acid supplementation in 15,480 people with diabetes. Am. Heart J. 198, 135–144 (2018).
pubmed: 29653635
pmcid: 5971211
doi: 10.1016/j.ahj.2017.12.006
Group, A. S. C. et al. Effects of aspirin for primary prevention in persons with diabetes mellitus. N. Engl. J. Med. 379, 1529–1539 (2018).
doi: 10.1056/NEJMoa1804988
Haynes, R. et al. Effects of sacubitril/valsartan versus irbesartan in patients with chronic kidney disease. Circulation 138, 1505–1514 (2018).
pubmed: 30002098
doi: 10.1161/CIRCULATIONAHA.118.034818
Sharp Collaborative Group. Study of Heart and Renal Protection (SHARP): randomized trial to assess the effects of lowering low-density lipoprotein cholesterol among 9,438 patients with chronic kidney disease. Am. Heart J. 160, 785–794.e710 (2010).
pubmed: 21095263
doi: 10.1016/j.ahj.2010.08.012
Lang, J. M., Buring, J. E., Rosner, B., Cook, N. & Hennekens, C. H. Estimating the effect of the run-in on the power of the Physicians' Health Study. Stat. Med. 10, 1585–1593 (1991).
pubmed: 1947514
doi: 10.1002/sim.4780101010
Landray, M. J. et al. Effects of extended-release niacin with laropiprant in high-risk patients. N. Engl. J. Med. 371, 203–212 (2014).
pubmed: 25014686
doi: 10.1056/NEJMoa1300955
Heerspink, H. J. L. et al. Atrasentan and renal events in patients with type 2 diabetes and chronic kidney disease (SONAR): a double-blind, randomised, placebo-controlled trial. Lancet 393, 1937–1947 (2019).
pubmed: 30995972
doi: 10.1016/S0140-6736(19)30772-X
Symplicity, H. T. N. I. et al. Renal sympathetic denervation in patients with treatment-resistant hypertension (The Symplicity HTN-2 Trial): a randomised controlled trial. Lancet 376, 1903–1909 (2010).
doi: 10.1016/S0140-6736(10)62039-9
Bhatt, D. L. et al. A controlled trial of renal denervation for resistant hypertension. N. Engl. J. Med. 370, 1393–1401 (2014).
pubmed: 24678939
doi: 10.1056/NEJMoa1402670
Howard, J. P. et al. Quantifying the 3 biases that lead to unintentional overestimation of the blood pressure-lowering effect of renal denervation. Circ. Cardiovasc. Qual. Outcomes 9, 14–22 (2016).
pubmed: 26758193
doi: 10.1161/CIRCOUTCOMES.115.002533
Meeker-O'Connell, A. et al. Enhancing clinical evidence by proactively building quality into clinical trials. Clin. Trials 13, 439–444 (2016).
pubmed: 27098014
pmcid: 4952025
doi: 10.1177/1740774516643491
Pogue, J. M., Devereaux, P. J., Thorlund, K. & Yusuf, S. Central statistical monitoring: detecting fraud in clinical trials. Clin. Trials 10, 225–235 (2013).
pubmed: 23283577
doi: 10.1177/1740774512469312
3C Study Collaborative Group. Campath, calcineurin inhibitor reduction, and chronic allograft nephropathy (the 3C Study) — results of a randomized controlled clinical trial. Am. J. Transpl. 18, 1424–1434 (2018).
doi: 10.1111/ajt.14619
Ndounga Diakou, L. A. et al. Comparison of central adjudication of outcomes and onsite outcome assessment on treatment effect estimates. Cochrane Database Syst. Rev. 3, MR000043 (2016).
pubmed: 26961577
Hlatky, M. A. et al. Use of Medicare data to identify coronary heart disease outcomes in the Women's Health Initiative. Circ. Cardiovasc. Qual. Outcomes 7, 157–162 (2014).
pubmed: 24399330
pmcid: 4548886
doi: 10.1161/CIRCOUTCOMES.113.000373
Frobert, O. et al. Thrombus aspiration during ST-segment elevation myocardial infarction. N. Engl. J. Med. 369, 1587–1597 (2013).
pubmed: 23991656
doi: 10.1056/NEJMoa1308789
Prentice, R. L. Surrogate endpoints in clinical trials: definition and operational criteria. Stat. Med. 8, 431–440 (1989).
pubmed: 2727467
doi: 10.1002/sim.4780080407
Levey, A. S. et al. GFR decline as an end point for clinical trials in CKD: a scientific workshop sponsored by the National Kidney Foundation and the US food and drug administration. Am. J. Kidney Dis. 64, 821–835 (2014).
pubmed: 25441437
doi: 10.1053/j.ajkd.2014.07.030
Fried, L. F. & Lewis, J. Albuminuria is not an appropriate therapeutic target in patients with CKD: the con view. Clin. J. Am. Soc. Nephrol. 10, 1089–1093 (2015).
pubmed: 25887070
pmcid: 4455218
doi: 10.2215/CJN.10681014
Lambers Heerspink, H. J. & Gansevoort, R. T. Albuminuria is an appropriate therapeutic target in patients with CKD: the pro view. Clin. J. Am. Soc. Nephrol. 10, 1079–1088 (2015).
pubmed: 25887073
pmcid: 4455219
doi: 10.2215/CJN.11511114
Heerspink, H. J. L. et al. Change in albuminuria as a surrogate endpoint for progression of kidney disease: a meta-analysis of treatment effects in randomised clinical trials. Lancet Diabetes Endocrinol. 7, 128–139 (2019).
pubmed: 30635226
doi: 10.1016/S2213-8587(18)30314-0
Smith, M. et al. Change in albuminuria and risk of renal and cardiovascular outcomes: natural variation should be taken into account. Kidney Int. Rep. 3, 939–949 (2018).
pubmed: 29988998
pmcid: 6035156
doi: 10.1016/j.ekir.2018.04.004
Thompson, A. et al. Proteinuria reduction as a surrogate end point in trials of IgA nephropathy. Clin. J. Am. Soc. Nephrol. 14, 469–481 (2019).
pubmed: 30635299
pmcid: 6419287
doi: 10.2215/CJN.08600718
Schluchter, M. D., Greene, T. & Beck, G. J. Analysis of change in the presence of informative censoring: application to a longitudinal clinical trial of progressive renal disease. Stat. Med. 20, 989–1007 (2001).
pubmed: 11276031
doi: 10.1002/sim.720
Vonesh, E. F., Greene, T. & Schluchter, M. D. Shared parameter models for the joint analysis of longitudinal data and event times. Stat. Med. 25, 143–163 (2006).
pubmed: 16025541
doi: 10.1002/sim.2249
Kular, D. & Macdougall, I. C. HIF stabilizers in the management of renal anemia: from bench to bedside to pediatrics. Pediatr. Nephrol. 34, 365–378 (2019).
pubmed: 29569190
doi: 10.1007/s00467-017-3849-3
McMurray, J. J. et al. Angiotensin-neprilysin inhibition versus enalapril in heart failure. N. Engl. J. Med. 371, 993–1004 (2014).
pubmed: 25176015
doi: 10.1056/NEJMoa1409077
Deo, A., Schmid, C. H., Earley, A., Lau, J. & Uhlig, K. Loss to analysis in randomized controlled trials in CKD. Am. J. Kidney Dis. 58, 349–355 (2011).
pubmed: 21778004
doi: 10.1053/j.ajkd.2011.04.023
Coronary Drug Project Research Group. Influence of adherence to treatment and response of cholesterol on mortality in the coronary drug project. N. Engl. J. Med. 303, 1038–1041 (1980).
doi: 10.1056/NEJM198010303031804
Eijkelkamp, W. B. et al. Albuminuria is a target for renoprotective therapy independent from blood pressure in patients with type 2 diabetic nephropathy: post hoc analysis from the Reduction of Endpoints in NIDDM with the Angiotensin II Antagonist Losartan (RENAAL) trial. J. Am. Soc. Nephrol. 18, 1540–1546 (2007).
pubmed: 17409317
doi: 10.1681/ASN.2006050445
Heart Protection Study Collaborative Group. MRC/BHF Heart Protection Study of cholesterol lowering with simvastatin in 20,536 high-risk individuals: a randomised placebo-controlled trial. Lancet 360, 7–22 (2002).
doi: 10.1016/S0140-6736(02)09327-3
Schmidt, M. et al. Serum creatinine elevation after renin-angiotensin system blockade and long term cardiorenal risks: cohort study. BMJ 356, j791 (2017).
pubmed: 28279964
pmcid: 5421447
doi: 10.1136/bmj.j791
Ohkuma, T. et al. Acute increases in serum creatinine after starting angiotensin-converting enzyme inhibitor-based therapy and effects of its continuation on major clinical outcomes in type 2 diabetes mellitus. Hypertension 73, 84–91 (2019).
pubmed: 30571562
doi: 10.1161/HYPERTENSIONAHA.118.12060
Vickers, A. J. & Altman, D. G. Statistics notes: analysing controlled trials with baseline and follow up measurements. BMJ 323, 1123–1124 (2001).
pubmed: 11701584
pmcid: 1121605
doi: 10.1136/bmj.323.7321.1123
Dember, L. M. et al. Pragmatic trials in maintenance dialysis: perspectives from the kidney health initiative. J. Am. Soc. Nephrol. 27, 2955–2963 (2016).
pubmed: 27401689
pmcid: 5042681
doi: 10.1681/ASN.2016030340
Hemming, K., Eldridge, S., Forbes, G., Weijer, C. & Taljaard, M. How to design efficient cluster randomised trials. BMJ 358, j3064 (2017).
pubmed: 28710062
pmcid: 5508848
doi: 10.1136/bmj.j3064
Torgerson, D. J. Contamination in trials: is cluster randomisation the answer? BMJ 322, 355–357 (2001).
pubmed: 11159665
pmcid: 1119583
doi: 10.1136/bmj.322.7282.355
Eldridge, S., Kerry, S. & Torgerson, D. J. Bias in identifying and recruiting participants in cluster randomised trials: what can be done? BMJ 339, b4006 (2009).
pubmed: 19819928
doi: 10.1136/bmj.b4006
Dember, L. M. et al. The time trial: a fully embedded, cluster-randomized, pragmatic trial of hemodialysis session duration. J. Am. Soc. Nephrol. 30, 890–903 (2019).
pubmed: 31000566
pmcid: 6493975
doi: 10.1681/ASN.2018090945
Hemming, K., Haines, T. P., Chilton, P. J., Girling, A. J. & Lilford, R. J. The stepped wedge cluster randomised trial: rationale, design, analysis, and reporting. BMJ 350, h391 (2015).
pubmed: 25662947
doi: 10.1136/bmj.h391
Selby, N. M. et al. An organizational-level program of intervention for aki: a pragmatic stepped wedge cluster randomized trial. J. Am. Soc. Nephrol. 30, 505–515 (2019).
pubmed: 31058607
pmcid: 6405151
doi: 10.1681/ASN.2018090886
Feldman, A. M. et al. Precision medicine for heart failure: lessons from oncology. Circ. Heart Fail. 10, e004202 (2017).
pubmed: 28611130
pmcid: 5643024
doi: 10.1161/CIRCHEARTFAILURE.117.004202
Baigent, C. et al. Efficacy and safety of more intensive lowering of LDL cholesterol: a meta-analysis of data from 170,000 participants in 26 randomised trials. Lancet 376, 1670–1681 (2010).
pubmed: 21067804
doi: 10.1016/S0140-6736(10)61350-5
Ruggenenti, P., Bettinaglio, P., Pinares, F. & Remuzzi, G. Angiotensin converting enzyme insertion/deletion polymorphism and renoprotection in diabetic and nondiabetic nephropathies. Clin. J. Am. Soc. Nephrol. 3, 1511–1525 (2008).
pubmed: 18550651
pmcid: 4571148
doi: 10.2215/CJN.04140907
de Zeeuw, D., Heerspink, H. J. L., Jardine, M. & Perkovic, V. Renal trials in diabetes need a platform: time for a global approach? Lancet Diabetes Endocrinol. 6, 356–358 (2018).
pubmed: 28797748
doi: 10.1016/S2213-8587(17)30263-2
Heerspink, H. J. L. et al. Rationale and protocol of the Study of diabetic Nephropathy with Atrasentan (SONAR) trial: a clinical trial design novel to diabetic nephropathy. Diabetes Obes. Metab. 20, 1369–1376 (2018).
pubmed: 29405626
pmcid: 5969254
doi: 10.1111/dom.13245
Park, J. J., Thorlund, K. & Mills, E. J. Critical concepts in adaptive clinical trials. Clin. Epidemiol. 10, 343–351 (2018).
pubmed: 29606891
pmcid: 5868584
doi: 10.2147/CLEP.S156708
Thorlund, K., Haggstrom, J., Park, J. J. & Mills, E. J. Key design considerations for adaptive clinical trials: a primer for clinicians. BMJ 360, k698 (2018).
pubmed: 29519932
pmcid: 5842365
doi: 10.1136/bmj.k698
Mann, J. F. et al. Avosentan for overt diabetic nephropathy. J. Am. Soc. Nephrol. 21, 527–535 (2010).
pubmed: 20167702
pmcid: 2831858
doi: 10.1681/ASN.2009060593
Hirakawa, A., Asano, J., Sato, H. & Teramukai, S. Master protocol trials in oncology: review and new trial designs. Contemp. Clin. Trials Commun. 12, 1–8 (2018).
pubmed: 30182068
pmcid: 6120722
doi: 10.1016/j.conctc.2018.08.009
Renfro, L. A. & Sargent, D. J. Statistical controversies in clinical research: basket trials, umbrella trials, and other master protocols: a review and examples. Ann. Oncol. 28, 34–43 (2017).
pubmed: 28177494
doi: 10.1093/annonc/mdw413
Saville, B. R. & Berry, S. M. Efficiencies of platform clinical trials: a vision of the future. Clin. Trials 13, 358–366 (2016).
pubmed: 26908536
doi: 10.1177/1740774515626362
Heerspink, H. J. L. & Perkovic, V. Trial design innovations to accelerate therapeutic advances in chronic kidney disease: moving from single trials to an ongoing platform. Clin. J. Am. Soc. Nephrol. 13, 946–948 (2018).
pubmed: 29700075
pmcid: 5989668
doi: 10.2215/CJN.01290118
Butler, C. C. et al. A trial like ALIC(4)E: why design a platform, response-adaptive, open, randomised controlled trial of antivirals for influenza-like illness? ERJ Open Res. 4, 00046-2018 (2018).
pubmed: 29761108
pmcid: 5938489
doi: 10.1183/23120541.00046-2018
Landray, M. Improving public health by improving clinical trial guidelines and their application. Eur. Heart J. 38, 1632–1637 (2017).
pubmed: 28329235
pmcid: 5837481
doi: 10.1093/eurheartj/ehx086
Sprosen, T. Major funders establish initiative to develop new trial guidelines. MoreTrials https://moretrials.net/major-funders-establish-initiative-develop-new-trial-guidelines/ (2019).
Clinical Trials Transformation Initiative. Who We Are https://www.ctti-clinicaltrials.org/who-we-are (2019).
Linde, P. G. et al. Overcoming barriers in kidney health-forging a platform for innovation. J. Am. Soc. Nephrol. 27, 1902–1910 (2016).
pubmed: 27127187
pmcid: 4926987
doi: 10.1681/ASN.2015090976