An introduction of the origin and development of N-of-1 trials_Chinese Journal of Evidence-Based Medicine

Authors：

LI Jiang ¹ , ZHAI Jingbo ² ,  SHANG Hongcai ³ , CHEN Jing ² , SHI Jufang ¹ , YANG Kehu ⁴ , TIAN Jinhui ⁴ ,  DAI Min ¹

1. Program office for Cancer Screening in Urban China, National Cancer Center/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100021, P.R.China;
2. Traditional Chinese Medicine of Tianjin University, Tianjin, 300193, P.R.China;
3. Key Laboratory of Chinese Internal Medicine of Ministry of Education and Beijing, Dongzhimen Hospital, Beijing University of Chinese Medicine, Beijing, 100700, P.R.China;
4. Evidence-Based Medicine Center, School of Basic Medical Sciences, Lanzhou University, Lanzhou, 730000, P.R.China;

Corresponding author：

SHANG Hongcai, Email: shanghongcai@foxmail.com; DAI Min, Email: daimin2002@hotmail.com

Keywords：

N-of-1 trial; Origin; Development

DOI：

10.7507/1672-2531.201701073

Video：

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N-of-1 trial is globally making rapid development as a patient-oriented individualized method with the development and improvement of methodology in clinical trial. The paper described the origin and development of N-of-1trial, so as to help improve awareness among medical researchers and clinicians, and improve the clinical medical quality and level of clinical diagnosis and treatment.

Citation： LIJiang, ZHAIJing-bo, SHANGHong-cai, CHENJing, SHIJu-fang, YANGKe-hu, TIANJin-hui, DAIMin. An introduction of the origin and development of N-of-1 trials. Chinese Journal of Evidence-Based Medicine, 2017, 17(2): 235-238. doi: 10.7507/1672-2531.201701073 Copy

1.	Nicholas J. Schork. Personalized medicine: Time for one-person trials. Nature, 2015, 520(7549): 609-611.
2.	李江, 田金徽, 石菊芳, 等. 单病例随机对照试验的研究现状与进展. 兰州大学学报(医学版), 2016, 42(3): 52-56.
3.	Scheen AJ. Integrating evidence-based medicine and personalized medicine. Rev Med Suisse, 2013, 9(395): 1499-1500.
4.	Kravitz RL, Duan N, Niedzinski EJ,et al. Whatever happened to N-of-1 trials? Insiders' perspectives and a look to the future. Milbank Q, 2008, 86(4): 533-555.
5.	Parry CH. Experiments relative to the medical effects of Turkey Rhubarb, and of the English Rhubarbs, No. I and No. II made on patients of the Pauper Charity. Lett Papers Bath Soc, 1786, 11: 431-453.
6.	Martini P. Methodenlehre der therapeutischen Untersuchung. Springer: Berlin, 1932.
7.	Hogben L, Sim M. The self-controlled and self-recorded clinical trial for low-grade morbidity. Br J Prev Soc Med, 1953, 7(4): 163-179.
8.	Kratchwill TR (ed). Single Subject Research: Strategies for Evaluating Change. Acad Pr, Orlando, Fla, 1978: 316.
9.	Kazdin AE. Single-Case Research Designs: Methods for Clinical and Applied Settings. Oxford U Pr, New York, 1982: 368.
10.	Barlow DH, Hersen M. Single Case Experimental Designs: Strategies for Studying Behavior Change, 2nd ed, Pergamon, New York, 1984: 419.
11.	Guyatt G, Sackett D, Taylor DW,et al. Determining optimal therapy-randomized trials in individual patients. N Engl J Med, 1986, 314(14): 889-892.
12.	Guyatt GH, Keller JL, Jaeschke R,et al. The n-of-1 randomized controlled trial: clinical usefulness. Our three- year experience. Ann Int Med, 1990, 112(4): 293-299.
13.	Guyatt GH, Heyting A, Jaeschke R,et al. N of 1 randomized trials for investigating new drugs. Control Clin Trials, 1990, 11(2): 88-100.
14.	Larson EB.N-of-1 clinical trials．A technique for improving medical therapeutics．West J Med, 1990, 152(1): 52-56.
15.	Larson EB, Ellsworth AJ, Oas J. Randomized Clinical Trials in Single Patients during a 2-Year Period. JAMA, 1993, 270(22): 2708-2712.
16.	Johannessen T, Fosstvedt D, Petersen H. Statistical Aspects of Controlled Single Subject Trial. Fam Pract, 1990, 7(4): 325-328.
17.	Senn S. Applying results of randomised trials to patients. N of 1 trials are needed. BMJ, 1998, 317(7157): 537-538.
18.	Nikles CJ, Glasziou PP, Del MCB,et al. Preliminary Experiences with a Single-Patient Trials Service in General Practice. Med J Aust, 2000, 173(2): 100-103.
19.	Nikles CJ, Mitchell GK, Del MCB,et al. An N-of-1 Trial Service in Clinical Practice: Testing the Effectiveness of Stimulants for Attention-Deficit/Hyperactivity Disorder. Pediatrics, 2006, 117(6): 2040-2046.
20.	Punja S, Eslick I, Duan N,et al. An Ethical Framework for N-of-1 Trials: Clinical Care, Quality Improvement, or Human Subjects Research? In: Kravitz RL, Duan N, eds, and the DEcIDE Methods Center N-of-1 Guidance Panel (Duan N, Eslick I, Gabler NB, Kaplan HC, Kravitz RL, Larson EB, Pace WD, Schmid CH, Sim I, Vohra S). Design and Implementation of N-of-1 Trials: A User’s Guide. AHRQ Publication No. 13(14)- EHC122-EF. Rockville, MD: Agency for Healthcare Research and Quality; January 2014: Chapter 2, pp. 13-22.
21.	Kravitz RL, Duan N, Braslow J. Evidence-Based Medicine, Heterogeneity of Treatment Effects, and the Trouble with Averages. Milbank Q, 2004, 82(4): 661-687.
22.	Kravitz RL, Duan N, White RH. N-of-1 Trials of Expensive Biological Therapies: A Third Way? Arch Intern Med, 2008, 168(10): 1030-1033.
23.	Kravitz RL, Duan N, Vohra S,et al. Introduction to N-of-1 Trials: Indications and Barriers. In: Kravitz RL, Duan N, eds, and the DEcIDE Methods Center N-of-1 Guidance Panel. Design and Implementation of N-of-1 Trials: A User’s Guide. AHRQ Publication No. 13(14)- EHC122- EF. Rockville, MD: Agency for Healthcare Research and Quality; January 2014.
24.	Vohra S, Shamseer L, Sampson M,et al. CONSORT extension for reporting N-of-1 trials (CENT) 2015 Statement. BMJ, 2015, 350: h1738.
25.	Li J, Gao W, Punja S.et al. Reporting quality of N-of-1 trials published between 1985 and 2013: a systematic review. J Clin Epidemiol. 2016; 76: 57-64.
26.	Zhai J, Cao H, Ren M,et al. Reporting of core items in hierarchical Bayesian analysis for aggregating N-of-1 trials to estimate population treatment effects is suboptimal. J Clin Epidemiol, 2016, 76: 99-107.
27.	Guyatt GH. N of 1 randomized trials: a commentary. J Clin Epidemiol, 2016, 76: 4-5.

1. Nicholas J. Schork. Personalized medicine: Time for one-person trials. Nature, 2015, 520(7549): 609-611.
2. 李江, 田金徽, 石菊芳, 等. 单病例随机对照试验的研究现状与进展. 兰州大学学报(医学版), 2016, 42(3): 52-56.
3. Scheen AJ. Integrating evidence-based medicine and personalized medicine. Rev Med Suisse, 2013, 9(395): 1499-1500.
4. Kravitz RL, Duan N, Niedzinski EJ,et al. Whatever happened to N-of-1 trials? Insiders' perspectives and a look to the future. Milbank Q, 2008, 86(4): 533-555.
5. Parry CH. Experiments relative to the medical effects of Turkey Rhubarb, and of the English Rhubarbs, No. I and No. II made on patients of the Pauper Charity. Lett Papers Bath Soc, 1786, 11: 431-453.
6. Martini P. Methodenlehre der therapeutischen Untersuchung. Springer: Berlin, 1932.
7. Hogben L, Sim M. The self-controlled and self-recorded clinical trial for low-grade morbidity. Br J Prev Soc Med, 1953, 7(4): 163-179.
8. Kratchwill TR (ed). Single Subject Research: Strategies for Evaluating Change. Acad Pr, Orlando, Fla, 1978: 316.
9. Kazdin AE. Single-Case Research Designs: Methods for Clinical and Applied Settings. Oxford U Pr, New York, 1982: 368.
10. Barlow DH, Hersen M. Single Case Experimental Designs: Strategies for Studying Behavior Change, 2nd ed, Pergamon, New York, 1984: 419.
11. Guyatt G, Sackett D, Taylor DW,et al. Determining optimal therapy-randomized trials in individual patients. N Engl J Med, 1986, 314(14): 889-892.
12. Guyatt GH, Keller JL, Jaeschke R,et al. The n-of-1 randomized controlled trial: clinical usefulness. Our three- year experience. Ann Int Med, 1990, 112(4): 293-299.
13. Guyatt GH, Heyting A, Jaeschke R,et al. N of 1 randomized trials for investigating new drugs. Control Clin Trials, 1990, 11(2): 88-100.
14. Larson EB.N-of-1 clinical trials．A technique for improving medical therapeutics．West J Med, 1990, 152(1): 52-56.
15. Larson EB, Ellsworth AJ, Oas J. Randomized Clinical Trials in Single Patients during a 2-Year Period. JAMA, 1993, 270(22): 2708-2712.
16. Johannessen T, Fosstvedt D, Petersen H. Statistical Aspects of Controlled Single Subject Trial. Fam Pract, 1990, 7(4): 325-328.
17. Senn S. Applying results of randomised trials to patients. N of 1 trials are needed. BMJ, 1998, 317(7157): 537-538.
18. Nikles CJ, Glasziou PP, Del MCB,et al. Preliminary Experiences with a Single-Patient Trials Service in General Practice. Med J Aust, 2000, 173(2): 100-103.
19. Nikles CJ, Mitchell GK, Del MCB,et al. An N-of-1 Trial Service in Clinical Practice: Testing the Effectiveness of Stimulants for Attention-Deficit/Hyperactivity Disorder. Pediatrics, 2006, 117(6): 2040-2046.
20. Punja S, Eslick I, Duan N,et al. An Ethical Framework for N-of-1 Trials: Clinical Care, Quality Improvement, or Human Subjects Research? In: Kravitz RL, Duan N, eds, and the DEcIDE Methods Center N-of-1 Guidance Panel (Duan N, Eslick I, Gabler NB, Kaplan HC, Kravitz RL, Larson EB, Pace WD, Schmid CH, Sim I, Vohra S). Design and Implementation of N-of-1 Trials: A User’s Guide. AHRQ Publication No. 13(14)- EHC122-EF. Rockville, MD: Agency for Healthcare Research and Quality; January 2014: Chapter 2, pp. 13-22.
21. Kravitz RL, Duan N, Braslow J. Evidence-Based Medicine, Heterogeneity of Treatment Effects, and the Trouble with Averages. Milbank Q, 2004, 82(4): 661-687.
22. Kravitz RL, Duan N, White RH. N-of-1 Trials of Expensive Biological Therapies: A Third Way? Arch Intern Med, 2008, 168(10): 1030-1033.
23. Kravitz RL, Duan N, Vohra S,et al. Introduction to N-of-1 Trials: Indications and Barriers. In: Kravitz RL, Duan N, eds, and the DEcIDE Methods Center N-of-1 Guidance Panel. Design and Implementation of N-of-1 Trials: A User’s Guide. AHRQ Publication No. 13(14)- EHC122- EF. Rockville, MD: Agency for Healthcare Research and Quality; January 2014.
24. Vohra S, Shamseer L, Sampson M,et al. CONSORT extension for reporting N-of-1 trials (CENT) 2015 Statement. BMJ, 2015, 350: h1738.
25. Li J, Gao W, Punja S.et al. Reporting quality of N-of-1 trials published between 1985 and 2013: a systematic review. J Clin Epidemiol. 2016; 76: 57-64.
26. Zhai J, Cao H, Ren M,et al. Reporting of core items in hierarchical Bayesian analysis for aggregating N-of-1 trials to estimate population treatment effects is suboptimal. J Clin Epidemiol, 2016, 76: 99-107.
27. Guyatt GH. N of 1 randomized trials: a commentary. J Clin Epidemiol, 2016, 76: 4-5.

Chinese Journal of Evidence-Based Medicine

An introduction of the origin and development of N-of-1 trials

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