Preview

Rational Pharmacotherapy in Cardiology

Advanced search

Clinical Trials Evaluating Drug Therapy: Types, Reliability of Results, Place in Modern Evidence-Based Medicine

https://doi.org/10.20996/1819-6446-2021-04-07

Full Text:

Abstract

The article discusses the main methods of evidence in modern medicine. Special attention is paid to randomized controlled trials and observational studies. The advantages of randomized controlled trials over observational studies are considered. A comparison of the informative value of randomized controlled trials and observational studies in assessing the effect of therapeutic interventions is made. Attention is drawn to situations when conducting randomized controlled trials is not possible and when they become the main source of information. It is emphasized that in order to verify the results of randomized controlled trials in real clinical practice, it is necessary to conduct observational studies. The basic principles of conducting observational studies are considered.

About the Authors

S. Yu. Martsevich
National Medical Research Center for Therapy and Preventive Medicine
Russian Federation

Sergey Yu. Martsevich - eLibrary SPIN 7908-9554

Moscow



N. P. Kutishenko
National Medical Research Center for Therapy and Preventive Medicine
Russian Federation

Natalia P. Kutishenko - eLibrary SPIN 7893-9865

Moscow



Yu. V. Lukina
National Medical Research Center for Therapy and Preventive Medicine
Russian Federation

Yulia V. Lukina - eLibrary SPIN 8949-4964

Moscow



M. M. Lukyanov
National Medical Research Center for Therapy and Preventive Medicine
Russian Federation

Mikhail M. Lukyanov - eLibrary SPIN 6842-9870

Moscow



O. M. Drapkina
National Medical Research Center for Therapy and Preventive Medicine
Russian Federation

Oxana M. Drapkina - eLibrary SPIN 4456-1297

Moscow



References

1. Jaillon P. L'essai Clinique controle randomise [Controlled randomized clinical trials]. Bull Acad Natl Med. 2007;191:739-56; discussion 756-758.

2. Pocock S, Elbourne D. Randomised trials or observational studies? Lancet. 2000;342:1907-9.

3. Concato J, Shah N, Horwitz RI. Randomized, controlled trials, observational studies, and the hierarchy of research designs. N Engl J Med. 2000;342(25):1887-92. DOI:10.1056/NEJM200006223422507.

4. Lang GF. Diseases of the circulatory system. Moscow: Gosudarstvennoye izdatel'stvo meditsinskoy lit- eratury; 1957 (In Russ.)

5. Echt DS, Liebeson PR, Mitchell LB, et al. Mortality and morbidity in patients receiving encainide, fle- cainide, or placebo: The Cardiac Arrhythmia Supression Trial. N Engl J Med. 1991;324:781-8.

6. Roelins M. De testimonio: on the evidence for decisions about the use of therapeutic interventions. Clinical Medicine. 2008;8(6):579-88.

7. Streptomycin treatment of pulmonary tuberculosis: a Medical Research Council investigation. BMJ. 1948;2:769-82. DOI:10.1136/bmj.2.4582.769.

8. Glasziou P, Chalmers I, Rawlins M, et al. When are randomized trials unnecessary? Picking signal from noise. BMJ. 2007;334:349-51.

9. Storm B. The use of pharmacoepidemiology to study beneficial drug effects. In: Strom B.L., Kimmel S.E., Hennessy S., eds. Textbook of pharmacoepidemiology. Philadelphia: Wiley-Blackwell; 2006. P.321-331.

10. Portioli I. I limited degli studi clinici controllata: il caso della reumatologia [The limits of controlled clinical studies: the case of rheumatology]. Ann Ital Med Int. 2000;15(1):39-46.

11. Benson K, Hartz AJ. A comparison of observational studies and randomized, controlled trials. N Engl J Med. 2000;342(25):1878-86. DOI:10.1056/NEJM200006223422506.

12. Penston J. Statistics-based research - a pig in a poke? J EvalClin Pract. 2011;17(5):862-7. DOI:10.1111/j.1365-2753.2011.01717.x.

13. Packer M, McMurray JJ, Desai AS, et al. PARADIGM-HF Investigators and Coordinators. Angiotensin receptor neprilysin inhibition compared with enalapril on the risk of clinical progression in surviving patients with heart failure. Circulation. 2015;131(1):54-61. DOI:10.1161/CIRCULATION-AHA.114.013748.

14. Colditz GA. Overview of the epidemiology methods and applications: strengths and limitations of observational study designs. Crit Rev Food Sci Nutr. 2010;50Suppl 1(s1):10-12. DOI:10.1080/10408398.2010.526838.

15. Abel U, Koch A. The role of randomization in clinical studies: myths and beliefs. J Clin Epidemiol. 1999;52(6):487-97. DOI:10.1016/s0895-4356(99)00041-4.

16. Guerrera F Renaud S, Tabbo F, et al. How to design a randomized clinical trial: tips and tricks for conduct a successful study in thoracic disease domain. J Thorac Dis. 2017;9(8):2692-6. DOI:10.21037/jtd.2017.06.147.

17. Glynn RJ, Schneeweiss S, Sturmer T. Indications for propensity scores and review of their use in pharmacoepidemiology. Basic Clin Pharmacol Toxicol. 2006;98:253-9. DOI:10.1111/j.1742-7843.2006.pto_293.x.

18. Martsevich SY, Kutishenko NP, Lukina YV, et al. Observational studies and registers. Their quality and role in modern evidence-based medicine. Cardiovascular Therapy and Prevention. 2021;20(2):61- 6 (In Russ.) DOI:10.15829/1728-8800-2021-2786.

19. Singal AG, Higgins PDR, Waijee AK. A primer on effectiveness and efficacy trials. Clinical and Translation Gastroenterology. 2014;5:e45. DOI:10.1038/ctg.2013.13.

20. Normand SLT, Sykora K, Li P, et al. Reader's guide to critical appraisal of cohort studies: 3. Analytical strategies to reduce confounding. BMJ. 2005;330:1021-3. DOI:10.1136/bmj.330.7498.1021.

21. Martsevich SY, Lukina YV, Kutishenko NP, et al. Observational multicenter trial of nicorandil use in stable coronary heart disease high risk patients (NIKEA): design and first results. Russian Journal of Cardiology. 2017;(9):75-82 (In Russ.) DOI:10.15829/1560-4071-2017-9-75-82

22. Martsevich SYu, Lukina YuV, Kutishenko NP, et al. Features and main problems of treating patients with high and very high cardiovascular risk with statins in real clinical practice (according to the data of the “PRIORITET” research). Cardiovascular Therapy and Prevention. 2018;17(6):52-60 (In Russ.) DOI:10.15829/1728-8800-2018-6-52-61.

23. Martsevich SY, Lukina YV, Kutishenko NP, et al. Adherence to Treatment with New Oral Anticoagulants in Atrial Fibrillation Patients in Real Clinical Practice (Results of the ANTEY Study). Rational Pharmacotherapy in Cardiology. 2019;15(6):864-72 (In Russ.) DOI:10.20996/1819-6446-2019-15-6-864-872.

24. Lukina YV, Kutishenko NP, Martsevich SY. Efficacy, Safety and Long-term Outcomes of Nicorandil Use in Patients with Stable Ischemic Heart Disease According to the Results of Randomized and Observational Studies. Rational Pharmacotherapy in Cardiology. 2019;15(5):641-8 (In Russ.) DOI:10.20996/1819-6446-2019-15-5-641-648.

25. Martsevich SY, Lukina YV, Kutishenko NP, et al. on behalf of the working group of the observational study PRIORITY. Adherence to Statins Therapy of High and Very High Cardiovascular Risk Patients in Real Clinical Practice: Diagnostics and Possible Ways to Solve the Problem (According to the PRIORITY Observational Study). Rational Pharmacotherapy in Cardiology 2018;14(6):891-900 (In Russ.) DOI:10.20996/1819-6446-2018-14-6-891-900.

26. Zhang Z, Peluso MJ, Gross CP, et al. Adherence reporting in randomized controlled trials. Clin Trials. 2014;(2):195-204. DOI:10.1177/1740774513512565.

27. Federal Law “On the Circulation of Medicines” dated 12.04.2010 N 61-FZ. Available at: http://pravo.gov.ru/proxy/ips/?docbody=&nd=102137440 (In Russ.)


For citation:


Martsevich S.Yu., Kutishenko N.P., Lukina Yu.V., Lukyanov M.M., Drapkina O.M. Clinical Trials Evaluating Drug Therapy: Types, Reliability of Results, Place in Modern Evidence-Based Medicine. Rational Pharmacotherapy in Cardiology. 2021;17(2):249-255. https://doi.org/10.20996/1819-6446-2021-04-07

Views: 168


Creative Commons License
This work is licensed under a Creative Commons Attribution 4.0 License.


ISSN 1819-6446 (Print)
ISSN 2225-3653 (Online)