Concordancia, eficiencia y aplicación regional de metodologías epidemiológicas frente al ensayo aleatorizado: revisión sistemática y meta-análisis meta-epidemiológico
DOI:
https://doi.org/10.71068/hs000106Palabras clave:
epidemiología, metodología de la investigación, meta-epidemiología, emulación de ensayo diana, evidencia de mundo real, razón de razones, equidad en saludResumen
Introducción: la elección del diseño condiciona la validez, la oportunidad y la utilidad de la evidencia epidemiológica. Estimamos la concordancia de los diseños no aleatorizados y emergentes con el ensayo controlado aleatorizado (ECA) y describimos su eficiencia y aplicación regional.Método: revisión sistemática y meta-análisis meta-epidemiológico según PRISMA 2020. Consultamos MEDLINE, Embase, Scopus, Web of Science y Cochrane (enero de 2020 a mayo de 2026). Se priorizó la comparación de cinco estimadores no aleatorizados frente a ECA; el agrupamiento heterogéneo de seis se mantuvo como exploratorio. Se aplicaron efectos aleatorios, REML, Hartung-Knapp-Sidik-Jonkman y exclusión conjunta de posibles fuentes solapadas. Se evaluaron riesgo de sesgo, certeza GRADE adaptada y matrices ordinales de distribución regional y eficiencia.Resultados: se incluyeron 24 estudios. Los cinco contrastes entre diseños no aleatorizados y ECA produjeron una RoR de 1.00 (IC 95 % 0.92–1.10); el agrupamiento exploratorio de seis estimadores fue 0.98 (0.89–1.06; I² = 77.9 %). Al excluir conjuntamente Toews y Kimachi, los tres estimadores no aleatorizados restantes dieron aproximadamente 0.95 (0.88–1.02), reconstruido desde valores publicados redondeados. Las RoR por familias fueron 1.05 para observacionales y 0.91 para emulación/evidencia de mundo real, con certeza baja y muy baja, respectivamente. Dieciséis de 28 puntuaciones regionales máximas se basaron en evidencia indirecta.Conclusiones: una RoR próxima a 1 no demuestra equivalencia con los ECA. Los contrastes entre diseños y las puntuaciones regionales requieren interpretación prudente y por familias; la combinación de métodos sigue siendo una hipótesis que debe evaluarse.
Referencias
1. Kim DH, et al. The impact of study size on COVID-19 treatment outcomes: a meta-epidemiological study comparing large and small randomized controlled trials. Rev Med Virol. 2026;36(1):e70125. https://doi.org/10.1002/rmv.70125.
2. Page MJ, McKenzie JE, Bossuyt PM, Boutron I, Hoffmann TC, Mulrow CD, et al. The PRISMA 2020 statement: an updated guideline for reporting systematic reviews. BMJ. 2021;372:n71. https://doi.org/10.1136/bmj.n71.
3. Hernán MA, Wang W, Leaf DE. Target trial emulation: a framework for causal inference from observational data. JAMA. 2022;328(24):2446-7. https://doi.org/10.1001/jama.2022.21383.
4. Hemming K, Taljaard M. Reflection on modern methods: when is a stepped-wedge cluster randomized trial a good study design choice? Int J Epidemiol. 2020;49(3):1043-52. https://doi.org/10.1093/ije/dyaa077
5. RECOVERY Collaborative Group; Horby P, Lim WS, Emberson JR, Mafham M, Bell JL, et al. Dexamethasone in hospitalized patients with Covid-19. N Engl J Med. 2021;384(8):693-704. https://doi.org/10.1056/NEJMoa2021436.
6. Toews I, Anglemyer A, Nyirenda JLZ, Alsaid D, Balduzzi S, Grummich K, et al. Healthcare outcomes assessed with observational study designs compared with those assessed in randomized trials: a meta-epidemiological study. Cochrane Database Syst Rev. 2024;1(1):MR000034. https://doi.org/10.1002/14651858.MR000034.pub3.
7. Concato J, Corrigan-Curay J. Real-world evidence: where are we now? N Engl J Med. 2022;386(18):1680-2. https://doi.org/10.1056/NEJMp2200089
8. Dean NE, Hogan JW, Schnitzer ME. Covid-19 vaccine effectiveness and the test-negative design. N Engl J Med. 2021;385(15):1431-3. https://doi.org/10.1056/NEJMe2113151.
9. Sanderson E, Glymour MM, Holmes MV, Kang H, Morrison J, Munafò MR, et al. Mendelian randomization. Nat Rev Methods Primers. 2022;2:6. https://doi.org/10.1038/s43586-021-00092-5.
10. Bergeri I, Whelan MG, Ware H, Subissi L, Nardone A, Lewis HC, et al. Global SARS-CoV-2 seroprevalence from January 2020 to April 2022: a systematic review and meta-analysis of standardized population-based studies. PLoS Med. 2022;19(11):e1004107. https://doi.org/10.1371/journal.pmed.1004107.
11. Brito AF, Semenova E, Dudas G, Hassler GW, Kalinich CC, Kraemer MUG, et al. Global disparities in SARS-CoV-2 genomic surveillance. Nat Commun. 2022;13(1):7003. https://doi.org/10.1038/s41467-022-33713-y.
12. Diamond MB, Keshaviah A, Bento AI, Conroy-Ben O, Driver EM, Halden RU, et al. Wastewater surveillance of pathogens can inform public health responses. Nat Med. 2022;28(10):1992-5. https://doi.org/10.1038/s41591-022-01940-x.
13. Skivington K, Matthews L, Simpson SA, Craig P, Baird J, Blazeby JM, et al. A new framework for developing and evaluating complex interventions: update of Medical Research Council guidance. BMJ. 2021;374:n2061. https://doi.org/10.1136/bmj.n2061.
14. Curran GM, Landes SJ, McBain SA, Pyne JM, Smith JD, Fernandez ME, et al. Reflections on 10 years of effectiveness-implementation hybrid studies. Front Health Serv. 2022;2:1053496. https://doi.org/10.3389/frhs.2022.1053496.
15. Ortiz K, Nash J, Shea L, Oetzel J, Garoutte J, Sanchez-Youngman S, et al. Partnerships, processes, and outcomes: a health equity-focused scoping meta-review of community-engaged scholarship. Annu Rev Public Health. 2020;41:177-99. https://doi.org/10.1146/annurev-publhealth-040119-094220.
16. Kimachi M, Onishi A, Tajika A, Kimachi K, Furukawa TA. Systematic differences in effect estimates between observational studies and randomized control trials in meta-analyses in nephrology. Sci Rep. 2021;11(1):6088. https://doi.org/10.1038/s41598-021-85519-5.
17. Stadelmaier J, Bantle G, Gorenflo L, Kiesswetter E, Nikolakopoulou A, Schwingshackl L. Evaluating agreement between individual nutrition randomised controlled trials and cohort studies: a meta-epidemiological study. BMC Med. 2025;23. https://doi.org/10.1186/s12916-025-03860-2
18. Schwingshackl L, Balduzzi S, Beyerbach J, Bröckelmann N, Werner SS, Zähringer J, et al. Evaluating agreement between bodies of evidence from randomised controlled trials and cohort studies in nutrition research: meta-epidemiological study. BMJ. 2021;374:n1864. https://doi.org/10.1136/bmj.n1864.
19. Bröckelmann N, Balduzzi S, Harms L, Beyerbach J, Petropoulou M, Kubiak C, et al. Evaluating agreement between bodies of evidence from randomized controlled trials and cohort studies in medical research: a meta-epidemiological study. BMC Med. 2022;20(1):174. https://doi.org/10.1186/s12916-022-02369-2.
20. Wang SV, Schneeweiss S; RCT-DUPLICATE Initiative. Emulation of randomized clinical trials with nonrandomized database analyses: results of 32 clinical trials. JAMA. 2023;329(16):1376-85. https://doi.org/10.1001/jama.2023.4221.
21. Heyard R, Held L, Schneeweiss S, Wang SV. Design differences and variation in results between randomised trials and non-randomised emulations: meta-analysis of RCT-DUPLICATE data. BMJ Med. 2024;3(1):e000709. https://doi.org/10.1136/bmjmed-2023-000709.
22. Franklin JM, Patorno E, Desai RJ, Glynn RJ, Martin D, Quinto K, et al. Emulating randomized clinical trials with nonrandomized real-world evidence studies: first results from the RCT DUPLICATE initiative. Circulation. 2021;143(10):1002-13. https://doi.org/10.1161/CIRCULATIONAHA.120.051718.
23. WHO Solidarity Trial Consortium; Pan H, Peto R, Henao-Restrepo AM, Preziosi MP, Sathiyamoorthy V, et al. Repurposed antiviral drugs for Covid-19: interim WHO Solidarity trial results. N Engl J Med. 2021;384(6):497-511. https://doi.org/10.1056/NEJMoa2023184.
24. Andaur Navarro CL, Damen JAA, Takada T, Nijman SWJ, Dhiman P, Ma J, et al. Risk of bias in studies on prediction models developed using supervised machine learning techniques: systematic review. BMJ. 2021;375:n2281. https://doi.org/10.1136/bmj.n2281.
25. Murad MH, Wang Z. Guidelines for reporting meta-epidemiological methodology research. Evid Based Med. 2017;22(4):139-42. https://doi.org/10.1136/ebmed-2017-110713.
26. Means AR, Kemp CG, Gwayi-Chore MC, Gimbel S, Soi C, Sherr K, et al. Evaluating and optimizing the consolidated framework for implementation research (CFIR) for use in low- and middle-income countries: a systematic review. Implement Sci. 2020;15(1):17. https://doi.org/10.1186/s13012-020-0977-0.
27. Breilh J. Critical epidemiology and the people's health. New York: Oxford University Press; 2021. ISBN: 9780190492793.
28. Ministerio de Salud Pública y Asistencia Social de Guatemala, Organización Panamericana de la Salud. Guía de diálogos interculturales en salud. Guatemala: MSPAS, OPS; 2024. Disponible en: https://www.paho.org/es/documentos/guia-dialogos-interculturales-salud
29. Page MJ, Moher D, Bossuyt PM, Boutron I, Hoffmann TC, Mulrow CD, et al. PRISMA 2020 explanation and elaboration: updated guidance and exemplars for reporting systematic reviews. BMJ. 2021;372:n160. https://doi.org/10.1136/bmj.n160.
30. Whiting P, Savović J, Higgins JPT, Caldwell DM, Reeves BC, Shea B, et al. ROBIS: a new tool to assess risk of bias in systematic reviews was developed. J Clin Epidemiol. 2016;69:225-34. https://doi.org/10.1016/j.jclinepi.2015.06.005.
31. Guyatt G, Oxman AD, Akl EA, Kunz R, Vist G, Brozek J, et al. GRADE guidelines: 1. Introduction-GRADE evidence profiles and summary of findings tables. J Clin Epidemiol. 2011;64(4):383-94. https://doi.org/10.1016/j.jclinepi.2010.04.026.
32. IntHout J, Ioannidis JPA, Borm GF. The Hartung-Knapp-Sidik-Jonkman method for random effects meta-analysis is straightforward and considerably outperforms the standard DerSimonian-Laird method. BMC Med Res Methodol. 2014;14:25. https://doi.org/10.1186/1471-2288-14-25.
33. Langan D, Higgins JPT, Jackson D, Bowden J, Veroniki AA, Kontopantelis E, et al. A comparison of heterogeneity variance estimators in simulated random-effects meta-analyses. Res Synth Methods. 2019;10(1):83-98. https://doi.org/10.1002/jrsm.1316.
34. Altman DG, Bland JM. Absence of evidence is not evidence of absence. BMJ. 1995;311(7003):485. https://doi.org/10.1136/bmj.311.7003.485.
35. Huang Bartlett C, Mardekian J, Cotter MJ, Huang X, Zhang Z, Parrinello CM, et al. Concordance of real-world versus conventional progression-free survival from a phase 3 trial of endocrine therapy as first-line treatment for metastatic breast cancer. PLoS One. 2020;15(4):e0227256. https://doi.org/10.1371/journal.pone.0227256.
Publicado
Número
Sección
Licencia
Derechos de autor 2026 Marco Antonio Ramos Espinal

Esta obra está bajo una licencia internacional Creative Commons Atribución 4.0.
Los artículos publicados en esta revista están disponibles bajo la licencia Creative Commons Atribución 4.0 Internacional (CC BY 4.0). Esta licencia autoriza a terceros a descargar, copiar, distribuir, adaptar y reutilizar los contenidos, incluso con fines comerciales, siempre que se otorgue el crédito correspondiente al autor original de la obra.


