The role of real clinical practice data in modern healthcare

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DOI: https://doi.org/10.29296/25419218-2021-03-01
Issue: 
3
Year: 
2021

E.A. Novoderezhkina, S.K. Zyryanov Peoples’ Friendship University of Russia, 6, Miklukho-Maklai St., Moscow 117198, Russian Federation

The paper defines the place of real world evidence (RWE) in evidence-based medicine and its role in modern healthcare by the example of psoriasis on the basis of the works published in the MedLine, PubMed, and Cochrane Library databases in 2010–2020. It considers the advantages and disadvantages of RWE studies and randomized clinical trials (RCTs). It is noted that the data of both types of studies can complement each other. In the foreseeable future, RCTs will remain necessary for the initial approval of drugs and treatments that must be initially demonstrated to be effective in the primary target group and for the primary indication in the RCTs before they can be introduced into the RWE. RWE studies will be able to more effectively identify the unresolved medical needs and molecular targets for further elaboration of therapeutic approaches, to register drugs, and to expand their range of indications. As the drawbacks in the structure of RWE studies are corrected, the importance of this method will grow in the clinical, regulatory, and economic areas of healthcare.
Keywords: 
real world evidence (RWE)
randomized clinical trials (RCTs)
psoriasis
References: 
  1. U.S. Food & Drug Administration. Framework for FDA’s Real-World Evidence program. 2018.
  2. Duke Margolis Center for Health Policy. Developing Real-World Data and Evidence to Support Regulatory Decision-Making. 2019.
  3. Duke-Margolis RWE Collaborative Methods Working Group. Adding Real-World Evidence to a Totality of Evidence Approach for Evaluating Marketed Product Effectiveness. Duke-Margolis Cent. Heal. Policy. 2019.
  4. U.S. Department of Health and Human Services Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER). Submitting DocumentsnUsing Real-World Data and Real-World Evidence to FDA for Drugs and Biologics. Guidance for Industry. 2019.
  5. European Medicines Agency. Guidance for companies considering the adaptive pathways approach. 2016; 44: 1–6.
  6. Davis B., Morgan J., Shah S. Getting real with real-world evidence: Deloitte’s Real-World Evidence Benchmark Survey shows life sciences companies have room for improvement. Deloitte Insights. 2017.
  7. Davis B., Morgan J., Shah S. Mission critical Biopharma companies are accelerating real-world evidence adoption, investment and application. Deloitte Insights. 2018.
  8. Eichler H.G. et al. Are Novel, Nonrandomized Analytic Methods Fit for Decision Making? The Need for Prospective, Controlled, and Transparent Validation. Clin. Pharmacol. Ther. 2020; 107 (4): 773–9.
  9. Sharma V. EMA Discusses Pivotal Role Of RWE For Six Drugs. The Pink Sheet. 2019.
  10. Yndigegn T. et al. Registry-based randomised clinical trial: Efficient evaluation of generic pharmacotherapies in the contemporary era. Heart. BMJ Publishing Group. 2018; 104 (19): 1562–7.
  11. James S., Rao S. V., Granger C.B. Registry-based randomized clinical trials – A new clinical trial paradigm. Nat. Rev. Cardiol. Nature Publishing Group. 2015; 12 (5): 312–6.
  12. Sheffield K.M. et al. Replication of randomized clinical trial results using real-world data: paving the way for effectiveness decisions. J. Comp. Eff. Res. 2020.
  13. Panayidou K. et al. GetReal in mathematical modelling: a review of studies predicting drug effectiveness in the real world. Res. Synth. Methods. 2016; 7 (3): 264–77.
  14. European Medicines Agency. Guideline on registry-based studies. Draft. 2020; EMA/502388/2020: 1–33.
  15. Sharma V. EMA Scouts Market To Procure Sentinel-Style RWD Database: Pink Sheet. [Electronic resource]. PinkSheet, Informa Pharma Intelligence. 2020. Access mode: https://pink.pharmaintelligence.informa.com PS142782/EMA-Scouts-Market-To-Procure-SentinelStyle-RW. D-Database (circulation date: 07.10.2020).
  16. EMA. HMA-EMA Joint Big Data Taskforce-summary report. 2019; EMA/105321/2019.
  17. EMA. HMA-EMA Joint Big Data Taskforce Phase II report: Evolving Data-Driven Regulation. 2020; 1–61.
  18. European Medicines Agency. Ten recommendations to unlock the potential of big data for public health in the EU. EMA/689902/2019, press release. 2020.
  19. Sentinel Initiative. [Electronic resource]. Access mode: https://www.sentinelinitiative.org (circulation date 07.10.2020).
  20. CNODES. Canadian Network for Observational Drug Effects Studies. [Electronic resource]. Access mode: https://www.cnodes.ca (circulation date: 07.10.2020)