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Case Study

Using Real Word Evidence to Enhance Drug Development

Real-world evidence (RWE) has emerged as an increasingly important tool in the drug development process,1 providing valuable insights into the effectiveness of treatments and potential areas for improvement. It can be derived from sources such as observational studies, medical records, administrative claims, and patient registries. By leveraging advanced analytics, RWE has the potential to further support decision-making throughout the entire drug life cycle from preclinical development through post-approval surveillance.

Challenge

Real-world effectiveness of a drug can differ from efficacy observed in randomized control trials (RCTs) due to differences in study populations, prescription patterns, as well as adherence to treatment.2,3 Certara was contracted by a sponsor to leverage RCT and RW data to generate evidence-based insights and bolster confidence in a particular medical product’s safety, efficacy, and cost-effectiveness in markets that are becoming increasingly more crowded.

Solution

Certara designed and implemented integrated drug and disease modeling using RCT data to maximize drug access, use, and uptake. This was done by:

  • Using RCT data to inform clinical decisions:
    • Optimal use, responders vs. non-responders
    • Optimizing clinical trial design (defining response variability and covariates impacting the most response)
    • Comparative safety/efficacy through model-based meta-analysis
  • Bridging RCT data to the RW setting with confidence by:
    • Characterizing sources of variability in RW
    • Effectively disseminating evidence
    • Leveraging RW data for improved modeling
    • Activating evidence to engage and enable clinical adoption and access

Figure 1. Modeling and simulation approaches were used to explore the clinical trial data and characterize and predict long-term effects.

Figure 1. Modeling and simulation approaches were used to explore the clinical trial data and characterize and predict long-term effects.

Benefits

Certara’s innovative simulation-based approach offers significant benefits in optimizing clinical trial design and decision-making. Using clinically and ‘RW’ relevant endpoints facilitates determining drug comparative safety and efficacy, as well as predicting effectiveness of a new drug in the RW setting, thereby quantifying its impact. The results of this study can be used to select the most relevant patient subgroups and optimize selection criteria and sample sizes for clinical trials, as well as to refine the specific indication for a drug and its positioning within its market.

References
  1. Real-world studies for the assessment of medicinal products and medical devices. https://www.has-sante.fr/upload/docs/application/pdf/2021-06/real-world_studies_for_the_assessment_of_medicinal_products_and_medical_devices.pdf. Published June 10, 2021. Accessed April 5, 2023.
  2. Ferrières J, Dallongeville J, Rossignol M, Bénichou J, Caro JJ, Getsios D, Hernandez L, Abenhaim L, Grimaldi-Bensouda L. Model-observational bridging study on the effectiveness of ezetimibe on cardiovascular morbidity and mortality in France: A population-based study. J Clin Lipidol. 2016 Nov-Dec;10(6):1379-1388. Doi: 10.1016/j.jacl.2016.08.015. Epub 2016 Sep 7. PMID: 27919355.
  3. Schiele F, Quignot N, Khachatryan A, Gusto G, Villa G, Kahangire D, Chauny JV, Ricci L, Desamericq G. Clinical impact and room for improvement of intensity and adherence to lipid lowering therapy: Five years of clinical follow-up from 164,565 post-myocardial infarction patients. Int J Cardiol. 2021 Jun 1;332:22-28. doi: 10.1016/j.ijcard.2021.03.007. Epub 2021 Mar 8. PMID: 33705845.

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