Translational Research in Cardiovascular Diseases: from Biomarker-based Adaptive Explanatory Studies to Pragmatic Trials


26-30 November

Cardiovascular disease produces immense health and economic burdens worldwide. It is the leading cause of death in OECD countries. The prevalence and control of cardiovascular health factors and risks remain a major issue.

The goal of translational research is the transfer of new understandings of disease mechanisms gained in the laboratory into the development of new methods for diagnosis, therapy, and prevention. The past two decades have brought a surge of interest for assessing the role of biomarkers in a wide range of cardiovascular disease. For instance, it appeared that circulating biomarkers can predict cardiovascular risk in large population-based cohorts. Testing of the circulating biomarkers combined with imaging biomarkers has become an integral part of everyday translational and clinical research.

Clinical research corresponds to any investigation in human subjects intended to (i) determine the clinical pharmacological effects of a drug and (ii) identify any adverse reactions to a drug. There are various types of clinical studies, which can be primarily categorized in observational versus interventional designs. Among interventional studies, randomized placebo-controlled double-blinded clinical trials (RCTs) are the gold-standard to assess the efficacy and safety of a drug. Nevertheless, several limitations of RCTs have been highlighted, including (i) the need for a large sample size and long study duration, (ii) the lack of power to evaluate efficacy overall versus in relevant subgroups, and (iii) the cost. Given the rapidly evolving field of biomarker-based personalized medicine, adaptive trial design has been proposed to increase the efficiency of explanatory RCTs and enhance the likelihood of finding a true benefit, if one exists, of the drug under investigation. In parallel, pragmatism has progressively arisen in in clinical research to question the effectiveness and safety of the investigational drug in real-world settings.

At the end of the present course, PhD candidates should be able to appreciate the importance of incorporating translational research towards clinical applications to increase the effectiveness and relevance of biomedical research in healthcare.

 

Aims of the course

  • To provide the PhD candidates with basic knowledge in cardiovascular diseases
  • To highlight key milestones in translational research in cardiovascular diseases
  • To teach participants about the strengths and limitations of RCTs, with a focus on adaptive explanatory and pragmatic designs

By the end of the course, the participants should be able to:

  • Conceptualize a translational research project
  • Propose logistic, methodological, and statistical approaches
  • Delineate the landscape of interventional clinical research;
  • Identify the strengths and limitations of RCTs;
  • Understand the concept of personalized medicine applied to RCTs
  • Appreciate the need for pragmatism in RCTs 

Target group:  PhD candidates (group limited to 15 participants)

Prerequisites:

  • Working knowledge of English;
  • Basic knowledge in clinical research;
  • Basic knowledge in translational research;
  • Basic knowledge in biomarkers.

Duration of the course and workload: 4 hours per day, over 5 days; 3h for attending and participating in each one-day course + 1h preparation before attending the course for the reading of the literature provided by the teacher. One day of practical course in the laboratory.  4x4+8 = 24 hours total workload

Location: GIGA B34, +5

 

Educators

François Jouret, Cécile Oury and members of her team (TBC), GIGA-Cardiovascular Sciences, ULiege

 

Course program

 

Day 1

9:00 – 13:00. Milestones of Translational research in Cardiovascular disease

Day 2

9:00 – 13:00. Role of circulating biomarkers in cardiovascular risk assessment

Day 3

9:00 – 13:00. Role of imaging biomarkers in everyday clinical practice – notions of radiomics

Day 4

14:00-14:00. Overview of clinical research, with a focus on interventional RCTs. The typical design of randomized placebo-controlled double-blinded clinical trials will be detailed, with emphasis on the potential biases of such an approach.

14:00-15:00. Overview of the personalized medicine, with a focus on “biomarkers”. The different types of biomarkers will be detailed in order to emphasize their potential use in clinical research. From these concepts, the adaptive trial design of RCTs will be explained.

15:00-16:00. Definition of “pragmatism” in clinical research, with a focus on the strengths and limitations of pragmatic RCTs.

Day 5

9:00 – 17:00. Practical course in the laboratory: participation in biobanking activity, patient sample collection, processing, and storage.  Critical reading and comparison of an adaptive versus pragmatic RCT.

 

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