Real-World Clinical Trials Themes and Projects

The themes and potential projects generated from the stakeholder consultations (1:1, planning event, webinars, teleconferences and individual meetings are::

1. Enhancing generalizability and individualized treatment: ensuring treatment needs in the broad population are addressed but with a focus on individual patient priorities (PROMs) and needs (precision medicine). Potential projects include:

  • Approaches to developing composite outcomes that reflect individual patient priorities (e.g. using discrete choice experiments and appropriate analytic models; combining individual level priorities with individual level trial outcomes to obtain individualized net benefit scores)
  • Approaches to designing “mini-trials” that can be aggregated into a “big” trial
  • Developing causal analysis methods for comparing per-protocol effectiveness that accounts for individual patient priorities
  • Integrating precision medicine considerations in trial design and analysis

2. Addressing real world limitations: making trials feasible and efficient in real world settings (constraints on blinding, randomization, sample size, operational procedures, ethical considerations). Potential projects include:

  • Developing hybrid effectiveness-implementation designs
  • Evaluating and optimizing power in non-standard trial designs (e.g. step-wedge designs with varying cluster sizes; realized power distribution of a randomization scheme; adaptive trial designs)
  • Improving recruitment and consenting processes (e.g. simplifying consent using technology; identifying patients’ subgroups with different preferences for consent procedures)

3. Leveraging external information sources: making use of non-trial information (published literature, health databases/medical records, expert opinion) to get answers more quickly and enhance the value of a trial. Potential projects include:

  • Analytic methods for combining heterogeneous data sources to support decision-making (both patient and policy)
  • Develop methods for conducting registry-based trials
  • New approaches to eliciting joint prior distributions for treatment effects

 

Projects that are moving forward

The RWCT MC is now working with teams to move five projects forward. There is intent subsequently to support additional projects for new areas of methodological research (depending upon resources).

The projects moving forward initially are (names of project leaders listed in alphabetical order):

  1. Approaches to developing composite outcomes that reflect individual patient priorities (e.g. using discrete choice experiments and appropriate analytic models; combining individual level priorities with individual level trial outcomes to obtain individualized net benefit scores). Co-Leads: Joel Singer & Nick Bansback
  1. Developing causal analysis methods for comparing per-protocol effectiveness that accounts for individual patient priorities. Lead: Ehsan Karim
  1. Increasing statistical efficiency including: (1) Evaluating and optimizing power in non-standard trial designs (e.g. step-wedge designs with varying cluster sizes; realized power distribution of a randomization scheme; adaptive trial designs) and (2) Improving the utilization of prior information (e.g. new approaches to eliciting joint prior distributions for treatment effects). Lead: Hubert Wong
  1. Improving recruitment and consenting processes (e.g. simplifying consent using technology; identifying patients’ subgroups with different preferences for consent procedures). Lead: Penny Brasher
  1. Develop methods for conducting registry-based trials. Co-Leads: Xibiao Ye & Penny Brasher