Top Study Designs for Late Phase Research

Top Study Designs for Late Phase Research

In a recent posting, we provided the Top 10 Reasons to Conduct Late Phase Research.  Focused and strategic peri and postapproval research, culminating in definitive evidence of product value, can make a significant impact on market access and product uptake. 

As the value demonstration evidence needs in support of a product take shape, important tactical questions begin to surface. 

  • Should outcomes measures be included in a mandated post-marketing global registry to address evidence needs beyond safety? 
  • Given the increasing focus on patient-centered outcomes, can direct-to-patient outreach be incorporated in the study design to help meet the specific needs of the program? 
  • Should a comparative effectiveness study be considered prior to product launch in order to address payor questions or requirements? 
  • How should the collection and analysis of data reflecting trends in patient compliance and adherence be executed? 

A diversity of study designs exists to address these types of research questions.  The challenge is to select the “right” design for the “right” research question(s).  UBC’s experience in scientifically rigorous study designs and methodologies across a wide range of patient populations and therapeutic areas can help you demonstrate and communicate your product’s value to a variety of decision makers and audiences.  UBC can offer leadership in the design and execution of:

  • Case-control Studies: to identify risk factors associated with adverse events
  • Multi-national retrospective Chart Review studies:  to evaluate burden of illness or drug utilization patterns including off-label use
  • Cross-sectional hybrid, retrospective and prospective observational studies:  these can be used to quantify burden of illness, ideal for data inputs to health economic models and other evaluations
  • Large Streamlined or Pragmatic Trials: well-suited for evaluations of comparative effectiveness with the strength of a randomized design and the evaluation of products under actual use conditions after randomization
  • Randomized, Blinded Clinical Trials: considered the “gold standard,” but with limitations on generalizability to real-world medical practice
  • Disease or Product Registries or Pregnancy Exposure Registries: to understand natural history of disease or risks and benefits of products under actual use conditions
  • Patient and Healthcare Provider Knowledge, Attitude and Behavior Surveys: cross sectional surveys used to evaluate patient and prescriber understanding of risk minimization initiatives
  • Standardized Multinational Resource Utilization Surveys:  using questionnaires geared to health economic assessments or to collect data on product use
  • Retrospective Burden of Illness or Drug Utilization Database Studies
  • National and Multinational Time and Motion Studies:  to quantify the efficiency and opportunity cost savings of novel drug formulations or medical devices

Strategic and synergistic real-world evidence gathering across the product lifecycle can and will contribute significantly to cost and timeline efficiencies.  Contact Krista Payne to learn more.