The National Heart, Lung, and Blood Institute (NHLBI) convened Working Group teleconferences on December 7 and 17th, 2010 to discuss the Value of Information (VOI). The purpose of the meeting was to identify the most effective use of VOI models in order to evaluate the potential usefulness and economic impact of proposed large-scale NHLBI clinical studies. The Working Group was charged with providing expert input on key issues regarding a proposed VOI program, as well as prioritized recommendations as to how to move the field forward. The Working Group consisted of experts in VOI modeling, epidemiology, biostatistics, and clinical research.
This working group is responsive to NHLBI Strategic Plan Goals 2 and 3.
NHLBI faces major challenges in determining how best to allocate investment in clinical research. Currently, there is no consistent method or mechanism to evaluate and compare proposed clinical studies for NHLBI to best determine where to invest its limited dollars. NHLBI decision-making relies on expert knowledge to assess potential impact and design of proposed clinical trials and studies. VOI analysis could supplement this expert knowledge to help NHLBI evaluate the scientific focus and design of proposed clinical studies and trials, and VOI models would provide NHLBI with an additional tool to aid decisions for allocating funding for research within and across disease areas, and to ensure most effective and economic study design.
VOI analysis puts a value on reducing uncertainty by calculating how the costs and consequences of decisions made with current evidence differ from those made with future evidence that resolves key unknowns. Should the future evidence prove more valuable than the current evidence, then undertaking research to obtain it makes sense, especially if the associated costs can be justified. VOI analysis expands on cost-effectiveness analysis and can be used to determine if more research is justified regarding a medical decision. VOI can help prioritize research investments, evaluate study design, and estimate optimal sample size.
The working group discussion started with a definition of value of information (VOI) modeling and an overview of the field. VOI is a modeling-based approach that quantifies uncertainty and evaluates the value of information derived from a potential study. VOI puts a population-based value on potential improvements in clinical knowledge or care, often in terms of quality-adjusted life years (QALY).
VOI is currently used to prioritize which studies to fund across interventions or across diseases, based on the potential impact on public health. VOI approaches are also used to determine study size and to assess clinical study design. The field of VOI is emerging, particularly in the United States, and the working group noted that there is a need for greater development of VOI methods and approaches, as well as training and workforce development.
The working group discussion addressed several questions, including what size clinical study should be evaluated by VOI, how long it takes to perform a VOI analysis, when during study development a VOI analysis should be performed, the effectiveness of VOI and how it is currently being used, as well as how to implement and evaluate VOI, and the role for training in VOI.
To address the challenges identified in VOI research, the working group identified several priorities for the development and application of VOI, including:
- A mechanism to facilitate the development and validation of novel research methodologies for VOI analyses.
- VOI networks or centers, each comprised of a multi-disciplinary team, that may (1) test VOI models against common problems to rapidly identify and share improved methods and approaches for VOI, (2) serve as a resource to the NHLBI for VOI analyses of proposed and ongoing clinical studies, (3) support cross-disciplinary training to ensure that VOI methods are informed by best practices and needs from multiple supporting fields of research, and (3) foster collaboration.
- A mechanism to support and foster training and workforce development in VOI research and application at the graduate, post-graduate, and early investigator levels.
- A mechanism to support application of VOI approaches retrospectively to completed NHLBI clinical studies and trials (such as those listed in the NHLBI BioLINCC resource) for research development purposes and feasibility studies.
- A mechanism to support VOI analysis on rapidly developing questions, such as analysis of a proposed major new clinical trial, or assessment of a developing area of clinical treatment, to identify the most critical clinical trial.
The group emphasized that these mechanisms and resources should be scalable, adaptive, and flexible to respond to the rapidly changing VOI field and needs of the NHLBI and the research community.
NHLBI website and peer-reviewed journal
Jennie Larkin, PhD
NHLBI, NIH, DHHS
Dina Paltoo, PhD, MPH
NHLBI, NIH, DHHS
Working Group Participants
- David O. Meltzer, MD, PhD, University of Chicago
- Anirban Basu, PhD, University of Washington
- Martin L. Brown, PhD, National Cancer Institute
- Karl Claxton, DPhil, University of York (UK)
- Kay Dickerson, PhD, Johns Hopkins University
- Eric J. (Rocky) Feuer, PhD, National Cancer Institute
- J. Sanford (Sandy) Schwartz, MD, University of Pennsylvania
- David L. Veenstra, PharmD, PhD, University of Washington
- Robin Boineau, MD
- Denise Bonds, MD
- Debbie Eng
- Jonathan Kaltman, MD
- Peter Kaufman, PhD
- Marissa Miller, DVM, MPH
- Lan-Hsiang Wang, PhD
- Barbara Wells, PhD
Last Updated: August 2011|