Three faculty members from the Pharmaceutical Outcomes Research and Policy Program (PORPP) and two faculty from the Division of Cardiology received a $3 million grant to develop a comprehensive toolkit of pragmatic value of information (VOI) approaches and the corresponding software that can readily be used by clinical researchers and funders to estimate the a-priori value of RCTs. Stergachis Family Professor of Pharmacy and Director of PORPP, Anirban Basu, is the principal investigator of the four-year project, which is being funded by the NHLBI. Assistant Professor of Pharmacy Josh Carlson and Professor of Pharmacy Dave Veenstra are key co-investigators for the project, along with Drs. Kelley Branch and Jeff Probstfield from the Division of Cardiology.
Like many other National Institutes of Health, the National Heart Lung and Blood Institute (NHLBI) faces major challenges in determining how best to allocate investments in randomized clinical trials (RCTs). Funding prioritization decisions rely on scientific literature, systematic reviews, meta-analyses and expert opinion to assess potential impact and design of proposed clinical trials and studies. This process likely fails to consistently account for 1) uncertainty around medical decisions, 2) health impact of making optimal treatment decisions, and 3) population level return on investment. These challenges are particularly acute for the NHLBI because of the breadth of interventions and size of the potential population impact within its disease areas. Novel approaches in decision science can provide valuable information to enhance the RCT prioritization process at NHLBI.
“We would develop a checklist that would indicate the feasibility of applying these methods to a proposed RCT,” said Basu. “When feasible, our software can enable researchers to demonstrate the expected population value of conducting this trial. When not, it would inform NHLBI that more nuanced discussions are needed to better understand the expected returns from the proposed research.”
Basu and the team will be conducting a broad range of health economics and outcomes research-based research activities. First, they will develop a hierarchy of commonly used end-points in HLB research based on the ease with which these endpoints could be translated to comprehensive population outcome measures such as overall survival and quality-adjusted life years. They will then develop a stakeholder-informed checklist that accounts for this hierarchy and informs researchers whether it is feasible to estimate the expected population value of a proposed trial using pragmatic VOI methods.
They will also develop a variety of pragmatic VOI methods based on simplified mathematical simulation models, building on their prior work in this area. These methods will be showcased in a user friendly web-based software as a proof-of-concept to perform these calculations for an RCT based on inputs received from NHLBI stakeholders.
“We think that the demonstration of these methods in the context of NHLBI trials can really add to the repertoire of tools that funders have to make informed decisions about prioritizing research studies” said Basu. “In the long-run, these methods could be extended to many other areas of research and hopefully help build a more robust portfolio of research investments in the United States and abroad.”