In September 2020, the Board of Governors for the Patient-Centered Outcomes Research Institute (PCORI) approved the proposed Principles for the Consideration of the Full Range of Outcomes Data for public comment. The goal of these principles is to outline PCORI’s compliance with its reauthorization legislation, which states that, in addition to clinical outcomes, research should also endeavor to capture patient-important outcomes that assess the economic burden of treatments and services.
While clinical effectiveness is still the priority, cost outcomes from the patient perspective are a reality that sometimes gets left behind. Patients struggle with affordability, travels costs associated with treatment, caregiver resources, etc. It is important to understand the existing evidence about which economic burden outcomes matter most to patients, as well as where PCORI may need to conduct additional research to fill in gaps. PCORI will need to better understand the landscape to ensure that research going forward captures accurate and actionable information about the economic burden of treatments from the patient perspective and how this information may be used to drive patient decision making when combined with the results of PCORI’s comparative-clinical effectiveness research.
In this new white paper, Xcenda conducted a targeted literature review in September 2020 to better understand how patient-important cost outcomes were considered in studies.
Key findings include:
- Patient preferences of economic burden outcomes are understudied in the literature
- Economic burden outcomes are often excluded from preference studies, and when they are reported, they are often assessed using a single catchall “cost” outcome
- More patient preference research is necessary to consider the full range of outcomes important to patients, including those that take affordability and economic impact into consideration
- Patients may prioritize economic burden outcomes differently based on a variety of factors, and their economic outcome preferences may factor into their treatment decisions
- Although treatment effectiveness and safety are major drivers behind treatment selection, economic burden also plays a major role in the decision-making process
Further research by PCORI may help to fill some of these evidence gaps and facilitate more informed healthcare treatment decision making.