Cost effectiveness research aims to determine if the benefits of a treatment outweigh the cost. But which benefits should be included? How should we weight improvements in different outcomes? What data and methods need to be used to measure benefits and costs?
These are questions that academics typically think of as within the domain of research experts. However, there is another group of experts that is often ignored: patients.
A 2012 JAMA commentary from Mullins, Abdulhalim, and Lavallee (2012) discuss specific points to involve patients in the process. In particular, they advocate for continuous cost effectiveness research. This process would incorporate patient feedback on direction through the entire research process. The 20 steps they outline are detailed below.
- Topic solicitation: Identify topics that are important to patients, caregivers, and the community; Propose topics to be investigated
- Prioritization: Solicit feedback on relevance and priority of topics; Discuss the urgency of addressing topics
- Framing the question: Ascertain questions’ relevance and usefulness; Assess “real-world” applicability
- Selection of comparators and outcomes: Identify comparator treatments of interest; Identify outcomes of interest; Incorporate other aspects of treatment
- Creation of conceptual framework: Provide a “reality check”; Verify logic of conceptual framework; Supplement with additional factors not documented in the literature
- Analysis plan: Verify importance of factors and variables; Ascertain whether there is a good proxy for a specific concept; Inquire about potential confounding factors
- Data collection: Determine best approaches for data collection (eg, trial, registry, medical charts); Assist with selection of data sources
- Reviewing and interpreting results: Assess believability of results; Suggest alternative explanations or approaches; Provide input for sensitivity analysis
- Translation: Interpret results to be meaningful; Document which results are easy or difficult to understand; Indicate which results are counterintuitive
- Dissemination: Facilitate engagement of other patients; Help other patients to understand findings
Mullins and co-authors lay out a convincing case. Although patients have hetergeneous clinical backgrounds, treatment preferences and technical sophistication, if cost effectiveness reserach aims to ultimately to improve the value of care patients receive, it would be a disservice not to include patients themselves explicitly in the research process.
- Mullins, C. Daniel, Abdulla M. Abdulhalim, and Danielle C. Lavallee. “Continuous patient engagement in comparative effectiveness research.” JAMA 307, no. 15 (2012): 1587-1588.