What makes a health care intervention like a medicine valuable? It all depends on who you ask.
A patient may prioritize improvement of their symptoms or their ability to function independently without a caregiver. An employer may prioritize their employee’s ability to maintain a consistent work schedule. A health insurance company may prioritize the cost of the therapy.
Value assessment seeks to determine the relative benefits, costs, risks, and broader impacts of health care interventions by measuring different value elements. Value elements can be divided into these buckets:
- Conventional value elements are those studied in randomized controlled trials. Examples include symptoms, safety, quality of life, and life expectancy.
- Broader contextual value elements are those relevant to all members of society. (This is sometimes referred to as the “plan member perspective.”) Examples include rarity and severity of disease, fear of contagion, and broader economic impact.
- Patient experience value elements are those particularly important to patients and caregivers who are directly impacted by the disease. Examples include impact of an illness on family/caregivers, ability to reach important personal milestones, and patient/family financial burden.
Value assessments of health care interventions can inform decisions made by patients and providers as well as insurance coverage and reimbursement. To ensure health care decisions are being guided by the best possible evidence, the PhRMA Foundation funds investigator-driven research that seeks to improve methodological approaches for value assessment and better incorporate diverse stakeholder perspectives and elements of value.
The Center for Pharmaceutical Value (pValue), funded by the PhRMA Foundation and the University of Colorado’s Data Science to Patient Value Initiative and headquartered in the CU Anschutz Medical Campus, is conducting research that examines methods for incorporating nontraditional value elements that are important to patients, payers, clinicians, or other health care stakeholders.
Currently, there is a lack of consensus and consistency across value frameworks regarding which value elements should be included and how they should inform decision-making. In September, pValue held a workshop seeking to better understand how much weight should be given to broader contextual value elements in value assessment practice in the United States. (The pValue team is also working on advancing the integration of patient experience value elements.)
The workshop brought together variety of stakeholders including patient advocates, expert researchers, industry representatives, and health plan representatives, with the goal of identifying and prioritizing broader contextual value elements. Participants discussed and weighted the importance of several broader contextual value elements in value assessment, including the impact of a health intervention on outcomes for patients with severe disease, patients with a rare disease, or patients in disadvantaged and vulnerable populations.
pValue Director R. Brett McQueen, PhD, assistant professor at the CU’s Skaggs School of Pharmacy and Pharmaceutical Sciences, explained that his team hoped to take the perspectives gathered at the workshop to develop supplemental decision aids to help prioritize these value elements and advance the application of value assessment.
McQueen said pValue seeks to create a common starting point — a guide to facilitate structured deliberation for value assessment frameworks to help prioritize discussions around broader value elements with the greatest weight. Such a tool could help increase the consistency in value assessment application but also allow for flexibility in situation-specific contexts and adaptability over time.
“Innovators developing new health care interventions need some level of predictability in what value elements and data they need to capture in their studies so that we have the evidence we need later on to inform evidence-based deliberations and analyses for value assessment.” McQueen said.
This pValue research exemplifies the type of investigator-led, solutions-oriented projects that the PhRMA Foundation funds to address challenges in the evaluation of medicines and other health care interventions. The Foundation offers predoctoral and postdoctoral fellowships and research awards for early- and mid-career faculty conducting research in value assessment and health outcomes research. Researchers interested in applying for these awards should visit our webpage here.