Prioritizing Factors in U.S. Value Assessment From a General Citizen Perspective 

With the implementation of Medicare drug price negotiation under the Inflation Reduction Act and the establishment of prescription drug affordability boards in some states, value assessment is likely to play an increasingly important role in patient access to medicines.

Value assessment research seeks to define and quantify the value of health care interventions like drugs by analyzing evidence about their benefits, risks, and costs. When used by health care decision-makers, value assessments can directly impact patient care, so it’s critical that they consider a variety of value elements important to stakeholders including patients, clinicians, and society broadly.

Value elements can include conventional factors like treatment safety and impact on patient quality of life, patient-centric factors like impact on caregivers and patient financial burden, as well as additional broader factors like disease severity and societal economic impact. However, these value elements are not all equally important concepts and stakeholders may prioritize different elements.

The PhRMA Foundation-funded Center for Pharmaceutical Value (pValue), headquartered at the University of Colorado Anschutz Medical Campus, brought together a group of 28 stakeholders including patient advocates, expert researchers, industry representatives, and health plan representatives to discuss and prioritize broader value elements from a U.S. general citizen perspective (also referred to as health insurance plan member or taxpayer perspective, as opposed to a personal/patient perspective).

The study results, recently published in Value in Health, showed that the multistakeholder group weighted the importance of additional value elements as follows:

• Severity of the disease (26%): The health improvements generated by the intervention are indicated for patients with severe disease (e.g., reduced length of life and/or quality of life).
• Disadvantaged and vulnerable target populations highly represented (22%): The intervention targets disadvantaged and vulnerable populations and improves their health more than other populations.
• Broader economic impact (17%): The intervention reduces the broader negative economic impact of the disease (i.e., on tax, education, legal, and environment system) considered from the perspective of the society.
• Risk protection (14%): The availability of the intervention reduces risk of disease and its unfavorable physical and financial consequences.
• Rarity of the disease (11%): The intervention targets those with rare (or ultra-rare) disease and improves outcomes as compared to existing alternatives.
• Scientific spillover of novel mechanism of action (10%): The intervention uses a new mechanism of action that could have positive spillover effects in other clinical areas.

“These results, in combination with future evidence in samples of general citizens, could inform health care policy decision-making by signaling stakeholder priorities to value assessors, manufacturers, and reimbursement decision-makers,” said pValue Director R. Brett McQueen, PhD, assistant professor at the CU’s Skaggs School of Pharmacy and Pharmaceutical Sciences.

Oftentimes, value assessments are limited by a lack of information, especially if they rely only on data collected during regulatory approval studies. “These findings could help manufacturers with efficient allocation of R&D funds by allowing them to focus on research and evidence generation for the highest priority value elements,” McQueen said.

In addition, the study demonstrates a pragmatic, structured approach for prioritizing value elements beyond the ones included in this research, the authors stated. This approach is already helping to improve the consistency and predictability of what’s important to stakeholders in Colorado prescription drug policy.

Read the study in Value in Health.