REIMAGINING HEALTH CARE VALUE ASSESSMENT

The Patient-Driven Values in Healthcare Evaluation Center of Excellence puts the patient first

By Christianna McCausland

The conventional model of value assessment used to facilitate health care decision-making has traditionally neglected to capture the patient perspective. While a treatment might be an effective therapeutic, it may also cause the patient to miss many days of work due to side effects, for example. These patient-informed factors have generally played a minor role in assessing the value of a treatment — if taken into account at all.

The School of Pharmacy’s Patient-Driven Values in Healthcare Evaluation (PAVE) Center of Excellence, a collaboration with the National Health Council, is bringing together the patient community and payer and industry leaders to put the patient perspective front and center in health care decision-making.

PAVE is more than a clever acronym; it represents a revolutionary way of thinking about this issue.

“As the PAVE Center, we are trying to ‘pave’ a new pathway for better understanding the value of health care interventions, and that means bridging two pathways of research and integrating information in a way that’s not been done before,” explains Susan dosReis, PhD ’99, BSPharm, a professor in the Department of Pharmaceutical Health Services Research (PHSR) and director of the PAVE Center, which was established in 2018 through a $500,000 grant from the Pharmaceutical Research and Manufacturers of America (PhRMA) Foundation.

“Understanding patient preferences and value assessment of health care interventions have typically been two separate lines of work and research,” she continues. “We’re trying to break new ground with empirical data to show how we can integrate these two and provide a more nuanced understanding of health care value. We do this by incorporating novel information about what’s important to patients that is not typically included in a traditional evaluation.”

One PAVE research study, for example, examined chronic obstructive pulmonary disease (COPD). Research showed that medication frequency is important to patients. If patients could take medication fewer times per day, they would have more flexibility to engage in life activities. Yet this aspect of treatment is not measured in COPD clinical trials, which focus more on outcomes like exacerbations.

Julia Slejko, PhD, is an associate professor of PHSR and codirector of the PAVE Center. Her work looks closely at the economics of health care decision-making. She explains that traditionally, when

Julia Slejko and Susan dosReis

pharmaceuticals or health care products have been evaluated, the value assessment has been based on clinical outcomes.

“Increasingly, new treatments are improving things for patients beyond what’s seen in clinical trials,” says Slejko. “So there’s a disconnect between the trial outcomes and things that are important to patients.”

Much of the research at PAVE is aimed at uncovering what patient-informed factors are missed in value assessment. PAVE developed a conceptual model of patient-informed value elements that includes five domains and 42 separate elements. These include social, economic, and quality of life impacts that are important to patients.

Another PAVE project analyzed hepatitis C virus (HCV) treatment and found that most HCV assessments focus solely on the cure. Yet patients with HCV expressed socioeconomic and psychological concerns, like how many workdays they miss and their ability to infect others, concerns that were not taken into account in the assessment of treatment. Based on this, a novel, more patient-centered methodology was developed in collaboration with a stakeholder advisory board, including HCV patients and the American Liver Foundation. Working with patients and patient organizations is a hallmark of PAVE’s approach.

“We go to patients who are living with the health conditions as the first step to help us discern how much a treatment decision relies on the effects of the treatment, the cost of the treatment, access to treatment, and the impact the treatment can have on their lives and social well-being,” says dosReis. “We listen to what patients tell us is important to isolate the key factors that are pivot points in decision-making and trade-offs people make.”

A significant active research project at PAVE is looking at major depressive disorder (MDD). Value assessments have resulted in a one-size-fits-all modality and that often leaves those with MDD with poor quality of life and high health care costs. PAVE’s research indicates that the amount of time it takes a patient to feel a treatment’s beneficial effect and their ability to remain hopeful are important factors to people with MDD, two factors that often are not taken into account in a traditional value assessment.

Put simply, many patients utilize treatment but do not actually feel better and are not able to engage in their lives in a healthy, productive way. PAVE’s research is working through a stakeholder-engaged process to identify the value elements important to patients and incorporate the information into a more informed, tailored economic model for MDD.

In addition to its research, PAVE is engaged in education both for professionals and patients. Education can correct myths and misconceptions about the impact patient input can have in value assessment. Particularly among payer groups, there is a concern that patients will naturally want the Continued on Page 16

THE PILLARS THE PILLARS OF PAVE OF PAVE

PAVE’s mission is to ensure patient engagement and perspective are included in the discussion of treatment value, something conventional models often lack. PAVE’s approach is grounded in three pillars:

Solicit meaningful input from patients on their preferences

QUANTIFY Create quantifiable measures of patient preferences

TRANSLATE: Apply empirical data into value assessment models

ELICIT

QUANTIFY

TRANSLATE

Continued from Page 15 most expensive treatment available. PAVE’s research indicates that is not always the case. What patients want are more tailored assessment models that conceive of value in a more sophisticated way. What PAVE provides is the empirical data to support this new approach. Director dosReis would like to expand PAVE’s work into health care policy. Although their research looks at this topic on a disease-specific basis, she sees potential and interest in applying this approach more widely to policy. There is also opportunity to work with patient groups to better understand how patient-driven values impact outcomes and how the patient experience changes — and treatment preferences change — over the course of a disease state. Co-director Slejko says this is a dynamic time in the field, one that is spawning productive collaborations and important data.

“The next steps will involve translating the information we’ve learned into our evaluations in a way that can be quantified,” says Slejko. “Currently, evaluations do mention what are sometimes called ‘contextual considerations,’ but they were rarely able to be quantified. That is what is exciting for everyone in this field.”

PAVE is establishing a patient-centered approach that can be adopted by researchers conducting assessments but also consistent with goals of decision-makers who use their results. This work can potentially have a long-term impact on how we elicit, quantify, and translate patient perspectives into future value assessments. This has the potential for downstream effects on access to and reimbursement for pharmaceutical products that can improve outcomes that matter most to patients. b

SOCIAL IMPACT

SOCIAL WELL-BEING

• Support Network • Relationship with Family • Relationship with Peers • Maintain Social Activities

PERSONAL VALUES

• Cultural Barriers • Religious Beliefs

SHORT- AND LONG-TERM TREATMENT EFFECTS

TOLERABILITY

• Medication Frequency • Length of Treatment • Side Effects

DISEASE BURDEN

• Age of Onset • Symptom Importance • Intermediate/Surrogate

Outcomes • Forecasting

IMPACT ON EDUCATION

• Impact on Career • Predictable Health

Care Needs

PATIENTINFORMED VALUE ELEMENTS

TREATMENT ACCESS

ACCESSIBILITY OF CARE/ TREATMENT

• New Therapeutic Option • Available Treatment • Provider Willing to Deliver Care • Proximity to Care Location • Appropriateness of Care • System Navigation

HEALTH CARE SERVICE DELIVERY

• Provider Relationship & Trust • Care Transitions • Consistency of Care • Explanation of Treatment (Risks & Benefits)

LIFE IMPACT

PERSONAL WELL-BEING

• Fatigue • Ability to Work • Physical Abilities • Emotional Status

STIGMA

• Embarrassment/Self-Consciousness • Rejection by Family • Rejection by Society

TREATMENT COSTS

COSTS INCURRED BY THE PATIENT

• Affordability • Cost of Treatment-Related Side Effects • Long-Term Costs • Reimbursed Care

COSTS INCURRED BY THE FAMILY

• Sibling Costs • Long-Term Effects on the Family • Relocation Costs • Autonomy/Dependence