For Patient Organizations

This Resource Guide for patient organizations provides information and research in key areas such as value assessment, heterogeneity and benefit design.

Value Assessment

Many patient organizations are being asked to participate in the value assessment process, and they are often unfamiliar with what that entails. What is it, and what do patient organizations need to know? How can patient preferences and inputs be better incorporated into value assessment?

NPC has conducted research and developed materials to address some of these questions.  

Value Frameworks

Current Landscape: Value Assessment Frameworks
This landscape assessment analyzes seven U.S. value frameworks and compares the strengths and limitations associated with each. Each were analyzed through the lens of six broad categories: development process, measures of benefit, measures of cost, methodology, evidence and assessment process.

Guiding Practices for Patient-Centered Value Assessments
Twenty-eight guiding practices were identified to address six key aspects of value assessments: the assessment process, methodology, benefits, costs, evidence, and dissemination and utilization. Seven guiding practices for budget impact assessment are outlined separately, as budget impact is not a measure of value.

NPC Infographic: How Should Value in Health Care be Assessed?

Patient Preferences and Inputs in Value Assessment

Multi-Criteria Decision Analysis: Can It Help Make Value Assessment More Patient Centered?
This white paper, developed with the National Health Council, identifies good practices and key considerations for integrating the patient voice into value assessment with multi-criteria decision analysis.

Do Patient Preferences Align with Value Frameworks? A Discrete-Choice Experiment of Patients with Breast Cancer
The study assessed patient preferences for aspects of breast-cancer treatments to test an assumption in the most commonly-used oncology value frameworks – that patients view treatment outcomes the same way.

Barriers and Solutions to the Inclusion of Broader Benefits in Biopharmaceutical Value Assessments
This study evaluated how institutional barriers prevent the inclusion of broader individual and societal benefits associated with new health care innovations from being considered when assessing the value of medical treatments and making health care coverage determinations, and potential solutions to those barriers.

Views from Patient Organizations
Patient organizations that have participated in value assessment processes have had very different experiences. In Q&As, the groups share their experiences and recommendations for improving the processes.

Individual Treatment Effects/Heterogeneity

Science has shown that patients living with a specific disease can react differently to the same treatment. Understanding when individual patient differences, or heterogeneity, can have a profound effect on patient care is important, especially with health benefit design and decisions about which treatments will work best for patients.

The Myth of Average: Why Individual Patient Differences Matter
This booklet explores the reasons behind differences in treatment effects and offers a checklist that patients can use when talking to their health care providers.

Variable Co-pays in Pharmacy Benefit Tiers: Ethics and Efficiency
This research identified the ethical, legal, actuarial implications associated with cost-sharing based on formulary tier rather than medical appropriateness for patients. A multi-stakeholder panel identified guiding principles for when it would be more (or less) acceptable to require patients with the same or similar condition to have variable out-of-pocket expenses.

A “Dynamic” Approach to Consumer Cost-Sharing for Prescription Drugs
This issue brief introduced and defined the concept of “rewarding the good soldier,” which refers to the scenario where a patient diligently follows step-therapy requirements but does not respond as desired, and needs a more expensive treatment option. This patient should, therefore, have reduced consumer cost-sharing for that treatment. This brief includes clinical examples, discusses the benefits from a more clinically nuanced cost-sharing approach and proposes next steps to move from cost-focused to value-based initiatives in formulary development.

Health Benefit Design

Health benefit design plays a critical role in patient health, access to care, and larger health system costs. But health benefit design that focuses narrowly on cost containment, without addressing patient needs or increased costs elsewhere, may come with unintended effects.

Stakeholders Find Step Therapy Should Be Flexible, Evidence-based and Transparent: Assessing Appropriateness Using a Consensus Approach
Research published in the Journal of Managed Care & Specialty Pharmacy shows that multiple stakeholders, including payer, provider and patient groups, disagree on when step therapy is appropriate but agree on a set of criteria about how to develop, implement, communicate, safeguard and evaluate step therapy protocols. The majority of these criteria can be achieved today.

Financial Impact of HSA-HDHP Reform to Improve Access to Chronic Disease Management Medications
This report demonstrates that expanding coverage of preventive services under Health Savings Account-eligible high-deductible health plans (HDHPs) could benefit millions of Americans suffering from chronic conditions.