Arthritis Foundation Principles on Patient-Centered Value Assessment

Introducing the Issue

The concept of assessing value for health care treatments has been in place for decades but has gained increased attention in recent years — as U.S. policymakers seek ways to reduce health care costs.

The Arthritis Foundation has long advocated for a patient-centered approach to value assessment that incorporates and prioritizes patient preferences and quality-of-life goals.

Multiple value assessments have been performed on rheumatoid arthritis (RA) drugs in recent years, prompting the Arthritis Foundation to publicly weigh in on the importance of patient engagement and patient data as a core component of the process.

Assessments of treatments for other forms of arthritis, including osteoarthritis (OA), may focus on procedures like joint replacements. Similarly, patient engagement is essential for incorporating patient treatment preferences and goals for post-surgical health outcomes.

Increasingly, value assessments are used by payers to help determine coverage and formulary decisions. Patient experiences, preferences, goals, benefit-risk tolerance and other factors must be incorporated into decisions, directly impacting patients’ access to care.

The patient experience must be considered holistically.

• Many people with arthritis have co-morbidities that impact their treatment choices and care. And the lifetime considerations of health costs and outcomes cannot be measured in isolated episodes or short-term windows of time.
• A good example comes from 2016 data as part of an RA drug review by the Institute for Clinical and Economic Review (ICER).
  • Survey responses showed that patients on average had to try between 2 and 3 drugs before finding one that worked for their disease. And often patients found the drug to be less effective over time, prompting an additional round of treatment changes.
  • Anecdotal evidence sheds light on what happens when patients have disruptions in their treatment: symptoms often worsen, leading to the need for further intervention and treatment, and sometimes hospitalization.
  • This data was critical to inform the ICER review process and led to recommendations in the final report about the inappropriate nature of step therapy in some cases.

 

Another successful patient engagement example came from a revision to the Innovation and Value Initiative (IVI) RA value model.

• Here, IVI conducted focus groups of patients to understand their patient experiences and considerations in seeking treatment.
• Listening to the patient journey helped model developers better understand how factors, such as hormonal changes or pregnancy, can impact treatment efficacy and adherence, which are important considerations.
• The results were published in a joint white paper in 2021 which offers a valuable template for other stakeholders seeking to better engage patients.  

The below principles are informed by these examples, in addition to myriad Arthritis Foundation patient surveys, focus groups, interviews and polls.

Principles for Patient-Centered Value Assessment

The objective of patient-centered value assessment is to inform health care decision making to include the goals and preferred outcomes of the patient. It considers all the details in a patient’s life that impact their care.

• A 2018 National Health Council dialogue, titled “Overcoming Barriers to Amplify the Patient Voice,” captured it well: Patient-centered value assessment exists when patients have been engaged, heard, understood and respected throughout the entire process, and their input is incorporated and guides decision making.
• To inform our principles on “value in health care,” we asked a select group of patient advocates what “value-based care” means to them, and what their primary health care goals include.

The diversity of responses reflects the diversity of the disease impact and of the patient experience. This highlights the importance of meaningful patient engagement in assessing value for any health care treatment.                     

 

Principles that policymakers and other stakeholders should consider in assessing value from this lens include:

1. Patient-Centered Methodologies.

• A widely used approach for estimating quality and quantity of life in economic mode is calculating Quality Adjusted Life Years (QALYs). QALYs may contribute to informing a value assessment. However, data inputs used to calculate QALYs do not holistically reflect patient experiences, preferences, goals and benefit-risk tolerance.

Current approaches to calculating QALYs often rely on generic questionnaires, which may not reflect health-related quality of life as defined by arthritis patients, nor where patients are in their disease or treatment journey.Further, QALYs can be discriminatory by placing a lower value on treatments that extend the lives of people living with disabilities and chronic conditions. Economic models calculated using QALYs should only be used in combination with other value assessment methods and should only play a partial role in the comprehensive assessment of treatments. Instead of using a QALY-only value assessment model, we would suggest the following:

• Value assessments must use multiple additional criteria and methods to account for patient preferences, goals and experiences.
• Value assessors and others who utilize QALYs should improve the way in which they use QALYs, ensuring that surveys are disease-specific and given at intervals that are most appropriate for that particular disease. Survey tools should be fit-for-purpose such that policymakers assessing arthritis treatments can evaluate:
  • Was the tool appropriate for arthritis?
  • Did it have questions related to the disease?
  • Did it consider validated joint-specific measurement tools?
• State and federal governments should incentivize state academic institutions to invest in patient-centered comparative effectiveness and cost-effectiveness research that moves beyond the QALY and can become standard practice.
• As new methods evolve, value assessors should be transparent about the limitations of specific models.

2. Real-World Evidence.

• Clinical trial data is insufficient to capture the heterogeneity of disease, market access factors and other environmental factors crucial for understanding the impact of the treatment on the disease population.
• Value assessments cannot fully incorporate all necessary and relevant data to be truly patient-centered until the treatment being assessed is on market. Value assessments should be updated regularly to take into account cost and formulary data, patient-reported outcomes data and any other real-world data that would inform true cost-effectiveness.

3. Comprehensive Claims Data.

•  All payer claims databases (APCD) are large state databases that include information, such as medical claims and pharmacy claims collected from public and private payers. Robust APCDs can help inform value assessment analyses by providing data across sites of care and longitudinally about patients, allowing value assessors to identify trends and patterns in health care costs and better tailor coverage and cost decisions.
• State legislatures should prioritize funding APCDs and using best practices from states that have utilized APCDs to help inform cost landscapes.

4. Transparency.

• Transparency across the health care ecosystem — from manufacturers to payers, pharmacy benefit managers and value assessors — is essential for implementing patient-centered value assessment. Currently, it is difficult to know the full set of processes and factors that contribute to any given value assessment — and importantly how payers and other stakeholders are utilizing them.
• Value assessors should be transparent about their methods and allow sufficient time for public input throughout the process.
• Payers should be transparent about how they are utilizing value assessments in their formulary decisions.
• Value assessors, payers and others should establish a continuous feedback loop with the patient community to inform post-value assessment decision making and any subsequent updates.

5. Meaningful Patient Engagement.

• A truly patient-centered value assessment would engage patients in a meaningful way from start to finish.
  • Patient engagement should never be considered a check-the-box activity. Instead, patients should be equal stakeholders throughout the process, and patient representatives should have voting privileges in any advisory councils or roundtables.
  • However, the value assessment should not be the beginning or end of patient engagement. Patients should be part of the decision-making process during clinical trial design to ensure manufacturers are measuring endpoints that matter to patients.
  • For example, a manufacturer of an osteoarthritis (OA) therapy may promise regeneration of cartilage, when what matters more to a patient may be pain reduction and functional improvement.
  • Likewise, pharmacy benefit managers and payers should include patients in their formulary review processes to ensure they have a robust understanding of the patient experience.
  • For example, detailed data on the impact of step therapy on patient health outcomes can more precisely guide appropriate step therapy protocols, including the number of steps included in a protocol and the appeals process.

 

• Manufacturers should incorporate patient preference data in their clinical trial design and should include patients in the identification of study endpoints.
• Patient representatives should be invited to serve on Pharmacy and Therapeutic (P&T) Committees and other forums that determine formulary coverage decisions.
• Any advisory committee considering cost effectiveness should include robust patient representation, including voting membership and extensive quantitative and qualitative patient data.
• Patient representatives should be invited to craft value assessment methodologies and strategies, including legislative and regulatory processes and value assessment methodology design.

6. Value-based Insurance Design.

• Value-based agreements and other value-based policies can help bridge the gaps in real-world value-based care. There are many examples of value-based care models in rheumatology, orthopedics and other arthritis-related specialties to draw from — and there are specific ways state and federal policymakers can promote value-based policies.  
  • States should incentivize Medicaid programs and state payers to use patient-centered value assessment and consider value-based agreements.
  • State reinsurance programs can be a good tool for ensuring better access in a way that reduces health care costs.
  • The Center for Medicaid and Medicare Innovation (CMMI) and other federal agencies should incorporate patient-centered value principles into their models.
  • Federal policymakers should reduce legal and administrative barriers to value-based agreements.

 

How policymakers and stakeholders can use these principles

• State legislators and stakeholders can
  • Utilize Prescription Drug Affordability Board (PDAB) legislation or other drug pricing-related legislation
  • Utilize model legislation to innovate around value-based care within state institutions and payers
  • Encourage development of additional patient-centered value assessment tools to properly inform decision making across the board
• Federal policymakers and stakeholders can:
  • Utilize these principles in the design of value models and demonstration projects
  • Fund research through federal agencies on best practices for using cost effectiveness to inform benefits and coverage decisions in insurance programs
  • Avoid creating bill provisions requiring agencies to cover only the most cost-effective drugs/treatments
    • Or require agencies to impose restrictions on less cost-effective treatments
  • Issue guidance to clarify that the ADA applies to state programs, including Medicaid, and clarify authority to HHS sub-agencies that QALYs should not be relied on for benefits/reimbursement decisions.
  • Issue guidance on what to do if a patient’s health insurance refuses to cover recommended treatment due to cost effectiveness
  • Use well-established alternatives to QALYs or cost-benefit analyses in combination, when considering cost effectiveness as a component of a condition-specific multi-criteria decision analysis