Brandy E. Fureman, PhD; Kathleen M. Farrell, MB BCh BAO; Alison Kukla, MPH; Jeffrey Buchhalter, MD, PhD; Epilepsy Foundation, Bowie, MD USA

 

SUMMARY

In the Epilepsy Learning Healthcare System, people with epilepsy and their care partners, community organizations, clinicians, researchers, and health system leaders work together. They design, implement, and share the results of collaborative research and quality improvement efforts, leading to better health outcomes and increased quality, experience, and value in care. In this article, the Epilepsy Learning Healthcare System demonstrates how coproduction can dramatically accelerate the ability to generate new knowledge and put it into practice from its work in planning the creation, validation, and implementation of a health-related quality of life for rare epilepsy populations.

 

What Is True Healthcare Value?

Value in healthcare has been defined as “quality of care ÷ costs,” with several components of quality that include clinical outcomes and the patient experience. Perhaps the important question about this definition is, “who determines what is an important outcome?” If the decision doesn’t incorporate the perspectives of the person with the condition and their care partners (if needed) into criteria for outcomes, the value equation fails to protect the interests of the most vulnerable of stakeholders in the healthcare complex: the patient. 

Patient-centered care has emerged as a dominant care philosophy placing the patient and family at the center of all decisions. Traditional healthcare models relied on the expertise of physicians to make appropriate decisions about diagnosis and treatment of their patients. “Shared decision making” is a means of incorporating patient preference and is an improvement towards patient-centered care. However, a limitation of the typical model is that shared decision making occurs at the treatment decision and doesn’t incorporate patient input into which outcomes are prioritized.

Likewise, the value-based approach to care has not yet incorporated “patient-centered” outcomes. In large part, the elements of the value equation are currently payer focused rather than patient focused. Incorporating patient preferences and priorities into the selection of outcome measures—both in clinical trials and in real-world evaluations of care—will better align the value equation with the philosophy of patient-centered care.¹

 

How Can Patient Preferences and Priorities Be Integrated Into the Development and Delivery of Improved Care?

The answer to this question is “coproduction.” Coproduction is a process of working together among people with epilepsy, care partners, healthcare providers, and community service providers to design a health system that optimizes the health outcomes that are most important to the affected person, in addition to recognizing the resources required. Coproduction means being involved in decision making about which outcomes are to be improved. Coproduction ensures the patient will have options presented that are most relevant from their perspective.

An example of meaningful coproduction comes from our experience in designing a learning healthcare system for people with the epilepsies. In 2019, the Epilepsy Foundation worked with multiple partners to launch the Epilepsy Learning Healthcare System (ELHS) to improve the quality of care and outcomes for people of all ages diagnosed with one of the epilepsies. Learning healthcare systems ensure that clinical care, science, informatics, incentives, and culture are aligned for continuous improvement, innovation, and research.²

 

"A limitation of the typical model is that shared decision making occurs at the treatment decision and doesn’t incorporate patient input into which outcomes are prioritized."

 

The epilepsies affect 1.2% of the population.³ Arriving at the correct epilepsy diagnosis is sometimes a complex process, often requiring extensive history, electroencephalogram monitoring, neuroimaging, and other diagnostic testing. Most forms of epilepsy require long-term follow-up and expensive treatments. Adverse effects of therapy are common and impair quality of life (QoL). Epilepsy is associated with high rates of comorbidities (eg, depression, anxiety, cognitive deficits) and excess premature mortality (eg, accidents, sudden unexpected death in epilepsy, suicide). Health disparities in epilepsy outcomes have been identified, driven in part by social determinants of health.⁴ Direct costs (medications, testing, emergency room visits, hospitalizations) and indirect costs (under/unemployment, lost productivity of patients, and caregivers) are high.⁵ Despite the clear negative impact of epilepsy on both QoL and health costs, few studies have addressed the value of epilepsy care, especially studies with purposeful inclusion of patient perspectives on value. In treatment trials for the epilepsies, the primary clinical outcome measure is nearly always a measure of seizure control,^2-6 operationalized as a 50% responder rate (eg, the percent of participants who had a 50% or more reduction in seizure frequency). Reductions in seizure frequency of this size have been traditionally accepted as a clinically meaningful difference in outcome, regardless of the specific population for the indication. However, rare epilepsies are characterized by much more frequent seizures, in which a 50% reduction in seizures could be of limited clinical significance. Counting seizures provides a quantitative measure of an intervention’s impact, which is a critical consideration for regulatory approval.

However, while seizures are the defining feature of the epilepsies, the broad spectrum of developmental impacts, QoL, comorbid conditions, and adverse effects of treatment are cited by patient and family partners (PFPs) as much as, or more important than, the number of seizures remaining.^6,7 For example, extensive international consultation with patients with Dravet syndrome and their caregivers has found that across cultures, families and patients have identified seizures as an important outcome, but view as equal impacts of the condition the effects on motor skills, expressive and receptive communication, learning, attention, emotional well-being, community functioning, daily activities, and sleep.^8,9 Similar themes have been reported by parents of children with other severe early life epilepsies.⁶ Measures of seizure frequency or severity alone do not capture the totality of experience for children or families living with the rare epilepsies. Clearly, evaluations of value in epilepsy must go beyond the number of seizures to incorporate more global measures of functional impact that are meaningful to patients.

 

Integrating Patient-Centered Outcomes in ELHS

In ELHS, we seek to coproduce the improvement objectives with patient and family partners who represent the diverse spectrum of people affected by the epilepsies. The unanimous message from PFPs was that measures of QoL were equally or more important than seizure frequency, which had been the measure prioritized by clinicians and researchers earlier in the process. Without this coproduction process, ELHS would have failed to appropriately prioritize an outcome that mattered as much as seizure control from the view of patients and families. 

In addition, we needed to identify reliable methods to measure QoL for everyone in our diverse population. Existing validated QoL scales, such as the PROMIS measures and the epilepsy-specific Quality of Life in Epilepsy–10 (QOLIE-10), can be employed with neurotypical adults and adolescents, and the Epilepsy-PedsQL can be used with neurotypical children (or children and adults who are severely developmentally delayed, as often occurs in many rare epilepsy syndromes); however, there are no validated scales that appropriately measure QoL.

The QOLIE-10 was developed to serve as a brief survey of health-related QoL (HRQoL) for adults with epilepsy. Ten questions are completed by the person who has epilepsy. The QOLIE-10 covers general and epilepsy-specific domains grouped into (1) epilepsy effects (memory, physical effects, and mental effects of medication), (2) mental health (energy, depression, overall QoL), and (3) role functioning (seizure worry, work, driving, social limits).¹⁰ The QOLIE-10 can be completed by a patient and reviewed by the physician within the timeframe of a visit. The scoring rubric allows for different weights to be attributed to different domains according to the patient’s individual prioritization of those domains.

For neurotypical children, youth, and young adults (2-25 years), the PedsQL-Epilepsy Module provides a validated measure of HRQoL that can be used in the clinic. The PedsQL Epilepsy Module is a 29-item measure with 5 subscales (ie, impact, cognitive, sleep, executive functioning, and mood/behavior) with parallel child and caregiver reports.¹¹ However, the development and validation samples for the PedsQL Epilepsy Module included only a small portion of individuals with rare epilepsies and intellectual disability, precluding differentiation of items and subgroup analyses for intellectual disability.

 

"Coproduction is the answer to the question, 'how do we unlock true value in healthcare?'”

 

For children and adults who are severely developmentally delayed, we are actively collaborating with international experts in QoL in relation to intellectual disability¹² and epilepsy¹¹ to develop and validate such a HRQoL scale for this most vulnerable population. This will be the first measure that can be used across the developmental spectrum (from 2-40 years of age) with parallel self- and caregiver proxy reports for patients with rare or severe epilepsies. The need for a caregiver report is especially important when children are too young or cognitively unable to complete measures, and for older individuals who are unable to complete questionnaires. We will use a coproduction approach across ELHS, rare epilepsy patient organizations (collectively known as the Rare Epilepsy Network), and industry partners and regulators (members of the Research Roundtable in Epilepsy) to collaboratively develop a PedsQL Rare Epilepsy Module. This innovative approach will require coordinated engagement and interaction between a consortium of every stakeholder group (ie, rare patients and families, rare epilepsy organizations, clinical trialists and healthcare providers, experts in outcome assessment, drug and device company sponsors, and regulators).

We will identify items for inclusion in a rare epilepsy HRQoL instrument by conducting extensive interviews with caregivers and affected individuals; confirm content validity using cognitive interviewing techniques and consulting with expert consortium members; and field test the HRQoL instrument with ~250 to 300 individuals ages 2 to 40 years with rare epilepsies and/or their caregivers, thereafter, conducting psychometric evaluations. We hypothesize that evaluation will yield a robust measure of HRQoL including internally consistent factors, the ability to detect differences between important clinical groups (eg, motor abilities, seizure severity, presence of comorbidities), and sensitive and reliable minimally clinically important difference values and clinical cut-off scores. Our work in this area will validate a measure for epilepsy-specific HRQoL for future clinical trials and clinical assessments of value of therapeutics to reduce severe and treatment-resistant epilepsy in a population for whom a validated HRQoL is not yet available.

 

Conclusion

In conclusion, we propose that integrating the appropriate QoL assessment into the standard of care for the epilepsies through improvement methodology will unlock true value in ELHS. Value assessment in a learning healthcare system that uses outcome measures that were identified by patient and family partners and ensures that those measures are both appropriate for the populations to be assessed and (ideally) individually weighted toward the patient/family partners, preferences for outcome becomes a truly “patient-centered value assessment” methodology. Such patient-centered assessments, whether carried out during clinical development as part of clinical trial endpoints or during clinical management/treatment decisions to improve the true value of epilepsy care, are sensitive to patient priorities in a way that seizure frequency outcomes alone are not.

We believe that coproduction, embedded within a learning healthcare system framework, is an essential and innovative approach to consistently and reliably incorporate patient-centered outcomes in health decision making and assessment of value. Coproduction is the answer to the question, “how do we unlock true value in healthcare?”

 

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