Identifying key unmet needs and value drivers in the treatment of focal-onset seizures (FOS) in patients with drug-resistant epilepsy (DRE) in Spain through Multi-Criteria Decision Analysis (MCDA)

Abstract

Introduction: Epilepsy is a serious neurological disease, ranking high in the top causes of disability. Approximately 40% of patients with epilepsy are pharmacoresistant after their seizures failed at least two antiseizure medications (ASMs). Adult patients experiencing focal-onset seizures (FOS) account for approximately 60% of all patients with epilepsy and they are more likely to become drug-resistant epilepsy (DRE) than those with generalized onset. Drug-resistant epilepsy is associated with mortality, morbidity, and reduced quality of life. The information available on the clinical management, health outcomes, and unmet needs of the disease within the Spanish healthcare environment is very limited. Multi-Criteria Decision Analysis (MCDA) allows determination of what represents value in a given indication considering all relevant criteria for healthcare decision-making in a transparent and systematic manner and from the perspective of relevant stakeholders. Purpose: The aim of this study was to identify the burden of DRE (clinical, quality of life, and economic) and the unmet needs in Spain and to determine what represents value in the treatment of FOS in DRE patients from the perspective of Spanish epileptologists. Methods: The steps taken to carry out the MCDA were based on previously published good methodological practices. A systematic literature review (combining biomedical databases and gray literature sources) was performed between March and April 2020. Results were reviewed and validated with three epileptologists in June 2020 and used to develop a MCDA value framework, adapted for FOS in DRE, composed of 12 quantitative criteria and 3 contextual criteria. A group of six Spanish epileptologists from four Spanish regions were trained in MCDA methodology before individually validating value criteria (and their definitions based on literature review findings) and assigned relative weights using an ordinal 6points scale. Results were analyzed and discussed in a group meeting through reflective MCDA discussion methodology. Results: Drug-resistant epilepsy is considered a very severe health problem with important unmet needs affecting a considerably sized population. While safety and impact on quality of life of available ASMs are considered adequate, efficacy remains insufficient for patients to achieve seizure freedom and maintain it over time. Hence, the therapeutic benefit of pharmacological treatments currently used is regarded as suboptimal. Drug-resistant epilepsy management is associated with moderate pharmacological, relevant direct medical and high indirect costs. Quality of evidence available for current treatments is moderate. It is considered that DRE does not currently stand as a key priority for the Spanish healthcare system.

Real‐world safety and effectiveness of cenobamate in patients with focal onset seizures: Outcomes from an Expanded Access Program

“…3 Drug-resistant epilepsy is a severe health condition associated with considerable unmet needs. 4 Patients with DRE may experience increased comorbidities, an increased risk of injuries, adverse events (AEs) from treatment and premature death. In addition, DRE may impact patients' employment status and quality of life.…”

Value contribution of blood-based neurofilament light chain as a biomarker in multiple sclerosis using multi-criteria decision analysis

“…Additionally, it functions as a valuable tool for Health Technology Agencies and pharmacotherapeutic committees, facilitating evaluations and decision-making processes. Using MCDA methodology enables a comprehension of the perceived value of a new health technology, achieved through the scoring of the evidence matrix, considering a diverse range of value attributes ( 33–35 ). It is therefore understandable that MCDA methodology is becoming increasingly popular to support healthcare decision-making, particularly in complex cases ( 26–28 , 31 , 32 ).…”

“…A non-hierarchical 5-point scale was used (+5 points = high relative importance; 0 point = no relative importance) for the disease and blood-based NfL-related criteria, except for the cost criteria, where 0 represents the best possible value, and 5 the worst possible value. Qualitative or contextual criteria were evaluated according to whether they represented a positive, neutral, or negative impact for the National Health System ( 33–35 ). The qualitative criteria score was displayed on a numerical scale of −1, 0, and + 1, representing negative, neutral, and positive impacts, respectively.…”

Recommendations for prescribing pharmacologic prevention during emergency department treatment of epileptic seizures

“…A pesar de los nuevos FACE, la epilepsia es farmacorresistente hasta en un 40% de pacientes 32 . Con frecuencia se prescriben dos o más FACE 7 dentro de una politerapia racional 33 .…”