Patient insights help transform lives

Jazz Pharmaceuticals aims to transform the lives of patients and their families by developing innovative, life-changing medicines for people with serious diseases that often have limited or no therapeutic options. Epidiolex® (the only FDA-approved cannabidiol [CBD] product) is approved to treat seizures associated with rare, severe forms of epilepsy: Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex.

Jazz Pharmaceuticals has a history of putting the patient first. The JazzCares patient support programs help with access to treatments and services, and the company gathers feedback about patients’ health journeys directly from patients and their families through  partnerships with patient advocacy groups and other external organizations.

There's growing evidence that cannabidiol reduces seizures and improves quality of life among individuals with epilepsy, but, still, little is known about the real-world use of the Epidiolex®, caregivers’ perceptions about their dependents’ condition after initiating Epidiolex®, and the impact on caregiver burden. Given the rarity of the conditions treated by Epidiolex and the burden of providing care for a chronic, often debilitating disease, Jazz Pharmaceuticals needed a partner who could survey this hard-to-reach population in a convenient, low-friction manner.

Evidation Patient Insights connects you to the right-fit community

Jazz Pharmaceuticals contacted our team to help develop and deploy a survey to better understand caregivers’ perspectives on the real-world implications of dependents’ use of Epidiolex and non-FDA-approved cannabinoid products. 

Using our Patient Insights solution within the Evidation platform, our team then led the scientific design of a one-time, cross-sectional survey co-developed with Jazz Pharmaceuticals.

For recruitment and pre-screening, we leveraged our well-labeled, continuously updated database to identify potentially eligible Evidation Members. Data sources included previous surveys on the Evidation platform such as:

• A single-question epilepsy caregiver survey: “Are you a parent or the main caregiver of a child or adult dependent who is being treated for seizures?” (~10,500 individuals)
• A single-question Epidiolex caregiver survey: “Does the person you’re caring for currently take Epidiolex, a prescription medication used to treat seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC)?” (6,986 individuals)

Analyses were performed on data from the entire cohort and from the following segments to explore differences in burden and experience by:

• Race /ethnicity (underrepresented vs not underrepresented)
• Sex (male vs female)
• Caregiver responsibility (sole caregiver vs shared caregiving responsibilities)

The process took less than six months to complete:

Rapid engagement delivered the insights Jazz Pharmaceuticals needed

Of the 346 Evidation Members who responded to the pre-screening survey, 204 self-identified caregivers (18 years and older) completed the CARE-EpiC survey. Each respondent had to provide care for an individual (age 1 year or older) who was being treated for epilepsy or a seizure condition with Epidiolex.

Key insights

• When seizures are better controlled, the caregiving experience also improves.
• Caregivers require additional support for dependents’ emotional/behavioral care and physically strenuous activities.
• Obtaining an Epidiolex prescription or waiting for insurance approval may be a treatment hurdle for some caregivers.

83% of study participants had wearable activity data from the past year

Caregiver perspectives highlight opportunities to improve the epilepsy treatment paradigm

Caregivers play an important role in epilepsy management. Therefore, characterizing their experience helps prioritize decisions about treatment development, distribution, and support services based on a deeper understanding of what is actually driving patient and caregiver behaviors and outcomes in everyday life. These insights uncover barriers and opportunities to improve access, such as patient programs to offset costs not covered by insurance in this population. Data collected from this representative sample enabled more nuanced segmentations of real-world experiences that can be used to inform caregiver programs for those with the highest burden (male caregivers, sole caregivers, or caregivers from underrepresented race/ethnic communities) and marketing strategies to individuals with epilepsy who have used other non-approved cannabinoid products.

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The project outcomes are also available via the:

American Epilepsy Society (AES) 2024 Annual Meeting

32nd Annual Education Conference held by the Developmental Disabilities Nurses Association (DDNA)