Long-term Follow-up of a Multicentre, Non-interventional, Prospective Cohort of Participants Prescribed Epidyolex in France in a Real-life Setting

Retention Rate Percentage After Initiation of Treatment With Epidyolex

Time frame: Up to 24 months post-dose.

Retention rate will be measured through assessing the frequency of seizures and the length of time on treatment before a seizure event.

Percent Change From Baseline in Seizure Frequency (Average Per 28 Days)

Time frame: Up to 24 months post-dose.

50% Responder Rate

Time frame: Up to 24 months post-dose.

75% Responder Rate

Time frame: Up to 24 months post-dose.

100% Responder (Seizure Freedom) Rate

Time frame: Up to 24 months post-dose.

Number of Seizure-Free Days (Average Per 28 Days)

Time frame: Up to 24 months post-dose.

Longest Duration of Seizure-Free Days in the Last 28 Days

Time frame: Up to 24 months post-dose.

Change From Baseline in Behavior Rating Inventory of Executive Function, Preschool Version (BRIEF-P) Score in Participants Aged ≤5 Years

Time frame: Baseline; Up to 24 months post-dose.

The BRIEF-P is a 63-item questionnaire which consists of a single Rating Form used by parents, teachers, and day care providers to assess a child's executive functions within the context of their everyday environments--home and preschool. High scores obtained on the BRIEF-P suggest a higher level of dysfunction in a specific domain of executive functions. The BRIEF rating scores is an ordinal set of a technically summarized degree of disorder in functionality: Never = 1 (Minimum), Sometimes = 2 and Often = 3 (Maximum).

Change From Baseline in Behavior Rating Inventory of Executive Function (BRIEF) Score in Participants Aged 6 to 18 Years

Time frame: Baseline; Up to 24 months post-dose.

BRIEF is an 86-item questionnaire used to assess the executive function behaviors at home and at school for children and adolescents ages 5-18. High scores obtained on the BRIEF suggest a higher level of dysfunction in a specific domain of executive functions. The BRIEF rating scores is an ordinal set of a technically summarized degree of disorder in functionality: Never = 1 (Minimum), Sometimes = 2 and Often = 3 (Maximum).

Change From Baseline in Behavior Rating Inventory of Executive Function, Adult Version (BRIEF- A) Score in Participants Aged >18 Years

Time frame: Baseline; Up to 24 months post-dose.

BRIEF-A is a patient-reported scale to measure various aspects of adult executive functioning and self-regulation in the person's everyday environment. It is a 75-item questionnaire that participants rate on a 3-point Likert scale (1 = behavior is never observed to 3 = behavior is often observed). Higher scores indicate greater impairment in executive functioning.

Modal Dose of Epidyolex Administered to Participants

Time frame: Up to 24 months post-dose.

Maximum Dose of Epidyolex Administered to Participants

Time frame: Up to 24 months post-dose.

Change from Baseline in Average Daily Dosage of Concomitant Anti-Epileptic Drugs (AEDs) and Other Medicines

Time frame: Up to 24 months post-dose.

Use of Rescue Medication Per Month

Time frame: Up to 24 months post-dose.

A month is defined as 28 days.

Physician Global Clinical Impression of Change (PGIC) Score

Time frame: Up to 24 months post-dose.

The PGIC comprises the following question: "Please assess the change in the patient's general functional abilities since enrollment," scored on a seven-point scale from 1 (Very Much Improved) to 7 (Very Much Worse). Lower scores on the scale indicate a better quality of life.

Caregiver Global Impression of Change (CGIC) Score

Time frame: Up to 24 months post-dose.

The CGIC comprises of the following question: "Since your child started treatment, please assess the status of your child's overall condition (comparing their condition now to their condition before treatment)," scored on a seven-point scale from 1 (Very Much Improved) to 7 (Very Much Worse). Lower scores on the scale indicate a better quality of life.

Change from Baseline in Health Utility Index Mark II (HUI-2) Score

Time frame: Up to 24 months post-dose.

The HUI-2 is a generic measure of health status and health-related quality of life. There are 15 questions in the questionnaire with 1-week recall completed by the caregiver. Answers to the questionnaire are mapped into a classification system of 7 sets of utility scores (sensation, mobility, emotion, cognition, self-care, pain, and fertility) with a scoring scale of a minimum score of 0.00 (lack of functional capacity or most disabled) to a maximum of 1.00 (full function or no disability). A higher score indicates a better outcome.