Dravet syndrome (DS) is a rare form of childhood epilepsy that is characterized by multiple, treatment-resistant seizures that subsequently lead to mental impairment, problems with motor skills and behavioral development issues.
Lennox-Gastaut syndrome (LGS) is also a rare and severe form of childhood epilepsy characterized by the presence of multiple seizure types but known for its drop seizures (where patients literally drop to the ground during a seizure), and mental deficiency or learning difficulties.[4-6]
Our objective is to estimate the number of individuals suffering from DS or LGS in the UK. We will also analyse their electronic health records to better understand which treatments are used by patients and when. . This will also include aspects like the use of medications, referrals to specialists and hospitalisations so that we can get the full picture of healthcare used by these patients. We will identify the cost of these resource items to estimate the financial burden associated with the current treatment of patients with DS or LGS.
We will also focus on the mortality of these patients and better understand the causes of death of DS and LGS patients.
Since these two conditions are considered as rare diseases, our findings are expected to inform the total cost of DS and LGS to the healthcare system and will be used in economic models which are used to demonstrate the value of a new drug.
The study objectives are (1) to estimate the prevalence by age groups suffering from DS or LGS; (2) to study treatment pathways; (3) to evaluate the financial burden of medical care of these two conditions; (4) to estimate the mortality rates by age groups and causes of death of DS and LGS patients.
Using the Clinical Practice Research Datalink (CPRD), Hospital Episode Statistics (HES) and Office National Statistics (ONS) Death Registration, we will study the patients with a coded general practitioner (GP) diagnosis of DS and those with a coded diagnosis of LGS. Since these two conditions are difficult to diagnose and can be miscoded by the GP, we will also focus on patients using some specific treatments (from CPRD only as no treatment information is available from HES data)
The main analyses will consist in evaluating the prevalence of the two conditions in adult and children populations. Resource use for both inpatient (number of stays and length of stay, etc.) and outpatient (prescription items, referral visits, etc.) will be estimated for these patients.
Finally, these estimates will be scaled up to the entire population and will be used to evaluate the financial burden of medical care of DS and LGS in the UK.
Prevalence of Dravet syndrome
- Specialist's referrals
- Mortality rates
- Prevalence of Lennox-Gastaut syndrome
- GP Visits
- Causes of Mortality
- Prescription of treatments
- Hospitalisations of patients
Monique Martin - Chief Investigator - Not from an Organisation
Monique Martin - Corresponding Applicant - Not from an Organisation
Florence Bianic - Collaborator - Syneos Health Consulting Inc (formerly inVentiv)
HES Accident and Emergency;HES Admitted Patient Care;HES Outpatient;ONS Death Registration Data;Practice Level Index of Multiple Deprivation