Pharmatelligence Ltd

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Craig Currie - Chief Investigator - Pharmatelligence LimitedEllen Berni - Corresponding Applicant - Pharmatelligence LimitedDavid Heaton - Collaborator - OPEN VIE ( Harvey Walsh Ltd )Matthew O'Connell - Collaborator - OPEN VIE ( Harvey Walsh Ltd )Michael Wallington - Collaborator - OPEN VIE ( Harvey Walsh Ltd )Rhiannon Thomason - Collaborator - OPEN VIE ( Harvey Walsh Ltd )Tracey Ellison - Collaborator - OPEN VIE ( Harvey Walsh Ltd )

CPRD888

Metachromatic leukodystrophy (MLD) is a rare, life-threatening inherited disorder. Patients experience a progressive deterioration in their neurological and motor function. Little information is available on the healthcare of the group of people with this condition. This study will describe the health and healthcare use of the population with this disease, before and after diagnosis, to better understand their clinical needs. The cost of their healthcare use will be estimated. General practice and hospital in- and out-patient and accident and emergency (A&E) healthcare use will be included. In addition, age at diagnosis, sex distribution, and number with siblings with the diagnosis, will be reported. The average survival from birth and from diagnosis, and the distribution of cause of death will be reported across the population.

MLD is a rare genetic disorder which affects the nervous system and shortens life-expectancy. The study will describe a population of patients with an MLD diagnosis recorded on CPRD. The study aims to describe the annual healthcare resource utilisation and costs associated with management of patients with MLD using CPRD (Aurum and Gold) for primary care resources, and HES inpatient, outpatient and A&E data for secondary care resources. Primary care resource analyses will include consultations, prescriptions and laboratory and diagnostic tests. Secondary care resource utilisation will include inpatient (including total admitted time, admissions/bed days), outpatient appointments and A&E. Speciality and elective/non-elective admissions and those to Intensive care and high dependency units, will be reported. Rates will be reported by year pre- and post-diagnosis and by year from diagnosis (including mean, standard deviation/ median, interquartile range, and range). Costing will be based on HES generated Health Resource Group (HRG), including inpatient, outpatient, and A&E tariff costs, and Personal Social Services Research Unit reference costs and the British National Formulary.

05/01/2021

Release