Amyotrophic lateral sclerosis (ALS), also known as motor neurone disease (MND) is a condition affecting the
muscles and nerves of the body. Individuals with ALS lose motor neurons which leads to loss of muscle
mass, strength and function. As ALS/MND progresses, individuals lose the ability to move, speak, eat and
breath. This condition is devastating for those who are affected as it gets worse over time, has a large impact
on quality of life, and is fatal. The average person with ALS or MND is only expected to live for 3 to 5 years
after the first symptom. Currently, there is no cure for ALS/MND and treatment is focused on slowing the
worsening of disease, managing symptoms, and improving the person’s comfort while living with the disease.
Managing the condition requires a range of resources such as visits to the doctor, hospital care, prescription
medications, and devices to help individuals move around (for example, wheelchairs) or breathe. To date,
there is no comprehensive study of the full extent of resource use and costs needed to care for those with
ALS/MND. The available research is based on surveys which may not capture all of the information. The
purpose of this study is to estimate the resources and costs needed to care for those with ALS/MND using a
population-wide database.
To describe and estimate the HCRU (frequency) and costs of outpatient visits, hospitalisations, medications, medical procedures, and assistive devices among adults newly diagnosed with ALS in England. The diseased group consists of those newly diagnosed with ALS based on Read/SNOMED codes and having no encounters with ALS diagnostic codes for 6 months prior to the index date. We will then match cases to controls without ALS diagnosis by age (at
randomly assigned index date), sex, and geographic region in a 1:5 ratio. We will use linked datasets to describe
and compare the annual mean HCRU of those with and without ALS in all categories.
Cumulative resource utilization from time of ALS diagnosis until occurrence of specific sentinel events (as
proxies for disease state/progression) such as use of parenteral nutrition (gastrostomy), ventilator, wheelchairs, will be
summarized through until death for the disease group. Descriptive analysis of proportions of patients who
have one or more sentinel events and time to occurrence of sentinel events from ALS diagnosis (index date)
will also be done using Kaplan Meier analysis. Mean HCRU in those diagnosed with ALS during the 6 months pre-diagnosis, month of diagnosis, and 6 months post-diagnosis, will be compared against controls. Descriptive statistics will be generated for all demographic and baseline variables (as available), including age, sex, marital status, and region. Continuous variables will be summarized by their means, standard deviations, median, interquartile range (25th, 75th percentiles), minima, and maxima. Categorical variables will be summarized with category-specific counts and percentages.
The linked data sources planned for use in the descriptive analysis include:
• ONS Death Registration data.
• HES datafiles:
o HES Admitted Patient Care (APC) data,
o HES Outpatient (OP) data,
• Small area-level data - specifically the Index of Multiple Deprivation (IMD) linked to patient postcode.
Mean and total annual resource utilisation, and associated costs for the following categories:
• Inpatient hospitalisations;
• Outpatient hospital visits;
• Primary care visits;
• Accident and emergency visits;
• Prescription medications;
• Medical procedures;
• Assistive medical devices (e.g., ventilators, wheelchairs)
Li Li - Chief Investigator - Biogen
Katie Stenson - Corresponding Applicant - Biogen
Kim Heithoff - Collaborator - Biogen
Susan Eaton - Collaborator - Biogen
Varant Kupelian - Collaborator - Biogen Inc.
HES Admitted Patient Care;HES Outpatient;ONS Death Registration Data;Patient Level Index of Multiple Deprivation;Practice Level Index of Multiple Deprivation