Exploring the cost of illness of a rare disease in the UK

Presentation of primary and secondary care database analyses


The challenge

Our client’s product indication was a rare, potentially life-threatening disease. Disease awareness was low which often lead to misdiagnosis and inappropriate management.

Our client wanted to generate evidence on the cost and burden of illness to both patients and the National Health Service (NHS), to ultimately raise disease awareness amongst external stakeholders. Published evidence on this disease was limited, particularly in the UK setting.

The solution

Firstly we conducted a brief literature review to demonstrate the limited availability of published evidence on the cost and burden of illness in the UK.

Working with our colleagues in Adelphi Real World, we analysed the following data sources to determine healthcare resource use in patients vs matched controls:

  • Hospital episode statistics (HES) database to indicate the burden of disease in secondary care (i.e. acute care), a database of all admissions, outpatient appointments and A&E attendances at NHS hospitals in England.
  • The Health Improvement Network (THIN) database which demonstrated disease burden within primary care, a representative sample of interactions with general practices in the UK.

We developed a strategic communications plan to disseminate the key findings of the study to all appropriate audiences through conference posters, presentations, and peer-reviewed publications. This was done in close collaboration with a group of clinical experts in the field.

Key results

The results helped quantify the incremental cost of burden of the disease within both primary and secondary care in terms of healthcare professional time, drug costs (not including specific treatments for this rare disease), hospital admissions and bed days.

Value to the client

This was the first UK cost of illness study for this disease. The results highlighted the additional cost to the health services of rare diseases in both primary and secondary care.

These results were used in discussions with UK payers to highlight the current unmet needs in the disease area.

Our publications were used to raise disease awareness amongst target external stakeholders and to highlight the need for appropriate disease management.

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