The Effectiveness and Cost-Effectiveness of Enzyme Replacement Therapies (ERT) for the Treatment of Late onset Pompe Disease (LOPD)

Project overview 

The recommended treatments are medicines called enzyme replacement therapy (ERT); three different ERTs are recommended for use in the NHS by NICE (National Institute for Health and Care Excellence). We used all available data to estimate the clinical effectiveness and cost-effectiveness of ERTs.

Status: completed

Contact: rob.hodgson@york.ac.uk

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What is the problem?

ERTs are very expensive and may not represent a cost-effective use of NHS resources at their current price.

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What did we do?

To investigate this issue, we conducted a study to estimate the cost-effectiveness of ERTs when compared to the best supportive care therapies available on the NHS (such as mobility aids and supplemental oxygen to help with breathing). We identified and analysed all the data from relevant clinical trials and other research studies on late-onset Pompe disease. These data were used in a health economic model to estimate the cost-effectiveness of ERTs.

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What did we find?

Findings suggest that, after one year, people who had never received enzyme replacement therapy before showed some improvement in walking distance compared to those receiving best supportive care. However, no significant difference was found in lung function (forced vital capacity % predicted). When comparing different types of enzyme replacement therapy, one drug appeared slightly better for walking distance, but this difference disappeared when adjusting for data variations.

Long-term studies showed that initial benefits of enzyme replacement therapy lasted 1–3 years, but muscle function gradually declined over 10–15 years. However, these results are uncertain due to small study sizes and missing data. Evidence on long-term best supportive care outcomes is also limited, making direct comparisons difficult.

Overall, enzyme replacement therapy provides short-term benefits, but its long-term impact on disease progression and the need for supportive care (such as walking aids or ventilation) remains unclear. Further research is needed to understand the lasting effects of enzyme replacement therapy.