Understanding the safety of the drug eliglustat over time for adults with Gaucher disease - Sanofi

What was the health issue?

Gaucher disease is a rare genetic condition where fatty substances build up in organs, like the liver, spleen and bones. Medicines such as eliglustat are used in the NHS to treat Gaucher disease after they have been tested for safety and efficacy in clinical trials. Medicines regulators require safety monitoring of new medicines in patients over time, after the new medicine has been approved for routine clinical use. As a newer medicine for a rare disease, there was limited data available on the long-term safety of eliglustat in the general population. 

What were the researchers trying to find out?

The primary goal of this study, carried out by Sanofi, was to assess the real-world safety of eliglustat in patients by monitoring for any side effects. The study also sought to understand how frequently eliglustat was being used in patients with Gaucher disease.

Why did the research require the use of health data?

For rare diseases such as Gaucher’s disease, data from multiple countries needs to be combined to ensure enough patients are included in the study for the results to be representative and accurate. This could only be done by combining hospital data from specialist centres in multiple countries with a specific Gaucher disease registry.

How was the research done?

Anonymised data was accessed through the International Collaborative Gaucher Group (ICGG) Gaucher registry which included hospital data from 20 centres across Europe, including the UK. These datasets included information on the number of patients taking the medicine, diagnosis details, and records of side effects, which could be used to assess the long-term safety of eliglustat. Patient records were analysed for any adverse effects that occurred after  being treated with eliglustat.  

What did the research find?

The research found that eliglustat remained safe over an extended period of time and that the safety profile in the real-world was consistent with that found in clinical trials. The research also showed that gastrointestinal disorders were the most common adverse effects. 

Why do the results matter?

Pharmaceutical companies are required to monitor the safety of their medicines once approved for use. By conducting these studies on anonymised patient data, companies can ensure doctors, local health systems, decision makers and the public have evidence of the benefits and risks of the medicines being prescribed.  

Further information

This case study has been adapted from the following study: A prospective observational post-authorisation safety sub-registry study to characterise the long-term safety profile of eliglustat in adult patients with Gaucher disease.

Glossary

Anonymised data: data where personal information has been removed so that individuals cannot be identified. This process ensures privacy and confidentiality, making it safe to use the data for research or analysis without revealing who the data belongs to.

Disease registry: Collections of health data related to patients with a specific diagnosis, condition or procedure.

Observational post-authorization safety sub-registry study: research conducted after a medicine is approved. It collects information over time from patients using a medicine in normal healthcare settings, without changing their treatment.

Safety profile: summary of all the known side effects and risks linked to a medicine.