Gene therapies for rare diseases
#WeWontRest until we can cure rare disease such as haemophilia
By replacing defective or missing genes, scientists aim to cure life-long and highly debilitating diseases such as haemophilia.
Around 69,000 people in Europe have Haemophilia A. Patients with moderate to severe Haemophilia require intravenous replacement therapy every 3-7 days to prevent bleeding incidents.
What is the potential breakthrough?
Gene therapy repairs the direct cause of genetic disease, by introducing genetic material into cells to compensate for abnormal genes. In the case of haemophilia, the gene therapy repairs the gene that makes the body produce its own protein that makes blood clot.
Gene therapy may improve patient quality of life by reducing the need to add the blood clotting protein intravenously, , reducing the stress of spontaneous haemorrhages and the long-term disabilities, specifically arthritis.
What is the potential impact on Europe’s healthcare systems?
What is the potential impact on Europe’s healthcare systems?
Hospital utilisation and drug expenditure could be reduced, due to a decline in the use of cost intensive replacement therapy and treatment of co-morbidities associated with frequent haemorrhages.
What is the potential impact on societies?
Gene therapy offers a potential life change for patients with haemophilia and their families, thereby reducing absenteeism and increasing economic productivity.
Download the full IQVIA/EFPIA Pipeline Review 2021
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WeWontRest
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