info@rareimpact.euGene and cell therapies represent some of the most advanced therapies currently being employed in biomedical research to improve the length and quality of life of patients with rare and complex diseases.
To date, patients’ access to these advanced therapies has been hampered by both practical and technical challenges, such as:
- The administrative and logistical challenges healthcare systems face in catering for advanced therapies.
- Satisfying national health technology assessment (HTA) data requirements, particularly when comparative and long-term efficacy data are difficult to generate due to the nature of the diseases these therapies aim to treat.
- The requirement to adjust traditional funding models due to the once-off treatment potential of many gene and cell therapies.
EURORDIS have recognised the issue of patients’ access to these therapies, and have responded by founding a patient-focused collaboration to assess the challenges along with proposing actionable solutions.