‘A miracle we have been waiting for many many years. But it must not become a privilege for some
instead of a right for all.’ Patient, United Kingdom
Gene therapy has been a long-awaited scientific development for thalassaemia patients and has given
hope, optimism, and opened new horizons.
It is imperative for ground-breaking scientific advances and innovative therapies to be sufficiently recognized by national health authorities in conjunction with not only an understanding of the impact of the disease on the daily lives of patients and their families but also in regards to the actual cost-of-illness for healthcare providers.
Read the Full TIF Position Statement on the Accessibility of Gene Therapy for Thalassaemia HERE
Gene therapy for thalassaemia is an expensive and complex procedure, with great efforts ongoing currently by all involved stakeholders, such as patients, healthcare professionals, etc. to ensure its accessibility and availability to as many patients across the world as possible.
Acknowledging that the approval of gene therapy for thalassaemia is now a reality, the Thalassaemia International Federation (TIF) embarked on a global effort with the purpose of further understanding and capturing the patients perspective on this matter, through a short survey which ran between March – October 2020.
The survey was translated in 7 languages and was distributed by electronic means only reaching over 10,000 individuals, including TIF Member organisations, Members of TIF Patient Advocacy Group, etc.