Thalassaemia International Federation
The Thalassaemia International Federation would like to welcome and strengthen the ongoing efforts to address the huge unmet needs of equal access to medicines and vaccines for patients with thalassaemia and other haemoglobinopathies.
Given the absence of national registries, the numbers of patients in each country are largely underestimated, which means that policy and budgeting are not based on true needs. Also, the number of new affected births is not known and is certainly significantly bigger than the published to-date, due to the absence of national prevention programmes.
Life-long regular red-cell transfusions and iron chelation, along with multidisciplinary care and improvement in healthcare services and public health have dramatically improved patients’ survival over the decades, transforming thalassaemia from a fatal paediatric disease to a chronic condition in countries where patients have access to quality healthcare services. Sadly, this is not happening everywhere.
According to estimates, less than 40% of children born with thalassaemia worldwide are regularly transfused and only 12% of them receive proper iron chelation. An even smaller percentage is currently receiving multiple care services.
Through this statement, we would like to urge Member States to:
* RAISE THE ALARM for the introduction of novel genome-related therapies in clinical protocols – these need to be affordable, accessible, adequate and safe;
* ENSURE that all patients have access to quality medicines for iron chelation and treatment of hepatitis C, including all relevant vaccines;
* ADVOCATE for the adoption by the pharmaceutical industry of the WHO fair-pricing approach to guarantee all patients’ access to affordable quality iron chelators and antiviral drugs;
* DEVELOP synergies to increase negotiating power for procuring medicines;
* ENCOURAGE the production and procurement of safe and effective generic medicines.