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Thalassaemia International Federation

TIF

Mission: To develop and establish National Control Programmes for the prevention and management of haemoglobinopathies in all affected countries.

Lobbying Activity

Response to Health technology assessment - Joint clinical assessments of medicinal products

29 Mar 2024

The Thalassaemia International Federation welcomes the draft Implementing Act on JCAs and the effort to define the exact role of patients. We firmly believe that patients should actively contribute to scoping, assessment and report review in a prominent manner, as per the Regulation and Implementing Act. More specifically, information disclosed to the Coordination Group (Art. 4) should contain information received from patient organisations (when invited to submit their input). The possible sources for the identification of patients are well defined (Art. 6) but the inability to identify a patient, either because of conflict of interests or disease rarity, is not taken into account nor appropriately mitigated. Moreover, patient organisations should be allowed to comment on aspects other than the disease or therapeutic area they represent, including patient experience, patient preference elicitation studies, surveys, etc. Overall, patients should be given the right to verify that assessments are based on science, medical evidence and the patient perspective.
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Meeting with Frédérique Ries (Member of the European Parliament, Shadow rapporteur) and FIPRA International SRL and

5 Dec 2023 · Revision of the Pharmaceutical Legislation

Response to Revision of the Union legislation on blood, tissues and cells

14 Dec 2020

The Thalassaemia International Federation represents a community of transfusion-dependent patients who rely on blood safety for their survival. We thus welcome the European Commission’s initiative to revise and update the Directive, especially with regard to the oversight of Member States in their respective practices to acquire adequate and safe blood supply. Indeed, the gaps and shortcomings identified in the roadmap correspond to a number of needs that need to be addressed through a flexible yet inclusive policy approach, having voluntary blood donation at its core. This principle that has been proven to be the best option for safe blood, based on data coming from healthcare professionals, practitioners and transfusion-dependent patient communities, should not be optional for Member States but mandatory and part of the policies uniting Member States under a common health umbrella. As the EU is highly heterogeneous with regard to the adoption of safety requirements and quality monitoring, we strongly recommend the fusion of Policy Options 2 and 3, as the EU needs to have the role of the auditor, ensuring that the BTCs in each Member State comply with those safety and quality standards defined by competent scientific bodies, i.e. the EDQM and ECDC. This will further strengthen the European Health Union agenda and support Member States in the adoption of policies that will ensure blood safety and adequacy. We need to highlight that Member States should not have the option to adopt measures that are prerequisites for optimal blood management and safety. These should be clearly specified in the revised version of the Directive. By adopting quality standards (and even certification) at European level, the qualification of staff members of BTCs will also be assessed. Competency lies at the core of every BTC hence the need to engage Medical Haematologists, renowned scientists and patients in the work of blood establishments. Patient-reported outcomes must be included in every policy that affects patients as end-users. Lastly, we welcome the following statements, as mentioned in the Roadmap, and hope to see them integrated into the revised version of the Directive: “The Commission will perform control in Member States, including audits of national systems of inspection, authorisation and vigilance.” “The Commission will develop common guidance and training activities on oversight in Member States.” “[…] EU monitoring and notification of sufficiency data and measures for emergency supply responses” “[…] rapid notifications in cases of serious impending shortage […] “The scope of the BTC legislation will be clarified to include novel substances of human origin currently used but not regulated at the EU level”. “Competent authorities will have to grant prior authorisation based on data demonstrating safety and benefit for patients that justifies any risks associated with treatment.” As end-users of blood products, we are ready to engage into bilateral discussions with the Commission to express the views of the haemoglobinopathy patient and contribute in strengthening the readiness and sustainability of national blood management bodies.
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Meeting with Stella Kyriakides (Commissioner)

10 Dec 2020 · Exchange on EU plans to revise the legislation on safety of blood and on other issues of interest/concern by Thalassaemia patients.