Part of the Alfa Europe Federation’s responsibility is to work on improving our
relations with institutions at National and European level to encourage a focus
on social, legal , financial and healthcare resources and treatments for the
needs of Alphas and their families, to support the healthcare professionals
working with AAT deficiency, and scientific research in the field.
To this end we have already begun to participate in EU Commission events, by
invitation, as lay experts in events such as workshops, held by European Platform for Patients' Organisations, Sciene and Industry (EPPOSI), in Brussels. We have become members of the European Organisation for Rare Diseases (EURORDIS). This is to be active in working groups in different areas of policy, such as orphan drugs and advanced therapies, future pediatric medicine ,
transplant and health issues which directly affect people with chronic rare diseases.
EPPOSI and Eurordis work with the EU Commission to organise events which include
all the stake-holders on Policy decision-making, patient representatives,
industry, institutions and the medical profession.
The European Medicines Agency (EMEA) works with national agencies to coordinate
the exiting scientific resources at the disposal of member states for the
evaluation, supervision and pharmacovigilance of medicinal products. The Agency
provides the Member States and the institutions of the EU with the best-possible
scientific advice on any question relating to the evaluation of the quality,
safety and efficacy of medicinal products for human or veterinary use referred
to in accordance with the provisions of EU legislation relating to medicinal
products. We have also become involved as lay experts, invited by the EMEA.
The Committee for Medicinal Products for Human Use (CHMP)
is responsible for preparing the Agency's opinions on all questions concerning
medicinal products for human use, in accordance with Regulation (EC) No 726/2004.
The Committee for Orphan Medicinal Products (
COMP) is responsible for reviewing
applications from persons or companies seeking 'orphan medicinal product
designation' for products they intend to develop for the diagnosis, prevention
or treatment of life-threatening or very serious conditions that affect not more
than 5 in 10,000 persons in the European Union.
Eurordis on behalf of its patient members and associations, works with the
Directorate General for Health and Consumer Affairs, DG Sanco, DG Research and
DG Enterprise to lobby the EU and gain recognised status for people with rare
disease such as AAT deficiency.
We have been consulted by
Orphanet, attending their meeting for Centres of
Excellence in Paris.
Efforts by AlfaEurope members to participate and advance in our goals are
important to our future concerns. Please contact us if you wish to be more
involved in a particular project or represent the Federation.