ECC attended the 4th International Alpha1 patient congress, 11-12 April in Barcelona, where presentations were made and discussions held on the nature of COPD caused by the of Alpha1 anti-trypsin deficiency, treatment options (if any), national and regional registries, research development and potential cure. During the interactive sessions, patients shared their first-hand experience with the disease and expressed their wishes for a better and earlier diagnosis of AATD, equal access to treatment and absence of stigma. The event was also an opportunity for AATD patient organisations from around the world to meet and mingle, to learn from one another’s activities and to display through an exhibition the scope of their association’s work.
(In the picture: Mr. John Walsh, President & CEO, Alpha-‐1 Foundation, USA, Prof.Sten Eriksson,Sweden, Honorary Guest Sr.Boi Ruiz,Catalonian Minister of Health, Spain; Sra. Mercedes Vinuesa Sebastian, Dir Gral Public Health,Quality & Innovation, Spain, Sr. Victor Grifols, President & CEO, Grifols, Spain)
During the meeting, the recommendations made in the report “Alpha-1 in the European Union – Expert Recommendations” presented in the European Parliament in 2011 was reiterated. The report was to identify the issues facing Alpha-1 patients and give concrete recommendations that would help the European institutions and Member States to choose the best way forward. The working party that prepared the report was called by Christopher Fjellner MEP (Sweden) and chaired by his colleague Carl Schlyter MEP (Sweden).
ECC supports the recommendations:
- Member States and the EU must ensure appropriate recognition of Alpha-1 as a rare condition and Alpha-1 related emphysema as a specific ultra-rare disease.
- Member States must raise awareness of Alpha-1 in the medical community and the general public in order to ensure a timely and fast diagnosis that will increase the chances of preventing irreversible tissue damage.
- Member States must prevent and put an end to health inequalities affecting patients suffering from Alpha-1 and other rare diseases.
- The EU must ensure that all Member States respect the EU definition of rare diseases.
- Future EU and national policies with a relevance to rare and ultra-rare diseases should respect the spirit and the letter of existing EU policies addressing these issues.
- EU Member States should ensure that policies and legislation in the field of rare diseases are not jeopardised by cost containment measures. Such measures should not have a negative impact on areas where long-term investments are needed to make a difference, such as public health.
- Each EU Member State should develop and implement ambitious national plans or strategies on rare diseases, as recommended by the Council of the European Union’s recommendations on action in the field of rare diseases.
- Member States should ensure that Alpha-1 patients can access the treatments they need, notably when implementing the Cross-Border Healthcare Directive.
- The EU should work towards better standardisation of treatments and devices supporting breathing to ensure that patients can enjoy their freedom of circulation.
- The EU should develop an ambitious strategy on information to patients so that all patients can make informed choices about their treatment options.
- Member States should ensure that the optimal guidelines for the treatment of Alpha-1 are implemented in order to reduce the need for lung transplants and thereby contribute to increasing the availability of lungs for transplantation.
- Patients should be given the possibility to decide with their physician whether and when they should undergo organ transplantation.
- Member States, national HTA* experts and policy makers must acknowledge the reality of clinical research on therapies for rare and ultra-rare conditions and accept alternative evidence validated by experts. Gold standard randomised, placebo-controlled and double-blind clinical trials with a sufficient number of patients are impossible to conduct and unethical. Physicians treating Alpha-1 patients should be asked about effectiveness when a therapy is being assessed.
- The EU and Member States must provide support to Alpha-1 expert groups, including academic and patients’ groups, in order to pool expertise and build on it.
- The EU and Member States should support the creation and the management of Alpha-1 patient registries and seek the advice of Alpha-1 experts who are already running them.
* HTA Health Technology Assessment produces research information about the effectiveness, costs and broader impact of healthcare treatments and tests for those who plan, provide or receive care in the NHS. HTA is used by the Department of Health and NICE.
This information was provided by Alan Heywood-Jones, Chair of the Alpha 1 Awareness UK .