Lisbon, 17 April 2015
As part of the celebrations of World a Day the Portuguese Association of Hemophilia (APH) strengthens its position against the fact that cost is the only selection criteria used for the purchasing of treatment for haemophilia. This represents a risk for the safety and quality of life of people affected by haemophilia.
Haemophilia is a disease that without proper treatment (coagulation factor concentrate VIII or coagulation factor concentrate IX infusion that is missing, allowing the patient to stop or prevent bleeding) may lead to major bleedings, disability and death.
Products for the treatment of haemophilia available in Portugal are purchased by public hospitals through public tenders. The choice of these products should always follow strict selection criteria guaranteeing the best treatment for patients. These criteria should be above all safety (the contamination of thousands of people with haemophilia with viral infections such as hepatitis C and HIV is still a daily reality for many patients in Europe) and effectiveness (the marketed products have different degrees of personalized effectiveness after administration) .
In 2013 Bill nº 13025-B / 2013 of 10 October established that the only criterion for the purchase of coagulation factor concentrate shall be price. This goes against Bill 28356/2008 that requires the presence of an immune-hemotherapist in the tender committee as well as mandatory advice of APH. Furthermore, the bill requires that product selection shall be based on safety and effectiveness of products aimed at treating people with haemophilia. These two most important criteria have been removed at the expense of the sole economic criteria of price.
Due to these changes in legislation, the health of people with haemophilia has become dependent on cheaper products that are not necessarily the most effective and safest products. On the other hand, medical experts in haemophilia, who should have the final say in the scientific and medical choice of these products, have been relegated to a completely secondary role in the choice of therapies that will be administered to their patients.
Furthermore, the referred Bill nº 13025-B/2013 goes against the recommendations of the European Council, of which Portugal is a member, for the correct treatment and care of haemophilia. These recommendation resulted from the Wild Bad Kreuth Initiative (an initiative of the University of Munich, the European Directorate for the Quality of Medicines and Healthcare and the Paul-Ehrlich-Institut) in April 2013 and they state that ‘price should not be the sole criterion in the choice of treatments for haemophilia.’
APH recognises that the economic criteria is important, however, it is not acceptable that price is the only and decisive criteria for purchasing treatment for haemophilia and that patients are not guaranteed to receive the safest and most efficacious treatment.
The Board of APH(Associação Portuguesa de Hemofilia)
Jim is aged 66 and has Haemophilia A with a high titer inhibitor which he developed at age 14.
Maria Elisa Mancuso (MD, PhD) is a Haematologist and works as a Senior Haematology Consultant at the Center for Thrombosis and Haemorrhagic Diseases of IRCCS Humanitas Research Hospital in Rozzano, Milan, Italy. She is Adjunct Clinical Professor at Humanitas University. She obtained a post-degree in Clinical and Experimental Haematology and a PhD in Clinical Methodology. She is involved in clinical research and has published several original articles in peer-reviewed journals a The Lancet, Blood, Journal of Thrombosis and Haemostasis, Haematologica, Thrombosis and Haemostasis, British Journal of Haematology and Haemophilia. She is reviewer for several peer-reviewed journals and member of the Editorial Board of JTH. She is a member of several scientific societies (ISTH, WFH, ASH, EAHAD, SISET, AICE) and was a medical member of the Inhibitor Working Group of the European Hemophilia Consortium. She is co-chair of the ADVANCE Study Group. She has acted also as co-chair of the Scientific and Standardization Subcommittee of ISTH on FVIII, FIX and rare bleeding disorders. She has been involved as principal and co-investigator in several clinical trials, and she takes care of both children and adults with hemophilia and other congenital bleeding disorders with a specific scientific interest in novel therapies, prophylaxis, inhibitors, and chronic hepatitis C.
As a patient with factor II deficiency, the diagnostic and treatment of rare bleeding disorders is a matter dear to my heart. My motivation to participate in the work of the ERIN committee is to improve both diagnostic and treatment for patients with rare bleeding disorders across Europe.
Economist and financial expert by profession, executive coach and trainer by passion and haemophilia advocate by every drop of my blood through my son (who has severe haemophilia A with inhibitors). Bringing a good decade of practical experience from the corporate insurance world, laser focus, growth mindset and resilience from my own experience, offering you anything I can just do, in hope that together we can make life more fulfilled for those impacted by bleeding disorders.
Amy Owen-Wyard is a Registered Mental Health Nurse. With experience working with children, young people, their families and adults with severe and enduring mental health conditions. Amy was also involved in a service improvement to provide a holistic care approach for those in general hospitals to support both their mental and physical health, whilst sharing her expertise and knowledge in mental health with the wider multidisciplinary team.
