Press Release
Brussels – December 22nd 2017
Kedrion Biopharma, an Italian plasma-derived haemophilia treatment manufacturer, and Swedish Orphan Biovitrum AB (Sobi™), supplier of extended half-life (EHL) haemophilia treatment products, signed a Memorandum of Understanding (MoU) with the European Haemophilia Consortium (EHC) in support of the EHC Procurement of Affordable Replacement Therapies – Network of European Relevant Stakeholders (PARTNERS) programme. Through this agreement, the companies commit to supporting the PARTNERS programme to increase access to vital treatment for over 5000 people with haemophilia in European countries that currently significantly under-treat haemophilia patients, substantially improving their quality of life.
Background
Through systematic data gathering and close collaboration with its National Member Organisations (NMOs), the EHC has long been monitoring the level of haemophilia care throughout Europe. Though there have been improvements in many areas, there remain disparities in access to treatment and quality of care for people with haemophilia. In several countries with developed health care systems but limited budgets, sustainable levels of factor replacement to effectively treat haemophilia are not available. This results in lack of treatment or under-treatment, which in turn can lead to debilitating, or even potentially fatal, consequences for the people with haemophilia in those countries. It also places a burden on those countries’ broader health and social systems.
PARTNERS programme
To address these urgent issues, the EHC has developed a new and innovative approach to create a sustainable procurement model for treatment for haemophilia A and haemophilia B in countries located both inside and outside of the European Union. The main objectives of the PARTNERS programme are to work with national health care systems in order to:
- Enable countries to provide adequate levels of factor concentrate that will result in improved levels of treatment for people with haemophilia. Governments will be able to purchase much larger quantities of quality treatment without significantly increasing their national haemophilia budget.
- Formally involve haemophilia clinicians and patient representatives in the national procurement process in an effort to inform selection and the sustainability of health care systems. In the experience of the EHC, this type of model leads to a more cost-effective process.
- Encourage collaboration between all stakeholders, including companies willing to participate in a fair and transparent tendering system that seeks to improve standards of care for people with haemophilia within these countries.
The criteria for country participation in PARTNERS are based on the latest set of recommendations from the Council of Europe’s European Directorate for the Quality of Medicines and Healthcare (EDQM) consensus meetings for the optimal treatment of haemophilia in Europe. Eligible countries are those that do not meet the recommended minimum treatment levels of 4 International Units (IU) factor VIII replacement therapy per capita (2016 recommendations), minimum treatment levels of 0.5 IU factor IX replacement therapy per capita (2016 recommendations) and do not provide prophylactic treatment for all children with severe haemophilia. These minimum levels of treatment are recommended on the basis that they allow for all children to be on some form of prophylaxis, all adults to be at least on on-demand treatment with home therapy, no restriction on a required surgery and possible access to Immune Tolerance Therapy for patients with inhibitors. Governments need to also agree to formally include haemophilia clinicians and patient representatives in national tender/procurement systems as part of the supplier bid evaluation process (2016 recommendations).
Brian O’Mahony, EHC president:
“Today in Europe we still see countries where people affected by haemophilia have the same level of joint damage and poor quality of life as we saw in patients before the advent of modern coagulation factor concentrates in the 1970s and this is unacceptable.
We believe that, with this programme, existing national haemophilia budgets can lead to increased quantities of treatment purchased through better tender systems. This ambitious project aims to provide an increased access to treatment but also to include patients and health care professionals in the decision-making process of the organisation of haemophilia care in any given countries. We are also very thankful for the support of all of our partners without which this project would not become a reality.”
Stakeholders
One of the most important aspects, without which this programme would not be possible, is the involvement of pharmaceutical companies. Companies supplying treatment products for haemophilia A and haemophilia B (namely Factor VIII and FIX replacement therapies, respectively) that have signed a Memorandum of Understanding with the EHC and who support the PARTNERS programme, express willingness to participate in national tender processes of the eligible and participating countries under standard, bi-lateral and confidential bidding conditions within the national procurement processes in a way that enables the increase in purchasing commitment from the national government in an affordable way.
Lorenzo Melani, Hemophilia Marketing Manager, Kedrion Biopharma:
“Nowadays, we know very well how a patient needs to be treated. Now that we know the goal, we have to speed up the process. We strongly believe that a breakthrough programme, such as PARTNERS, will help patients in a number of countries where treatment levels are not optimal.”
Philip Wood, Head of Haemophilia, Sobi:
The PARTNERS programme has the potential to create one unified voice amongst physicians and patients in the tender process. This approach takes sustainable access to a whole new level as people living with haemophilia will have the possibility to access the latest treatments on a long-term basis, raising the standard of care.
Current standing
PARTNERS has received endorsement from the European Patients’ Forum and EURORDIS – Rare Diseases Europe, as well as from many Members of the European Parliament (MEPs). To solidify this support on both a European and national level, the EHC officially launched the PARTNERS programme on November 28th in the European Parliament in the presence of MEPs, EU institutions, pharmaceutical companies and patient organisations involved in PARTNERS.
In 2017, the EHC visited several of the eligible 14 countries for a meeting with representatives of their Ministries of Health, Insurance Funds and haemophilia patient organisations to discuss interest in the programme, and an implementation schedule is now being elaborated on.
About Haemophilia
Haemophilia is a rare congenital bleeding disorder resulting from a deficiency in either clotting factor VIII (haemophilia A) or clotting factor IX (haemophilia B), which are essential proteins for the blood clotting process.
Having haemophilia means that when a blood vessel is damaged in that individual’s body, the bleeding that occurs lasts for a prolonged amount of time. These bleeds can either be spontaneous or they can be caused by trauma and stress. If the bleed is not stopped with treatment products and properly managed, it can severely and permanently damage the patient’s joints (such as ankles, hips and knees), causing affected individuals to have significantly reduced mobility, increased pain and decreased quality of life. Untreated bleeds can also lead to death.
About Kedrion Biopharma
Kedrion Biopharma is an international company that collects and fractionates blood plasma to produce and distribute plasma-derived therapeutic products for use in treating and preventing serious diseases, disorders and conditions such as haemophilia, primary immune system deficiencies and Rh-sensitization.
Kedrion acts as a bridge between donors and the people who need treatments, and works on a global scale to expand patients’ access to available treatments.
Additional information about Kedrion Biopharma can be found at www.kedrion.com.
About Sobi
Sobi is an international specialty healthcare company dedicated to rare diseases. Sobi’s mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primarily focused on Haemophilia, Inflammation and Genetic diseases. Sobi also markets a portfolio of specialty and rare disease products across Europe, the Middle East, North Africa and Russia for partner companies. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2016, Sobi had total revenues of SEK 5.2 billion (USD 608 M) and about 760 employees. The share (STO: SOBI) is listed on Nasdaq Stockholm. More information is available at www.sobi.com.
Additional Resources
For a detailed description of the consensus recommendations coming out of the EDQM meetings, also known as the “Wildbad Kreuth” meetings, please visit:
For more information on the PARTNERS programme, please visit:
To learn more about haemophilia and other congenital bleeding disorders, visit:
Haemophilia Stories is a documentary produced for the EHC, which depicts the daily lives of people with haemophilia in five European countries. It shows the different outcomes that disparities in access to treatment lead to:
Inhibitor Stories is a follow-up documentary that focuses on people with haemophilia and inhibitors and the various challenges they face:
https://www.ehc.eu/community/2017/10/30/inhibitor-stories-documentary-living-haemophilia-inhibitors/
Media Contact
Brian O’Mahony, EHC President
+353 1 6579900
To download the press release, click here.
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.
