As part of its continued efforts to improve the quality of life of people living with rare bleeding disorders in Europe, the European Haemophilia Consortium (EHC) officially launched the Procurement of Affordable Replacement Therapies – Network of European Relevant Stakeholders (PARTNERS) programme on November 28th in the European Parliament. Members of the European Parliament, European Union (EU) Institutions, patient organisations and representatives of the pharmaceutical industry attended in support of the programme, which aims to increase access to vital replacement therapy for over 5000 people with haemophilia in countries that provide little or no such treatment.

Background
The EHC has been systematically gathering data on all aspects of haemophilia treatment and care in Europe from its National Member Organisations (NMOs) on a regular basis. Surveys conducted in 2009, 2012 and 2015 displayed clear disparities in access to treatment and quality of care for people with haemophilia. In several countries with developed health care systems but highly 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 public health care and social systems.

PARTNERS programme
To turn its advocacy efforts into action and address these urgent issues, the EHC developed a new and innovative approach to create a sustainable procurement model of treatment products for haemophilia A and haemophilia B in countries located both inside and outside of the EU. The PARTNERS programme is a collaborative effort by multiple stakeholders, with the main objectives being to work with national health care systems in order to:

  1. 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 immediate or significant increase in their national haemophilia budget.
  2. Formally involve haemophilia clinicians and patient representatives in the national procurement process in an effort to improve the selection process and the sustainability of health care systems. In the experience of the EHC, this type of model not only leads to the purchase of safer and more efficacious products, but also to a more cost-effective process.
  3. Encourage collaboration between stakeholders, including encouraging factor concentrate suppliers to participate in fair and transparent tenders that seek to improve standards of care for people with haemophilia.

In the time period of carrying out the programme’s objectives, the EHC would also work to improve access to comprehensive care and better organisation of care by utilising the structures established or optimised in each country by this programme, and by encouraging and supporting the certification of comprehensive care and treatment centres, the establishment of national haemophilia committees, the promulgation of national treatment protocols and encouraging equality of access to treatment inside each country.

Stakeholders
One of the most important aspects of PARTNERS is the involvement of pharmaceutical companies. Companies manufacturing treatment products for haemophilia A and haemophilia B (namely Factor VIII and Factor IX replacement therapies, respectively) that have signed a Memorandum of Understanding(MoU) 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. Kedrion Biopharma and Swedish Orphan Biovitrium (Sobi) have expressed their commitment to the project and are set to sign MoUs.

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:
“What makes the PARTNERS programme different is the comprehensive approach with the full inclusion of physicians and patients in the treatment tender process, which is important in creating a unified voice when it comes to improving access to treatment. The programme is really taking things to the next level in terms of securing sustainable access for patients who have not been able to obtain factor treatment.”

Countries participating in PARTNERS
The criteria for country participation in PARTNERS were selected based on the latest set of the Council of Europe’s European Directorate for the Quality of Medicines and Healthcare (EDQM) recommendations 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 (EDQM 2016), minimum treatment levels of 0.5 IU factor IX replacement therapy per capita (EDQM 2016) 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 (EDQM 2016).

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 1970’s 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.”

Endorsements
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) who will assist on a European and national level with the implementation of the programme.

Norica Nicolai, MEP, ALDE Vicechair, Romania
“In public affairs, when something is rare, it is not paid attention to. I think this is the wrong approach: when people have problems, they need solutions.

Haemophilia is a rare disorder, which needs proper solutions from the public sector. Therefore, I decided to support the organisations, which sustain programs meant to increase the quality of life for people suffering from haemophilia and to raise awareness of this subject.

I am very proud of the work that the European Haemophilia Consortium, along with their partners, are doing and I hope that one day, every European patient will have proper access to the best medical treatment for their condition, even if it is a rare one.”

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Additional Resources

For a detailed description of the EDQM Wildbad recommendations, please visit:

For more information on the PARTNERS project, please visit:

To learn more about haemophilia and other inherited bleeding disorders, visit:

www.EHC.eu

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:

Media Contact
Brian O’Mahony, EHC President
brian@haemophilia.ie
+353 1 6579900

Image courtesy of EHC | EHC - European Haemophilia Consortium