Brussels, 20.2.19, Round Table
The Future of Comprehensive Care and Outcomes in Haemophilia
EHC is pleased to bring two perspectives from the near capacity Round Table today in Brussels
The Honourable MEP Gesine Meissner and MEP Norica Nicolai graciously hosted a Round Table at the European Parliament. Ms. Meissner said, “The true value of the EU is demonstrable in rare diseases.”
Evelien Mauser-Bunschoten, UMC Utrecht and members of the EHC Medical and Scientific Advisory Group (MASAG), set the scene with a compelling speech about what is comprehensive care and the Dutch experience with it. In Setting the Scene: haemophilia treatment paradigm shift – how will novel therapies impact the role of CCCs, Dr. Mauser-Bunschoten stressed that in the future, specialised haemophilia centres will be needed more, not less, as their expertise will be needed to address several challenges arising out of the complexity both of rare bleeding disorders themselves as well as of the introduction of novel therapies. Amongst these challenges, she highlighted the ageing of patients and the complexity of their multi-specialist management, including of co-morbidities not previously seen in haemophilia (which accompany today’s increased life expectancy).
EHC Medical Advisory Group (MAG) member Prof. Johannes Oldenburg, University of Bonn, followed with Haemophilia treatment outcomes: raising the bar in the rise of novel treatments. While prophylaxis remains the gold standard for therapy there is a need for novel therapies. Prof Oldenburg outlined the potential of true personalisation of haemophilia treatment with novel therapies, underscoring the important role of Comprehensive Care Centres in the delivery of such personalisation of treatment and care.
The European Association for Haemophilia and Allied Disorders’ (EAHAD) Sébastien Lobet, Chair of their Physiotherapy Committee and member of Cliniques Universitaires St Luc, highlighted the importance of a multidisciplinary approach and individualisation in the provision of haemophilia care. To illustrate his point, he underscored the discrepancy between the structural condition of joints and their functionality, highlighting that novel therapies may improve joint function and mobility, but the underlying damage to joint structures from previous bleeds will remain unimproved and may indeed continue to deteriorate despite mobility gains. For this reason, even in a world of novel therapies, the close evaluation of patients and sensitive diagnostic tools made possible by Comprehensive Care Centres will remain vital to monitor the true health status of patients and to be in a position to respond quickly if needed.
Anna Griesheimer, staff person from the Deutsche Hämophilie Gesellschaft (DHG), the EHC’s German National Member Organisation, raised the alarm on a proposed law in Germany during her talk on The role of comprehensive care centres in the distribution of treatment. This draft law – ‘Gesetz für mehr Sicherheit in der Arzneimittelversorgung’ – was introduced in November 2018 and if passed would come into effect before the summer of 2019. While the intention behind the law was to make pricing more transparent and homogenous, Ms Griesheimer highlighted the many ways in which this proposed law if enacted would in fact be detrimental and negligent in the haemophilia space, including:
1) Effectively limiting patient visits to haemophilia Treatment and Comprehensive Care Centres, which would lead to the weakening of these Centres and therefore to the endangerment of safety and traceability of treatment products, quality of treatment, safeguarding of documentation and erosion of scientific research, training and development. Ms Griesheimer reminded audience members that the move to distribute haemophilia treatments through treatment centres was the direct result of the contamination of blood-based treatment products with HIV and hepatitis C in the 1980s and early 1990s and that by reversing this protection, the proposed law would create gaps in patient monitoring and gaps in traceability and thereby threaten the current reliable and provenly safe system. In the eyes of the patients, such a move would be grossly negligent on the part of the government.
2) Very likely leading to increases in morbidity and mortality as haemophilia is a rare disease that can be life-threatening and for both reasons requires specialised treatment and medical expertise. Adequate treatment of haemophilia and its frequent co-morbidities (including HIV, hepatitis C and orthopaedic problems) is only possible in Comprehensive Care Centres. Additionally, the introduction of novel products with their completely new modes of action should take place exclusively through Comprehensive Care Centres as is the joint position of the EHC and EAHAD. Not doing so would lead to expert clinicians losing full control, oversight and documentation abilities, which would also lead to the erosion of data reporting into the national registry and overall quality and safety controls will become more difficult.
3) Finally, all of the above will lead to the loss of national expertise as, up until present, the centralisation of treatment through Comprehensive Care Centres has ensured that expert healthcare providers are retained and able to grow their expertise through seeing a sufficient number of patients regularly. Regular interaction with patients is also a pre-condition for conducting scientific research and observational studies and, as is true for all rare diseases, good data collection is essential for the continued study and improvement of treatment protocols.
Ms Griesheimer concluded her talk with a call to preserve the current model that works, and works very well, namely to maintain the distribution of haemophilia treatment products via haemophilia treatment centres, such as Comprehensive Care Centres. Concerns about transparency may be addressed in other ways, she suggested, and cited one proposed strategy, namely to ensure that contracts between haemophilia centres and national health insurers are established.
It was certainly a ‘comprehensive’ Round Table of which the key takeaways include that, while it may seem that there are viable alternatives, the long-term effects of circumventing or reducing Comprehensive Care Centres would lead to increased costs and negative outcomes. “We’ll (certainly) need the European Union and others to further strengthen our hard-built, highly expert Comprehensive Care Centres in a world of post-novel therapies,” Amanda Bok tweeted, and further reflected on the irony that, “On one hand, we are building (European Reference Networks) ERNs to centralise European expertise in rare diseases, but on the other hand in haemophilia, which has trail-blazed this centralisation in comprehensive care centres, we’re fighting to keep them.”
MEP Meissner closed the meeting with words of gratitude for having learned many new angles to this complex disease state and flagging her support for the continued role of Comprehensive Care Centres in Europe. She reiterated European Union Health and Food Safety Commissioner Vytenis Andriukaitis’ position that by working together and exchanging views on rare diseases, European expertise can be a world leader. It will take a partnership approach, but one thing today’s Round Table made clear is that the partners are there, and ready.
@photos by Elliott Laub
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.
