Haemophilia care in Central and Eastern Europe: challenges and ways forward from clinicians' perspective.

Abstract

As a rare disease, haemophilia requires continuous support from public health care systems, financially as well as organizationally. More than two decades after the fall of the Berlin Wall, health care systems of countries of Central and Eastern Europe (CEE) still face challenges when it comes to providing a high level of haemophilia treatment. This letter discusses the main areas for improvement in haemophilia care in CEE countries from a clinicians’ perspective, as initially identified by the authors during the first Central European Haemophilia Interactive Forum for Opinion Leaders in Haemophilia, which took place in May 2013 in Budapest, Hungary. By offering the clinicians’ perspective, our goal is to complement recent publications by O'Mahony et al. 1 and Fischer and Hermans 2. on similar topics. Our focus on the CEE region intends to supplement findings of studies such as ESCHQoL 3. In the nine CEE countries represented at the forum (Bulgaria, the Czech Republic, Estonia, Hungary, Latvia, Lithuania, Poland, Romania and Slovakia) haemophilia care faces a double challenge: first, to improve access to quality care, and second, to strengthen the quality of governance of haemophilia care. One area where progress is necessary to minimize bleeding episodes and improve the lives of patients in these countries is the quality of care, namely access to high standards of treatment and prophylaxis. In general, given the differences in consumption of units per capita per year between CEE countries, this is a challenge primarily for the Baltic countries, Bulgaria and Romania. Prophylaxis for children is available in Hungary, Poland, Czech Republic, Slovakia and Bulgaria. Although also available, prophylaxis is not officially approved and recognized as standard treatment in Estonia, Latvia and Lithuania. The utmost priority for these countries is increasing the supply of factor concentrates and introducing prophylaxis as standard, formally recognized way of treatment. There are also disparities in access to immune tolerance induction therapy (ITI) between individual countries surveyed. As a consequence of the historical lack of resources, all CEE countries have a specific population of patients with long-lasting history of inhibitor who do not have any prior experience with ITI. Some countries of the region (Hungary, Czech Republic, Slovakia) have initiated ITI even in this population despite poor prognostics and have seen cases of inhibitor eradication. While in these countries all patients have access to ITI including high-dose regimens (e.g. Bonn protocol), the situation is more difficult in all three Baltic countries as well as in Poland, Romania and Bulgaria, where ITI is not guaranteed. There is no doubt that it is necessary to enable access to ITI (at least for children) also in these countries. Based on the recent recommendation of EDQM, every EU country should be able to supply people with haemophilia with the factor FVIII equivalent to 3 IU per capita per year 4. The rationale behind this recommendation is mainly to sufficiently cover the factor consumption needed for prophylaxis and ITI in children with haemophilia. Those CEE governments and authorities which do not yet follow this recommendation need to tackle this issue as a priority. A significant source of factor concentrate for certain CEE countries, for instance Bulgaria, is participation in international clinical trials. While this ‘clandestine prophylaxis’ is far from a systemic solution, clinical trials play a non-negligible role in assuring access to treatment, in addition to providing an opportunity for training of younger staff and developing international collaboration. Some CEE countries, namely Poland, Bulgaria and Romania face also the problem of an insufficient number of properly trained and experienced specialists (orthopaedists, gynaecologists, hepatologists, urologists, infectious diseases specialists) familiar with haemophilia patients. This seems to be less of an issue in other CEE countries but where difficult access to specialists persists, attention needs to be paid to improving professional education of specialists about haemophilia, as well as undoing any structural disincentives to collaboration (e.g. overly complex financing mechanisms, such as in Bulgaria). The other area relates to the formal and practical recognition of central haemophilia organizations 5 by regulators, local/national authorities and other important stakeholders. In countries where similar organizations are lacking (Latvia, Lithuania, Estonia), the most burning issue is to formally establish them in collaboration with health authorities and to achieve official recognition. In Bulgaria, despite the existence of the Working Group of the Bulgarian Scientific Society for Clinical Haematology, official designation and recognition of a National Haemophilia Council by the state is needed. In a positive development, Romania has in 2014 seen the establishment of a central council composed of representatives of Ministry of Health, the payer, patients and care-givers. In other CEE countries (Hungary, the Czech Republic and Slovakia) the challenge is to improve the integration of central haemophilia organizations in the decision-making system, so that they serve as a real partner co-shaping policies without whose agreement decisions cannot be made. Parallel to this, there are also more efforts needed to refine the role of Comprehensive Care Centres (CCC) and Haemophilia Treatment Centres (HTC) – or even to formally establish them where necessary, especially in Bulgaria, Poland and the Baltic countries. A European Treatment Centre was recently accredited in Timisoara, Romania, but regional disparities in access to care in the country persist. CCC and HTC networks function well in the Czech Republic, Slovakia and Hungary. In Poland the existing network is judged insufficient, especially for adults. It is crucial to follow the suggestions of EU haemophilia principles 2 and set up and formally recognize networks of CCCs and HTCs in all CEE countries. One possible way to do this is to cooperate within the current initiatives of European Haemophilia Safety Surveillance (EUHASS) and EUHANET in this field 6, 7. In order to give haemophilia clinicians and patients more legitimacy in discussions with decision-makers, up-to-date and accurate nation-wide data are required. However, central haemophilia patient registries are non-existent or underdeveloped in Bulgaria, Estonia, Latvia and Lithuania. In the Baltic countries, e.g. several institutional registries exist in individual hospitals, while in Romania and Poland, registries exist but are often incomplete or not up to date and not publicly accessible. In contrast, sophisticated and well-established registries used by medical professionals, as well as by other stakeholders and regulators, work well in the Czech Republic, Hungary and Slovakia 8. In general, collaborative data-gathering efforts by health care providers, supported by central administrations, would greatly enhance knowledge about haemophilia in CEE countries. Cooperation within the EUHASS registry is also crucial within the European Union context. Given that not all CEE countries face the same problems (main findings per country are summarized in Table 1), it is clear that steps towards improvement of haemophilia care will have to be taken predominantly at the national level. However, some changes can be initiated or supported by international collaboration – both with other European countries and within CEE countries themselves. Twinning projects and collaborations are of invaluable importance for improving the clinical part of haemophilia care; at the same time international collaboration could also point to best practices in stakeholder involvement, governmental action and health care governance in haemophilia in general. International initiatives such as the World Federation of Hemophilia and its regional arm the European Haemophilia Consortium, or the European Association for Haemophilia and Allied Disorders, can play an active role in promoting and bettering haemophilia care in the CEE region. The authors thank the participants of the expert survey: Valeria Kaleva, Angelina Stoyanova, Denka Stoyanova, Svetla Kostova, Maria Podolak-Dawidziak, Krystyna Zawilska, Joanna Zdziarska, Witold Kruger, Elżbieta Stasiak- Zając, Wanda Badowska, Magdalena Górska-Kosicka, Fatiha Griffi, Attou Khadidja, Meriem Bensadok, Světlana Köhlerová, Kateřina Lejhancová, Lukáš Stejskal, Ines Vaide, Kadri Saks, Irena Voitovica, Gunta Kluce, Egle Karalkeviciene, Audrone Stonkiene, Angelika Bátorová, Janka Hulíková, Pavol Hollý, Alena Rovňáková, Natália Galóová, Alexander Varga, Marta Kvočáková, Shadan Lalezari, Yariv Fruchtman, Savita Rangarajan, Ioana Franc, Ingrid Crenguta Miron, Madalina Schmidt, Luminita Rusen, Valentina Uscatescu, Emilia Ursu, Claudia Ciobanu, Maria Kardos, Istvan Szegedi, Katalin Hunyadi, Gyorgy Pfliegler, Aniko Marosi, Marianna Zombori, Hajna Losonczy, Ferenc Horvath, Zsuzsa Agoitai, Judit Farkas, Erzsebet Marian, Nacira Cherif. Logistical and financial support to the first Central European Haemophilia Interactive Forum for Opinion Leaders in Haemophilia preceding our survey was provided by Baxter. However, Baxter did not influence or interfere with the content of this publication. No Baxter employees were present during the confidential group discussions. Baxter covered the OnlineOpen fee for this article. LN and JB contributed equally to shaping the structure and content of this article. LN, JB, NC, AK, STS and MSp led group discussions, provided written conclusions and critically reviewed earlier versions of this paper. MSe provided detailed written critical input. All authors approved the final version. JB has received in the past a speaker's and/or consultation fee from Baxter Novo Nordisk, Bayer, LFB, Griffols. Mse has worked on clinical trials funded by Baxter, Bayer, Novo Nordisk and Octopharma, and has received reimbursement or attendance fees from Baxter, Bayer, Novo Nordisk, Biotest and Octopharma, and has organized educational meetings funded by Baxter and Novo Nordisk. AK and MSp have received speaker's fees and reimbursement for attendance of the forum from Baxter. SST has acted as a paid consultant for Bayer. The authors stated that they had no other interests which might be perceived as posing a conflict or bias.