2010 November - EHC

NEWSLETTER 

NOVEMBER 2010 

www.ehc.eu 

EHC Newsletter| NOVEMBER 2010 1

In the November 2010 issue… 

THE PRESIDENT’S REPORT p.3 

EHC NEWS p.5 

EHC CONFERENCE LISBON p.7 

NMO NEWS p.9 

EHC SUPPORTERS NEWS p.11 

EU HEALTH POLICY UPDATE p.15 

OTHER EVENTS OF INTEREST p.20 

ABOUT EHC: The European Haemophilia Consortium (EHC) is a 

European umbrella organisation representing national member 

organisations from 43 countries in Europe. The EHC is working to reduce 

the burden of the disease on both the individual and on society. Its 

mission is to improve the quality of life of people with Haemophilia in 

Europe. For more information contact info@ehc.eu 

Please note that the EHC Supporter News section contains news 

submitted by our corporate supporters and is not compiled by EHC. 

European Haemophilia Consortium 

EU Representation Office 

Rue du Luxembourg 22-24 

BE - 1000 Brussels 

Phone: +32 (0)2 761 66 27 

Direct Fax: +32 (0)2 777 05 10 

Email: info@ehc.eu 

Website: www.ehc.eu 

European Haemophilia Consortium 

Headquarters 

National Member Organisations Office 

Rue Grisar 38 

BE - 1070 Brussels 

Phone : +32 (0)2 521 11 50 

Direct Fax : +32 (0)2 520 68 66 

Email : office@ehc.eu 

Website: www.ehc.eu 


THE PRESIDENT’S REPORT 

Dear Readers, Members and Friends, 

Back from Lisbon, I thank all of you who attended the 23 th 

EHC-conference. In the first place I thank the hosting NMO. I 

congratulate them for the success of the Conference, for the 

warm welcome and for the smooth course of the Conference. 

With them I congratulate the Conference organizer, without 

whom this couldn‟t have been done. I thank the sponsors for 

supporting us in the organization of the Annual European 

Haemophilia Conference. They provide us the financial possibility to attend the 

meetings, to lay contacts at the exhibition hall, they encourage us to improve 

our taking care of patients with haemophilia and other bleeding disorders. I also 

would present my gratitude to all who have contributed to the scientific level of 

this conference: speakers, doctors, nurses, physiotherapists, patients 

themselves, … Thanks for those who nursed the patients during the Conference. 

Yes, thank you all for your presence and your contribution. 

The conference this year aimed some new start in our working. The business 

meeting was a sign of this „renewal‟. We had several elections. The General 

Assemble elected a new Steering Committee. I congratulate those who are 

elected: Mr. Jordan Nedevski as vice-president finance; Dr. Gabriele Calizzani, 

Mr. Brian O‟Mahony, Dr. Gábor Varga and Mr. Alain Weill as Steering Committee 

members. Dr. Uwe Schlenkrich was asked to join the SC as a co-opted member. 

I thank them in advance for their engagement towards the patient organizations 

and for all the work they will do. Although they weren‟t elected, I also would 

express my gratitude to those who accepted to be a candidate. As Prof. P. 

Giangrande expressed after the presentation of all the candidates: “it is 

remarkable how strong the candidates are and what a high level of capacities 

they present.” This means that they too wanted to put their skills into the 

service of the EHC and the improvement of patients care. After this election, the 

General Assemble choose also the hosting NMO for the EHC-conference in 1012. 

A tidy competition between three candidates resulted in the election of the Czech 

Republic NMO. They will host the 25th EHC-Conference in Prague. 

The General Assembly approved the relocation of our headquarters to Rue Grisar 

38, BE - 1070 Brussels and the fact to work with a CEO. He will take over some 

tasks of the EU Representation Office, as for example the newsletter. We thank 

the staff of the EU Representation Office for the excellent work they have done 

and we appreciate that we still may reckon on their collaboration. 


Together with them, the EHC organized in last September the third Round Table 

focusing on : “Patient Safety and Haemophilia Care. How to optimize access to 

safe medicines and treatment, creating the value of patient safety between 

stakeholders.” You will find an excellent report in this newsletter. We had to 

postpone the European Parliament Event to next year for the reason that too 

many activities were scheduled and managing this would asking too much effort 

and resources. 

You also will find an article on the event in Poland, where the EHC sustained the 

Polish Haemophilia Association in its action of better treatment. 

The EHC , together with Mr. Mark Skinner the president of the World Federation 

and the Portuguese Society, had a meeting with the Minister of Health of 

Portugal on the occasion of the EHC-Conference in Lisbon. They contributed with 

success to the strategy for haemophilia care in Portugal. 

We just received the news that MEP Mrs. Serracchiani has tabled a written 

question on Haemophilia treatment to the European Commission. The 

Commission has to reply to this question in 6 weeks. We will keep you informed. 

I wish you the best for those two last months of this year and hope to meet you 

again at one of our events. 

Sincerely yours 

Ad Veldhuizen 

President EHC 

(some SC-members in discussion at the SC-meeting in Lisbon) 


EHC NEWS 

EHC Round Table on Patient Safety and Haemophilia Care 

On 28 September the EHC held a Round Table on Patient Safety and Haemophilia Care– How to 

optimize access to safe medicines and treatment, creating the value of patient safety between 

stakeholders. 

The Round Table’s well-timed debate focused 

on access to safe medicines and treatment and 

followed the European Parliament’s vote on 

the pharmacovigilance legislation, which aims 

to strengthen transparency and 

communication on medicine’s safety issues and 

most importantly empower patients to report 

adverse effects of new medicines. 

Dr. Mike Makris, started his presentation with 

the words “ it cannot be any other patient 

group that has suffered so much from adverse 

events than Haemophilia patients”. The risk 

factors, which are well known for this group of patients, have changed significantly for the last three 

decades. The pharmacovigilance system helps to identify the new risk factors and make sure that 

patient safety is safeguarded. Dr. Markis presented the EUHASS (European Haemophilia Safety 

Surveillance) project, which aims to establish a pharmacovigilance programme to monitor the safety 

of treatments for people with Haemophilia and develop and maintain a database of all the 

Haemophilia centres in Europe. Presenting the first results of the project, Dr. Makris confirmed that 

so far, the two main treatments studied, plasma-derived products and recombinant products (i.e. 

medicinal products not made from blood, but issued from a manufacturing process used to replicate 

the factor molecule), show the same risk. 

Prof. Alessandro Gringeri from the University of Milan, further addressed the issue of plasma 

derivatives vs. recombinant products. The use of plasma-derived products to treat Haemophilia 

patients is still very much associated with the tragedy of the 80s, when many patients were infected 

with HIV or Hepatitis B and C through blood donations. Nowadays, the safety product issue balances 

between the hypothetical risk of blood borne infections (such as prions) and the real risk of 

contracting inhibitors (antibodies). He therefore stressed that the issue of safety concentrates is still 

important, and it is crucial for all stakeholders to remain vigilant on the methods and measures 

adopted. 

Mr. Charles Waller, Vice- President of PPTA Europe (Plasma Protein Plasma Associations) stressed 

that the issue of supply products was underestimated in the safety debate, reminding the audience 

that “contemporary care should be made available to everyone”. Indeed, the cost of manufactured 

products, financial pressure on healthcare authorities, and reimbursement policies will have a 

growing impact on the supply of products for patients. These issues should be addressed through 

more education, communication and collaboration between industry and patients. 


Views from the regulators, were expressed by the EMA representative, Monica Vinhas, who 

explained the importance of the proactive 

pharmacovigilance and Risk Management Plan as 

set out by the Agency, and the requirements that 

need to be provided by companies for marketing 

authorisation. The idea behind this process is to 

“minimize risks and optimize possible benefits”. The 

Risk Management Plan is to evolve with the 

adoption of the pharmacovigilance legislation, 

which is set to come into force in 2012. In 

particular, there will be an evolution toward 

benefit risk management plans, more transparency, 

better information to patients, and better data 

collection. 

Finally, the patient views were given by Dr. Uwe Schlenkrich, EHC Steering Committee, and Board 

member of the German Haemophilia Society. Dr. Schlenkrich presented the history of safety 

treatments of Haemophilia patients in Germany since the 70s, and the results of unsafety 

treatments, stressing the important role of the patient. Patients, he said, should be included in all 

safety, supply and treatment issues, particularly when addressing reduction of costs in the 

healthcare system, and the issue of HCV compensation of patients. 

Echoing the Round Table exchange, the EHC calls for: 

� High and constant protection of safety treatments and 

products in Haemophilia care 

� Proactive pharmacovigilance system which will 

empower patients to actively report adverse effects 

� Quality information to patients on safety issues 

� Equal access to supply products and to treatment 

across the EU 


EHC-CONFERENCE 22 - 24 OCTOBER LISBON 

Advocacy meeting with Government 

Centers for hemophilia can already start this year The Health 

Ministry will examine the proposal it received to create three 

centers specializing in the treatment of hemophilia and other 

clotting diseases. The idea is to move forward in 2011. In the 

same week that Lisbon hosts for the first time the conference 

of the European Haemophilia Consortium (EHC), the Assistant 

Secretary of State and Health, Manuel Pizarro, received the 

president of the WFH and EHC and members of other 

organizations, including the Portuguese Hemophilia Association 

and other congenital coagulopathies who tried to warn for a gap. "By centralizing the monitoring of 

patients we ensure a greater expertise and an integrated approach that includes not only the 

haematological situation, but also the approach in emergency situations, complications (orthopedic 

and other), the psychological counseling, the genetic counseling" explained the Department of 

Public Health to PUBLICO. The Ministry could not advance concrete dates nor would confirm that the 

centres will be situated in hospitals of Lisbon, Coimbra and Porto. 

European Haemophilia Consortium’s point of view 

The European Haemophilia Consortium (EHC) will bring some ideas to implement haemophilia 

centers in Portugal. «The aim is to facilitate the discussion over this matter», says Ad Veldhuizen, 

president of EHC. «Comprehensive haemophilia care comprises many elements that affect the 

quality of the patient’s life». Different treatment methods, different medicines, different 

organizational structures 

and specialists including 

medical doctors, specialist 

nurses, physiotherapists 

and psychologists play, all 

together, an important 

role in comprehensive 

care», he adds. 

(F.l.t.r. Mrs. Maria de Lourdes 

Fonseca, vice president of the 

Portuguese Haemophilia 

Association, Ad Veldhuizen 

President EHC, Dr. Alice Tavares, 

chairman of the Conference) 

«These centers will be 

helpful, not only for 

people with haemophilia, 

but, also, to other bleeding 


disorders. Comprehensive health care centers (that were born six years ago) are implanted already 

in more than 20 countries in Europe like in: Ireland, UK, Germany, Belgium, Netherlands, France, etc. 

And comprehensive health care will cover all sites of treatment», completes. 

In his perspective, «comprehensive health care is more cost-effective and for instance a good way to 

improve quality of life in patients with haemophilia». Taking the example of other countries, patients 

should go twice a year to centers to receive a medical treatment plan. After that, they can follow the 

treatment in local hospital. «In this centers we have all the disciplines and the treatment is 

concentrated. » 

This measure is very useful not only for children but patients with co-morbidities such as HIV positive 

and infected with hepatitis. «Although patients have to travel to the centre, at the end, patients can 

achieve a lot of benefits. We have learned by following examples of other countries that the cost 

have crash down with comprehensive health care centers, because in this places experts are 

available to treat patients who suffered of haemophilia in a efficient way.» Also as hospitalisation 

went down with 40%, quality of live for the patient is much better now. 

The social cost is lower because when the patient has a better treatment his functionality to work or 

to go to school, reducing the days missed, increases, for instance. «This multidisciplinary approach 

needs to be applied in efficient care and treatment centers to ensure its full benefits. The EHC would 

like to improve the progress on these elements in Portugal, giving some advices and tools to start 

implementation soon», concludes Ad Veldhuizen. 

(text published in the newspaper edited during the EHC-conference) 

Welcome of the President of the “Associação Portuguesa 

de Hemofilia e de outras Coagulopatias Congénitas” (APH) 

Lisbon is the chosen place to perform the 23rd Annual Conference of European Consortium of 

Haemophilia (CEH), a meeting organized by Portuguese Association of Haemophilia and other 

Congenital Coagulopathies (APH) and CEH. According to João Paulo Silva, the president of APH, this 

meeting aims «to disclose haemophilia and promote the understanding of all the issues related to 

this disease, in order to improve haemophilia and other congenital bleeding disorders’ treatment, as 

well as implement references centers to treat patients». 

João Paulo Silva hopes that this event «can be an opportunity to boost universal criteria for the 

treatment of these diseases throughout the country – guidelines». In addition, this meeting «can be 

a change to attract more medical doctors and teams with specific expertise in the multidisciplinary 

treatment of haemophilia and other congenital coagulopathies», he says. 

«For APH, the Conference of the European Consortium of Haemophilia has a great value. We cannot 

forget that this meeting is one of the most significant scientific events in Europe and all over the 

world in this area. For us, is an honor to receive the leading experts in haemophilia and congenital 

bleeding disorders in Portugal», indicates. 


The goal of this meeting is to expand the knowledge «not only for technicians and physicians who 

manage these problems in clinical practice, but either to policymaker and general public». 

As a patient with haemophilia, the president of APH highlights the importance of an event with this 

dimension, such as CEH, patient’s quality of life. This is an opportunity to update information. João 

Paulo Silva also remarks the importance of driving the information to general public – a way to 

demystify haemophilia. «One of our major concerns is the fact that haemophilia stills unknown to 

the generality of medical doctors, nurses, paramedics and INEM». 

Finally, the president of APH leaves a message of hope: «My desire is to expand haemophilia and 

other congenital bleeding disorders out of borders, in order to improve the treatment and the 

patients’ quality of life.» 

(text published in the newspaper edited during the EHC-conference) 

The complete version of this publication is downloadable on www.ehc.eu 

NMO NEWS 

Ireland - New Publications on Women and Bleeding 

Disorders and Rare Bleeding Disorders 

The Irish haemophilia Society has published new booklets 

on Women and Bleeding Disorders and on rare Bleeding 

Disorders. These comprehensive booklets are aimed at the 

large number of these demographic groups who do not 

always avail of the information and services available from 

national haemophilia patient organizations stated Brian o 

Mahony, Chief Executive of the I H S. The booklets can be 

downloaded from the I H S website www. haemophilia.ie. 

The Irish Haemophilia Society are also happy to mail 

copies free of charge to any Haemophilia Society. 


Haemophilia Care in Poland 

On 7th September 2010 an international conference “Haemophilia Care in Poland – Current State of 

Affairs” took place in Warsaw, Poland. The conference was jointly organized by the Polish 

Haemophilia Society and the EHC, and the Polish Minister of Health Ewa Kopacz was its official 

patron. The speakers included Dr Paul Giangrande, haematology consultant from Oxford Radcliffe 

Hospital, Brian O’Mahony, Chief Executive of the Irish Haemophilia Society, Dr Joanna Zdziarska, a 

haematologist from the University Hospital in Cracow, Poland, and Bogdan Gajewski, President of 

the Polish Haemophilia Society. Also representatives of people with haemophilia and some parents 

of children with haemophilia had a chance to express their opinions. The debate was efficiently 

moderated by journalist Malgorzata Wisniewska. 

The speakers underlined that the introduction of prophylactic treatment for children in 2008 was a 

very significant step forward in haemophilia care in Poland. However, there remained many 

problems to be solved. 

The most important were: 

� lack of comprehensive haemophilia care, including problems with access to physiotherapy; 

� lack of secondary prophylaxis for adults with serious joint changes; 

� wrong costing of medical procedures by the National Health Fund, which was the reason 

that hospitals often lost money when treating patients with haemophilia; 

� no comprehensive treatment for patients with inhibitors; 

� necessity of continuing the existing National Programme of Haemophilia Treatment, which 

will expire in 2011; 

� no access to recombinant factor concentrates. 

When referring to the latter point, Dr Giangrande stressed the fact that prices of plasma-derived and 

recombinant factor concentrates had become comparable, and in the most recent tender organized 

in the United Kingdom plasma-derived factor concentrates (bought only for patients with von 

Willebrand disease as otherwise British patients rely on recombinants) were more expensive than 

recombinants. 


Dr Giangrande pointed also out that switching over from plasma-derived factor concentrates to 

recombinants did not cause any increase in inhibitor frequency. 

Praising the progress made in Polish haemophilia care, Brian O’Mahony added that still further steps 

were necessary to fulfill the European Principles of Haemophilia Care. He underlined that patients 

should have access to the full range of concentrates, including both recombinant and plasma 

derived. Recombinant concentrates form 100 per cent of the supply in Ireland and 40 per cent in 

Hungary. Patients should also have a chance to participate in making decisions concerning the state 

health policy as far as haemophilia was concerned. 

During the debate parents and journalists asked about reasons for the existing delays in introducing 

a modified version of the programme of prophylaxis for children. Representatives of the National 

Health Fund and the Ministry of Health explained that the delay had been caused by organization 

problems and that soon the new version should be implemented. 

The conference found a large echo in the media, with articles published in several magazines and 

some materials, including an interview with Dr Giangrande and Brian O’Mahony, broadcast by state 

television. 

There was also a direct gain for people with haemophilia in Poland as the National Health Fund 

decided to revise its costing of endoprosthetic surgery in haemophilia. This in turn resulted in a 

change of the attitude of hospitals. Currently up to eight such surgeries a month are made in the 

Institute of Haematology and Transfusion Medicine in Warsaw, and some more are made in Cracow. 

Adam Sumera 

Vice-President, Polish Haemophilia Society 

EHC SUPPORTERS NEWS 

How comprehensive care creates value for individuals and society 

(Baxter Symposium held at the 23 th EHC-conference in Lisbon) 

Comprehensive care adds significant value 

for individuals and society and requires 

appropriate funding. As the management 

of haemophilia is complex, patients should 

have access to a range of services, which 

are described by the European Principles 

of Haemophilia Care. Clinicians, patient 

groups and industry must make their voice 

heard so that payers not only know the 

cost of treatment, but also understand its 

values. This was the strong opinion of the 

speakers at the Baxter symposium. 

(f.l.t.r.: Declan Noone, Kate Khair, Brian O’Mahony, 

Prof. Cedric Hermans) 


Today, effective treatment of haemophilia goes far beyond stopping acute bleeding. 

Comprehensive care helps patients lead an active live that is as normal as possible. At the EHC- 

Conference 2010 in Lisbon on October the 23th a symposium, sponsored by Baxter, focused 

precisely on this question: “Governments know the cost of treatment, but do they know the 

value?” 

Prevention of life-threatening bleeds and joint damage, fewer missed work days, freedom from 

pain and anxiety – these are just some examples of how comprehensive care creates value. Its 

cornerstones are described in the European Principles of Haemophilia Care which were 

developed by an interdisciplinary working group of 45 leading haemophilia physicians from 19 

European countries. The Principles call for a national haemophilia organization, patient registries, 

comprehensive care centres, the supply of safe medicines, home treatment, prophylaxis and 

education and research programs in every country. 

Level of funding determines quality of healthcare 

“Funding is the biggest determinant of the quality of healthcare provided in any country,” said 

Professor Cedric Hermans of the Catholic University of Louvain, Belgium. Despite much variation 

in healthcare systems across Europe, every country is experiencing increasing pressure on 

healthcare funding, which may impact patient care, prescriber freedom and research and 

innovation in haemophilia. 

At the same time, major challenges to maintaining and improving the standard of care in this 

region need to be resolved. “To ensure consistency of care throughout Europe, increasing 

access to recombinant products, prophylaxis and immune tolerance therapy has to be made 

available,” emphasized Cedric Hermans. “The implementation of the European Principles of 

Haemophilia Care should contribute to getting the best value out of haemophilia care.” 

A variety of EU patient interest groups and medical organizations increasingly lobby for adequate 

funding to ensure access to care. However, unlike other medical conditions, haemophilia is a 

lifelong disease which requires extensive care and careful management. Adequate funding 

needs to be protected to not only preserve patients’ physical care, but also their overall quality of 

life for their lifetime. 

Cedric Hermans called upon the audience to make their voices heard so that comprehensive 

care, as described by the European Principles, is ensured: “Clinicians, patient groups and 

industry must educate payers on haemophilia and engage with them so that physicians and 

patients are heard when funding decisions are made.” 

Home care – better quality of life and fewer hospital visits 

Home therapy as a treatment option has existed for 30 years. It is a compelling story of living a 

nearly normal life despite a severe disease. Access to home treatment and delivery is one of the 

European Principles of Haemophilia Care. 


In her presentation on home care, Kate Khair, a nurse at the Great Ormond Street Hospital for 

Children in London, explained what a difference home care has made in the life of Jenny and 

James: James was 18 months old when he first came to the hospital. His three-and-a-half-yearold 

sister Jenny had to accompany him on his many visits and could not go to nursery. A nearly 

normal life for Jenny only began when James started home treatment. 

As a result of home care, the family caregivers generally experience some tangible benefits too: 

due to the reduced number of hospital stays, they have to take less time off work and disruption 

to family life is minimised. Moreover, fewer hospital visits have a clear financial advantage. Kate 

Khair: "Home care has saved Great Ormond Street Hospital £1.3 million per year for the past six 

years.” 

Home care is also a continuous learning experience for patients, physicians, nurses and 

caregivers who become valued partners of the healthcare team. Today, comprehensive home 

care programs educate patients and caregivers about the treatment, the medicines and most 

importantly when to contact a centre for help and support. 

New innovations are being developed to make home treatment as easy as possible, for instance, 

through home delivery of products, physiotherapy and smartphone applications that help to 

ensure compliance. Kate Khair summarized: “Home care is here to stay, we can prove it is 

effective and benefits patients and their families.” 

The foundation of comprehensive care – safe and effective medicine 

Factor replacements have revolutionized the treatment of haemophilia. The European Principles 

of Haemophilia Care refer to medicines as one important element of comprehensive care: 

“People with haemophilia need to have access to safe and effective treatment at optimum 

levels.” 

The economic value of safe and effective medicines is huge: fewer orthopaedic surgeries and 

days in hospital, less time taken off work and education, the ability to pay taxes and generally to 

be an active member of society are just some examples. “In the past, unsafe medications 

resulted in high costs because patients needed, for instance, to be treated for HIV and hepatitis,” 

Declan Noone, a person with haemophilia and member of the Irish Haemophilia Society in 

Dublin, Ireland, reminded the audience. 

Criteria for selection of a specific product include safety, quality and efficacy. Declan Noone 

concluded his presentation with a personal plea: “The human cost of safe and effective treatment 

should always outweigh the monetary costs.” 

Prophylaxis for adults – better quality of life and fewer days off work 

The benefits of primary prophylaxis for children are now widely accepted: children with severe 

haemophilia who begin prophylactic treatment at an early age and continue into early adulthood 

have very little or no joint damage and excellent quality of life . Many European countries 

recognize this fact by making prophylactic treatment available for children and even adults. One 

example is Sweden: the quality of life of young adults with haemophilia in Sweden today is 

significantly better than in other countries due to early and sustained prophylaxis. 

“Prophylaxis for adults should also be considered,” stated Brian O’Mahony, Chief Executive of 

the Irish Haemophilia Society in Dublin, Ireland, in his presentation. “Secondary or intermittent 

prophylaxis in adults is beneficial and cost effective. It should be looked at on a case-by-case 

basis.” 


Study results support his view. As demonstrated in Italy and the UK, secondary prophylaxis was 

effective in preventing total bleeds and joint bleeds in adults with severe haemophilia . Access to 

prophylactic therapy is also one of the European Principles of Haemophilia Care. 

Whereas some benefits, for instance “missed work days”, are easily measurable and can be 

balanced with costs, others are not. Avoidance of pain and suffering, the pursuit of an education 

or career, raising a family, enjoying normal activities, the ability to travel and freedom from 

anxiety and restrictions – the list of benefits without a price tag is long. Brian O’Mahony 

concluded his presentation by commenting: “We need to work to ensure that governments, 

health ministries and health economists focus on the value of haemophilia treatment in addition 

to cost.” 

Expert Meeting on von Willebrand disease on Åland Islands 

Thirty-eight experts from 18 countries gathered on the Åland Island Meeting to discuss the treatment 

and science of von Willebrand disease on 22nd- 25th September, 2010, on invitation of Prof. Erik 

Berntorp, University of Lund, Sweden with financial support from Octapharma . 

One of the first patients described with von Willebrand disease was Hjördis S., a little girl from the 

Åland Islands, located halfway between Finland and Sweden in the Baltic Sea. In 1924, she was taken 

to the doctor because of a history of serious bleeds. Only one year old, she fell and had a nose bleed 

for an “unusually long time”. At age three, she fell again and had a cut in her upper lip. After three 

days of heavy bleeding, the child became almost unconscious and recovered only after 10 weeks in 

bed. Other family members also suffered from severe bleeds. All but two of her 11 siblings had a 

history of bleeding. Three of Hjördis’s siblings died at age two and four from uncontrolled bleeds. 

Erik von Willebrand published his observations of Hjördis family and other families on Åland islands 

what he called “hereditary pseudohaemophilia”, occurring equally in men and women, and showing 


a strong family manifestation. He described the new bleeding disorder in the publication ”Hereditary 

Pseudohaemophilia in ”Finska Läkaresällskapets Handlingar” in 1926. 

Tragically, Hjördis bled to death during her fourth menstrual period only 14 years old. But the 

publication on her disease initiated research that furthered significant developments in the field of 

von Willebrand disease over the world. 

International experts therefore chose this historic scene to meet and discuss aspects of VWD 

research in eight sessions covering biochemistry, immunology, diagnosis, classification, genetics, 

treatment and disease management, gene therapy, and current guidelines for the treatment of 

VWD. 

The four-day meeting also included a study excursion to the Föglö Islands visiting the house of 

Hjördis. Exceptional lectures given by Otto Lindberg, the great grandson of E. von Willebrand 

familiarizing the audience not only with the biography but also with the person Erik. von Willebrand 

and Margareta Blombäck, a pioneer in the research on VWD, made this journey an extraordinary 

experience. 

The closing session gave an overview of the different national and regional guidelines for the 

treatment of VWD and resulted in the decision to institute the Åland Island working group with 

drafting an up-to-date European guideline based on the results of the Åland Islands Meeting in 

cooperation with EAHAD (European Association of Haemophilia and Allied 

Disorders). 

Proceedings are planned to be published in the beginning of 2011. 

EU HEALTH POLICY UPDATE 

European Parliament adopts second reading the draft 

directive on patient rights in cross-border healthcare 

On 27 October the European Parliament’s Public Health Committee 

adopted in second reading (by 47 votes to 2 with 1 abstention) the 

draft report by MEP Françoise Grossetête (EPP, FR), on the 

proposal for a directive on patients rights in cross border 

healthcare. This second vote reading pave the way for the vote in 

plenary of the draft legislation, scheduled for 18 January 2011. 

The vote follows the adoption by the Council on a compromised text in first reading under the lead 

of the Belgium Presidency on 13 September. The compromise reached by the Council reflects the 

Council's intention to fully respect the case law of the European Court of Justice on the patients' 

rights in cross-border healthcare while preserving Member States' rights to organise their own 

healthcare systems. The vote in Committee also reflects the will by MEPs to guarantee that patients 

covered for a treatment in their home country can receive the same treatment in another EU 

country, and be reimbursed without prior authorisation up to the level they would have received in 

their home country. 

“The European Parliament wished to avoid the risk of legal uncertainty for the patient, stating clearly 

the cases where a Member State may refuse a reimbursement or the right to seek treatment 

abroad", said Rapporteur Françoise Grossetête. 


Most importantly, MEPs decided to add special rules for patients with rare diseases, stressing that 

"patients affected or suspected to be affected by rare diseases have the right to access healthcare in 

another Member State and to receive reimbursement even if the diagnosis and/or treatment in 

question is not provided for by the legislation" of the home Member State. Such treatment should 

however, be subject to prior authorisation. 

According to a Eurobarometer study, 4% of Europeans received medical treatment in another EU 

Member State in 2006-2007. Cross-border healthcare is in fact more frequent for the treatment of 

rare diseases and in border regions, smaller Member States and areas with large numbers of 

tourists. 

Next Steps 

� 18 January 2011: EP plenary sitting, 2nd reading (indicative date) 

Additional information 

� EP Draft Recommendation for Second Reading: 

http://www.europarl.europa.eu/sides/getDoc.do?pubRef=-//EP//NONSGML+COMPARL+PE- 

443.081+02+DOC+PDF+V0//EN&language=EN 

� The Council first-reading position on a draft Directive concerning the application of patients' 

rights in cross-border healthcare: 

http://www.consilium.europa.eu/uedocs/cms_Data/docs/pressdata/en/lsa/116482.pdf 

Better protection of patients in the use medicines: the 

European Parliament adopts a legislation on 

pharmacovigilance 

Substantial progress was made on the pharmacovigilance dossier of the 

“pharmaceutical package” leading to its adoption at the European 

Parliament plenary session on 22 September 2010. 

The European Parliament report drafted by Rapporteur Linda McAvan, 

adopted by the Parliament on 22 September strengthens the initial 

proposal of the European Commission towards more public health safety, transparency and 

communication on medicine’s safety issues. In particular, it empowers patients who will be able to 

report adverse effects of new medicines using national web portals and a European portal. All new 

or updated medicines will be intensely monitored for five years, and doctors and patients will be 

informed and encouraged to report all problems. 

The Community pharmacovigilance database, “EudraVigilance” will also be further developed and 

strengthened, to become a single point of receipt for pharmacovigilance information. 

Besides, post-authorisation safety studies will be conducted in real-world populations and real-life 

conditions. The new legislation will also allow for stricter, but simpler rules on monitoring and 

reporting by drug companies. 


Finally, a “Pharmacovigilance Risk Assessment Committee” will be created within the EMA, which 

will be solely responsible for pharmacovigilance and risk assessment, and will be made up of one 

representative per member state, six additional members appointed by the Commission, a patients' 

representative and another representative of the health sector. The European Medicines Agency 

welcomed the vote: “this is a major step towards the legislation coming into force, currently 

expected for mid-2012”. 

Next Steps 

� Council to formally endorse the proposal 

� Legislation come into force mid 2012 

Additional Information 

� Please click here to access the ENVI report of Linda McAvan on pharmacovigilance 

Progress on Information to Patients proposal 

On 28 September 2010, the Environment and Health Committee voted on the Information 

to Patient report, opening the way forward for the adoption of the second legislative 

proposal of the “Pharmaceutical Package”. 

The draft report of Rapporteur Christopher Fjellner (EPP, Sweden) was adopted by a 

majority of MEPs in the ENVI Committee. The new draft focuses on patients’ rights 

rather than industry’s rights. It introduces in particular amendments to the legislation, 

which oblige Member States to ensure that "objective, unbiased information" is 

available to the general public on medicinal products sold in that Member State. 

Information should include: a summary of product characteristics, a labeling and package 

leaflet, a publicly accessible version of the assessment report of the medicinal product, the diseases 

and health conditions which are to be treated with the medicinal product, and information on how 

to prevent such diseases and conditions. 

Pharmaceutical companies should be required, and not just have the option as in the Commission 

proposal, to make available the approved and most recent contents of summaries of product 

characteristics, labelling and package leaflet and a publicly accessible version of the assessment 

report. 

In addition, pharmaceutical companies will be allowed to provide the general public with other welldefined 

non-promotional information on the environmental impact of the product or its effect on 

prices, following prior authorisation from the competent authorities. 

This information would have to be supplied both in electronic and printed form and appropriate 

format for the disabled, but it will be banned from printed newspapers or magazines. 

Besides, MEPs voted that the Commission will have to produce a report on Member States’ practices 

three years of the legislation enters into force. The Commission will also have to publish an 

assessment of improvements that could be made to the summary of product characteristics and the 

information leaflet after two years. 

Ahead of the plenary vote on 24 November, the Association Internationale de la Mutualité (AIM), 

Health Action International (HAI) Europe, the International Society of Drug Bulletins (ISDB) and the 


Medicines in Europe Forum (MiEF) have called MEPs to oppose the Commission’s proposal with 

regard to the public provision of information relating to drugs subjected to medical regulation, 

specifically for a directive to modify Directive 2001/83/CE and a regulation to modify (EC)726/2004. 

According to them, the proposal would generate more bureaucracy, expose patients to greater risks 

and bring additional costs for member states, which would have to deal with the consequences. 

Next Steps 

� 24 November: first reading vote of the European Parliament plenary 

European Health Forum Gastein addresses healthy ageing 

The 13 th European Health Forum 

Gastein (EHFG) was held on 6-9 

October 2010 and focused on 

“Creating a better future for Health”. 

Issues such as health literacy and investment in a healthy workforce, the growing ageing population, 

cost-saving measures were tackled. On the issue of ageing, EHFG president Prof. Günther Leiner, 

called the EU and governments to put in place a European fund for the aged that would help 

compensate for the deficit in available care for older people. “Everyone should be able to age in 

dignity in Europe, not just in a few member countries” he stressed. He believes such a fund could be 

created at the image of the European Regional Development Fund (EFRE) which aims to reduce 

economic development inequalities across European regions. He also believes that “such a measure 

would be fully within the spirit of the European Commission’s Europe 2020 strategy, a leading 

initiative for ensuring economic, social and territorial cohesion by helping the poor and socially 

excluded.” 

(poster presented by EUHASS at the Forum) 

Health Commissioner Dalli also addressed the issue of 

ageing in his keynote speech. He mentioned in particular 

that as part of the European Commission initiative on an 

“Innovation Union” by 2020, an “Innovation Partnership 

on Healthy and Active Ageing” will be created. 

Commissioner Dalli stressed that the aim of the 

partnership is to “increase by two years the number of 

years that Europeans can live an active and healthy life.” 

The new initiative will bring together governments, civil 

society, industry and the European Commission to deliver 

concrete innovative solutions to meet the needs of 

patients. 

(Poster made and presented by EUHASS at the Forum) 

Additional information 

Information on the 13 th European Health Forum Gastein: 

http://www.ehfg.org/index.php 


European Medicine Agency publishes first review of orphan 

designation documents 

The Agency published on 17 September the first of its ‘review of 

orphan designation’ documents. These documents summarise 

the review of the orphan designation carried out by the 

Committee for Orphan Medicinal Products (COMP) whenever an 

orphan medicine reaches marketing authorisation. The review is 

carried out to check that the criteria underpinning the 

medicine’s orphan designation still apply. 

The publication of these documents has been introduced in order to increase transparency over the 

Agency’s orphan designation process. The documents summarise the COMP’s position on whether 

the orphan designation for a medicinal product that is receiving marketing authorisation should be 

maintained or revoked, and include a discussion on the justification of benefit over other authorised 

treatments. 

The first review document concerns Vpriv (velaglucerase alfa), which was authorised for the 

treatment of Gaucher disease on 26 August 2010. The COMP concluded that Vpriv’s orphan 

designation can be maintained on 8 July 2010. 

The Agency will continue to publish these documents for all orphan medicinal products at the time 

of marketing authorisation, and for all extensions of indication approved for orphan medicines 

already on the market. 

Additional Information: 

The EMA press release and supporting materials are available at : 

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2010/09/news_det 

ail_001114.jsp&murl=menus/news_and_events/news_and_events.jsp&mid=WC0b01ac058004d5c1 


OTHER EVENTS OF INTEREST 

EUROPLAN rare disease national conferences, May- 

December 2010, across Europe 

15 national conferences organized within the framework of EUROPLAN, a EU funded project, to 

promote national plans and strategies for rare diseases based on EU Council Recommendation 

For more information here 

11th Partnering for Rare Diseases Therapy Development – 

Workshop, 29-30 November, Prague, Czech Republic 

The workshop will concentrate on: 

� Science - the determinants of research in rare disease in Europe: 

understanding the current and future European Research and 

Development challenges and opportunities. 

� Regulation - the Europe and beyond: developing future regulations 

and policies for better rare disease therapies. 

� Sustainable Access - The reality we face to improve access to and 

affordability of orphans. 


Please note that the World Orphan Drug Congress will take place on Monday 29 November - 

Wednesday 1 December, Crowne Plaza, Geneva, Switzerland. 

Please click here to access all information about the congress. 


A European Strategy for Chronic Conditions – Presidency 

Conference, 9 December, Brussels 


EPPOSI invites all stakeholders to its Second Workshop on Chronic 

Conditions to help formulate the principal elements of a holistic new 

model which can actively reduce the debilitating personal and economic 

burden of chronic conditions. The model will be developed by a Task 

Force, to be set up after the workshop and based on its outcomes, 

which can be adapted and implemented in all Member States. 

2nd Workshop on Registries for Rare Disorders, 25-26 

January 2011, Brussels, Belgium 

The workshop will focus on: 

� The use of registries to support patients access 

� The use of registries to meet payers' needs 

� The role of registries to support scientific advances 


4 th Annual Congress of EAHAD, 20-4 February 211, Geneva 

More information see www.eahad2011.ch

2010 November - EHC

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