Network, capacity building & education programmes
BUILDING STRONGER CF ASSOCIATIONS for better acces to care

CFE is convinced that better access to better CF care can only be ensured by building strong local associations and networks.  Therefore we organize network and capacity building meeting where CF representatives from across Europe can exchange experiences and receive training in all aspects relating to building strong, representative and successful patient associations. CFE also organizes, supports or participates regularly in regional and national education programmes on CF care (physiotherapy, nutrition, treatment...) and functioning of a national association (lobbying, fundraising, information...) for CF associations, families, medical and allied health professionals.

3rd Pan-European CF network building conference on June 8 and 9, 2009 in Paris
Cystic Fibrosis Europe organized its third European CF network building conference on June 8 and 9, 2009 at Vaincre la Mucoviscidose in Paris. 25 CF patient associations from 24 European countries attended in a very dynamic and interactive meeting. The primary aim was to support capacity and network building, define the needs and develop strategies to improve care and quality of life for people with CF throughout Europe. We focused on exchange about successful experiences & existing problems of CF associations, building strong patient associations and European collaboration with special workshops and training on media campaigns and lobbying for better access to care.
To ensure that association from all over Europe could afford to attend the meeting, participation was free.  We all enjoyed the nice facilities and food offered by the French CF association.
Sharing experiences, learning from each other and realising that every association in Europe is fighting to improve the quality of life and CF care proved a very stimulating experience. 
Participants believed progress is possible and took home a lot of ideas to make this happen.

CF Seminar Moscow 2-3 July 2009
CFE helped the CF association and foundation to organise a CF seminar in Moscow during the Europeadiatrics Conference in Moscow in July.
The meeting was very well organised and it was a great pleasure to meeting so many dynamic people fighting for better and longer lives for people with CF in Russia. I was most pleasantly surprised by some of the people working in the clinic, the head of the lung department was really a dream doctor, such a huge heart AND expertise. The actual situation is better than expected, knowing the harsh conditions limiting access to quality care in the country, thanks to the families active in the association and foundation: Irina, Vladimir and other parents and a few very good, motivated health care professionals active in several Russian regions.  Thanks to a mayor who is very social and understanding, in Moscow all families seem to have access to care (although it takes a lot of time and bureaucracy to get the necessary care and medication).  But there are no physiotherapists who want to work with CF (they’re all afraid of infections). President Medjedev opened the paediatric conference with a nice, supportive, child oriented speech, but access to CF care is really bad in some regions (e.g. in some far away regions -since they introduced neonatal screening they detected several baby’s with CF but they all died within a year; some families have no access to nebulizers, creon etc).  Luckily Irina acts as a good ‘Godmother’ for all Russian families and fights for each and everyone to have to basic care they need, together with parents (mostly mothers) from around the country. 
But there is still a lot of work going on, the families have to fight constantly for access to care, to keep CF on the rare disease agenda, to obtain the results they obtained (e.g. Creon was taken of the list of medication recently).
This Russian example again showed that a regional approach and plan, designed together with local families is essential.  A physiotherapy training for instance must first address the fear of bacteria and infection of other patients before focusing on techniques...

“On behalf of all cystic fibrosis patients of  Russia and their families  I`d like to  cordially thank  Cystic Fibrosis Europe and   you  personally   for your kind  help in organizing master classes in lung respiratory  techniques  (kineziotherapy) and nutrition in CF which took place at Children`s Health Center in Moscow, July 2nd,2009. 34 doctors working with CF patients in different parts of Russia, a doctor from Moldavia  and 42 family members of Russian CF patients and  3 mothers of CF children from the Ukraine took part in the seminar. The knowledge they got from the master classes was very important  for making CF care in Russia  and  in those countries better.I am certain that this will  make lives of  Russian CF patients longer and better.I hope that we will continue our collaboration in helping CF patients in the future.”
Irina Myasnikova, President of NGO “Help to CF Patients”, Moscow, Russia, July,7th, 2009 

CFE also helped the Bulgarian CF association to organise a CF seminar in Plovdiv during the rare Diseases meeting there in May 2010. It was the very first meeting ever organised for CF professionals and families, but the meeting was a major success.  It was very well organised and organized nearly 100 families (a high number considering only 170 patients are diagnosed with CF in the country). Unfortunately not many health professionals attended.  Only Dr Galeva (Sofia), since long interested in CF, a young paediatrician, now working on a thesis about CF and 2 physiotherapists who voluntarily work with CF patients were present.  Officially 4 CF clinics exist in the country but I get the impression only the physician in Sofia has a good knowledge about CF care. Unfortunately she can’t ensure regular follow-up according to CF standards of care, because of the administrative regulations in the clinic and Bulgaria she can only attend certain patients when hospitalised.
But I was pleasantly surprised by the interest and level of understanding of the families who attended the conference.  Their hunger for more knowledge and understanding of the disease and treatment was big and it was clear they had managed to obtain a lot of information through their own resources.  But of course reading about treatment on the internet etc is not sufficient and may be misleading, so this first medical course and physiotherapy training by international experts was very much needed and appreciated.  Karleen could also stimulate the families to collaborate in building a strong association as the main way towards better access to care. In theory Bulgaria is well advanced in its rare disease strategy, being applauded by the EC for its national plan on rare diseases, but in reality access to care is very difficult for all rare disease patients.  One of the parents expressed it very strongly: ‘I’d wish we were only fighting against a wall, at least a wall can be broken through sometimes; but for us we don’t even know whom we’re fighting, whom we can talk to, negotiate and lobby...’ CFE will continue working with the association to find ways to improve care and access to care.
 “On behalf of the Bulgarian Association “Mucoviscidosis,” please accept our sincere thanks for your participation in the First Bulgarian Educational Seminar on Cystic Fibrosis, May 29, 2010 in Plovdiv. Your presentations were profoundly informative and expertly delivered, and contributed greatly to the event’s success. Your leadership and personal involvement sets a very high standard for commitment to the mission of preserving and improving the lives of people with cystic fibrosis, and is an inspiring example to all of us. We deeply appreciate all the time and effort you invested in preparing for and traveling to attend our event, and all your generous support. We look forward to continue working with you to enhance the standard of care for CF in Bulgaria. Thank you again for all you have done for us!” Svetlana, secretary CF Association in Bulgaria

CFE provided nebulizers and physiotherapy devices such as flutters for all participating families. This enables the families to have a hands-on training with the necessary equipment and to practice what they leanred at home with the CF patients. This was made possible thanks to a donation of Romedic (Netherlands-nebulizers) and the Belgian CF association (flutter+transport costs of all equipment).

CFE has also put a lot of time and effort in helping Anja Kladar, president of the Croation association, to prepare the Southeastern Europe CF Conference to be held from 1 to 3 October 2010 in Dubrovnik, where more than 300 people are expected to attend the lay, allied health and medical seminars.
So far the programme (for CF associations, physicians and allied health) looks very promising with renowned international speakers on pulmonology, gastro-enterology, physiotherapy, nutrition, psychology... so block these dates in your agenda now!  As soon as the Croatian association has finalised the programme we’ll send it around to you all with more practical information about registering and attending this meeting.

Unfortunately the V4-CF conference in Budapest was cancelled this year because the organizers feared the date chosen for the Southeast European conference was too close to theirs to attract sufficient participants.  We are very sorry about this problem, due to miscommunication and misunderstanding about the dates.  We hope to be able to collaborate for the organization of the next V4-CF conference at a later date.

We started plan the next Southeastern European CF conference in Romania in 2011.

David Versteeg, leader of OEF, a CF adult group in the Netherlands, has been very supportive of our solidarity actions for Eastern Europe, by stimulating companies to donate free nebulizers and looking for sponsoring for our educational programme (we’re waiting for the results now). Website: www.oef.nl

Better awareness and more support for Cystic Fibrosis patients in Europe

From 9 to 15 November 2009 Cystic Fibrosis Europe organized its 1st  European CF Awareness Week.  Our goal was to fight for equal access to appropriate care for all children and people living with this disease in Europe. The CF care in Europe can and has to be improved.  Early diagnosis, regular follow-up by specialists, correct treatment of early symptoms and proper hygiene can prolong and save lives, but also save extra health costs. In several countries basic treatments are not available while other, more expensive but less essential, medication is reimbursed. Ongoing efforts of CF patients, families, associations and health care workers will lead to better and more accessible CF care, and better and longer lives for all CF patients in Europe.  But the patients don’t have the time to wait too long...  We also wanted to make the public aware of Cystic Fibrosis and the problems patients have to cope with.  Although 1 of 30 Europeans carries the gene that can cause Cystic Fibrosis few people know the disease and the patients and their families have to fight for understanding and support every day. All over Europe CF associations took this opportunity to make the public and politicians in their country aware of the needs of people with CF. More than 20 countries reported on media, lobbying and fundraising campaigns in their country.
A very successful event was held in the European parliament on November 11 with patient representatives from 15 countries and MEPs from 11 countries attending!

200.000 CF solidarity bracelets were distributed all over Europe, making people aware of the fight for better and longer lives of CF patients. In total 170 events and dozens of publications/TV showings were registered on the special action website www.cfweek.eu  (although the majority were from Belgium) and 33 movies posted on Youtube.

The website was visited by more than  50.000 people last year. The statistics show that the site was still being visited frequently in the beginning of 2010. The site is a very good instrument to inform and motivate people...it cost us a lot of time and effort and it’s a pity that your input and activities are not to be found on it.

Survey on “Comparative demographics of the European cystic fibrosis population: a cross-sectional database analysis” published in The Lancet 2010; 375: 1007–13.

Pan-European research led by the University of Dundee has led to an impassioned plea being issued to close a healthcare gap amounting to a “death sentence” for Cystic Fibrosis (CF) children born in Eastern Europe.
Dr Anil Mehta, from the University’s Centre for Cardiovascular & Lung Biology, led a team from 35 countries that examined outcomes for almost 30,000 CF patients.  The resulting paper in The Lancet, the world renown leading medical paper, shows that there is a striking difference in number and health status between CF-patients in the old EU-countries (members of the EU before 2003) and in the countries joining after 2003 despite similar population sizes and underlying gene frequencies for CF. In the new EU-countries there were far fewer children and young people with CF than could be expected, i.e. they die earlier and are diagnosed later.
Dr Mehta presented his results for the first time in the European Parliament during the CFE meeting organized during the CF Week in November and we continue collaborating to inform the European Parliament and stimulate them to take action to diminish the gap and improve access to care.
We, the board of CFE, do welcome this survey and appreciate the dedicated work performed by Dr. Anil Mehta and his group. We are most grateful to the European Community’s Sixth Framework Programme for Research, and the Czech Ministry of Health for sponsoring such an important study. In the past our members could only assert that care and treatment were poorer in the new EU countries; now they are able to prove it with data and that better results are possible. That is a great step forward.

We are now looking for a MEP who will present a question to the European parliament concerning the status of CF care and treatment and patients’ access to it in the different European member countries. The members of the European parliament should be informed about these findings given that the European commission funded this research. Cystic fibrosis can now be used as a key example in the impact of the European Commission Communication of the in the field of rare diseases. This could create conditions for a quantum leap towards better treatment and care for patients affected by rare diseases.
CF is a condition which can be treated well today at reasonable cost (as shown in an earlier Lancet paper from 2007, also from Dr Mehta), as the data in this latest European Registry survey show. We think that this fact should be brought to the knowledge of the European parliament members. It must not be that in Europe the country where you are born as a CF-patient determines whether you are able to live or will die prematurely.

You find more info in the press release in the annex ‘EU health news’. The article in The Lancet unfortunately is not yet free, but you can listen to the podcast on the Homepage of The Lancet: http://download.thelancet.com/flatcontentassets/audio/lancet/2010/9719_20march.mp3

10th anniversary of the European Orphan Drug Regulation

The European Medicines Agency (EMA) celebrated the tenth anniversary of Regulation (EC) No 141/2000 of the European Parliament and of the Council on Orphan Medicinal Products (Orphan Drug Regulation) which came into effect in early 2000, along with Commission Regulation (EC) No 847/2000 defining the provisions for implementation of the criteria for orphan designation. The legislation was brought forward in order to improve the offer of products for patients with rare conditions by providing drug developers with a bouquet of economic and regulatory incentives, including 10-year market exclusivity, protocol assistance, and access to the Centralised Procedure for Marketing Authorisation.

CFE participated in several meetings and surveys celebrating but most of all evaluating this regulation and looking for further progress in the years to come.

EPPOSI: 10th Workshop on Partnering for Rare Disease Therapy Development 
The topic of this year’s workshop on 26-27 October 2009 in Brussels, Belgium of the European Platform for Patients’ Organisations, Science and Industry is “10 years after the adoption of the EU Orphan Medicines Regulation: Where do we go? Three key themes were addressed: “What is the impact of the economic crisis on the field of rare diseases – and what is the vision on further progress in R&D, diagnosis and patient care? Building on the public policy base of the last 10 years: how to advance policies in the next five years? Rare cancers: specific challenges within rare diseases.

Karleen attended the event: "Together for RareDiseases", 1 June 2010 in the  European Parliament, Brussels, hosted by MEP Grossetête on  “Together for Rare Diseases” held at the European Parliament on 1 June 2010 to celebrate the 10 years of success of the EU Regulation on orphan medicines. Françoise Grossetête, MEP showed the positive impact of the OMP Regulation for rare disease patients and patients’ expectations for the future. Yann le Cam, CEO, Rare Diseases Europe (Eurordis) discussed the future for rare diseases and orphan medicines in Europe following the EC Communication and Council Recommendation on rare diseases. 

In light of the 10th anniversary the European Medicines Agency (EMA, formerly known as EMEA) underwent a year-long evaluation process. "Both the industry and other stakeholders tend to agree that the creation of COMP and related incentives have had a positive impact on research and development for specific products for orphan diseases. ...” Although the current orphan products policy is recognized as having very positive outcomes, where CF is concerned, the clinical development of new therapeutic agents for CF is hampered by the lack of a final guideline of EMA. It does not appear that a lot of progress has been made in moving forward the draft guidance issued in 2008. Clearly, a regulatory guidance helping to overcome important barriers in clinical development (e.g. defining meaningful yet achievable clinical endpoints) is of great importance to bring new therapies to patients as quickly and as safely as possible.
By establishing the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN), where CFE is an active partner, the ECFS has made an important step towards efficient and high-quality clinical development for CF in Europe. Creating the ECFS European CF-Therapeutic Development Task Force (Euro-CFTDT) should help improving the context for regulatory decisions in the field of CF drug development in Europe, by providing scientifically sound and convincing information.  CFE is involved in both the CTN and the creation of the Task Force (see next pages).

CFE as partner of clinicians and researchers

CFE is more and more recognized as patient representative and as such we’re involved in common European projects, where we represent and defend the interest of people with CF and their families.

3rd European CF Young Investigator’s Meeting (EYIM) - France, 08.2009

The 3rd European CF Young Investigator Meeting was held in Lille (France) from 25 – 28 August 2009. For this 2009 edition CFE invited all member associations from across Europe to send out a call for abstracts to their young investigator’s resulting in candidates from 12 countries (from as far north as Sweden and as far south as Oman and from France to Ukraine!). This scientific meeting is organized in collaboration with the CF associations of France, Germany, Holland, Belgium and Italy and the ECFS. It aims at fostering interactions between young scientists in order to create a long-term collaborative European network of CF investigators. We stimulate young researchers to continue investing in CF Research by offering them a forum for exchange and a change to present their work at an international meeting, covering all fields in research and clinical CF research. Fifty young investigators (under 35 year-old) are selected by an international scientific committee in order to present their work on CF. Sessions per topic are introduced and moderated by leading European senior scientists. The EYIM provides a unique opportunity for young scientists for training, sharing experiences and networking. Enclosed you find a report of the Young Researcher Olena Bilevych who had to travel the longest distance to attend the meeting, all the way from Ukraine.

ECFS (European Cystic Fibrosis Society)
(www.ecfsoc.org)

CFE works together with ECFS, the organization of CF professionals in several programmes and projects, mainly:

• Patient’s point of view on conferences
• Participation in European consensus meetings & surveys:
• European CF registry
• European Clinical Trial Network
• Collaboration in EU projects

For the Brest Conference (06/2009) Franck Dufour, CFE Board member, was part of the scientific committee. This way CFE can promote topics which can improve the quality of care of CF patients and present the patient’s point of view in the scientific conference. Franck presented the role of patient associations and CFE in clinical trials in a well attended plenary session.

ECFS Patient Registry

Angeliki Preftitsi represents CFE in the European CF Registry Steering Committee and reports on the meetings  of June 11, 2009  in Brest and  the 25th of January 2010, in Amsterdam (detailed report in annex).
The Eurocare CF WP2 Registry came to an end on the 30th of June 2009. The report for EU has been finalized with anonymous demographic data of more than 30 countries which have been collected over a period from 2003 to 2008. Following the end of the EurocareCF project, the members of the steering Committee decided to rename the Registry with an ECFS related name: ‘ECFS Patient Registry’, instead of using the name ‘ECFS Registry’ or ‘European CF patients Registry’, since ECFS is the data user of this registry that consists of CF patients’ data.

They also decided to abandon PortCF, the registry software that has been given for free by the American Cystic Fibrosis Foundation to ECFS. Unfortunately, several reasons, lead to its abandonment, mainly because the technical support from CFF was too expensive and belated and there were still remaining many other issues that  needed to be solved, in order to use  this software.
The new software that was selected was the Registry Software that has been used for a lot of years by Italian CF centers, complies with the EU rules and patients’ data can be codified at a center level and then sent to the central server at the University of Milan in Italy. From the 1st of February the new version of the software is available at the net. The software’s name is ‘ECFRecord’, it was created by the Italian company and anyone who would like to participate in the registry, can download it and use it.
During the meeting in Brest major interest of the ECFR Steering Committee members was focused on legal issues and issues regarding patients’ codes and anonymity.

A new ECFR website www.ecfregistry.eu presented. This site contains all the details about the registry formerly placed at the ECFS homepage, and from now on, it will be the direct online access site for the registry.
Furthermore, some clarification on data protection issues was added to the new guidelines (compliance with national data protection laws, ethics etc).
In Amsterdam the new composition of the Steering Committee has been discussed. The board will be partially renewed, replacing alternatively every 3 years two/three members.

It was decided to have two patient representatives, rather than one, appointed solely by CFE and not by CFWW, since ECFR interferes more with European patients. CFE has to appoint the second representative during the meeting in Valencia.
The possibilities of cooperation between CTN and ECFS Registry were discussed as well as the cooperation method between the researchers and ECFRegistry. It was decided that questions will be made to the European CF Registry Scientific board via a standard request form. As a preliminary scientific committee, the following members were appointed: Anil Mehta (elected member representative), Eitan Kerem (ECFS representative), Angeliki Preftitsi (patient representative and legal expert) and Laura Viviani (statistical expert

ECFS-CTN, the European Clinical Trial Network

In 2008, a milestone has been reached in our fight against CF: for the first time ever drugs aiming at the cause of the disaese, called ‘disease modifying drugs’ were shown to be non toxic, well tolerated and to have some efficacy in small and moderate size trials. They now need to be tested on large numbers of patients. In order to mobilize all resources to make these hopes become new efficient therapies and finally defeat CF safely and efficiently, ECFS has formed a ‘European clinical trial network’, the ECFS-CTN. The aim of this new network is to implement high quality clinical research in order to facilitate the progress of drugs from clinical trials to the patient. ECFS has now selected 18 CF centres across Europe fulfilling criteria of state of the art care of CF patients and showing commitment to undertake clinical research together. More CF centres will join the network within the next years. Together, they will be able to safely carry out clinical trials including large numbers of patients and this is critical to know quickly and reliably if a drug can be of real benefit to the patients.
CF Europe has been a partner of the ECFS-CTN from the very beginning. Promoting a positive attitude towards clinical research is one of the great challenges for our associations for the coming years. We invited member associations to inform patients about clinical research, about the coming treatments, the possible improvement in the patients' daily life and about possible new medications that these studies might bring to patients and show the importance of patients’ participation in clinical studies.
ECFS will provide most of the money to finance the Clinical trial Network and hopefully some money will be found in EU projects. But major support is also been forwarded by our member by supporting the partners of the network in your country and the network itself (€125.000 will be donated this year after the ECFS agreed to a memorandum of Understanding developed by all participating patient associations).

We hope that if the network is working well, with many patients participating and high standards of performance, the pharmaceutical industry will be more willing to invest in CF research and to work in partnership with CF clinicians and researchers. CFE Board member Franck Dufour was nominated as representative of the European patient association in the  Executive Committee (besides Kris De Boeck as chair, Harm Tiddens as co-chair, Steve Conway, Isabelle Fajac and Thomas Wagner as medical representatives) . Veerle Bulteel has been engaged as coordinator.
You can find more information about the network and how to get involved at the ECFS website www.ecfs.eu (under the ECFS Supported Initiates link), or contact ECFS-CTN@uz.kuleuven.ac.be.

ECFS Consensus Conference on Outcome Parameters in CF, 19-21 March 2010, Venice, Italy

Reto Weibel represented CFE and the patient’s point of view at the consensus meeting and reports.
“A new collaboration between medical professionals and their CF patients is developing, a new way of thinking is growing and the direct dialogue during the consensus conferenec in Venice seems a good sign of this development.
The principle is to bring expertise and experience together.  For the medicals people this is their great knowledge about CF in medical terms, our knowledge as patients is the daile experience how it feels to practice the many treatments as tehrapy or in clinical studies. As patients we often know too little about the thoughts, deeds and struggle for progress from physicians  a,d they know too little about our doubts and fears.
ECFS organizes Consensus Meetings to develop treatment guidelines. Now we have the opportunity to presnet a point of view medical specialists don’t know: the point of view of patients who experience this treatment. 
There was a good exchange between the medical specialists present at the meeting and the 2 patient representatives.  They involved us in their discussions and mostly followed our input.
But I was surprised to hear remarks such  as „in my daily work i hardly have contact with people with CF PWCF)“... “This point of view of this problem I didn’t know“... „Good you’re here, because I didn’t knwo that...”  It is clearly important to have this dialogue at these meetings and urgent for medical specialists and patients to talk more to each other.  It raises better understanding and diminishes conflict potential whic will ultimately lead to a more global, human and better treatment of the disease!”

ECFS European CF-Therapeutic Development Task Force (Euro-CFTDT)

The clinical development of new therapeutic agents for CF is hampered by the lack of a final guideline of the European Medicines Agency (EMA). It does not appear that a lot of progress has been made in moving forward the draft guidance issued in 2008. Clearly, a regulatory guidance helping to overcome important barriers in clinical development (e.g. defining meaningful yet achievable clinical endpoints) is of great importance to bring new therapies to patients as quickly and as safely as possible.
By establishing the European Cystic Fibrosis Society (ECFS) Clinical Trial Network (ECFS-CTN), the ECFS has made an important step towards efficient and high-quality clinical development for CF in Europe. ECFS wishes to join forces to keep the momentum also in the regulatory context.
The main objective of Euro-CFTDT is to help improving the context for regulatory decisions in the field of CF drug development in Europe. This is achieved by providing scientifically sound and convincing information to the EMA and applicable national competent authorities thereby helping these institutions to set up appropriate guidance documents and to discuss specific applications for marketing authorization related to CF.
The members, who should act as independent experts, are to be appointed by the Board of the ECFS. Two patient representatives will be nominated by Cystic Fibrosis Europe. A first meeting was planned for May 2010 in Paris.

CFE as PATIENT experts & DRUG DEVELOPMENT

EMA, the European Medicines Agency (formerly known as EMEA).
Ulrike Pypops (CF adult) and Karleen were invited as experts for 2 EMA dossiers: Anti-Pseudomonas IgY Colistimethate sodium (Colobreathe). CFE is now recognized as a partner and will be involved in future EMA dossiers concerning CF products.

Karleen and Ulrike Pypops were invited at a meeting with Vertex Pharmaceuticals at the Brest conference to disuss patient’s expectations and needs concerning clinical trials and the development of new medication.

Karleen represented CFE at Novartis International AG’s stakeholder dialogue on the 29th of September 2009 in Brussels, for a meeting of a group of experts from patient groups and senior topical experts from Novartis. As an international company with approximately 50 000 associates in Europe, Novartis wanted to engage with its stakeholders to help further refine the company’s Corporate Citizenship communications approach. 
Corporate Citizenship at Novartis follows a four-pillar approach, reflecting commitments to patients, ethical business conduct, people and communities, and environmental care. (a copy of the CC section of the 2008 Novartis Annual Report is available at http://novartis.com/annualreport2008).

ECORN-CF: European Centres of Reference Network for CF

This project was accepted by the EU and officially started on May 1, 2007 and finished in April 2010.
The aims of this project are:

1. access to info and expert advice for patients & families
2. expert advice for health care professionals
3. quality control system: ensuring similar level of quality and consensus for all answers independent of language and country/decide on priorities - where do we need a new consensus

CFE is an active partner in this project and is actually funded for its work within the project.
ECORN-CF is active in 11 European language regions now.

Birgit and Karleen represented CFE at the Quality Round Table and Consensus Meeting in Prague (6+ 7 Oct 2009). The Consensus paper "CF and Travelling" was finalized. CFE was responsible for editing the chapters Infrastructure Abroad (Patient Organisations, CF Competence, Hospitals, Emergencies), CF health/travel Insurance (incl. Repatriation) and Specific Documents (Customs, Forms, Medical Letter) but also took an active part in defending the patient’s point of view and interests in all other chapters.

ECORN-CF was finished in April 2010. Everyone agrees the project should be continued because it provides a good instrument for quality advice and information for patients, families and health professionals and for developing a consensus on patient related guidelines. 
Unfortunately the future of ECORN-CF is unsure.  TOF Wagner wants to pass on ECORN-CF to CFE... but is still looking for funding through new EU grants, support from ECFS and patient association. 
In the Annex on EU projects you can find some conclusions on ECORN-CF from a patient’s view by CFE secretary Birgit Dembski after this project has been running for almost three years.

ENCE-CF-LAM-LTX - European Networks of Centres of Expertise for CF (Cystic Fibrosis), LAM (Lymphangioleiomyomatosis) and LTX (Lung Transplantation)

The aim of the project (set up by Thomas Wagner, Frankfurt) is the drafting of a blueprint for the implementation of Networks of Centers of Expertise for Rare Diseases as exemplified by CF, LAM, and lung transplantation (LTX). Since the different factors have to be weighted differently for the respective diseases (there is almost no need for crossborder care in CF, but a substantial one in LTX, for instance), the three entities complement each other well as model entities for a blueprint. CFE is a collaborative partner.
Based on the European Commission’s DG Research call "HEALTH-2007-3.4-1: Disease networks of centres of reference", the main goal of ENCE-CF-LAM-LTX is to further the optimisation of the delivery of health care to European citizens by developing a general blueprint of how to implement quality management and guidelines for rare diseases and how to develop and build European Networks of Centres of Expertise for rare diseases. This will be done by looking at three exemplary rare entities: Cystic Fibrosis (CF), Pulmonary Lymphangioleiomyomatosis (LAM), and Lung Transplantation (LTX).
CFE is an associated partner in ENCE.

Birgit and Karleen participated for CFE in the ENCE workshop on "How to Build Expertise Networks in Europe" – held on September 23 & 24, 2009 in Frankfurt. The minutes, along with the presentations given and the documents mentioned during discussion, are available for download at www.ence-plan.eu.  ENCE has greatly profited from the discussions and exchange of information and the coordinators are busy working the participant’s feedback into the next steps of the project.

ENCE's first project report (April 2009 - March 2010) has been completed and submitted to the European Commission.

ENCE's goals for the first project year were mapping and analysis of available resources and structures and a scoping exercise to design a framework of what such networks should look like  A set of elements for the formation of such European networks of centres of expertise has been determined for each entity. Among these elements are patient registries, clinical trials networks, the involvement of patient organizations, basic research, biobanking, etc. In a next step, these criteria will be recorded in separate consensus statements for each patient group.

For more info see Annex EU projects.

IMPACTT (IgY project APPLICATION)  

“PROSPECTIVE, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND,MULTICENTER STUDY (PHASE III) TO EVALUATE CLINICAL EFFICACY ANDTOLERABILITY OF POLYCLONAL ANTI-PSEUDOMONAS CHICKEN ANTIBODIES (IgY) IN PREVENTION AND TREATMENT OF RECURRENCE OFPSEUDOMONAS AERUGINOSA INFECTIONS IN CYSTIC FIBROSIS (CF)PATIENTS”

IMPACCTT is currently being evaluated to be accepted as a EU granted project and mainly concerns a  study where children gargle with an egg solution to prevent pseudomonas. CF Europe has been asked to join this project as work package leader for the dissemination of the EU project, in close contact with the clinical trial participants (in particular patient groups and physicians) as well as national, European and worldwide organizations, groups and institutions who represent the CF patient.

Primary efforts of the dissemination part of the WP includes

1. Improving the quality of life of Cystic Fibrosis (CF) patients and their families by representing and defending the interests both at the start, during, under the project. This, by informing and educating the patient CF families with regard to protocol instructions and clinical trials, but also by disseminating knowledge to the scientific community.
2. Raising  public awareness and understanding in Europe of the therapeutic aspects of the clinical trial anti-Pseudomonas IgY  applications and disseminate project results
3. Promoting appropriate medical care assigned to the Cystic Fibrosis patients during the study by performing site visits

For this a policy of wide dissemination of project findings and results will be pursued; in particular, with focus on potential users. Relevant project information will be disseminated to established and a website will be set up and maintained during the course of the project, including clinical trial relevant information, project findings, links to background information (publications), a discussion forum and a consortium area (the site will be created by another partner, CFE will be the main decision maker on what goes on the website). If the project is accepted, CFE would receive enough funding to engage staff to coordinate this workpackage.

Polka - Patients‘ Consensus on Preferred Policy Scenarii for Rare Diseases
CFE is contributing to Polka, when involved via Eurordis, not as an active partner.
Polka project- Patients‘ Consensus on Preferred Policy Scenarii for Rare Diseases: participation in the selection of topics the European rare disease community will be working on in the next three years. Polka is a project coordinated by Eurordis and partners: the Fundacio Doctor Robert, Rare Disorders Denmark and the National Commissioning Group NHS UK. It is supported by the Public Health Programme of the European Commission DG Sanco. CFE replied and send out a questionnaire to all our member associations.
Polka is launched in September 2008, to ensure patient input in the development of strategies and plans for rare diseases which are currently being developed by the European Union and many of its Member States. The project will facilitate the consultation of the European rare disease community, with the aim of building consensus on preferred public health policy scenarios for rare diseases in key areas such as (pre-implantation) genetic diagnosis, information to the patient and genetic counselling, centres of expertise, cost of treatments, stem cells, neonatal screening and cross-border healthcare.
For more info see Annex EU projects.

Eurordis (European Association for Rare Diseases)
(www.eurordis.org).

Eurordis Survey : Patient organizations & Research

CFE was one of the 309 patient organisations who responded to the EURORDIS Survey on the role and priorities of patient organisations in rare disease research that was conducted by EURORDIS in October and November 2009. As with previous surveys, the high response rate (40%) recorded for this survey made it possible for EURORDIS to fulfil one of its main missions: to carry the voice of rare disease patients in Europe. The results should be useful to highlight the role that patient organisations play in research, advocate for public funding corresponding to the needs of the patients and -optimise collaboration with research.

CFE signed the EURORDIS paper on the proposal to set up a Working Party at the EMEA dedicated to the common assessment of the Clinical Added Value of Orphan Drugs (CAVOD). EURORDIS and other interested parties have identified the delays in national decisions on pricing & reimbursement of Orphan Drugs as a major element reducing and delaying real patient access to orphan drugs.

In this context, the proposed Working Party would facilitate, and therefore accelerate, the decisions to be taken at national level on pricing & reimbursement (as they would be based on reports made by experts gathered at EU level), thereby improving patient access to orphan drugs. In short this working party would have to prepare the assessment of newly acknowledged drugs by national health authorities to accelerate patients’ access, by collecting all data which are available at the time of market authorization (and not start new surveys themselves). Collaboration between this working group and national authorities shall  be included in the national plans for rare diseases (which are to be established according to the Communication from the Commission on “Rare diseases: Europe’s challenges” and  the “Communication on a European Action in the Field of rare Diseases”). Patients representatives are to be members of this working group.
CFE co-signed this paper, together with EURORDIS, other patient associations and academic leaders in the field of rare diseases, to give it stronger political relevance. You can read more background info in the article "Orphan drugs: rising to the challenge to ensure a better future for 30 million patients in Europe" , written by François Faurisso on www.eurordis.org.

Workshop “Bridging Patients &Researchers to Build the Future Agenda for Rare Disease Research in Europe”

As part of this year’s Rare Disease Day 2010 awareness-raising campaign “Researchers & Patients: Partners for Life!” EURORDIS organised a Workshop to bring together the main players to discuss the future of rare disease research in Europe. The meeting, entitled “Bridging Patients and Researchers to Build the Future Agenda for Rare Disease Research in Europe”, was held on March 1st, 2010 at the Residence Palace, Centre de Presse International in Brussels. The European Workshop, was co-organised with E-RARE in partnership with the European Commission, ORPHANET and EUROPLAN. European and national research authorities, researchers, policy-makers and representatives from patients’ organisations and industry met to identify the future priorities in rare disease research and define concrete steps to ensure better collaboration of all interested parties. Karleen represented CFE.
The Workshop offered the opportunity to outline a detailed picture of the rare disease research landscape in Europe and to start shaping the future agenda for rare disease research both at the national and EU level. All attendees had the opportunity to contribute to the ultimate goal of this meeting: to analyse how to increase and secure long term resources for real patient-centred rare disease research for the upcoming years, research that is expected to improve the health of 30 million patients affected by one of the 5 000 (possibly 8 000) different rare diseases in Europe.
This European Workshop confirmed that rare disease research is dynamic, high-quality and highly competitive. Rare disease research contributes to the medical advancement of both rare and frequent diseases. But also that there exists a huge gap between the level of ongoing research and patients needs. The concentration of research activities on few rare diseases (such as Cystic fibrosis!) provides a showcase to demonstrate that research on rare diseases is doable and productive, and that these models can be transposed to other rare diseases or group of rare diseases.
The studies presented at the Workshop confirm that the existence of patients’ organisations sparks off rare disease research. All areas of rare disease research that are successful have a register, a network, a critical mass of publications and an active patient organisation behind them: there is a dynamic between all of these elements and they all need to be in place for research to thrive.
However, these determinants of research are alone not sufficient to face the issue of continuity and sustainability of research projects. Public authorities are necessary to support rare disease research with new, adapted policy and financial instruments at both national and European levels.
In light of the appointment of the European Committee for Rare Diseases (EU CERD) in May 2010, EURORDIS proposed to create a EU CERD Working Group on Research Policy, allowing the integration of the recommendations under development for national strategies with an EU comprehensive research policy agenda taking into account the other aspect of rare disease EU policy, preparing for the 8th EU Research Framework Programme.

On May 15th, the EU Committee of Experts on Rare Diseases (EU CERD) was publicly presented for the first time at the European Conference on Rare Diseases - ECRD 2010 Krakow. This assembly of over 50 members will bring the Commission's DG Health, DG Research, DG Enterprise, the European Medicines Agency, the Committee for Orphan Medicinal Products and the European Centre for Disease Control together with representatives of all 27 EU Member States, as well as 4 representatives of patient organisations, 4 representatives of industry and 15 representatives of academia, selected from a variety of areas of expertise.
For a detailed report and conclusions see the Annex EU health news.

Rare! Together Network Workshop in Barcelona, 19 June 2009

CFE was invited to participate in the Rare! Together Steering Committee. The aim of this project is to help the selected networks build and operate their European Federation, and, ultimately, to develop a “Guide to Establishing and Developing a European Rare Disease Federation.” The advisors share their experience in the creation of their federation, methodology and best practices and the questions and answers will be published on a wiki on the Eurordis website, to share the acquired knowledge and allow other patient representatives to add their comments. End goal is the “Guide to Establishing and Developing a European Rare Disease Network.
Karleen works as an expert is this Eurordis programme to build better and stronger European rare disease federations (as CFE is considered as an exemplary RD disease federation!). 
At the second meeting in Barcelona several topics were discussed with Eurordis and other European federations such as OIFE, Debra, ECHDO and Euro-HSP: the Pros & Cons of a European Federation, the possibilities of Wiki (Create article about topic and everyone can work and comment on the net), EU Funding of Health Research and Public Health, Advocacy  and Empowering the community: How can we have power in the drug development and healthcare decision-making? Main conclusion of the workshop was: Yes we can! What can be done, must be done… We can make a difference! We have the right and duty to act.  We can have more effect then we think... ‘Patients’ are primary stakeholders - We have to do it, together with all stakeholders (researchers, health care professionals, authorities, other associations-Eurordis…). Don’t be afraid you’re too small or weak; turn ‘rare’ into advantage. Plan, be there, do it! 
More info on http://raretogether.eurordis.org and the report in annex.

We will also take part in the newly launched “European Network of Rare Diseases European Federations”.  Both initiatives will certainly help CFE to grow stronger as a European association and better understand the functioning or European (and EU) organizations.

Updating of the entry on Cystic Fibrosis Europe for the latest European Patient Group Directory

The 2009-2010 edition of the European Patient Group Directory is authored by PatientView, and due to be published by Burson-Marsteller Brussels for public release at the European Health Forum Gastein. The Directory will be free in PDF to all members of the public. Print copies of the Directory will be distributed to the European Commission and to other major European policymakers, healthcare stakeholders, and industry.
The 2008 edition of the Directory was warmly welcomed by health professionals, policymakers, and industry across Europe. The 2009-2010 edition will contain about 100 more pan-European groups than the 2008 Directory. We hope that the Directory will provide our group with valuable publicity.
The Directory is available at: http://www.patient-view.com/directories.htm

• Free medical equipment:
• Thanks to the support of David Versteeg (CF Adult group) & Romedic Netherlands 150 PortaNebs (withVentstream en sidestream) were distributed in Russia and Bulgaria.  CF Belgium funded the transport costs.
• Thanks to a grant of the Belgian association free flutters and nasal rinsing cans were distributed during training sessions in eastern European countries.

• Central European contact point: several times a week CFE is contacted to help find answers to questions and problems related to CF (on CF in general, patient data and needs, CF care, services.. in Europe) by patients and families, CF associations, health care professionals, health agencies and organizations, research institutions, pharma companies, authorities and so on. We have regular contacts with all members and individuals from all over Europe and reply to questions via mail and telephone or participate in meetings.

• Website: regular updates by the board, but more input of the national associations would be welcome!

Website

• CFW is very helpful in providing us a place on their website, we have a good visibility and the CFW webmaster puts our materials on the site as soon as we ask for it. To enable easier access CFE acquired the domain www.cfeurope.org  which leads you directly to the CFE page.

Administration & finances

• No paid staff: members of the board work as volunteers for CFE; most of the costs are paid by their national associations. The Belgian and German CF Associations provide regular logistic and administrative help (eg accounting in Germany)
• CFE recognized as charity in Germany: CFE is officially acknowledged as charity and is a registered non-profit organisation with tax reduction status.
• No active fundraising yet: resources depend on payment for participation in EU projects (ECORN-CF) and donations by national associations (Belgium + France and Spain for the European conference and general assembly). Membership is free. Applications were made to Dutch foundations (through OEF) and the German Bosch Foundation.
• Financial Report : see annex

CFE Annual Report 2009-10 Annual meeting June 16 2010
For more info contact karleen@muco.be – www.cfeurope.org