Phaenews spring 2010

MariposaNews 1 Spring 2010n.
SPRING ISSUE OF THE NEWSLETTER OF PHA EUROPE,
EUROPEAN PULMONARY HYPERTENSION ASSOCIATION
Dear friends,first of all I would like to thank you again for electing me President of PHA Europe. I am honoured by the trust you placed in me and look forward to working closely withthe Board and Members of PHA Europe in promoting the cause of Pulmonary Hyper- tension patients in Europe.
I am delighted to introduce the first issue of our newsletter which will be circulated in PDF format to our members and other interested parties.
This issue includes a report on our recent elections and biographical notes of the newBoard, news from PH patient associations in Europe and around the world, updates on PH treatment and research, information about recent conferences and European Unionpolicy and NGO activities in PH and related areas (rare diseases, orphan drugs, organ donation and transplantation). The newsletter also includes a summary of upcoming eventsfor the year and a section on useful links and reading suggestions.
2010 is already proving a very interesting and challenging year for our association. We will take part to both the European Society of Cardiology (ESC) and European Res-piratory Society (ERS) Annual Congresses, respectively in Stockholm at the end of August and in Barcelona in mid September. We are going to hold our Annual General Assembly within the context of the ERS Con- gress, from 18th to 20th September, and are very pleased with the exceptional turnout,40 confirmed delegates from 18 European countries. The General Assembly has a very interesting and wide ranging program and we will be holding our first corporate meeting with representatives of pharmaceutical industries.
There are many positive developments at European Union level, which we will be mon- itoring closely. In particular, we are very pleased that the Council Recommendation onEuropean Action in the field of Rare Dis- eases will provide more focus on thisentire field. We also welcome the new trend towards closer involvement of • PHAE elections and new Board patient associations in the European Committee of Experts on Rare Diseases • News from European PH patient (EUCERD) and in the European Medi- associations p. 03 cines Agency and look forward to new • News from PH patient associations developments with regard to improving access to orphan drugs for patients in • Patient invovlement in ESC/ERS Guidelines for PH I would like to thank our member asso- • Update on PH treatments ciations and their leaders for their pre- and research on PH drugs cious input without which this newsletter • European Workshop on bridging would not have been possible and Pisana patients and researchers Ferrari for putting it all together and • News from European Institutions editing the texts as well as for her contri- butions on EU and NGO activities.
I look forward to your comments and to Upcoming events for 2010 working with you.
• Interesting links and suggested Gerry Fischer Wilhelmstraße 19 • 1120 Vienna • AUSTRIA • +43 1 4023725 • +43 1 4093528 • [email protected] • www.phaeurope.org

PHAE elections and new Board 2010-2012
Elections for the new Board of PHA Europe were held in January 2010. PHA Europe wishes to thank Past President BrunoKopp and the other members of his team for the work carried out in the two years of his Presidency and is delighted tointroduce the new Board of Members, composed of extremely qualifed and dedicated individuals who are very active atnational level in providing invaluable support to Pulmonary Hypertension patients and their families. All have been affected,either directly or indirectly, by Pulmonary Hypertension. PRESIDENT AND
Ebru Arisoy
Economics graduate. Has been working for Undersecre- University studies in world trade. Lives in Vienna teriat of Foreign Trade since 1996. Lives in Ankara. Has where he works in tourism and, since 2001, manages been involved in PH since 2008 when she was working the family liquor and schnaps business. Married and for the Turkish Embassy in Vienna and she met Umit father of a 21 year old boy and a teenage girl with PH.
Atli, an old school friend, recovering from lung tran- Founding Member and President of the Austrian PH splant surgery, and other PH patients. Member of the Board of Turkish PH patient association.
1ST VICE PRESIDENT
Political Science graduate. Has worked for EU insti- Engineering studies and Master in Management. Lives tutions and consultancy firms and then in event mana- in Lyon and works as Managing Director of a company gement. Lives in Milan. Has a 21 year old daughter.
which produces Air Conditioning systems for special Diagnosed with PH in 1988, underwent double lung vehicles. Diagnosed with PH in 2004 he started colla- transplant surgery in 2002. Founding Member and borating with the French PH association in 2006.
President of AIPI, Italian PH patient association.
Current President of HTAP France, the French patientassociation.
2ND VICE PRESIDENT
AUDITOR I
Umit Atli
Chemistry graduate. Has been working for the Turkish PhD in Biology and Homeopathy. Lives in a small Prime Minister's Press and Public Relations Office as town near Haifa. Married and mother of two girls.
a consultant since 2000. Lives in Ankara. Diagnosed Diagnosed with PH in 1999. Member of the Board of with PH in 2001, he underwent double lung tran- the Israeli PH patient association.
splant surgery in 2007. Founding Member and Presidentof the Turkish PH patient association.
AUDITOR II
Luisa Sciacca della Scala
Mélanie Gallant Dewawrin
University studies in Languages and Literature.
Lawyer and Business School Professor. Is married and Married and mother of three grown up children, lives has a young daughter. Lives in Beaune, near Dijon and works in Rome. Has been involved in PH since (Bourgogne/Burgundy). Was diagnosed with PH as a 1998, when her close friend Maria Pia Proia was dia- child. She was on Flolan treatment for 16 years before gnosed with PH and when in 2000 she founded double lung transplant surgery in 2009. Past President AMIP, Italian Patient association. President of AMIP.
of the French PH patient association, she is currentlyManaging Director. During the 2009 General Assembly held in Paris in Sep- Pisana Ferrari, who is also first Vice President, will be tember, there was a discussion about future activities working in close contact with the President and the and strategies for PHA Europe and it was felt that PHA Board, will maintain contacts with the members, coor- Europe would greatly benefit by having a European coor- dinate common activities and projects, organize meetings dinator working for the association. and participation in conferences, take charge of the web The decision was taken to employ someone for the site, newsletter and printed material and general organi- position and the name of Pisana Ferrari was put forward.
zation of the association.

News from European PH patient associations
n. 1 - Spring 2010
Awareness campaign
Vienna's bus stops last January were plastered with bill-
boards from the awareness campaign promoted and
organized by the Austrian PH Association. The cam-
paign deliberately included very powerful and
provocative images of women, men and children
standing with one foot in a coffin whilst carrying our
every day activities (see photos). This campaign aims to
convey the message that there are many people who risk
dying, without even knowing it! Shortness of breath can
be a symptom of a very dangerous disease, PH. By
increasing awareness of the disease, the Austrian Asso-
ciation hopes to encourage more people with breathing
problems to see their doctor and ask if their symptoms
From left: Eva Huetter, Prof Walter Klepetko, Dr. Irene Lang. are consistent with PH. As we know, early diagnosis and Third from right Prof. Martin Langer next to Gerry Fischer treatment can make all the difference. The campaign number of tickets for their concert of the 15th April in provoked a lot of comments and even some criticism, the Wiener Konzerthaus. Proceeds from the ticket sale but it certainly reached its objective, that is to raise were entirely for the benefit of the Initiative. awareness of PH.
Photos from other fundraising events: Circus Roncalli PH Gala in front of the PHA Austria held a PH patient meeting on the 22nd of Vienna City Hall (Gerry and his daughter January in Vienna. Special guests included Prof. Irene Maleen with Bernhard Paul, ringmaster) Lang, cardiologist and PH specialist, and Prof. WalterKlepetko, world renowned lung transplant surgeon,both at the AKH, Vienna General Hospital, and Prof.
Martin Langer, gynaecologist, who spoke about PH andwomen issues.
Maleen becomes Wiener Philarmoniker
a little Fash- ionista and was As part of the events of the able to meet with Vienna Marathon, the Wiener Karl Lagerfeld, have given to the Initiative Philipp Plein & Heilung für Lungenhochdruck a Tang de Hoo Billboards in Vienna with PHA Austria's latest awareness campaign News from European PH patient associations
BELGIUM - HTAP BELGIQUE
HTAP Belgique's President at Eurordis
HTAP Belgique's President, Rosie Matthysen, con- Erasme University regularly organizes "Master Classes" tributed to raising awareness of PH by recently sharing on PAH for medical professionals (cardiologists, pneu- her personal expe- mologists, rheumatologists) from all over the world. The training program includes a two hour PH patient-led workshop organized by HTAP Belgique. Through the workshop the patients association has ation for Rare Dis- contributed to the training of professionals from Venezuela, Greece, Spain, Italy. Its work was greatly features in six lan- appreciated and, in particular, the syllabus written by a PH patient with information about the disease in every day life of patients.
Good news from Hôpital Erasme
Hôpital Erasme-ULB Brussels has created an inde-pendant Clinique d'Hypertension Pulmonaire, headed by Participation at scientific events
Pr. Jean-Luc Vachiéry, fully and only dedicated to PH HTAP Belgique has recently taken part in a workshop organised by the Cellule d'Education Permanente desMédecins (Belgian ongoing education for medical pro- Other Belgian hospitals
fessionals) on the safe use of pharmaceutical products.
A number of Belgian hospitals have a Cellule It has also participated to the Rare Disease Day organized Hôpital-relais HTAP for PH patients. HTAP Bel- by the Belgian Ministry of Health for physicians, nurses gique representatives often visit PH patients in and patient representatives.
these hospitals.
BELGIUM - VZW
Vlaanderen and Limburg) to give the opportunity to The Flemish Belgian patient association has a new Pres- patients to talk to each other in a very informal ident since April 2009, Mr Gust Caelen. We are very context, without doctors or other medical profes- happy that our friend and past President Hendrik sionals present. Ramaker will continue to represent the association inPHA Europe.
New Patient Day
In May all new patients will be invited to a meeting
Patient to patient meetings
where they will be provided with basic information Meetings were held in February in five regions of about the disease and the patient organisation and its Flanders (Antwerp, Brussels, West-Vlaanderen, Oost- Photos from the patient meeting in Limburg n. 1 - Spring 2010
New Member
for PH be paid for by public funding. PHA Europe is very happy to welcome the Bulgarian PHpatient association as a new member. The President is Mr Future plans
Todor Mangarov. The organization was set up at his ini- Mr Mangarov is planning to hold a PH patient meeting tiative at the beginning of 2009 and is a part of National in mid May in Plovdiv, with the support of the National Alliance of People with Rare Diseases in Bulgaria. Alliance for Rare Diseases and the Information Centre for In the last months of 2009 Mr Vladimir Tomov, Rare Diseases and Orphan Drugs. He is also planning to Chairman of the National Alliance for Rare Dis- set up a website in order to increase awareness of the dis- eases, launched a campaign to ensure that treatment eases and provide information to patients. New President and Board
year's event is scheduled is from 29 to 30 May 2010 in The Czech Association has a new Board as of February Seã. There will be lectures on PH diagnosis and 2010. The President is Katerina Novakova, the Vice- treatment, surgery and rehabilitation programs.
President is Veronika Schächterova. Other members Patients will take part in workshops where they can of the Board are Dr. Josef Osˇkera and Jirˇí Bunesˇ. Andrea discuss their problems. The Saturday evening after Hy´blova is the manager. the first part of the conference is always free andpatients may, if they wish, relax and enjoy beauty Annual Congress of the Czech Society
treatments in the wellness area.
of Cardiology
The Czech asso-
Physical rehabilitation program-stay
The third fitness/rehab program for PH patients will be taking part in the organized during the summer of 2010 in Podebrady with the support of the Czech Gov- ernment. Physical rehabili- tation will represent the main part of the program which brings together doctors and Shiri Ben Artzi at the 2009 General Assembly with the association.
gress is scheduled in Brno from 16 to 19 May. The Con-gress is a very important opportunity to interact withdoctors, nurses and PH specialists. The association willbe distributing brochures and other material and thuscontributing to spreading awareness about the organi-zation.
Patient weekend and General Assembly
Each year theCzech associ-ation organizes aweekend for its Photos from 2009 rehab program members. The General A brochure on "PH - diagnosis and treatment" has recently been printed and another is planned on the social and legal aspects of living with PH.
News from European PH patient associations
Annual General Assembly
HTAP France held its Annual
General Assembly on the 13th of
March in Paris. About 110 people
attended the meeting, including
patients, family members and
healthcare professionals. Items on
the agenda included elections of
the new Board
and a break-out
President Sylvain Reydellet medical profes-sionals. The President presented the Annual Second from left Prof. Gerald Simonneau, fourth from left Prof. Marc Humbert, third from right Dr Xavier Jaïs Report with details of 2009 activ-ities and program for 2010. TheGeneral Assembly also voted on the 2009 financial friends can meet and report. The meeting ended with drinks and snacks and share experiences at the time for socializing. Village des Vacances inChédigny, in Touraine.
Study on PH patients and families, March
During the weekend A study on PH on everyday life of patients started in patients and their fam- March. It will cover all aspects of the disease: diagnosis, ilies will be able to meet referral, treatments, consequences of the disease on the Professeur Bonnet of the family, social and professional life including psycho- Necker Hospital.
logical and emotional aspects. All French PH patientsand their families can take part in this study.
PH Awareness day, Lyon, 6th of June
The 5th edition of "Running for PH" (À pleins poumons)
Regional patient meetings, March-April
is being held in Lyon in June. The race is open to Patient meetings were held on the 27th of March in Mar- anyone over 6 years of age. Adults will run 10km. This tillac, in Aquitaine, and on the 24th of April in Mol- event is being organised in a park in the centre of Lyon lkirch, in Alsace.
and will be supported by local press and TV. Fundsraised will be devolved to HTAP France.
PH Awareness day, Sète, 8th of May
This event is being organized by Xavier Gine, father of
PH Marathon,Toulouse, 11-12th of June
a little girl who just passed away last April, in collaboration The 6th edition of the HTAP marathon is being run on with HTAP France, in Sète, a town by the sea in the South 11th and 12th of June. The marathon starts in Toulouse of France, in Languedoc-Roussillon. Alexandra Rosenfeld, and ends in Port La Nouvelle (205 km). Last year's Miss France 2006, is patron of the event, which is sup- marathon was very successful and managed to raise con- ported by the local press, TV and radio, tourist office, siderable attention on PH in TV, radio and press.
hotels and other commercial activities. The funds raisedwill be devolved to HTAP France for research. The First French speaking Patients Conference Con-
program includes a walking/cycling excursion and other gress for PH patients and their families, Lyon, 8-
activities and the highlight of the day is a concert by 10th of October.
Michel Fugain, a very famous French singer and composer.
HTAP France is organizing its first French speaking The first edition, in 2009, was a great success. patient Congress in Lyon 8-10 October. The 2-dayevent is specifically organized for patients and their Family weekend, Chédigny, 8-9th of May
families. PH experts from the PH reference centres will HTAP France is organising a weekend where children make presentations and there will sessions where with PH (up to 20 years of age) and their families and patients can meet doctors, psychologists, nurses etc. n. 1 - Spring 2010
René Baumgart Foundation award
• interaction of drugs The 7th edition of • risks and advantages of pharmaceutical studies the Prize instituted • German health policy issues e.g. advanced health • surgery for chronic thromboembolic pulmonary hyper- • physical exercise in patients with PH without Pulmonary Hypertension" was March in Hannover. • workshops for parents of PH children The two winners with their award • workshops for family members of patients • workshops on quality of life and psychological aspects context of the 51st Congress of the German Society for of the disease.
Respiratory Medicine. For the second time the prize wasawarded to a project in basic research (Dr. Matthias Brock from the University of Zurich) and in clinical In 2011 the PH Association will celebrate its 15th practice (Dr. Nika Skoro Sajer, University Clinic, anniversary and the René Baumgart Foundation its AKH Vienna). Dr. J. Winkler from Leipzig, Chairman 10th anniversary. of the René Baumgart Foundation, and Professor Dr.
Two big events are being scheduled to raise awareness E. Grünig, Member of the Executive Committee of the and funds for research: a football match with charity run Foundation, presented the award.
and autograph session on 19 July 2010 and a concert on7 May 2011. National Patient meeting
In October 2010 the association will hold its 13th
national patient meeting in Frankfurt with lectures President Bruno Kopp was invited to present the PH given by PH experts and discussions on PH related patient point of view in January at a Lilly event in issues, with an expected attendance of 300 to 400 Prague and in March at a Pfizer event in Munich. He will be speaking on the 27 April at a Bayer Vital event inBerlin, on 25/26th of June at an Actelion event in The program is expected to include sessions about: Cologne and on 28th of June at a Bayer Schering event • combination therapy The Hungarian PAH Association is planning to start improve their web site by uploading a that would provide tunity for patients essary contacts in ences and network. order to have their Eszter Csabuda, President and Sara Marton the links section of The association is different Hungarian medical web pages.
News from European PH patient associations
Mr. Joni Berg,Chairman of the IsraeliPH Association Chamber music beingplayed after theAnnual meeting Annual meeting
PHA-Israel held its Annual Meeting last December in
Hertzelia (near Tel-Aviv). About 100 people attended,
including patients, family members and medical stuff. Mr.
Joni Berg (Chairman) and Mr. Aryeh Copperman organizers had to (Director) reported on the year's activities and pre- sented the program for 2010. One of the highlights of the meeting was the presentation by a speaker from the pieces of paper! National Transplant Service who explained the proce- During the dinner dures through which people are listed for transplant, a pianist and flutist played background music. donors found and organs allo-cated. There was great interest from the patients on PHA Israel runs a professionally facilitated emo- this issue and a lot of ques- tional support group for its patients. This support tions were put forward. group was started with the help of generous funding A PH patient who had from PHA USA in the form of a seed grant. The undergone a lung transplant group meets monthly and it provides a unique oppor-tunity for PH patients to share and support eachother and to deal with the emotional issues involvedin living with PH. In January the ladies of the groupwere treated to a morning of relaxation, fun and cre-ativity. They started with relaxation exercises andguided imagery. A professional jewelry designer thenexplained the basic techniques of making a beaded Learning how to make beaded necklace. She had brought along hundreds of assorted beads in all different colours, shapes and sizes and theladies enjoyed making necklaces whilst chatting and then described her experience and how it affected sharing coffee and cakes. her life. The last speaker was a PH specialist whospoke about combination therapy, describing the dif- ferent pathways and mechanisms by which the drugs The Israel PH Association plans to sponsor a series of operate. He also spoke about side effects of combi- consciousness-raising events this spring and summer nation therapy.
as part of the international (Pucker-Up) for Blue After the conference, for the very first time, a raffle Lips campaign. These include a "Blue Lips" wall was held. The first prize was an iPod Touch, donated by pasted with a hopefully astronomical number of paper a pharmaceutical company. The initiative was so suc- slips adorned with supporter kisses during a major cessful that people where queuing up to buy tickets. media event in one of Tel Aviv's most central squares.
n. 1 - Spring 2010
ITALY – AIPI
Annual Patient meeting
AIPI held its Annual As part of its efforts to increase awareness of the disease AIPI patient meeting and has recently offered articles on PH to a number of health General Assembly in and health related magazines and succeeded in having Bologna on 11 April.
them published in a number of magazines, some of which Very qualified speakers are distributed free of charge in Italian pharmacies. The next provided updates on issue of the Italian Organ Donor Association (AIDO) PH treatments and national magazine will carry an interview with Cristina research. Italian jour- Gandola, an ex PH patient who underwent lung transplant nalist and writer Margherita De Bac presented her second surgery 15 years ago. book on rare diseases, recently published, which con-tains a section on PH based on an interview with AIPI Walking for PH
President Pisana Ferrari. As in past Board member Cristina Gandola took years, there was a buffet lunch fol- part in the Milano STRAMILANO lowed by a raffle and time for social- 10 km walk on 21 March to celebrate izing. Over 100 patients and family 15 years since her lung tranplant and members attended.
to contribute to raise awareness of thedisease. She walked 10 km in 2 h and Pisana Ferrari, President of AIPI, and Margherita De Bac, journalist and writer ITALY – AMIP
Annual patient weekend
This equipment is normally supplied by the National AMIP is holding its annual patient day in Pavia from Health Service, but it can take years before it does - this 14th to the 16th of May. The three day program includes is not the first time that patient associations step into a meeting with the physicians at the PH specialist centre the role of the public institutions… of the San Matteo Hospital in Pavia and a social program
with a visit of Pavia and its famous 14th century Certosa. Footbal for PH
AMIP members Vittorio and Laura, parents of two PH on Sky TV
children with PH, are extremely active in fundraising An 8 minute video on PH featuring interviews with for PH. On 21 December 2009 they took part in the Luisa Bonelli and Maria Pia Proia (President and annual Derby del cuore, a match where famous singers member of the board) from AMIP Italy and PH physi- and actors compete against each other to raise money cians from the Umberto I Hospital in Rome was shown on 23 February on SKY TG 24 as part of a program onrare diseases conducted by well-known Italian jour- PH and Facebook
nalist Paola Saluzzi. Check out and join Vittorio and Laura's Bambin Gesù Hospital and Echocardiogram
Facebook page called equipment for Umberto I Hospital in Rome
Ipertensione Polmonare: sai Over the last few years AMIP has organized a number cos'è? (Do you know what of fundraising events. With part of these funds it has Pulmonary Hypertension financed a project at the Bambin Gesù Paediatric Hos- pital in Rome to support medical professionals dedi- It has over 1,200 members and an extremely lively news cated to paediatric PH. AMIP is also donating a VIVID S6 - echocardiogram equipment - to the Umberto IHospital in Rome. This equipment will be invaluable to the hundreds of PH patients being treated in the PH unit.
News from European PH patient associations
Useful and interesting merchandising ideas
PH and the need to raise money for research. Patients Medikeeper is a USB-stick in the shape of can paste a photo of themselves on the postcard under a medical doctor on which one can store the wording "Am I familiar to you?'' This postcard relevant personal information: address, should be sent to ten friends/aquaintances or family contact details, name of persons to be members, with the request to donate and to make ten warned in case of accident, illnesses, copies of the postcard in order to send them on to medicines being used, type of blood, addresses of the other friends. In turn these friends should also send ten treating medical specialists and hospitals, vaccina- cards to their friends. This way PHA NL hopes to tions, allergies and so on. It is available on request from create a "chain of donators". PHA NL web site.
Subsciption fees and fundraising
PHA NL has created a "PH Club of 100" for donors who An interesting initiative is due to be launched shortly.
contribute either individually or as a group 100 euros. PHA NL will be sending to all known patients 10postcards. These postcards contain a brief text about Situation in Norway
opportunity to find each other and to exchange valuable Norway's population is information and encouragement with each other.
less than 5 million. Thismeans that the number PHA Norway
of PH patients is very Due to the low membership number (about 25), PHA small (it is estimated that Norway decided to join forces with the much bigger about 100 people in Heart and Lung Association (LHL) in 2009. Norway are diagnosed PHA Norway is defined as an independent interest group with PH). One patient's search for other patients to within LHL and has its own web page and forum.
share experiences with initiated the start of the Nor-wegian organization about two years ago. A web page and Report from Hall Skara, President of the PH group. a forum were set up, and patients had, in this way, an New Board
It includes a patient The Polish patient association held elections in March forum and a calender of 2010 and has a new Board of Members. The President future events. The associ- is Wlodzimierz Galuszkiewicz and the Vice-President is ation plans to upload a Marek Babecki. Other members of the Board are Piotr video interview with a Manikowski, Dariusz Sakowski, Roman Cichon´, PH expert in the near Agnieszka Bartosiewicz and Mal´gorzata Serwicka- future. Walczak. The annual meeting of PHA Poland is planned for The PHA Poland web site was launched in the second May 2010. This is intended to be a social occasion and half of 2009. The address is www.tetniczenadcisnie-
will include family members and friends, with time for relaxation and socializing n. 1 - Spring 2010
Respiratory Medicine Congress
sionals, hospitals, etc. To apply click on www.fotovi-
The 8th Winter Congress of the Spanish Society of Pul- monology and Thoracic Surgery (SEPAR) was held in The photos submitted will be posted on this web site.
Madrid in January. Irene Delgado, President of theSpanish PH association, was invited to present the PH booklet for parents of children with PH
patient perspective during the session on Pulmonary The Spanish PH Association has recently published a This was the first booklet for parents of children with PH Guia para padres: Tengo un hijo con Hipertension Pulmonar (see photo). The publication contains not only scientific information about the disease, but also practical tips for day to day everyday life of a matters and advice on how to Irene Delgado speakiing at the Congress deal with the emotional aspects of the condition. Ithas a very attractive graphic layout, beautiful and evocative photos and it includes touching quotations The Spanish PH Association's Annual General from famous authors, poems and lyrics. Assembly will take place from 11-13 June in Marina The booklet was distributed in a number of Spanish D'Or (Castellón). The meeting will bring together hospitals. Both PHA USA and Latina American PH patients from all over Spain to talk about PH and PH Associations have requested it and put it on their web- related issues in an informal atmosphere and pleasant sites. At the moment the booklet is available only in location (by the sea) which will also allow for social- izing and relaxing.
Last year's Spanish Association on Youtube
The Spanish Association has recently made a brief video about its activities which can be viewed on area and was such a success that itwas decided to Spanish Association on Facebook
The Spanish Associ- Yoga session at 2009 Assembly ation's Facebook pagehas over 1.000 fans. Photo competition
The Spanish Association has launched a photo contest
open to patients and families on the subject of "Living with PH". The winners will be announced during the General Assembly (see above). The 12 best photos willbe used in the Spanish Association's 2011 calenderwhich will be distributed to members, medical profes- A PH patient association has recently been set up in President, Annika Nordberg, as well as her colleagues Sweden. PHA Europe wishes the best of luck to its Britt-Marie Forsstrum and Malin Ekstromm.
News from European PH patient associations
SWITZERLAND - Schweitzer PH-Verein
PH Association set up in January
On January 15 the Swiss PH self help group (PPH Selb-
sthilfegruppe Schweiz) which Bruno Bosshard has run
successfully for the last ten years, has become the
Swiss PH Association (Schweizer PH-Verein). Mrs
Therese Oesch is the new President. For further infor-
mation please see www.lungenhochdruck.ch (PH)
and www.novaria.ch (transplant). Warmest congrat-
ulations to Bruno and his colleaugues from all at PHA
Europe!
Office moved and patient meeting
have concentrated their efforts on raising awareness PHA Turkey recently moved its central office from of the disease and of the difficulties PH patients face Istanbul to Ankara. in day to day life by contacting national and local On January 10 it held a "Patient-info Meeting" during which future plans for 2010 were discussed. Umit Atli, PHA Turkey President and other members One of the aims for the coming year is to open offices of the Turkish association recently appeared on TV in Istanbul, Izmir and other regions of Turkey. programs in Istanbul and Ankara (see photo).
Participation at scientific events
PH Turkey took part in the "Patient Partner Central-
Eastern European Regional Workshop on Defining the
needs and means for more partnership between Patients,
Patient Organisations and Other Stakeholders in Clinical
Trials" on 24th and 25th March in Athens.
Web site
PHA Turkey has two new web-sites. The official site is
www.phdernegi.org.tr and the web site for the support
group is www.pha-turkey.com
Awareness campaign
Over the last few months PHA Turkey members
On left: PHA Turkey members on national TV Above: new web site n. 1 - Spring 2010
Breathe freely campaign
PHA-UK's "Breathe Freely" campaign is in its final stage and has achieved considerable success in helping recently updated its persuade British Airways, Cathay Pacific, Emirates and leading charter holiday provider Thomson Airways to Guide and has pub- drop charges for the provision of supplemental oxygen.
It has also succeeded in persuading many others carriers to change their policies and allow people to bring their own oxygen equipment on board.
patient partici-pation in clinical PHA UK is now focusing on the global Pulmonary Hyper- tension awareness campaign, PuckerUp4PH 2010. The important issue for original 2008 initiative to PH patients in their collect the largest number of day to day life with the disease, but is rarely addressed.
"Blue Kisses" with the aim of This booklet provides very useful information and entering the Guinness Book advice. The publications are available on request by of records was thwarted by the contacting the association. H1N1 virus pandemic. The alternate online Pucker For further details see: www.phassociation.uk.com
Up4PH search for "The Lipsof 2010", however, proved a Spring issue of newsletter
great success and generated wideranging media interest.
The Spring issue of the PHA 24 PH patient support groups from around the world par- UK newsletter is now out with ticipated and almost a thousand photo were uploaded to many interesting articles.
the www. puckerup4ph.com /gallery.php photo gallery
Topics covered include: Most significantly, there were over 30,000 hits on the • news of the Puckerup4 www.puckerup4ph.com website.
On the 14th of May 2010 PHA UK is launching this • results of the Breathe freely year's PuckerUp4PH Campaign, which will run through till the start of International PH Awareness Month in • disability living and November 2010, with the participation of a number of attendace allowances; PHA Europe member associations. • a Guide to accessible Britain; Blue Lips Kits will be sent to each participating PH • details of meetings planned; patient group in order to help their members collect • fundraising events for 2010.
kisses from friends and loved ones, but also to encouragePH awareness raising and "kiss collecting" events.
The newsletter also features a number of patient stories. Each Blue Lips Kit will contain enough forms to collect100 kisses.
For further details see: www.phassociation.uk.com
Next PHAE newsletter
The Summer issue of the PHA Europe newsletter is due out around the end of July/beginning of August. It will cover May-June-July. We would be very grateful to you if you could send a brief report and possibly some photos of any activities of yourpatient association that you wish to share with us. You may send your contributions in your own language and we can providefor translation. Many thanks in advance! News from PH patient associations around the world
Sociedad Latina de Hipertensión Pulmonar
e-newsletter containing infor- The Latin America PH Society was founded by Migdalia mation on its activities and Denis, a Venezuelian PH patient living and working out interesting reports from of Miami. She is the current President. PHA Europe its member assocations members had the opportunity of meeting her at the of Argentina, Colombia, International PH association meeting held in Barcelona Brazil, Mexico, Puertorico, Venezuela as well as from theSpanish association.
Quarterly newsletter
Migdalia Denis has recently sent us its second quarterly
New website
aspects of PH and PH in every day life. PHA USA's web site has recently undergone major The three day Conference is packed with interesting restyling and has many new and interesting features. The events such as patient and caregiver meet-ups, support international section is particularly interesting: the page group meetings, helpline volunteers training sessions and on international commitment contains a time line mapping the traditional PH Fashion Show. Two sessions on PH the development of the PH associations in different coun- and PH treatments will be held in Spanish. tries and their main activities over the last 30 years. PHA Europe President Gerry Fischer has been invited tospeak about "Fundraising for PH" at the Leaders Meeting. 9th International Conference on PH
PHA's 9th International Pulmonary Hypertension
Advances in Pulmonary Hypertension
Conference is taking place in The title of the new and Garden Grove, California, extremely interesting issue of the from the 25th to the 27th Official Journal of PHA USA is June. This year's scientific ses- "Living with PH: multidisci- sions for medical professionals plinary collaboration for patient will be on "Inflammation and success". Items covered include: Growth factors in PAH".
• health-related quality of life There will be numerous as perceived by patients; patient/ family led sessions as • depression and PH.
well as medically led sessionsfor patients covering different Jornada Annual de Hipertension Pulmonar
held in Barcelona in 2006. She sent us In February the Venezuelian Society for Cardiology, in a report and photos of the meeting.
collaboration with the Society for Respiratory Med-icine, organized a conference on PH. One of the speakers Legislation on the disabled
was Prof. Nazzareno Galiè, from the University of The PH patient association is lob- Bologna, Italy (see photo with Annabel Sivira). bying for PH to be included in the Annabel Sivira is the President of the Venezuelian PH new law on the disabled due to be approved in April 2010.
Association. PHA Europe members had the opportunity ofmeeting her at the International PH Association meeting n. 1 - Spring 2010
Patient involvement in ESC/ERS Guidelines for PH
• Updated clinical classification.
Guidelines on the diag- • Revised diagnostic algorithm. nosis and treatment of • Revised therapeutic algorithm (with indication for Pulmonary Hypertension early treatment, functional class II).
were the object of a • Detailed analysis of all drugs and possible drug interac- course organized by the European Society of Car- • Specific recommendations for treatment for different diology (ESC) in Sophia types of pulmonary hypertension.
Antipolis, France, in • Definition of the characteristics of centers of excel- October 2009, attended by over 300 medical pro-fessionals. These courses Also, compared to prior versions, the new guidelines take place annually but contain more details on associated forms, risk and prog- the 2009 edition repre- nosis factors, diagnosis and treatment in children, etc. sents a landmark event as it included, for the first time Obviously, the limits of the Guidelines were also brought ever, presentations from all the relevant stakeholders: to light, with indications for possible improvements. One medical professionals, patient representatives, pharma- limit in particular is the fact that the Guidelines, as ceutical industry and regulatory authorities. detailed as they may be, cannot cover all the possible Over the course of the one day event, qualified speakers from forms of the disease, especially in cases of various eti- all over the world discussed possible gaps in evidence of the ologies or co-morbidity. current guidelines and future challenges in treatment andresearch for PH. The course faculty included medical pro- Need for further evidence
fessionals and researchers Marion Delcroix and Jean Luc Another issue raised during the discussions is the need for Vachièry (Belgium), Marius Hoeper (Germany), Adam further evidence and problems related to recruiting vol- Torbicki (Poland), Gerald Simmoneau and Oliver Sitbon unteers for clinical studies. The reasons for these difficulties (France), Simon Gibbs (U.K.), Nazzareno Galiè (Italy).
include the following: Frédéric Bodin (Actelion France) represented the industry, • Three classes of effective drugs already exist and patients Amani El-Gazayerly (Netherlands) the European Medicines are reluctant to enter new studies. Agency (EMA) and Pisana Ferrari (Italy) PH patients and • Many patients still arrive at medical centers in an advanced stage of the disease and cannot be enrolled; • The presence of a control group with placebo replacing Role of patient associations
the active principle is considered by some to be ethically Very interestingly, one of the gaps identified in the course of the discussion was involvement and input from patients • Clinical trials on naïve patients (not treated with other in the drawing up of the guidelines. This represents an drugs) are almost impossible to conduct: the drugs being important step towards increased patient involvement and tested are usually added to the treatment already we look forward to further developments in this respect. The role of patient associations in providing invaluable • The proliferation of small centers makes the organization support to patients is explicitly recognized by the new of clinical studies more difficult. ESC/ERS Guidelines in several instances:• Paragrah 7.3.1 General measures: "Encouraging patients Improving future clinical trials
and their families to join patient support groups can have For all of these reasons and despite the fact that there are positive effects on coping, confidence and outlook" already several substances ready for experimentation, it was and "Patient support groups may also play an important felt that it will be difficult to complete clinical studies to role in this area (psycho-social support) and patients verify their efficacy in the short-medium term. should be advised to join such groups" (page 3).
Suggestions were put forward on ways to improve clinical • Paragraph 12.7 "(Medical) Referral centers will have a link to their national and/or European pulmonary • Redefine the endpoints of clinical studies (replacing or hypertension patients association" (page 38). adding to the 6 minute test); for example, evaluating thenumber of patients who improve significantly or intro- Main changes in the Guidelines
ducing an absolute value.
Without going into details, we would briefly like to • Diversify the population of clinical study participants review the main changes/improvements introduced by (currently, most are patients with the idiopathic form the new Guidelines: of the disease).
Patient involvement in ESC/ERS Guidelines for PH
• Differentiate the clinical studies by their functional therapeutic strategies. class: perhaps specific tests for class II (mild symptoms) PHA Europe will be monitoring future developments.
• Carefully evaluate the effects of first line combination Next ESC course on PH, Sophia Antipolis
therapy compared to the system currently in use in PHA Europe President Gerry Fischer has been invited to which the second or third drug is subsequently added. attend the next ECSC course on PH being held in SophiaAntipolis in the Fall of 2010.
ESC/ERS Guidelines and PH patient perspective
From the patients' perspective, the existence and the use
* A joint task force of the ESC, European Society of Cardi- of Guidelines by physicians who are involved with pul- ology, and of the ERS, European Society of Respiratory Med- monary hypertension are, without a doubt, to be received icine have developed these guidelines with the participation of the International Society of Transplantation and the Association Guidelines summarize and evaluate all of the latest and for European Paediatric Cardiology who have subsequently most up to date evidence on a given disease. If they are cor- endorsed them. Full text at www.escardio.org/guidelines-
rectly implemented they guarantee the application of sys- tematically and widely tested diagnostic techniques and Update on PH treatments and research on PH drugs
In this section of the newsletter we will be reporting on New substances being tested
ongoing research on PH treatments, both on approved • Riociguat, NO-independent stimulators and activators drugs and new substances being tested. We will also advise of cGMP in PAH (PATENT study) or CTEPH (CHEST on any new indications or new ways of administering study) (Bayer).
approved drugs. Clinical trials involve long procedures for • Imatinib - tyrosine kinase inhibitors - platelet-derived design, recruitment of volunteers, testing period and pub- growth factor inhibitors - IMPRES study (Novartis).
lication of results so we do not expect to have significant • PF-00489791 - novel long-acting phosphodiesterase 5 developments to report in each issue. inhibitor (Pfizer).
• Macitentan - a new, potent tissue-targeting endothelin At the present moment new developments include: receptor antagonist (Actelion) - SERAPHIN study.
• Selixipag (previously ACT-293987), a potent non- prostanoid prostacyclin receptor agonist which exerts • In January 2010 ambrisentan/Volibris' (GSK) use was vasodilating effects (Actelion) - GRIPHON study.
extended to functional class II. New ways of administering approved drugs
• Sildenafil/Revatio has been recently approved for intra-
venous use (Pfizer).
• Clinical trials under way to assess efficacy and safety of oral treprostinil/Remodulin (United Therapeutics) -FREEDOM-C2 and FREEDOM M-M studies.
Combination therapy clinical trials
• Effects of combination of bosentan/Tracleer (Actelion)
and sildenafil/Revatio (Pfizer) Versus sildenafil/Revatiomonotherapy - COMPASS-2 study. For further information and details: • Upfront combination of tadalafil/Adcirca (Eli Lilly) and ambrisentan/Volibris (GSK) versus monotherapy with either tadalafil or ambrisentan - AMBITION • www.emea.eu
• web sites of pharmaceutical companies European Workshop on bridging patients and researchers
n. 1 - Spring 2010
The European Organization for Rare Diseases (Eurordis) of orphan drugs as a proxy. Five hundred and eighty one organized a workshop on 1st of March in Brussels on designations have been made for potentially treating 343 Research in Rare Diseases (RD) in Europe. PHA Europe diseases affecting 8.2 million people. There are 666 ongoing delegates Rosie and Luc Matthysen (Belgium) and Pisana national or international unique clinical trials relating to Ferrari (Italy) attended this meeting which provided an 312 diseases potentially affecting 6.8 million people.
excellent opportunity to listen to some of the key players Looking at products on the market, there are 99 mar- on the Brussels scene in the area of rare diseases: EU keted drugs for 141 diseases. Interestingly, pulmonary Commission DG SanCo (Health and Consumer Pro- arterial hypertension is second on the list of (non tumor) tection) and DG Research officials, representatives of RD list (after cystic fibrosis) with 13 orphan drug desig- European Medicines Agency (EMA), Eurordis, Orphanet, nations (see slide below). Ségolène Aymé stressed the E-rare network, Europlan, and national research Insti- importance of Registries, European networks and patient tutes. About 100 persons attended the workshop, including organisations and disease-specific scientific output as many patient organisations, both national and European, determinants of R&D. Research on RD requires a com- national health authorities and research institutes and rep- munity of interested-parties to reach the critical mass. resentatives of the pharmaceutical industry. What followsis a brief summary of three of the presentations, felt to be For further details see: the most relevant as they refer to research on PH or to the role of patient associations in Europe. There is only a brief mention of the other topics covered but furtherdetails can be found at: Eurordis survey on patient role in research
Fabrizia Bignami This survey was carried out in 2009 by Eurordis in collab-oration with the Centre de Sociologie de l'Innovation (Ecole des Mines, Paris, France) and the aim was to: Ségolène Aymé • measure patient organisations' (POs) interest for RareDiseasePlatform (RDPlatform) is 3-year support (2008-2012) action project of the EU involving 13 coun- • evaluate POs support to research: in what ways and to tries. RD Platform provides information to the RD research community on: databases, bio-banks, patient registries, • learn about POs experience of collaboration with technology platforms and academic and industrial know- how. The aim is to provide decision makers and the com- • collect POs opinion on priorities and difficulties for RD munity with a clear view of what is currently funded at the national and international levels in the field of RD. Theproject leader is Ségolène Aymé, and the coordinating team Seven hundred and nine patient associations were involved is composed of members of Orphanet Paris. Ségolène in this survey. The response rate was 40%, with 309 valid Aymé in her presentation reviewed the current situation responses from 29 European countries representing 110 dis- with regard to RD research in the EU. eases affecting 1.3 million persons. As far as basic research is concerned at the present moment There were considerable differences between the POs, in there are 4.770 ongoing projects covering 2121 diseases.
particular in terms of annual budget, number of members, The situation varies greatly according to the disease. Very time since setting up.
few diseases have more than 10 research projects, a few dis- As far as budget is concerned, about 6% of POs have no eases have over 5 projects going on, the vast majority of budget, 10% have less than 300 euros, 25% have less diseases have only one or two teams than 3.000 euros. 25% of POs have working. Cystic fibrosis is the disease over 100.000 euros, and 5% has with the highest number of research more than 1.200.000 euros. There projects going on, followed by neuro- are big differences also in number of muscular diseases, scleroderma, some members, with 6% having less than rare forms of non rare diseases, rare 50 members, 21% between 50 and cancers, etc. In this area the main a 99 and 33% between 100 and 499.
progresses have been made in gene In other words, 60% of POs have identification, which translates into less than 500 members. Another diagnostic tests. 26% have between 500 and 3000 With regard to products in devel- members and only 6% over 3.000.
opment Mrs Aymé takes the example The number of years of activity European Workshop on bridging patients and researchers
varies between 1 and 30 (only very because of the numbers (1 in 10.000), few POs are older than this) with even if there are designations of drugs.
the median being 11 years.
European networking, the existence of The main conclusions of the survey registries and role of patient associa- are that POs have a very high com- tions are three major determinants mitment to research are very con- for research on RD and should be put scious of obstacles and priorities.
at the centre of any policy on RD.
56% of POs have a scientific board Research on RD being carried out is and thus integrate external scientific of high quality, there is excellence competences within their structures.
and this is a very positive message Overall, 37% of POs finance for all involved. Despite this, there is research. Of these a total contri- a big issue of discontinuity of funding bution of 13 M euros was provided over the last year. The and insufficient funding and this has emerged as a regular majority of POs financially support research using their own message throughout the workshop. This is particularly budget, others organise specific fundraisers for this purpose.
evident for infrastructure and tools (eg registries). More The above slide shows some of the types of research which awareness needs to be raised about funding on RD. Funding is funded by POs. should follow two parallel paths: One very interesting point made by Fabrizia Bignami is that • openness of all research on the basis of excellence POs contribute to research in many other ways and not just financially. They also contribute by: • specific lines of funding for RD research for networking, • creating links between patients, researchers and physi- tools, bio-banks, registries, clinical trials.
• helping to identify patients to participate in clinical trials It is also important to ensure long term sustainibilty of and providing information for them; research through continuity in funding.
• participating in scientific committees within institutions;• collaborating in clinical trials design.
Finally, POs can contribute to defining research projects The European Project for Rare Diseases National Plans by highlighting patient's needs and expectations.
Development (EUROPLAN) is a 3-year project (2008- PO concern about funding clearly emerged in the survey 2011). The main goal is to provide National Health as a key issue. POs do not expect to (and cannot) replace Authorities with supporting tools for the implementation public or private research institutions, but rather aim to of the Council Recommendation on an action in the collaborate with them on an equal footing as fully recog- nised partners bringing important contributions.
For further details: For further details: E-Rare (ERA-Net for research programs on rare diseases) Yann Le Cam is a network of 10 partners - public bodies, ministries Mr Le Cam concluded the day's debate by pointing out that and research management organizations - from 8 coun- nothwithstanding the significant amount of research on tries, responsible for the development and management RD going on there are 500 RD for which there are only of national/regional research programs on rare diseases.
five projects and 3.000 RD with no research at all being E-Rare is a 4-year carried out! Most research is focussed on few diseases and more must be done to progressively address other RDs.
There have been two major achievements: • gene and mutation identification has led to tests foster research on • almost 100 products for RD are on the market in Europe.
Basic research is not linked to prevalence of the disease. As For further details: far as clinical trials are concerned this is more difficult, n. 1 - Spring 2010
News from European Institutions and NGO's
In this section of the newsletter we would like to highlight COLLABORATION TOWARD COMMON SCIENTIFIC ASSESSMENT some of the items currently on the EU agenda which can OF THE CLINICAL ADDED VALUE OF ORPHAN DRUGS (Eurordis be of interest, either directy or indirectly, to PHA Europe call it CAVOD).
members and PH patients in Europe. These include: The proposed Working Party would facilitate collabo- • an increased focus o rare diseases through the implemen- ration amongst EU authorities and Member States in tation of the Council Recommendation to the Members order to make the most of already existing scientific eval- States (2009-2013); uations and information at the EU level and to help • new developments with respect to access to orphan national health authorities make their pricing and reim- drugs throughout Europe; bursement decisions, thus improving effective access to • closer involvement of patient assocations in the orphan drugs by rare disease patients. Working Party in the EU Committee of Experts on The Commission responded to Eurordis recognizing that Rare diseaases and in the European Medicines Agency; access to authorized medicinal products in Europe is a • expected approval of the proposed Council and EP horizontal issue concerning all healthcare interventions directive on Organ Donation and Transplantation.
but one that is particularly acute for orphan drugs. It alsoannounced its decision to address the issue by launching Greater focus on rare diseases in Europe over the
a Call for Tenders to help identify and assess possible next few years
ways to implement a mechanism of coordination between The Council of EU Health Ministers adopted in June 2009 a European strategy* that calls upon Member States The Executive Agency for Health and Consumers has to implement national plans for rare diseases before the end now published eight calls for tender in the area of public of 2013. The EU recognizes the specificities of rare diseases health, one of which relates to the creation of such a - limited number of patients and dirth of relevant mechanism. The aim is to: knowledge and expertise - and singles them out as a field • describe the regulatory process followed by an orphan with very high European added value. The objective is to medicine, from orphan designation at the European support Member States by ensuring effective and efficient level to reimbursement in the Member State and identification, recognition, prevention, diagnosis, examine to what extent the information produced by treatment, care, and research for rare diseases in Europe. the authorities responsible for orphan designation and The Council Recommendation also addressses the issue MA is directly useful for the medicine reimbursement of difficulties in access to orphan drugs (paragraph 17(e)) decision process; and suppports the sharing of national assessment reports • describe the Health Technology Assessment (HTA) on the therapeutic or clinical added value of these drugs expertise (focusing on relative efficacy and relative in order to minimize delays for rare disease patients (see effectiveness) used at national level for this purpose and also below). We will be monitoring developments in this level of involvement of existing international net- *COUNCIL RECOMMENDATION ON EUROPEAN ACTION IN • describe what expertise is used when the medicine is pre- THE FIELD OF RARE DISEASES (2009/C 151/02) scribed to all the targeted population of patients affectedby a certain rare disease.
To access information about the calls for tenders see: http://ec.europa.eu/eahc/health/tenders.html
For more information on Eurordis position see:
Equal access to orphan drugs throughout Europe
Eurordis has frequently addressed the issue of rare disease patients in Europe not accessing life-saving EU-approvedorphan drugs. This happens mainly because national Patient associations in the Eu Committee of
authorities take too long to decide on the pricing/reim- Experts on Rare Diseases
bursement of the drugs, thus delaying their being put on Four representatives of patient associations will be sitting the market, and also because companies are faced with dif- on the recently created European Committee of Experts ferent procedures in the different countries. on Rare Diseases (EUCERD). The aim of this Committee In order to overcome this problem, Eurordis, representa- is to assist the Commission in formulating and imple- tives of industry and academic leaders in the field of menting the Community's activities in the field of rare dis- orphan drugs have put forward a proposal to the European eases. It should help all EU countries to tackle rare diseases Commission and the European Medicines Agency (EMA) more effectively and maximise the limited resources for the establishment of a WORKING PARTY FOR EUROPEAN available by avoiding fragmented efforts and duplication. News from European Institutions and NGO's
The Committee will be composed of 51 members including • contribution to the Agency's safety communication.
representatives from departments or government agenciesresponsible for rare diseases (one for each Member State), The strategy also foresees financial support to facilitate the from the pharmaceutical industry (4); from ongoing and/or participation of patients' and consumers' representatives.
past Community projects in the field of rare diseases (9); The Reflection Paper specifically recognizes that "patients representatives of ongoing and/or past rare diseases projects and consumers bring a unique input which contributes to financed by the Community Framework Programmes for achieve the best possible results within the regulatory Research and Technological Development (6); and one rep- process. Their contribution not only builds trust in the men- resentative of the European Centre for Disease Prevention tioned process but also incurs higher levels of transparency". and Control. According to a recent issue of Orphanews Europe (19 For further information see: March), because of the delay in establishing the new European Commission (Barroso II), the EUCERD appoint-ments have not yet been finalised and the first meeting has Organ Donation and Transplantation
been postponed until May 2010. Organ donation and transplantation (ODT) is a major issuein the EU as the demand for organs currently exceeds the For more information see: number of available organs in all Member States. The EU organized an Open Consultation on ODT that took place in June 2006. Patient representatives through Eurordis and the RAPID database of press releases at : responded to this consultation. Eurordis underlined the importance of ODT for rare disease patients and explained how these were worse-off than the average organ recipient.
In that same contribution Eurordis suggested that the Closer patient involvement in the European Medi-
Commission fund a pan-European awareness-raising and cinal Agency
information campaign involving patient representatives, The European Medicines Agency (EMA) has recently so as to educate the public and enlarge the pool of potential taken an important step forward in the direction of a closer involvement of patients and consumers. In December 2009 the Management Board of EMA ODT is currently on the EU agenda with two specifc pro- endorsed a a REFLECTION PAPER (EMA/10723/2009) with proposed actions that will considerably widen the • an Action Plan (2009-2015) on ODT which aims to scope of patient and consumer contribution to to the reinforce coordination between Member States in this Community legislation already allows for participation • a proposed Directive of the European Parliament and of patients as members of three scientific committees: of the Council on standards of quality and safety of • COMP (COMMITTEE ON ORPHAN MEDICINAL human organs intended for transplantation.
• PDCO (PAEDIATRIC COMMITTEE); The ACTION PLAN identifies ten priority areas in order to • CAT (COMMITTEE FOR ADVANCED THERAPIES). reinforce the cooperation between Member States, byidentifying and developing common objectives and guide- The legislation relating to patient and consumer partici- lines, jointly-agreed benchmarks, regular reporting, and pation in these Committees has been implemented, and defining and sharing of best practices. Actions at Com- all related activities are well established. However, in munity level will complement Member States' efforts to other areas where there is no specific legal basis patient improve the quality and safety of organ donation and involvement occurs but is not well structured (eg. at the transplantation, combat the organ shortage and make level of the CHMP, COMMITTEE FOR MEDICINAL PRODUCTS transplantation systems more efficient. FOR HUMAN USE).
The PROPOSAL FOR A DIRECTIVE covers human organs The measures proposed by the Reflection Paper will used for transplantation, during all the phases of the provide a more structured framework for patient and con- process - donation, procurement, testing, preservation, sumer involvement. The proposed measures include: transport and use - and aims to ensure their quality and • consultation during benefit-risk evaluation of medi- safety, and hence a high level of health protection.
The European Parliament Committee on the Envi- • participation as observers in meetings of the PHARMA- ronment, Public Health and Food Safety was asked for its COVIGILANCE WORKING PARTY; opinion (within the context of the co-decision procedure n. 1 - Spring 2010
- see below). At its meeting of 16th of March it agreed on the task of further training more transplant donor coordi- a number of comments and suggestions on the Action Plan nators in specific regions of each Member State.
and has proposed amendments to the proposed Directive.
These reflect concerns with regard in particular to the def- For further information see: inition of donor, responsible authorities, post transplan- tation period, living living donors.
Rare Disease Blogs: a joint project of EURORDIS
The Proposal for a Directive Directive is due to be approved by the European Parliament in plenary session 17-20 May. Rare Disease Blogs is a recent joint project of Eurordis(European Organization for Rare Diseases) and NORD For further information see: (National Organization for rare diseases, USA). It aims to bring together key international opinion leaders in the fields of rare diseases and orphan drugs in order to promote debate and foster interaction with members of the public. Invited bloggers include patient representatives, rare disease national and international patient federations and national health andresearch authorities. Comments or questions Training transplant donor coordinators trainers
The Executive Agency for Health and Consumers has recently published eight calls for tender in the area of public health (see also page 19). One of these is related to the wishes to join.
training of transplant coordinators.
The purpose of this contract is to train from one to three For further infor- persons from each Member State, who have been desig- nated as organ transplant coordinator by their hospital/local/regional/national government. These persons will become "national trainers" and will be charged with HOW THE EUROPEAN PARLIAMENT LEGISLATIVE PROCESS WORKS IN PRACTICAL TERMS
From the European Parliament web site: provides that consultation is obligatory, being required by the • A Member of the European Parliament, working in one of the legal base, and the proposal cannot acquire the force of law parliamentary committees, draws up a report on a proposal for unless Parliament has delivered an opinion. In this case the a ‘legislative text' presented by the European Commission, the Council is not empowered to take a decision alone.
only institution empowered to initiate legislation. The parlia-mentary committee votes on this report and, possibly, amends Parliament has a power of political initiative: it can ask the Com- it.When the text has been revised and adopted in plenary, Par- mission to present legislative proposals for laws to the Council.
liament has adopted its position.This process is repeated one It plays a genuine role in creating new laws, since it examines the or more times, depending on the type of procedure and Commission's annual programme of work and says which laws it whether or not agreement is reached with the Council.
would like to see introduced.
• In the adoption of legislative acts, a distinction is made between the ordinary legislative procedure (codecision), which puts Par- For further details
liament on an equal footing with the Council, and the special leg- islative procedures, which apply only in specific cases where Par- liament has only a consultative role.The codecision procedurewas introduced by the Maastricht Treaty on European Union Find your country's MEP sitting on the EP Committee
(1992), and extended and made more effective by the Ams- on the Environment, Public Health and Food Safety at:
terdam Treaty (1999).Two thirds of European laws are adopted jointly by the European Parliament and the Council, including the fields of transport, the environment and consumer protection.
• On ‘sensitive' questions (e.g. taxation, industrial policy, agricul- Find your contry's MEP's at:
tural policy) the European Parliament gives only an advisory opinion (the ‘consultation procedure'). In some cases the Treaty Upcoming events for 2010
13-15 May, Cracow, Poland
■ INTERNATIONAL CONFERENCE ON RARE DISEASES
The biennial European Conference on Rare Diseases is organised by Eurordis and its partners, and is supported by the
European Commission's Health Programme. It is the most important conference in Europe on rare disease policy and has
attracted over 400 participants to each of the three last conferences. On May 13th, the day before the Conference offi-
cially starts, Eurordis will hold its General Assembly. Several satellite workshops will also take place on May 13th at the
same venue, such as the 10th Council of National Alliances, the annual Orphanet Workshop, and the EuroPlan
Workshop, which will gather health authorities from all 27 Member States. PHA Europe (Eurorids Member) will be rep-
resented by Mrs Marzia Predieri, Member of the Board of AIPI, Italian PH Patient Association.
24-27 June, Garden Grove, USA
■ PHA'S 9th INTERNATIONAL CONFERENCE ON PULMONARY HYPERTENSION
PHA's 9th International Pulmonary Hypertension Conference is taking place in Garden Grove, California, from the 25th
to the 27th June. This year's scientific sessions for medical professionals will be on Inflammation and Growth factors in
PAH. There will be numerous patient/family led sessions as well as medically led sessions for patients covering different
aspects of PH and PH in every day life. The three day Conference is packed with interesting events such as patient and
caregiver meet-ups, support group meetings, helpline volunteers training sessions and the traditional PH Fashion Show.
Two sessions on PH and PH treatments will be held in Spanish. PHA Europe President Gerry Fischer has been invited
to speak at the Leaders Meeting on Fundraising for PH. A number of other PHA Europe Member Associations will also
be attending.
28 August - 1 September, Stockolm, Sweden
■ ESC ANNUAL CONGRESS
The European Society of Cardiology Annual Congress is currently the largest cardiovascular
medicine meeting in the world. It covers all disciplines, from basic research to clinical practice. Over
30.000 persons attended the 2009 ESC meeting in Barcelona. As in past years it is expected that
there will be a number of sessions dedicated to Pulmonary Hypertension. PHA Europe will be present
as an exibitor with a booth and will be represented by Mrs Pisana Ferrari, Vice President of PHA
Europe, and Mrs Luisa Bonelli, Secretary of PHA Europe and President of AMIP, Italian PH
Patient Association.
18-22 September, Barcelona, Spain
■ ERS ANNUAL CONGRESS AND PHA EUROPE GENERAL ASSEMBLY
PHA Europe will be present with an exibitor booth also at the European Society for Respiratory
Medicine's Annual Congress in Barcelona. This Congress is the world's broadest respiratory
gathering. It involves not only leading experts in respiratory medicine, but also in other areas, covering
the entire spectrum of research and practice in respiratory medicine. Over 18.000 persons attended
the 2009 edition in Vienna. As in past years it is expected that there will be a number of sessions
dedicated to Pulmonary Hypertension. PHA Europe will be holding its 2010 General Assembly within
the context of the ERS Congress (see below).
18-20 September, Barcelona, Spain
PHAE GENERAL ANNUAL ASSEMBLY
The three day program of the 2010 General Assembly includes presentations from national associations, sessions for sharingbest practices and defining future strategies, updates on PH treatments and research from top medical experts and a meetingwith representatives of the industry. There is also a social program and delegates will have to opportunity to visit Barcelonaand spend an evening in town. All but one of our member associations have confirmed their attendance. We also have guestsfrom other PH patient associations in Europe. Fourty delegates from eighteen countries will be present. All European PH patientassociations are invited and it is still possible to apply. For further information please contact Pisana Ferrari at [email protected], tel +39 3484023432. n. 1 - Spring 2010
Interesting links and suggested reading
EU institutions:
European Parliament: www.europarl.europa.eu/
European Parliament Legislative Observatory: www.europarl.europa.eu/oeil/
European Council: www.consilium.europa.eu/showPage.aspx?id=&lang=en
European Commission: http://ec.europa.eu/
Economic and Social Committee: www.eesc.europa.eu/
European Health Portal
The Health-EU Portal contains a wide range of information and data on health-
related issues and activities at both European and international level. The portal
is intended to help meet EU objectives in the Public Health field, it is an important
instrument to positively influence behaviour and promote the steady improvement
of public health in the 27 EU Member States.
http://ec.europa.eu/health-eu/index_en.htm
EU Commission Directorate General for Health and Consumers (DG SanCo)
The EU Commission Directorate General for Health and Consumers has the task of keeping laws on the safety of food
and other products, on consumers' rights and on the protection of people's health up to date. It is also in charge of ver-
ifying that EU laws are correctly implemented in the 27 Member States.
http://ec.europa.eu/dgs/health_consumer/index_en.htm
Europe in action for healthier, safer, more confident consumers action
This booklet published by the EU Commission Directorate General for Health and
Consumers provides a very interesting overview of EU action in this field.
European Patients Forum - EPF
EPF is the umbrella organisation of pan-European patient organisations active in
the field of public health and health advocacy. It was founded in 2003 to become
the collective patients' voice at EU level. It currently represent 42 patients organ-
isations - which are chronic disease specific patient organisations operating at EU
level and national coalitions of patients organisations. Eurordis is a Member of EPF.
www.eu-patient.eu
Pharmaceutical Forum
The Pharmaceutical Forum is a high-level political platform for discussion set up in 2006 by Commission Vice-President
Günter Verheugen and Commissioner Markos Kyprianou. The aim is to enhance the competitiveness of the pharmaceu-
tical industry in terms of its contribution to social and public health objectives. The Forum brings together Ministers from
all European Union Member States, representatives of the European Parliament, patients, the pharmaceutical industry,
health care professionals, and insurance funds. European Patient Forum (see above) represents patients' organisations in
the Steering Group and in the three expert working groups: Relative Effectiveness, Pricing and Reimbursement and Infor-
mation to Patients.
http://ec.europa.eu/pharmaforum/
Aims of PHA Europe
Pulmonary Hypertension causes breathlessness and is a rare, progressive and currently incurable lung disorder. PHA EUROPE,European Pulmonary Hypertension Association, was founded in 2003 and is registered in Vienna, Austria, as an inter-national non profit organisation. PHA EUROPE is an umbrella organization bringing together Pulmonary Hypertensionpatient associations across Europe. The primary objective of PHA EUROPE is to establish a narrow cooperation betweenthe members and the European institutions, international organizations and public institutions worldwide and work towardsachieving the best possible standards of care for all European Pulmonary Hypertension patients.
Members of PHA Europe
PH Israel
Wilhelmstraße, 19 - 1120 Wien 13, Hashoshan st. Bet Shemesh - 99590 Israel www.lungenhochdruck.at - [email protected] www.phisrael.org.il - [email protected] Patiëntenvereniging Pulmonale Hypertensie vzw
Associazione Ipertensione Polmonare Italiana onlus
Cantincrodelaan, 48 - 2150 Borsbeek (Antwerpen) Via Vigoni, 13 - 20122 Milano www.ph-vzw.be - [email protected] www.aipiitalia.org - [email protected] HTAP Belgique, Asbl
Associazione Malati di Ipertensione Polmonare
Première avenue, 83 - 1330 Rixensart Via Bagnoregio, 51 - 00189 Roma www.assoamip.net - [email protected] National Association of PPH
PHA Polska
Hadzi Dimitar str. 37, B, 3 - 5800 Pleven - Bulgaria Duracza, 8/164 - 01-892 Warsaw PORTUGAL
Associação Portuguesa de Hipertensão Pulmonar
Sdruzˇení Pacientu˚ s Plicní Hypertenzí
Avenida Dr. Luis Navega, 38-42 - 3050 Mealhada P.O. Box 47, Praha 4, 14021 www.aphp-hp.org - [email protected] Schweizer PH-Verein (SPHV)
HTAP France
für Menschen mit pulmonaler Hypertonie
10, rue des Chardonnerets - 57365 Ennery Im Rossweidli, 1 - CH-8045 Zürich www.htapfrance.com - [email protected] Association HTAP Revivre
Pulmonale Hypertonie e.V.
Promenade de la Borgne, 23 - 1967 Bramois/VS Wormser Straße, 20 - 76287 Rheinstetten www.phev.de - [email protected] Associacion Nacional Hipertension Pulmonar
Association of Support and Mutual Help for PH Patients
Avenida de las Artes, 7 - 28300 Aranjuez - Madrid 32, Lokridos Str. - 17778 Tayros, Athens www.phagr.com - [email protected] Pulmoner Hipertansiyon Dernegi
Meriç Sokak Milas 2000 Is Merkezi 5A 19, Cházár András utca - 1146 Budapest Blok/26 - Bestepe Ankara csabada.eszter@t-online-hu - www.tudoer.hu www.pha-turkey.com - [email protected] Contacts
Pisana Ferrari - Vice President and European coordinator: Via G. Vigoni 13, 20122 Milano (Italy) [email protected] - tel. + 39 0258318547 - mob. + 39 3484023432 - fax + 39 02 29512476

Source: http://www.plicni-hypertenze.cz/downloadfile.php?id=35