Umbrella Organization of European Waldenström patient supportgroups and patients


Newsletter June 2014

In this Newsletter you can find four short reports of: the EURORDIS Membership Meeting and Conference (Berlin, May), the Lymphoma Coalition Europe Meeting and the Congress of the European Hematology Association (Milan, June). These conferences were attended by EWMnetwork’s secretary. The information in this Newsletter may give you some idea about EWMnetwork’s networking and advocacy at the European level: often not disease specific for Waldenström but in collaboration with other patient organizations in the field of Lymphoma and rare cancers.
EURORDIS (Eur. Organization for Rare Diseases) Membership Meeting (MM) Berlin, May 8
Between 150-200 representatives of patient organizations participated in the Membership Meeting  and attended the “Learning from each other sessions” and one of the “patient advocates capacity building workshops”.
The workshops were a good introduction to the difficult  scientific language that followed the next two days. The “Patient advocates capacity building workshop” that I attended was dedicated to “Patient advocacy to improve access to orphan medicinal products”. Some opinions and trends:
- A new sustainable business model for marketing authorization and access to drugs is necessary: in the present situation too much energy and money is wasted because of lack of flexible regulations and lack of collaboration between different stakeholders.
An important initiative in this field are the pilots of the Mechanism of Coordinated Access (MOCA) to Orphan Drugs, developed within the EU Stakeholders Forum on Corporate Responsibility in Pharmaceuticals. MOCA  promotes pan-European collaboration between EU Member States on Orphan Drugs, based on a common approach to the Value of new medicines, pricing, volume and post-Marketing Authorization data generation.
- Collaboration  is also needed in the field of Health Technology Assessments (HTA). A European network for HTA  for the whole cycle could be the answer.
- Potential and limits of patient progressive access to/ adaptive licensing of new medicines,applied to Rare Diseases, was discussed.
- Last but not least , the power of setting up a patient organization was shown by a young Cystic Fibrosis patient who told her impressive story how, pushed by patient actions in Ireland, the development of a new drug for this disease ánd the reimbursement  of it were reached.
EURORDIS, EUPATI a.o. support volunteers with trainings
Discussions on topics like the above mentioned “marketing authorization and access to drugs, Health Technology Assessments, adaptive licensing (for faster access to drugs) etc” are not easy to grasp for a volunteer.
Eurordis supports volunteers with trainings (e.g. a Summer Academy) to be increasingly involved in Eurordis activities, participating in NGO partnerships and representing patients in working groups
and committees of the European Commission and EMA (European Medicine Agency).
Eurordis also promotes greater convergence and collaboration between the 33 National Patient Alliances in Europe, as well as between Eurordis and these National Alliances. The same applies to the 41 disease specific European/ International Federations & Networks (like EWMnetwork).
If you are interested have also a look at internet what EUPATI offers .
More  information about Eurordis you can find at .
 EURORDIS’ European Conference on Rare Diseases (ECRD), May 9-10
A very fruitful conference brought together around 800 stakeholders from all interested parties across all European countries: patients’ representatives, academics, healthcare professionals, industry, payers, regulators and policy makers.They meet each other and discuss relevant themes and actual problems like unity in Healthcare Technology Assessments, Research and Access to orphan drugs (off label use etc.). So many motivated specialists together, but so many problems to solve, because in many cases each European country is entitled to make his own decisions in healthcare matters.
During these two days the topics mentioned above and many more, were (divided in 6 themes)  discussed in parallel sessions with introductions from renowned speakers, followed by panel discussion with the participants. Twelve sessions to choose from on Friday and sixteen on Saturday.
you can find the speaker presentations via mobile app and website at It is worth to have a look!
One topic I like to mention is the importance of the National Plans for Rare Diseases and the fact that by 1 january 2014 most EU countries have adapted their national plan. Next step is action for adequate patient registries for use on the national level but also to be used on the European level.
I conclude with mentioning the ambition of the International Rare Disease Research Consortium (IRDiRC) in Horizon 2020 : 200 new therapies for RD (at the moment 100) and a diagnosis for  most Rare Diseases!
1st meeting Lymphoma Coalition Europe (LCE,, Milan June 12,
Summary LCE  meeting: this meeting was the start of more collaboration between lymphoma organizations in Europe (e.g. plans for data collecting by Lymphoma Coalition Worldwide on clinical trials and the availability of medicines per European country). As a result of meeting each other in Milan the Swiss- and a German Lymphoma Organization joined EWMnetwork as affiliates; Portugese and Croatian Lymphoma Organizations consider to join EWMnetwork when they have information on Waldenström’s disease available on their website.
This first meeting in Europe, organized by Lymphoma Coalition Wordwide, was attended by representatives of Lymphoma Organizations from Croatia, Denmark, France, Italy, Netherlands, Portugal, Romania, UK, Switzerland and by me as a representative of EWMnetwork.
Collaboration patient organizations and health care professionals
The meeting started with a discussion between patient organizations and five doctors how one might work together to support the newly diagnosed patient in their journey. The role of nurses apart from doctors was discussed as well as new ways of psycho-social care (e.g. cognitive therapy for fatigue),
special consulting hours by nurses or by volunteers from patient organizations and apps for patients. Lymphoma Coalition will develop a toolkit so we can learn from each other.
Lymphoma Coalition Europe (LCE), information project
Next was discussed how LCE might move forward to create a plan of action to advocate together. All agreed that best practices and data collecting  play an important role.  A Lymphoma Information Project with data per country specified on Lymphoma Survey results, on access to and follow up of clinical trials, access to and reimbursement of medicines etc. and the start of a group for an Education Program would be a good base for collaboration.
LCE: Possible conclusion for the role and position of EWMnetwork
 It might be good to make a difference between disease specific advocacy (only for WM) and advocacy for lymphoma or rare cancers more in general. By joining a collaboration in Europe of Lymphoma organizations that also include WM, EWMnetwork gets a stronger basis, while not loosing sight of WM specific issues. We will be able to get support not only from WM advocates but also from Lymphoma advocates, if applicable.
Sessions for patient organizations at the congress of the EHA (European Hematology Association), Milan June 11-14, 2014
Summary EHA congress: the voice of Patient Organizations gets little by little stronger as could be experienced at the 2014 congress of EHA (European Hematology Association). Topics this year were: access to effective drugs and the position of generics in hematology; psycho-social care, the design of and access to clinical trials.
Patient Advocacy Meeting, June 13
The EHA congress 2014 counted around 10.000 (!) attendants. EHA had offered 50 free registrations to patient organizations, around 35 patient organizations participated.


In the Patient Advocacy Session on June 13, Jan Geissler (CML advocates) called to mind that in 2010 35 minutes for patient organizations were available in the EHA program and in 2014 150 minutes, so we are making progress. Main results of this meeting were the discussion and selection of patient advocacy topics for the EHA congress 2015 and the formation of a working-group to organize the
EHA  Advocacy Program 2015. Important is to attract doctors to the EHA patient advocacy sessions with the program.
Patient Advocacy Session, June 14: Generics in Hematology: the doctors and patients perspective.
Some conclusions/ opinions:
  •  Pharmacological research shows that there are good generics, but also a great variety of substandard generics, copies and even complete counterfeits;
  • Control by governments on the quality of generic drugs is at the moment practically impossible (too many, quickly changing new generics; post marketing studies are important for effects and side-effects of medicines;
  • From the hematologists’clinical perspective an example was given of a much less effective generic medicine that without clinical trial was given to CML patients with “systematic mastocytosis mutation” (the mutation comprises nearly 90% of cases of mastocytosis).
  • From the patient view the advise was given: if you “have to” change to a generic medicine, be very watchful on side effects and discuss them if necessary with your specialist and with fellow patients.
Patient Advocacy Session,  June 14, The challenges of young patients with old people’s diseases
A patient and a nurse told their experiences (consequences for sexuality, for self esteem etc), followed by a discussion about the need to pay more attention to these aspects.
Symposium, June 14:”What do you mean he can’t have treatment?”, An interactive session for hematologists and patients
In a played clinical consultation (a doctor with a patient, an older man of 74 accompanied with his very emancipated daughter, the last two played by professional actors), the doctor told the patient that he could not participate in a clinical trial because of restrictions by the manufacturer.
A lively discusssion was held around the issue:
Access to an effective drug relies on innovative research and access to clinical trials (CT’s).
The design of CT’s for registration purposes is frequently constrained by either the pharmaceutical company’s or the regulatory authority’s perceived requirement to assess the efficacy of the new agent on a homogenous patient population. Consequently major exclusions related to older age and prior malignancy are frequent and render many patients ineligible for recruitment.
Regulatory issues also profoundly affect access to medicines in Europe; a lack of anticipated return on investment leads companies to disregards smaller markets.
Under the current legislation there is no mechanism to ensure that, once a medicinal product is authorized in the European Union, it is marketed in all Member States. The resulting non-availability of certain medicines in some countries is a major source of health inequalities in the EU.
The issues of eligibility that effect elder patients may be further affected by attitudes of both clinicians and patients to embarking on treatment with newer (and potentially more effective) therapies with on the background an awareness that resources are not unlimited. The greatly increased availability of information about new and effective treatment on internet means that even if older people are not well informed, other family members may well placed to act as their advocates.  
Doctors and patient organizations can play here a prominent part.
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