MESSAGE FROM THE CEO
Welcome to our Spring edition of the quarterly IPPOSI newsletter. We welcome articles and event notices from any interested members just contact us on:
and we will be happy to include items of interest.
We began the year with Europlan which was held on the 20 January 2011, more than 200 delegates with an interest in rare diseases gathered in Farmleigh House in Dublin for the Irish conference. EUROPLAN is a three year (2008-2011), European-funded project, involving 25 countries, the main goal of which is to develop recommendations on how to define a strategic plan for rare diseases.
The EUROPLAN conference was organised by the Genetic and Rare Disorders Group (GRDO), IPPOSI and the Medical Research Charities Group (MRCG). The conference played a valuable part by bringing together all stakeholders with an interest in rare diseases and provided a forum for the many issues affecting rare-disease patients in Ireland. The topics of discussion were wide-ranging and diverse but focused primarily on the broad areas of research, centres of excellence for rare diseases, orphan drugs and access to treatment, and patient empowerment and support.
Since the Europlan meeting, the Department of Health has set up a policy group to work towards developing a National Strategy for Rare Diseases by 2013. Representatives from the three Umbrella Groups been appointed to this policy group. More news on this initiative in the coming weeks.
HTA Information Day
The IPPOSI Information Day entitled; Health Technology Assessment for Pharmaceuticals and New Medical Technologies - Where are we now? is scheduled to take place on TUESDAY 5TH APRIL 2011, EUROPEAN UNION HOUSE between 11am and 2pm. We have an interesting lineup of speakers and participants from patient groups, industry and science/health. IPPOSI have had assistance from Fionnuala Gibbons in Molecular Medicine Ireland, Dr. Martin Flattery and Dr. Mairin Ryan in HIQA and Jenny Hughes in GSK in organising the event, many thanks to them.
As you are well aware, over the last decade innovative medicines and new health technologies have contributed to significant improvements in health status and quality of life. All across Europe people are living longer. Treatments are also improving while becoming more expensive and resources to pay for such treatments are not increasing. We are well aware that these changes are putting our health system under increasing pressure.
This IPPOSI Information Day is an attempt to follow on from the IPPOSI Access to Medicines meeting held in 2008 where IPPOSI recommended the following:
- Ireland needs to foster a consensus based approach to the development of HTAs/PEAs rather than a prescriptive approach.
- The scope of HTA/PEA should not be limited to economic considerations only. Social, ethical, legal considerations are equally important.
- Patients and patient organisations need to be assisted and empowered to better understand HTA/PEA system and to formally engage in the HTA/PEA process.
The event is open to IPPOSI members and you can register with
Conference Programme available on http://www.ipposi.ie/index.php/events/event-articles
IPPOSI and the Medical Research Charities Group (MRCG) are organising a round table discussion entitled; "Towards a National Strategy for Patient Registers in Ireland". The aim of the meeting is to develop a briefing paper and list of concrete, achievable recommendations on the issue of Patient Registries in Ireland to present to the Minister for Health and key decision makers in the HSE.
The Round table seminar is open to a max of 50-60 people with short inputs from key individuals to stimulate discussion and debate. The meeting will have a rapporteur who will present the key issues and discussion points.
The event is scheduled to take place on Tuesday, 10th May, 2011, 12.30pm lunch, to start @1.00pm sharp in European Union House, Dawson St, Dublin 2.
Please register by emailing
Finally, I am pleased to announce two recent appointments in my capacity as CEO of IPPOSI, patient-led platform;
- The HIQA HTA Programme Advisory Group
- The Department of Health Rare Disease Policy Group
I will work to the best of my ability to represent the IPPOSI well developed and approved consensus views on Rare Disease Policy and Health Technology Assessments respectively.
EUROBAROMETER SURVEY REVEALS AWARENESS AND SUPPORT FOR RARE DISEASES AMONGST EU CITIZENS
Published on 28 February, in honour of the fourth International Rare Disease Day, the European Awareness of Rare Diseases Report presents the results of a Eurobarometer survey conducted by TNS Opinion & Social at the request of the Directorate General for Health and Consumers (DG Sanco) and coordinated by Directorate General Communication.
Seeking to gauge the awareness for rare conditions as well as the level of public support for European-level measures, the survey found that "...approximately 2 out of every 3 respondents know that rare diseases affect a limited number of people and require very specific care. Almost 1 in every 5 personally knows of someone suffering from a rare disease". While there were "significant differences" in awareness between the Member States, some "...95% of respondents believe there should be more European cooperation in this area and that rare disease patients should have the right to access appropriate care in another Member State". The survey, in the form of questionnaire, was undertaken in each of the 27 European Union Member States, with approximately 1000 citizens from each country participating, for a total of 26,574 interviews. The release of the report coincides with the formal adoption of the Cross Border Healthcare Directive - legislation of particular relevance to rare disease patients and their families (consult the accompanying news article). In a press release, John Dalli, European Commissioner for Health and Consumer Policy, stated: "I am encouraged to see that EU citizens want more European co-operation on rare diseases. This is important, because the required medical expertise may not be available within national borders. ...I want to stress that the European Commission is engaged in added value action to help citizens access the care they need across the EU".
(Ref: www.orpha.net )
THE EUROPEAN UNION COMMITTEE OF EXPERTS ON RARE DISEASES (EUCERD)
EUCERD was established following the publication of the European Commission decision of 30 November 2009 in the Official Journal of the European Union. Consisting of 51 members, including one representative coming from the ministries or government agencies responsible for rare diseases designated by the government of each Member State; four patient organisation representatives; four pharmaceutical industry representatives; nine representatives of ongoing and/or past Community projects in the field of rare diseases financed by the programmes of Community action in the field of health, including three members of the pilot European Reference Networks on rare diseases; six representatives of ongoing and/or past rare diseases projects financed by the Community Framework Programmes for Research and Technological Development; and one representative of the European Centre for Disease Prevention and Control, the EUCERD replaces the European Commission's Rare Diseases Task Force. The EUCERD held its first meeting on 9-10 December 2010 in Luxembourg. At the meeting, the Internal Rules for the EUCERD were discussed, along with the Road Map for the Implementation of the EU Policy on Rare Diseases 2010-2013, the Past activities of the Rare Diseases Task Force, Tools for the EUCERD, and much more. The public summary report for this meeting is now available online. Dr. John Devlin, Department of Health and Children is the Irish representative on EUCERD.
THE WORLD ORPHAN DRUG SUMMIT
Date: 31 May - 1 June 2011
Venue: Frankfurt, Germany
The World Orphan Drug Summit will tackle challenges that are currently slowing the development, market authorisation and patient access to orphan drug treatments. The event will focus on delivering in-depth discussion and practical solutions to provide participants with the strategies to grow their orphan drug business.
For Information on other International Rare Disease and Genetics related events please check out What's on Where section of http://www.orpha.net/actor/EuropaNews/2011/110316.html#20526
RARE DISEASE UK LAUNCH THEIR STRATEGY
More than half of people living with rare diseases in the UK are left to research and manage their conditions by themselves because of a lack of accessible medical expertise in the health service, according to a report released on the 28th February by Rare Disease UK (RDUK).
RDUK's report, Improving Lives, Optimising Resources, illustrates how patients are too often given insufficient information on their medical, psychological and social needs after being diagnosed. The lack of medical professionals who understand rare conditions results in patients finding unreliable, often frightening information and researching their own treatment and care paths.
A rare disease affects fewer than one in 2,000 people. Collectively, rare diseases are not rare. A total of 3.5m people in the UK live with a rare condition at some point in their lives - the equivalent to 1 person in 17. Rare diseases are often chronic, progressive, degenerative and often life-threatening.
The report shows the UK lacks the systems and structures to respond to the needs of people affected by rare diseases and sets out recommendations for a national strategy that will ensure the most effective use of NHS resources while immeasurably improve the lives of millions of people.
RDUK's recommendations cover the diagnosis, care, treatment and research of rare diseases. They bring together existing scarce knowledge and expertise - in the NHS, universities, industry, patient groups and medical research charities - and include:
- A UK online portal for rare disease information, linking to reliable sources and guidelines
- Inclusion of a rare disorders module on the medical curriculum and ongoing training
- Improvement of access to diagnostic tests, including a revision of the newborn screening programme
- Development of rare disease registries
- Personalised care plans for patients with rare conditions
LOW PROTEIN LIVING WEEKEND
The 4th Annual SHS Low Protein Living Weekend will take place on the 15th to the 17th of April in the Johnstown House Hotel, Enfield, Co. Meath. If you would like to receive a registration pack please call 1800 923 535 (ROI) or 0800 973 216 (NI).
Eibhlin Mulroe, CEO of IPPOSI will be addressing the participants on the work of IPPOSI in the development of a strategy for Rare Diseases in Ireland.
IMI CALL 4 - JUNE 2011 LAUNCH
The scientific priorities on which the 4th call will be based are now available. Topics include:
- Assessment of drug-induced toxicity in relevant organs - surrogates for early drug failure
- Enhancing translation in neurological disease
- European Medical Information System
- Stem Cells for Drug Development and Toxicity Screening
- Beyond high-throughput screening
- Disease heterogeneity / Taxonomy of disease
- Generic mapping of extreme genotypesCombination therapy development
Not all of these areas will be covered in the call.
For more information on the topics and how to participate contact Ciarán Duffy:
DUBLIN HOSTS THE EUROPEAN CITY OF SCIENCE, JULY 2012
Euroscience Open Forum - ESOF2012 will take place in Dublin from the 11th to the 15th of July. The event will bring together over 5,000 scientists, business leaders, government officials, policy-makers and international scientific media representatives to discuss the best of European science and to address all of the current major global scientific challenges, including Energy, Climate Change, Food and Health.
The call for scientific session proposals is now open. Go to www.esof2012.org to find the call with instructions on how to make a submission. The deadline is the 30th of June 2011.
ESOF2012- Where creativity and great science meet
Should you require any additional information, please do not hesitate to contact the ESOF Hub on
CONSENSUS DOCUMENT ON BRAIN RESEARCH
The European Journal of Neuroscience has published the 2011 version of Consensus Document on European Brain Research (see below).
The consensus document on European Brain Research, prepared by European Brain Council in collaboration with more than 200 European scientists and patients organizations, contains both proposals for future research programmes and describes the needs and achievements of research in Europe. The document highlights the need for strong basic research in neuroscience and the development of a strong European platform for brain research which is needed to address brain diseases, representing nowadays a societal emergency in European countries.
For more details see http://onlinelibrary.wiley.com/ doi/10.1111/j.1460-9568.2010.07596.x/pdf
CROSS-BORDER HEALTHCARE: THE EUROPEAN PARLIAMENT ADOPTS THE DRAFT DIRECTIVE
On the 28th February 2011, the Council approved the European Parliament's amendments on a draft directive aimed at facilitating access to safe and high-quality cross-border healthcare and promoting cooperation on healthcare between member states. Member states will have 30 months to transpose the directive's provision into national legislation. The new directive provides clarity about the rights of patients who seek healthcare in another member state and supplements the rights that patients already have at EU level through the legislation on the coordination of social security schemes (regulation 883/04).
It meets the Council's wish to fully respect the case law of the European Court of Justice on patients' rights in cross-border healthcare while preserving member states' rights to organise their own healthcare systems.
- As a general rule, patients will be allowed to receive healthcare in another member state and be reimbursed up to the level of costs that would have been assumed by the member state of affiliation, if this healthcare had been provided on its territory; instead of reimbursing the patient, member states of affiliation may also decide to pay the healthcare provider directly;
- For overriding reasons of general interest (such as planning requirements for ensuring permanent access to a balanced range of high-quality treatment or the wish to control costs and to avoid any waste of resources) a member state of affiliation may limit the application of the rules on reimbursement for cross-border healthcare;
- Member states may introduce a system of prior authorisation to manage the possible outflow of patients. This is, however, limited to healthcare that is subject to planning requirements, such as hospital care (defined as care involving overnight hospital accommodation) and healthcare that involves highly specialised and cost-intensive medical infrastructure or equipment, healthcare that involves treatments presenting a particular risk for the patient or the population, or healthcare which would be provided by a healthcare provider which could raise serious concerns with regard to the quality or safety of the care;
- In order to manage incoming flows of patients and to ensure sufficient and permanent access to healthcare within its territory, a member state of treatment may adopt measures concerning access to treatment where this is justified by overriding reasons of general interest (such as planning requirements for ensuring permanent access to a balanced range of high-quality treatment or the wish to control costs and to avoid any waste of resources);
- Member states will have to establish national contact points that must provide patients with information about their rights and entitlements and practical aspects of receiving cross border healthcare, e.g. information about healthcare providers, quality and safety, accessibility of hospitals for persons with disabilities, to enable patients to make an informed choice;
- Cooperation between member states in the field of healthcare has been strengthened, for example, in the field of e-health and through the development of a European network which will bring together, on a voluntary basis, the national authorities responsible for e-health; another example is rare diseases, where the Commission will have to support member states in cooperating in the field of diagnosis and treatment capacity;
- The recognition of prescriptions issued in another member state has been improved; as a general rule, if a product is authorised to be marketed on its territory, a member state must ensure that prescriptions issued for such a product in another member state can be dispensed in its territory in compliance with its national legislation;
- Sales of medicinal products and medical devices via internet, long-term care services provided in residential homes and the access and allocation of organs for the purpose of transplantation fall outside the scope of the directive.
PATIENT SAFETY AND QUALITY OF CARE
The Commission's Patient Safety and Quality of Care Working Group met on the 26th of January 2011. The Group discussed a questionnaire to Member States to gather information on the progress of the implementation of the Council Recommendation on patient safety. The Council Recommendation includes a number of specific recommendations concerning the empowerment and involvement of patients.
The European Patients Forum considers it is important to gather feedback also from the perspective of patient organisations as to what extent these provisions are being implemented in Member States, and we will be inviting their members' views on these shortly.
The second part of the meeting focused on several presentations on patient safety-related initiatives and projects, including the HANDOVER project, EPSO, the European Forum of Primary Care and the WHO initiative on patients' rights and patient safety, in which EPF is a participant.
The agenda and presentations from this meeting are available to the public on the Commission's website: http://ec.europa.eu/health/patient_safety/events/ev_20110126_en.htm
(Ref: EPF newsletter)
DIRECTIVE ON COUNTERFEIT MEDICINES ADOPTED BY EUROPEAN PARLIAMENT
The EU legislation on the prevention of the entry into the legal supply chain of counterfeit medicinal products, amending Directive 2001/83, was adopted by the European Parliament on 16 February, following informal agreement reached by the EU Institutions at the end of December. The vote comes after months of delay due to several issues that needed to be ironed out in the negotiations. The Directive is now awaiting formal adoption by the Council.
More information available from EPF newsletter
EUROPEAN PATIENTS' RIGHTS DAY
The 5th European Patients' Rights Day will be celebrated this year on 11 and 12 April in Brussels with a European Conference focusing on best practices in civic participation in the field of health, and cross-border healthcare. Please visit www.activecitizenship.net/content/blogcategory/72/179/ for more information on the Patients' Rights Day, how to get involved, and how to submit best practices and link in with national patients' rights events.
CONSULTATION ON TRANSPARENCY DIRECTIVE
The European Commission is initiating a process of review of the "Transparency Directive" (89/105/EEC). In this framework, it is seeking the views of interested parties to determine how the existing EU rules on the transparency of pricing and reimbursement procedures for medicines may be modernised.
Although the prices of medicines and their reimbursement status are decided by each Member State, the Transparency Directive was adopted in 1989 to facilitate the free movement of medicines in Europe. It has never been amended since its entry into force. Nevertheless, the pharmaceutical market and national policies to control pharmaceutical expenditure have considerably evolved in the last twenty years.
This consultation will help the Commission propose initiatives to ensure that the directive continues to achieve its objectives in the current context. At present, the Transparency Directive applies exclusively to medicinal products. The consultation also aims to assess its relevance to the medical devices sector.
How to submit your contribution
In the interests of transparency, organisations (including, for example, NGOs, trade associations and commercial enterprises) are invited to provide the public with relevant information about themselves by registering in the Interest Representative Register and subscribing to its Code of Conduct.
If your organisation is not registered, you have the opportunity to Register now. Then return to this page to submit your contribution as a Registered organisation.
View the questionnaire
The questionnaire is available in English, French and German.
The maximum time to complete the questionnaire allowed by the system is 90 minutes. Partial responses will not be saved. This means once you start filling in the questionnaire, you have to answer all compulsory questions to submit your response. You can, however, preview/print a PDF version of the online questionnaire to help you to prepare your response.
Please note that the questionnaire is organised in distinct modules adapted to different target groups, based on their identification in the first section. As each target group only receives the questions relevant to it, this may result in apparent inconsistencies in the numbering of the questions viewed on the screen. This will not affect the treatment of your replies.
Transparency Directive 89/105/EEC of 21 December 1988 relating to the transparency of measures regulating the prices of medicinal products for human use and their inclusion in the scope of national health insurance systems.
DG Enterprise and Industry, Unit F/5: Food and Healthcare Industries, Biotechnologies
European Commission, BREY 10/220, B-1049 Brussels, Belgium
2011 PUBLIC HEALTH CALL NOW OPEN
The Executive Agency for Health and Consumers (EAHC) has just published a call for proposals for projects, operating grants, conferences and joint actions.
The EAHC programme focuses on public health. The main priorities are:
- To improve citizens' health security
- To promote health, including the reduction of health inequalities
- To generate and disseminate health information and knowledge
Proposals are expected to address important problems in European public health and be in line with the Commission's priorities as expressed in the Work Plan. Successful proposals should concentrate on aspects of public health that cannot be achieved on a national level and should ensure a significant impact across the EU. The deadline for submission of proposals is 27 May 2011. Some priority areas covered this year include: rare diseases, health determinants, cross-border healthcare and legislation on products and substances.
Details on the different funding instruments as well as the Work Plan can be downloaded from the EAHC website. The funding priorities for 2011 are listed on pages 9-21 of the Work Plan document.
Note: Calls for Tenders to be published during the course of 2011.
The Work Plan 2011 foresees a significant number of calls for tenders for the provision of services, in particular preparation of surveys, analyses, training programmes and studies concerning public health.
The calls for tenders will be launched during the course of 2011. In order to be alerted of when open calls for tenders are launched, please sign-up to the mailing list by writing to:
If you require further information please contact:
Kay Duggan-Walls, HRB - National Focal Point for the Public Health Programme:
EUROPEAN GENDER SUMMIT 2011
The European Science Foundation, genSET - gender in science, and the European Cooperation in Science and Technology would like to invite you to the first European Gender Summit on 8-9 November 2011 in Brussels. The first European Gender Summit provides a forum for stakeholders from research, industry and policy to jointly explore how gendered methodologies can stimulate innovation and advance scientific excellence. The summit will map the current state of knowledge, provide a forum for interdisciplinary debate and draw up concrete action plans for research and innovation policy.
More information: http://www.gender-summit.eu/index.php
2011 INTERNATIONAL CLINICAL TRIALS' DAY
The International Clinical Trials Day meeting organised by ECRIN (European Clinical Research Infrastructures Network) is to be held at the Palais Harrach, Freyung 3, in Vienna, Austria on 20th May 2011.
The International Clinical Trials' Day is an annual event developed by ECRIN. The 2011 event will be an opportunity to further the patients' and patient organisations' understanding of the methods, current issues, and importance of clinical research, and to facilitate debate between participants, physicians, researchers and the drug industry. ECRIN is an infrastructure, which interconnects national networks of clinical research centres in Europe. ECRIN offers information, consultancy and services to sponsors and investigators in order to facilitate high quality multinational clinical trials in Europe www.ecrin.org.
Clinical Research Centers in Ireland also plan to host events and details will follow as soon as we know.
PATIENTS' RIGHTS TO SUPPORT AND REDRESS: IMPLICATIONS FOR POLICY AND PRACTICE
One day conference - Friday 10th June, Edinburgh
Outlined in the vision for a 'mutual NHS' the conceptual shift from viewing patients as 'service users' to 'active partners' is having a profound effect on healthcare policy and practice.
Developed in partnership with the Scotland Patients Association, this national one-day conference will focus on three core strands of patient rights and responsibilities and will explore, through examples of good practice, how NHS providers can successfully engage and empower patients in the design, delivery and evaluation of local services.
Session 1 - Understanding the patient experience
Exploring efforts at a local and national level to gain a greater understanding of the patient experience and how service providers can encourage patient feedback
Session 2 - Valuing patient complaints
Considering the importance of empowering patients and valuing patient complaints - including an update on the work being undertaken by the SPSO to develop a new fit-for-purpose complaints system
Session 3 - Offering effective redress
A review of the findings of the no-fault compensation review group headed-up by Professor Sheila McLean and a discussion on the way forward for Scotland in offering effective redress to patients
The programme will also include a number of case studies featuring real-life patient experiences.
To find out more contact Jane on 0131 556 1500 email Jane Wilson
MMI SEMINAR - CAPTURING THE BENEFITS OF RESEARCH FOR HEALTH, JOBS AND INNOVATION
Capturing the benefits of research for health, jobs and innovation -Thursday 7 April from 18:00 to 19:30 hrs
Advances in genomic and proteomic research are changing our understanding of human health and disease. How do we ensure that these advances in research are translated into better outcomes for patients and advances in population health? How do we link the expertise of our researchers with that of our health services so patients benefit, new jobs are created and Ireland's knowledge economy grows?
Molecular Medicine Ireland invites you to attend a seminar to explore these issues on Thursday 7 April from 18:00 to 19:30 hrs at Studio 1 in the Science Gallery, Naughton Institute, Trinity College, Pearse St, Dublin 2.
Two international leaders in genomic and proteomic research will address the seminar:
Dr Alan Winter, President and CEO of Genome BC, will speak from a Canadian and British Columbian perspective on the best way to encourage the translation of health research into the health system and the potential benefits to society; and
Dr Frank Giles, recently appointed Professor of Cancer Therapeutics at NUI Galway and Trinity College Dublin and an international authority on the development of new drugs to treat chronic myeloid leukaemia (CML), will address the challenge of translating the benefits of health research in Ireland.
Tom Lynch, Chairman of Molecular Medicine Ireland, will introduce the speakers and chair the discussion.
A wine reception will follow the seminar.
Please RSVP to
by Monday 4 April.
ICRIN NEWS: RESEARCH GOVERNANCE AND SPONSORSHIP
ICRIN Seminar on Research Governance and Sponsorship was held on 16 September in the UCD Clinical Research Centre at the Mater Misericordiae University Hospital. The report of the proceedings can be found and downloaded at:http://www.molecularmedicineireland.ie/show/item/1/1565
The seminar addressed the difficulty principal investigators face in acting as sponsors of clinical and translational research studies in Ireland and abroad and the underdeveloped nature of our system of research governance. Both are major obstacles to the development of clinical research in Irish hospitals, universities and the broader research community. The report describes how these issues are being tackled in Ireland and abroad.
The report summarises the presentations of the speakers including Professor Bill Powderly, Dublin Academic Medical Centre, Professor Stephen K. Smith of Imperial College Healthcare NHS Trust, Professor Paul Browne, ICORG and Dr Geraldine Boylan, UCC. It also highlights the key messages of the seminar for a more effective system of research governance in Ireland
CLINICAL TRIALS ONLINE
The EU Clinical Trials Register (https://www.clinicaltrialsregister.eu) was recently launched by the European Medicines Agency (EMA).
The online register gives for the first time public access to information on interventional clinical trials for medicines authorised in the 27 EU Member States and Iceland, Liechtenstein and Norway. The database also allows the public to search for information on clinical trials authorised to be carried out outside the EU if these trials are part of a paediatricinvestigation plan.
The press release document referring to this matter can be found here.
And Finally - Tell Us What You Think
We welcome any feedback or ideas, please let us know. Contact Karen on