EUCERD replaced by new Commission Expert Group

May 30, 2014 in News

The European Union Committee of Experts on Rare Diseases was replaced from 2014 onwards by the European Commission Expert Group on Rare Diseases, established via Commission Decision 2013/C 219/04 of 30 July 2013 which had its first meeting on 11-12 February 2014 and which will meet again on 3-4 July 2014.

Theexpert group on rare diseases helps the European Commission formulate and implement policy to combat rare diseases.

The Commission expert group may:

  • assist the Commission in the drawing up of legal instruments and policy documents, including guidelines and recommendations;
  • advise the Commission in the implementation of Union actions and suggest improvements to the measures taken;
  • advise the Commission in the monitoring, evaluation and dissemination of the results of measures taken at Union and national level;
  • advise the Commission on international cooperation;
  • provide an overview on Union and national policies;
  • foster exchanges of relevant experience, policies and practices between the Member States and the various parties involved

A new site for the Expert Group is currently being created.

The members and their representatives to the Commission expert group on rare diseases were appointed by a Commission Decision of 7 January 2014 following a call for expression of interest.

The group is composed of one representative per Member State, stakeholder organisations and rare diseases experts.

The list of members of the new Expert Group can be found here.

Rare Disease Day 2014: Join together for better care

February 20, 2014 in News

Join us in celebrating the 6th international Rare Disease Day on 28 February 2014.

On 28 February 2014 we commemorate 7 years of Rare Disease Day and once again rare disease communities around the world are joining hands to show solidarity and generate awareness. Rare Disease Day is an annual, awareness-raising event co-ordinated by EURORDIS at the international level and by National Alliances and Patient Organisations at the national level.

The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients’ lives.

Rare Disease Day 2014 focuses on care and encourages everyone in the rare disease community to Join Together for Better Care. The theme for 2014 highlights that the many different facets of rare disease care represents a universal need for patients and their families around the world. Advocating at the local and national level to improve the lives of people living with a rare disease and their families is necessary.

The campaign targets primarily the general public but it is also designed for patients and patient representatives, as well as politicians, public authorities, policy-makers, industry representatives, researchers, health professionals and anyone who has a genuine interest in rare diseases.

Since Rare Disease Day was first launched by EURORDIS and its Council of National Alliances in 2008, more than 1000 events have taken place throughout the world reaching hundreds of thousands of people and resulting in a great deal of media coverage.

Take a look at the official Rare Disease Day website to find the activities being organised in your region.


Why accomplish you first need free Musically tastes?

January 12, 2014 in News

CLEVELAND, Ohio aIf you have children here at centre school, it’s likely that you realize of about musical. Ly, the very video sociable marketing, and its own brand-new spinoff, live. Ly. But are all these applications protect in order for children?

Here’s the very rundown. What’s musical. Ly?

Musical. Ly, the very Shanghai-based social-media marketing, is definitely extremely favored by teenagers and then preteens. Critiques here at app’s choice of members vary from 60 mil right to 120 mil. Irrespective, millions of children are using that it to build up 15-second video clips of themselves singing, lip-syncing and then dancing. The very app, that was released here at 2014, is definitely for free in order for Apple and then Android gadgets. Members, or “Musers” mainly because fans of a app call themselves, are more likely to follow finance to keep tabs on the favorite artists. Mega-popular superstars, involving Ariana Grande and then Selena Gomez, use the app. Musers are more likely to dream posts, comment, call on questions and invite people to release duets. They can level mention their musical. Ly content material here at other sociable press platforms, and then like the majority of of a platforms, musical. Ly is definitely hashtag centric. This past year, the very #DontJudgeChallenge proceeded to go viral arising from musical. Ly’s musers. The very sociable press advertising campaign was going to conquer comparison shaming, according to MTV, but level has done the opposite simply by reinforcing cosmetic “ideals. “

Musical. Ly members shared video clips of themselves seeking “ugly” by having glasses, pimples, distance oral, thick eyebrows and then intelligence hair. Watch the very video above to view some of the #DontJudgeChallenge, and then realize that video has recently almost 120 mil destinations here at YouTube. Essentially, musical. Ly accepts its members right to express themselves here at a really creative trick. But lots of the app’s members are all young. What’s more, a man or woman if ever its very best stars, Jacob Sartorius, is definitely 13. He has recently above 10 mil fans on the app. Right to download and then sign up for the very app, members need to be at the very least 13. But why don’t prove to be accurate, it isn’t next to impossible right to rest of about your age here at the net. What’s live. Ly?

Live. Ly, the very live-streaming video app, is definitely fairly brand-new. It turned out released in may and then racked up above 2 mil downloads here at monthly. Mainly because we have most seen, and that traveling acknowledgement in order for live-streaming sociable press by having Twitter’s Periscope and then Facebook Live. Mainly because musical. Ly expectations music, live. Ly has recently musers for free reign on how they wish to introduce fans and then accepts musers right to talk to fans suddenly. Musers can pick obtained from a mix of categories, involving make-up, way of life, sing, dance, and the like. , right to tag within their live video clips. So can be these applications protect for that centre schoolers?

Perhaps. However the next to impossible role is definitely your choice. You must have right to make sure your guy disables distance new products over the app here at their mobile phone controls. Then during the app, you must have right to release three tabs during the app settings:

Only friends are more likely to direct. Ly me

Cover up distance detail

Confidential believed

First of all here at live. Ly, members transmission here at off their musicl. Ly believed, and so the environment likes are more likely to still exist an equal over the live-streaming app. In order for live. Ly, strain the very requirement for locating clever with all the app. Live streaming is definitely exactly what that it appears like; your own body’s guy will be live on the net, without the the capability to edit the very video before now everything else view it. Distance of a live-stream is definitely crucial as it can provide away information about where your own body’s guy lives and then spends length. Here’s one more thing to keep in mind. Musical. Ly is definitely certainly then a music-based app, but not all the music are all guy friendly. You’ll get tens of thousands of song categories to choose from and then what is likely dream millions of music, so your guy perhaps has not been going to prove to be fans hack singing Disney music.

2013 edition of the State of the Art of RD Activities report now online

July 9, 2013 in News

The 2013 edition on annual report on the State of the Art of Rare Disease Activities in Europe, covering activities up to the end of 2012 is now available online. Previous editions can be found in here

Report on the state of the art of rare disease activities in Europe – 2013 edition

This report covers activities up to the end of 2012.

Individual reports on the activities in each of the European countries covered by this year’s report are also available via the country pages of the ‘National resources’ sub-tab.


All previous reports of the EUCERD are accessible here.

IRDiRC delivers a successful and inspiring conference: a common goal emphasised

May 14, 2013 in News, Research

The first IRDiRC conference was held on April 16-17 2013 in the charming city of Dublin, Ireland. Thought leaders from all over the world captured the audience with stimulating,  inspirational and highly informative talks on the past, present and future of rare disease research. IRDiRC was unanimously hailed as need of the time to advance the cause of rare disease research. IRDIRC promises to contribute in development of 200 therapies for rare disease and means to diagnose all of them by 2020. Rare disease stakeholders discussed the ways and means to help reach these goals. They provided examples of success stories as well as suggestions of the innovative ways in which stakeholders can come together to fulfill this goal.

This truly international conference was attended by more than 400 participants representing Europe, North America, Australia and Asia. It was recognised by one and all that expertise in rare disease, like the patients, are scattered across the globe. It is thus imperative for the global rare disease community to put their heads together to solve the great big jigsaw of rare disease, of which everyone holds a piece. This meeting boasted of attendance from policy makers, industry leaders, academicians as well as patient organisations from around the world. Representatives from regulatory bodies such as FDA, EMA, the Canadian and Australian regulatory agencies shared their expertise and pushed the need for more regulatory success for orphan drugs. Patient organisations such as Rare Voices Australia, Eurordis and Genetic Alliance US, gave an overview of the current need of patients and how coming together with a common agenda is urgent, but also achievable. Industry partners emphatically expressed the need to work with academicians, patient organisations and regulatory bodies to significantly increase the number and quality of drugs that is accessible to all. The buzz word was collaboration, collaboration, collaboration.

Mirroring the scientific committees of IRDiRC, the conference panels were divided into 3 tracks- therapies, diagnostic and interdisciplinary track-with experts in each area presenting the current outlook and the way forward. The therapies track addressed issues that deal with providing better treatment for patients such as drug repurposing, developing innovative therapies and ensuring an open dialogue with the regulatory bodies so that the treatments developed painstakingly see the light of day. The diagnostic track articulated the current projects that are underway to identify the causes of the rare diseases, the tremendous advancements in Next Generation Sequencing including the use of data generated from it and the crucial understanding of the Human Phenome. The speakers in the Interdisciplinary track highlighted the means by which successful collaborations can lead to successful treatments and diagnostics. They also endeavoured to delineate how future challenges in the extremely complex world of rare disease research can be overcome. Economic and ethical issues such as those that have cropped up due to recent advancements in technology, especially next generation sequencing, were also discussed at length.

Dr Ruxandra Draghia-Akli who is a champion of the rare disease cause and served IRDiRC as its chairperson with impressive results, passed on the baton to Dr. Paul Lasko, a distinguished member in the field of rare disease research. The success of this conference promises a much brighter future for rare disease patients. IRDiRC has 32 members representing research funding bodies.  Organisations interested in becoming members of IRDiRC are encouraged to apply.

New EUCERD Recommendation on RD European Reference Networks

February 21, 2013 in News, Recommendation

On the 31 January 2013, during the seventh meeting of the European Union Committee of Experts on Rare Diseases, the EUCERD Recommendations on European Reference Networks for Rare Diseases (RD ERNs) were unanimously adopted by the 51-member EUCERD.

There are around 6000 rare diseases and most are unknown to healthcare professionals so rare diseases patients suffer from not knowing where to consult. To overcome this, some Member States have established centres specialised in some rare diseases/groups of rare diseases which have proven to be very efficient in improving quality of care. Now the European Commission is planning to link these centres together through European Reference Networks (ERNs) in order to gather expertise and improve healthcare for rare disease patients.

The development of centres of expertise and European Reference Networks in the field of rare diseases is encouraged in the Council Recommendation on an Action in the Field of Rare Diseases (2009/C 151/02) (8 June 2009) and in the Directive on the application of patients’ rights in cross-border healthcare (2011/24/EU) (9 March 2011) as a means of organising care for the thousands of heterogeneous rare conditions affecting scattered patient populations across Europe. European Reference Networks (ERNs) are one of the structures foreseen by the aforementioned Cross-Border Healthcare Directive to share knowledge, facilitate the mobility of expertise, and to allow Member States to provide highly specialised services of high quality for patients where this would have been impossible without European networking, such as in the case of rare diseases.

The EUCERD, formally the EC Rare Diseases Task Force, has already issued a series of reports investigating the state-of-the-art in the field, and a recommendation in 2011 on Quality Criteria for Centres of Expertise for Rare Diseases in Member States The 21 recommendations on RD ERNs build upon these previous achievements and will serve both to inform the Commission services and expert groups working on criteria for the creation and designation of ERNs in the context of the Cross-Border Healthcare Directive, as well as the Member States who are developing their healthcare pathways at both the national and EU levels in the field of rare diseases in the context of national plans/strategies for rare diseases which the Council has urged all Member States to elaborate by 2013.
The recommendations cover a range of points including the mission, vision and scope of ERNs, their governance, their composition, their funding and evaluation, as well as their designation.

The recommendations have been presented to the Cross-Border Healthcare Expert Group at the European Commission to inform their work on the designation criteria for ERNs on 6 February 2013.

Consult the recommendation.

Rare Disease Day 2013 – Watch the video!

February 8, 2013 in News

Join us in celebrating the 6th international Rare Disease Day on 28 February 2013.

On 28 February 2013 we commemorate 6 years of Rare Disease Day and once again rare disease communities around the world are joining hands to show solidarity and generate awareness. Rare Disease Day is coordinated by Eurordis with national alliances across 24 European countries and was celebrated by 63 countries worldwide in 2012. This year several new countries– including Bahrain, Iceland, Israel, Palestine and Singapore-will also be partipipating. For the past 6 years, this day has been a significant benchmark of how far we have come in our quest to help rare disease patients. It is also a day of reflection to acknowledge how much more can be achieved if we work collectively. In the spirit of global collaboration, the slogan this year “Rare disorders without borders” expresses the need for reaching across and sharing knowledge and resources to further the cause of the rare disease community.

This year’s official video for Rare Disease Day 2013 has been received enthusiastically, with more than 13,000 views on youtube! Several people provided their services free of charce to produce this video which is available in 10 languages. Patient organisations featured in this video are members of the Associazione Italiana Sindrome di Noonan, Duchenne Parent Project and Xeroderma Pigmentosum Society. Other than participating in the events and activities organised by the rare disease community in your part of the world, you can also upload your story and photos on the Rare Disease Day website.

A prelude to the Rare Disease Day was the meeting in Brussels on 26 February 2013 where “Eurordis and Members of European Parliament Ms Antonyia Parvanova (Bulgaria) and Mr Cristian Silviu Buşoi (Romania) co-hosted a multi-stakeholder policy event to examine how different policy measures can help improve access to therapies for rare diseases”. This meeting precedes the vote in the plenary session of the Parliament for “EU Transparency Directive on medicinal products”. The participants highlighted “the need for transparency around pricing and reimbursement of medicinal products” and discussed how faster, more equitable access to medicines can be achieved.

Take a look at the official Rare Disease Day website to find the activities being organised in your region.


Summary report of 7th EUCERD meeting now available

December 18, 2012 in EUCERD Meeting, News

The European Union Committee of Experts on Rare Diseases (EUCERD met for the seventh time on 31 January 2013 – 1 February 2013.

The meeting saw the unanimous adoption of the EUCERD Recommendations on European Reference Networks for Rare Diseases, which will feed into the work of the Cross-Border Healthcare Expert Group, currently in the process of aiding the European Commission implement the Cross-Border Healthcare Directive, adopted on 28 February 2011.

One of the main topics of discussion was the area of registries in the field of rare diseases. Members had a chance to discuss the first draft of the EUCERD Recommendations on Common Principles and Consensus on Patient Registries and Data Collection for Rare Diseases which will be further refined and discussed at a workshop foreseen in the scope of the EPIRARE /EUCERD Joint Action workshop on 22-23 April 2013 in Brussels. This workshop will also be aimed at working on a technical document of possible policy scenarios for rare disease patient registration concerning the direction the proposed European registry platform to be based at the EC’s Joint Research Centre in Ispra could take. Members were also provided with an update on the EUCERD Joint Action activities after one year.

In particular an update was given on the activities in the field of the coding and classification of rare diseases, the work being carried out on specialised social services, and work on the elaboration of an EUCERD Recommendation on Indicators for National Plans/Strategies for Rare Diseases.

Other topics discussed included newborn screening, the outcomes of an expert workshop held at the Joint Research Centre in Ispra with the collaboration of Eurogentest and the EUCERD on the genetic testing offer in Europe, and progress in the Member States to elaborate, adopt and implement rare disease plans/strategies before the end of 2013.

The public executive summary of the 7th meeting of the European Union Committee of Experts on Rare Diseases, held on 31 January 2013 – 1 February 2013 is now available online (Read the summary)


New recommendation for a CAVOMP Information Flow

October 10, 2012 in News, Orphan medicinal products, Recommendation

EUCERD’s recommendation for improving the assessment of the Clinical Added Value of Orphan Medicinal Products encourages the creation of an Information Flow

Equitable and timely access to market-authorised orphan medicinal products is an ongoing issue for thousands of rare disease patients and their families across the European Union (EU). Large disparities in access exist between and even within the European Member State countries. Increasing cooperation between EU-level authorities and the Member States (MS) has been identified as a means of improving access to Orphan Medicinal Products (OMP) and reducing inequities. Global auditing firm Ernst & Young was mandated by the European Commission to identify and assess the possible options for the creation of a mechanism for the exchange of knowledge between the MS and European authorities on the scientific assessment of the relative effectiveness of orphan medicines and issued a reportin December 2011. The European Commission consequently asked the European Union Committee of Experts of Rare Diseases (EUCERD) to make recommendations in the area of facilitating the exchange of scientific information on orphan medicinal products that would support the clinical assessment processes undertaken by the MS.
No need to reinvent the wheel…
The EUCERD recommendation involves the creation of an Information Flow between individual MS and between MS and the EU that would bridge existing knowledge gaps, especially at the time of marketing authorisation. This flow would fit into existing regulatory, clinical, Health Technology Assessment (HTA), pricing and reimbursement processes. The recommendation thus capitalises on existing mechanisms, procedures, and regulatory frameworks, and does not bring new obstacles to the assessment process. The EUCERD Recommendation emphasises the continuum of evidence generation that takes place during the life cycle of an individual OMP and the Information Flow would be harboured by the corresponding institution responsible during each particular point in time (see the diagram).
The EUCERD Recommendation Improving Informed Decisions Based on the Clinical Added Value of Orphan Medicinal Products (CAVOMP) Information Flow will ultimately accelerate access to approved orphan medicinal products, while encouraging pricing and reimbursement decisions based on the value of the OMP and promoting good medical practices throughout the EU. The Recommendation has been submitted to the European Commission.

5th Meeting of the EUCERD

August 6, 2012 in EUCERD Meeting, News

The fifth meeting of the European Union Committee of Experts on Rare Diseases (EUCERD) took place in Luxembourg from 20-21 June 2012 with many important issues on the agenda. The Clinical Added Value of Orphan Medicinal Products – Information Flow (CAVOMP-IF) took centre stage with unanimous agreement to adopt the EUCERD recommendations by written procedure following minor modifications that arose from discussion during the meeting. This latest EUCERD Recommendation is the result of several years of effort and input from key stakeholders from the EUCERD, the European Medicines Agency, the European Commission and the EUnetHTA. The CAVOMP Information Flow is a process for the exchange of knowledge between Member States and European Authorities to help improve informed decisions based on the clinical added value of orphan medicinal products. The EUCERD recommendation highlights the fact that the life cycle of an orphan medicinal product is a continuum of evidence generation, necessary to assessors and decision makers, as well as being necessary to improve the good use of medicines. Other important issues were addressed, including the Cross-Border Healthcare directive and the European Reference Networks (ERNs). The EUCERD is developing recommendations for the ERNs, to be finalised later this year. Other key topics included the EUCERD Joint Action work plan, an update of the situation for rare diseases in international nomenclatures, the revision of the World Health Organization’s International Classification of Diseases, and the upcoming workshop of cross-referencing terminologies. There was also discussion on the potential for sharing knowledge on newborn screening practices, the evolution of the International Rare Disease Research Consortium (IRDiRC), and the European Commission’s Health for Growth Programme (2014-2020). A report from the EUCERD’s fifth meeting will be forthcoming.

The Executive Summary is now available online.