Eurordis - Rare Disease Europe

Therapeutic Action Group (TAG)

The Therapeutic Action Group (TAG) was created in January 2009 and is composed of EURORDIS volunteers in the scientific committees and working party at the European Medicines Agency (EMA). These committees include the Committee for Orphan Medicinal Products (COMP), the Paediatric Committee (PDCO), the Committee for Advanced Therapies (CAT) and the Patients’ and Consumers’ Working Party (PCWP)

These patients’ representatives dedicate their time, experience and expertise to the task of evaluating dossiers of medicinal products requesting orphan designation, evaluation for use in paediatric patients, evaluation of advanced therapies, as well as ensuring accurate, transparent and available information to patients on authorised medicinal products.

A monthly report of activities of EMA Committees and EURORDIS patients’ representatives in these Committees is produced and disseminated to all members of the TAG, EURORDIS Task Forces, the EURORDIS Open Academy alumni and the EURORDIS Board.

Social Policy Action Group (SPAG)

The Social Policy Action Group (SPAG) supports EURORDIS’ work to shape policies and practices that improve the access of people living with a rare disease and their families to holistic care, and the guarantee of their social and human rights.

The SPAG is composed of 20 volunteers, representing 13 European countries and a diversity of rare diseases, disabilities, ages, and backgrounds. Its members are individuals living with a rare disease and family members, some of whom are also experts in social policy and human rights.

Why is the SPAG important?

Most people who live with a rare disease face serious everyday challenges and many societal barriers, while also living with visible or invisible disabilities. Such barriers prevent people living with a rare disease and their families, often the main carers, from living more independently and from fully participating in various areas of society, while also exposing them to a high level of psychological, social and economic vulnerability.

The SPAG supports EURORDIS’ work to change that and to progress towards a world where all people living with a rare disease can achieve their full potential, in a society that leaves no one behind.

Find out more about the SPAG in its Terms of Reference, published within the EURORDIS By-Laws.

SPAG members

The mandate of these SPAG members is of 3 years, from January 2024 to December 2026.

DITA Task Force

The Drug Information, Transparency and Access (DITA) Task Force closely follows the work done by patients and consumers at the European Medicines Agency (EMA) and in the European Network of HTA agencies (EUnetHTA) in the areas of product information, transparency of the regulatory process and access to medicines.

There are 18 volunteer members of DITA, from EURORDIS member patient organisations, led and supported by EURORDIS staff members Francois Houÿez, Director of Treatment Information and Access, Health Policy Advisor and Anne-Mary Bodin, Operations Assistant.

DITA volunteers bring invaluable knowledge of their own rare disease and the national health system. Many are patients themselves living with a rare disease. DITA works and gives input into several EU projects that EURORDIS is involved in and that concern the rare disease patient community. The task force meets twice yearly with regular telephone conferences and email correspondence to maintain the workflow.

Newborn Screening Working Group (NBS-WG)

Newborn screening is the process of systematically testing newborns just after birth for certain treatable diseases. Ideally, this practice is part of a larger programme that includes confirmatory diagnosis, immediate care, treatment and follow-up. The EURORDIS Newborn Screening Working Group (NBS-WG) was set up to review current policy and practice in the field of NBS, in order to develop principles for harmonious uptake/adoption of the NBS programs across the Member States with a view to delivering maximum benefit and improving outcomes for babies born with rare diseases.

Members of NBS-WG include representatives from patient organizations, international screening societies and international and national federations with a focus on NBS.

In January 2021, EURORDIS, alongside its Council of National Alliances, Council of European Federations and its members, set out 11 Key Principles to support a harmonised European approach to Newborn Screening.

The EURORDIS Health Technology Assessment (HTA) Task Force is a group of 12 persons who live with a rare disease or are parents of child with a rare disease.

They are EURORDIS’ members experienced and/or trained at different extent in HTA, with various educational background and stories.

The focus of the Task Force is to discuss any aspect of the assessment and reimbursement of health technologies.

The existence of this Task Force ensures that EURORDIS’ position on any HTA-related matter is built on the knowledge and the input of patients. Moreover, its members are engaged in advocating for a scientific, inclusive, and European-shaped HTA, as well as for the legitimate role of patients in this exercise and in the consequent decision-making.

They act under the rules of the EURORDIS Chart of Volunteers

The Task Force could also welcome external experts as guests if and when that can benefit the work of the group.

Why it is important for patients

How do healthcare systems decide what are the best treatments and the ones it’s worth to pay for?

Health Technology Assessment (HTA) is the process by which national authorities evaluate the available healthcare options. They look at medical, social, economic and ethical factors to determine healthcare practices at a policy level.

The EURORDIS HTA Task Force is meant to focus on any aspect of HTA that is of interest for patients, experts, and health authorities, at national/local level as well as at the European level.

What work does the task force do

The mission of the Task Force includes informing EURORDIS about how health technologies are assessed, which one are reimbursed, how patients are engaged and how decisions are made. Sharing views on the future of HTA at the European level and interact with experts is also part of their mandate.

The Task Force may raise and discuss specific cases about reimbursement and access of medicines or medical devices, reply to public consultations, attend conferences, and advocate for legislative initiatives about HTA and the engagement of patients.

The knowledge shared and acquired by the Group is also meant to be shared in liaison with the Task Force member’s communities and networks, so as to raise awareness regarding HTA.

Digital and Data Advisory Group (DAG)

Recognizing the importance and potential impact on rare disease patients of digital technologies, patient data usage as well as artificial intelligence, EURORDIS identified the necessity of bringing rare disease patients’ voices into these fields in order to help shape better tailored, ethical and sustainable solutions.

The Digital and Data Advisory Group (DAG) will pursue all aspects regarding digital policies and procedures and will advise EURORDIS in matters that range from situation assessment to strategic decision making.

The DAG is comprised of 11 volunteer patient advocates nominated for a term of 3 years.