Eurordis - Rare Disease Europe

Our work

EURORDIS advocates for change to improve both the development of medicines and the systems that obstruct patients’ access to rare disease medicines.

Our objectives are:

• Objective one: Improve patients’ access to orphan medicinal products and treatments for rare diseases through a sustainable development model.
• Objective two: Ensure legislation that affects treatment options for people living with a rare disease (the EU legislation on orphan medicinal products, EU cooperation on health technology assessment, and the EU pharmaceutical strategy to name a few) take into account the needs of the 30 million people living with a rare disease in Europe.
• Objective three: Engage rare disease patient advocates in the research and development of rare disease treatments as well as in regulatory, assessment and appraisal processes.

Advocate

Through interactions with policy makers, companies, regulators and payers, EURORDIS advocates for better access to rare disease treatments and also improved engagement of patients in the R&D process for rare disease treatments.

Our advocacy and the positions we take on important topics around rare disease treatments is bolstered by the views and experience of our member patient organisations, who collectively represent the 30 million people living with a rare disease in Europe. We collect their input through consultations (often held through webinars and events) or through Rare Barometer surveys, as can be seen in the results of the 2019 treatments survey.

EURORDIS has proposed a four-pillar approach to tackling the challenges that prevent patients’ access to medicines, including the ambition to have 3 to 5 times more new rare disease therapies approved per year, 3 to 5 times cheaper than today by 2025.

In cooperation with our member patient organisations and on behalf of the wider rare disease community, EURORDIS advocates for better access to treatments at several stages:

• Prior to market authorisation of the treatment

EURORDIS advocates for improved compassionate use programmes in EU Member States. Compassionate Use Programmes (CUP) are established to regulate access to medicines prior to their marketing authorisation, to help treat patients who have no other options and who do not have the time to wait for the end of clinical trials and the authorisation process. More information on how to access a CUP.

• At the stage of marketing authorisation (granted at the EU level)

EURORDIS is involved in initiatives to make the evaluation of medicines more efficient.

• Post-marketing authorisation of the treatment (at the stage when decisions are taken at the national level on pricing & reimbursement of the treatment)

EURORDIS has had a fundamental role in promoting dialogue between all major stakeholders involved in improving access to patients with particular focus on getting health technology assessment bodies and payers engaged in different platforms and mechanisms, such as the Mechanism of Coordinated Access to orphan medicinal products (MoCA). Read more about EURORDIS’ work to improve EU cooperation on health technology assessment.

EURORDIS represents the patient voice in conversations around improving access with companies that develop rare disease therapies through events held with the EURORDIS Round Table of Companies, as well as through the EURORDIS-organised biennial European Conference on Rare Diseases & Orphan Products.

Patient engagement in medicines R&D

EURORDIS participates in projects such as PARADIGM (Patients Active in Research and Dialogues for an Improved Generation of Medicines), through which a series of tools that all stakeholders involved in the development of medicines — including patient organisations —  can use to facilitate patient engagement in medicines R&D.

Improving access to gene and cell therapies

Gene and cell therapies offer ground-breaking new opportunities for patients with rare and complex diseases. As chair and participant in the RARE IMPACT consortium, EURORDIS has helped lead conversations with companies producing advanced therapy medicinal products (ATMPs), focused on how to improve patients’ access to these innovative and potentially live-saving products that are now reaching the market.

Repurposing of treatments

When appropriate, EURORDIS also supports the repurposing of medicines that are authorised for treatment of one disease so that it can be used to treat a rare disease. This can save on money and time usually needed for the lengthy development of a rare disease treatment.

Safety of treatments

EURORDIS believes in the importance of pharmacovigilance – the reporting of side effects and adverse events caused by a medicine so as to improve its safety in the future. We participated in the WEB-RADR project to create an app for this purpose.

Partner

It is vital that patients are engaged in the development of treatments for rare diseases. By providing feedback on their real-life needs throughout the life-cycle development of medicines, patients can play a role in ensuring the end product truly serves its purpose, to improve their health outputs and quality of life.

At the European Medicines Agency (EMA)

EURORDIS facilitates the participation of patient representatives in several committees and working parties at the EMA that play a role in the development of orphan medicines. Patients can be involved as full members (or alternates) of the Committees or as adhoc experts.:

• Committee for Orphan Medicinal Products (COMP)
• Paediatric Committee (PDCO)
• Committee for Advanced Therapies (CAT)
• Scientific Advice Working Party (SAWP)
• Committee for Medicinal Products for Human Use (CHMP)
• Pharmacovigilance Risk Assessment Committee (PRAC)
• Committee on Herbal Medicinal Products (HMPC)
• Patients’ and Consumers’ Working Party (PCWP)

EURORDIS also has several internal working groups that bring together patient representative volunteers from EURORDIS member organisations at the EMA:

• The Therapeutic Action Group (TAG) is composed of EURORDIS volunteers in the scientific committees and working parties at the EMA.
• The Drug Information, Transparency and Access (DITA) Task Force closely follows the work done by patients and consumers at the 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.

Empower

EURORDIS empowers patient advocates through the EURORDIS EUROCAB Programme, which helps patient advocates to set up and run community advisory boards in their disease area, which create a bridge between patient community and sponsor of the clinical research, to integrate the view of the patient into the research and development of a rare disease treatment.

EURORDIS also empowers rare disease patient advocates, many of whom go on to serve on the committees of the EMA responsible for developing treatments, through the EURORDIS Open Academy trainings. Patients gain the knowledge and skills they need to sit at the table in discussions with regulators and companies.