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Register for EURORDIS Multi-Stakeholder Symposium on Improving Patients’ Access to Medicines

Dezember 2018

Participants at previous event

Few treatments are available for rare diseases. Even when a treatment does exist, patients are often unable to access it due to issues that create a deadlock at the national level.

Over the last two decades, EURORDIS and its member patient organisations have dedicated much of their advocacy efforts to improving patients’ access to medicines.

Thanks to advancements in science and a favourable legislative environment including the EU Regulation on orphan medicinal products, the number of medicines for rare diseases has increased substantially in recent years.

However, major difficulties in accessing approved treatments still exist and make clear the need for urgent action. We continue to witness cases when patients and families cannot access a medicine, even though the very same medicine is available in a neighbouring country.

“If a medicine is developed and approved for market but does not reach those who need it, it has failed in its primary purpose. People living with a rare disease must be able to hope for new therapies and to benefit from them rapidly and fully, all across Europe, as soon as possible after they are approved for market”, stated Yann Le Cam, Chief Executive Officer, EURORDIS.

Register for EURORDIS symposium on improving access

The 3rd EURORDIS Multi-Stakeholder Symposium on Improving Patients’ Access to Rare Disease Therapies will take place 13 – 14 February 2019 in Brussels.

The event brings together all stakeholders that play a role in ensuring patients can access medicines – patient advocates, payers, health technology assessment bodies, national competent authorities, policy makers, regulators, clinicians, academics, and pharmaceutical and biotech industry leaders.

Register for the symposium to take part in discussions on tangible next steps that can be taken to improve access and to learn more about actions planned ahead of the European elections in May 2019.

Views expressed by participants will go towards producing a roadmap that will offer implementable solutions and set out the commitment of all players working together towards the goal of guaranteeing timely and universal access to rare disease medicines. This roadmap will be broadly disseminated to European and national institutions ahead of the 2019 European parliamentary elections. Read the event concept paper.

EURORDIS position on access

Earlier this year, EURORDIS launched a position paper on how to improve patients’ access to rare disease medicines (available in English and Spanish), which dispels misconceptions about the price of orphan medicines and sets out a four-pillar approach to improving access:

  1. A new blueprint to cut costs and fast track R&D (read more);
  2. Early dialogue and cooperation between healthcare systems on the determination of value of a medicine and on patient access (read more);
  3. A transparent European cooperation framework between national healthcare systems for the determination of fair prices and of sustainable healthcare budget impacts (read more); and
  4. A continuum approach to evidence generation linked to healthcare budget spending (read more).

Yann Le Cam, Chief Executive Officer, EURORDIS, said, “Patients around Europe are tired of living the consequences of a fragmented system that exists across 28 separate markets, and which is not conducive to effective access to medicines. All stakeholders have an urgent collective responsibility to shape a new approach that will accelerate the translation of major scientific advancements into new therapies.”

Simone Boselli, Public Affairs Director, EURORDIS, commented, “We believe that a new ecosystem is possible, a framework based on a global approach to innovation for unmet medical needs and on sustainability for healthcare systems, as well as financial attractiveness to industry and investors. We do not believe that this is a “futuristic” dream: a structured approach can be built with practical solutions and methods that have already been put in practice at varying levels and on different scales. Our objective is to unlock the access deadlock so as to leave no one living with a rare disease behind. This 3rd symposium is a step in that direction.”

Join Rare Barometer Voices to share your views on access

EURORDIS will launch a new Rare Barometer survey on access in early 2019.

Rare disease patients, family members and carers can join the over 8,000 Voices that have already signed up to share their views on the issues that matter most to the rare disease community.

Sign up to Rare Barometer Voices (available in 23 languages) and you will be sent the access survey as soon as it launches in 2019!

The results will be made available for your country and language for you to use when you advocate for better access to medicines for people in your country.

The European Commission is also conducting a public consultation for individuals and healthcare professionals on the EU Regulation on Orphan Medicinal Products ahead of a possible revision of the regulation. Read why EURORDIS thinks it is important to maintain this regulation and respond to the consultation by filling out the online questionnaire.  

Eva Bearryman, Communications Manager, EURORDIS