31st EURORDIS Round Table of Companies Workshop

Pharmaceutical Strategy for Europe: sustainable therapeutic development to leave no rare disease behind

ONLINE

22-23 March 2021 

12:00-19:00 CET 

 

Programme

Preparatory documents

 

See the Agenda

Presentations

This document is password protected. 

 

Workshop Description

In the context of the ongoing consultation on the new public health programme from the European Commission, this workshop will convene  patients, policy makers, regulators, industry and payers to reflect on and discuss ways in which Europe’s competitiveness and attractiveness can be enhanced and safeguarded. Exchanges will also focus on key elements of the EU Regulation on orphan medicinal products which can help to shape a sustainable ecosystem addressing rare disease patients’ unmet medical needs.

Taking stock of 20 years of therapeutic development in rare diseases and looking at the forecast if we keep operating in a static environment, we will look at the changes needed to fulfil patients’ unmet medical needs and to unlock the potential of research & development for currently disregarded diseases.

In particular, we will reflect on reframing the system of incentives within - and outside of - the EU regulation on orphan medicinal products, so that Europe provides an attractive environment to de-risk investment in medicines development. We will examine how multi-stakeholder collaboration can help to bridge public and private research and how important it is to ensure that an early dialogue with regulators, HTA bodies and payers is taking place.

Beyond the regulation, we will discuss which conditions are needed to ensure a sustainable therapeutic development and a coordinated and accurate funding agenda/investment strategy, which places an emphasis on pre-competitive knowledge generation and on the efforts required to reconcile development and access to medicines.

The discussions will also reflect on a common European approach that highlights the importance of continuous data generation to support health systems in providing equitable access to rare disease treatment and care.

In this workshop, we will call on decision makers at all levels and across stakeholder groups to commit to and develop the necessary framework and mechanisms to ensure people living with a rare disease in Europe ultimately benefit from the therapies that are developed.

Let us collectively recognise the need to implement new policies that are fit-for-purpose to actively design our preferred future for people living with a rare disease, and make Europe the next world leader in research and access for rare disease medicines.

 

Objectives

This workshop will provide valuable information to its participants with the following objectives:

Day 1:

  • Identify the changes needed in the rare disease R&D environment to fulfil unmet medical needs
  • Discuss how to reframe the system of incentives within - and outside of - the EU Regulation on OMP so that Europe provides an attractive environment to de-risk investment in medicines development
  • Explore how to unlock the potential of research and development for currently disregarded diseases and ensure sustainable therapeutic development so to reconcile development and access to medicines

Day 2:

  • Recognise the urgency and need to implement new policies that are fit-for-purpose to actively design our preferred future for people living with a rare disease
  • Understand the benefits of a common European approach to access rare disease treatments beyond the regulation 
  • Acknowledge the importance of continuous data generation to support health systems in providing equitable access to treatment and care

 

Registration

Registration for the 31st ERTC Workshop is now open!

ERTC members have received an email with the link to register.

If you are a patient, patient advocate or healthcare professional and you wish to take part, please contact Martina Bergna <martina.bergna@eurordis.org>

 

Questions

Please send any questions concerning registration to Martina Bergna at: martina.bergna@eurordis.org

We look forward to welcoming you to the EURORDIS Round Table of Companies.

 
 
La voz de los pacientes de enfermedades raras en EuropaEURORDIS La voz internacional de las personas que tienen enfermedades rarasRare Disease International Reuniendo a pacientes, familiares y expertos para compartir experiencias en un foro moderado en distintos idiomas. RareConnect El Programa Rare Barometer es una iniciativa de EURORDIS que realiza encuestas para transformar las experiencias de los pacientes con enfermedades raras en cifras que puedan compartirse con los responsables políticos.Rare Barometer Una campaña internacional de sensibilización que se celebra todos los años el último día de febrero, El Día de las Enfermedades Raras es una iniciativa de EURORDISRare Disease Day Únete al mayor grupo de partes interesadas en toda Europa, en la Conferencia Europea sobre Enfermedades Raras y Medicamentos Huérfanos que se celebra cada dos años. ECRD es una iniciativa de EURORDISEuropean Conference on Rare Diseases