31st EURORDIS Round Table of Companies Workshop
Pharmaceutical Strategy for Europe: sustainable therapeutic development to leave no rare disease behind
22-23 March 2021
- Rare 2030 Recommendations
- European Commission Inception Impact Assessment Report
- EURORDIS position on Inception Impact Assessment
* This document is password protected.
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.
This workshop will provide valuable information to its participants with the following objectives:
- 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
- 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
Please send any questions concerning registration to Martina Bergna at: email@example.com
We look forward to welcoming you to the EURORDIS Round Table of Companies.