Event categories: EFPIA

Questions to be addressed include:

• What can the health care sector expect from the revamped orphan and pediatric rules ?
• Does the definition of rare disease still make sense?
• How can incentives be balanced with the need to create financially sustainable health systems in Europe? And how can incentives stimulate innovation in areas of real unmet needs?
• Do the current incentives match the difficulties companies face when developing orphan drugs?
• How can Europe achieve faster, more equitable access to orphan medicines across the bloc?

As all of these questions are high priority on the current France’s EU Council Presidency agenda, this event will take a critical look at the current model and deep dive into the most salient issues the revamp of pharmaceuticals rules will need to tackle and explore ways to overcome them.

Complex Clinical Trials (CCTs) have the potential to accelerate drug development and enable patients to get timely access to transformative therapies.

The EFPIA Pipeline Review has found that in 2020 alone, around 5,000 clinical trials were initiated to investigate and develop new therapies and vaccines. Almost 50% of therapies in development are new substances, with 40% of the pipeline targeting rare diseases, and groundbreaking cell and gene therapies growing in importance. This exciting new wave of medical innovation will play a key role in addressing the challenges faced by patients and healthcare systems. To coincide with the publication of EFPIA’s annual “Pipeline Review”, we are organising a high-level panel discussion around the latest medical research outcomes.