Session Details

Co-Chairs:

• Miguel Forte, MD, PhD, mC4Tx, Belgium
• Paula Salmikangas, PhD, NDA, an SSI Strategy Co., Finland

Speakers:

• lona Reischl, PhD, Assessor, AGES MEA, Austria - Regulatory agencies and the development and authorization of Advanced Therapies in Europe
• Manel Juan, MD, PhD, Hospital Clinic Barcelona, Spain - ARI program: "driving" efficient treatments through challenges in academic/non-profit environments
• Douglas Lundin, PhD, The Swedish Dental and Pharmaceutical Benefits Agency, TLV, Sweden - Uncertainty in duration of treatment effect: How can we assess the degree of risk reduction a payment model offers?

How to overcome the challenges in academic/non-profit and biotech environments to enable efficient clinical translation and commercialization of ATMPs to ensure broad and sustainable patient access to novel therapies?. In this session, we will discover how early regulatory engagement with EMA can accelerate the successful ATMP development journey and gain insight on how the HTA bodies are considering and reacting to the assessment challenges posed by ATMPs. We will also hear a real-life example of an academic CAR-T development, highlighting how the hospital exemption pathway and proactive interactions with regulatory agencies have enhanced the patient access to advanced immunotherapy locally in Spain.

Session Objectives

1. How to overcome the challenges getting ATMPs approved in the EU and what are the hurdles that take products out of the market?
2. Understand how an ATMP, manufactured locally in an academic environment, can reach the patients.
3. Understand the challenges that ATMPs are introducing to HTAs and how they can be tackled.