Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies

• Date:
  Monday, 26 November 2018
• Location:
  European Medicines Agency, Amsterdam, the Netherlands

The aim of this workshop was to discuss scientific and regulatory approaches which could be used to facilitate development and preparation of robust quality data packages, to enable timely access to medicines for patients whilst assuring that patient safety and product quality are not compromised. 

Participants looked at specific industry case studies covering chemicals, biologicals and advanced therapies and experiences across different regions. 

EMA and the United States Food and Drug Administration (FDA) launched PRIME and Breakthrough Therapy schemes to strengthen their support to the development of medicines for unmet medical needs to help patients to benefit from these therapies as early as possible. This workshop is a joint collaboration between EMA and the FDA.

EMA has published a report on the workshop, containing recommendations from participants on next steps and areas to be further explored by EMA and the FDA. 

Documents

EMA–FDA joint Q&As on Quality and GMP aspects of PRIME/Breakthrough therapy applications

Reference Number: EMA/CHMP/531552/2023

Report: Workshop with stakeholders on support to quality development in early access approaches (i.e. PRIME, Breakthrough Therapies)

Reference Number: EMA/CHMP/BWP/812924/2018

Agenda - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies

Reference Number: EMA/493240/2018

Presentation - Perspective from European Union - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (V. Jekerle)

Presentation - Session 1 - Setting the scene - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (E. Alteri, V. Jekerle)

Presentation - Session 1 - FDA perspective - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (Ramesh K. Sood)

Presentation - Session 2 - Process validation and accelerated access - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (S. Barry)

Presentation - Session 2 - Innovative validation - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (S. Finn)

Presentation - Session 3.1 - Control strategy: Regulators perspective - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (M. Wellin)

Presentation - Session 3.2 - Control strategy: Industry perspective - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (D. Wilkinson)

Presentation - Session 3.3 - Control strategy: Industry perspective - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (C. Campa)

Presentation - Session 4.1 - Good manufacturing practice - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (G. Lorenti)

Presentation - Session 4.2 - Good manufacturing practice considerations for accelerated access - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (M. Popkin)

Presentation - Session 5.1 - Setting specification acceptance criteria - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (R. Keane, N. Frantz)

Presentation - Session 5.2 - Ebola case study- Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (T. Pepper)

Presentation - Session 5b - Case study on control strategy: impurity control strategy for an oncology drug - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (A. Teasdale)

Presentation - Session 6.1 - Risk-based assessment of comparability for a mAb undergoing accelerated development - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (A. Clinch)

Presentation - Session 6.2 - Advanced therapy medicinal products (ATMPs) comparability challenge case study - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (M. Jeschke)

Presentation - Session 6.3 - Advanced therapy medicinal products (ATMPs) comparability challenge case study - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (M. Alai-Safar)

Presentation - Session 6b - Stability approaches - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (R. Ogilvie)

Presentation - Session 7.1 - Predictive stability models to extrapolate shelf-life - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (A. Lennard)

Presentation - Session 9.1 - Regulatory tools - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (Y. Momonoi)

Presentation - Session 9.3 - Regulatory tools - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (C. Blanc, C. Bouygues)

Presentation - Session 9.4 - Regulatory tools - FDA - Stakeholder workshop on support to quality development in early access approaches, such as PRIME and Breakthrough Therapies (A. Byrnes)

Video recording