Home » Event agendas » Agenda – Cell & Gene Therapy » Day 2 – Cell & Gene Therapy


"Realising the promises of next generation therapies"

Day 2 - Friday 10th March 2023


  • EU legal requirement and regulatory framework for classification of advance therapies
  • Comparative analysis between EU and US guidelines
  • Classification Criteria in Europe and US
  • Nuances that may affect development


  • Challenges in quality and regulatory perspective
  • Main sources of quality issues, and solutions that have been developed?
  • Best practices for robust regulatory strategies
  • Harmonisation among the various regulatory agencies
  • Opportunities for businesses in EU to influence changes in EU law and policy that could make development and commercialization of transformative new treatment less costly.
  • Policy, regulations, and legislative changes to help overcome barriers to more widespread adaptation of CGTs towards its full potential and industries steady progress
  • Ethical issues involved in current gene therapy research and problems inherent to future gene therapies
  • Moral responsibilities of those involve in the development and use of CGTs
  • How to recognize individual’s right to choose to accept or reject testing or treatment?
  • Possible dilemmas involved in the use of genetic science in ways that go beyond prevention and cure of disease
  • Economic impact associated with advance therapies


  • Conducting early research to better understand patient and indications
  • Smarter clinical trial study designs that support implementation
  • Relieving barriers in manufacturing and supply chain
  • Accelerated pathways for the investigation and approvals of CGTs
  • Strategies and tactical approaches that companies can undertake to aid in achieving consistent and appropriate reimbursement levels
  • How can CGT developers expand capabilities for broader adaptation?


  • Challenges and obstacles in bringing this highly complex therapies to market
  • Stakeholder collaboration on the road towards a solution
  • Lessons learned and applications for new entrants
  • Strategies that can be taken to improve timely market access and access for patients 
  • Can pharma companies demonstrate the cost-effectiveness of this therapies?
  • What are the innovative payment plans between pharma and payers?
  • How to leverage, engage, and coordinate patient care to enable individual patient outcomes
  • Addressing the need for patient assistance programmes
  • Exploring alternative distribution capabilities for reimbursements
  • Changes in value assessment framework to improve adaptation
  • Encouraging payer decision-making while supporting patient access
  • How to ensure sustainability for healthcare system and patients
  • Innovative payment models to ensure timely access and alleviate the financial burden


  • Breakthroughs and innovations in research and development
  • Implementing next-generation technologies
  • Novel pricing and market access strategies pharma will employ to increase patient access
  • Various CGTs in the pipeline
  • What will the next decade hold for these life-changing therapies?


Wenliang  Dong, Chief Operating Officer, ORCA Therapeutics


Scroll to Top