Day 2
2nd CELL & GENE THERAPY WORLD CONGRESS 2024 EUROPE
"Driving Innovation, Addressing Significant Unmet Medical Needs, and Ultimately Improving Patient Outcomes"
Day 2 - Friday 4th October 2024
COLLABORATION AND PATIENT ACCESS
- The importance of stakeholder engagement: perspectives across different functions
- Creating a CGT platform: stakeholder engagement as a tool to support not only the development/commercialization of specific assets, but to build a sustainable platform
- How national level activities can impact on patient access to ATMPs
- Further national level collaboration between academic, clinical and industrial players is necessary to overcome the existing hindrances and boost further development of ATMPs in Europe.
Panellist:
Dr. Alexander Natz, Secretary General, EUCOPE
- To Follow
Marco Traub, CEO, The Trans European Stem Cell Therapy Society
- The significance of rationalizing clinical trials to reduce logistical and financial burdens for participants
- Leveraging technology to enhance access to treatment and specialist care
- Data-driven strategies to make informed decisions
- Patient-centred approach for better experience and outcomes
- Focusing on ensuring the equitable access to biologics and specifically RNA based drug products by everyone, everywhere.
Prof Harris Makatsoris, Professor of Manufacturing Systems, Department of Engineering, King’s College London
- Unique public-private partnership that has increased the access to new treatment options for patients
- Promote further innovation in clinical research using the strengths of the partnership
- Align potentially conflicting organizational cultures to maximize efficiencies and share project leadership
- Utilize a precision medicine master protocol to bring cutting-edge advances
- Allow for broader patient access and clinical trials that are more inclusive than standard industry-run trials
- Significant milestones in the development of gene therapy medicinal products that have facilitated the treatment of a significant number of rare diseases
- Bottlenecks and potential solutions that can improve access to gene therapy for rare diseases.
- Stakeholders engagement to bring about changes to improve access to gene therapy
MARKET ACCESS AND PRICING
- Circumventing operational complexities and regulatory barriers
- Consider near- and longer-term implications and opportunities to influence the design of the model
- Ability of the model to reduce costs and improve access
- Monitoring the development to better understand successful contracting arrangements
- Assess how potential therapeutic area expansion may impact pricing, reimbursement, and access for other gene therapy products.
- Past (learnings…)
- Benefit Structures / Cost / Contracting (e.g., Outcomes Based)
- Evidence & its Evaluation
- Special Considerations (e.g.,
– Dealing with surrogate endpoints
– Modifications & Flexibilities for Evaluation of Rare Diseases)
- Introduction of GCT Market Potential and Trends in Japan (commercial, regulatory and reimbursement)
- Opportunities in Japanese GCT market (political considerations and incentives)
- Challenges and Recommendations
Mohamed Oubihi, Founder and CEO, YAKUMED LIMITED
- Current landscape
- Existing innovative contracting with gene therapies
- How to overcome key challenges to innovative payment models.
- Leveraging real-world evidence to define outcome measures
- Implications to gene therapy manufacturers
- 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?