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Agenda | Day 3 Printer friendly version - full agenda
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DAY 3 | Wednesday, January 26th 2011
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Morning plenary session
Who's next to market? Evaluating the prospects of cell & gene therapies in pivotal trials
How are both lessons from the past, and cutting edge technologies, informing clinical development and regulatory strategies?
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- Case studies: Clinical development updates on leading gene therapy, stem cell therapy and tissue engineered product candidates in pivotal trials
- What is the latest clinical data?
- What specific trial designs are being employed and why? What is the regulatory rationale? How have we engaged with the regulators?
- How are we tackling the increasingly acute challenge of patient recruitment? How to structure your clinical development program given the comparative lack of patients for orphan disease indications?
- How does our clinical development strategy integrate with our overall business/commercialization model?
- In the case of the stem cell therapies, specifically: How are the companies involved differentiating their cell platforms through manipulation/enhancement of the cells? What are the regulators asking for in terms of tracking data?
- Questions for the speakers, followed by industry/regulator/investor roundtable discussion
- What insights regarding the selection of pivotal trial endpoints can be drawn from past trials in both cell and gene therapy?
- What work can be done pre-phase III to inform the endpoint selection process?
- What are the keys to successfully undertaking a multinational clinical trial programme?
- What are the mechanics, step-by-step?
- What are the major regulatory/strategic considerations in running trials simultaneously in the US, Europe and other regions of the world?
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FOLLOWED BY YOUR CHOICE OF 3 PARALLEL BREAKOUT SESSIONS:
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Cell therapy road to commercialization stream
Focus session 1
Piecing together the technology jigsaw to enable the economically-feasible commercial scale-up of cell therapies
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- When should you delay clinical advancement in favour of further process/product development to improve the cost/performance aspect?
- How do you assess what your product price point might be, and then accurately estimate the improvements that might be made on the unit cost and product performance?
- What impact might the continued development have both on costs and on potential price?
- Cell therapy company case study: Assembling technology components into a platform - what were the decision-making processes and rationale behind our choices?
- Which specific components are required to balance the cost of goods/manufacturing vs. product performance equation, and how are we strategizing to optimally access and integrate them?
- How much to invest in optimizing the process with consideration to CoG?
- How to optimize strategic decision-making in terms of biomaterials sourcing?
- When/where/how should automation technology be applied to minimize costs and improve quality/regulatory compliance?
- Short presentation(s) followed by panel discussion: Addressing scale-up issues with tissue/cell therapy-device combination products
- When to engage a device specialist and when to develop your own? Examining the pros and cons of each approach, and the decision-making processes for companies that have chosen either pathway
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OR | Gene therapy R&D strategies stream
Focus session 2
Focus on the eye: Comparing and contrasting the scientific approaches and clinical data for innovative gene and RNAi therapies in the ophthalmology area
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- Case studies: Clinical development updates in the age related macular degeneration (AMD) area with varying gene and RNAi therapy technologies/methods of delivery
- Overcoming targeted delivery issues: What specific approach is each company employing and why?
- How do the clinical development strategies tie in with the company's overall business models?
- Questions for the speakers, followed by panel discussion
- Comparing and contrasting viral and non-viral delivery platforms for ophthalmology therapeutics - which are achieving the desired safety and efficacy profiles? Where do challenges remain?
- Examining clinical trial endpoints for gene and RNAi therapies in ophthalmology indications - are they good enough? What do these candidates really need to show in trials to ensure there is going to be a viable product at the end?
- What is the ideal combination of delivery device and vector for ophthalmology indications?
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OR | Workshop
(Highly interactive session for a maximum of 25 participants)
In-depth regulatory workshop
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- Addressing specific issues arising from disharmonization between US and European regulations for cell & gene therapies
Close of the Phacilitate Cell & Gene Therapy Forum 2011 followed by buffet lunch in the exhibition area
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