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April 30 – May 2, 2018

Boston, MA
Earlybird Rate Closes Friday – Save $400

Day One
Tuesday, May 1, 2018

Day Two
Wednesday May 2, 2018

08.50
Chair’s Opening Remarks

Discussing Pricing, Reimbursement & Market Access Considerations

09.00
Establishing Value for Gene Therapy Products by Implementing Effective Reimbursement Strategies

  • Sven Kili VP, Head of Cell & Gene Therapy Development, GSK

Synopsis

  • Considering the patient and carers with broader implications, not just the therapy in reimbursement planning
  • Demonstrating durability through factors like long-term efficacy and safety, which are primary differentiators from current treatment options
  • Investigating new reimbursement models for gene therapy – strengths and weaknesses
  • Learning first hand from experiences in pricing Strimvelis in the European landscape and lessons that can be taken into the US environment

09.30
Evaluating Potential Pricing Strategies for Gene Therapies

Synopsis

  • Understanding the relative advantages of up-front payments versus performance over time
  • Striking the optimum balance between access and affordability
  • Investigating how to best compare the cost of treatment with current therapy
  • Evaluating what a patient costs to the health system and factoring this into pricing and value assessments

10.00
Panel Discussion: Global Commercialization Considerations

Synopsis

  • Evaluating the challenge of multiple healthcare systems globally – how can pricing and reimbursement structures adapt and change to suit each geo?
  • Investigating how follow-ups can adapt to handle the unexpected: patients who die of natural causes, move or change insurer
  • Learning from the reimbursement of cell and gene therapy products in the US and Europe to inform future strategies

10.30
Morning Refreshments & Networking

Enhancing the Efficacy & Safety of Gene Therapy Vectors

11.30
The NAV Platform of AAV Vectors for Gene Therapy: Current Applications & Beyond

Synopsis

  • Case studies illustrating clinical applications with the current vector technology platform
  • Critically evaluating current vector technology – what are the key limitations and where are the greatest opportunities for enhancement?
  • Understanding the opportunities enhanced vector technologies will open up

12.00
Establishing Robust Approaches to Quantify AAV Vectors and Build a Robust Path to the Clinic

Synopsis

  • Contrasting multiple approaches (qPCR, ddPCR, capsid ELISA) used to characterise and determine vector titer
  • Reflecting on the down-stream effects of robust vector characterization
  • Why is this so important? Understanding the influence of vector titering in deciding on initial clinical starting dose

12.30
Lunch & Networking

Putting the Patients First

13.30
The Patient Perspective – Understanding Common Misconceptions & Frequently Asked Questions About Gene Therapy

Synopsis

  • Understanding how patients really view the gene therapy space and how organizations can use this information to engage with them more effectively
  • Cutting through the hype – evaluating strategies to inform patients of the reality of inclusion in gene therapy trials
  • Discussing examples of patient misconceptions and questions about the field

14.00
Using Feedback from Patient Groups to Shape Gene Therapy Protocols

  • Sharon Hesterlee Research Project Lead, Gene Therapy, Bamboo Therapeutics, A Pfizer Company

Synopsis

  • Patients and patient organizations are a rich source of real world information
  • Patient preferences are increasingly recognized and valued by regulators
  • Gene therapy creates unique benefit/risk situations where patient preference information can inform protocol design

14.30
Gene Therapy Clinical Trials: Can They be Compelling for Patients/Families, Investigators and Health Authorities?

Synopsis

  • Understanding the key constituencies of the clinical equation at every level – how and why are gene therapy studies different?
  • Educating patients and families of the realities and risks associated with clinical trials
  • Addressing key health authority questions: are they compatible with relevant clinical programs in rare disorders?

15.00
Afternoon Refreshments & Networking

15.30
Advancing Gene Therapies to Patients by Forging Mutually Beneficial Alliances

Synopsis

  • Sharing how internal and external capabilities and expertise can contribute to effective clinical development in the gene therapy space
  • Identifying key companies and organizations to collaborate with to advance the development of gene therapies
  • How can organisations in the field collaborate more effectively to bring therapies to patients more rapidly?

16.00
Panel Discussion: The Investor’s Perspective – Evaluating How VCs View the Gene Therapy Field

Synopsis

  • Understanding the gene therapy investment landcape – what are the characteristics and considerations unique to this field?
  • To what extent are new investors looking to dilute out the founders?
  • How do VCs view indications with very small patient populations?
  • What are investors looking for in companies in this space?
  • How flexible are investors in defining investment opportunities?
  • Investigating the barriers to entry in the gene therapy field

16.30
Chair’s Closing Remarks