Conference Day Two: Thursday, March 28
7:30 am Registration, Coffee & Breakfast Briefings
8:20 am Chair’s Opening Remarks
PROGRESSING THE PRICING, REIMBURSEMENT & MARKET ACCESS LANDSCAPE
8:30 am Federal-Led Models for Lowering Costs & Improving Access to Gene Therapies (Virtual)
Synopsis
- Exploring key goals for the Center for Medicare and Medicaid Innovation’s (CMMI) ‘Cell & Gene Therapy Access’ model
- How are the CMMI approaching quality measurement for gene therapies, including patient-reported outcomes?
- Addressing the short- and long- term implications of these two models for gene therapy drug developers
- Considerations for what reimbursement pathway for gene therapies actually looks like
9:00 am Highlighting the Many Innovative Payment Tools to Enable Market Access
Synopsis
- Exploring the wide of variety of new payment models available
- Considerations for selecting a model for your unique condition and therapeutic
- Planning to overcome the legal and implementation barriers
9:30 am Outlining Success Stories in Value Demonstration to Private Insurers
Synopsis
- The Duchenne Muscular Dystrophy Story: Driving forward access of an approved gene therapy by working with patients and insurers
- Leveraging publications from healthcare professionals around value-based assessments and the therapeutic need
- Addressing the need for consistent messaging between stakeholders in one disease community
10:00 am Panel Discussion: Gene Therapy Health Plan Coverage & Reimbursement Challenges for Private Payers
Synopsis
- Understanding payer concerns around gene therapy coverage: why are some therapies deemed too high-risk to cover?
- Comparing current methods payers are introducing to help make value-based contracts work
- Exploring novel financing options to ensure these high-cost speciality drugs can be made accessible to those in need
10:30 am Morning Refreshments & Networking
MANUFACTURING
Benchmarking Innovation in Manufacturing
11:15 am Navigating Complexities of LV Vector from Clinical to Commercial Production: Lesson Learnings
Synopsis
- Learn about the journey from clinical manufacturing to process validation
- Importance of characterization and supportive studies through a case study
11:45 am HEK293 Suspension Based Production as a Scalable Platform for AAV Manufacturing
Synopsis
- Improvements in HEK293 productivity over the years
- Major cost drivers when scaling up to larger scales
- Opportunities for process intensification and reduced costs
12:15 pm Efficient Viral Vector Manufacturing with Microcarriers
Synopsis
- Understanding the viral vector manufacturing challenges often faced by drug developers
- Advantages of using microcarriers
- Upstream and downstream considerations for microcarriers cell culture system
CLINICAL
Ensuring Safety in Clinical Trial Design & Mitigating the Immune Response
11:15 am Monitoring Safety & Efficacy of Rare Gene Therapies During Clinical Development & in the Commercial Setting
Synopsis
- Reviewing the potential direct and indirect risks of genetic therapies
- Implementing safety monitoring before and after approval of bluebird’s gene therapies
- Long-term follow-up of patients receiving genetic therapies now and in the future
11:45 am IgG Cleaving Enzymes as a Potential Solution to the NAb Challenge
Synopsis
- An overview of IgG cleaving enzymes: their MOA and translatability for use in conjunction with gene therapies
- Discussing clinical data from non-gene therapy indications
- Considerations for treatment timing and dosing of gene therapy post-administration of enzyme
12:15 pm Leveraging the Latest Understanding of Viral Vector Immunogenicity to Better Inform Clinical Strategy
Synopsis
- Highlighting the latest understanding of viral vector immunogenicity seen in the clinical setting
- Outlining new trends and emerging standards in immune mitigation strategies
- Guidance for clinical assay selection to monitor the humoral and cellular immune response to a gene therapy
REGULATORY
Mapping Out Key Regulatory Touchpoints
11:15 am Maximizing the Benefits of a Type D Meeting
Synopsis
- Understanding the scope, associated timelines, and practicalities of FDA Type D meetings and any follow-up opportunities
- Reflections from engaging in a Type D meeting and advice for others seeking to engage
- How do these meetings fit into an overall development plan?
11:45 am Designing & Implementing a Robust Late-Clinical Stage Regulatory Action Plan
Synopsis
- Reflecting on lessons learned preparing for and undertaking a BLA/MAA filing
- Top tips for data demonstration earlier in clinical development to support a marketing application
- Facilitating smooth translation of your therapy into a commercial-ready product
12:15 pm Assessing In-Market Performance: Lessons Learned from an Approved Gene Therapy
Synopsis
- Presenting a case study on an in-market gene therapy: challenges and strategies in reaching the target patient population
- Lessons learned in improving gene therapy access to patients in a timely manner
- In the context of more gene therapies due to hit the market, how can industry ensure these are covered by insurers?
12:45 pm Lunch & Networking
Negotiating Manufacturing Challenges Under Compressed Timelines
1:45 pm The Evolution of Vector Production – Is Suspension Really the Solution?
Synopsis
- Assessing adherent and suspension process impact on critical quality attributes
- Leveraging innovation to drive increased performance of adherent process
- Economic evaluation of adherent and suspension processes
2:15 pm Panel Discussion: Establishing a Commercial-Ready CMC Checklist for Expedited Pathways
Synopsis
- Defining key sticking points to ensuring CMC and clinical development are aligned when a programme is under an expedited pathway
- Considerations for CMC-focused meetings with the regulators when moving through the clinic
- Laying out a commercial manufacturing plan upon receiving an expedited pathway designation
Clinical Endpoints for Rare Disease Studies
1:45 pm Outlining How to Establish & Run an Effective Natural History Study
Synopsis
- How to effectively incorporate multiple stakeholder perspectives when planning and designing natural history data collection efforts
- Challenges faced and solutions implemented in running a natural history study
- Top tips for maximizing the use of natural history data in clinical trial design and outcome assessments
2:15 pm Development of Primary Endpoints for Rare Gene Therapy Clinical Studies: The Lovo-Cel Case Study
Synopsis
- Reviewing the selection of endpoints for lovo-cel clinical studies
- Exploring the background and impact of changes made to endpoints during clinical development
- Future direction of clinical endpoints as the use of genetic therapies expands
Defining the Role of Companion Diagnostics
1:45 pm Strategies for Effective Companion Diagnostic Co-Development with Gene Therapies (Virtual)
Synopsis
- Key considerations for CDx assays used in Gene Therapy development
- Coordination of regulatory strategies and submissions for the drug and CDx
2:15 pm Regulatory Considerations for Gene Therapy-Specific Companion Diagnostic Devices
Synopsis
- FDA oversight of in vitro diagnostic devices, including companion diagnostics (CDx)
- Importance of Co-development of a CDx and planning for contemporaneous approval
- Overcoming challenges associated with CDx development
2:45 pm Afternoon Refreshments & Networking
BRIDGING THE GAP BETWEEN DRUG SPONSORS & PATIENT COMMUNITIES
3:30 pm First-Hand Experiences of the Clinical Trial Process
Synopsis
- Outlining the decision-making process: assessing risks, benefits, and drivers for commencing a clinical trial
- Reflecting on the burden of participating: which elements of the experience were de-stressed and where could improvements be made in the future?
- Assessing levels of communication from healthcare providers throughout
- How can drug developers help to empower patients to partner with them on drug development?
4:00 pm Panel Discussion: Evolving the Patient Engagement Paradigm in Rare Disease
Synopsis
- Outlining how all stakeholders stand to benefit from patient groups being involved throughout the drug development path
- How can drug developers ensure that the patient voice is never lost throughout the development process?
- Where in the drug development pathway are their further opportunities for drug developers to engage rare disease patient groups?
- Stories of successful patient engagement and potential areas for industry to improve on