*All times are shown in EST
COMMERCIALIZATION OF GENE THERAPIES FOR BLOOD DISORDERS
9:00 am Standardization to Create Stable Relationships Between Industry Developers & Hospitals
Synopsis
- Anticipating a surge in hospital demand and preparing to manage upcoming gene therapies which require certain degrees of resources and unique requests
- Standardization of blood collection processes, labelling, traceability systems, formatting for data printing, contracting processes and real-world evidence generation
- Encouraging conversation between standardization bodies and industry partners, and bringing in regulators to negotiate with hospitals to implement standardization where possible
9:20 am Differentiating Your Development Strategy for Gene Therapies
Synopsis
- Encouraging industry collaboration to share lessons learned
- Considering the impact of gene therapy landscape for your development strategy
- Collaboration to ensure support of clinicians and patients
- Differentiating against competitors
9:40 am Q&A Panel Discussion
10:10 am Morning Break
PRICING & REIMBURSEMENT: SETTING UP FOR THE PUSH TO MARKET
10:30 am Developing a Gene Therapy Payment Model & Pricing Strategy for Hemophilia when Alternative Treatments are Available
Synopsis
- Changing a system to allow for onetime payment that captures the value of the product
- Exploring creative payment models with payers and supplying compelling data to payers to prove long term benefit
- Discussing the commercial implications of having other treatments available
10:50 am Evaluating Different Payment Models for Different Blood Indications
Synopsis
- Explaining a payer’s perspective on pricing and reimbursement for gene therapies for blood disorders
- Discussing their requirements and views on innovative payment models
- Predicting state changes and national policy implications
- Highlighting the differences between sickle cell disease, beta-thalassemia and hemophilia
11:10 am Gene Therapy Precision Financing Models for Blood Conditions
Synopsis
- Addressing Value Demonstration Challenges for Gene Therapy for Blood Disorders
- Bringing a one-time potentially curative therapy to a chronic disease with history of limited treatment options has inherent value demonstration challenges
- Proper investment in real-world evidence to best characterize disease unmet needs is critical to ensure patient access and commercial success
- Novel payment models that are linked to credible estimates of product value can help overcome access hurdles
11:30 am Q&A Panel Discussion
12:00 pm Lunch Break
ADDRESSING RISK CONCERNS & LONG TERM SAFETY STRATEGIES
1:00 pm Antibody-Based Conditioning for Safer Hematopoietic Stem Cell Transplantation
Synopsis
- Exploring the current challenges of patient conditioning for ex vivo products
- Highlighting new research in antibody-based conditioning regimens for hematopoietic stem cell transplantation, including those for gene therapies
1:20 pm Developing a Long Term Follow Up Strategy for Genetic Therapies for Blood Disorders
Synopsis
- Considering the long term risks of gene therapies for blood disorders
- How do the risks differ by patient population and specific approach?
- Discussing the current FDA and EMA guidelines for long term follow up
1:40 pm Q&A Panel Discussion
2:00 pm Afternoon Break
NAVIGATING LOGISTICAL & MANUFACTURING CHALLENGES FOR DIFFERENT THERAPEUTICS
2:30 pm Ready for Launch? – Operationalizing your Supply Chain to Deliver Cell Therapies Commercially
Synopsis
• Supply Chain design and key considerations for treating 1000’s of patients
commercially
• The complexity and challenges of planning and operationalizing your supply
chain for commercial launch
• Preparing for what happens next after launching your product, and key
considerations for lifecycle management
2:50 pm Addressing Manufacturing Challenges for Sickle Cell & Beta Thalassemia
3:10 pm Q&A Panel Discussion
3:30 pm Chair’s Closing Remarks
