Day Two
Thursday, March 31 | 08.50 AM - 5.40 PM
DEMONSTRATING VALUE WITH GENE THERAPIES FOR BLOOD DISORDERS
8:50 am Chair’s Opening Remarks
9:00 am Value Demonstration Challenges for Gene Therapy for Blood Disorders: Lessons Learned & New Approaches
Synopsis
- Bringing a one-time potentially curative therapy to a chronic disease with history of limited treatment options has inherent value demonstration challenges
- Learnings to date from European HTAs indicate remaining high hurdles to convince payers of paying appropriately for gene therapies
- Proper investment in real-world evidence, and related communications of that evidence, to best characterize disease unmet needs is critical to ensure patient access and commercial success
9:30 am Value-Based Purchasing: Explosion or Fizzle from New MBP Rules?
Synopsis
- What the new CMS MBP rule will enable, and won’t
- Go it alone or together? Metrics and outcomes tracking
- How payment innovation can also generate evidence
10:00 am Real World Data & How Its Use Can Shape Our Long Term Understanding of Gene Therapy
Synopsis
- An overview of the utility of RWD to satisfy long term follow requirement in those
who receive gene therapy - How long term value can be demonstrated using RWD
- Approaches to engage patients beyond infusion
10:30 am Joint Q&A Session
11:00 am Morning Break & Structured Networking
COMMERCIALIZATION OF GENE THERAPY PRODUCTS IN THE BLOOD DISORDERS FIELD
12:00 pm Global Opportunities & Challenges for Gene Therapies & Gene Editing Modalities in the Blood Disorders Space: An ASC Therapeutics Case Study
Synopsis
- What are the current hurdles to further drug development in the Blood Disorders space at a global scale
- Development in the gene therapy and gene editing spaces
- Demonstrating value at a global scale
12:30 pm Delivering the Innovation of Gene Therapy to Patients – Considerations & Learnings in Commercial Launch Preparation
Synopsis
- Building a long term plan to approach launch in the right manner
- Lessons learned from our journey so far
1:00 pm Lunch Break
2:00 pm Launch of Gene Therapies in Europe: How Medical Affairs Teams are Connecting the Dots in Haemophilia
Synopsis
- Role of medical affairs team in the pre-launch, launch and lifecycle management of gene therapies in blood disorders
- How medical affairs brings value to patients, HCPs, healthcare systems and companies in Europe
- How European medical affairs teams collaborate with internal stakeholders (market access, clinical development, regulatory affairs, marketing, policy, patient engagement & other teams)
- How European medical affairs teams collaborate with external stakeholders (such as patient organisations, HCP organisations, local and international authorities
2:30 pm Joint Q&A Session
3:00 pm PANEL: Clinical, Approval & Commercialization Challenges in a World with Non-Gtx Competitors
Synopsis
- Demonstrating value to practitioners and physicians to recruit relevant patients for your clinical trials
- What does the approval process look like in a field with active approved competitor therapies
- Building an effective commercialisation strategy for your gene therapy where current alternative therapies already exist
- What can we learn from alternative approvals within the blood disorders field?
- Can approved alternative drugs be used to accelerate drug development in the
Gtx field for blood disorders?
3:30 pm Afternoon Break
4:00 pm Standards for Gene Therapy Including Cell Collection of Apheresis & Viral Vector Quantification
Synopsis
- Current standards for gene therapy including collection and viral vector quantification
- Resources for finding and implementing standards for gene therapy
- Standards for gene therapy under development and how to get involved
4:30 pm Knowing What You Don’t Know: Utilizing Medical Affairs to Facilitate Patient-Centric Drug Development
Synopsis
- Developing medical affairs teams to enter into new therapeutic areas
- Benefits of patient engagement early in the development process
- Bringing an epigenetic therapy into a gene therapy treatment landscape