Day Two

Thursday, March 31 | 08.50 AM - 5.40 PM


8:50 am Chair’s Opening Remarks

9:00 am Value Demonstration Challenges for Gene Therapy for Blood Disorders: Lessons Learned & New Approaches


  • 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?


  • 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


  • 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


12:00 pm Global Opportunities & Challenges for Gene Therapies & Gene Editing Modalities in the Blood Disorders Space: An ASC Therapeutics Case Study


  • 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

  • Antti Kourula Global Vice President Commercial President , CSL Behring


  • 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


  • 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

  • Karen Pinachyan Head of Medical Affairs Europe , CSL Behring
  • Adam Wufsus Medical Director Rare Blood Disorders, Novo Nordisk
  • Antti Kourula Global Vice President Commercial President , CSL Behring


  • 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

  • Dawn Henke Director, Standards Coordinating Body


  • 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

  • Adam Wufsus Medical Director Rare Blood Disorders, Novo Nordisk


  • 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

5:00 pm Joint Q&A

  • Dawn Henke Director, Standards Coordinating Body
  • Adam Wufsus Medical Director Rare Blood Disorders, Novo Nordisk

5:30 pm Chair’s Closing Remarks

5:40 pm End of Conference