Conference Day One

Conference Day One

Wednesday 26th June 2024

7:50 am Check-In & Coffee

8:50 am Chair’s Opening Remarks

Dissecting the Global Cell & Gene Landscape by Understanding the Current State & Address Critical Challenges to Improve Global Patient Access

9:00 am Panel Discussion: Reviewing Global Market Access & Challenges to Streamline Ongoing Strategies & Improve Patient Access

  • Jeff Chaffin Market Access Strategy Leader, Novan Inc.
  • Carlos Martin Chief Commercial Officer & Head of International, Rocket Pharma
  • Jie Zhang Vice President, Global Value & Access, Cell & Gene, Novartis


  • What are the key challenges that are currently hindering effective pricing and reimbursement globally
  • What challenges have we overcome during the commercialization of currently approved cell and gene therapies?
  • What challenges will be a priority to tackle in the next 5 years?
  • What is the global state of cell and gene therapies?

9:45 am Discussing the Blueprint for a Successful Commercial Launch by Exploring Challenges & Uniting Industry, Government, & Payers to Address Challenges Together


  • Discussing unique payer challenges for cell and gene therapy: how do you prepare for sustainable commercialization of these promising therapies?
  • Key considerations for a successful commercial launch
  • Knowing your customer (industry, government, payers and finding a way together)

10:15 am Morning Break & Speed Networking


As this community unites for the first time, this session will provide valuable networking time with your peers, enabling you to forge new and lasting connections

Evaluating Value-Based Pricing to Amortize the Financial Burden & Support Manufacturer’s Profitability

11:15 am Integrating Single or Multiple Public & Private Payers in the Payment Policy to Enhance Broader Adoption


  • Reviewing payer response to providing access to cell and gene therapies
  • Exploring options to improve affordability
  • Stepping away from the “single site, single problem, single solution” system

11:45 am Fireside Chat: Discussing Outcome-Based Contracts to Prepare for Therapy Failures by Sharing Risk with Health Insurers

  • Kristin Wolff Vice President, Market Access, bluebird bio
  • Sumeet Panjabi Senior Director, Real World Value and Evidence, Janssen Pharmaceutical Companies of Johnson & Johnson


  • How to provide an alternative to buy-and-bill?
  • Exploring the variety of payers in the US landscape detailing the structures for government payers and commercial payers, and addressing the high variability of state government plans
  • How to go into pricing agreements with each health plan to guarantee the best price

12:30 pm Lunch & Networking Break

Tackling Health Economics & Real-World Evidence to Establish a Robust Delivery Model for Expediting Approvals of Transformative Therapies

1:30 pm Navigating the Need for Substantial Evidence Beyond Small, Initial Trial Populations or Limited Follow-Up Periods to Tackle Evidence Gaps & Maintaining Access to Patients

  • Sumeet Panjabi Senior Director, Real World Value and Evidence, Janssen Pharmaceutical Companies of Johnson & Johnson


  • The single trial: absence of comparative data sets to understand the added benefit vs standard of care
  • Beyond market access: maintaining access to patients, re-evaluations and reward evidence
  • Providing real-world evidence that is generalizable to the Medicare beneficiary population that are the intended recipients of the intervention
  • Tackling material gaps in the evidence base relative to the reasonable and necessary standard as defined required for Medicare beneficiaries

2:00 pm Panel Discussion: Analyzing the CMS Coverage with Evidence Development Model for Enhanced Access to Beneficiaries: A Real-World Evidence & Health Economics Perspective

  • Marianne Hamilton Lopez Senior Research Director, Biomedical Innovation, Duke-Margolis Center for Health Policy
  • Terry Cothran Senior Pharmacy Director, Oklahoma Health Care Authority
  • Mark Trusheim Strategic Director, NEWDIGS, Tufts Medical Center
  • Erica Cischke Vice President, Government Affairs, Alliance for Regenerative Medicine


  • Assessing the scientific merit and clinical validity of interventions or therapies within Medicare/Medicaid-covered populations
  • Exploring the reasonability and necessity of Medicare/Medicaid coverage for specific therapies
  • Examining coverage contingent upon randomized control trials or real-world evidence studies to validate pre-market evidence in the applicable Medicare beneficiary population
  • Aligning coverage with evidence development (CED) requirements and their relationship to outcomes expected in risksharing contracts
  • Strategizing the integration of CED studies with existing risk-sharing contracts for optimal real-world evidence and health economics impact

3:00 pm Afternoon Break & Networking

Navigating the Complexities of Real-Wold Evidence by Addressing Patient Enrollment & Data Collection for Increased Access

4:00 pm Gaining In-Depth Insights into the US Market Landscape to Uncover Hidden Challenges & Anticipate Future Benefits for Strategic Decision-Making

  • Laura Okpala Executive Director, US Reimbursement Policy, Gilead Sciences


  • How to tackle patient enrolment hurdles such as co-pay concerns or fear to enhance treatment onboarding?
  • Lessons learned about the barriers to utilization posing difficulties to broad access and utilization of cell and gene therapies as we prepare for more of these products to enter the market
  • Assessing the potential value-based agreements, such as CMMI’s cell and gene therapy model in supporting increased access

4:30 pm Addressing the Evolving Role of Real-World Evidence & its Impact on the Sustainability of the Cell & Gene Space for Long-Term Viability


  • How can real-world data support our understanding of durable benefits?
  • How to address the uncertainty around the durability of treatment and true clinical value of these therapies?
  • How to use the same data collection vehicle to also demonstrate benefit alongside safety?
  • Understanding which data is representative of benefit and how to collect it

5:00 pm Chair’s Closing Remarks

End of Conference Day One