Conference Day Two

8:00 am Registration & Coffee

8:50 am Chair’s Opening Remarks

Exploring State and Federal Level Innovation

9:00 am A State Authority Perspective on Medicaid Reimbursement for Cell & Gene Therapies

  • Josh Moore Director Of Pharmacy - MO HealthNet, State of Missouri

Synopsis

  • Sharing experiences on negotiating payment terms with manufacturers
  • The new Medicaid Drug Rebate Program Final Rule & CMMI Access Models: how are these impacting CGT reimbursement at the state-level in 2025?
  • Discussing CGT patient data evidence generation efforts

9:30 am Roundtable Discussion: Driving Policy Reforms and Multistakeholder Actions to Progress the Field

  • Marianne Hamilton Lopez Senior Research Director, Biomedical Innovation Core Faculty Member, Duke Margolis Institute for Health Policy - Duke University

Synopsis

Roundtables include a larger focus on group discussion. A moderator will introduce the session topic to attendees with a presentation, before they split into groups to work through a series of topic areas. At the end, all groups report back on their discussions, and findings are collated.

This roundtable will cover:

  • Necessary steps for fair pricing mechanisms to both recoup manufacturing costs and reduce payer uncertainty
  • Developing robust data infrastructures to support patient outcome tracking
  • Suggesting policy reforms to improve equitable access across geographic regions and socioeconomic backgrounds

10:00 am Morning Break & Networking

Improving Value Demonstration & Evidence Generation

11:00 am Beyond the Price Tag: The Hidden Financial Dynamics of Cell and Gene Therapies Impacting Payers

Synopsis

  • Using Milliman’s proprietary closed claims dataset: the payer’s perspective on the patient journey associated with cell and gene therapy treatment
  • Exploring differences in treatment episodes and payer costs for ex vivo versus in vivo therapies and the potential impacts on the access ecosystem
  • Unpacking the distinct roles of primary and secondary/re-insurers related to treatment costs to drive understanding of their perspectives and financial exposure

11:30 am Hemgenix: Developing Methods to Model US Population Uptake

Synopsis

  • Cross-functional design of a long-term impact study on Hemgenix adoption in the US, and resulting budget impact forecasts
  • Fine-tuning the model to make it as predictive as possible
  • Leveraging data to help payers understand the economic benefits of the therapy

Developing Effective Payer Engagement Strategies

12:00 pm Panel Discussion: Refining Drug Manufacturer-Payer Engagement Strategies

  • Marianne Hamilton Lopez Senior Research Director, Biomedical Innovation Core Faculty Member, Duke Margolis Institute for Health Policy - Duke University
  • Chuka Ndukauba Director, Strategy & Innovation, Optum
  • Jodie Wehling Vice President - Market Access, Cargo Therapeutics
  • Christen Volk Head, National Accounts US Health Systems, Kite Pharma Inc
  • Kevin Brooks Senior Director, Market Access & Commercialization, Red Nucleus

Synopsis

  • Setting out a timeline through clinical development for touchpoints with payers
  • Practical advice to develop meaningful relationships with both large, national; and small, regional payers
  • Which areas of the CGT value demonstration story are often overlooked during payer interactions?

12:40 pm Lunch & Networking

1:40 pm Tailoring Value Propositions to Overcome Access Barriers in Cell & Gene Therapies

  • Joel Iff Executive Director - Global Access, Value & Evidence, Sarepta Therapeutics

Synopsis

Showcase how some Gene Therapies (GTs) are facing significant barriers to successful price and reimbursement

Understand key payer value barriers for GTs and how these vary by type of gene therapy, namely:

  • Durability of effect
  • Unmet need
  • Cost offsets

Discuss evidence generation strategies to mitigate each of these payer barriers and optimize payer value propositions for GTs

2:10 pm Navigating the Current Trends in Stop-Loss Insurance

Synopsis

  • Clarifying these policies: their goals, methods, benefits and drawbacks in providing access to CGTs, particularly for self-insured plan sponsors
  • How are reinsurance companies thinking about risk-based agreements for the success of these therapies?
  • Is there an opportunity for manufacturers to directly contract with them?
  • How can manufacturers work to ensure their therapies aren’t lasered out of plans?

2:40 pm Chair’s Closing Remarks

2:45 pm End of Conference