Because gene therapies are high cost, a key challenge for manufacturers is to demonstrate value to payers in a health technology assessment (HTA) setting.

Gene therapies are potentially curative and are generally developed for diseases with devastating consequences, where there is no treatment or sub optimal treatment options. Because they are high cost, a key challenge for manufacturers is to demonstrate value to payers in a health technology assessment (HTA) setting.

For this session, we will look at the challenges and characteristics of products that have low frequency of administration, high up-front cost and value demonstration that occurs over a long-time horizon. We will discuss clinical trial design, evidence generation and the regulatory environment.

Through specific gene therapy examples we will look at how HTA agencies in France, Germany and the United Kingdom considered the disease being treated; how they evaluated the technology including clinical trial design, outcomes, health economics data and overall evidence package; and how they managed uncertainty. We also discuss the evolution of HTA evaluations over time as more products have come to market.

In addition, we will share ICON original research on payer perspectives on the assessment of our example products and their views on the changing landscape for advanced therapy medicinal products (ATMPs). Attendees will come away with a review of the HTA and the broader market access implications for gene therapies, including:
 

The unique characteristics of ATMPs; the potential value they offer and the challenges of demonstrating that value to payers

  • A review HTA processes for gene therapy products
  • A critique of the HTA review of selected gene therapy products
  • An understanding of payer perceptions and broader market access implications for gene therapy products
  • Insights on HTA, pricing and reimbursement for future gene therapies
     

Audience

This program is intended for professionals from pharmaceutical, biotech, and medical device companies involved in:

  • ATMPs
  • Pricing, reimbursement market access
  • Health economics and outcomes research
  • Commercial development
  • Medical affairs

Presenters

Caroline Delaitre-Bonnin, Pharm. D.

Senior Principal, Global Health Economics, ICON plc.

Caroline joined ICON in 2009 and has over 15 years of healthcare experience spanning industry and consulting. Her primary focus is on market access and pricing projects with extensive experience in oncology, haematology, gastroenterology and orphan drugs. She has been involved on projects in health policy, health technology assessment, public health programs assessment including disease management. Her activities include due diligences for mergers and acquisitions, licensing.

Bob Swann, MBA.

Senior Principal, Global Pricing and Market Access, ICON plc.

Bob has over 25 years of experience in market access consultancy and industry. At ICON is a project lead for global pricing and market access client engagements. His experience includes all access including pricing, reimbursement, HTA support, multiple therapy areas and product types including ATMPs.

Moderator

Patti Peeples, RPh, PhD.

CEO, HealthEconomics.Com

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