Business Trends Impacting the Health Care Marketplace

[B1] Managing Access for Gene Therapy: Learnings from an International Perspective

ACPE UAN: 0233-0000-19-017-L04-P | Contact Hours: 1.25 | Activity Type: Knowledge-based

Faculty(s)

• 

  Sophie Schmitz, BA, MA

  Managing Partner
  Partners4Access
  Hilversum, Noord-Holland, Netherlands

Moderator(s)

• KR

  Kellie Rademacher, PharmD

  Vice President, Access Experience Team
  Precision for Value
  Costa Mesa, California

Gene therapy is an innovative and complex technique that has the potential to improve the health of millions of people with potentially terminal diseases, such as cystic fibrosis, hemophilia, multiple sclerosis, sickle cell disease, and spinal muscular atrophy. According to analyst reports published this year, the global gene therapy market is estimated to generate anywhere between $2.3 billion and $4.4 billion by 2023-2024. While the United States is currently the highest contributor in the gene therapy market, Europe has gained a great deal of commercial experience and understanding into patient access issues since the first gene therapy launch in 2012. Some of the main commercial barriers to adoption of gene therapy in both Europe and the U.S. are high treatment costs and the uncertainty of evidence over the long-term benefits. As a result, key stakeholders in the U.S. are proposing and evaluating new financing and payment models for these therapies. Many of these have been tried and tested in Europe, with differing levels of success.

This session will provide an overview of key commercial learnings of gene therapy from an international perspective and highlight strategies that could be beneficial in the United States.

Learning Objectives:

At the completion of this activity, participants should be able to:

1. Explain the key financing and payment models used in gene therapy globally.
2. Describe the experiences of stakeholders in adopting financing and payment models for gene therapy.
3. Discuss proposed financial models for managing the drug spend associated with gene therapy in the United States.