Gene Therapy Management in Europe Versus the United States

The United States and Europe face many similar challenges in the management of gene therapies, including high upfront costs and the logistics of getting patients through complex care, according to Jeremy Schafer, PharmD, MBA, director of payer access solutions at Precision for Value in New York, and Alex Grosvenor, vice president of global pricing and product strategy at Precision Xtract in London, who compared notes on the handling of gene therapy in Europe and the United States during AMCP Annual Meeting 2018.

Since 2009, the European Medicines Agency has classified 57 products as gene therapy medicinal products, and three have been approved: alipogene tiparvovec for familial lipoprotein lipase deficiency, talimogene laherparepvec for unresectable melanoma, and Strimvelis for severe adenosine deaminase severe combined immunodeficiency (ADA-SCID). Approval in Europe, however, has no guarantee of a novel agent’s success, as these regimens still need to be evaluated for reimbursement in each market.

Route of administration can affect market access and funding pathways in Europe. The National Institute for Health and Care Excellence’s evaluation of Strimvelis shed some light on the complexities of incorporating these specialized treatments into existing funding mechanisms. Value demonstration in the European Union (EU) is based on incremental benefits and unmet need. France and Germany are the only EU5 markets that have evaluated alipogene tiparvovec, and neither recognized a long-term benefit with the therapy. Despite the curative potential of gene therapies, the evidence at launch may not support long-term value.

The United States faces many of the same challenges as Europe, including high upfront costs, an inability to calculate long-term cost-effectiveness, questions surrounding long-term durability, and the logistics of care. The speakers presented a Payer and Integrated Delivery Network (IDN) survey that included 25 responders from 20 health plans in five health systems. When asked what the biggest learning experiences have been in implementing gene therapies, responses included:

  • Thinking outside the box to manage costs
  • Regulations lag the drug market
  • Drug companies are not experienced in contracting
  • There is not much demand for these therapies yet
  • These agents were being lumped under transplant
  • The industry is not yet prepared to handle this treatment, both financially and operationally

Payer respondents’ top concerns with gene therapy include selecting the appropriate patients (35%), the potential need for retreatment (30%), member leaving the plan shortly after treatment (30%), and the potential ongoing need to treat the condition with other drugs (5%). Top concerns among all responders included a need for more information on durability of response (72%), the gene therapy pipeline (20%), long-term safety (4%), and retreatment guidance (4%).

To prepare for these therapies, payers and IDNs can begin experimenting with new payment models, pilot organizational goals with a long-term focus, and consider analogs outside traditional drug therapy to build new approaches. Meanwhile, manufacturers can assist by building a real-world evidence strategy, offering flexible payment options, providing education and resources, and helping to define the long-term value.

Presentation S5: From Europe to the U.S.: The Challenge and Potential of Gene Therapies. AMCP Annual Meeting 2018.