PRMA Consulting Expert Advisory Group on Complex Pricing (EAGCP): Focus on gene therapies

A recent meeting of complex pricing experts focused on the challenges and solutions for pricing and reimbursement in the area of innovative cell and gene therapy products. The bespoke group shared knowledge from a range of countries and different roles within healthcare systems, to bring a practical and pragmatic approach to issues related to pricing.

Here are our highlights from the meeting:

  • With the launch of products such as Strimvelis, Kymriah, Yescarta, and
    Luxturna, gene therapies are rapidly becoming a reality for payers rather than a future concern.
  • The introduction of gene therapies presents particular challenges for pricing and reimbursement due to:
    • the high overall costs in a “single shot” of treatment, combined with
      uncertainty over long-term outcomes
    • concerns around the level of future demand for gene therapies and the sustainability issues this may present for health systems.

These issues suggest a need for the development of new reimbursement and funding models if gene therapies are to become more widely adopted in future. Various options have been explored:

  • in the EU, performance-based reimbursement and mechanisms to spread the cost of gene therapy over time
  •  in the US, financial innovation, including “mortgage” payments, reinsurance, and innovative third-party financing models.

Despite this dialogue, radical new payment models have not been adopted by payers for reimbursement of the first wave of gene therapies, and payers have largely continued with or adapted current pricing mechanisms, which may be combined with formal and informal access restrictions, to manage the entry of gene therapies.

This conservative approach appears sustainable in the short term, but as payers and policy makers gain experience of the implications for value benchmarks and sustainability, alternative approaches may come back into focus.

Manufacturers introducing products as part of the “second wave” of gene therapy development may need to propose innovative payment models. The highly specialist nature of some gene therapy treatments produce challenges for delivering access for patients. New regulations, infrastructure, and processes are required; specialized centers must be established, staff trained, and new data collection methods introduced. Unit manufacturing costs and the feasibility of scale economies are key considerations for commercial strategy and the pricing potential of gene therapies.

  • The commercial model for delivery of gene therapies should not be considered static. Disruptive approaches are being explored by both commercial and academic organizations and concerns over sustainability may compel policymakers to evaluate alternatives for IP protection and evidentiary requirements of regulatory and pharmacovigilance regimes.
  • The “one-shot” nature of treatment means that the potential for health tourism may become a more important consideration in pricing strategies, as the geographic footprint of gene therapy provision expands.

Our expert advisory group on complex pricing meets regularly to discuss topics that we identify or that are raised by our clients. This means that, as a client, you have a valuable opportunity at regular intervals to engage with and elicit feedback from experts on specific issues you face in complex pricing. In addition, we can provide bespoke projects, including primary and secondary research, advice, and support for strategy development. If you would like to discuss how the PRMA Consulting expert group on complex pricing could benefit your strategy, we would be happy to have a confidential conversation with you or your colleagues.


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