Market Access Hurdles for Cell and Gene Therapies

The emergence and rise of gene and cell therapies has the potential to significantly change the way we manage and treat disease. While payers recognize that gene and cell therapies are likely to be the next big step in the evolution of medicine, the upfront costs of treatment and the uncertainty over long-term outcomes will present major pricing and market access hurdles. Individual therapies can be challenging for payers to fund but the potential for dozens of high cost cell and gene therapies would significantly impact already constrained budgets.

The lack of certainty between the data at launch and the ongoing clinical performance expectations top the list of clinical and reimbursement concerns for both HTA reviewers and the payers they influence. Short follow up time and low numbers of patients contribute to this uncertainty as do the nature of the single arm studies which tend to overestimate the benefits.

The NICE commissioned research into conducting assessments of regenerative medicines and cell therapies advocated the use of more flexible “spline-based” survival models rather than conventional parametric survival functions (e.g. exponential, Weibull, log-normal etc.).  They determined that these models were better able to approximate the hazard function across each of the various evidence sets.  Although these are yet to be used in practice, this shows that HTA bodies may be open to adapting their current methodologies for the unique challenges of cell and gene therapies.

The therapeutic impact over time leaves payers looking for innovative pricing and contracting agreements that mitigate their risks of these unknown benefits over time. Payers will look for agreements that protect their budgets against any patient relapse before the full value of the drug is realized. 

These new agreements could take the shape of pay-for-performance agreements, possibly tied to annuity-based payments. In these agreements, payers would monitor real world evidence for agreed outcomes and pay only for the therapy while it produces the expected effect. New stakeholders such as third-party lenders might be needed to cover the full upfront cost of the therapy, providing a benefit to the manufacturer who could then utilize the income immediately through reinvestment.

Assessing existing vs. expected data, monitoring and managing the therapeutic outcomes over time, and forging a pricing and contracting strategy that meets all stakeholder interests are needed if cell and gene therapies are to be adopted, funded, and utilized in today’s budget-constrained marketplace. Where there is a combination of great uncertainty but potentially very substantial patient benefits, innovative market access methodologies need to be developed to manage these challenges and facilitate timely patient access to these transformative therapies.

Key insights include:

• How payers are reacting to emerging cell and gene therapies
• How to address payers evidence concerns
• The types of agreements that payers will want in order to facilitate market access
• How market access solutions vary across countries

• Market Access
• HEOR
• Marketing
• Pricing & Contracting