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. How will payers assess such innovative therapies which still have to prove their duration of efficacy? How do manufacturers price a drug that has the potential to cure, or confer lifelong benefit from a single administration? How will already financially stretched healthcare authorities fund patient access to these therapies? 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.