Gil Van Bokkelen, Athersys CEO, and Micheal Werner, Alliance for Regenerative Medicine Co-founder and Executive Director, explained the requirements for the delicate interphase between regulatory front and business reimbursement for a stem cell therapy company.
Regulatory process complexity versus reimbursement presents a major challenge: these business components should operate in synch but in reality they very rarely do.
In 1992, the FDA Accelerated Approval Framework regulated the filling of unmet clinical need based on a surrogate end point for defined conditions. Last summer the application of the framework was broaden to the so-called breakthrough therapies (which are designed for the treatment of life threatening conditions) and the time for the FDASIA to review a therapy application was cut down to 60 days. This impressive progress now allows those early stage therapies whose rationale is strongly data driven to significantly develop forward and to provide the regenerative medicine community with critical results in a totally novel way than before.
This leads to shorter, smaller effective clinical trials and allows to save millions of dollars for the overall process, included the follow-ups. The ultimate impact is an efficient mechanism to work with the FDA and other regulatory bodies: but what would happen if we make more efficient therapies that go quicker into clinics if on the other hand we cannot be paid back at the same speed? In other words, how do we tie payment of the delivered product to accelerated approval?
The reimbursement principles are the same but the solution strategy is different. Regenerative medicine products have unique challenges due to novelty of the field, therefore the reimbursement plan needs to be planned accordingly and most importantly in advance or at least by Phase II.
Before presenting the reimbursement plan to potential investors, a provident CEO should tick all the boxes of the "reimbursement checklist" such as:
- Address the issues before Phase II (definitely before Phase III)
- Gather health economics and comparative clinical effectiveness data (NB: going back to research won't be reimbursed!!)
- Conduct a meeting with CMS/NHS or equivalent, private insurers, or both
- COMPETITIVNESS: why is our product worth paying for?
- Build relationships with physicians and patients advocacy groups
Start planning early enough and identifying gaps as well as investors interests are the key to secure soon incomes and at the same time shape the costs curve in the right direction for a company. Once again, only if the regulatory bodies, the manufacturers and the final users work as a community the outcome can be of shared benefit.
Guest blog provided by Giulia Detela, UCL