Time to review authorisation and funding for new cancer medicines in Europe? Inferences from the case study with olaratumab

The potential benefits of early patient access to new medicines in areas of high unmet medical need are recognised, but uncertainties concerning effectiveness, safety, and added value when new medicines are authorised and subsequently funded based on initial preliminary data only have important implications. In 2016, olaratumab received accelerated conditional approval from both the EMA and FDA for the treatment of soft tissue sarcoma based on the claims of a substantial reduction in the risk of death with an 11.8 month improvement in median overall survival in a phase II trial in combination with doxorubicin versus doxorubicin alone. The failure to confirm these benefits in the post authorisation pivotal trial has highlighted key concerns regarding early access and conditional approvals for new medicines. Concerns include potentially considerable opportunity clinical and economical costs, so that patients may have received suboptimal treatment and money spent has foregone the opportunity to improve access to effective treatments. As a result, it seems reasonable to reconsider current marketing authorisation models and approaches. Potential ways forward include closer collaboration between regulators, pharmaceutical companies and payers to enhance the generation of rapid and comparative confirmatory trials in a safe and fair way with minimal patient exposure to achieve robust evidence. Also, it may be time to review early access systems and to explore new avenues regarding who should pay or part pay for new treatments whilst information is being collected as part of any obligations for conditional marketing authorisation. Greater co-operation between countries regarding the collection of data in routine clinical care, and further research on post marketing data analysis and interpretation, may also contribute to improved appraisal and continued access to new innovative cancer treatments.