Abstract
If the disease is serious and there is no effective treatment option, delayed patient access to treatment leads to significant patient risk. Conditional approval regulatory frameworks have been established to accelerate the patient with serious disease and no effective therapeutic option access to innovative therapeutic options such as regenerative medical products, pharmaceuticals and medical devices. It is often difficult to conduct confirmatory clinical trials on a statistically reasonable scale for the severe disease with little effective therapeutic option. The point to be considered in the conditional approvals is how to conduct an approval condition-based post-marketing evaluation of safety and/or efficacy of the product and to ensure the product safety during the conditional approved period. However, regulatory concerns are unclear in the conditional regulatory frameworks. Here, we looked into dossiers of the conditional approved medical products. Pre-marketing clinical evidences appeared to be lower in conditional approvals than direct full approvals. In contrast, post-marketing requirements may be much more in conditional approvals than in direct full approvals. Together, the conditional approval regulatory framework may be a regulatory framework balancing between pre- and post-marketing regulatory burdens by approval requirements, leading to address a serious or life-threatening rare unmet medical need.
