ABSTRACT
The conduct of conventional phase-based clinical trials to collect reliable and robust evidence could be more challenging in comparison with traditional drugs. Regenerative medicines represent significant promises for rare genetic disorders lacking alternative options. Undoubtedly, this leads to difficulties with regard to patient recruitment in relation to small patient populations, as well as the selection of appropriate comparators to make direct comparison. In contrast to the regulator’s enthusiasm to facilitate timely market approval based on limited evidence, health technology assessment (HTA) bodies hold more conservative attitudes towards regenerative medicines. HTA bodies concluded that the limited clinical evidence increased the uncertainties surrounding the curative potential, the magnitude and the durability of clinical benefits, and the potential unfavorable effects in the long run, which raised barriers for payers to reimburse RMs within tight budgets. The objective of this chapter is to investigate the HTA decisions of approved RMs made and further analyze the limitations of the clinical evidence for RMs that are criticized by the HTA bodies studied.
