Challenges in the evaluation of safety and efficacy of human tissue and cell based products.

Human tissue and cell-based therapeutic interventions provide efficacious alternative treatment where traditional drugs or medical devices have proved unsatisfactory. Yet human tissue and cell-based products (HTC/P) still present a series of challenges regarding their safety and efficacy. By reviewing the history of development and current regulatory standards and observing activities related to the manufacture of HTC/P, we have described the common characteristics of HTC/P, including the risk of pathogen transmission, transportation methods of human tissues, quality control in HTC/P manufacturing, and the concern of ancillary materials. The ethical issues of cadaveric donation and clinical trial standards for HTC/P have also been discussed. Overall, we have provided a general standard, which articulates the essential requirements on the safety and efficacy of HTC/P.

Transparency in drug regulation: public assessment reports in Europe and Australia.

Openness and transparency are important considerations for medicines regulators, where public health is of paramount concern. As part of their commitment to transparency, the European Medicines Agency (EMA) and Therapeutic Goods Administration (TGA) in Australia publish information relating to their evaluation of medicines via public assessment reports. European Public Assessment Reports (EPARs) and Australian Public Assessment Reports (AusPARs) provide information about the considerations that led the regulator to approve or refuse the application. The reports summarise assessments by each regulator of the information provided on the quality, safety, and efficacy of the medicine under evaluation. Here, we describe the experiences of two established medicines regulators in publishing public assessment reports, and reflect on their future role in communicating medicines information.