The translation of cell-based therapies:clinical landscape and manufacturing challenges

Cell-based therapies have the potential to make a large contribution toward currently unmet patient need and thus effective manufacture of these products is essential. Many challenges must be overcome before this can become a reality and a better definition of the manufacturing requirements for cell-based products must be obtained. The aim of this study is to inform industry and academia of current cell-based therapy clinical development and to identify gaps in their manufacturing requirements. A total of 1342 active cell-based therapy clinical trials have been identified and characterized based on cell type, target indication and trial phase. Multiple technologies have been assessed for the manufacture of these cell types in order to facilitate product translation and future process development

On the process capability of the solid free-form fabrication: a case study of scaffold moulds for tissue engineering.

This study applies the methodology and procedure of process capability to investigate a solid free-form fabrication technique as a manufacturing method to produce scaffold moulds for tissue engineering. The process capability Cpk and process performance Ppk of scaffold mould manufacture using a solid free-form fabrication technique has been analysed with respect to the dimension deviations. A solid free-form fabrication machine T66 was used to fabricate scaffold moulds in this study and is able to create features that ranged from 200 microm to 1000 microm. The analysis showed that the printing process under the normal cooling conditions of the printing chamber was in statistical control but gave low process capability indices, indicating that the process was 'inadequate' for production of 'dimension-consistent' scaffold moulds. The study demonstrates that, by lowering the temperature of the cooling conditions, the capability Cpk of the printing process can be improved (about threefold) sufficiently to ensure the consistent production of scaffold moulds with dimension characteristics within their specification limits

Achieving reimbursement for regenerative medicine products in the USA

Achieving reimbursement for regenerative medicine products is potentially a greater challenge than gaining US FDA approval, making it a decisive factor in the success or failure of small businesses. However, the mechanisms by which reimbursement is achieved are still seen as something of a ‘black box’, especially to those outside of the USA. This report aims to provide insights into the mechanisms of reimbursement and variety of payers in the USA, and to act as a starting point for a successful US reimbursement strategy. Fundamental concepts such as coverage, payment and coding are explained and linked with the factors that potentially determine the successful reimbursement of regenerative medicine products, including cost of goods and clinical study design. Finally, important considerations for the design of clinical studies that satisfy both the payers and the FDA are discussed and the key elements of a successful company strategy identified. </jats:p