Identifying Viable Regulatory and Innovation Pathways for Regenerative Medicine
Authors : J.Mittra, J.Tait1, M.Mastroeni, M.L.Turner, J.C.Mountford, K.Bruce
Abstract
The development of red blood cells for the blood transfusion market is a groundbreaking advancement in regenerative medicine. This article explores a research project that aims to produce red blood cells from human embryonic stem cells. The project faces significant challenges related to regulatory protocols and the manufacturing process, which must adhere to Good Manufacturing Practice (GMP) standards for clinical studies. The research analysis employs the innovative approach of the Analysis of Life Science Innovation Systems (ALSIS), which combines social and natural sciences to anticipate product development pathways.
The study highlights various issues encountered during the development process, particularly in cell manufacturing and scaling up, as influenced by regulatory requirements within the innovation ecosystem, such as preclinical testing and clinical trials. The article also discusses the endeavors of regulators to adapt the existing pharmaceutical-based regulatory model to accommodate allogeneic regenerative medicine products. Moreover, it emphasizes the broader implications of this case study for successful innovation and translation of regenerative medicine therapies. The role of methodological and regulatory innovation in future advancements in the field is also considered in the discussion.
Method
The method applied for this project, Analysis of Life Science Innovation Systems (ALSIS) [2] uses a strategic mapping approach to project future business models and product development pathways (defined as the full range of activities required to bring a product from conception to end use, including design, production, marketing, distribution and support to the final consumer). These factors, broadly discussed under the control of the innovator, are embedded within an innovation ecosystem that includes the economic, regulatory, societal and political contexts that are beyond the control of the innovator, with either positive or negative impacts on the product development business plan. For the Blood pharma project, critical decision points within the product development pathway arose from the scientific and technological challenges of differentiating sufficient quantities of RBCs from stem cells meeting clinical grade GMP standards for different stages of pre-clinical and clinical testing; and the implications for product development and regulatory science of targeting the niche Thalassaemia market. The main innovation ecosystem components discussed in this paper are the regulatory system and the challenge of meeting requirements related to the use of conventional preclinical animal models and to the conduct of human clinical trials.
Results
The sequence of decisions related to the scientific and technical challenges in developing the cultured product, showing the critical points in the overall pathway at which manufacturing and scale-up decisions would need to be finalised and where such decisions would interact with the regulatory system. The first concepts at the start of the critical path, obtaining a starting cell line and developing a working cell bank bring up the question of the expected starting material. In Between 2010-12 the project was using human embryonic stem cell (hESC) lines as the starting material, but the long-term plan was to use induced pluripotent stem cell (iPSC) lines. Factors favouring that choice included: the requirement for open-source hESC banking in the UK.
Conclusion
We found two vital observations from this research: (i) related to the innovative capacity of regenerative medicine therapies; and (ii) related to the potential role of interdisciplinary social science methods in supporting innovation processes.
Citation: J. Mittra , J. Tait , M. Mastroeni , M.L. Turner , J.C. Mountford and K. Bruce Identifying Viable Regulatory and Innovation Pathways for Regenerative Medicine doi:10.1016/j.nbt.2014.07.008
Copyrights: © 2014 the Authors. This article is available under the terms of the Creative Commons Attribution License (CC BY). You may distribute and copy the article, create extracts, abstracts, and other revised versions, adaptations or derivative works of or from an article (such as a translation), to include in a collective work (such as an anthology), to text or data mine the article, including for commercial purposes without permission from Elsevier. The original work must always be appropriately credited.
Received: August 2014 Accepted: January 2015 Published: January 2015
Competing interests
The authors declare that they have no competing interests.
Funding: Open Access funded by Economic and Social Research Council.
