Prospects for Harmonizing Regulatory Science Programs in Europe, Japan, and the United States to Advance Regenerative Medicine

Abstract

Just as consistent and predictable regulatory support founded on common principles in regulatory science provide the confidence and certainty required to bolster investment in regenerative medicine, harmonization is essential to building that framework on a global scale.

Evolution of Business Models in Regenerative Medicine: Effects of a Disruptive Innovation on the Innovation Ecosystem

“…Turning to the European setting, the firms reported that they get central regulatory approval from the European Medicines Agency (EMA), but they require nation-by-nation reimbursement, with each applying different criteria, making this expensive for under-resourced RM firms. However, in their discussion of regulatory systems in Europe, Japan, and the United States, Milne et al 15 reported that there are attempts at harmonization.…”

A decade of marketing approval of gene and cell-based therapies in the United States, European Union and Japan: An evaluation of regulatory decision-making

“…Parallel scientific advice meetings with multiple regulatory agencies could clarify which evidentiary requirements are needed for global registration strategies. Furthermore, regulatory agencies are holding joint meetings and other forums to harmonize regulatory strategies for GCT approval [43]. However, early access to GCTs is ultimately dependent on payment structures, such as national reimbursement schemes, rather than adaptive approaches to licensing.…”

“…Our results indicate that these advances will mostly take place within adaptive approaches to licensing, under regulatory standards of varying expedited and adaptive pathways [23]. Early access and long-term uncertainties urge authorities to cautiously consider and enforce appropriate post-marketing risk management and collection of confirmatory evidence.Thus, knowledge sharing between agencies and opportunity for parallel scientific advice need to be further strengthened to facilitate clinical development and suitable regulatory standards throughout the GCT life cycle [13,43].…”

Global Regulatory Differences for Gene‐ and Cell‐Based Therapies: Consequences and Implications for Patient Access and Therapeutic Innovation

“…New models of precompetitive collaboration can be utilized to increase R&D efficiency and innovation in the GCT field . At the same time, ongoing collaborations between regulatory agencies and interactions with developers need to be further strengthened . To facilitate global development by larger companies, existing opportunities for parallel scientific advice could be increasingly utilized and extended to other agencies.…”