Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Global regulatory dynamics in response to the challenges of innovation in gene and cell therapy products: An overview

Angela Zambrano (Abschlußjahr: 2018)

Summary
Language: English
The impact of globalization on health systems and the growing technological pressure for the entry of new therapies for medical unmet needs as the Advanced Therapy Medicinal Products (ATMPs), constitute a worldwide challenge for the health authorities, especially in countries with emerging economies. The National Regulatory Authorities (NRA) must guarantee access to innovative medicines for their citizens, with compliance of quality, efficacy and safety standards, without compromising the sustainability of the health systems. Further to the more evolved regulatory development from Japan, the United States and the European Union, there are advances in other countries of the Americas and Asia, which have relevant regulatory frameworks for those novelty products. Furthermore, given the speed of scientific advances in the research and development of ATMPs in recent times and having identified the barriers and challenges faced by regulators, industry and other actors involved in this process, several initiatives have been generated to take advantage of the synergy that entails the association of diverse stakeholders to expedite the clinical and commercial translation processes.
Therefore, the aim of this thesis is to perform a comparative analysis of the framework of the legislation of ATMPs in 4 countries: Canada, Brazil, Korea and Singapore; the regulatory dynamics on this topic is described and moreover, a compilation of existing collaborative initiatives among stakeholders is carried out, bearing in mind the point of view of the industry, academic system, patients advocacy groups and regulators, to strengthen the development of ATMPs on the stages of the product’s lifecycle.
Regulatory authorities around the world must make great efforts to guarantee that ATMPs met properly quality, safety and efficacy standards to be approved, protecting the public health of the population and responding to the progress of science. In jurisdictions that rely on stringent regulatory authorities and have a strong as well as consolidated regulation, there were few authorizations for ATMPs commercialization in the last decade. But, by means of scientific guidelines, the authorities give recommendations to developers about the regulation. It is so as a result of an intense work in updating/generating guidance documents during the last 2 years, and as part of a joint work with stakeholders, together with a more flexible regulatory model oriented towards the risk analysis approach on a case-by-case basis and the strengthening of post-marketing risk management beside simplifying access to expedited regulatory pathways, new ATMPs have been approved during this period. It is also worth noting that jurisdictions from countries with emerging economies are making progress in the generation or strengthening of regulatory initiatives focused on ATMPs, such as the development of specific frameworks or the implementation of early access schemes and other options for developers’ support.
Classification of those products and their legal definition, helps to delimit in each jurisdiction the scope of the regulation. Moreover, their classifications by risk approach include factors related with the degree of manipulation, the intended use, if produces local or systemic effect, the developer and the source of cells. ATMPs intended for nonhomologous use and with extensively manipulation require a NRA’s approval.
To strengthen the international cooperation initiatives that promote convergence and/or regulatory harmonization in ATMPs specific topics is necessary to build up the regulatory systems of the countries, considering the increase of new responsibilities faced by the NRA. Also, available interactions schemes between regulatory agencies with developers must be strengthened; furthermore, some existing regulatory procedures could be up-to-dated and extended to be put into action for other authorities.
It is expected that the increasing number of key partnerships will continue meanwhile the sector reaches a degree of maturity, the regulatory environment evolves, and a profitable technology translation from bench to bedside comes true, with support of dedicated public funding and regulatory authorities, and the integration of academic systems.
Pages: 72, Annexes: VI pages: 26