Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

How to develop a fictitious combined ATMP regarding non-clinical and early clinical phase: A possible SME Scenario ***

Dr. Helene Kern (Abschlußjahr: 2015)

Summary
Language: English
The development of ATMP in the EU represents a critical challenge, the success of which, more than in case of conventional innovative medicinal products, depends on a meticulously thought-out study program and a farsighted regulatory strategy under continuous involvement of the EMA and other regulatory stakeholders. A recommendation for a universally valid non-clinical and clinical study program is not feasible for ATMP because of their high diversity and the risks, limitations and chances related thereto.
Such circumstances in connection with limited capacities within often small and medium-sized developing companies in terms of regulatory expertise and financial means regularly put ambitious development projects for novel treatments of life-threatening or debilitating diseases at risk of failure along the difficult road to authorised patient-access. 
This thesis intends to provide an overview of the current regulatory and legal circumstances pharmaceutical companies, particularly SME, are confronted with and can make use of during the development process of an ATMP. In a further step it outlines potential regulatory and scientific milestones of development of a fictitious ATMP, the product in question being a combined GTMP with an implantable capsule-like non-biodegradable drug delivery device incorporating genetically modified human allogeneic cells, which produce and secrete a therapeutic protein deficient in a rare eye disease.
The focus of the regulatory development is laid on SME-specific procedures in association with the chosen fictitious product, such as SME registration and incentives, the Innovation Task Force Briefing Meeting, product classification and certification, the collaboration with Notified bodies and respective guidance documents. Scientific development focusses on non-clinical and clinical aspects and the demonstration of the product’s components meeting the legally laid down essential requirements. Relevant guidelines in relation with characteristics of the product in terms of manufacturing, application and mechanism of action offer the framework for the suggestion of important investigations and points to consider in both study phases.
The thesis aims at offering a perspective of the challenges and chances that the development of a highly complex combined ATMP implies for an SME within the current dynamic regulatory landscape. Multidisciplinarity, flexibility and transparency in collaboration with regulatory stakeholders and intern experts are considered to be key for the efficient and successful development of a combined ATMP.
Pages: 51