Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Making an old biologic great again - a regulatory survey about new indications for an old product and their requirements

Dr. Eva Skerhutt (Abschlußjahr: 2017)

Summary
Language: English
The development of new indications for already established and long-term known medicinal products is especially for medium-sized pharmaceutical entrepreneurs not daily business and may reveal some pitfalls that need to be solved.
"Making an old biologic great again" - the slogan adapted from recent politics stands in this survey for a lot more than the usual expected empty phrases in politics. A well-known and -tolerated, meanwhile very versatile biological substance should be further developed in terms of a new, completely different indication.
For answering the question of regulatory strategy and of course the profitability, this survey includes a situation appraisal, followed by decision analysis, analysis of potential problems, definition of preventive and corrective actions for the problems likely to occur and not at least an estimation about return on investment.
In order to answer the most urgent questions, the current situation of the authorised pharmaceutical product is analysed. This is done with a short introduction of the mechanism of action of the enzyme, further the background of the proposed new indication.
Further a description of the existing marketing authorisations for the medicinal product, followed by analysis of non-clinical and clinical data already available and evaluated by competent authority in Europe is given, focusing on reusability of the different studies and data. This survey then describes the manufacturing of the authorised product and the resulting quality thereof. Some obvious restrictions and last but not least the involved functions for this project within the company complete the situation appraisal.
For the decision analysis at first the decision to be taken is defined, followed by the definition, categorisation and weighting of relevant criteria. The afterwords listed regulatory alternatives are compared against the criteria. The individual requirements for each alternative are defined, as well as chances and risks for each alternative are presented. The most tentative choice is then given as a result of the comparison of alternatives against criteria.
With the essential definition of preventive and corrective action, the essential points for the different alternatives are considered. Including these preventive and corrective action a road map for the development of a new indication special for this old biological product is given.
Pages: 37, Annexes: pages: 14