Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Changing landscape in clinical drug development - New regulatory challenges in clinical trial designs

Elisabeth Kuhn (Abschlußjahr: 2020)

Summary
Language: English
Over the past, great advances have been made in genomics and computer analyses, which greatly improved our molecular understanding of tumors and other diseases. However, the molecular decryption of diseases renders the traditional clinical trial designs less practicable, as it would be impossible and unfeasible to investigate all these disease sub-types, mutations and combination of mutations by means of traditional clinical trial designs. Instead, new clinical trial designs are necessary that allow increasing the efficiency of clinical trials and answering multiple scientific questions within one clinical trial, instead of having to perform multiple clinical trials. One way to achieve this is via Complex Clinical Trial (CCT) designs.
After introducing the CCT concept and the different CCT designs (basket, umbrella and platform) and their characteristic features, the thesis presents and discusses the major points to consider when planning, conducting and reporting a CCT in the EU, such as the preparation, submission and approval of a Clinical Trial Application, safety reporting, amendments and results reporting, followed by a comprehensive list of key recommendations for sponsors of a CCT. In addition, possible impacts of the upcoming Clinical Trials Regulation on this process are discussed. The second part of the thesis summarizes and compares the current regulatory landscape for CCTs in the EU and the US, and the similarities and differences between both regions are shown. Furthermore, a systematic review of CCTs via a database analysis has been performed and results are described herein, including description of any detectable trends. At the end of the thesis, the opportunities and challenges of CCTs are summarized and critically discussed.
Overall, one can say that CCTs offer a variety of opportunities and represent a powerful tool to make the drug development process more efficient. This is also reflected in the growing number of CCTs and their increased use across multiple disease areas over the last years. But at the same time, CCTs come with challenges that should not be underestimated and need to be accounted for early on, rendering the planning of such clinical trials more complex, cumbersome and lengthy. But if sponsors are successful in overcoming these challenges, CCT designs can deliver on their promise to make the drug development process more efficient and bring new medicinal products to patients faster.
Pages: 56
Annexes: 0