Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Biomarkers in oncological drug development: the potential and the challenges leading the way towards personalized medicine

Dr. Georgeta-Irina Sava-Piroddi (Abschlußjahr: 2017)

Summary
Language: English
In the last decade, high throughput biotechnologies experienced a fall in processing times and costs, but also an improvement in precision and accuracy of the results. These developments led to the identification of a large number of relevant molecular pathways and biological markers and to a better understanding numerous disease mechanisms. Especially in the therapeutic area of oncology, significant progresses have been made to understand tumor biology and to identify new biomarkers which can serve as potential drug targets for cancer treatment.
This master thesis aims to discuss the role played by the identification of biomarkers in the oncology drug development and the contributions of the main stakeholders involved in the advance of personalized medicine. Discovery, validation, qualification and use of biomarkers have an enormous potential to reshape drug development by identifying the patients who are most likely to respond to a certain therapy, by increasing the number of approved drugs with better patient outcomes and (in a best-case scenario) by accelerating the development time and decreasing the costs of new therapeutic compounds. The discovery and incorporation of "successful" biomarkers in the design of clinical trials plays a crucial role in shifting the "one drug fits all" paradigm towards the "right drug at the right dose and in the right patient population" approach. Nevertheless, validation of biomarkers requires new strategies during the clinical phase: the linear, conventional drug development has to be replaced with a more flexible approach, taking into account smaller patient populations and novel endpoints. The regulatory authorities have to permanently adapt to all the new knowledge in order to be able to offer scientific guidance and incentives to support the industry in placing new biomarker-based drugs on the market. The development of targeted therapies increased the need for collaboration between the pharmaceutical industry and the diagnostic companies in order to develop the corresponding companion diagnostic tools. Although regulatory authorities have recognized this trend and issued several guidelines to support and optimize the development of biomarker diagnostic tools, the lack of harmonization regarding co-approval of companion diagnostic tools and their reimbursement continue to pose challenges. Moreover, the costs of the innovative cancer therapies escalated in the last two decades, making the problem of the market access for new drugs and their reimbursement extremely challenging.
Finally, clinical translation of biomarkers in the pathology of lung cancer and available therapeutic options will be discussed in the last chapter of the thesis.
Pages: 45        
Annexes: -