Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Real World Evidence - Impact on Regulatory Decision Making ***

Dr. Elmar Wegener (Abschlußjahr: 2018)

Summary
Language: English
In recent years, the pressure in the healthcare systems of the majority of developed countries worldwide has significantly increased due to a variety of pharmacoeconomic reasons. All stakeholders involved in this complex environment, including regulators, health technology assessment (HTA) bodies, and pharmaceutical companies are strongly dedicated to balance patient’s expectations and the economical sustainability of the healthcare system with the primary objective to enhance the access to innovative, effective, and affordable medicines.
This master thesis provides an in depth analysis to elucidate how regulators in the European Union are increasingly basing their decision making on evidence from the real world to expedite the development process of innovative medicines. This thesis refers to

• current areas of impact of real world evidence on the medicine’s life cycle,
• various activities initiated to foster the scientific progress in this area,
• a set of representative case studies to demonstrate under which circumstances real world data has already been utilized in health care decision making,
• and the challenges associated with the use of real world data in the pre- and post-registration phase.

Based on these insights, it can be estimated that the impact of real world evidence on the regulatory decision making process will further rise in the near future. With special focus on innovative medicines, the classical clinical development will most probably be replaced by a more adaptive and seamless transition from the clinical proof of concept to the post-authorization phase incorporating an increasing level of real world data sources. Especially taking into account the recently strengthened cooperation between competent authorities and HTA bodies in regulatory mechanisms like the Adaptive Pathways leveraging real world evidence, potential synergies between marketing authorization requirements and market access considerations may intensify the harmonization in healthcare decision making processes at European level.
Pages: 60