Tobias Polak

Introduction: Background and outline of this thesis 19 PERSONAL MOTIVATION This thesis was inspired by the practical challenges encountered in my daily work of designing, implementing, and analyzing expanded access programs. I was working for the company myTomorrows, a service provider in the field of expanded access.1 myTomorrows helps patients and physicians find and access treatment options, and simultaneously designs and conducts expanded access programs for the biopharmaceutical industry. In my daily work, I was responsible for overseeing the data collection and analysis for our clients. In 2019, I was working on designing a data collection program for pritelivir,48 an antiviral therapy for treatment-resistant herpes simplex virus, when I started searching for information on how others had implemented expanded access programs. I stumbled upon a single report highlighting examples of the regulatory use of expanded access data for efficacy assessments.37 This report included lutetium 177Lu dotatate (Lutatheraâ„¢), a radioactive treatment for pancreatic and gastrointestinal cancers.49 The Lutathera program provided access to and collected data from 1,214 patients at a single center,50 the Erasmus MC in Rotterdam, making my hometown an ideal starting point for future research. Although some informal information was available online, I realized that there was a lack of systematic groundwork to help identify and avoid common pitfalls in executing expanded access programs, as well as an absence of literature on best practices. This sparked my interest and prompted me to embark on the journey of this thesis, aiming to strengthen the theoretical foundation of utilizing expanded access with the hope of addressing my day-to-day issues. On a personal level, back in 2020, I received a call from an old teammate of my bridge team facing bile duct cancer. By accompanying him to his weekly doctor appointments in Rotterdam, I witnessed firsthand the challenges faced by patients and doctors when no further treatment options are available. Sadly, my friend passed away on March 9th, 2021. I will forever remember our lively discussions on making the most of his remaining days, only a negligible part of these talks was dedicated discussing expanded access and clinical trials, putting the relative importance of the topics discussed in this thesis in perspective. 1 https://mytomorrows.com

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