Chapter 1 22 We here shift our focus from theoretical concepts to practical examples by examining the challenges faced when designing an expanded access program and associated data collection. We will illustrate the design, analyses, and limitations of running an expanded access programs through the lens of two distinct products: pritelivir and tabelecleucel. 1. Pritelivir, an investigational product in infectiology to treat treatment-resistant herpes simplex viruses. 2. Tabelecleucel, a cell therapy for relapsed/refractory patients with a variety of (cancerous) diseases caused in part by Epstein-Bar Virus re-activation in immune compromised patients. The two programs differ in a variety of aspects, such as the nature of the disease areas, type of product, development stage, and type of company. Pritelivir is a small molecule drug, taken as pill orally by patients at home. Tabelecleucel is an advanced therapy medicinal product, derived from human cells and can only be administered intravenously in specialized hospitals. Second, the marketing authorization submission of tabelecleucel was long underway while the expanded access program started, whereas the phase III trials for pritelivir are still ongoing at the time of writing this thesis. On a company level, AiCuris is a privately owned smaller biotech company operating from Wuppertal, Germany. In contrast, Atara Biotherapeutics is a NASDAQ (ATRA) listed, publicly traded company, operating from their headquarters in California in the United States. As a result, the reader receives two separate and diverse expanded access examples from both programs.
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