Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Optimization of post-trial patient treatment - A challenge of highest regulatory need ***

Ariane Bruche (Abschlußjahr: 2022)

Summary
Language: English
Every new drug development is based on the outcomes of the related pivotal clinical trial. The generation of robust safety and efficacy data is only possible due to patients´ voluntary participation in research and in turn results in the obligation to care for the patients beyond clinical trial termination. For this reason, the provision of the investigational medicinal product must be ensured in case of treatment success until national market entry and finalization of reimbursement negotiations. Whereas the conduct of clinical trials is highly regulated, the post-trial phase is not harmonized among European countries and underlies national legislation. An alignment outside the clinical trial framework is only viable on country level resulting in different access opportunities for the citizens of the individual countries. Even though clinical trials are undoubtedly the best means to learn about the effects of an innovative medicinal product and simultaneously protect the trial participant, the conduct is both elaborate and expensive for all stakeholders. A gradual transition from the strictly monitored and also restricted clinical trial environment to routine treatment conditions would be optimal for the assurance of continuous provision of an investigational medicinal product. However, neither an overarching framework nor Europe-wide guidance is momentarily applicable for sponsors to meet their responsibility of post-trial treatment until commercial product launch.
Compassionate use programs (= CUP) for patient cohorts or provision of investigational medicinal products on a named patient base are often used to bridge this gap. But this route is relatively uncontrolled, hence a hard and risky switch for patients coming out from a clinical trial and not endorsed by all sponsors and investigators. Additionally, there is no common agreement within Europe about the chargeability of unauthorized medicinal products within a CUP or any other form of early access. This ambiguity can lead to criticism whatever option is chosen: if the CUP medicinal product is provided free of charge this can be regarded as a commercial tactic to capture a market share prior to the grant of marketing authorization whereas the decision to prize a CUP medicinal product might lead to the allegation to make profit from compassion. Consequently, there is a high need to find a common pan-European solution and finally regulation to address this lack of clarity and to harmonize national legislation in order to keep Europe attractive for pharmaceutical research and development.
The purpose of this master thesis is to outline the current options within several European countries including Switzerland and the United Kingdom and decide how to provide post-trial access to an innovative medicinal product prior to the grant of marketing authorization in a practical, but fictional example. In this context the implications and necessary preparatory actions were defined based on a structured decision analysis, the legal background explained and the need for decisive and overarching regulation highlighted. The applicable requirements on country level have been summarized and listed in a table at the end of this master thesis. Information was gathered via publicly available databases, Cortellis and direct communication with health authorities and trade associations primarily via email.
Pages: 81
Annexes: 5, Pages:10