Chapter 11 234 clinical trials, which is at odds with the recently stated vision of EMA executive director Emer Cooke who argued: ‘We believe that the binary discussion between clinical trials and RWE is unhelpful as each approach brings its own strengths and weaknesses.’58 The historical distinction between ‘research’ and ‘treatment’ intent is not always clear – nor should this imply that the primary intent (treatment) should prevent other (research) usages. Electronic health records are clearly intended to aid in the treatment of patients, but have been harnessed on a grand scale to simultaneously facilitate research.59 Third, the conduct of multinational observational studies warrants simplification. The European Clinical Trial Regulation expedites interventional studies via a shared assessment by member states. For non-interventional studies no such pathway exists, which hinders the set-up of studies. This potentially explains why publications frequently cover only the national experience within international compassionate use programs. The burden of setting up separate studies within each individual country or region affects rare diseases in particular, where the effort of initiating an observational study may not outweigh the limited data collection benefits. A centralized noninterventional study procedure could resolve these issues. Fourth, we call for the creation of a unified EU expanded access pathway. The main goal of compassionate use is to provide ‘early access’ to investigational medicine for patients in need. The current set-up consists of a non-binding, optional advice procedure from the EMA, as well as 27 member states with multiple different pathways per member state. To provide expanded access, some countries require ethics committee approval, others do not. Some countries pay for treatment cost, others demand fees from manufacturers. Some countries allow liberal data collection, while others do not allow data collection at all. Harmonization and standardization of compassionate use pathways could reduce costs to regulators and manufacturers and resolve issues of equity in patient access, while also facilitating data collection to supplement trial data, which can be especially important for patients with rare diseases.
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