The development of novel health technologies is a complex and costly process that follows an extensive set of regulatory guidelines and legal provisions intended to ensure that the treatments reach the patients that need them in a highly controlled and standardised manner. In the European Union (EU), the first step in the path to market access for a new therapeutic intervention is the marketing authorisation procedure coordinated by the European Medicines Agency (EMA), in which applicants have to provide evidence of the safety, quality and efficacy of their product, which is mainly derived from the conduct of clinical trials. Once approval has been granted, each individual EU Member State will decide on how the drug will be introduced into their healthcare systems based on nationally diverging criteria determining its price setting, reimbursement conditions and clinical application.

However, in recent years, this established paradigm has faced increasing criticism from authors in the field, especially in relation to cancer treatment, who have highlighted its contribution to the existence of a research gap between the pre-approval development of anticancer medicines and their post-approval use in real-life practice. A drug-centred attitude dominates the present framework, leaving important patient-focused aspects relating to the real-world utilisation of antitumor therapies unaddressed, including how to combine them with existing health technologies, how their effectiveness compares to that of therapeutically relevant alternatives, how long they have to be administered to achieve the desired effects, whether a lower dose could produce the same results with potentially fewer toxic side effects and how they perform in terms of patient-relevant outcome measures such as quality of life and overall survival.

This situation has led to calls for a transition towards a new paradigm that puts the patient at the centre of clinical drug development and places a strong emphasis on treatment optimisation.Treatment optimisation, which has also been called applied research, seeks to optimise the way health technologies are used in real-world conditions through the conduct of studies designed to provide an answer to one or more of the above mentioned questions. It is not intended to replace the current clinical research framework; instead, it aims to generate results complementing those of the registrational trials as a way to bridge the research gap. However, a number of crucial questions remain regarding the ideal features of treatment optimisation studies, as well as their acceptability among the actors involved in the development and adoption into practice of novel therapies.


Read the complete study on ‘Treatment optimisation in drug development‘ in the Think Tank pages of the European Parliament.