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Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Views of European Drug Development Stakeholders on Treatment Optimization and Its Potential for Use in
Robbe Saesen1,2, Stéphane Lejeune1, Gianluca Quaglio3
1European Organisation for Research and Treatment of Cancer, Brussels, Belgium.
Background:
The current drug development paradigm has been criticized for being too drug-centered and for not adequately focusing on the patients who will eventually be administered the therapeutic interventions it generates. The drug-driven nature of the present framework has led to the emergence of a research gap between the pre-approval development of anticancer medicines and their post-registration use in real-life clinical practice. This gap could potentially be bridged by transitioning toward a patient-centered paradigm that places a strong emphasis on treatment optimization, which strives to optimize the way health technologies are applied in a real-world environment. However, questions remain concerning the ideal features of treatment optimization studies and their acceptability among key stakeholders.
Objectives:
The aim of this study was to explore the views of key stakeholders in the drug development process regarding the concept of treatment optimization.
Methods:
Semi-structured interviews were conducted between December 2018 and May 2019 with 26 participants across ten EU Member States and six different stakeholder groups, including academic clinicians as well as representatives of patient organizations, regulatory authorities, health technology assessment agencies, payers, and industry.
Results:
Based on the input of the experts interviewed, clarification was obtained regarding the optimal features of treatment optimization studies in terms of their conduct, funding, timing, design, and setting. Moreover, a number of opportunities and challenges of undertaking such trials were identified. Inter-stakeholder discussion during their design was seen as desirable. There was also broad support among the participants for regulatory measures to facilitate treatment optimization, although there was no agreement on the optimal scale and nature of these initiatives. Furthermore, the interviewees believed that the evidence strength of well-designed treatment optimization studies performed according to rigorous quality standards is greater than or at least equal to that of classical clinical trials. In addition, there was a strong consensus that the results of treatment optimization studies should be taken into account during the decision-making of regulators, payers, and/or clinicians.
Conclusions:
Stakeholders involved in drug development consider treatment optimization studies to be valuable tools to address current evidence gaps and support their implementation into the existing research framework.
Insights
Stakeholders find treatment optimization studies valuable for bridging evidence gaps in drug development. These patient-centered approaches are seen as crucial for improving real-world therapeutic application and informing regulatory decisions.
Area of Science:
- Pharmacoeconomics and Health Technology Assessment
- Clinical Trial Design and Methodology
- Oncology Drug Development
Background:
- Current drug development is criticized for being too drug-centered, creating a gap between pre-approval and real-world use of anticancer medicines.
- A patient-centered paradigm emphasizing treatment optimization is proposed to bridge this gap.
- Key stakeholders' views on the ideal features and acceptability of treatment optimization studies require exploration.
Purpose of the Study:
- To explore stakeholder perspectives on the concept of treatment optimization in drug development.
- To identify optimal features for treatment optimization studies.
- To assess the acceptability and potential challenges of implementing these studies.
Main Methods:
- Semi-structured interviews were conducted with 26 participants from diverse stakeholder groups across ten EU Member States.
- Stakeholder groups included academic clinicians, patient organizations, regulatory authorities, health technology assessment agencies, payers, and industry representatives.
- Interviews took place between December 2018 and May 2019.
Main Results:
- Clarification was obtained on optimal features for treatment optimization studies regarding conduct, funding, timing, design, and setting.
- Opportunities and challenges for these trials were identified, with a consensus on the desirability of inter-stakeholder discussion during design.
- Participants believed treatment optimization studies provide evidence strength equal to or greater than classical clinical trials and should inform decision-making.
- Broad support exists for regulatory measures to facilitate treatment optimization, though specifics were not agreed upon.
Conclusions:
- Stakeholders view treatment optimization studies as valuable tools to address evidence gaps in drug development.
- These studies are considered essential for optimizing the real-world application of health technologies.
- Implementation of treatment optimization studies into the existing research framework is supported by key stakeholders.
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