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Making clinical trials more efficient: trial costs, budget planning, and platform trials

Griessbach, Alexandra. Making clinical trials more efficient: trial costs, budget planning, and platform trials. 2024, Doctoral Thesis, University of Basel, Faculty of Medicine.

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Abstract

Conducting high quality clinical trials is complex, time consuming and expensive. Consequently, making clinical trials more efficient will play an integral part in their sustainability in the future, in addition to reducing research waste. In this thesis we aimed to make clinical trials more efficient by conducting three research projects. Firstly, we determined the costs and resources required to plan, conduct and finalize an investigator initiated randomized controlled trial in Switzerland, Germany and the United Kingdom. Secondly, we investigated the tools and approaches used by Swiss Clinical Trial Units (CTUs) and the Swiss National Science Foundation (SNSF) to calculate trial budgets and compare them to internationally available tools and determine areas of improvement. Thirdly, we explored the platform trial design, to determine if this innovative approach may improve clinical trial efficiency by conducting a systematic review, and through practical experience gained as a trial coordinator for the Corona VaccinE tRiAL pLatform (COVERALL-2).
For the first project, we determined that writing of the protocol, data management and patient follow up contributed the highest proportion to total trial costs. Reduction in these areas has the potential to lead to more cost-effective investigator initiated randomized controlled trials (RCTs). In addition, based on the actual trial costs determined in our work, investigators now have cost estimates and proportions available, on which they can base future budget calculations, since 83% of these RCTs exceeded their original budget. Secondly, we determined that budgeting tools and approaches used by Swiss CTUs and SNSF are lacking, not tested, and do not include all cost relevant items or provide cost ranges for investigators to rely on. As such, we discuss standardizing and improving budgeting approaches and tools across Swiss CTUs. Thirdly, our systematic review identified 127 platform trials in various medical fields. These platform trials also showed lower rates of trial discontinuation and answered many research questions by adding new arms to the trial. Nevertheless, some did not add new arms, and consequently incur higher planning costs and not benefit from the cost savings of adding new arms. The prolific nature of platform trials was confirmed firsthand by the COVERALL trial, which resulted in 3 subprotocols and 7 publications.
In conclusion of this work, we emphasize the importance of confronting the challenge of collecting and sharing clinical trial cost data, ultimately contributing to future success of clinical research. We discuss efforts being made to make trial costs more transparent, such as the 72nd World Health Assembly (WHO, 2019) and The Pharmaceutical Research Transparency Act of 2022 and 2023. Secondly, we also highlight the growing significance of platform trials and identified barriers such as infrastructure challenges, methodological issues, and lack of regulatory guidelines. We discuss recommendations for the future of platform trials and include ongoing efforts made by stakeholders (such as EU-PEARL and EU-Response) to transform platform trial methodology and streamline regulatory processes.
Advisors:Briel, Matthias
Committee Members:Szucs, Thomas and Schwenkglenks, Matthias and Speich, Benjamin and Buyse, Marc
Faculties and Departments:03 Faculty of Medicine > Departement Public Health > Pharmazeutische Medizin ECPM > Pharmazeutische Medizin (Szucs)
UniBasel Contributors:Briel, Matthias and Schwenkglenks, Matthias
Item Type:Thesis
Thesis Subtype:Doctoral Thesis
Thesis no:15601
Thesis status:Complete
Number of Pages:180
Language:English
Identification Number:
  • urn: urn:nbn:ch:bel-bau-diss156014
edoc DOI:
Last Modified:14 Feb 2025 05:30
Deposited On:31 Jan 2025 11:27

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