Towards the new generation of clinical trials – trials methodology research

Deadline: Apr 7, 2020  
CALL EXPIRED

Efficient and effective clinical trials^[1] are the primary means to provide scientific evidence to ensure optimal health interventions. Although the randomized controlled trial (RTC) design is regarded as the gold standard for evaluating the effectiveness of intervention in clinical research, there is a need for new trial methodologies that address current challenges such as:

• Globalization of clinical research;
• Use of emerging health technologies^[2],[3],[4];
• Defining patient populations and patient enrolment strategies;
• Data management^[5].

Given that all clinical research relies on voluntary contribution of patients, new designs may reduce the operational complexity, assure transparency and build trust, meeting all ethics standards and protecting the individuals’ personal identity and privacy^[6].

Additionally, non-commercial trials often show suboptimal performance as compared to large commercial trials in terms of data collection, management and processing, good clinical practice compliance, and pharmacovigilance, there is a need of a new methodology that improves their legislative compliance and encourage clinical trials conducted by non-commercial sponsors.

Proposals should focus on methodology research and develop innovative solutions to improve the design, conduct and analysis of clinical trials. Proposals should identify and validate methods that will improve the generalizability of evidence generated through differently designed trials, including personalized medicine approaches and combinatorial interventions^[7]. In order to draw meaningful conclusions following state of the art of statistical analyses, applicants need to demonstrate access to adequate clinical trial data sets that will be included into the proposed research.

The proposed methodology should allow sound extrapolation in various subgroups of disease of high public health burden as well as integration of RTC data and post-approval evidence generation. Furthermore, applicants should identify best practices to prevent bottlenecks in execution of clinical trial, including issues related to patient recruitment, adherence and compliance, governance, ethics, sex and gender-based analysis as well as data sharing.

The special attention should be put on non-commercial trials, including quantifiable indicators to measure the qualitative improvement in terms of trial management, data processing, and reporting. Whenever relevant, proposals should cover different aspects of training exercises, including hands-on trainings and closer monitoring of the scientific and technical staff involved in the conduct, management and analysis of the trial.

All literature analyses to define the current state of the art in the clinical trial methodology research must be completed at the time of submission of the proposal. Methodology research related to clinical studies exclusively on medical devices is not in the scope of this topic.

In this topic, the European Medicines Agency^[8] (EMA) and the Commission Expert Group on Clinical Trials^[9] will support the selected applicant consortium in the implementation of the action. Successful applicants under this topic are also expected to liaise with the successful applicants of the relevant coordination and support action (CSA) topics^[10],[11], in order to exchange information, avoid potential overlapping activities, create synergies and support the CSA goals. To maintain the interactions with the CSA consortia, specific tasks and a dedicated budget should be foreseen in the proposal. Additionally, consultations with the European Centre for Disease Prevention and Control^[12] should also be envisaged as additional relevant activities of the successful proposals.

Please note that this topic will take the form of lump sums as defined in Commission Decision C(2017)7151 of 27 October 2017^[13]. Details of the lump sum funding pilot scheme are published on the Funding & Tenders Portal^[14]together with the specific Model Grant Agreement for Lump Sums^[15] applicable.

The Commission considers that proposals requesting a contribution from the EU of between EUR 4 and 6 million would allow this specific challenge to be addressed appropriately. Nonetheless, this does not preclude submission and selection of proposals requesting other amounts.

• Improved relevance, quality and efficiency of clinical trials conducted with public funding.
• Potential to establish a novel clinical trial methodology supported by regulatory authorities.

Trials Methodology Research refers to research into the methods used in the design, conduct, analysis, reporting and knowledge translation of clinical trials to ensure that effective and efficient methods are available for the conduct of clinical trials.

Gender

[1]Clinical Trial Regulation EU No. 536/2014; https://ec.europa.eu/health//sites/health/files/files/eudralex/vol-1/reg_2014_536/reg_2014_536_en.pdf

[2]https://www.who.int/medical_devices/innovation/new_emerging_techs/en/

[3]Regulation 2017/745 on Medical Devices; https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32017R0745&from=EN

[4]Regulation 2017/746 on In-Vitro Diagnostic Devices;

[5]Eichler HG and Sweeney F. The evolution of clinical trials: Can we address the challenges of the future? Clin Trials. 2018 Feb;15(1_suppl):27-32. doi: 10.1177/1740774518755058. PubMed PMID: 29452522

[6]Regulation (EU) 2016/679 of the European Parliament and of the Council of 27 April 2016 on the protection of natural persons with regard to the processing of personal data and on the free movement of such data (General Data Protection Regulation); http://data.europa.eu/eli/reg/2016/679/2016-05-04

[7]European Council Conclusion on personalised medicine for patients (2015/C 421/03) (https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:52015XG1217(01)&from=EN) for combinatorial interventions, see https://www.england.nhs.uk/wp-content/uploads/2015/03/test-bed-prospectus.pdf

[8]https://www.ema.europa.eu/

[9]http://ec.europa.eu/transparency/regexpert/index.cfm?do=groupDetail.groupDetail&groupID=1464&NewSearch=1&NewSearch=1

[10]Proposals funded under the SC1-HCO-18-2020 topic: “Developing an adaptive methodological framework for improved clinical investigation and evaluation of high-risk medical devices”

[11]The consortium of the project STARS (825881) “Strengthening training of academia in regulatory sciences and supporting regulatory scientific advice”; the project website is not yet established.

[12]https://ecdc.europa.eu/en/home

[13]http://ec.europa.eu/research/participants/data/ref/h2020/other/legal/lump_sum/lumpsumdecision-2017-7151_en.pdf

[14]https://ec.europa.eu/info/funding-tenders/opportunities/portal/screen/home

[15]http://ec.europa.eu/research/participants/data/ref/h2020/mga/lumpsum/h2020-mga-lumpsum-pilot-multi_en.pdf