Useful links & resources for investigators

Useful documents for investigators

To optimise the chance for a successful application,read the top 10 tips and tricks here.

Each call has its own specific forms and documents to be used. You can find the forms for the 2020 investigator-led call here under the section 'Downloads (all documents Call 2020)'.

Useful links on Pragmatic trials

Pragmatic trials are trials that are designed to compare treatment options in a real-world setting, as opposed to explanatory trials that test a physiological or clinical hypothesis, typically to develop new drugs or other treatments. Trials can be pragmatic with regards to several features, such as the way sites and patients are recruited, the choice of outcomes, the interventions and their delivery etc.  

Other resources

Pragmatic Trials

• This NEJM paper gives more information on pragmatic trials: http://www.nejm.org/doi/full/10.1056/NEJMra1510059
• The PRECIS (Pragmatic-Explanatory Continuum Indicator Summary) is a tool to help trialists designing clinical trials consider where they would like their trial to be on the pragmatic or explanatory continuum. It can be found here: https://www.precis-2.org/
• The authors of the GetReal consortium (workpackage 3) have a series of interesting publications on pragmatic trials:
  • Zuidgeest M, Goetz I, Groenwold et al. Pragmatic trials and real-world evidence-an introduction to the series. J Clin Epidemiol 2017 [in press] http://www.jclinepi.com/article/S0895-4356(17)30540-1/fulltext
  • Worsley S, Oude Rengerink K, Irving E et al. Challenges in pragmatic trials: selection and inclusion of usual care sites. J Clin Epidemiol 2017 [in press] http://www.jclinepi.com/article/S0895-4356(17)30478-X/fulltext
  • Oude Rengerink K, Kalkman S, Collier S et al. Participant eligibility, recruitment, and retention in pragmatic trials. J Clin Epidemiol 2017 [in press] http://www.jclinepi.com/article/S0895-4356(17)30480-8/fulltext
  • Kalkman S, van Thiel G, Zuidgeest M et al. Challenges of informed consent for pragmatic trials. J Clin Epidemiol 2017 [in press] http://www.jclinepi.com/article/S0895-4356(17)30479-1/fulltext
  • Welsing P, Oude Rengerink K, Collier S et al. Outcome selection and measurement in pragmatic trials. J Clin Epidemiol 2017 [in press] http://www.jclinepi.com/article/S0895-4356(17)30481-X/fulltext
  • Irving E, van den Bor R, Welsing P et al. Collecting and reporting safety data and monitoring trial conduct in pragmatic trials. J Clin Epidemiol 2017 [in press] http://www.jclinepi.com/article/S0895-4356(17)30482-1/fulltext

  • Meinecke A, Welsing P, Kafatos G et al. Data collection in pragmatic trials. J Clin Epidemiol 2017 [in press]
    http://www.jclinepi.com/article/S0895-4356(17)30776-X/fulltext

  • Zuidgeest M, Welsing P, van Thiel, J et al. Pragmatic trials: defining questions, choosing comparators, allocating treatments. J Clin Epidemiol 2017 [in press]
    http://www.jclinepi.com/article/S0895-4356(17)30749-7/fulltext

Presentation on pragmatic trials by Professor Sandra Eldridge

In June 2017 KCE Trials was delighted to welcome Professor Sandra Eldridge (Professor of Biostatistics; Director, Pragmatic Clinical Trials Unit, Centre for Primary Care and Public Health, Blizard Institute, Queen Mary University of London, UK) to KCE to give a talk on pragmatic trials. This focused on an introduction, some issues, and emerging designs. She also did a Q&A where she gave short answers on many key questions in pragmatic trials. Her talk and Q&A can be viewed via the link below.

Patient Involvement

• European Patients' Academy on Therapeutic Innovation: https://www.eupati.eu/
• Ligue des usagers des services de santé: http://www.luss.be/
• Vlaams Patiëntenplatform: http://www.vlaamspatientenplatform.be/

Sample size

• You can also find a questionnaire, developed by Pr. Cook from Oxford University, to help applicants better justify their underlying justification of the sample size used in a randomised controlled trial grant proposal. The document can be found here.
• DELTA2 Guidance – Difference Elicitation in trials https://www.csm.ox.ac.uk/research/methodology-research/delta2

Trial Design & reporting

• NHS Health Research Authority Guidance – Specific questions that need answering when considering the design of clinical trials https://www.nihr.ac.uk/funding-and-support/documents/current-funding-opportunities/hta/guidance-questions-considerations-clinical-trials.pdf
• The COMET (Core Outcome Measures in Effectiveness Trials) initiative: http://www.comet-initiative.org/
• International Consortium for Health Outcomes Measurement (ICHOM): http://www.ichom.org
• The CONSORT (Consolidated Standards of Reporting Trials) website: http://www.consort-statement.org/
  • CONSORT extension for pragmatic trials: http://www.consort-statement.org/Media/Default/Downloads/Extensions/CONSORT%20Extension%20for%20Pragmatic%20Trials.pdf

Risk-adapted approaches

• MRC/DH/MHRA Joint Project - Risk-adapted Approaches to the Management of Clinical Trials of Investigational Medicinal Products

 Health Economics

• Cleemput I, Neyt M, Van de Sande S, Thiry N. Belgian guidelines for economic evaluations and budget impact analyses: second edition. Health Technology Assessment (HTA). Brussels: Belgian Health Care Knowledge Centre(KCE). 2012. KCE Report 183C. https://kce.fgov.be/sites/default/files/atoms/files/KCE_183_economic_evaluations_second_edition_Report_update.pdf
• EUnetHTA: Endpoints used for Relative Effectiveness Assessment: health-related quality-of-life and utility measures http://www.eunethta.eu/outputs/endpoints-used-relative-effectiveness-assessment-health-related-quality-life-and-utility-mea

Other useful links

•  Website of the Federal agency for medicines and health products (FAGG/AFMPS): https://www.fagg-afmps.be/en

• mHealthBELGIUM, the Belgian platform for mobile applications that are CE-marked as a medical device https://mhealthbelgium.be/en/about-us/.  The requirements of Levels I, II, and III (Level I: the app is a medical device; Level II: the app ensures a safe connection; Level III: the app shows clinical benefit and is reimbursed) are described in the menu on top  “Publish your app”.

• ICH Good Clinical Practice http://www.ich.org/products/guidelines/efficacy/article/efficacy-guidelines.html

  • ICH GCP E6(R1) http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E6/E6_R1_Guideline.pdf
  • ICH GCP E6(R2) http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E6/E6_R2__Step_4.pdf