Klin Farmakol Farm. 2026;40(2):97-101 | DOI: 10.36290/far.2026.024

Innovative clinical trial designs

Adriána Papiež1, 2, Lenka Součková1, 2, Jitka Rychlíčková1, 2, 3
1 Farmakologický ústav, Lékařská fakulta, Masarykova univerzita Brno
2 Centrum excelence CREATIC, Lékařská fakulta, Masarykova univerzita Brno
3 Fakultní nemocnice u sv. Anny v Brně

Innovative clinical trial designs represent an important shift in the methodology of clinical research, reflecting the growing complexity of diseases, the development of personalized medicine, and the emergence of advanced therapy medicinal products. In addition to traditional randomized controlled trials, approaches such as adaptive designs, master protocols or single-arm studies are increasingly being used, enabling more flexible and targeted evaluation of new therapeutic interventions. They may accelerate the identification of effective therapies and improve the efficiency of resource utilization; however, they also introduce methodological and organizational challenges, particularly in relation to statistical planning, control of bias, and interpretation of results. In the Czech Republic, the implementation of innovative trial designs remains limi­ted, mainly due to insufficient methodological expertise, limited funding in the academic sector, and the lack of systematic education in clinical trial methodology. Capacity-building initiatives may help strengthen the methodological background and support a more active involvement of Czech institutions in the international research landscape. Innovative trial designs represent a key tool of modern clinical research, and their further development will depend on systematic methodological support and interdisciplinary collaboration.

Keywords: innovative designs, clinical research, adaptive designs, clinical trial methodology.

Received: April 16, 2026; Revised: June 3, 2026; Accepted: June 11, 2026; Published: July 1, 2026  Show citation

ACS AIP APA ASA Harvard Chicago Chicago Notes IEEE ISO690 MLA NLM Turabian Vancouver
Papiež A, Součková L, Rychlíčková J. Innovative clinical trial designs. Klin Farmakol Farm. 2026;40(2):97-101. doi: 10.36290/far.2026.024.
Download citation

References

  1. Bothwell LE, Avorn J, Khan NF, et al. Adaptive design clinical trials: a review of the literature and ClinicalTrials.gov. BMJ Open. 2018;8:e018320. Go to original source...
  2. Bhatt DL, Mehta C. Adaptive Designs for Clinical Trials. N Engl J Med. 2016;375:65-74. Go to original source...
  3. Kaizer AM, Belli HM, Ma Z, et al. Recent innovations in adaptive trial designs: a review of design opportunities in translational research. J Clin Transl Sci. 2023;7:e125. Go to original source...
  4. European Medicines Agency. Methodological issues in confirmatory clinical trials planned with an adaptive design - Scientific guideline [Internet]. European Medicines Agency; 2007 [cited 2026-04-15]. Available from: https://www.ema.europa.eu/en/methodological-issues-confirmatory-clinical-trials-planned-adaptive-design-scientific-guideline.
  5. Center for Drug Evaluation and Research. Adaptive Design Clinical Trials for Drugs and Biologics Guidance for Industry [Internet]. U.S. Food and Drug Administration; 2020 [cited 2026-04-15]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/adaptive-design-clinical-trials-drugs-and-biologics-guidance-industry.
  6. European Medicines Agency. ICH E20 adaptive designs for clinical trials - Scientific guideline [Internet]. European Medicines Agency; 2025 [cited 2026-04-13]. Available from: https://www.ema.europa.eu/en/ich-e20-adaptive-designs-clinical-trials-scientific-guideline.
  7. Chow SC, Chang M. Adaptive design methods in clinical trials - a review. Orphanet J Rare Dis. 2008;3:11. Go to original source...
  8. Miller E, Gallo P, He W, et al. DIA's Adaptive Design Scientific Working Group (ADSWG): Best Practices Case Studies for 'Less Well-understood' Adaptive Designs. Ther Innov Regul Sci. 2017;51:77-88. Go to original source...
  9. Kaizer AM, Belli HM, Ma Z, et al. Recent innovations in adaptive trial designs: a review of design opportunities in translational research. J Clin Transl Sci. 2023;7:e125. Go to original source...
  10. Woodcock J, LaVange LM. Master Protocols to Study Multiple Therapies, Multiple Diseases, or Both. N Engl J Med. 2017;377:62-70. Go to original source...
  11. Bogin V. Master protocols: New directions in drug discovery. Contemp Clin Trials Commun. 2020;18:100568. Go to original source...
  12. Park JJH, Siden E, Zoratti MJ, et al. Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols. Trials. 2019;20:572. Go to original source...
  13. Wang Y, Zhai H, Wang J, et al. Study protocol of an open-label prospective phase II umbrella study of precise neoadjuvant therapy for patients with stage II-IIIB resectable non-small cell lung cancer (PURPOSE). Front Oncol. 2022;12:1052774. Go to original source...
  14. Center for Drug Evaluation and Research. Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics Guidance for Industry [Internet]. U.S. Food and Drug Administration; 2024 [cited 2026-04-18]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/master-protocols-efficient-clinical-trial-design-strategies-expedite-development-oncology-drugs-and.
  15. Lu C, Li X, Broglio K, et al. Practical Considerations and Recommendations for Master Protocol Framework: Basket, Umbrella and Platform Trials. Ther Innov Regul Sci. 2021;55: 1145-1154. Go to original source...
  16. Cohn AL, Day B-M, Abhyankar S, et al. BRAFV600 mutations in solid tumors, other than metastatic melanoma and papillary thyroid cancer, or multiple myeloma: a screening study. Onco Targets Ther. 2017;10:965-971. Go to original source...
  17. Roustit M, Demarcq O, Laporte S, et al. Platform trials. Therapies. 2023;78:29-38. Go to original source...
  18. Burki T. Platform trials: the future of medical research? Lancet Respir Med. 2023;11:232-233. Go to original source...
  19. Saville BR, Berry SM. Efficiencies of platform clinical trials: A vision of the future. Clin Trials. 2016;13:358-366. Go to original source...
  20. RECOVERY Collaborative Group, et al. Dexamethasone in Hospitalized Patients with Covid-19. N Engl J Med. 2021;384:693-704. Go to original source...
  21. Hobbs FDR, Dorward J, Hayward G, et al. The PRINCIPLE randomised controlled open label platform trial of hydroxychloroquine for treating COVID19 in community based patients at high risk. Sci Rep. 2025;15:23850. Go to original source...
  22. Waite AAC, Peto L, Gordon AC, et al. Platform Trials to Assess Therapeutics in Patients Hospitalized With Influenza. J Infect Dis. 2025;232:S254-S261. Go to original source...
  23. Angus DC, Berry S, Lewis RJ, et al. The REMAP-CAP (Randomized Embedded Multifactorial Adaptive Platform for Community-acquired Pneumonia) Study. Rationale and Design. Ann Am Thorac Soc. 2020;17:879-891. Go to original source...
  24. UCSF. UCSF Alzheimer's Disease Trial: Alzheimer's Tau Platform: Master Protocol [Internet]. University of California San Francisco; 2025 [cited 2026-03-14]. Available from: https://clinicaltrials.ucsf.edu/trial/NCT06957418.
  25. Berry SM, Petzold EA, Dull P, et al. A response adaptive randomization platform trial for efficient evaluation of Ebola virus treatments: A model for pandemic response. Clin Trials. 2016;13:22-30. Go to original source...
  26. Lillie EO, Patay B, Diamant J, et al. The n-of-1 clinical trial: the ultimate strategy for individualizing medicine? Pers Med. 2011;8:161-173. Go to original source...
  27. Wang M, Ma H, Shi Y, et al. Single-arm clinical trials: design, ethics, principles. BMJ Support Palliat Care. 2024;15:e004984. Go to original source...
  28. Yao S, Shang Q, Ouyang M, et al. Designing Single-Arm Clinical Trials: Principles, Applications, and Methodological Considerations. Ann Clin Epidemiol. 2025;7:90-98. Go to original source...
  29. Thorlund K, Dron L, Park JJH, et al. Synthetic and External Controls in Clinical Trials - A Primer for Researchers. Clin Epidemiol. 2020;12:457-467. Go to original source...
  30. Pignatti F, El-Galaly TC, Kaiser M, et al. Assessing Overall Survival Benefits in Advanced Cancers: The Role of External Comparator Cohort Studies with Real-World Data. Clin Pharmacol Ther. 2026;119:1080-1087. Go to original source...
  31. European Medicines Agency. Single-arm trials as pivotal evidence for the authorisation of medicines in the EU [Internet]. European Medicines Agency; 2023 [cited 2026-04-22]. Available from: https://www.ema.europa.eu/en/news/single-arm-trials-pivotal-evidence-authorisation-medicines-eu.
  32. Center for Drug Evaluation and Research. Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products [Internet]. U.S. Food and Drug Administration; 2023 [cited 2026-06-03]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-use-real-world-data-and-real-world-evidence-support-regulatory-decision-making-drug.
  33. Center for Drug Evaluation and Research. Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products [Internet]. U.S. Food and Drug Administration; 2025 [cited 2026-06-03]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/real-world-data-assessing-electronic-health-records-and-medical-claims-data-support-regulatory.
  34. European Medicines Agency. Use of real-world evidence in regulatory decision making - EMA publishes review of its studies [Internet]. European Medicines Agency; 2023 [cited 2026-06-03]. Available from: https://www.ema.europa.eu/en/news/use-real-world-evidence-regulatory-decision-making-ema-publishes-review-its-studies.
  35. Almirall D, Nahum-Shani I, Sherwood NE, et al. Introduction to SMART designs for the development of adaptive interventions: with application to weight loss research. Transl Behav Med. 2014;4:260-274. Go to original source...




Clinical Pharmacology and Pharmacy

Madam, Sir,
please be aware that the website on which you intend to enter, not the general public because it contains technical information about medicines, including advertisements relating to medicinal products. This information and communication professionals are solely under §2 of the Act n.40/1995 Coll. Is active persons authorized to prescribe or supply (hereinafter expert).
Take note that if you are not an expert, you run the risk of danger to their health or the health of other persons, if you the obtained information improperly understood or interpreted, and especially advertising which may be part of this site, or whether you used it for self-diagnosis or medical treatment, whether in relation to each other in person or in relation to others.

I declare:

  1. that I have met the above instruction
  2. I'm an expert within the meaning of the Act n.40/1995 Coll. the regulation of advertising, as amended, and I am aware of the risks that would be a person other than the expert input to these sites exhibited


No

Yes

If your statement is not true, please be aware
that brings the risk of danger to their health or the health of others.