Directions for new developments on statistical design and analysis of small population group trials

Orphanet Journal of Rare Diseases

Volumn 11, Issue 1, 2016, Pages

  Hilgers, Ralf Dieter ^a   Roes, Kit ^b   Stallard, Nigel ^c   Alberti, Corinne ^d   Van Baal, Caroline ^d   Benda, Norbert ^d   Biesheuvel, Egbert ^d   Burmann, Carl Fredrik ^d   Bogdan, Malgorzata ^d   Comets, Emmanuelle ^d   Day, Simon ^d   Dette, Holger ^d   Dmitrienko, Alex ^d   Friede, Tim ^d   Graf, Alexandra ^d   Karlsson, Mats ^d   Koch, Armin ^d   König, Franz ^d   Van Der Lee, Johanna H ^d   Lentz, Frederike ^d   Madan, Jason ^d   Male, Christoph ^d   Mentré, France ^d   Miller, Frank ^d   Molenberghs, Geert ^d   Neuenschwander, Beat ^d   Posch, Martin ^d   Oosterwijk, Cor ^d   Röver, Christian ^d   Senn, Stephen ^d   Torres, Ferran ^d   Zohar, Sarah ^d   more..

^a RWTH AACHEN UNIVERSITY   (Germany)

^b UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^c UNIVERSITY OF WARWICK   (United Kingdom)

^d NONE

Author keywords

EMA CHMP Guideline on clinical trials in small populations; Rare disease; Small population clinical trials; Statistical analysis; Statistical design; Statistical methods

Indexed keywords

ARTICLE; BAYES THEOREM; CLINICAL TRIAL (TOPIC); DISEASE CLASSIFICATION; METHODOLOGY; POPULATION GROUP; PRACTICE GUIDELINE; PROGRAM DEVELOPMENT; RARE DISEASE; SMALL POPULATION; STATISTICAL ANALYSIS; HUMAN;

CLINICAL TRIALS AS TOPIC; DATA INTERPRETATION, STATISTICAL; HUMANS; POPULATION GROUPS; RARE DISEASES;

EID: 84975132550     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-016-0464-5     Document Type: Article

References (32)

1. Joppi R, Bertele V, Garattini S. Orphan drug development is progressing too slowly. Br J Clin Pharmacol. 2006;61(3):355-60. doi: 10.1111/j.1365-2125.2006.02579.x.
2. EU. FP7-HEALTH-2013-INNOVATION-1. [Online] 2013. [Cited: 23 03 2016.] https://ec.europa.eu/research/participants/portal/desktop/en/opportunities/fp7/calls/fp7-health-2013-innovation-1.html. Accessed 8 June 2016.
3. Molster C et al. Key outcomes from stakeholder workshops at a symposium to inform the development of an Australian national plan for rare diseases. Orphanet J Rare Dis. 2012;7:50. doi: 10.1186/1750-1172-7-50.
4. Orphanet. Orphanet Report Series - List of rare diseases - listed in alphabetical number. [Online] December 2012. [Cited: February 1, 2013.] http://www.orpha.net/orphacom/cahiers/docs/GB/List-of-rare-diseases-in-alphabetical-order.pdf. Accessed 8 June 2016.
5. Eurordis. Fact Sheet. [Online] 2007. [Cited: February 1, 2013.] http://www.eurordis.org/sites/default/files/publications/Fact-Sheet-RD.pdf. Accessed 8 June 2016.
6. Colledge VL, Solly J. The rare disease challenge and how to promote a productive rare disease community: Case study of Birt-Hogg-Dube Symposia. Orphanet J Rare Dis. 2012;7:63. doi: 10.1186/1750-1172-7-63.
7. Breithaupt C. Contribution to the treatment of rare diseases: orphan drugs. J Eur Medical Writers Assoc Write Stuff. 2009;18(1):21-3.
8. EMA. Orphan Medicines Figures 2000. Orphan Medicines Figures 2000/2014 Update 1/1/2015. [Online] 01 01 2015. [Cited: 26 02 2015.] www.ema.europa.eu/docs/en-GB/document-library/Other/2015/04/WC500185766.pdf. Accessed 8 June 2016.
9. irdirc. Current Results of Research. Current Results of Research. [Online] 2016. [Cited: 25 05 2016.] http://www.irdirc.org/rare-diseases-research/current-results-of-research/. Accessed 8 June 2016.
10. CHMP. Guideline on clinical trials in small populations. [Online] 2007. [Cited: February 1, 2013.] www.ema.europa.eu/docs/en-GB/document-library/Scientific-guideline/2009/09/WC500003615.pdf. Accessed 8 June 2016.
11. EMA. Concept paper on extrapolation of efficacy and safety in medicine development. [Online] 2012. [Cited: 1 February 2013.] www.ema.europa.eu/docs/en-GB/document-library/Scientific-guideline/2012/06/WC500129285.pdf. Accessed 8 June 2016.
12. FDA. Rare Diseases: Common Issues in Drug Development Guidance for Industry. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM458485.pdf. [Online] 08 2015. [Cited: 28 02 2016.]. Accessed 8 June 2016.
13. Karlsson, K E. Benefits of pharmacometric model-based design and dnalysis of clinical trials. Acta Universitatis Upsaliensis. 2010;133. http://www.diva-portal.org/smash/record.jsf?pid=diva2%3A360473&dswid=1152. Accessed 8 June 2016.
14. Barrett J. Pharmacometrics: a multidisciplinary field to facilitate critical thinking in drug development and rranslational research settings. J Clin Pharmacol. 2008;48:632.
15. Burzykowski T, Molenberghs G, Buyse M. The Evaluation of Surrogate Endpoints. New York: Springer; 2005.
16. pcori. Patient-Centered Outcomes Research Institute. Patient-Centered Outcomes Research Institute. [Online] 2016. [Cited: 25 may 2016.] http://www.pcori.org. Accessed 8 June 2016.
17. Frank L, Basch E, Selby JV. The PCORI perspective on patient-centered outcomes research. J Am Med Assoc. 2014;312(15):1513-4. doi: 10.1001/jama.2014.11100.
18. Gargon E et al. Choosing important health outcomes for comparative effectiveness research: a systematic review. PLoS One. 2014;9:6. doi: 10.1371/journal.pone.0099111.
19. Kiresuk TJ, Sherman RE. Goal attainment scaling: A general method for evaluating comprehensive community mental health programs. Community Ment Health J. 1968;4(6):443-53. doi: 10.1007/BF01530764.
20. Pocock SJ, Geller NL, Tsiatis AA. The analysis of multiple endpoints in clinical trials. Biometrics. 1987;43(3):487-98.
21. Posch M, Futschik A. A uniform improvement of Bonferroni-type tests by sequential tests. J Am Stat Assoc. 2008;103(481):299-308. doi: 10.1198/016214508000000012.
22. Bretz F et al. A graphical approach to sequentially rejective multiple test procedures. Stat Med. 2009;28(4):586-604. doi: 10.1002/sim.3495.
23. eurordis. European Organisation for Rare Diseases.[Online] [Cited: 23 03 206.] http://www.eurordis.org/. Accessed 8 June 2016.
24. Kennes LN et al. The impact of selection bias on test decisions in randomized clinical trials. Stat Med. 2011;30(21):2573-81. doi: 10.1002/sim.4279.
25. Bretz F et al. Adaptive designs for confirmatory clinical trials. Stat Med. 2009;28(8):1181-217. doi: 10.1002/sim.3538.
26. Gerss JW, Köppke W. Clincal trials and rare diseases. Adv Exp Med Biol. 2010;686:173-90. doi: 10.1007/978-90-481-9485-8-11.
27. FDA. Guidance for the Use of Bayesian Statistics in Medical Device Clinical Trials. [Online] 2010. http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm071121.pdf. Accessed 8 June 2016.
28. Wästfelt M, Fadeel B, Henter JI. A journey of hope: lessons learned from studies on rare diseases and orphan drugs. J Intern Med. 2006;260(1):1-10.
29. van der Graaf R, Roes KC, van Delden JJ. Adaptive trials in clinical research: scientific and ethical issues to consider. JAMA. 2012;307(22):2379-80. doi: 10.1001/jama.2012.6380.
30. Emanuel EJ, Wendler D, Grady C. What makes clinical research ethical? JAMA. 2000;283(20):2701-11.
31. Gupta S et al. A framework for applying unfamiliar trial designs in studies of rare diseases. J Clin Epidemiol. 2011;64(10):1085-94. doi: 10.1016/j.jclinepi.2010.12.019.
32. Cornu C et al. Experimental designs for small randomized clinical trials: an algorithm for choice. Orphanet J Rare Dis. 2013;8:48. doi: 10.1186/1750-1172-8-48.