The new EMEA-CHMP guideline on clinical trials in small populations - methodological and statistical considerations with published examples

• Autorzy: Gerß J. , Köpcke W.
• Języki publikacji: EN

Abstrakty

EN

The EMEA-CHMP guideline on clinical trials in small populations considers problems associated with clinical trials, when there are very few patients available to study. In such conditions, less conventional and/or less common methodological approaches may be acceptable. In the present report, selected strategies for approaches to trials in small populations are briefly outlined. It focuses on Chapter 6 of the guideline – Methodological and statistical considerations. Methods are addressed, that may increase the efficiency of the design or analysis of a trial where large studies are not feasible. In addition to general principles, specific examples of published studies are presented.

Słowa kluczowe

EN

EMEA-CHMP guideline; clinical trial; small population; rare disease; small sample; response-adaptive randomisation methods; group sequential designs; n-of-1 designs

PL

próba kliniczna; choroba; metoda losowa; statystyka

• Wydawca: Nałęcz Institute of Biocybernetics and Biomedical Engineering of the Polish Academy of Sciences ; Elsevier
• Czasopismo: Biocybernetics and Biomedical Engineering
• Rocznik: 2007
• Tom: Vol. 27, no. 3
• Strony: 59--66
• Opis fizyczny: Bibliogr. 5 poz., tab., wykr.

Twórcy

autor

Gerß J.

autor

Köpcke W.

• Münster University Hospital, Departament of Medical Informatics and Biomathematics, Domagkstr. 9, 48129 Münster, Germany,

Bibliografia

• 1. International conference on harmonisation of technical requirements for registration of pharmaceu-ticals for human use - ICH harmonised tripartite guideline E11: Clinical investigation of medicinal products in the pediatric population, 2000. http://www.ich.org/LOB/media/MEDIA487.pdf
• 2. European Medicines Agency (EMEA), Committee for Medicinal Products for Human Use (CHMP): Guideline for Clinical Trials in Small Populations, 2005. http://www.emea.eu.int/pdfs/human/ewp/8356105en.pdf
• 3. Giles F.J., Kantarjian H.M., Cortes J.E., Garcia-Manero G., Verstovsek S., Faderl S., Thomas D.A., Ferrajoli A., O'Brien S., Wathen J.K., Xiao L.-C., Berry D.A., Estey E.H.: Adaptive randomized study of Idarubicin and Cytarabine versus Troxacitabine and Cytarabine versus Troxacitabine and Idarubicin in untreated patients 50 years or older with adverse karyotype acute myeloid leukemia. Journal of Clinical Oncology 2003, 21, 9, 1722-1727.
• 4. Trnavský K., Fischer M., Vögtle-Junkert U., Schreyger F.: Efficacy and safety of 5% Ibuprofen cream treatment in knee osteoarthritis. Results of a randomized, double-blind, placebo-controlled study. Journal of Rheumatology 2004, 31, 565-572.
• 5. Wegman A.C.M., van der Windt D.A.W.M., de Haan M., Devillé W.L.J.M., Chin A Fo C.T., de Vries Th.P.G.M.: Switching from NSAIDs to Paracetamol: A series of n of 1 trials for individual patients with osteoarthritis. Annals of the Rheumatic Diseases 2003, 62, 1156-1161.