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

Ralf-Dieter Hilgers, Kit Roes, Nigel Stallard and for the IDeAl, Asterix and InSPiRe project groups. Orphanet Journal of Rare Diseases 201611:78 DOI: 10.1186/s13023-016-0464-5.

Open Access. Creative Commons Attribution 4.0 International License

This paper aims to raise awareness of the ongoing research and stimulate other groups to work on statistical methodology on design and analysis of clinical trials in small populations. Having well informed researchers, physicians and biostatisticians will result in the use of more efficient methods to conduct a clinical trial in small population groups and thus bring approved treatments faster to our patients.

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