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Table 1 Available Phase II data for the control group for a hypothetical pivotal Phase III design of a new drug in Progressive Supranuclear Palsy

From: Application of Bayesian methods to accelerate rare disease drug development: scopes and hurdles

Study

N

Mean change from baseline to week 52 in PSPRS

Standard error

Boxer et al. [51]

153

10.9

0.99

Tolosa et al. [52]

31

11.4

1.13

Höglinger et al. [53]

59

10.5

1.00

  1. PSPRS is the PSP Rating Scale (PSPRS) which is a disease specific quantitative measure of disability
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172