Lee et al. [ 21] performed efficacy studies using BALB/c mice bearing C-26 colon carcinoma tumors.
To ensure that these learning opportunities are achieving their intended goals, researchers must perform efficacy studies and use evidence to support evaluative judgments (Dolan, 2008).
We next performed efficacy studies in vivo, using two models of intracranial GBM xenografts, established by a low number (5 × 10) of GSC lines 3832 and 387.
Alternatively, one could perform efficacy studies of a lead compound in disease hiPSC-CM of multiple genetic backgrounds, in essence conducting a 'clinical trial in the dish'.
The study investigators performed efficacy (clinical, radiological, and mycological examinations) and safety evaluations every 3 months, which helped to determine treatment duration.
A fundamental issue with anti-VL chemotherapy is that although the incidence of VL in India is such that high-quality studies can be performed, efficacy against Indian VL does not convey to disease from other endemic regions.
For a number of orphan diseases that are, in particular, the target of gene transfer protocols, it is difficult to obtain material from a large cohort of patients with which to perform efficacy and safety studies.
Moreover, hPSCs also offer a unique, novel platform by which to augment, and even redefine, current drug discovery and drug screening programmes by the provision of a human in vitro tool on which to perform efficacy and toxicity screens for novel chemical entities (NCEs) [ 4, 5].
In general, studies in children will not be performed until efficacy and safety has been demonstrated in adults.
Although more work is needed to improve vaccines against this disease, progress is hampered by the need to perform vaccine efficacy studies in horses.
