Orphan drug clinical trials often are adversely affected by a lack of high quality treatment efficacy data that can be reliably compared across large patient cohorts derived from multiple governmental and country jurisdictions.
Morphine is accepted as the gold standard for cancer pain treatment, however high-quality placebo-controlled efficacy data in cancer pain is lacking.
Such trials will need to be conducted in a high incidence population, to obtain efficacy data within a realistic time frame.
It appears none of these molecules were tried during the epidemic in Africa, likely due to the lack of efficacy data in higher order species.
The German CAO/ARO/AIO-94 study showed that preoperative chemoradiation was well tolerated and carried no higher risk of postoperative morbidity, but efficacy data are awaited (Sauer et al, 2001).
39, 42, 43 We used the efficacy data in this subpopulation of high-risk patients in FREEDOM for sensitivity analyses.
Overall results suggest moderate to high rates of acceptability and feasibility of using technology to treat headaches, although efficacy data are more limited.
In the social sector there is a real poverty of efficacy data.
When a new biologic was produced, the manufacturer usually obtained data exclusivity to protect the clinical trial data for the medicine, including safety and efficacy data.
Efficacy data will be available in 2001.
