HD researchers have put tremendous effort into looking for biomarkers that track with disease progression, potential endpoints that show differential signals across stages of the disease, and, more recently, pharmacodynamic biomarkers that provide mechanistic readouts. Many of these tools were identified with convergent evidence from studies of varying sizes, from multiple countries with a broad spectrum of researchers, and generally with a high standard for scientific integrity. The ultimate goal of this effort is to provide tools that facilitate clinical development by refining patient selection, demonstrating early signals of therapeutic benefit, and ultimately establishing efficacy of new treatments. The lack of existing effective treatments complicates this task. We are now in the fortunate position of being able to evaluate these tools in the context of ongoing and upcoming clinical trials. Clinical development of a new therapeutic proceeds through a sequence of studies where results at each step impact decisions for future studies. Because of the impact that false positives and false negatives will have on future decisions, it is our responsibility to use the most rigorous standards for the design, analysis and interpretation of these studies. Hopefully over the next few years we will have not only validated tools, but also effective treatments with which to evaluate future tools.
- clinical trials
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