Cystic Fibrosis - Heat Map and Analysis
Cystic fibrosis (CF) is a progressive genetic condition and is the result of a patient inheriting a faulty cystic fibroses transmembrane conductance regulator (CFTR) gene from both parents. The defective CFTR genes produce faulty CFTR proteins on the body’s epithelial cells, and result in the buildup of a thick and sticky mucus in the lungs and pancreas. Historically therapeutic options have focused heavily on treating the symptoms, but despite being clinically and commercially successful have failed to address the faulty CFTR proteins that cause the disease. This unmet need has led to the development of CFTR modulators.
The treatment algorithm patients receive is likely to consist of an antimicrobial, a mucolytic agent, pancreatic enzymes and possibly a CFTR modulator. This tabular heatmap framework, designed to provide an easily digestible summary of these clinical characteristics, provides detailed information on all late-stage clinical trial results for products in the CF market and Phase III or Preregistration stages of the pipeline. These are split along drug class lines, and are therefore reflective of the treatment algorithm.
All safety and efficacy endpoints reported in these trials are displayed. In addition, key study characteristics such as the size, composition and patient segment of the study population are provided. These results are presented in a visually accessible, color-coded manner in order to maximize ease of use.
The accompanying text provides a detailed analysis of the clinical benchmarks set by the current market landscape, and the anticipated changes to these benchmarks, and to the treatment algorithm, as a result of the late-stage pipeline.
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