Extract
The exponential advance in biological and medical science, with the use of molecular and genetic testing as well as various “omics” approaches, has led to the elucidation of many disease pathways. It has also led to the identification of marked differences in the mechanisms, phenotypes and genotypes of diseases that were once thought to have similar characteristics and required similar therapies. An example of this approach is in cystic fibrosis, where patients with the specific G551D mutation respond extremely well to ivafactor (Kalydeco) [1]. Although this mutation is only found in about 5% of all cystic fibrosis patients, their response to treatment is so dramatic that it is truly life changing. However, the cost of this medication is extremely high and it is difficult to imagine that similar costs can be sustained in the long term for other, more common diseases. The number of available monoclonal antibodies and targeted drugs for severe or life-threatening disease, such as cancer and rheumatoid arthritis, is increasing rapidly and expected to continue to rise in the next decade [2, 3]. Moreover, we are now faced with the severe end of the spectrum of many diseases, in which the clinical characteristics and response to treatment differ considerably from mild disease.
Abstract
First results from the U-BIOPRED studies of adults and children with severe asthma http://ow.ly/RKJZ7
Footnotes
Conflict of interest: None declared.
- Received August 26, 2015.
- Accepted August 26, 2015.
- Copyright ©ERS 2015