Failure of Drug Development Programs for Chronic Diseases
Original article published on Assay Depot Blog, on 13 May 2014 here.
95% failure rate! Statistics like this for a business would not only justify but also truly warrant shutting down that enterprise. However, this is what drug development in the pharmaceutical world looks like .
Failure of new drugs in late stages of development is a major concern for the pharmaceutical and medical community. Not only does this lead to prohibitive costs for drug development, but it also is a cause for concern for diseases that lack effective treatment options. Chronic diseases like hypertension, diabetes, Alzheimer’s, cancer, autoimmune disorders remain difficult to treat in spite of major technological and biomedical advances. So why have the scientific discoveries from genomics, proteomics, molecular biology, pharmaceutics not translated into effective therapies? Why are effective drugs not being developed? This is a troubling issue that needs to be addressed urgently.
One major growing problem in the 21st century is type II diabetes; it is associated with abnormal responses to insulin (including resistance) leading to uncontrolled blood glucose levels. This leads to a range of pathologies affecting the eyes, kidney, brain etc. Diabetes represents a billion-dollar drug market and has been the subject of intense research, both at the basic and translational levels. The pharmaceutical and biotech industries have been attempting to develop a cure for this condition, with little success.
Similarly, Alzheimer’s disease is affecting an increasing number of people; it is a degenerative disease characterized by dementia and other neurological abnormalities. Basic research has shown the protein amyloid-beta to be responsible, at least in part for the pathology leading to this disease. Millions of dollars were spent on trying to translate these findings into a cure for Alzheimer’s. However, none of these efforts were successful.
Another recalcitrant disease group is that of autoimmune disorders, where the body develops antibodies against its own proteins. This results in a variety of different diseases with moderate to severe disability and can be controlled to some extent by immunosuppressant therapy. However, specific drugs for these diseases would likely achieve better control and need to be developed. Among the most debilitating of the autoimmune disorders are rheumatoid arthritis and systemic lupus erythematosus. There is no effective treatment strategy for these disorders and current therapies are associated with severe adverse effects.
The common theme for all the three disease groups described here is a lack of effective treatment options. New strategies for drug development are needed, in order to develop therapies against these conditions with minimal adverse effects. Stay tuned next week to find out what these new strategies are!
- Phase II and Phase III attrition rates 2011–2012. (2013) Nature Reviews Drug Discovery. Volume 12: 569. doi:10.1038/nrd4090