Pharmaceutical Companies Increasingly Adopting Orphans

Orphan diseases had been traditionally neglected. By their definition, they were often rare and so it was not financial sound to pursue developing a treatment for them. In 1983 however, the US government introduced the Orphan Drug Act and things began to change. According to PhRMA, which represents pharmaceutical manufacturers in America, “In the 29 years since the Orphan Drug Act was established, 398 orphan drugs have been approved.” This trend is set to continue according to new research by BCC Research.

The global market grew to $86 billion in 2012 and at a 5 year compound annual growth rate (CAGR) of 5.4% is set to grow to $112 billion by 2017.

According to the research oncological drugs (both biological and non-biological) make up the largest proportion of the growth in orphan drugs. It may seem strange to categorise cancers as ‘orphans’ because they appear to be relatively common, causing 12.5% of all deaths, but it is with good reason. Researchers are increasingly categorising cancers into more specific sub-types based usually on genetic factors. The increasing availability of sequencing technology means that patients can be more easily diagnosed with a particular sub-type of cancer and so their treatment can be more personalised. Many see this use of pharmacogenomics as the early stages in a truly personalised medicine.

Another trend in this field which mirrors a more general trend in pharmaceuticals is the growing amount of biologicals. Biologicals in the orphan drug market were valued at $57.7 billion in 2012 and at a 4.3% CAGR are set to reach $71.4 billion in 2017. While the non-biologicals market will rise from $28.3 billion to $40.8 billion at an impressive CAGR of 7.5%.

One of the reasons for growth in this area is in the application of the drugs for off-label uses. According to research published in PLOS ONE, “three of four top-selling orphan drugs were used more commonly for non-orphan indications.” This suggests that these drugs may have a much wider application than just the indication for which they were developed.

Overall the trend away from a focus on blockbuster drugs towards new sources of revenue is set to be a defining one over the coming years and it may result in better treatments for those who 40 years ago may not have been deemed worthy of consideration.