Consider it done.
From 2015 Was Another Good Year for Orphan Drugs & Pharma Marketers
Although overall approvals show no trend, the number of orphan drug approvals is steadily increasing. They now constitute nearly half of all new drug approvals. Expect this trend to continue as the requests for orphan drug designation (< 200K US patients) are climbing even faster
From The 2014 Numbers Are In: FDA’s Orphan Drug Program Shatters Records
The Orphan Drug Act of 1983 provides 50% R&D tax credits and a 7-year window of exclusivity to designated drugs. It effectively adds a year of monopoly pricing power for the drug manufacturer[1] .
But the real breakthrough – the one that is causing the gold-rush mentality among pharmas – is the demonstration that the market will bear exorbitantly high prices for orphan drugs, especially cancer therapeutics. This graph pretty much tells the story:
From The World of Orphan Drugs
The other key development is the rise of genomic profiling of cancer. Breast and lung and colon cancers – which are nobody’s idea of rare diseases – can now be diced into orphan-friendly sub-diseases according to their driver mutations.
“Personalized medicine” creates only modest cancer survival benefits, but drives spectacular increases in pharma company revenues:
From The business of orphan drugs is booming
Even though this pharma business model is not sustainable, I think we can agree that the orphans are doing quite well for themselves. In fact, they are coming to resemble true orphans in only one characteristic:
[1] Incentives for orphan drug research and development in the United States