Making Rare Disease Drug Research Pay Off

With drug companies investing huge amounts of time and money to produce viable products, it is not surprising that they look for drug candidates that will provide a return on the investment.  Whereas rare diseases may not have been the way to go before, there are indications that the trend may be changing in some sectors. A case for developing drugs for rare, orphan diseases can be found in the most recent Spotlight On report, a quarterly pharmaceutical review of the key market players, deals and drug developments within a specific disease area from Thomson Reuters Competitive Intelligence.  The most recent issue focuses on the economic potential of ultra-orphan diseases by looking at lysosomal storage disorders (LSDs), a group of more than fifty rare inherited metabolic disorders, each occurring in less than 10,000 people worldwide. According to the report, the pharmaceutical industry had limited interest in orphan drugs until the introduction of the Orphan Drugs Act in 1983.  Now, said the author’s chief editor, Ulrike Jahnke, “The pharmaceutical industry has shown an increasing interest in developing drugs for rare, orphan diseases.  Amongst the orphan diseases is a subgroup of extremely rare disorders, termed ultra-orphan.  The lysosomal storage disorders (LSDs), each of which occurs in less than 10,000 people worldwide, are examples of ultra-orphan diseases.  Despite the very low prevalence of these conditions, therapies for ultra-orphan drugs have yielded high returns for the developing companies, as exemplified by Genzyme’s blockbuster Cerezyme (imiglucerase) for the ultra-rare Gaucher disease.  This short review will assess the LSD market as an example for the market opportunity of targeting such ultra-orphan treatments.” The report shows that developing ultra-orphan therapies can have advantages.  For instance, with such a small target population, treatments for LSDs can command ultra-high prices to recoup R&D expenditure.  There are significant market opportunities.  Many LSD therapies are limited by their inability to treat neurological symptoms, and only 10 of the disorders have a treatment option.  Therapies in the pipeline include Genzyme's substrate reduction therapy Cerdelga (eliglustat tartrate). Since the launch of Ceredase and Cerezyme, 13 orphan drugs targeting 10 different LSDs have entered the market.  Combined sales revenue of 10 of these drugs was $4.055 billion in 2013 and is expected to reach $6.387 billion by 2019.  Revenue for more than half of these drugs could exceed $500 million each by 2019.