INDUSTRY
The Orphan Drug Outlook Orphan drugs are going through an interesting era. Although they are intended to treat rare conditions – orphan diseases that affect less than 200,000 persons in the US – this area has taken on a special importance in recent years. For one thing, the investigation of orphan drugs often leads to medical breakthroughs that might not otherwise have been achieved, sometimes for populations facing life-threatening conditions. It’s also usually easier to gain both government support and marketing approval for an orphan drug, in order to offset the eventuality that the drug may not be profitable in the short run (even though the market cost of these drugs has been rising steadily). And those medications, once in the marketplace, may lead to further indications down the road.
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In 2019, nearly half – 21 of 48 – novel drugs approved by the FDA in the U.S. were for rare or orphan diseases. The companies producing them often get longer exclusivity periods, tax credits and lower regulatory fees. This encourages them to take on what would otherwise be risky research studies. It also helps them conduct clinical trials, which are more difficult and costly with smaller populations of potential trial participants (the Clinicaltrials.gov website reports that 32% of uncompleted rare disease trials cited lack of available patients as the reason for their failure).
Clinical trial design is more difficult for uncommon diseases, but this can lead to innovation in trial design that will help other companies in the future. Lavinia Meloni, spokeswoman for the European Federation of Pharmaceutical Industries and Associations, says that “The use of real-world data (RWD) can also be particularly useful in rare disease trials, for example by utilizing the information in patient registries for patient stratification and sometimes replacing one study arm with information from RWD and even in some cases replacing the traditional clinical trial when the population is too low to run a randomized trial.”
