January 22, 2015—The Food and Drug Administration approved 41 “novel new” drugs in 2014, of which 17 – a record high – were for orphan diseases or conditions, the federal agency says in a new report. [ms-protect-content id=”2799″]
Hundreds of medicines are approved every year, most of them variations of drugs already on the market. The FDA reports annually on the small subset it approves that are unprecedented, serve previously unmet needs, or otherwise stand out. The large proportion of orphan drugs on the list (41 percent) is another sign of the pharmaceutical industry’s turn toward orphan products, which have a streamlined approval process, have a longer exclusivity period, and typically command high prices.
Drug manufacturers are suing the Health Resources and Services Administration over its position that the 340B statute permits rural and cancer hospitals to purchase orphan drugs at 340B prices when the products are used to treat common conditions.
According to the FDA’s orphan drug database, there are 484 drugs with orphan designations approved for sale in the United States, up from 362 at the end of 2010.
One of the newly approved orphan drugs, the advanced melanoma treatment Keytruda from Merck, costs about about $150,000 a year, according to The New York Times. Bristol-Myers Squibb’s rival cancer drug, Blincyto, went on sale in Japan in September at an average annual cost of $143,000, The Wall Street Journal reported.
Orphan drugs will account for almost 20 percent of total worldwide drug sales (excluding generics) by the end of the decade, with year-to-year sales growth more than double that of the overall prescription drug market, the market intelligence firm EvaluatePharma reported last fall. In the U.S., the average per-patient annual cost for orphan drugs in 2014 was $137,782, more than six times the cost for non-orphan products, it said. [/ms-protect-content]