According to a story from BioSpace, a report on the results of the Orphan Drug Act of 1983 that was originally commissioned by the National Organization for Rare Disorders (NORD) was published recently. The report was specifically focused on the impact of the period of market exclusivity that orphan drugs enjoy if they are approved.
Why Market Exclusivity?
Generally, the analysis indicated that the exclusivity period was working as the original law intended. One of the major benefits of Orphan Drug designation, which is reserved for therapies designed to treat rare diseases, is a seven year period of market exclusivity if the drug gains approval for public use. This measure is intended to guarantee drug developers a return on investment for the drugs, which often take a lot of time and money to develop.
The study found that market exclusivity did not prevent competitors, such as generics or biosimilars, from entering the market. The study instead linked the dearth of generic competitors to the fact that the return on investment was not substantial enough; after all, generics of orphan drugs do not qualify for special designations and are still limited by the small market that a treatment for a single rare disease often has.
Rising Prices? Stop Blaming Orphan Drugs
Some critics have claimed in the past that orphan drugs are part of the reason that drug prices are continuing to climb, but several facts that the report found appear to refute this claim.
First off, adding an orphan designation to an existing drug did not result in a sudden jacking up of the price, at least most of the time. In fact, the price tended to rise more slowly. In addition, prices of drugs designed to treat more common diseases tended to rise at a faster pace in comparison to rare disease drugs since 2009. Of course, it is also worth noting that prices for many rare disease drugs tend be extremely high in the first place.
Depending on how you break down the data, it may be easy to make it look like orphan drugs are part of the problem. After all, many of them are among the most expensive on the market. But this fact alone does not mean that they are somehow causing other drugs to get more costly.
The report also listed several other findings. Of the 217 drugs which had lost patent protection of orphan exclusivity, 116 currently have biosimilars or generics. In addition about 25 percent of orphan drugs that gained approval were in indications with less than 5,000 patients in the country.
Overall, the report indicates that the Orphan Drug Act of 1983 is still working more or less as intended. Learn more about the report here.
