This post kicks off a series about drug prices in the orphan drug space. We initially wanted to get a sense of orphan drug price trends over the last 30 years — since the passage of the Orphan Drug Act of 1983. There were a few published industry reports regarding orphan drug approvals, but the data was already in aggregate form so it was impossible to do any real analytics. I could either give up or I could go out and try to figure out approximate orphan drug prices manually. There are only 498 FDA approved drugs with Orphan designated status. How hard could it be?
Really hard, it turns out. The lack of transparency in the end user pharmaceutical space is appalling. It’s a nightmare trying to work out exactly how much a patient with a single disease pays per year for their medications. I’m amazed there isn’t a comprehensive data set for information about pharmaceutical prices over time. Data.gov/DjPatil, I’m looking at you on this one. Ultimately, I resorted to limiting my search to only Mendelian (single-gene) diseases. A lot of the orphan approved drugs are for specific cancer indications, and these were removed from the dataset. I was left with ~70 approved orphan drugs.
After a few days of furious Googling, reading and calling pharmacies, I had a semi-comprehensive list of orphan drug prices per patient per year. (All the graphs below were generated in Tableau, per my previous post on data analysis).
Initially the data doesn’t seem to present any obvious trends. However if you filter this data by drug type (biologic or small molecule) an appreciable trend starts to emerge for one group.
If we dig deeper and eliminate the biologics in blue we start seeing a really striking trend of small molecule orphan drug prices.
At some point after 2008, orphan drug prices for small molecules start increasing rapidly. This data represents the estimated cost per patient per year for a certain drug, which is a standard for looking at pharmaceutical trends. It means, however, that I had to make certain assumptions about patient weight, dose requirements, etc. A more robust metric, I believe, is the cost/dose data.
This data tells a really clear story: the price per dose for small molecule orphan drugs for Mendelian disease indications is increasing exponentially. In our next few posts, we’ll look at why that might be the case. Stay tuned for updates and more!
