As reported in NewsWise; rare drugs are few and far between, with a cliental of less than 200,000 Americans dissuading drug companies from taking on the enormous charge of developing novel treatments. In an effort to propel rare progress, the U.S. government started the “orphan drug” program 1983, designed as a financial and regulatory boost to make the development of new treatments for rare diseases more economically feasible for drug companies.

However, researchers pointed out— the orphan drug program often finds its way into subsidizing top drugs that can be used for a rare disease in addition to more common conditions, through an extension of the program known as the “partial orphan drugs.” A team of researchers from the University of Michigan and Boston University in the study “Spending For Orphan Indications Among Top-Selling Orphan Drugs Approved To Treat Common Diseases,” examined how much of partial orphan drug spending actually went to treating rare disease patients.

Their findings showed that just over 21% of the money spent on the top 15 selling partial orphan drugs in 2018 actually went to rare diseases, leaving the vast majority of the money (70%) to ultimately treat more common diseases. This discrepancy points out a loophole incentivizing drug companies to take advantage of the looser regulations and financial subsidies.

“Our findings raise questions regarding whether the benefits of orphan drug status are always warranted for partial orphan drugs,” said Kao-Ping Chua, M.D., Ph.D., the study’s lead author and researcher at UM.

Chua explained that the portion of the money that ultimately goes to the treatment of rare disease patients depends on whether that research originally was created for the rare disorder and later repurposed for a common disease, or if the drug was first made for a common disease before it was discovered to treat a rare disease too and deemed an orphan drug. This is an important distinction because in the former situation, the drug would have still prioritized rare patients, and the by-product benefits a broader community. However, in the latter, drug companies could use the orphan drug status as a loophole to use funds for a more common and lucrative project.

The researchers want to call attention to this distinction because it might illuminate a way that rare disease research could still be made a secondary priority within a category specifically created to help bridge the gap in research dedicated to rare patients. This might imply that some of the partial orphan drugs should be reconsidered as qualifying for orphan drug status funding.

The researchers set out to find how the top 15 partial orphan drugs were ultimately used by patients, considering the records of 12.8 people patients under 65 from a database compiled on insurance claims. Overall, they found a mere 21% of the total spending actually went to treating rare disease patients in 2018. That left the bulk of money to other ends, namely, over 70% of the funds supported the treatment of common diseases.

Amongst the 15 partial orphan drugs reviewed in the study, they found only eight of the drugs were originally created and FDA approved to treat a rare disease. Amongst the eight drugs tailored for rare diseases, around 40% of the spending ultimately went to rare patients.

Even more concerned were the remaining seven drugs, which were created for common disorders before being repurposed for rare drugs and in turn making use of orphan drug benefits. Amongst the seven, a mere 8% of the funding went to rare disease patients, of which a whopping 92% was dedicated still to common diseases. The drug pegafilgrastrim, a chemeotherapy drug claiming partial orphan drug status dedicated a mere 0.6% of their funds to rare disease use, while the remaining 99.4% was portioned to the common diseases.

Chua explained that the use of this status to treat such a large number of common disorders causes a call for concern. For one, because orphan drugs can pass through FDA regulations with lower barriers due to the difficulty of accumulating thorough data on rare disorders. For this reason, a drug company might use the orphan drug status to create a drug for a rare disorder that they know will easily be adapted for a common disease as a loophole to by-pass regulation and costs.

They also point out that companies might use the orphan status in order to protect an already existing medication since orphan drugs have more barriers of time protection against competitors.

The authors suggest a number of policy changes that could dissuade drug companies from misusing the orphan drug status, including providing fewer incentives for partial orphan drugs, and getting off the 340B exemption, which makes orphan drugs exempt from the otherwise mandatory discounts given to hospitals in order to purchase drugs in bulk for cheaper.

While these cutbacks may draw money away from these partial orphan drugs, that would allow more of the funding to be dedicated to the intended beneficiaries of the policy, rare patients. Chua said,

“The tradeoff is that each of these changes could lower investment in discovering new treatment options for people with rare disease.”