The Orphan Drug Act originated in 1983 as an incentive for pharmaceutical companies who worked on developing treatments for rare disease. Since rare diseases are well, rare, choosing to work on a rare condition was not at the forefront of the plan for developers who needed to make a living themselves. This Act gave pharmaceutical companies seven years of market exclusivity to reward them for making that jump.

At the time, it was a great solution. In reality, it still is. However, we have now recognized that there are some loopholes that need fixing.

When a company is able to hold on to market exclusivity for longer than seven years due to loopholes in the Act, it eliminates the possibility of cheaper generics coming to the market. For patients struggling to make financial ends meet, this is a huge drawback.

Thankfully, we have good news to share today.

The House of Representatives has just unanimously passed a bill which will help to eliminate this loophole of the Orphan Drug Act while maintaining the integrity of the legislation.

The Bill

This bill requires drug developers to prove that they don’t think they will be able to recoup their research and development costs with 12 years of sales in the U.S.

This will prohibit drug developers who have revamped an old drug that has already received orphan drug designation from obtaining the exclusivity. How the Act stands now, any repurposed orphan drug can have the same exclusivity that the original was afforded, regardless of expected profit.

That was because the Act created two ways to obtain the designation. One way is if the drug they were developing had to support a population of fewer than 200,000. But, this designation could also be provided if the developers didn’t think that they could recover their costs for developing the therapy. The current Act does not require developers to prove this when developing a repurposed version of an orphan drug.

Looking Forward

The hope is that this new bill will allow for continued drug development for rare diseases while providing patients greater choices when it comes to drug generics.

You can read the full explanation of this new, unanimously passed bill here.