A new bipartisan bill aims to help accelerate the development of targeted drugs to treat rare diseases, including Duchenne muscular dystrophy, cystic fibrosis, some cancers and other genetic diseases.

Although it's unclear how exactly the bill will aid in the development of targeted rare disease treatments, one of the cosponsors, Sen. Elizabeth Warren (D-MA), said the bill would clarify the US Food and Drug Administration's (FDA) current authority to consider research supporting previously approved targeted treatments.

Background

The FDA Office of Orphan Products Development (OOPD) currently incentivizes the development of products for rare diseases by offering orphan status to drugs and biologics intended to treat rare diseases/disorders that affect fewer than 200,000 people in the US.  

If a company develops a new rare disease treatment, it is eligible for seven years of marketing exclusivity during which time the product is shielded from generic competition. Other incentives include tax credits, grants and user fee waivers.

The program has successfully enabled the development and marketing of more than 500 drugs and biologics for rare diseases since 1983, including 49 in 2014 alone. Still, there are about 30 million Americans who suffer from a rare disease, and about half of those are children, according to a recent FDA blog post.

Late last month, FDA issued additional guidance to industry to help sponsors tackle common issues tied to orphan drug development. Among the suggestions and hopes of the agency, FDA said it's looking to see more drugmakers conduct natural history studies to gain greater knowledge about the specific diseases.

In 2014, FDA also offered more guidance to sponsors on how to improve their meetings with OOPD.

Bill and Support

Senators said that the new bill introduced Tuesday, known as the Advancing Targeted Therapies for Rare Diseases Act of 2015, affirms FDA's authority to "allow innovators to use their own data supporting the approval of a targeted therapy to help facilitate additional targeted therapies to treat patients with the same rare disease."

"It is our expectation that this bill will enable sponsors developing therapies for Duchenne - and other devastating rare diseases and disorders with profound unmet medical needs – to leverage similar or closely related underlying technologies and/or data to accelerate the development pathway for additional targeted therapies,” Parent Project Muscular Dystrophy President Pat Furlong said.

The senators noted in a statement that the bill does not seek to change FDA's current approval standards.

"Companies developing these targeted therapies are saving and lengthening lives, and if we allow them to expand the scope of their current testing we can potentially save even more lives," Michael Bennet (D-CO), one of the sponsors of the bill, said in a statement. 

Sen. Orrin Hatch (R-UT) also explained that the bill would help to "remove difficulties involved in conducting conventional trials for genetic subgroups of many rare diseases," thereby incentivizing new drug development, creating greater efficiency in the drug review process and bringing needed treatments to patients faster.

The Parent Project Muscular Dystrophy (PPMD), the Muscular Dystrophy Association, the Duchenne Alliance and the National Organization for Rare Disorders (NORD) all support the bill.

Bill Necessary?

A lack of examples and specifics on how exactly the bill would help speed the development of rare disease treatments, and whether those ideas would pan out, particularly since it won't impact FDA's review process for orphan drugs, may impede its passage.

The bill's introduction also follows the introduction of a number of bills with roots that can be firmly traced back to the landmark Orphan Drug Act, which paved the way for all of the recent approvals of rare disease treatments. Among those bills is the House-passed 21st Century Cures Act, which could also help to advance rare disease research and treatments. The Senate is currently working on its version of the bill.

In addition, a study from May found that FDA has been increasingly flexible in its approach to rare diseases.  Among 27 orphan drugs approved between July 2010 and June 2014 for non-cancer indications, study authors found that flexible approvals were the rule—not the exception—for orphan drugs.

Regardless, even the introduction of such a bill will help to raise awareness around the difficulties involved in developing orphan drugs and running clinical trials for such small patient populations.

Bill Announcement