Adopting Orphans More than Ever
Jim Kompare

At an agency working with several orphan-drug companies, GA knows that this would never have been possible without the effort of rare-disease organizations who pushed for the Orphan Drug Act more than 30 years ago.

In the 1970s and early 80s, very few treatments for rare diseases were brought to market by industry. In response, rare-disease organizations banded together, calling for legislation to encourage orphan drug development. Thanks to those efforts, in 1983 Congress passed the Orphan Drug Act, which provides a number of incentives for manufacturers to develop and commercialize orphan drugs: tax breaks, enhanced patent protection, exclusive marketing rights, waiver of FDA user fees, and often expedited reviews.

Over the past 3 decades, more than 450 drugs and biologic products for rare diseases have gained FDA approval, and more than 3300 orphan drug designations have been granted. And the momentum behind pharma’s shift to orphan drugs is real.

Across pharma big and small, research dollars and expertise continue to move from common-disease blockbusters to orphan drugs. The number of new orphan drug designations in 2010 to 2014 rose from 195 to a record 286. In the first two months of 2015, there have already been 53 new orphan drug designations, according to the FDA’s website.

As a result of this shift in research, expertise, and commercialization, orphan drugs are projected to be nearly 20% of global prescription sales by 2020, according to EvaluatePharma’s Orphan Drug Report 2014. The growth rate for orphan drugs is nearly triple that of drugs treating larger populations, 11% vs 4%.

GA is proud to work with several leading orphan drug companies—Baxter, Johnson & Johnson, Pharmacyclics, Shire, Biomarin, and Sarepta Therapeutics. We’ve helped launch and relaunch orphan drugs in hematology-oncology (Imbruvica), immunology (HyQvia), inherited metabolic disorders (Kuvan, Elaprase, Replagal, and VPRIV), and others.

To learn more about the latest trends in the orphan drug market, you can access EvaluatePharma’s Orphan Drug Report 2014 at www.evaluategroup.com/orphandrug2014.