Earlier this month in a post concerning the Orange Book we mentioned our love of data and discussed how solid figures can reveal some interesting conclusions. After all, it’s rather difficult – and dangerous – to come to a conclusion without having as many facts and as much data as possible. Or, as Sir Arthur Conan Doyle wrote in The Adventure of the Copper Beeches, one of the Sherlock Holmes series of stories: “‘Data! Data! Data!’ he cried impatiently. ‘I can’t make bricks without clay.’” Indeed! And the clay we have to play with today to make some bricks are the latest data from FDA’s Office of Orphan Products Development (“OOPD”) on orphan drug designations and approvals.
Although FDA’s agency-wide performance management system, known as FDA-TRACK, provides some information on orphan drug designations and approvals, that system is not up-to-date. So we have to get our data from other sources – primarily (though not exclusively) from FDA’s Orphan Drug Designations and Approvals database. As we’ve done in some previous years, we culled information from that database, which OOPD populates with data on a rather frequent basis. The database is also constantly being refined to better reflect what orphan drug designations have been granted, what orphan drugs have been approved, and what the relevant periods of orphan drug exclusivity apply to. We understand that the database will be revamped soon (perhaps later this year) to make it a more useful and user-friendly resource.
The three metrics we’ve historically followed are: (1) the number of orphan drug designation requests received by OOPD; (2) the number of orphan drug designation requests granted by OOPD; and (3) the number of orphan drugs approved. In 2014, records were shattered for all three metrics, with an astounding 467 designation requests (a nearly 35% increase over 2013), an astonishing 293 orphan drug designations granted (a nearly 13% increase over 2013), and a whopping 49 orphan drug approvals (a 53% increase over 2013). Wow! That’s an amazing output for FDA’s orphan drug program (and, in particular, for OOPD).
Below are three tables – one for each metric – showing the year-by-year numbers since 1983.
When we add up all of the numbers since 1983, FDA has approved 511 orphan drugs, granted 3,280 orphan drug designations, and received 4,738 orphan drug designation requests. Of the 511 approvals, some drugs have been approved for more than a single rare disease, and sometimes a single orphan drug designation has been the platform for multiple orphan drug approvals (and multiple periods of 7-year exclusivity).
So what does it all mean? Well, clearly orphan drugs are trending up – way up! And there’s no indication of a slowdown any time soon. (In fact, we understand that OOPD is already on pace to break the 2014 record for the number of orphan drug designation requests received by the Office.) The data also show that the Orphan Drug Act has been an overwhelming success (for both patients and the drug and biotechnology industries). Of course, a successful program breeds copycats. We’ve seen that with the creation of orphan drug programs in other countries modeled after the Orphan Drug Act. It’s also happening on out own backyard, however. Consider, for example, the Generating Antibiotic Incentives Now Act (“GAIN Act”) (FDC Act § 505E), and the Dormant Therapies Act provisions included in the draft 21st Century Cures Act (see our previous post here). The roots for both of those items can almost certainly be traced back to the Orphan Drug Act.
In celebration of Rare Disease Day, NORD is excited to host a special event for the rare disease community that includes a screening of the new documentary, “Banner on the Moon,” about Cindy Abbott and narrated by Valerie Harper.
Join us in D.C. for a reception and to see this inspiring story about love and determination. Space is limited - RSVP to reserve your tickets today!
Thursday, February 26, 2015 | 2:00 – 5:00 p.m.
National Press Club | 529 14th Street, NW, 13th Floor |Washington, D.C. 20045
For the sixth year, we’re proudly partnering with the pharmaceutical company Lundbeck on the “Raise Your Hand to Fight Rare Diseases” campaign, which supports rare disease research in celebration of Rare Disease Day.
The campaign invites individuals to visit the Rare Disease Day U.S. website, http://rarediseaseday.us, and click on the “Raise Your Hand” icon during the month of February. Each unique click triggers Lundbeck to make a $1 donation to NORD’s research grant fund (up to a maximum donation of $10,000).
Rare Disease Day is an international awareness day held annually on the last day of February. NORD is proud to be the official U.S. sponsor for this important event. read more >
The bleeding disorders community will be recognizing Rare Disease Day on February 28, Hemophilia Awareness Month during the entire month of March, and World Hemophilia Day of April 17. These important dates were created to raise awareness and education about rare conditions like hemophilia and bleeding disorders.
NORD is thrilled to partner with our member organization, Hemophilia Federation of America, to raise awareness. HFA is offering shirts that say “I Am Rare. I Matter.” to help you raise awareness during these important upcoming community dates! Proceeds will be donated to HFA and NORD.
Don’t miss out, the limited edition shirts (available in grey and red, crew and v-neck) will only be available until Sunday, February 15. Order here: http://www.booster.com/rarediseaseday2015.
The National Organization for Rare Disorders (NORD)®, a leading independent, non-profit organization committed to theidentification, treatment, and cure of rare disorders and official U.S. sponsor of Rare Disease Day® on February 28, announces a new social media campaign, #1in10, to raise awareness for the 1 in 10 Americans living with a rare disease.
“It’s astounding that there are just as many Americans living with a rare disease as there are people who are left-handed,” said Lisa Phelps, director of marketing and community relations at NORD. “We are launching this campaign for everyone who has a rare disease, their families, caregivers, nurses and doctors to help raise important awareness for this major public health issue and the need for safe, new treatments.”
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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