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 a new interview with Medscape, Marshall L. Summar, chief of genetics and metabolism at Children’s National Medical Center in Washington, D.C. and NORD board member, talks about the importance of patient registries for rare diseases, and the role that NORD’s patient registry program has in helping patients and educating doctors. According to Dr. Summar, registries can accelerate the process of treatment and help physicians address the big knowledge gap about what happens in the day-to-day lives of patients. read more >
Today, NORD Vice President Diane Dorman is providing remarks before the FDA Oncologic Drugs Advisory Committee on the importance of distinguishable names for biologics as a fundamental core of maximizing the benefits for patients and minimizing any potential harm from biosimilars. This is part of NORD’s work to provide advocacy on behalf of the entire rare disease community. Read Diane’s remarks here and follow the conversation online under the hashtag, #ODAC.
If you have supported NORD with a donation at any time during its 31+ year history, we thank you. The wonderful work that NORD has accomplished since 1983 on behalf of rare disease patients and their families wouldn’t have been possible without the support of many caring individuals.
NORD works very hard to be a careful steward of donated funds, keeping operating costs low so that 95 cents of every dollar goes directly to programs and services for patients.
We know that you receive many solicitations for contributions at this time of year, but we believe that NORD has earned a place at the top of your list through its history of dedicated leadership and service.
And we are excited to tell you about two unique opportunities to drive progress for patients in 2015: promoting state-based advocacy through NORD’s Rare Action Network™ (RAN) and advancing research with an innovative new platform NORD has developed for patient registries and natural history studies. read more >
Alena Galan is a happy 16-year old girl full of life and wise beyond her years. She can light up a room with her quick-witted charm and energetic personality. She loves to sing, dance, play guitar and violin, and like most teenagers, enjoys spending time with friends. Alena has a deep-rooted appreciation of life and cherishes every moment – all with good reason.
On January 24, 2002 Dr. Robert Marion, director of Children’s Evaluation & Research Center, Albert Einstein College of Medicine diagnosed Alena with mucopolysaccharidoses VI (MPS VI) or Maroteaux-Lamy syndrome, a rare inherited lysosomal storage disorder. Individuals with MPS VI do not produce the enzyme that carries all the impurities out of their body. This causes thickening of the bones, breathing difficulties, and ceases normal human growth and development. Symptoms gradually worsen over time, and individuals with MPS VI usually do not survive past 25-years old.
The news devastated Marcia Galan, who adopted three-year old Alena from a Russian orphanage just seven weeks earlier. At the time, Alena – who remarkably resembled Marcia – seemed to be healthy and happy. They immediately bonded as mother and daughter. Shortly after bringing Alena home to the U.S., a pediatrician who specializes in Russian adoptees observed several abnormalities, including a heart murmur, which led to further evaluations and the MPS VI diagnosis. read more >
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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