Orphans with Many Parents

Apparently FDA’s Office of Orphan Products Development wants to keep its record year quiet

By Steven E. Kuehn

Isn’t it funny how sometimes you stumble on to something and it quietly astonishes you? I did recently while catching up on current events. One of my news feeds brought forth a headline that really caught my eye: “The 2014 Numbers Are In: FDA’s Orphan Drug Program Shatters Records.” I clicked on it and up came “FDA Law Blog” published by patent law firm Hyman, Phelps & McNamara. And I paused for a second. I’m not sure why I was surprised by the source. In retrospect suppose I was expecting a more mainstream industry media outlet, you know, like, ahem, PharmaManufacturing.com (if you haven’t visited lately, click on over, we’ve upgraded the whole place).

Shameless plugs aside, I read with astonishment a brilliant little piece of data-driven journalism that for once painted the U.S. regulators’ activities in a positive light offering clearly data points that succinctly revealed just how successful the FDA’s Office of Orphan Products Development (OOPD) has been at supporting drug innovation since 1983.

Self-identified blog “master” Kurt R. Karst explained he and his colleagues often cull data from the FDA’s “Orphan Drug Designations and Approvals database” to get metrics relevant to their practice and their clients’ interests. According to Karst, the firm historically follows three metrics: 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,” writes Karst, “records were shattered for all three metrics, with an astounding 467 designation requests (a nearly 35 percent increase over 2013), an astonishing 293 orphan drug designations granted (a nearly 13 percent increase over 2013), and a whopping 49 orphan drug approvals (a 53 percent increase over 2013). ‘Wow!’ That’s an amazing output for FDA’s orphan drug program (and, in particular, for OOPD).” Nice cull Hyman, Phelps & McNamara. Karst sums up OOPD’s track record: “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.”

According to OOPD its mission is to advance the evaluation and development of products (drugs, biologics, etc.) that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. “The program,” declares the OOPD’s home page, “has successfully enabled the development and marketing of more than 400 drugs and biologic products for rare diseases since 1983.”

Wait just a minute I thought to myself, that figure is 111 approvals out of date. My astonishment continued as I cast about for any other coverage of this better-than-average news, especially for a government agency. To my surprise, I could not search my way to another source reporting the fact that the OOPD was able to approve a record 49 orphan-status drugs in 2014. My search of the FDA’s website revealed little more than the database mentioned in Karst’s blog, and even OOPD’s news page had nothing mentioning their big year. In fact, the closest thing I found was a brief mention of the program’s recent success (Q1 performance metrics) in a power point presentation. Apparently, the agency wants to keep this news quiet; I think somebody should call their PR communicators.

All that notwithstanding, it’s a shame the news isn’t getting national attention. For years Orphan Drug Designation program has provided orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people or that affect more than 200,000 persons but their owners are not expected to recover the costs of developing or marketing the drug.

“So what does it all mean?” asks Karst. “Clearly orphan drugs are trending up — way up! And there’s no indication of a slowdown any time soon.” Unequivocally, Hyman, Phelps & McNamara’s analysis reveals that the Orphan Drug Act has been a true success. Orphan drugs have many parents, but you’d think at least one them would want to brag about their kid’s accomplishments!

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