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Approval, even for an orphan drug will be major. This article is a must read:

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On a late summer day, Tim Coté sat in a corner office of his Sandy Spring, Md., consulting firm, Coté Orphan. He leaned into his computer microphone to dispense insider knowledge about the orphan drug approval process on a webcast hosted by FDAnews, a trade news organization. Listeners paid about $300 a head, but Coté said he wasn’t paid for doing it.

The FDA is more flexible in evaluating drugs for rare diseases, he said, explaining that “about half of them get through with just one pivotal clinical trial. Not so for common diseases.” The FDA, citing a report from the National Organization for Rare Disorders, said about two-thirds of orphan drugs were approved with one adequate and well-controlled trial with supportive evidence. It typically requires two or three such trials to approve a mass market drug.

Coté also told the webinar audience that clinical trials for orphan drugs are usually smaller and the approval is a “different scientific and regulatory experience.”

Coté knows his stuff. He was Rao’s immediate predecessor as chief of the FDA’s Office of Orphan Products Development. It’s not unusual for government officials to leave FDA and other regulatory agencies and obtain jobs as consultants or industry executives.
Dr. Tim Coté, former head of the FDA’s orphan drug office and now principal and chief executive of Coté Orphan (Courtesy of Coté Orphan)

Dr. Tim Coté, former head of the FDA’s orphan drug office and now principal and chief executive of Coté Orphan (Courtesy of Coté Orphan)

Coté’s website, headlined “The Inside Track,” notes that he oversaw applications that led to the approval of at least 150 orphan drugs when he was at the FDA and that his firm is now the largest submitter of orphan drug applications.

“We write the entire application,” the website for Coté’s company notes, adding that his staff of 25 includes regulatory scientists with deep knowledge and experience in FDA’s “unwritten rules” regarding orphan drugs.

Many of Coté’s more than 300 clients are small biotech companies begun by researchers or even passionate parents who found investment backing. Parents Ilan and Annie Ganot, for example, started Solid Biosciences to find treatments and potentially a cure for their son with Duchenne muscular dystrophy.

Coté guides them through the regulatory process since most don’t have the expertise. He can offer his expertise and develop an application that makes it easier for the FDA to designate and approve the drug.

“When you make the FDA smile, the value of your asset goes up. And that’s how the game is played,” he said in an interview, adding quickly, “It’s really not a game because people’s lives are what is in balance.”

Coté and other ex-FDA officials play a vital role in helping drugmakers choose rare disease targets and get through the FDA approval process.

http://khn.org/news/drugmakers-manipulate-orphan-drug-rules-to-create-prized-monopolies/