Scott Gottlieb

FDA Commisioner Scott Gottlieb (Credit: scottgottlieb.com)

Many are keen to see a more adaptable, modern drug approval system but a keen eye is being held over any signs of personal relationships and attitudes tainting the need for proper procedure.

A letter obtained by the independent watchdog group the Project on Government Oversight (POGO) in a Freedom of Information Act request reveals that Amicus Therapeutics CEO John Crowley sent Gottlieb a friendly note—just months before the experimental drug Amicus makes was fast-tracked by the FDA.

“Scott, Congratulations, again!” the note from Crowley opens. “Thanks for your attention to the attached.” The attachment was a more formal letter suggesting that the FDA withdraw its request for additional research into one of Amicus Therapeutics’ drugs.

Crowley’s company makes a drug to treat Fabry disease, a rare, inherited disorder that often leads to fatal organ damage. The company had already raised a few eyebrows after the FDA withdrew a request to run additional clinical trials following a meeting between Crowley and President Donald Trump. Crowley’s daughter Megan suffers from another rare disorder, Pompe disease. At his first address to a joint session of Congress, Crowley’s daughter sat in the first lady’s box and Trump mentioned her by name, promising to speed up treatments for other rare diseases.

“If we slash the restraints, not just at the FDA but across our government, then we will be blessed with far more miracles like Megan,” Trump said at the time. (Amicus also developed a drug that cured Megan.)

Weeks later, Crowley authored an article in the Observer praising Gottlieb’s nomination.

Last year, the agency asked for another study on the drug’s side effects that would have delayed approval by at least two years. Withdrawing such a request is an extremely rare move. Then in September, Amicus won another victory with the agency, earning special fast-track status to expedite the drug’s regulatory review. Fast-tracking is no guarantee that a drug will be approved, but it does give a drug the opportunity to hit the market sooner. The drug was slated to be approved as early as this month. The letter provides additional insight into how personal relationships may have played a role in those decisions behind the scenes.

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The venture firm where Gottlieb was a partner prior to his FDA position, New Enterprise Associates, was also a significant investor in Amicus Therapeutics, POGO notes. A more formal note from Crowley to Gottlieb was augmented by a second one in which he addressed the commissioner in familiar terms and references an earlier conversation about speeding up approval for rare diseases. In the formal note, dated May 11—before the FDA withdrew its request for additional trials in July—Crowley wrote that research was “not feasible in a reasonable amount of time.”

In a statement to Gizmodo, the FDA said Gottlieb “was not involved in decisions related to this product review.”

Furthermore, the agency said that Gottlieb didn’t even read the letter.

“Dr. Gottlieb was not involved in this matter and further, he was not aware of this letter,” the agency said. “Product-specific correspondence that is sent to Dr. Gottlieb by external parties is referred to the relevant centers as a matter of routine procedure. Further, decisions on whether to accept to file an application or grant a drug application priority review (or any of the other pathways for drug review) is based on specific criteria applied to the product’s data and determined by appropriate FDA career staff.”

Either way, the correspondence is a reminder of the role that chummy relationships between regulators and industry (or industry and academia) can sometimes wind up directly impacting public health. It’s not a new concern—but it is a very real one.

Since taking office in May 2017, Food and Drug Administration head Scott Gottlieb has set about working to make drug approval a less “slow and burdensome” process. In September, Sarepta Therapeutics released early data from a study of a new drug to treat some patients with the rare muscle-wasting disease, Duchenne Muscular Dystrophy (DMD). 

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The FDA are in the process of outlining exactly how efficiency can be created within the regulatory process. Gottlieb gave some clues about the direction of travel: “We need to make sure that our approach to regulation is efficient, and doesn’t become an obstacle to the translation of scientific discoveries into practical solutions for patients. We need to make sure that we’re using the best science so we maintain our gold standard for determining safety and benefit.”

FDA Commissioner Spills His Thoughts on Drug Development Costs

Furthermore, he went on to explain a way in which costs and time can be cut is through the use of combined-phase studies, otherwise known as seamless trials.

He commented, “Instead of conducting the usual three phases of study, seamless trials encompass one adaptive study where the phases are separated by interim looks. By using one large, continuous trial, it saves time and reduces costs. It also reduces the number of patients that have to be enrolled in a trial.”

Under Rick Pazdur, the FDA’s Oncology Centre of Excellence is “taking steps to better evaluate and cultivate these new approaches as one part of our ongoing efforts to modernize our approaches,” Gottlieb said.

Also in the pipeline are plans to modernise, via advanced computing methods. The agency is also working on the use of ‘Master Protocols” to enable greater coordinated ways to use the same trials structure to evaluate treatments in more than one subtype of a disease or type of patient.

He also warned the industry that “by front-loading the cost of drug discovery, the broader biomedical community is making it harder to advance new ideas. It’s economically harder to capitalise the cost of an early stage drug program, relative to funding a later stage project. So, front-loading the costs is a recipe for reducing the amount of new ideas that can be advanced.

“We need to do these things to make sure we’re providing an efficient path for the translation of cutting-edge science into practical treatments that are going to benefit patients,” he said. “We need to do these things because the rising cost of drug development is unsustainable.

“Unless we find ways to modernize how we approach our work, and make more efficient use of our resources, then we’re going to get fewer medicines, and higher costs.”

In the era of Trump’s “buddy business” style scrutiny will be intense over any changes within authorities, especially those with the safeguarding of public health at the heart of their mandates.