Like his predecessors, Scott Gottlieb, the newly sworn-in commissioner of the US Food and Drug Administration (FDA), will have a full plate to tackle as he takes the reins of the short-staffed agency.
At the forefront of the agenda is what Gottlieb called his "highest immediate priority" in the confirmation hearing: the opioid crisis.
While acknowledging that FDA’s past actions may have been too incremental, Gottlieb will now have to deal with a situation that FDA has little control of, though the agency will be able to re-think the approval of new pain medications, some of which may have abuse-deterrent features, as well as shift how health providers are trained and educated on opioids.
Gottlieb will join with Health and Human Services Secretary Tom Price, who has been on a listening tour on opioid abuse, with stops in Michigan, New Hampshire, Maine and West Virginia this week.
Reauthorized User Fees and Cures Act Implementation
The five-year reauthorization of the user fees paid by the pharmaceutical, generic drug, medical device and biosimilar industries to FDA is currently making its way through the Senate and expected to be set in stone before the August congressional recess, or else thousands of FDA employees could receive pink slips.
Included in those agreements are an array of new deadlines and goals that FDA will have to meet in exchange for additional funding. Gottlieb will be tasked with how to juggle the new funds and what should be prioritized.
In addition, implementation of provisions from the recently passed 21st Century Cures Act will open FDA up to new challenges, like deciding how to spend $500 million in new innovation funds.
Under the law, FDA has already begun designating certain regenerative medicines as treatments that could potentially address unmet needs. And beginning in June, FDA will start making public statements on patient experience data as part of all new drug and biologic application approvals. Other deadlines are coming, too.
Off-Label Marketing, Communications
Just before Trump took office, FDA attempted to clarify its position on off-label promotion with a new memo, guidance documents and public meeting. Gottlieb’s close ties to industry will be on full display (see more on conflicts of interest further below) when the agency has to work through some of the disconnects between FDA and industry, especially as FDA in recent years has lost court cases on its regulation of "truthful and non-misleading" speech from drug and device companies.
Generic Drug Labeling
Although speeding up generic drug approvals (specifically first generic drug approvals) may be more at the forefront of Gottlieb’s mind than labeling, FDA has punted on the contentious generic drug labeling rules several times in recent years after first proposing a rule in 2013.
Gottlieb told Focus last year: "FDA needs to bear more responsibility for updating the labels of old generic drugs and can take on that role, rather than foisting it onto generic drug makers in a way that will not only increase generic costs and expose them to the same sort of failure to warn suits that plague branded companies, but will undermine the generic model by creating incongruities between the labeling of the same generic drug, solely by virtue of the manufacturer and what CBE [changes-being-effected] supplements they happened to file. I think this is the most inefficient way to achieve what FDA purports to be their public health goal here, which is why I believe this was politically contrived, or at least politically influenced."
Drug Approval Speed, Patient Voices and Efficacy
One of the biggest issues for FDA in 2016 was figuring out how to handle an overwhelming call from patients to approve a new drug despite serious qualms raised by a committee of outside experts and others within the agency.
The case is part of a larger push from the new Cures law and elsewhere to get FDA to approve pharmaceuticals and biologics more quickly, even though in most cases, FDA is the fastest in the world. How Gottlieb balances FDA's job to remain the world’s top regulator in approving safe and effective drugs, while helping patients deal with unmet needs in a timely manner, will be a tricky one.
Also interesting will be to see how the agency enforces guidance, set to be released in 2017, on "Civil Monetary Penalties for Failure to Meet Accelerated Postmarketing Requirements."
Skyrocketing brand-name and generic drug prices has been at the center of America’s discussion of rising health costs, and though prices and costs generally remain outside FDA’s scope, expect to see some sort of agency action directed at drug prices.
However, Gottlieb has been critical of an idea shared by Democrats and Trump – that allowing more drug imports from Canada or other countries with government cost controls will bring down the cost of US medicines. Other past FDA commissioners and current CDER Director Janet Woodcock have raised safety questions about allowing such imports, and it remains to be seen which drug imports will be allowed and which will not be.
FDA has fallen behind its European regulatory counterparts on its transparency, particularly on clinical trial transparency, though there have been glimmers of hope that FDA might begin releasing more information. First on that transparency wish list is complete response letters, which if released, would provide a better picture of why certain products are rejected by the agency.
A major agency transparency initiative, begun under previous commissioner Margaret Hamburg in 2009, has not seen too many recent updates.
Conflicts of Interest
Like his predecessor Robert Califf, Gottlieb has come under fire for his close ties to the industry he’ll regulate, which is at least part of the reason why 42 democrats voted against his nomination.
And though he has said he will recuse himself from certain agency decisions where conflicts might appear, many FDA observers will be waiting to see how lenient the agency becomes under Gottlieb’s leadership.
The global drug and medical device industries are only getting more global, and that means further collaboration between regulators will be needed, particularly for the most advanced ones in the US, Canada, EU, Japan and Australia. FDA currently regulates products from more than 150 countries.
In March, the US and EU forged a landmark mutual recognition agreement on pharmaceutical manufacturing inspections. Further collaboratve work could help to reduce duplicative clinical trials and manufacturing inspections. FDA and EMA also just wrapped up a joint Quality by Design pilot program, and currently work together on some parallel assessments. At the end of 2016, they also discussed the establishment of a joint working group to further consider the clinical development and data requirements for new antibiotics.
Regulation of LDTs
Like with the generic drug labeling rule, FDA has pushed its decision on how to regulate lab-developed tests (LDTs) down the road multiple times, trying to find the right equilibrium between ensuring LDTs are safe and work properly and how to not stifle their development.
Two months after saying it would not finalize guidance on the regulation of LDTs, FDA in January published a discussion paper with some hints as to how it would like the discussion and regulations on this thorny issue to move forward.