Regulation

Gottlieb’s durable reforms

How Gottlieb’s reforms will persist after he walks away from FDA

While most of the changes Scott Gottlieb has set in motion in just under two years as FDA commissioner are works in progress, and some may falter in his absence, an important number are well underway and will bring lasting changes to FDA, as well as to companies that develop medicines and patients who rely on their products.

His reforms will be durable, and as the rest of the world looks to FDA as the gold standard, they will be emulated around the world.

Gottlieb, like FDA, is held in high esteem. He was not pushed out of office, enjoys strong support in the White House and is wildly popular on both sides of the aisle on Capitol Hill, with advocacy groups, the press and with biopharma companies. The next commissioner -- acting or permanent -- will not have a mandate to roll back policies or pursue a radically different agenda.

Because the changes Gottlieb championed were largely based on proposals from agency staff, FDA’s civil servants will follow through on the blizzard of initiatives the agency has launched under his leadership (see Figure: “FDA Guidance Documents since 2000”).

Crafting and releasing guidance documents is one of the ways progress gets incorporated into FDA’s DNA.

Crafting and releasing guidance documents is one of the ways progress gets incorporated into FDA’s DNA. It makes policies lasting, and it makes them transparent.

Transparency has been a hallmark of Gottlieb’s tenure. He has communicated more, and more effectively, than any previous FDA commissioner -- and arguably, any other U.S. government agency leader.

One marker of his communications prowess is his use of social media as a tool to educate, amuse and interact. Gottlieb has sent nearly 5,000 tweets since being sworn in on May 11, 2017 from his official Twitter account, averaging 7.6 tweets per day. He has used the medium to communicate about the status of FDA operations during the partial shutdown, to promote vaccination, and to banter with journalists about socks and soy milk.

Gottlieb has embedded cutting-edge science in the agency through senior staff recruitments, tipping the odds that some of those changes will outlast him. Among them is his hiring of Amy Abernethy to the No. 2 spot at FDA, principal deputy commissioner, bringing deep expertise in digital health and as an oncologist. Abernethy was CMO of Flatiron Health Inc., now a unit of Roche, where she led initiatives to turn electronic health records into real world data.

But Gottlieb’s attempt to revamp diagnostics regulation may not have the same momentum, unless his successor picks up the mantle.

Promoting the social contract

Enactment of the 21st Century Cures Act and user fee reauthorizations during the Obama administration created tailwinds for the initiatives Gottlieb has championed, such as integrating real world evidence into product development and streamlining reviews of life-changing cell and gene therapies.

Those effects will last beyond his tenure as well.

The laws also helped FDA attract talent by giving the agency leeway to offer salaries that are more competitive with the private sector.

Gottlieb leveraged his personal popularity with members of Congress and his standing in the Trump administration to achieve a substantial budget boost starting in FY19 that will fund new regulatory science initiatives (see “Budget Deal Includes Massive FDA Funding Boost”).

The results of some of the pro-competition activities Gottlieb pushed forward will not become obvious until after his departure.

Gottlieb has sought to stretch laws to make them fit circumstances that weren’t envisioned when they were written. He has championed new paradigms for regulating digital technologies based on assessments of developers’ quality systems, allowing rapid iteration rather than freezing products by requiring premarket review of proposed modifications.

More fundamentally, he has introduced new ways of thinking about FDA’s mission, for example, by making a public health argument for promoting competition and fighting anticompetitive practices.

As commissioner, Gottlieb has embraced the idea that medical product manufacturers should comply with implicit social contracts (see “Breach of Contract”).

And in that light, he has pushed staff to increase the numbers of generic drugs reviewed and approved (see Figure: “Generic Drug Approvals”). 

The results of some of the pro-competition activities Gottlieb pushed forward will not become obvious until after his departure. These include policies that encourage and prioritize applications to launch generic versions of off-patent drugs that have little or no competition, as well as agency investments in regulatory science to facilitate development of complex generics.

Similarly, the effectiveness of organizational changes will take time to manifest, including a complete rewiring of the Office of New Drugs at the Center for Drug Evaluation and Research and restructuring the Office of the Commissioner. The restructuring, designed by CDER Director Janet Woodcock, is intended to make reviews dramatically more efficient and consistent, and enhance the scientific expertise and stature of its scientists and physicians (see “Getting Flatter”).

In contrast to reforms FDA can put into effect independently, the future is unclear for reforms Gottlieb has championed that require action from Congress.

The proposed scheme for regulating diagnostics would focus FDA’s oversight on tests with the highest risks, shift regulatory focus from premarket to postmarket, and create systems for voluntary precertification of technologies and quality systems. The agency also would integrate external expertise into regulatory decisions and increase scientific and medical transparency (see “Debating Diagnostics”).

In September 2018, Gottlieb predicted it would take about a year to gain consensus in Congress on the proposal. That timeline will depend on who succeeds him.

Companies and Institutions Mentioned

Roche (SIX:ROG; OTCQX:RHHBY), Basel, Switzerland

U.S. Food and Drug Administration (FDA), Silver Spring, Md.