Prices, Opioids, and Ethics Dominate FDA Confirmation Hearing

If confirmed as FDA commissioner, Scott Gottlieb said he will be guided by the science and by the advice of the agency’s career staff to avoid any appearance of bias or political pressure on his regulatory decisions. He states he will move quickly to address the devastating opioid epidemic and to implement provisions of the 21^st Century Cures legislation to “improve the efficiency of the development process” and to ensure that FDA is “an absolutely objective watchdog over this field.”

At his confirmation hearing before the Senate Health, Education, Labor and Pensions Committee on Wednesday, April 4, 2017, Gottlieb fended off charges that he would cave to Trump administration proposals for overhauling FDA regulations and permit his close ties to pharma companies and investment firms to influence his decisions. A main concern of both Republicans and Democrats is how FDA would fare under a hiring freeze and severe budget cuts from the White House. Gottlieb emphasized his commitment to maintaining a strong work force at FDA, and that these experts will lead efforts to update agency review and regulatory practices. While acknowledging that some parts of the agency are more receptive to the “new opportunities” to speed new breakthrough drugs to market, he said that efforts to encourage “a broader embrace of this pathway” must utilize “a bottoms-up approach” led by career staff.

A main concern for the Senators was how FDA can do more to address the nation’s deadly opioid epidemic, which Gottlieb agreed was “a staggering human tragedy” and the “biggest crisis” facing the agency. He pledged to make opioid abuse his top priority, and would address multiple issues, including a review of the framework for developing alternative opioid dosage forms, developing alternative pain therapies, and supporting people in treatment. He raised the question of whether FDA has authority to consider abuse and diversion potential as criteria for approval, and the need to clarify generic-drug standards for older drugs that lack abuse-deterrent features.

The closest Gottlieb came to rejecting a Trump position was in response to a question about whether he believes that vaccines cause autism. Gottlieb said that while he understood the concerns of some parents, he has seen enough research that disputes any connection between immunization and autism. He commented that he has not been shy about making his views known, and will give his “science-based judgment” on this issue, “in the proper venue.”

Prices and generics

In response to multiple comments that FDA should do more to address widespread public concerns about high drug prices, Gottlieb focused on efforts to speed more generic drugs to market, particularly those complex therapies where blood tests are not sufficient to demonstrate similarity and interchangeability. While looking to make changes internally, Gottlieb also suggested that the agency may need additional authority from Congress in this area. And he pledged to have FDA issue promised guidance documents on developing biosimilars on time.

This focus on more generics, though, did not deter Sen. Bernie Sanders of Vermont from expressing his “amazement” that President Trump would nominate an FDA commissioner who disagreed with administration proposals for expanding drug importing and Medicare drug pricing negotiations, as Gottlieb has stated in his many articles and commentaries on these issues.

Gottlieb also acknowledged the possible need for a new framework to prevent companies from using the regulatory process to delay competition and gain a commercial advantage. Here, the candidate said that his background in industry and in the investment field would be valuable in understanding and designing policies related to inappropriate use of the regulatory policies in commercial disputes.

Gottlieb also addressed queries about whether faster, more efficient clinical trials could help lower costs and drug prices. He supported efforts to modernize the conduct of clinical studies, without compromising FDA’s gold standard for ensuring product safety and effectiveness. While he will examine proposals for a compressing Phase II and Phase III studies, he maintained the need for a “proper clinical program.”

HELP Committee chairman Sen. Lamar Alexander (R-Tenn.) opened the hearing by emphasizing the need for timely reauthorization of FDA user fees, but few other panel members brought up this imperative. Gottlieb said that he will advocate for a strong FDA, with proper resources and an “appropriate user fee program.”  He is expected to have a chance to do so, with confirmation expected in a few weeks, after the candidate responds in writing to an expected wave of additional questions from the Senate panel.