FDA, Politics

Gottlieb tackles speculators, FDA transparency and the R&D gold standard in a last round of queries ahead of confirmation vote

Fielding a final barrage of written questions from a group of skeptical Democratic US Senators ahead of today’s committee vote on Scott Gottlieb’s nomination as head of the FDA, the nation’s likely next Commissioner of Food and Drugs vowed to square off against financial speculators who are gaming the system to charge high prices, reaffirmed his unwavering support for the country’s gold standard on drug development and reviews and called for publishing most of what the agency’s CRLs say about regulators’ reasons for rejecting a marketing application.

This interchange between the prospective head of the FDA and lawmakers helps illustrate key points Gottlieb is likely to raise in the coming years with the world’s drug developers. And they mark a clear line in the sand that illustrates where the agency is likely to be more — and less — accommodating for companies charged with handling the world’s drug pipeline.

Democratic Senators Robert Casey (D-PA) and Al Franken (D-MI) both pressed him on drug pricing and the role he could play at the FDA in bringing costs down. Franken in particular cited hundreds of generic drugs with no current competition, leaving them wide open to players like Martin Shkreli, the former CEO at Turing, who hiked the price of generic Daraprim by more than 5000%, triggering a lingering controversy.

Here is Gottlieb’s answer, which he repeated in other responses to the array of questions he faced (emphasis added):

While drug pricing does not fall directly within FDA’s purview, I believe the agency can play an important role on this important issue by taking steps to improve product competition. If confirmed, I will work to ensure FDA has the appropriate policies and processes in place to effectively facilitate generic market entry and competition, especially for complex drugs that sometimes don’t face effective generic competition even long after patent expiries. Reforming the regulatory pathway for complex generic products would address one key policy deficiency that results in unnecessary barriers to the development and review of generic competitors for some innovator products for which traditional bioequivalence and bioavailability testing alone are sometimes insufficient for proving sameness. FDA should also explore options to improve the efficiency and consistency of ANDA review processes and timelines, so that financial speculators cannot engage in a regulatory arbitrage, by dramatically hiking the price of some very old generic drugs because they know it can take years for new generic competitors to enter the market.

Senator Sheldon Whitehouse (D-RI) noted that there have been occasions when generic manufacturers protested that branded drug manufacturers were raising hurdles on accessing drugs to delay cheap knockoffs.

Part of Gottlieb’s response:

If manufacturers inappropriately refuse to provide their product to prospective generic competitors, this would be a concern to FDA and become a matter for potential enforcement action by the Federal Trade Commission.

Also, he added, reforming the pathway on complex generics could also help address this.

Tammy Baldwin (D-WI) encouraged Gottlieb to get behind new rules to use patient-reported outcomes measures in the review process, to “capture data beyond just disease symptoms and physical functioning to include psychosocial health measures, including distress screening (e.g., concerns related to disruption of work/family life [due to the regimen], concerns related to nutrition, financial impact and others). This would provide meaningful patient feedback about issues that may not be identified through the current measures being used in the clinical trial process.”

Gottlieb:

I strongly agree that patient experiences, preferences, and perspectives should play an important and appropriate role in FDA’s regulatory policy-making and decision-making. Among other approaches, I have advocated that we continue to advance well-validated, scientific tools for incorporating Patient Reported Outcomes (PROs) as endpoints in clinical trials. I support these and other measures that would allow meaningful patient feedback to be incorporated into regulatory decision making to better define patient-experience data that may not be identified through the current measures being used in the clinical trial process.

Senator Christopher Murphy (D-CT) raised Gottlieb’s stand on transparency, an issue that comes up routinely for an agency barred by law from discussing details on drug data — and much more.

I am a strong proponent of data transparency—for patients, physicians, and manufacturers. I have long advocated that the FDA release more information related to its own review process that could be used to better inform consumers and product developers alike. If confirmed, I will be committed to working with Congress, patients, industry, and stakeholders on the issue of data transparency and new ways that FDA could potentially make important of its own information and deliberations more readily available to the public.

And in another answer to one of Senator Elizabeth Warren’s (D-MA) questions, he added an important point on his commitment to opening up more.

This includes the complete response letters, after proper redaction of commercial confidential information.

Through it all, Gottlieb repeatedly committed to maintaining the agency’s standards on proving a drug’s efficacy and safety ahead of an approval. And he stuck to that as Senator Patty Murray (D-WA) questioned where he would draw the line and new drug approvals, citing some criticism from Gottlieb five years ago.

FDA has made significant progress in recent years to ensure that patients in the U.S. have access to new, innovative therapies thanks to new legislative pathways like Breakthrough Therapy designation. The adoption of the Breakthrough pathway addressed many of the concerns I raised in that 2012 article. I believe we should continue to look for ways to improve the efficiency of FDA’s medical product review program, modernize the scientific standards used in drug regulation, and seek more uniform adoption of pathways created by Congress like the Breakthrough Designation, and build on these opportunities through adoption of the new provisions in Congress in 21st Century Cures. We need to do all these things while continuing to ensure that new products meet FDA’s Gold Standard for safety and efficacy.

In another response, he noted:

Maintaining the Gold Standard of safety and efficacy for medical products is fundamental to FDA’s mission to protect and promote public health.

Gottlieb also repeated that his often cited business ties to companies like GlaxoSmithKline, which had paid him $60,000 a year plus travel expenses for his advice, Vertex, Celgene and others were already or about to be severed, vowing to seek counsel from ethics advisers after recusing himself from any decisions about his former clients and business investments.

Gottlieb is digging in for the duration.


Image: Commissioner-designate Scott Gottlieb at his nomination hearing in early April Zach Gibson/Getty Images


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