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Pink Sheet – Gottlieb Q&A: Off-Label Communications And Right-To-Try

Pink Sheet – Gottlieb Q&A: Off-Label Communications And Right-To-Try

Executive Summary

US FDA Commissioner nominee favors making up-to-date, accurate information available to patients and providers, but will that translate to more freedom for sponsor communications?

Scott Gottlieb admitted to not fully briefed on the US FDA’s off-label communications issues, but seems interested in policy tilting toward more information disclosure on drug uses.


The nominee for FDA Commissioner indicated in answers to written questions submitted by members of the Senate Health, Education, Labor and Pensions Committee that while it is important that drug labels have scientifically-based and accurate information, patients and providers also deserve as much data as possible when making decisions.


“It is crucial that manufacturers continue to develop and submit to the agency clinical data demonstrating the safety and efficacy of medical products for new indications they seek to include on labeling and in their marketing communications with patients, payers, and providers,” Gottlieb wrote in an answer to an off-label communication question from HELP Committee Ranking Member Patty Murray (D-Wash.). She asked whether Gottlieb would “preserve physician and public trust in approved medical products and the important incentive of FDA approval.”


Editor’s note: This story is part of an ongoing series examining Scott Gottlieb’s responses to Senators on a variety of FDA issues. The first story is here: (Also see “Gottlieb Q&A: Sen. Murray Wants To Know About Generic Labeling Rule” – Pink Sheet, 30 Apr, 2017.).


“I also believe that patients and physicians make the best decisions when they have access to as much truthful, non-misleading, scientifically based information as possible,” Gottlieb said.


The Friends of Cancer Research has raised the idea of finding a more streamlined way of adding postmarketing data to drug labels, which could help deal with the off-label communications problem. (Also see “Label Expansion: Could Clinical Experience Data Find Supplement Work-Around?” – Pink Sheet, 20 Apr, 2017.)

Getting Up To Speed

Gottlieb was careful in his answer to recognize FDA’s position on the issue and not signal a radical shift.


In a way, it encapsulates FDA’s dilemma with off-label communication. The agency is interested in ensuring providers and patient can access the latest information on prescription drugs. But in doing that it also does not want to destroy the incentive for sponsors to conduct clinical trials and submit applications to add indications.

Sen. Elizabeth Warren (D-Mass.) indicated in a written question that she was concerned Gottlieb would further loosen off-label promotion restrictions. He gave largely the same answer provided to Murray.


“FDA has consistently acknowledged that there is some public health benefit of allowing certain non-promotional communication about truthful, non-misleading, clinical data that is not already incorporated into FDA-approved product labeling,” Gottlieb said. “FDA has carved out certain ‘safe harbors’ for such communications.”


Gottlieb also said in the answer to Murray’s question that he intended to work with FDA staff “to get up to speed on the agency’s latest thinking and actions on these matters,” as well as provide “clarity to manufacturers, payers, providers, and patients about acceptable truthful and non- misleading communications related to clinical data not already incorporated in a label.”


FDA released draft guidances earlier this year on communication of health care economic information to payers and similar entities (Also see “Industry Communications With Payors: US FDA Okays Info On Investigational Drugs” – Pink Sheet, 19 Jan, 2017.), as well as when communication about a product is considered consistent with approved labeling. (Also see “Off-Label Communication: When Does It Align With Approved Labeling?” – Pink Sheet, 17 Jan, 2017.)


The agency also outlined its position on First Amendment considerations in relation to off-label communications, where it said in some cases off-label communication can advance public health, but did not give its solution to the problem. (Also see “First Amendment Does Not Block Off-Label Enforcement Actions, FDA Reiterates” – Pink Sheet, 22 Jan, 2017.)


The guidances and memo were part of a flurry of documents published in the final days of the Obama Administration. (Also see “FDA’s Document Dump: Guidance Release Skyrockets Ahead Of Trump’s Arrival” – Pink Sheet, 22 Jan, 2017.)

Will Off-Label Experience Influence Gottlieb?

Gottlieb’s personal experience with off-label drug use may suggest he would favor allowing more off-label information to be made available than less.


In another answer to a question from Murray, Gottlieb wrote that while fighting cancer, he used a “commercially available combination therapy in an off-label manner.”


The comment came in the context of right-to-try legislation. Murray said in the question that she has concerns about the issue and asked for his perspective.


Gottlieb said he understood “at a very personal level that patients who are fighting serious or life-threatening diseases want the flexibility to try new therapeutic approaches, including access to investigational medical products, particularly when there is no other FDA-approved treatment option.”


But he also said that “the clinical trial process is crucial to the development of innovative new medical products.”


“If confirmed, I would commit to ensuring FDA has the right policies and processes in place to appropriately balance individual patients’ needs for access to investigational therapies while recognizing the importance of maintaining a rigorous clinical trial paradigm for testing investigational products and demonstrating safety and efficacy,” Gottlieb wrote.


Right-to-try laws have gained attention in state legislatures and Congress as patients with life-threatening conditions with no other options have fought for access to potential treatments still under development. (See sidebar for related story.)

Off-Label Not Addressed At Hearing

Off-label communication was one of the few major FDA topics that was not broached during Gottlieb’s Senate confirmation hearing. Senators did question him on subjects such as the opioid epidemic (Also see “Opioid Policy At US FDA: Gottlieb Seeks More Activist Role To Combat Abuse” – Pink Sheet, 6 Apr, 2017.), agency efficiency (Also see “Gottlieb Promotes ‘Bottom-Up’ Review To Increase FDA Efficiency, Consistency” – Pink Sheet, 6 Apr, 2017.), and seafood consumption. (Also see “FDA’s Commissioner Fish Story Continues” – Pink Sheet, 18 Apr, 2017.)


Many Senators’ questions also dealt with drug development issues. (Also see “Drug Development, Not Conflicts, Dominates Gottlieb’s Confirmation Hearing” – Pink Sheet, 10 Apr, 2017.)


Ultimately, the HELP Committee voted to send Gottlieb to the Senate floor for confirmation with only two Democratic votes. (Also see “Gottlieb Advances, But FDA’s Future Seems Increasingly Partisan” – Pink Sheet, 27 Apr, 2017.)…