FDA Guidance On Health Care Economic Data Is Coming In 2015

Agency originally said it would issue a draft guidance on industry’s interactions with formulary committees in 2014, but CDER Director Woodcock says “extremely contentious” issues have slowed progress.

Having failed to meet its year-end 2014 target date, FDA is now eyeing 2015 to issue guidance governing pharmaceutical sponsors’ interactions with formulary committees on health care economic information.

In a Dec. 22 letter to two law firms that represent the Medical Information Working Group (MIWG), Associate Commissioner for Policy Leslie Kux said the agency’s current goal is to issue guidance that addresses health care economic information after it releases guidance “that addresses manufacturer dissemination of information regarding unapproved uses during the first part of 2015.”

This timetable has slipped from the agency’s June 2014 response to an MIWG citizen petition in which FDA announced plans to issue draft guidance about health care economic information by the end of the year (Also see "FDA Promises More Off-label Guidances As Part Of First Amendment Harmonization" - Pink Sheet, 10 Jun, 2014.).

MIWG, a coalition of pharmaceutical companies, first petitioned the agency in 2011 seeking to clarify agency policies and regulations in four areas of communication – scientific exchange, sharing information with formulary committees and payers, providing third-party clinical practice guidelines and responding to unsolicited requests for information (Also see "FDA Pressed To Clarify Permissible Formulary, Clinical Guideline Communications" - Pink Sheet, 5 Jul, 2011.).

In a statement to “The Pink Sheet” DAILY, FDA said: “Our intention is for guidance on manufacturer dissemination of information regarding unapproved uses to address unsolicited requests, distributing scientific and medical information on unapproved new uses, and manufacturer discussions regarding scientific information more generally. The issues above include what is often referred to as ‘scientific exchange’ for unapproved new uses and the distribution of scientific and medical publications on unapproved new uses.”

18 Years In The Making

The issue of interactions with formulary committees and payers has been a longstanding quandary for the industry.

Section 114 of the FDA Modernization Act of 1997 allows manufacturers to provide health care economic information not in product labeling to formulary committees if it is based on “competent and reliable scientific evidence” and pertains directly to an approved indication.

FDA has never issued formal guidance spelling out how the “competent and reliable” standard should be interpreted, and there has been little agency enforcement related to health care economic information. However, industry stakeholders say the agency has applied a more restrictive “substantial evidence” standard in its interpretation of Sec. 114 (Also see "Will FDA’s View On Health Economic Claims Change With More Government-Funded Research?" - Pink Sheet, 27 Feb, 2012.).

Although FDA originally told MIWG it would issue guidance by the end of 2014, Center for Drug Evaluation and Research Director Janet Woodcock told the FDA/CMS Summit Dec. 11 that the document was taking longer to get out than expected because it has been “an extremely contentious issue” internally at FDA and externally.

Industry Presses Its Case

In 2012, the Pharmaceutical Research and Manufacturers of America submitted a white paper that said guidance on health care economic information should allow for use of a wide range of data sources and not be limited only to the types of adequate and well-controlled trials that would satisfy the substantial evidence requirement (Also see "Requiring Clinical Trial Support For Every Sec. 114 Economic Claim “Unwarranted,” PhRMA Tells FDA" - Pink Sheet, 15 Oct, 2012.).

Industry representatives continued to press this point during a Dec. 12 panel discussion at the FDA/CMS Summit.

Adequate and well-controlled trials “practically never happen in health economic studies because they’re open label, they’re post hoc, they’re not randomized, and we have to select which data we’re looking at,” said Sanofi Chief Medical Officer Paul Chew.

In an interview with “The Pink Sheet” DAILY, Chew said clarification on Sec. 114 is critical to ensuring that sponsors can secure prompt product reimbursement and access for patients.

“The number of traditional … clinical trials after approval is being counterbalanced by a surge in health economic analyses, and we need guidance because … we’re working in an atmosphere of no guidance and no [enforcement] letters, but that’s not a really solid basis of going forward,” Chew said.

“We always talk about accelerated/streamlining development. The whole thing is not just to get the approval, it’s to get it into the hands of patients and doctors. And that piece, the post-approval piece, we need to streamline that so that people are not doing that part of the cycle inefficiently and with inappropriate risk.”

The lack of clarity on Sec. 114 is undermining the growing need to focus on the value of new treatments, Bay City Capital Managing Director David Beier said from the audience during the panel discussion.

The absence of FDA guidance is “getting in the way of communicating value stories to people who are making billions of dollars in purchasing decisions. There’s something wrong with that.”

Beier suggested FDA could, in cooperation with industry, draft a safe harbor clarifying the things that “are possible to do, things that are prohibited to do, and things that are a grey area. It may be possible from the context of health care reform to think about what’s the best way to inform sophisticated purchasers about the economic reality of using the product on- and off-label.”

Other Guidances On FDA’s Priority List

Kux’s Dec. 22 letter also states that the agency is reviewing comments received on several recent draft guidances related to First Amendment, advertising and promotional issues.

In January, the agency issued a draft guidance on post-marketing submissions of interactive promotional media (Also see "FDA’s Social Media Guidance Gives Firms A Pass On User-Generated Content" - Pink Sheet, 20 Jan, 2014.).

In June, FDA released a recommended practices guidance on distribution of reprints with new risk information (Also see "Journal Reprints: Risk-Mitigating Info Should Be “At Least As Persuasive” As Existing Sources" - Pink Sheet, 9 Jun, 2014.). The document was drafted quickly in response to stakeholder questions about a revised guidance issued in February on distribution of scientific articles about unapproved uses (Also see "FDA Reprint Policy Expands To Clinical Practice Guidelines, Medical Reference Texts" - Pink Sheet, 1 Mar, 2014.).

Also issued in June were two social media guidances on presentation of risk and benefit information in platforms with character space limitations, and correction of independent third-party misinformation (Also see "FDA’s Twitter Formula Allows Tweets About Products Without Excessive Risks" - Pink Sheet, 17 Jun, 2014.).

Kux said the agency is considering “all the perspectives reflected in the comments” on the draft documents and vowed that the guidances “are one of FDA’s highest priorities in the coming year.”