The document is in the form of a Q&A and the FDA will accept comment for 60 days, until about March 15, 2017. The FDA PDF is here. The matching comment website is here.
Outgoing Commissioner's Califf's remarks on the document release, one of his last acts in office, is here.
A blog by Amy Dow of Epstein Becker Green is here at NLR; one at RAPS here.
For a detailed blow-by-blow analysis by Tufts policy guru Peter Neumann, see Health Affairs, February 2017, here.
In Amy Dow's brief summary:
FDA states that it will not object to manufacturers providing payors with “unbiased, factual, accurate and non-misleading” information regarding investigational drugs and medical devices, provided that those communications include a clear statement of the investigational status of the product and that its safety and effectiveness have not been established, along with information regarding the stage of product development of the product.
Information that may be provided by manufacturers in accordance with FDA’s recommendations in the draft guidance includes information about the product such as its drug class or design, the indication sought and the patient population under investigation, a factual presentation of the results of clinical and pre-clinical studies without any conclusions regarding the product’s safety and effectiveness, the anticipated timeline for FDA approval, product pricing information, and anticipated marketing strategies and product-related programs and services, such as patient assistance programs.
More details and links after the break.
Payer Communications Task Force (PCTF)
The FDA recently established a "Payer Communication Task Force" and wants to build relationships with payers that go further than its existing Parallel Review program, used a few times for CMS NCDs. The "PCTF" website is here.
November 2016 Two Day Meeting on Payer Communications
The draft guidance follows a two-day meeting on the topic (November 9-10, 2016; see a Reed-Smith blog summarizes that, here; the NLR blog on the topic, here; the MWE blog here; and an earlier blog on the topic, here.)
The FDA's webpage for the November meeting is here and includes archived webcasts and transcripts. The meeting agenda here.
How 21st Century Cures Updated Prior Guidance at FDAMA 1997 Section 114
The 1997 law gave some authority for pharma-payer communications, but the statute itself was brief and the FDA hadn't issued good regulatory or white paper guidance on implementation.
- Section 114 (1997) had defined healthcare economic data in a few words, the new law Section 3037 (2016) uses a lot of words and open-ended phrases.
- Any analysis (including the clinical data, inputs, clinical or other assumptions, methods, results, and other components underlying or comprising the analysis) that identifies, measures, or describes the economic consequences, which may be based on the separate or aggregated clinical consequences of the represented health outcomes, of the use of a drug.
- Such analysis may be comparative to the use of another drug, to another health care intervention, or to no intervention.
- Section 114 had required info to be "directly related" to on label indications; the new law Section 3037 requires the info be only "related" to on label indications.
- The new law requires a disclaimer about what new economic/clinical information is beyond the label; this is a cup half full because it clearly allows info that is not on-label.
- Section 114 was for "formulary committees or similar entity," while Section 3037 is for "drug information centers, technology assessment panels, pharmacy benefit managers, and other multidisciplinary entities that review scientific and technology assessments to make drug selection, formulary management, and/or coverage and reimbursement decisions on a population basis for health care organizations."
- Comparisons based on indirect studies with competing drugs are allowed, as long as within the framework of competent and reliable evidence.
- The drug approval standard is "substantial evidence from well controlled trials," while the HEOR standard is "competent and reliable scientific evidence."
New Acronym: CARSE
Learn this one: Competent and Reliable Scientific Evidence. In the FDA Q&A on payer communications, CARSE is:
Health care economic information [HCEI] developed using generally accepted scientific standards, appropriate for the information being conveyed, that yield accurate and reliable results. In evaluating whether the amount and type of evidence that forms the basis for a particular communication of HCEI [meets the standards], FDA will consider the merits of existing current good research practices for substantiation developed by authoritative bodies (e.g. ISPOR, PCORI). For example, when evaluating indirect treatment comparisons in the absence of data from head-to-heard controlled clinical trials, FDA may refer to guidelines issued by external expert bodies regarding current rigorous methodologies and best practices for such comparisons, e.g. network meta-analysis.
FDA and First Amendment - Redux
The FDA has also reopened a prior public comment docket on communications and First Amendment rights, which has an 88 day comment period until April 19, here. This one comes with a 63-page memo, download from here. 225 footnotes, and includes discussion of the Amarin and Caronia court cases.
A Different Draft Guidance: "Communications Consistent With Required Labeling."
In another just-released guidance, perhaps of even bigger long term impact, the FDA strikes some positions regarding communications about data that is "consistent" with FDA approved indications but not literally on-label. That guidance is online here. The matching comment website is here.
Despite prior rules that seemed to restrict any product claims prior to approval, the feds have turned a blind eye to some very public pre-approval payer communications. For CMS inpatient DRG add-on payments, CMS requires detailed submissions, which require the manufacturer to make hard-nosed clinical claims of benefit. These usually occur for pending "new" technology before its FDA approval, and these exchanges have occurred for years without a squeak of complaint, and before any formal exemption in FDA guidance. ...Just sayin.
A consolidated listing of the cited blogs is in the cloud, here.