The PIE Act – A Win for Patients, Payors, and Sponsors

A win for patient access!   Prescription drug and medical device Pre-Approval Information Exchange (PIE) now has specific legal protection allowing for sponsors to proactively communicate to payors certain information about products in development to help expedite patient access upon product approval.

The PIE Act, otherwise known as section 3630, “Facilitating Exchange of Product Information Prior to Approval” of H.R. 2617, the “Consolidated Appropriations Act, 2023,” was signed into law by President Biden on December 29.   The provision amends the Federal Food, Drug, and Cosmetic Act section 502 to provide explicit protection for conveying certain information about products in development to payors, including unapproved uses of approved products.

While pre-approval payor communications are not entirely new, the legal protection now afforded to them will help (hopefully) expedite the creation and reviews of such communications that, have to date, been often fraught with internal company debate.  As background, 21 C.F.R. §312.7 explicitly prohibits the “promotion” of investigational drugs:

A sponsor or investigator, or any person acting on behalf of a sponsor or investigator, shall not represent in a promotional context that an investigational new drug is safe or effective for the purposes for which it is under investigation or otherwise promote the drug.  This provision is not intended to restrict full exchange of scientific information concerning the drug, including dissemination of scientific findings in scientific or lay media.  Rather, its intent is to restrict promotional claims of safety or effectiveness of the drug for a use for which it is under investigation and to preclude commercialization of the drug before it is approved for commercial distribution.

While the focus of the regulation seems to be a sponsor’s suggestion that its investigational drug is safe or effective, the language of the regulation more broadly states that a sponsor shall not “otherwise promote the drug” and that its intent is “to preclude commercialization of the drug before it is approved.”  Industry has grappled with interpreting this regulation – what is promotion? what is commercialization? We know that, in the context of payor communications, the Office of Prescription Drug Promotion (and before it, the Division of Drug Marketing, Advertising and Communications – DDMAC) has long considered the provision of healthcare economic information about drugs to payors to be “promotional,” requiring this content to be submitted to FDA on Form 2253 as promotional labeling.

When FDA published its draft guidance on payor communications in 2017, it seemed revolutionary – introducing the concept of pre-approval information exchange for wholly unapproved products. Upon finalization of the guidance a year and a half later in June 2018 (which gave those who follow ad/promo guidance documents whiplash given the number of still-in-draft guidances) FDA expanded its enforcement discretion to include communications about unapproved uses of approved products.  Per the final guidance, sponsors could provide payors with information about unapproved products and unapproved uses of approved products including information about indications sought, descriptions of clinical studies, anticipated timeline for possible FDA approval/clearance, product pricing, patient utilization projections, and product related programs or services.  FDA recommended that communications include a clear statement that the product or use is not FDA-approved and that safety or effectiveness of the product or use has not been established.

As we noted back in our 2018 blogpost , the expanded scope of the final guidance – covering unapproved uses of approved products – was significant, as these discussions are tantamount to off-label discussions and discussions with Medicare and Medicaid providers could raise False Claims Act questions.  While helpful to have an FDA guidance, there was nothing specifically in the law or regulations that would otherwise protect these communications (other than the First Amendment which, let’s face it, is always a bit risky when litigating).  Reconciling the guidance with 21 C.F.R. §312.7’s prohibition on promoting investigational drugs was difficult.  We had stated, at that time, that “[r]egardless of these new, more permissive guidelines, industry should be careful of suggesting an unapproved use for an approved product.”  FDA had put industry in a quandary.

The PIE Act largely adopts the language from FDA’s guidance and provides the legal protection for these communications desperately sought by sponsors, payors, and patients.  Until now, since the publication of the final guidance, we have witnessed internal company struggles regarding whether and how to provide this important information to payors.  We are hopeful that this critical information will facilitate earlier patient access to needed treatments once they are approved.