By Ankush Patel, MD, Senior Strategic Planner, Mosaic
PIE Act approval
On December 29, 2022, the Pre-approval Information Exchange (PIE) Act was signed into law as part of the Consolidated Appropriations Act.1,2 This legislation amends the Federal Food, Drug, and Cosmetic Act (FD&C) to formally permit both drug and medical device companies to communicate various types of product information to payers, formulary committees, and other similar entities prior to receiving FDA approval or clearance for the relevant product or indication. Previous FDA guidance on PIE was communicated in June 20183; however, the new legislation offers further clarity and provides legal protections to drug and device manufacturers about ways they can responsibly structure such communications. Earlier preapproval communication between pharmaceutical manufacturers and healthcare decision-makers will further expedite payer coverage decisions and lead to improved patient access to new pharmaceuticals and medical devices.
The new legislation largely mirrors the 2018 final FDA guidance3, but provides several key updates:
Manufacturers of investigational drugs and medical devices are now statutorily permitted to engage in preapproval communications with payers, formulary committees, and similar entities1
In addition to drug manufacturers, medical device manufacturers can now explicitly disseminate healthcare economic information about approved or cleared products with payers, whereas previous guidance only expressly included drugs4
The PIE Act adds a new section (gg) to the FDA’s misbranding authority (Section 502 of the FDCA), which specifically exempts from misbranding the exchange of “truthful and not misleading product information” for both investigational drugs and devices, as well as investigational uses of already approved or cleared drugs and devices to approved entities5
Provisions of the PIE Act5,6
See below for specific product information that can be shared, and criteria that must be followed when engaging in preapproval communications:
Product information that can be shared*
- Information describing the product (eg, drug class, device description, features)
- Information about the indication(s) being investigated
- Anticipated timeline for possible approval, clearance, marketing authorization, or licensure of the investigational product or investigational use
- Product pricing information
- Patient utilization projections
- Product-related programs or services
- Factual presentations of study results that neither characterize nor make conclusions regarding safety or efficacy of the investigational product or investigational use
Information shared must meet the following criteria*
- Statement that the investigational drug/device or investigational use of drug/device has not been approved and safety/effectiveness has not been established
- Include information relating to the stage of development of the drug or device
- Where factual presentations of study results are shared, describe all material aspects (ie, study design, methodology, results, limitations). Results cannot be selectively presented
- Where applicable, include statement disclosing indication(s) approved, granted marketing authorization, cleared, or licensed. A copy of the most recent label should also be included
- If previously communicated information becomes outdated, include updated information
- Do not represent that investigational drug or device has been approved, cleared, granted marketing authorization, licensed, or has been determined safe and effective
*This information has been summarized to represent general guidance for informational purposes and does not constitute legal advice. Reference the full PIE Act for more information.
Payer and manufacturer implications of the PIE Act
The PIE Act confirms payers’ ability to conduct rigorous reviews of drugs and devices concurrently with the FDA without sacrificing safety or quality of review. This will continue to allow payers to anticipate a new therapy or indication, and plan for potential expansion of eligible patient populations and budget impacts. Publicly funded payers such as Medicare and Medicaid will benefit from the strengthened ability the PIE act affords to review data and update formularies before a plan year begins, as there is statutorily limited flexibility to make changes outside the formulary submission period.
From a value-based purchasing and contracting agreement standpoint, PIE will allow payers more time to negotiate these agreements before a drug comes to market, which is especially important for new high-cost treatments, such as cell and gene therapies. The approved legislation contains no prohibition against follow-up requests for information after the initial exchange, which means that payers will be able to continue having some level of open communication with manufacturers throughout the preapproval stage and to request follow-up questions to inform coverage and purchasing.
Approval of the PIE Act will likely further empower manufacturers of all sizes, including manufacturers of medical devices, to engage in preapproval communications with legal protections in place. With these protections, manufacturers may take additional steps to change their internal policies and update their regulatory guidance and processes to reflect this new legislation.
Additionally, manufacturers of all sizes are more likely to invest in systems and processes to develop and disseminate PIE materials in a more proactive fashion. The PIE Act approval will also likely have downstream effects on patients by allowing them to receive new therapies and devices more quickly, potentially leading to improved care.
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