Explaining Unapproved Drugs to Payors: FDA Guidance Helps Pharma Avoid Pitfalls
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The FDA has issued new guidance on how drug manufacturers can communicate information about unapproved drugs to payors, aiming to reduce legal risks. This article explains the key provisions, including what can and cannot be said about study results, and offers strategic insights for pharma teams.
FDA’s guidance on drug and device manufacturer communications with payors sets practical boundaries for sharing health care economic information and data on unapproved products or uses with formulary committees that have economic analysis expertise.
Contents8 sections
Key Takeaways
- FDA’s final Q&A guidance covers HCEI and communications to payors about unapproved products or unapproved uses of approved products.
- Firms should state clearly that safety and effectiveness are not established for unapproved products or uses and disclose development stage.
- Study presentations to payors should include material design limitations and avoid selective reporting of only positive results.
- Separate 2025 SIUU guidance addresses scientific information on unapproved uses shared with health care providers, a related but distinct audience.
What does FDA’s payor communications guidance cover?
The agency’s document Drug and Device Manufacturer Communications With Payors, Formulary Committees, and Similar Entities – Questions and Answers answers common questions about firms’ communications of health care economic information (HCEI) to sophisticated payor audiences.
It also addresses dissemination to payors of information about medical products not yet approved or cleared for any use, and information about unapproved uses of approved or cleared products.
The PDF guidance text on fda.gov/media/133620 recommends that communications remain truthful, non-misleading, and paired with specific disclosures.
What must companies disclose about unapproved products?
When communicating about unapproved products or unapproved uses, FDA recommends:
- A clear statement that the product or use is not approved/cleared/licensed and that safety or effectiveness has not been established
- Information on development stage, study status, and whether a marketing application has been submitted or is planned
- For study-result presentations, material aspects of design and methodology plus material limitations, without selective positive-only reporting
For unapproved uses of already approved products, firms should also disclose approved indications and provide current FDA-required labeling.
FDA further suggests follow-up if previously shared information becomes materially outdated — for example, after a failed primary endpoint or a clinical hold.
How does this relate to SIUU communications with clinicians?
Payor guidance is not the same as FDA’s January 2025 final guidance on communications of scientific information on unapproved uses (SIUU) to health care providers.
OPDP lists both documents among current promotional policy materials on its regulatory information page. Teams should map audience first: formulary economists versus treating clinicians.
Using payor HCEI slides with HCP audiences, or SIUU reprints with payors without the recommended disclosures, recreates the enforcement risk the guidances try to cabin.
Medical affairs and market access should keep separate approved slide libraries, with version control that records which disclosures accompanied each payor meeting. That operational discipline matters more than slogan-level “FDA allows payor talk” interpretations.
When a pivotal trial fails after payor discussions, FDA’s suggestion to provide follow-up information becomes a practical recall problem: who received the prior deck, and how quickly can the company push a correction?
What remains a compliance judgment call?
Guidance describes FDA’s current thinking; it is not a statute. Characterizations that imply superiority, predictive clinical benefit, or approved status for unapproved uses remain high risk even when an HCEI deck is otherwise technical.
Companies should still involve medical, legal, and regulatory review before payor meetings on Phase 2 assets or supplemental indications.
Nothing in the payor Q&A converts an unapproved product into a reimbursable covered drug. Coverage decisions still depend on FDA labeling after approval, compendia, and payer medical policy — communications guidance only reduces one category of promotional uncertainty.
Pharma pricing and HEOR teams should treat payor meetings on investigational assets as structured scientific exchange with an audit trail, including attendees, materials version, and follow-up obligations if trial status changes in 2026 or later.
Where companies previously relied on verbal “safe harbor” folklore, the written Q&A now supplies a checklist that compliance officers can map to training decks and field-force monitoring scripts.
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Frequently Asked Questions
What is FDA’s payor communications guidance about?
It is a Q&A guidance explaining how manufacturers may share health care economic information and certain data on unapproved products or uses with payors and formulary committees that have economic analysis expertise.
What disclosures does FDA recommend for unapproved products?
Firms should state that the product or use is not approved and that safety and effectiveness are not established, describe development stage, and present study results with material limitations and without selective reporting.
Is payor guidance the same as SIUU guidance for clinicians?
No. SIUU guidance addresses scientific information on unapproved uses shared with health care providers. Payor guidance targets formulary and similar economic decision-makers.
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