Industry groups seek clarity, flexibility in manufacturer-payor communications guidance
Industry and other stakeholders want the US Food and Drug Administration (FDA) to provide more clarity and ensure flexibility in a revised guidance on how drug and medical device makers should communicate with payors and other stakeholders about their products.
In early June, FDA published the proposed updated guidance, which addresses common questions about health care economic information (HCEI) that companies are able to share with payors, formulary committees, and similar entities conducting healthcare economic analysis of their approved and cleared products and products before they've been approved or cleared by the agency. Several stakeholders have provided feedback on the changes, including drug, device and regulatory groups. (RELATED: FDA updates manufacturer communications guidance to emphasize application to devices, Regulatory Focus 3 June 2026)
AdvaMed
The device lobby group AdvaMed asked FDA to clarify the scope of the guidance and ensure it accurately reflects device-specific data generation and terminologies. The group said that while the guidance states it is intended to cover communications between healthcare product manufacturers and payors, technology assessment committees, and similar entities, it wants it to be clear that other audiences are not covered in the document. It notes that the guidance refers to other guidance that may be relevant to how companies communicate, such as guidances on off-label and unapproved uses of products, which may create confusion.
"Such scientific exchange and related communications with other audiences are recognized and appropriate and all efforts should be made so as not to infer this guidance is applicable, particularly in light of the broad definition of HCEI," said AdvaMed. "While we believe that it is FDA’s intent, we want to reinforce the importance of distinguishing from other audiences for purposes of this policy—all for whom scientific discourse is not only important, but in the best interests of patient care and the overall public health."
AdvaMed noted that it is important for FDA to differentiate the HCEI information covered in the guidance from payor-directed economic communication, which has a separate statutory framework, and proposed language to make that clearer. The group said such information is particularly important because manufacturers frequently engage with payors to discuss a range of topics.
AdvaMed proposed the guidance add language that when HCEI is being evaluated with indirect treatment comparisons, FDA will also consider information such as generally accepted scientific standards applicable to device-generated real-world evidence, device and procedure registries, analytical performance and accuracy studies, evidence derived from predecessor technologies, patient-reported outcomes, adherence and persistence analyses, workflow and healthcare resource utilization assessments, and other scientifically valid methodologies that are commonly used to assess product performance and value. The group said the language currently used in the guidance focusing on competent and reliable scientific evidence (CARSE) is method-agnostic, with examples that tend to focus on drugs. The proposed language is intended to reflect the range of evidence typically used to evaluate medtech products.
"Medical device evaluations frequently rely on real-world data, registries, analytical performance studies, patient-reported outcomes, and evidence from predecessor technologies among others," said AdvaMed. "These evidence types are recognized within FDA's medical device evidentiary framework and are commonly used to support regulatory and payer decision-making.
"Explicit recognition of these methodologies would reduce uncertainty regarding their eligibility under CARSE and reinforce that evidence should be evaluated based on methodological rigor, fitness for purpose, transparency, and relevance to the decision-making context—not the evidence source alone," the group added. "This clarification would promote consistent application of CARSE across product categories while aligning the guidance with FDA's established approach to medical device evidence."
AdvaMed also said that the guidance should state that HCEI information for medical devices may also include material assumptions, data source characteristics, device-specific limitations, uncertainty analyses, and factors that affect the applicability of results across variables such as patient populations, sites of care, and clinical settings. The group emphasized that the information doesn't have to be all-encompassing but rather should provide sufficient context to understand the strengths, limitations, and relevance of the information so that they can be used in population-level healthcare decision-making.
Furthermore, AdvaMed asked that the guidance clarify that medtech comparator products may be predecessor devices, alternative technologies, diagnostic strategies, digital health platforms, procedures, sites of care, clinical pathways, or standard management, rather than specific products. The group said that such comparisons may be more useful in understanding the value of the sponsor's product.
"Unlike pharmaceuticals, medical devices are updated through iterative innovation," said AdvaMed. "For example, a [continuous glucose meter (CGM)] platform may undergo multiple software updates, algorithm improvements, changes in features, sensor generation advances, and expanded indication reviews within a coverage contract period.
"Payors routinely require advance information about planned product enhancements, new indications under development, interoperability capabilities, software upgrades, and expanded patient populations to make multi-year coverage decisions," the group added.
PhRMA
PhRMA wrote to FDA asking it to continue allowing flexibility in the communication sponsors are able to have with payors and similar entities, revise the discussion of investigational versus unapproved to better align with statute, clarify what pre-approval information exchange (PIE) communication formats are permissible, and provide additional recommendations on clinical outcome assessments (CAO). The drug lobby group said it appreciates the examples in the guidance on situations where HCEI analyses may be related to an approved indication because they take into account real-world factors, but asked FDA to include evidence of patient preference in that list of examples.
"Preference evidence can, among other things, assist payors, formulary committees, and similar entities to understand the aspects of treatment and outcomes that are most important to patients, contextualize health impacts that generic measures may not fully capture, identify preference heterogeneity, and support uptake estimates," said the group.
PhRMA said the guidance appears to apply different standards to investigational and unapproved products and their uses and noted there isn't a significant statutory distinction between those different standards. The group explained that Congress uses the terms interchangeably in statute and, therefore, there's no basis in law to treat them differently. It said the guidance should harmonize the terms to align with their statutory treatment.
"In practice, FDA could strengthen the Draft Guidance’s statements regarding unapproved products and uses, for example to state that the Agency 'will not object' to communications related to unapproved products or uses that satisfy the requirements of section 502(gg)," said PhRMA. "Such clarification would more clearly reflect the legal effect of the statute and further Congress’s objective of facilitating appropriate information exchange with payors and similar entities.
"At minimum, PhRMA encourages FDA to clarify that it views the terms 'investigational' and 'unapproved' products and uses as interchangeable for purposes of the Draft Guidance," the group added. "As drafted, it is not entirely clear to what extent FDA intends these terms to carry different substantive meanings or the practical consequences that flow from this distinction."
PhRMA asked FDA to state that PIE communication may be presented in the same way as HCEI communications. The group argued that PIE communications often serve similar planning and budgeting functions as HCEI communications and would be beneficial to manufacturers.
"PhRMA therefore recommends that FDA expressly state that product information may be presented through a variety of formats, including, but not limited to, the communication methods discussed in the HCEI section of the Draft Guidance," said PhRMA. "Providing this clarification would promote consistency across the guidance and help ensure that manufacturers can communicate useful information in formats that are most relevant to payors’ decision-making needs."
PhRMA also listed several recommendations to update the guidance to allow the use of COA information, including recognizing responder definitions and meaningful within-patient change (MWPC) thresholds, adding more COA examples, clarifying how trial COA endpoints that are not in labeling may be treated, updating COA references, and addressing digital health technology (DHT)-derived endpoints.
C-Path
The Critical Path Institute (C-Path) also commented on the revised guidance and said it supports including COA information in HCEI analyses submitted to payors. The regulatory science advocacy group argued that COA data provides important insights into patient experience and treatment and encouraged payors to consider them in their assessments.
C-Path, however, said the guidance could be strengthened by clarifying whether communication consistent with labeling can be used to support HCEI communication to payors. It also recommended referencing another guidance on FDA-required labeling to explain the relationship between such communication and the HCEI communication discussed in the revised guidance and provide contrasting examples of when such communication is not permitted.
On a couple of occasions, C-Path recommended that the FDA include a reference to FDA’s patient-focused drug development guidance because it includes the definition of COA. The group also notes that the guidance clarifies that there are four types of COAs: patient-reported outcomes, clinician-reported outcomes, observer-reported outcomes, and performance outcomes. It said the guidance provides valuable characteristics of valid and reliable assessments of COAs.
