Patient preference information: Stakeholders seek clarity on terms, postmarket usage

Stakeholders are asking the US Food and Drug Administration (FDA) to clarify its recently published draft guidance on using voluntary patient preference information (PPI) for premarket submissions. Commenters asked for several changes, including emphasizing that the guidance applies to devices and device-led combination products, using PPI throughout the product lifecycle, and clarifying terms such as care-partner.

FDA recently published an updated PPI draft guidance that it developed based on its Medical Device User Fee Amendments (MDUFA V) agreement and on experience with a growing number of industry-sponsored PPI studies, better understanding of PPI trial designs, and other factors. (RELATED: FDA updates patient preference guidance to span the product life cycle, Regulatory Focus 5 September 2024)

Nineteen comments, including comments from the medtech lobby group AdvaMed, were submitted to the docket. The group suggested changes, including making it clear that the guidance should also apply to device-led combination products and adding a glossary of key terms, especially for sponsors who are not familiar with patient preference studies.

AdvaMed also recommended that a section on the potential limitations of patient preference studies and how to address them be added to the guidance. The group further recommended addressing issues such as the subjectivity of the qualitative data, ensuring the heterogenicity of the patient population, and cognitive biases when developing patient preferences.

AdvaMed also emphasized more clarity when defining “preference sensitive.” The group noted that the guidance should clearly state that while one treatment may be superior on average within a patient population, there may be subpopulations that benefit more from a different treatment.

“When [the] Medical Device Innovation Consortium (MDIC) developed the definition of ‘preference sensitive,’ which is also referenced in the 2016 FDA [patient preference information (PPI)] final guidance, the intent behind ‘uncertain or variable’ evidence was not variability between different patient populations but uncertainty within a population,” said the group. “Within a population, while a treatment may be superior on average, the uncertainty can result in a chance that it is not superior on one or several attributes, hence requiring an application of PPI within the population – making such a problem preference sensitive.”

AdvaMed asked that when providing preference sensitive examples in evaluating patient decisions, the guidance should also include an example where the benefits of the treatment may not clearly outweigh the risks but there is still a great unmet need for it.

AdvaMed and the drug maker Pfizer both recommended changing the guidance's title to explicitly state that it applies to devices.

"We recommend revising the title to be 'Incorporating Voluntary Patient Preference Information over the Total Product Life Cycle for Medical Devices' or 'Incorporating Voluntary Patient Preference information over the Total Product Lifecycle for Medical Technologies,'" said Pfizer. "The draft guidance focuses on patient preference information as it relates to medical devices; however, this is not immediately clear from the title."

AdvaMed and Pfizer also addressed the use of PPI during the lifecycle of the device. AdvaMed asked that the guidance clarify PPI will be applicable in the post-approval phase of the product as new adverse events are observed and the risk-benefit profile becomes clearer over time.

Similarly, Pfizer noted that the guidance states that knowledge gleaned from the PPI can be used across the total product lifecycle (TPLC); a perspective that it says is consistent with the agency’s benefit-risk assessment of products over their lifecycle.

“We recommend that the Agency reference the lifecycle approach to benefit-risk assessment as one reason for considering PPI across the total product lifecycle and cite the 2023 Final guidance on Benefit-Risk Assessment from FDA and the forthcoming consensus guidance from [the Council for International Organizations of Medical Sciences working group (CIOMS XII)].”

Pfizer also recommended FDA include a section in the guidance that specifically addresses how to analyze patient preference data. In particular, it wanted guidance to address issues such as model specification, model goodness of fit, internal consistency/validity, data quality assessments, and pre-specification of analysis sets and software.

AdvaMed and the drugmaker AbbVie addressed how patients and their healthcare providers or care-partners may have differing views on the effectiveness of a device. AbbVie said that the proposed guidance does not elaborate on how to reconcile such differences. It noted that the guidance only states such differences should be taken into account during premarket reviews.

“We recommend that FDA identify the factors a sponsor should take into consideration when evaluating preferences which may be in conflict particularly in situations where caregivers are in a decision-making role (e.g. conditions that affect younger individuals or individuals with cognitive impairment) impairment,” said AbbVie. “We also recommend that FDA clarify how these factors will be taken into consideration during FDA’s review.”

AdvaMed took issue with the term "care-partner" and argued that caregiver is a more commonly used term. The group provided definitions and examples of the terms caregiver and care-partner.

"However, if FDA thinks 'care-partner' is different than 'caregiver' and is the more appropriate term, we suggest defining the term and using it consistently throughout the document," said AdvaMed. "Further, due to this topic being referenced throughout the document, consider adding more context or resources about care-partner or healthcare professional preferences."

Patient Focused Medicine Development also commented on the guidance and asked for more clarity in several areas. It noted that while FDA asks for patient engagement (PE) early on in PPI studies, it said PE should be emphasized in the concept, design, generation and use of PPI research.

"PE can be informative starting at the inception/ ideation of device development to help identify unmet patient needs," said the group. "We encourage the agency to specify further that good PPI research requires the integration of PE throughout the full spectrum of the PPI study to produce robust and meaningful results."

"PE supports patient preference studies (PPS), and all other methodologies used to collect [patient engagement data (PED)], to ensure that what is most important and meaningful to the patient community is measured and that data is contextualized," the group added. "Consistent and regular monitoring and evaluation of patient engagement in PPS is needed to ensure continuous feedback and improvement on the patient engagement process, outcomes, and impacts."

PFMD also noted that it has helped develop tools for integrating patient engagement throughout the PED research phases.

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Patient preference information: Stakeholders seek clarity on terms, postmarket usage

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