The new article emphasizes the important role of patient preference information.
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The Food and Drug Administration (FDA or the Agency), the US regulating authority in the sphere of healthcare products, has published a guidance document dedicated to incorporating voluntary patient preference information over the Total Product Life Cycle (TPLC). The guidance provides a general overview of the applicable regulatory requirements, as well as additional clarifications and recommendations to be taken into consideration by medical device manufacturers and other parties involved in order to ensure compliance.
At the same time, provisions of the guidance are non-binding in their legal nature, nor are they intended to introduce new rules or impose new obligations. The authority explicitly states that an alternative approach could be applied, provided such an approach is in line with the existing regulatory framework and has been agreed with the authority in advance.
Patient Input and Its Role in FDA Device Decision-Making
According to the document, the FDA actively incorporates patient input into its decision-making processes for medical devices. Patients can provide vital insights into their overall health conditions, the natural history of their diseases, and how these conditions impact their quality of life.
Patients’ experiences with treatments, their unmet medical needs, and their perspectives on desired outcomes and disease management strategies can offer essential guidance in evaluating medical devices. These insights allow the FDA to better understand patient priorities and preferences, particularly when patients face treatment options that require personal value-based decisions.
Patient experience data, which can come from sources like literature reviews, healthcare professionals, or care-partners, can be helpful throughout the entire life cycle of a medical device. This data provides essential insights into disease understanding, helps define design inputs, and contributes to assessing outcomes that are most important to patients.
Defining Patient Preference Information (PPI)
According to the guidance, patient preference information (PPI) refers to both qualitative and quantitative evaluations of how patients value different alternatives, outcomes, or attributes of health interventions. Essentially, PPI captures the preferences of patients regarding the benefits and risks associated with specific devices or medical treatments.
PPI differs from patient-reported outcomes (PROs), which are direct reports from patients about their health condition. While PROs measure the patient’s assessment of health status, PPI studies aim to understand the trade-offs patients are willing to make between different treatment options or attributes.
PPI studies are designed to reveal which device attributes are most important to patients, the significance of these attributes, and how willing patients are to accept certain risks in exchange for potential benefits. PPI is sometimes referred to as health-preference assessment, patient-centered research, or health-preference research in scientific literature.
It can be especially useful in providing the FDA with an understanding of the patient’s viewpoint on risk tolerance and preferences in benefit-risk assessments. In addition to patients, the FDA may also consider the preferences of care-partners and healthcare professionals, especially when they are directly relevant to the device’s benefit-risk evaluation.
Why Include Patient Preference Information in FDA Decision-Making?
The inclusion of PPI in FDA decision-making acknowledges that individual patient preferences can vary significantly. Patients may weigh the benefits and risks of a medical device differently from healthcare professionals, family members, or regulators.
Some patients may be willing to accept greater risks for potential benefits, while others may be more risk-averse and require higher benefits before accepting certain risks. Factors such as personal values, disease stage, family circumstances, age, and other demographic characteristics can also influence patient preferences.
In some cases, PPI may help inform FDA decisions regarding device approval for specific subpopulations. If scientific evidence indicates that a significant number of well-informed patients would accept the potential risks in exchange for the benefits, this could support a favorable benefit-risk profile.
Conversely, if the statutory standard is not met for any sub-population, the FDA will not approve the device.
Differentiating Patient Preference Information from Patient-Reported Outcomes
While PPI and patient-reported outcomes (PROs) may seem similar, they serve different purposes in the FDA’s decision-making process. A PRO is a direct report from a patient about their health condition, such as a pain score or a disability index.
These measurements capture the patient’s perceptions of their health status at a specific moment in time but do not convey the patient’s values regarding different outcomes or therapies. PPI, on the other hand, seeks to understand the relative importance of various outcomes or therapies to patients and explores how they compare and trade off between them.
For example, a PPI study might assess whether a patient would prefer a therapy that causes a slight reduction in physical function in exchange for significant pain relief. These studies, which employ quantitative methods, can provide crucial data about patient preferences and tolerance for different device characteristics.
When Should Patient Preference Information Be Considered in Device Decisions?
Although the submission of PPI to the FDA is voluntary, it can be particularly useful for certain types of devices, especially when patient preferences play a central role in treatment decisions. Devices with a direct patient interface, those designed to improve health or appearance, devices that affect the quality of life, high-risk life-saving devices, and those developed to address unmet medical needs or rare conditions could all benefit from PPI studies.
PPI can also assist the FDA in understanding population-level differences in patient perspectives, especially when demographic characteristics, disease stages, or phenotypes vary widely within the target population. For diseases with significant public health impacts, such as those with high mortality or morbidity rates, patient preferences may be crucial in shaping device evaluations.
Examples of PPI Studies Supporting Device Review Decisions
Several PPI studies have played a pivotal role in FDA device reviews. One example is a study sponsored by the FDA’s Center for Devices and Radiological Health (CDRH), which evaluated patient preferences for weight-loss devices.
The study sampled more than 500 obese patients and assessed their risk tolerance levels in relation to different weight-loss outcomes. This study demonstrated that many patients were willing to accept the risks associated with surgically implanted devices if they achieved a significant reduction in weight.
The results provided valuable data that helped inform clinical trial designs and benefit-risk assessments. Another example involves a study conducted to support the expansion of indications for a hemodialysis device.
Initially cleared for home use with a care partner, the manufacturer sought to modify the label to allow for solo home hemodialysis (HHD). A PPI study surveyed 142 patients and revealed that they were willing to accept the increased risks associated with solo HHD in exchange for the benefit of increased treatment accessibility.
This study contributed to the FDA’s decision to clear the device for solo use. Such PPI studies provide valuable insights into the relative importance of device attributes to patients, as well as how patients weigh risks and benefits.
These studies can inform patient-centric decision-making and even shape the design and analysis of clinical trials.
Important Considerations for Designing PPI Studies
As further explained by the authority, when designing a PPI study to address an FDA decision-making question, sponsors should carefully consider several factors. These include defining the scientific question, study objectives, parameters of interest, study design, and study population.
For example, early in the product life cycle, a study may focus on understanding patient preferences for clinical endpoints, while later in the process, it may assess patients’ willingness to accept risks for specific benefits. The choice of study design – whether qualitative or quantitative – depends on the research question, and different patient preference parameters may be needed at various stages of the product life cycle.
For example, if the objective is to support a benefit-risk assessment, both Maximum Acceptable Risk (MAR) and Minimal Acceptable Benefit (MAB) parameters may be necessary. Ultimately, well-designed PPI studies provide invaluable insights into patients’ values and preferences, contributing to more informed, patient-centered regulatory decision-making for medical devices.
Conclusion
In summary, the present draft guidance provides an overview of the key aspects of patient input and its role in FDA decision-making, highlighting the importance of patient preference information and offering examples of how PPI studies have supported regulatory decisions.
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