As the U.S. Food and Drug Administration (FDA) increasingly focuses on patient engagement in its product design and premarket review processes, a recent FDA guidance1 document outlines how device companies can utilize patient preference information (PPI) in premarket submissions for inclusion in device labeling. The guidance is limited in scope to companies submitting premarket approval applications, humanitarian device applications (for conditions or diseases impacting fewer than 4,000 patients), or de novo requests, the latter of which often involves new technologies not previously classified by the agency.
The FDA says PPI might be useful for devices that have direct patient interfaces; are intended to directly affect health-related quality of life; are lifesaving but high-risk; are designed to fill an unmet medical need or treat a rare condition; or include novel technology. The FDA recommends that device companies submitting PPI for review also describe PPI in device labeling to support a complete picture of the benefit-risk profile for patients and HCPs making treatment decisions. In the context of presenting PPI to patients, the FDA also offers recommendations for device companies about how to generally approach patient labeling and promotional materials.
Patient Preference Matters
In issuing guidance for industry on the premarket submission of PPI to the FDA, there is a clear signal from the FDA that patient preference matters, particularly when treatment option decisions for patients are “preference sensitive.” This was a welcome signal for industry from an agency that just last year issued a warning letter to a prescription drug company for, among other things, its patient preference data regarding capsule vs. liquid treatment options.2 The FDA’s device guidance identifies three scenarios when treatment decisions are preference sensitive: 1) When multiple options exist and no treatment option is clearly superior for all patients; 2) When evidence supporting one option vs. others is uncertain or variable; and 3) When patients’ or healthcare professionals’ views differ on the most important benefits or acceptable risks.
In its guidance, the FDA says PPI can help FDA better identify a device’s risks and benefits from the patient’s perspective, understand how patients think about a technology’s benefits and risks, and assess whether preferences vary among subgroups of patients. As patient advocacy groups have become more and more vocal about the importance of the patient voice in benefit-risk calculations, especially where prognoses may warrant elevated risk tolerances among patients, the FDA’s device guidance is a step in the right direction for more effective device labeling and, perhaps, quicker access to new technologies. Although currently limited in scope to devices submitted for review under the FDA’s premarket approval, humanitarian device, or de novo review mechanisms, the FDA highlights how preference data can be useful to companies in the design, nonclinical, and presubmission stages of new product or next generation product development as well. Patient perspectives are only likely to grow in utilization and importance within the industry as a result.
Considerations When Obtaining Patient Preference
The instrument used to collect PPI is integral to how meaningful the FDA may regard those data and, essentially, may control whether those data can be promoted post-market. In gauging a patient’s risk tolerance, some of the factors the FDA’s guidance recommends device companies consider in the design of patient preference studies and collection of PPI include:
- Examining the characteristics of the patient population, including disease stage, age, gender, race, ethnicity, socioeconomic status, personal values, family circumstances, and availability of treatment options.
- Ensuring a sample size that is representative of the patient population and large enough that results can be generalized.
- Assessing the tradeoffs patients are both willing and unwilling to take regarding measures of effectiveness, safety, means of use, duration of benefits, and frequency of application.
- Keeping the central focus of the preference study on the patient rather than the HCP.
- Presenting numbers and communicating benefit-risk information so that patients participating in the study can easily process and understand the probabilities of outcomes and risks (e.g., avoid verbal descriptors that patients may interpret differently such as “low” or “high,” avoid fractions and decimals, use multiple formats to present the same information in order to increase comprehension, present positive and negative percentages for the same information, such as “20% chance of adverse events” alongside “80% chance of no adverse events”).
- Testing information presentations pre-study to improve the presentation format used in the study.
- Removing cognitive biases in the study design (such as describing changes as gains or losses).
- Ensuring statistical modeling and sensitivity analyses for reliable and interpretable data.
Promoting Patient Preference Data
In reviewing device premarket submissions where PPI is included, the FDA indicates that its publicly posted decision summaries typically will include a summary of the PPI that the FDA reviewed when deciding whether to approve or allow marketing of a device. Beyond inclusion in decision summaries, the FDA recommends that PPI be included in device labeling to assist patients and healthcare professionals in making treatment decisions and understanding benefit-risk tradeoffs. For device companies, the inclusion of PPI in device summaries and labeling establishes authoritative reference points for those data in support of any future marketing use for the PPI.
While PPI studies may pose challenges for industry in the premarket phase to ensure study questionnaires or other instruments objectively elicit the information they are designed to poll, the opportunity to leverage those data post-market to more effectively communicate with patients and providers could have significant benefits for companies. As the FDA notes in its guidance document, post-market decision-making tools and patient-directed communications that include PPI can help inform treatment decisions, signaling to industry the ability to develop promotional materials around PPI.
Finally, the FDA notes that post-market PPI monitoring can inform the development and design of future products. The challenge of designing a PPI instrument may have promotional rewards not only in shaping patient- or doctor-directed communication tools, but also in keeping the company in tune with the patient voice and at the cutting edge of where product development needs to be moving for patients’ preferences and needs.
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