As of October 2016, the FDA’s guidance on patient preference information in device submissions[1], came into effect. Up to this date, patient-reported data have more commonly been collected and reported in association with pharmaceutical submissions; especially since the FDA released its Guidance for Industry on Patient-Reported Outcome (PRO) Measures in 2009[2].

What does the FDA Patient Preference Guidance mean for device companies?

Although the FDA made reference to including patient preference data in submissions for device products in their 2012 Guidance on Benefit-Risk Determinations in Medical Device Premarket Approval[3], the new guidance moves from theoretical proposition to practical implementation. This guides how FDA staff may use patient preference information in their decision making for medical device applications.

The guideline recognises that the opinions of medical device users, i.e. patients and their care partners, can provide additional insight on the benefits and risks of those devices. This may be especially true for devices that:

  • Are used directly by patients
  • Directly intend to affect health related quality of life
  • Are potentially high-risk devices or involve high risk procedures
  • Address an unmet need or particularly rare condition
  • Offer alternative benefits to those already available
  • Involve novel technologies.

What is patient preference data and why is it important?

The FDA define patient preference information as: “qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions”.

In addition to patient preference, ‘patient perspective’ refers to a specific type of patient input, and includes information relating to patients’ experiences with a disease or condition and its management. Although they reiterate that submission of patient data is voluntary, they specifically suggest that it can be very useful for evaluating a benefit-risk profile when patient decisions are ‘preference-sensitive’, for example:

  • Where multiple treatment options exist, with no clear superior option for all patients;
  • Where supporting evidence for one treatment option over others is not definitive; and/or
  • When there is considerable variation in the views of risk/benefits adopted by patients or health care professionals.

Patient preference and outcomes data is also important after the initial evaluation of a medical device. The guidance highlights that patient input regarding their condition and its management may be useful throughout the total product lifecycle for certain devices; for example, by improving understanding the impact a disease or condition exerts on patient life, defining design inputs to meet patient needs, assessing the impact of treatments or unmet needs from the patient perspective, assessing outcomes most important to patients, priorities for disease management, plus more. This highlights the potential benefits that device companies stand to gain when patient preference and outcomes data are utilised.

What about caregivers and healthcare professionals?

Patients are not the only stakeholders in the development and use of medical devices. The FDA guidance also suggests that preference data from care-partners (e.g., parents) and healthcare professionals is acceptable. These preference assessments should focus on the patient’s willingness and reluctance to accept the risks associated with the device, both of which are informative to the FDA’s assessment of the benefit-risk profile of the device.

So what information should device companies collect?

The FDA appear open to the type of data that can support patient preference, suggesting that qualitative and quantitative data can be utilised to demonstrate patient preference. For example, in the context of benefit-risk assessments, qualitative data may be useful in identifying which outcomes, endpoints or other attributes are primarily valued by patients, and what factors affect patients’ perspectives on risk and benefit. However, quantitative preference data can help estimate how different outcomes, endpoints or other attributes are valued by patients, and also the trade‑offs that patients are willing to make.

At DRG Abacus, our COA experts can advise and assist on the collection of qualitative and quantitative patient data for any type of product, either drug or device related. Feel free to contact us if you feel we could be of assistance at Access@TeamDRG.com.

[1] Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders: Patient Preference Information – Voluntary Submission, Review in Premarket Approval Applications, Humanitarian Device Exemption Applications, and De Novo Requests, and Inclusion in Decision Summaries and Device Labeling. October 2016. Available from: http://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm446680.pdf

[2] Guidance for Industry. Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. December 2009. Available from: http://www.fda.gov/downloads/drugs/guidances/ucm193282.pdf

[3] Guidance for Industry and Food and Drug Administration Staff; Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications issued on March 28, 2012. Available from: http://www.fda.gov/ucm/groups/fdagov-public/@fdagov-meddev-gen/documents/document/ucm517504.pdf

How Glympse Bio oversubscribed their Series B funding amidst the pandemic

View Now