FDA Issues Draft Guidance Regarding Patient Preference Information for Medical Device SubmissionsMay 17, 2015
In March 2012, FDA issued a guidance document, Factors to Consider when Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications. As we discussed in our blog post on that guidance, one important discussion in that guidance had to do with patient perspective of benefit-risk and how FDA might take that into consideration when deciding whether to approve premarket approval (PMA) applications or de novo submissions. On May 13, 2015, FDA expanded this discussion with a 32-page draft guidance addressing how FDA might consider patient preference information, the types of data and information to include in a submission, and how to incorporate patient preference information into a product’s labeling. See Patient Preference Information – Submission, Review in PMAs, HDE Applications, and De Novo Requests, and Inclusion in Device Labeling. Note that the draft guidance applies only to PMAs, de novo petitions, and humanitarian device exemption (HDE) submissions. It does not apply to 510(k)s, consistent with the Benefit-Risk Guidance.
FDA makes clear in the draft guidance that including patient preference information in a premarket submission is voluntary, but can be useful in the following ways:
1) to help identify the most important benefits and risks of a technology from a patient’s perspective; 2) to assess the relative importance to patients of different attributes of benefit and risk, and clarify how patients think about the tradeoffs of these benefits and risks for a given technology; and 3) to help understand the heterogeneity or distribution of patient preferences regarding benefits and risks of various treatment or diagnostic options.
FDA defines “patient preference information” as “qualitative or quantitative assessments of the relative desirability or acceptability of attributes that differ among alternative diagnostic or therapeutic strategies.” One of the key ways in which this information may assist FDA during the review process is to help reviewers understand circumstances in which certain patient populations may be willing to accept a greater risk for a smaller benefit, for example, if there are no other treatment options available or a sub-group of patients may be more likely to benefit from a device even if the data do not support approval for use in the population as a whole.
The guidance discusses methods to elicit patient preferences. While acknowledging that qualitative data may be sufficient at times, the guidance emphasizes the importance of quantitative data “to ensure that different outcomes are properly weighed in the same scale and therefore can be compared.” It also states that while submission of patient preference information is voluntary, it may be particularly helpful where decisions about device usage are “preference-sensitive.” This may occur, for example, when a patient may have multiple treatment options and none is clearly superior for all preferences, when the evidence supporting one option over others is uncertain or variable, or when patients’ views about the benefits and risks of a device vary considerably within a population.
In order to accept patient preference information provided in a premarket submission, the information must constitute “valid scientific evidence.” FDA will make this determination based on the following study qualities:
- Representativeness of the sample and generalizability of results;
- Capturing heterogeneity of patients’ preferences;
- Established good research practices by recognized professional organizations;
- Patient centeredness;
- Effective communication of benefit, harm, uncertainty, and risk;
- Minimal cognitive bias;
- Logical soundness;
- Robustness of analysis of results;
- Study conduct; and
- Comprehension by study participants.
Like other studies conducted to support a PMA, patient preference studies should be described in the device’s labeling upon approval. The labeling should contain information to help patients understand:
- If they might benefit from use of the device;
- The potential benefits from use of the device;
- The potential risks or complications from use of the device, and the likelihoods of each;
- Any relevant contraindications, warnings, and precautions;
- If they share characteristics with the group of patients who view the benefits as outweighing the risks; and
- Any additional information about what is known and not known about patient outcomes (e.g., long-term outcomes, rare complications).
The information in the draft guidance has the potential to be helpful to sponsors of PMAs, de novo petitions, and HDEs, particularly for sponsors of new or novel devices. While collecting patient preference data of a quality and quantity described in the guidance will pose an additional burden and expense on the sponsor, it may also allow for the possibility of device approval when such approval was previously unlikely.