Benefit-Risk from the Lens of the Patient

Decisions around therapeutic options should be informed by the ultimate expert assessor of benefits and potential harms associated with the product-- the patients themselves-- whether considered alone or in the context of shared decision-making.

Recognizing the patient as the true expert in his or her condition, and in the relevance of information to his/ her healthcare decisions, individual and shared decision-making tools can elicit valuable trade-off information (e.g., oral vs injection); monitoring preferences; one side effect vs another. Meaningful application of tools to assess benefits vs harms also can be used to help patients determine the appropriateness of products available over-the-counter, supplementing the existing drug facts label. In the broader approval context, a patient’s perspective on FDA’s benefit-risk assessment (if already available) for a product is useful to help inform shared decision-making around the product, given the possibility of differences in the individual benefit-risk tradeoffs from a patient perspective as compared with the Sponsor's or FDA's perspective. Patient views on expectations around product features, treatment benefits experienced, and relative acceptability of harms and/or burdens associated with treatment, should be able to be captured in a manner that will be readily accepted by health authorities. Although no health authority recognized patient-level benefit-harm tools, or methods by which to aggregated tradeoff data gleaned at the individual patient level exist, standardized approaches for development of those tools, if not models themselves, should be considered and put forward. This session will review existing examples and potential criteria to support development and use of these tools in a regulatory context.