Evolving Food Allergy Clinical Trials to Become More Patient-Centered.

The current FDA paradigm may not fully capture important patient-centered outcomes or measure a primary outcome that is truly meaningful to patients. Patient reported outcome measures (PROMs) are standardized tools measuring the patient's experience in food allergy clinical trials, which can help support shared decision-making (SDM) and further our understanding of treatment impact. Food allergy PROMs include quality of life (QoL), health state utility (HSU), severity, and self-efficacy measures. Currently, FDA registration trials for product approval only consider a fixed increase in allergen threshold from pre-to-post intervention as a primary outcome (vs. a more flexible "X-fold" increase not accounting for an upper and lower specific threshold), though many use QoL as a secondary outcome for patient-centered assessment of treatment impact. Currently used QoL PROMs were not designed to measure change on therapy nor measure HSU (e.g., quantitative risk a patient may be willing to take to improve their current health), which can be used to determine therapy value. While the current paradigm for primary and secondary outcomes in food allergy clinical trials was appropriate at the early stages of food allergy therapy development when conceived in the late 2000's and early 2010's, in the 2020's these outcome choices risk being stagnant and outdated. As such, the current paradigm for food allergy outcomes should evolve to incorporate more patient-centered primary outcome measures which patient data indicate are meaningful, so outcomes more realistically reflect a therapy's impact. This evolution will better support SDM discussions as patients consider their therapy options and can inform new product development.