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Abstract:
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Allergen immunotherapies (AITs) have been increasingly developed for treating a variety of allergy diseases and some have demonstrated significant clinical benefits through clinical trials. Since AIT directly targets the pro-inflammatory immune response, it is hoped to potentially provide sustained and disease modifying effects by altering the course of disease. The European Medicines Agency’s guideline on AIT distinguishes four levels of treatment benefits: efficacy of symptom relief in the first year, sustained efficacy over 2-3 years, long-term efficacy and disease modifying effects in post-treatment years, and allergy curing efficacy in post-treatment years. Due to lack of robust randomized double-blind, placebo-controlled long-term trials, sustained and disease modifying effects of AIT products have not been formally evaluated. Therefore, we propose a three-stage design for sequentially evaluating the short-term, sustained, and disease modifying effects of an AIT in respective stages. Depending on the type of endpoint and timings of the measurements, appropriate statistical approaches are proposed for analyzing different levels of treatment effects at individual stages.
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