An important area of pediatric research and development is peanut allergy immunotherapy. Researchers are pursuing oral, sublingual, subcutaneous, and epicutaneous products that activate the immune system through allergen exposure to desensitize individuals to peanuts. One such treatment is now FDA approved, and another is poised to receive market approval. However, immunotherapy conveys significant risk to patients, such as high rates of treatment-induced anaphylaxis. This project's objective is to identify empirically the on-the-ground ethical challenges that emerge in pediatric peanut immunotherapy (PIT) clinical trials. To date, there has been little attention to ethics within the broad domain of food allergy research and practice. Yet, PIT research involves many features that warrant ethical consideration, including clinical trials that enroll children, some as young as 1 year old, and risk-benefit profiles that differ from many other therapeutic areas. To explore these ethical issues, the project has 2 specific aims: (1) Document how stakeholders?investigators, caregivers, adolescents, patient advocates, FDA officers, and pharmaceutical company representatives?define and understand acceptable risks and benefits from peanut immunotherapy, both in terms of research protocols and the eventual products to be used in clinical practice; and (2) Identify the ethical challenges that emerge from these stakeholders' stances on peanut immunotherapy risks and benefits.
For Aim 1, the project uses an ethnographic approach?consisting of observational studies and 125 semi-structured interviews?that focuses on 6 leading U.S. PIT research centers, academic food allergy conferences, patient advocacy events, and FDA hearings for PIT products. To accomplish Aim 2, the project employs analytic methods to ground the empirical findings from Aim 1 in bioethical frameworks, including principlism and research on vulnerable or complex populations. The proposed research is significant because it will develop an initial framework for the ethical conduct of PIT clinical trials that attends to the myriad interests that drive biomedical research, including the search for therapies that meaningfully improve the lives of children and adults with peanut allergy, the need to design and conduct clinical trials to meet the regulatory standards required by the FDA for the approval of products, and the commercial context in which investigators and companies have financial conflicts of interest that motivate the successful development of new therapies. This project is innovative because it explores a novel area of social science and bioethics research and because it investigates the entire sphere of PIT research and a wide array of stakeholders. This application is directly responsive to NIH's Notice of Interest in High Priority Research in Bioethical, Legal, and Societal Implications of Biomedical Research (NOT-LM-17-001), focusing in particular on new and emerging technology development and use, pediatric research, and the current and emerging regulatory environments.
The proposed research is relevant to public health because it investigates the risks, benefits, and ethical chal- lenges of pediatric peanut immunotherapy research. Our findings could be used to better protect children who enroll in these clinical trials and to maximize the benefits of peanut immunotherapy for future clinical use.
