Translational Justice and Trickle Down Equity: Centering Patient Priorities for Equitable Therapeutic Development

Abstract: Biomedical translation has historically prioritized developing novel therapeutics with demonstrable clinical endpoints, namely safety and efficacy. Although considered the regulatory gold standard, this approach has often led to a mismatch between the priorities of industry and regulators and those of patient communities, which may exacerbate inequities in access. In some cases, therapeutics developed for a narrow subset of patients for speedy translation may fail to meet the needs of broader communities. In others, clinical endpoints targeted by innovators may be different from, or even offensive to, patient communities.

In this panel we will discuss the complementary concepts of “translational justice” and “trickle down equity” which clarify the need for more equitable and just translational pathways centered on those who are the most morally impacted: namely patient communities and neglected rare diseases. First, a bioethicist and sociologist (session moderator) will introduce the concept of translational justice, and how it addresses existing normative gaps in therapeutic development. Second, a bioethics doctoral student will describe the operationalization of the translational justice framework and present empirical findings from diverse patient communities exploring ideal processes for therapeutic development. Third, a genetic counselor and advocate with achondroplasia, a rare genetic condition causing short stature, will build on the concept of translational justice by discussing potential successes and failures of incorporating community voices in clinical trials and therapeutic interventions. Finally, a public health bioethicist will discuss the interrelated concept of trickle down equity, reimagining how rare disease research is conducted by integrating equity throughout the translational pipeline.

Keywords: clinical translation, biomedical research, health justice