Progress and Criteria in Public Health Applications of Gene Therapy and Gene Editing: Beyond the White Paper.

Introduction: This Review examines the pace of public health-related gene therapy and gene editing development since the publication of a key British white paper in 2003.

Methods: A case approach to assessing progress is used, where the cases represent illustrative health conditions we have discussed with our public health students. Three overarching baskets are considered: (1) gene therapy and editing for rare, single gene disorders (e.g., homozygous familial hypercholesterolemia and hereditary amyloidosis polyneuropathy); (2) gene therapy and editing for high prevalence conditions (e.g., sickle cell disease); and (3) genetic engineering and gene editing of mosquitoes transmitting tropical disease.

Results: The largest number of public health-related trials listed in this purposive inspection have made Phase III (comparing treatments), with several in Phase II (establishing efficacy) and Phase I (assessing safety). Cost-benefit considerations will limit the bulk of gene therapies from receiving Medicaid support which would benefit diverse groups, though criteria do exist for public backing.

Discussion: A scenario is described where several gene therapies relating to public health conditions could be supported by public insurance and justifiably meet the public health goals of effectiveness and equity. These instances are contrasted with human germline gene editing using CRISPR/Cas9 technology, and localized efforts to control vector populations with gene drives.

Conclusion: Future debates will reflect extensions of the current applications, with secondary research undertaking cross-sectional inspections of sets of gene therapy and gene editing clinical trials. A limited number of protocols could advance to public financing and receive public health support; indications for such usage need further defining. Considerations of benefit to diverse populations are an important hallmark of public health-related regimens.