OBJECTIVES

Curative cell and gene therapies (C&GTs) are transforming disease management approaches, offering potential cures for previously chronic/lethal diseases. However, by drastically reducing the patient population size (through cures), such therapies may lead to market failure, with competition limited and monopoly pricing maintained far longer than with traditional therapies. This research investigates potential market failure with currently licensed C&GTs, and whether market failure may be more likely in indications with smaller patient populations.

METHODS

The European Medicines Agency and US Food and Drug Administration websites were searched to identify currently licensed C&GTs on December 12, 2019. The clinicaltrials.gov website was subsequently searched using terms for C&GTs, and all phase 1/2/3 trials ongoing or completed since 2015 in the indications of currently licensed C&GTs were identified. US incidence rates (per 100,000 population) were derived from searches in PubMed.

RESULTS

In total, 7/8 indications with licensed C&GTs had only one C&GT approved (diffuse large B-cell lymphoma [DLBCL] being the exception). For spinal muscular atrophy (incidence: 0.12/100,000), there were no ongoing or completed trials for competitor C&GTs, and for retinal dystrophy (unreported incidence; US prevalence: <2,000), there were no trials for competitor C&GTs with results due by December 2021. Of the six indications that had >1 ongoing competitor C&GT trial, four were oncology-based. Although the number of competitor C&GT trials was not found to have a linear correlation with disease incidence, indications with disease incidence >1/100,000 (DLBCL, acute lymphoblastic leukemia, melanoma) had >20 ongoing or recently completed competitor C&GT trials, whereas indications with disease incidence <1/100,000 had 0–7 ongoing or recently completed competitor C&GTs trials.

CONCLUSIONS

There is potential for market failure with C&GTs in non-oncology indications due to a lack of competitor C&GT products. Further research will continue to explore potential market failure in indications for which new C&GTs are in development.