Investigating Potential Market Failure with Curative Cell and GENE Therapies
Author(s)
Shaw S1, Wickstead R2, Woodhouse F1, Micallef J1, Brooks Rooney C3
1Costello Medical, Cambridge, UK, 2Costello Medical, London, UK, 3Costello Medical, #23-01/02 Chinatown Point, 01, Singapore
OBJECTIVES Curative cell and gene therapies (C>s) are transforming disease management, offering potential cures for previously chronic/fatal diseases. However, by drastically reducing patient population sizes (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>s, 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 licensed C>s on 12 December 2019 and again on 11 June 2020. Clinicaltrials.gov was then searched using terms for C>s, and all Phase 1/2/3 trials ongoing or completed since 2015 in the indications of currently licensed C>s were identified. US incidence rates (per 100,000 population) were derived from searches in PubMed. RESULTS There is currently only one indication (diffuse large B-cell lymphoma [DLBCL]) with >1 C> approved; the remaining 7/8 indications of licensed C>s have only 1 C> approved. Notably in spinal muscular atrophy (incidence: 0.12/100,000), there are no ongoing or completed trials for competitor C>s, and in retinal dystrophy (unreported incidence; US prevalence: <2,000), there are no trials for competitor C>s with results due by December 2021. Of the 6 indications with >1 ongoing competitor C> trial, 4 are oncology-based. Although the number of competitor C> trials does not have a linear correlation with disease incidence, indications with an incidence >1/100,000 (DLBCL, acute lymphoblastic leukemia, melanoma) have >30 ongoing/recently completed competitor C> trials, whereas indications with an incidence <1/100,000 have 0–9 ongoing/recently completed competitor C> trials. CONCLUSIONS There is potential for market failure with C>s in non-oncology indications due to a lack of competitor C> products. Further research will continue to explore potential market failure in indications for which new C>s are in development.
Conference/Value in Health Info
2020-11, ISPOR Europe 2020, Milan, Italy
Value in Health, Volume 23, Issue S2 (December 2020)
Code
PBI33
Topic
Epidemiology & Public Health, Health Service Delivery & Process of Care
Topic Subcategory
Disease Management
Disease
Genetic, Regenerative and Curative Therapies