To identify future indications for CAR T-cells, we searched ClinicalTrials.gov for all registered studies on CAR T-cell therapies (search term: “chimeric antigen receptor”) for hematological cancers on May 3, 2019. This search included early phase 1, phase 1, phase 2, phase 3, and phase 4 trials. All studies were ranked according to the indication studied (most to least often studied indication). Through a semi-structured interview, several clinical experts were asked to validate this ranking and to (re)arrange it according to the sequence of expected market entry.

To estimate the eligible patient population for CAR T-cells, we focused on the 2 indications for which CAR T-cells already have market authorization (pediatric ALL [pALL] and DLBCL) and the top 5 potential future indications identified by the clinical experts. The eligible patient population was calculated based on previous population forecasts by using 2 data sources, namely Eurostat and Globocan.3

In the Eurostat forecast, several assumptions were made on the future development for fertility, mortality, and net migration to predict the population of European member states to the year 2080 (based on the population in 2016).4 We assumed a linear trend between the 2016 and 2080 Eurostat data and calculated the yearly population per country of interest. For our purpose, we defined the “disease incident population” by estimating the yearly crude incidence rate (IR) per 100 000 for each disease and country of interest. For pALL and DLBCL, the yearly disease IRs were taken from HTA/BIA reports. For future indications, or in the absence of published data from HTA/BIA reports, we used data from the European Cancer Information System (ECIS).5 Subsequently, the crude IRs were applied to projected population data by Eurostat.4

The online database Global Cancer Incidence, Mortality and Prevalence offers information on projected IRs of different cancer types for the time between 2018 and 2040 for several countries.6 To derive the number of patients for each cancer subtype of interest, we applied proportions based on the literature.3,79

Both forecast approaches are depicted in Figure Figure11.

Flowchart of forecast approaches. CAR-T = chimeric antigen receptor T cell.

The proportion of “patients eligible for CAR T-cell therapy” per country was calculated based on HTA/BIA reports. Most publications stated the yearly number of incident cases and the total number of patients eligible for CAR T-cell therapy. From these numbers, we calculated the proportion of eligible patients and applied this rate to all incident cases to derive the total yearly number of eligible patients for CAR T-cells per disease and country. The CAR T-cell therapy eligible patient population for all future indications was based on expert opinion.

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