Pediatric-onset rare disease therapy pipeline

This data is described in detail in the Technical Appendix. Supplementary File S1 contains the technical appendix describing the functional structure of the forecasting model a bibliography organized by disease and, within diseases, by antigen or gene.

Supplementary File S2 contains examples of code (mostly SQL) used to extract and process data from the Clinical Trials Transformation Initiative's database for Aggregate Analysis of ClinicalTrials.gov (AACT). (AACT is a dynamic entity and was substantially changed on November 15, 2021.) Supplementary File S3 contains participant inclusion and exclusion criteria for clinical trials in our sample. Supplementary File S2 is an Excel workbook containing the clinical trials data used in the Markov Chain analysis, modalities, delivery vectors, targeted discriminators for each disease, trial success rates and totals of completed trials by phase and modality, peak penetration and years to peak for indication/modality/discriminator groups, modality level data, basic epidemiological data, and empirical probability density functions for months-in-phase for completed.