Supplementary Materials

Supplementary Material for:

Financing translation: Analysis of the NCATS rare-diseases portfolio

David E. Fagnan, N. Nora Yang, John C. McKew, Andrew W. Lo*

*Corresponding author. E-mail: alo-admin{at}mit.edu

Published 25 February 2015, Sci. Transl. Med. 7, 276ps3 (2015)
DOI: 10.1126/scitranslmed.aaa2360

This PDF file includes:

  • Table S1. Observations of NCATS rare-disease projects including success, duration, and cost of trials.
  • Table S2. Prior weight given to literature data for orphan diseases (4), with lower values relying more on the NCATS observations.
  • Table S3. Posterior estimates of parameters for simulating an NCATS rare-disease megafund, combining literature estimates for orphan diseases (4).
  • Table S4. NCATS portfolio of rare-disease projects in TRND and BrIDGs.
  • Table S5. Summary of key comments from valuation panel respondents when asked to value a portfolio of rare-disease projects within NCATS.
  • Table S6. Panel median valuations compared with literature estimates for orphan diseases (4).
  • Table S7. Parameters and distributions used in simulation framework for an NCATS raredisease megafund.
  • Fig. S1. Plot of density functions for various Phase 2 clinical trial time distributions calibrated by matching first and second moments.
  • Table S8. Performance metrics for RBO structure (without guarantee) from Table S2 for alternative clinical trial time distributions.
  • Table S9. Calibrated parameters for valuation distributions at phase 3.
  • Fig. S2. Plot of density functions for various Phase 3 valuation distributions calibrated using first and second moment matching.
  • Table S10. Performance metrics for RBO structure (without guarantee) from Table S2 for alternative valuation distributions.
  • Table S11. Performance metrics for RBO structure (without guarantee) from Table S2 for adjusted probability of success applied to all stages.
  • Fig. S3. Performance metrics for RBO structure (without guarantee) from Table S2 for adjusted probability of success applied to all stages.
  • Fig. S4. Performance metrics for RBO structure (without guarantee) from Table S2 for adjusted mean and standard deviation of Phase 3 valuation.
  • Table S12. Performance metrics for RBO structure (without guarantee) from Table S2 for adjusted mean and standard deviation of Phase 3 valuation.
  • Fig. S5. NCATS rare disease portfolio diversity by disease and drug modality.

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