Financing repurposed drugs for rare diseases: a case study of Unravel Biosciences

Table 3 Development stage assumptions used for the portfolio of rare disease compounds and the probability distributions used to perturb our assumptions

		Normal case		"Me-too" case
(a) Probabilities of success, PoS	Distribution	PoS	Prob. of arriving to phase	PoS	Prob. of arriving to phase
Discovery, preclinical development, and phase 1	Bernoulli	67%	100%	72%	100%
Phase 2	Bernoulli	53%	67%	63%	72%
Phase 3	Bernoulli	69%	36%	79%	45%
FDA submission-to-launch	Bernoulli	94%	25%	94%	36%
Total cumulative PoS		100%	23%	100%	34%

(b) Time for development (years)	Distribution	Lower bound	Base case	Upper bound
Discovery, preclinical development, and phase 1	PERT	1	1	1
Phase 2	PERT	1	2	3
Phase 3	PERT	1	2	3
FDA submission-to-launch	PERT	1	1	1
Total		4	6	8

(c) Cost of development ($, in millions)	Distribution	Lower bound	Base case	Upper bound
Discovery, preclinical development, and phase 1	PERT	1	4	7
Phase 2	PERT	9	14	40
Phase 3	PERT	22	35	99
FDA submission-to-launch	PERT	37	37	37
Total		69	90	183

We consider drugs that are and that are not a “follow-on” of other drugs for the same disease that have been proven to be effective and reached phase 2 (i.e., "me-too" drugs)

ISSN: 1750-1172