Table 1.

Assumptions about three development pathways.

Case A	I. Some firms will be experienced in development of licensed biological products, II. Some experienced firms will be willing and able to invest in the proposed cell therapy to ensure rapid development and licensure of the product. This can lead to compressed timelines but it may also place investment at risk even prior to achieving clinical proof-of-concept. This is especially relevant for regenerative medicine advanced therapies (RMAT) and Breakthrough-eligible products, which could be licensed with modest sized pivotal clinical trials similar to Phase 2 studies, if substantial clinical benefit is demonstrated for high unmet medical needs in Phase 1/2 studies. III. If the firm has internal manufacturing capability, no investment is needed for a commercial plant. If not, then capacity must be found at a CMO or built.
Case B	I. Although experienced with the development of biological products, some firms may not be willing or able to invest at risk to ensure rapid development and licensure. Investment beyond that to enable Phase 1 and 2 studies will not occur, except for critical items, until clear achievement of clinical proof-of-concept. II. If the firm has internal manufacturing capability, no investment is needed for a commercial plant. If not, then capacity must be found at a CMO or built.
Case C	I. Some firms and other non-commercial organizations will not invest beyond the minimum level necessary to initiate Phase 1/2 studies until clinical proof of concept is achieved II. In many of these cases, the strategy is to sell the product or the organization to a pharmaceutical company that will then invest in late development.