Function

The aim of these functions is to develop a business case and guide the development of the new TB vaccine. Indeed, an overall business model is crucial for TB vaccines, which have a high global impact but an un-validated commercial value which will vary depending upon the Target Product Profile (TPP).

A key objective of the business function is to develop the business approach for the TB vaccine candidate, to establish partnerships, and to mobilise resources. The objective of legal is typically to support negotiations during the establishment of partnerships, to manage intellectual property and to implement policies. The market function develops market analysis and assessment, monitors the competition, defines product positioning and the communication strategy.

Stage 
B
Perform POC studies in animals
Gate 
B
Progress to Pre-Clinical
Main Activities
  • Describe Intellectual Property (IP) status
  • If necessary, identify potential partners to support development
CRITERIA REQUIRED
  • No major IP obstacles identified or strategy for resolution in place
  • As necessary, viable partners identified; Material Transfer Agreement (MTA) and other agreements established
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Guidance

Intellectual Property (IP) is an important element in business. If not done already, it is wise at this early stage to analyse the status of IP of the technology or vaccine. This includes a review of the ‘state of the art’, what is known and published, and the status of own patents versus competition. It is important to identify any obstacle due to IP. The IP situation and strategy for resolution (of e.g. existing prior patent, uncertain freedom to operate) is defined at this stage and becomes part of the Product Development Plan (PDP).

The developer should consider if a partner will be needed in the near- or long-term to assist and support the development of the vaccine candidate. If so, a strategy for establishing and management of partnerships should be developed and this becomes part of the PDP.

An early analysis of the market potential of the product and the countries where it would be used should be performed and summaries should be part of the PDP. This analysis is important for TB vaccines which have a high global impact but an unvalidated commercial value. Further information is provided in an AERAS and TBVI publication TB vaccine Research and Development, a Business Case for investment.

Stage 
C
Perform Pre-Clinical evaluations
Gate 
C
Progress to preparation for Phase 1, First-In-Human
Main Activities
  • Establish acceptable Intellectual Property (IP) position
CRITERIA REQUIRED
  • Freedom to operate investigated and an acceptable level of risk demonstrated
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Guidance

A Patent Attorney should evaluate the freedom to operate and provide an opinion on risk to further confirm/solidify an acceptable IP position.

Stage 
D
Perform GMP and toxicity studies and prepare Clinical Trial Application
Gate 
D
Progress to First-In-Human/Phase1
Main Activities
  • Identify and secure funds for First-in-Human (FIH)/Phase 1
CRITERIA REQUIRED
  • Funding for FIH/Phase 1 secured
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Guidance

Funding must be secured and available for the first-in-human (FIH)/Phase1 before the start of the study.

The decision to move the candidate(s) into clinic should trigger a more formal analysis and detailed analysis of the potential market and target countries are re-evaluated.

Stage 
E
Perform, First-in-human/Ph1
Gate 
E
Progress to Ph2
Main Activities
  • If necessary, identify potential partners to support late development (Chemistry, Manufacturing and Controls (CMC), scale-up validation)
CRITERIA REQUIRED
  • As necessary, viable partners identified; contractual agreements and other agreements established
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Guidance

As the cost of development and the need for capacity increases, if necessary, potential partners to support mid and late clinical (CMC, scale-up validation) development are sought.

Stage 
F
Perform Ph2 (including Pre-POC) studies
Gate 
F
Progress to Ph2b Efficacy
Main Activities
  • Analyse and update Intellectual Property (IP)
  • Conduct initial forecasting and Cost of Goods (CoG) analysis for pricing parameters, scalability of process, and product viability in targeted countries
CRITERIA REQUIRED
  • Acceptable IP status to support commercialisation confirmed
  • Initial forecasting and CoG in targeted (low-, middle- and high-income) countries analysed and supportive of a viable product
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Guidance

An initial forecasting and cost of goods (CoG) analysis for pricing parameters, scalability of process and product viability in targeted countries should be performed and the analysis integrated back into the TPP and PDP. An analysis of the IP situation should confirm that the IP status would support commercialisation.

Stage 
G
Perform Ph2b Efficacy
Gate 
G
Progress to Ph3
Main Activities
  • Analyse and update Intelectual Property (IP)
  • Update partnership agreement(s) if necessary
  • Define pathway to commercialization
  • Obtain support from Principal Investigator(s) (PI) and Key Opinion Leaders (KOL)
  • Establish budget and secure financial plan to complete Phase 3
  • Fine-tune Cost of Goods (COG) and supply and demand plan
  • Update market assessments in potential early (middle and late) adopters
CRITERIA REQUIRED
  • Acceptable IP status to support commercialization confirmed
  • Strong industrial partnership in place
  • Pathway to commercialization defined
  • PI and KOL community are supportive
  • Phase 3 budget and funding plan in place
  • COGs and supply and demand analysis available, and support introduction of a viable product
  • Market assessments updated
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Guidance

As the product progresses to Phase 3, the IP situation is further analysed as needed. Performing a large Phase 3 efficacy study and anticipating registration and the potential market requires new business development, and a pathway to commercialisation is defined. It might include seeking partnership, obtaining support from principal investigators (PI) and key opinion leaders (KOL). The market assessments made in the previous Stages are updated in potential early adopters as first priority, in middle and late countries as second priority. The initial COGs, supply and demand plan are also fine-tuned.

Stage 
H
Perform Ph3
Gate 
H
Progress to licensure
Main Activities
  • Analyse and update Intellectual property (IP)
  • Establish budget and financial plan to licensure
  • Finalise demand and supply plan
CRITERIA REQUIRED
  • IP obstacles to commercialization resolved
  • Budget for licensure established
  • Demand and supply plan validated
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Guidance

The IP is updated. Any obstacles to commercialisation must be addressed. Budget and financial plan to licensure have been established and post-market commitments and funding have been secured. The plan between demand and supply is finalised.