Function

Three areas of expertise are gathered in Function 2, business, legal and market. Overall, their aim is to develop a business case and guide the business development of the new TB vaccine candidate, according to the objectives stated in the Target Product Profile (TPP). An overall business model is crucial for TB vaccines, which have a high global impact but an un-validated commercial value.

The objectives of the business function are 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 the establishment of contracts and legal agreements, 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 the business case. If not done already, it is wise at this early stage to analyse the IP status of the technology and of the vaccine candidate(s). 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 be part of the PDP.

It is prudent at this early stage to initiate a market analysis with epidemiology data, burden of disease, regions/ countries and population where the vaccine would be used, and marketing aspects. This preliminary market assessment is summarised in the PDP and is important for TB vaccines which have a high global impact but an unvalidated commercial value.

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 intellectual property 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

As best practice, funding should be secured and available to cover the whole First-In-Human (FIH)/Phase1 before the start of the study.

With the decision to move the vaccine candidate into clinical development, a formal market assessment should be performed at this stage, refining the market analysis initiated earlier (Stage B). The assessment includes medical need (from epidemiology data, incidence and disease burden), market characteristics and dynamics (market size, competition, procurement and delivery processes), and positioning of the vaccine candidate versus standard of care (SOC), niche indications, and competitive advantages. The selection of the population and of the potential early adopter countries as first priority, and of middle and late adopter countries as second priority should also be made. An initial business case that validates the upcoming investments should be developed. It will highlight drivers and barriers that will contribute to the medical and economic values of the vaccine and ensure that a viable market will exist. A comprehensive review of recent literature would be a relevant way to gather data needed for market assessment and business case.

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 manufacturing and clinical development are sought. Market analysis and assessments are refined according to availability of new data.

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 intellectual property (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 intellectual property situation is further analysed as needed. Performing a large Phase 3 efficacy study and anticipating marketing authorisation 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 assessment and the business case are updated. The selection of potential early adopters as first priority, and of middle and late countries as second priority is re-evaluated. The initial cost of goods, 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 intellectual property is updated, and obstacles to commercialisation addressed. The activities leading to licensure are budgeted and covered. The plan between demand and supply is further defined together with cost of goods assumptions. An initial market plan is developed at this stage. The initial market plan is developed at this stage focusing as a priority on the the early adopter countries.

Stage 
I
Register vaccine with relevant Regulatory Authorities
Gate 
I
Obtain MA and Progress to launch
Main Activities
  • Update partnership agreement(s), if necessary
  • Establish budget and financial plan for post-market commitments
  • Review and complement business case
  • Validate COGS, demand and supply plan
  • Refine Market plan
CRITERIA REQUIRED
  • Commercial partnership established
  • Budget for post-marketing commitments established
  • Business case complemented
  • COGS, demand and supply plan validated
  • Market plan ready
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Guidance

The PDP is updated to include activities of each functional area to launch and implement vaccination programmes.

Stage 
J
Launch
Gate 
J
Implement vaccination programs
Main Activities
  • Validate Business case, demand and supply plan
  • Launch in early adopters countries
CRITERIA REQUIRED
  • Business Case, demand and supply plan validated
  • 1st commercial launch achieved
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Guidance

The PDP is updated to include activities of each functional area to launch and implement vaccination programmes.