Efficacy is evaluated at this stage, leading to a major decision at Gate H to progress to registration. The PDP is focusing on the performance of Phase 3 and regulatory. Hopefully, efficacy and safety data meet TPP attributes and support the related product information in the leaflet. Additional indications should be sought in the Phase 4 clinical studies.
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.
Stability data have been generated from the first R&D lot and for each manufacturing lot. These stability data should be compared, as part of the characterisation of the product. A sufficient amount of vaccine product from the Phase 3 production has to be stored to serve as a long-term reference standard for QC product assays.
Provided that all pre-set criteria for product and process are confirmed, meaning that no major out-of-specification issues occurred, the production process can be executed for routine manufacturing.
The Phase 3 study has been completed; safety and efficacy data are analysed. The complete study report is prepared. If vaccine consistency has not been established at this stage, a study of safety and immunogenicity consistency of lots should be carried out.
Draft a plan for Phase 4 studies to assess vaccine safety and effectiveness in field conditions and in populations that have not yet been studied during Phase 3 studies.
During the Phase 3 trial biospecimens have been collected. The investigational plan to identify a CoPshould be developed based on the analysis of Phase 3 immunogenicity andefficacy data.
During the Phase 3 trial bio specimens have been collected. The investigational plan to identify a CoP should be developed based on the analysis of Phase 3 immunogenicity and efficacy data.
Analysis of the data from the Phase 3 trial confirmed protective efficacy against the primary case definition endpoint greater or equal to the minimum predefined efficacy as set in the TPP in the target population for licensure.
The objective is to implement as part of or in parallel to Phase 3 the HEOR activities that will enable gathering of key data and the elements to subsequently develop the value dossier to support the medical and economic rationale of the vaccination programme.
A draft of this value dossier should be developed during stage H and contain a summary of the clinical, economic and societal value, and supporting evidence (studies) for the new TB vaccine as well as background and information on TB (i.e., burden of illness, epidemiology, etc.). The core dossier is developed as an evolving document that can serve as a template for customising submissions to local, national and/ or supranational customers and other stakeholders at a later stage.
At this stage, the initial pricing strategy should be drafted and developed. The strategy is supported by a value-based approach documented by the HEOR rationale and data. It will need to encompass an international vision, taking into account for example specificities of high, middle and low income countries, (multi-tiered pricing strategy). The planning and prioritisation in gathering relevant data to support this value-based approach is crucial because of the timing and costs of these activities.
Regular consultations with stakeholders that were identified in earlier stages are crucial to prepare regulatory submissions, WHO pre-qualification, policy recommendation, implementation studies, financing and product launch.
For adolescents/ adult vaccines, a tiered pricing strategy should be considered according to potential use in high-risk groups in developed countries.
For neonates/infant vaccines, there is a need to take current BCG pricing and volumes into account.
For therapeutic vaccines, a tiered pricing strategy should be considered taking into account use in TB patients in developed countries. Existing drug pricing and reimbursement mechanisms will be key parameters to analyse and integrate into the analysis.