Development Strategy

Target product profile

Who are we going to treat or vaccinate?

The TPP summarizes the indication and many of the expected or desired product characteristics. Defining a TPP can be an ongoing, iterative process: the TPP guides early decision making whereas early data may result in TPP adaptations

• Define the indication(s) and product characteristics
• Define the Phase 3 data package required for licensure
• Define the clinical pathway needed to design and derisk the pivotal studies
• Define the data that need to be generated in Ph1, 2 and 3 to support licensure
• Define the assays and assay quality needed to generate data linked to specific endpoints

Clinical development

The CDP documents the pathway from FTIH towards licensure, including early safety and dose-finding studies, age (de)-escalation studies and pivotal registration studies. Clinical development is usually made up of 3 phases, with Phase 3 studies aiming to generate critical efficacy and safety data. Phase 4 studies are done after licensure and can be critical to generate effectiveness and/or further safety data. VaxxCellence can provide advice on:

• CDP and clinical study design
• Clinical and immunological endpoint definition

Readings:

• Roy-Ghanta S et al, 2014. Responses to A(H1N1)pdm09 Influenza vaccines in participants previously vaccinated with Seasonal Influenza vaccine: A randomized, observer-blind, controlled study,  J Inf Dis 210(9): 1419
• Ryser M et al, 2019.  Post-hoc analysis from phase III trials of human papillomavirus vaccines: considerations on impact on non-vaccine types, Exp Rev Vaccines 18(3)
• Bruxvoort K et al, 2022. Recombinant Adjuvanted Zoster vaccine and reduced risk of Coronavirus disease 2019 diagnosis and hospitalization in older adults, J Inf Dis 225(11):1915

Benefit/Risk analysis

Assessing the product’s safety profile

Benefit/Risk assessment is central at every step of the R&D process because it provides the framework for stage-gate decision making. It is clear that benefit should always outweight the risks but understanding both and raising awareness of the balance can inform product and study design decisions at each phase

• Each phase of product development is subject to careful Benefit/Risk assessment and review of the plans/documents in place
• Taking Benefit/Risk evaluation into account from the start can facilitate stage-gate discussion and results in better preparedness for late phase development

Readings:

• Buonocore SM & van der Most RG, 2022. Narcolepsy and H1N1 influenza immunology a decade later: what have we learned?, Front Immunol 13
• Cohet C et al, 2019. Safety of AS03-adjuvanted influenza vaccins: A review of the evidence ,  Vaccine 37: 23, 2006

Cost/Benefit analysis

What is the product's impact on the individual and on the public health system?

Vaccines do not save lives, but vaccination does. Market access is critical for success and implementation relies on careful cost-benefit analysis. This is the area of Health Economic Outcome Research (HEOR) that hinges on modelling and real world data to estimate the impact of a treatment/vaccine on the healthcare system and quantifies how this compares with other, competing approaches   

• Cost/Benefit (C/B) ratios can be modelled based on the indication and can inform the expected efficacy
• These C/B ratios can predict the product's affordability and place in the competitive landscape
• HEOR is critical for market access decisions

• Specific expertise supported by an MSc at LSE (2020): LSE Dissertation (pdf)