The three key processes
- intelligence and project sourcing
- and project & portfolio management
will be further implemented by DNDi to fill the pipeline by seeking leads and drug candidates at all stages of development (Figure 10). At the post-registration stage, existing and new mechanisms will be leveraged to ensure patient access to treatments through partnerships with industrial partners, international organizations, and national programmes.
Projects will be divided into five categories:
- New treatments (involving NCEs) developed from novel compounds identified through screening, lead optimization, or licensing. These drugs must meet target product profiles (TPPs) and may be used in monotherapy or as part of combination therapies when appropriate;
- New treatments developed from compounds with known antimicrobial/antiparasitic activities (could start at lead optimization or pre-clinical development);
- New indications for existing treatments (therapeutic switching);
- Combinations or new formulations of existing drugs that are better adapted to field conditions and patient needs (paediatric dosage forms, long-acting, new route of administration, fixed-dose combinations, copackaging, or co-administration);
- Geographical extension of existing treatments, including completion of regulatory dossiers in new countries.
Selecting for success in the field: the Target Product Profile
As a prerequisite to building a portfolio strategy, the desired R&D outcome for each disease is defined as the target product profile (TPP). Each R&D project in the portfolio is selected, progressed, and managed according to well-defined decision matrices based on these TPPs.
Used properly, the TPP can play a central role in the entire drug discovery and development process. This role includes effective optimisation of drug candidates, decision-making within an organisation, design of clinical research strategies, and constructive communication with regulatory authorities.
Characteristics of a TPP
Indications: which diseases?
Population: which patients and where?
Clinical Efficacy: does it kill the parasite effectively?
Safety and Tolerability: what kind and how many adverse events?
Stability: how long can it be stored in the field?
Route of Administration: how is it given to patients?
Dosing Frequency: how often and how long must it be given?
Cost: will it be affordable to target population?
Time to Availability: how long will it take to develop?