Target Product Profile (TPP)
As a prerequisite to building a portfolio strategy, the desired R&D outcome, or the target product profile (TPP), has been defined. Based on discussion with various HAT players and experts, a dual strategy is proposed: 1. The first priority is to develop a safe, effective, and practical stage 2 HAT drug to replace current first-line treatments, and to improve and simplify the current case management. The aim is to develop one drug that is effective against both stage 1 and stage 2 of HAT. 2. The second priority is for a very simple stage 1 treatment, to be used at the local health centre level, which in turn could lead to increased access to treatment and coverage of HAT. Depending on the availability of a simple diagnostic, such a drug would allow for mass screening + treatment campaigns in endemic areas, thus preventing disease progression to stage 2 and reducing disease transmission. Each R&D project in the portfolio will be selected, progressed, and managed according to well-defined decision matrices based on these TPPs. TPP 1: Towards a new stage 1+2 treatment
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TPP 2 : To be developed only if a second stage treatment fails to show efficacy and is already in advanced clinical trials
Review of these TPPs will occur on a regular basis through consultation with stakeholders including WHO, HAT control programs in endemic countries, and specifically with physicians and health workers who deal with this disease on a daily basis. |