ImpactU Versión 3.11.2 Última actualización: Interfaz de Usuario: 16/10/2025 Base de Datos: 29/08/2025 Hecho en Colombia
Involving Patients in Drug Development for Neglected Tropical Diseases (NTDs): A Qualitative Study Exploring and Incorporating Preferences of Patients with Cutaneous Leishmaniasis into Target Product Profile Development
Background: Target Product Profiles (TPPs) are instrumental to help optimise the design and development of therapeutics, vaccines, and diagnostics – these products, to achieve the intended impact, should be aligned with users’ preferences and needs. However, patients are rarely involved as key stakeholders in building a TPP.Methods: Thirty-three cutaneous leishmaniasis (CL) patients from Brazil, Colombia, and Austria, infected with New-World Leishmania species, were recruited using a maximum variation approach along geographic, sociodemographic and clinical criteria. Semi-structured interviews were conducted in the patient’s mother tongue. Transcripts, translated into English, were analysed using a framework approach. We matched disease experiences, preferences, and expectations of CL patients to a TPP developed by DNDi (Drug for Neglected Diseases initiative) for CL treatment.Findings: Patients’ preferences regarding treatments ranged from specific efficacy and safety endpoints to direct and significant indirect costs. Respondents expressed views about trade-offs between efficacy and experienced discomfort/adverse events caused by treatment. Reasons for non-compliance, such as adverse events or geographical and availability barriers, were discussed. Considerations related to accessibility and affordability were relevant from the patients’ perspective.Interpretation: This exploratory study identified preferences in a broad international patient spectrum. It provides methodological guidance on how patients can be meaningfully involved as stakeholders in the construction of a TPP of therapeutics for NTDs. CL is used as exemplar, but the approach can be adapted for other NTDs.Funding: WHO-TDR, DNDi and the Austrian Academy of Sciences.Declaration of Interest: We declare no competing interests.Ethical Approval: Ethical clearance of the protocol was obtained from the following institutional review boards (IRBs) and ethics committees (ECs): World Health Organization Research Ethics Review Committee (WHO ERC), Geneva, Switzerland. Comité Institucional de Ética de Investigación en Humanos (CIEIH), Ethics committee of the Centro Internacional de Entrenamiento e Investigaciones Médicas (CIDEIM), Cali, Colombia. Instituto René Rachou, Fundação Oswaldo Cruz (FIOCRUZ), Minas Gerais, Brazil and Comissão Nacional de Ética em Pesquisa—CONEP, Brasília, Brazil. Comité de Bioética Sede de Investigación Universitaria (CBE-SIU), Universidad de Antioquia, Medellín, Colombia. Oxford Tropical Research Committee (OxTREC), University of Oxford, Oxford, UK. Ethics Committee of the Medical University of Vienna, Vienna, Austria. Only patients above the age of consent were interviewed. Consent was obtained by signature, or an appropriate alternative, as specified by the relevant IRBs. Ethical procedures at each site observed the respective IRB guidance, and are further detailed in the study protocol.