Author | Castro, María del Mar | |
Author | Erber, Astrid C. | |
Author | Arana, Byron | |
Author | Cota, Gláucia Fernandes | |
Author | Denkinger, Claudia M. | |
Author | Harrison, Nicole | |
Author | Kutyi, Julia | |
Author | López-Carvajal, Liliana | |
Author | Plugge, Emma | |
Author | Walochnik, Julia | |
Author | Olliaro, Piero | |
Access date | 2024-04-05T18:08:50Z | |
Available date | 2024-04-05T18:08:50Z | |
Document date | 2024 | |
Citation | CASTRO, María del Mar al. 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. PLoS Negl Trop Dis, v. 18, n. 2, p. e0011975, 2024. doi.org/10.1371/journal.pntd.0011975 | en_US |
ISSN | 1935-2735 | en_US |
URI | https://www.arca.fiocruz.br/handle/icict/63425 | |
Language | eng | en_US |
Publisher | Public Library of Science | en_US |
Related document | https://www.arca.fiocruz.br/handle/icict/59878 | en_US |
Rights | open access | en_US |
Title | 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. | en_US |
Type | Article | en_US |
Abstract | Background: Target Product Profiles (TPPs) are instrumental to help optimise the design and development of therapeutics, vaccines, and diagnostics – these products, in order 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.
Methodology: 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 respective 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.
Principal 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.
Conclusions/Significance: NTDs affect disadvantaged populations, often with little access to health systems. Engaging patients in designing adapted therapies could significantly contribute to the suitability of an intervention to a specific context and to compliance, by tailoring the product to the end-users’ needs. 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 an exemplar, but the approach can be adapted for other NTDs. | en_US |
Affilliation | Centro Internacional de Entrenamiento de Investigaciones Médicas. Cali, Colombia/Universidad Icesi. Cali, Colombia/ Division of Infectious Diseases and Tropical Medicine, Center of Infectious Diseases. Heidelberg University Hospital. Heidelberg, Germany/German Center of Infection Research. | en_US |
Affilliation | Department of Epidemiology. Center for Public Health. Medical University of Vienna.Vienna, Austria/Infectious Diseases Data Observatory. Oxford, United Kingdom | en_US |
Affilliation | Drugs for Neglected Diseases Initiative. Geneva, Switzerland | en_US |
Affilliation | Fundação Oswaldo Cruz. Instituto René Rachou. Belo Horizonte, MG, Brazil | en_US |
Affilliation | Division of Infectious Diseases and Tropical Medicine, Center of Infectious Diseases, Heidelberg University Hospital, Heidelberg, Germany/German Center of Infection Research. | en_US |
Affilliation | Division of Infectious Diseases and Tropical Medicine, Department of Medicine I, Medical University of Vienn. Viena, Austria | en_US |
Affilliation | Division of Infectious Diseases and Tropical Medicine, Department of Medicine I, Medical University of Vienn. Viena, Austria | en_US |
Affilliation | Programa de Estudio y Control de Enfermedades Tropicales. Universidad de Antioquia. Medellín, Colombia | en_US |
Affilliation | Primary Care. Population Sciences and Medical Education. University of Southampton. Southampton, United Kingdom | en_US |
Affilliation | Institute of Specific Prophylaxis and Tropical Medicine. Center for Pathophysiology. Infectiology and Immunology. Medical University of Vienna. Vienna, Austria | en_US |
Affilliation | International Severe Acute Respiratory and Emerging Infection Consortium. Pandemic Sciences Institute. University of Oxford, Oxford, United Kingdom | en_US |
Subject | Drug Therapy | en_US |
Subject | Leishmaniasis | en_US |
Subject | Neglected Tropical Diseases | en_US |
Subject | Adverse events | en_US |
Subject | Drug research and development | en_US |
Subject | Drug administration | en_US |
Subject | Colombia | en_US |
Subject | Brazil | en_US |