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https://www.arca.fiocruz.br/handle/icict/63425
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.
Leishmaniasis
Neglected Tropical Diseases
Adverse events
Drug research and development
Drug administration
Colombia
Brazil
Author
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.
Department of Epidemiology. Center for Public Health. Medical University of Vienna.Vienna, Austria/Infectious Diseases Data Observatory. Oxford, United Kingdom
Drugs for Neglected Diseases Initiative. Geneva, Switzerland
Fundação Oswaldo Cruz. Instituto René Rachou. Belo Horizonte, MG, Brazil
Division of Infectious Diseases and Tropical Medicine, Center of Infectious Diseases, Heidelberg University Hospital, Heidelberg, Germany/German Center of Infection Research.
Division of Infectious Diseases and Tropical Medicine, Department of Medicine I, Medical University of Vienn. Viena, Austria
Division of Infectious Diseases and Tropical Medicine, Department of Medicine I, Medical University of Vienn. Viena, Austria
Programa de Estudio y Control de Enfermedades Tropicales. Universidad de Antioquia. Medellín, Colombia
Primary Care. Population Sciences and Medical Education. University of Southampton. Southampton, United Kingdom
Institute of Specific Prophylaxis and Tropical Medicine. Center for Pathophysiology. Infectiology and Immunology. Medical University of Vienna. Vienna, Austria
International Severe Acute Respiratory and Emerging Infection Consortium. Pandemic Sciences Institute. University of Oxford, Oxford, United Kingdom
Department of Epidemiology. Center for Public Health. Medical University of Vienna.Vienna, Austria/Infectious Diseases Data Observatory. Oxford, United Kingdom
Drugs for Neglected Diseases Initiative. Geneva, Switzerland
Fundação Oswaldo Cruz. Instituto René Rachou. Belo Horizonte, MG, Brazil
Division of Infectious Diseases and Tropical Medicine, Center of Infectious Diseases, Heidelberg University Hospital, Heidelberg, Germany/German Center of Infection Research.
Division of Infectious Diseases and Tropical Medicine, Department of Medicine I, Medical University of Vienn. Viena, Austria
Division of Infectious Diseases and Tropical Medicine, Department of Medicine I, Medical University of Vienn. Viena, Austria
Programa de Estudio y Control de Enfermedades Tropicales. Universidad de Antioquia. Medellín, Colombia
Primary Care. Population Sciences and Medical Education. University of Southampton. Southampton, United Kingdom
Institute of Specific Prophylaxis and Tropical Medicine. Center for Pathophysiology. Infectiology and Immunology. Medical University of Vienna. Vienna, Austria
International Severe Acute Respiratory and Emerging Infection Consortium. Pandemic Sciences Institute. University of Oxford, Oxford, United Kingdom
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.
Keywords
Drug TherapyLeishmaniasis
Neglected Tropical Diseases
Adverse events
Drug research and development
Drug administration
Colombia
Brazil
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