| Author | Castro, Maria del Mar | |
| Author | Erber, Astrid | |
| Author | Arana, Byron | |
| Author | Cota, Glaucia Fernandes | |
| Author | Denkinger, Claudia | |
| Author | Nicole, Harrison | |
| Author | Julia, Kutyi | |
| Author | Carvajal, Liliana López | |
| Author | Plugge, Emma | |
| Author | Walochnik, Julia | |
| Author | Olliaro, Piero L. | |
| Access date | 2023-08-02T13:49:55Z | |
| Available date | 2023-08-02T13:49:55Z | |
| Document date | 2023 | |
| Citation | CASTRO, Maria del Mar et 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. The Lancet, p. 1-24, 2023. doi.org/10.2139/ssrn.4380035. | en_US |
| ISSN | 0140-6736 | en_US |
| URI | https://www.arca.fiocruz.br/handle/icict/59878 | |
| Language | eng | en_US |
| Publisher | Elsevier | en_US |
| Rights | restricted 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 | Preprint | en_US |
| Abstract | 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. | en_US |
| Affilliation | Centro Internacional de Entrenamiento de Investigaciones Médicas. Cali, Colombia | en_US |
| Affilliation | Medical University of Vienna. Department of Epidemiology. . Vienna, Switzerland | en_US |
| Affilliation | Drugs for Neglected Diseases Initiative. | en_US |
| Affilliation | Oswaldo Cruz Foundation. Instituto Rene Rachou. Belo Horizonte, MG, Brasil | en_US |
| Affilliation | Heidelberg University Hospital. Division of Infectious Disease and Tropical Medicine and German Centre for Infection Research. Heidelberg, Germain | en_US |
| Affilliation | Medical University of Vienna. Division of Infectious Diseases and Tropical Medicine. Vienna, Switzerland | en_US |
| Affilliation | Medical University of Vienna. Division of Infectious Diseases and Tropical Medicine. Vienna, Switzerland | en_US |
| Affilliation | Universidad de Antioquia. Program for the Study and Control of Tropical Diseases. Antioquia, Colômbia. | en_US |
| Affilliation | University of Southampton. School of Primary Care, Population Sciences and Medical Education. Southampton, UK | en_US |
| Affilliation | Medical University of Vienna. Institute of Specific Prophylaxis and Tropical Medicine. Vienna, Switzerland | en_US |
| Affilliation | University of Oxford. Nuffield Department of Medicine. Oxford, UK | en_US |
| Subject | drug development | en_US |
| Subject | target product profile | en_US |
| Subject | cutaneous leishmaniasis | en_US |
| Subject | neglected tropical diseases | en_US |
| Subject | patient involvement | en_US |
| Subject | patient consultation | en_US |
| Embargo date | 3100-12-31 | |
