1. 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.
- Author
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Castro, María del Mar, Erber, Astrid C., Arana, Byron, Cota, Gláucia, Denkinger, Claudia M., Harrison, Nicole, Kutyi, Julia, López-Carvajal, Liliana, Plugge, Emma, Walochnik, Julia, and Olliaro, Piero
- Subjects
CUTANEOUS leishmaniasis ,NEGLECTED diseases ,PATIENTS' attitudes ,PATIENT preferences ,DRUG development ,LEISHMANIA mexicana - 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. Author summary: Our study addresses the challenge of involving patients in defining which medical product would work for their condition. When designing a new medical product, it is customary to identify a "target product profile" (TPP), which identifies the characteristics the product should have to meet in order to address the medical need it is intended for. For the product to be used as intended and achieve the desired effects, it should be adapted to the conditions and the people who will use it, so, patients' views are important, but rarely heard. Here, we use as an example cutaneous leishmaniasis, a skin and mucosal disease caused by a protozoan parasite, which disproportionally affects poor people across tropical and subtropical areas of the world. We collected patients' views about product safety, efficacy, costs, treatment administration, and perceived barriers, that contribute to specifying product characteristics in the TPP. Overall, our study contributes to the limited body of knowledge with an example and an adaptable methodology to give patients a voice in designing adapted medical products. Patients input may also contribute to redefining aspects of a TPP, such as affordability, instead of just the cost per unit. The methodology used here can be adapted and used for other neglected diseases to give patients a voice in designing medical products. [ABSTRACT FROM AUTHOR]
- Published
- 2024
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