Rational and practical considerations to guide a target product profile for patient-centric drug product development with measurable patient outcomes – A proposed roadmap

Stegemann, Sven; Sheehan, Liz; Rossi, Alessandra; Barrett, Andrew F.; Paudel, Amrit; Crean, Abina M.; Ruiz, Fabrice; Bresciani, Massimo; Liu, Fang; Shariff, Zakia; Shine, Margarete; Schmelzer, Christel; Pensé‐Lhéritier, Anne‐Marie

doi:10.1016/j.ejpb.2022.06.006

Cited by 16 publications

(9 citation statements)

References 26 publications

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“…Central among the barriers to involving patients in drug development is the lack of methodological guidance, as reported by drug developers, patients and patient advocates, regulators and funders [ 47 , 48 ]. Recent initiatives have addressed this at a general level [ 16 , 17 ], and our study provides methodological guidance using an open, exploratory approach taking an NTD as an example. We designed and published an interview topic guide that allows an in-depth exploration of experiences and preferences [ 26 ], and reflections upon these, instead of focusing on TPP categories a priori .…”

Section: Discussionmentioning

confidence: 99%

“…Few studies describe patients’ involvement as stakeholders in the construction of a TPP [ 15 ]. A high-level guideline [ 16 ] and a roadmap [ 17 ] have been published recently. Adepoyibi et al [ 18 ] conducted a survey among laboratory personnel, national tuberculosis control program managers, donors, technical experts, patients and researchers, and asked them to rank the items in a TPP for tuberculosis diagnostic tools by their perceived importance.…”

Section: Introductionmentioning

confidence: 99%

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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

Castro,

Erber,

Arana

et al. 2024

PLoS Negl Trop Dis

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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.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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

Castro,

Erber,

Arana

et al. 2024

PLoS Negl Trop Dis

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“…Therefore, adequate acceptability is essential for the development of drug products [7]. The needs of the intended subset of patients have to be identified at the beginning of drug product development [7,8]. Drug product characteristics influencing the acceptability of medicines in older adults are stated in a reflection paper by the European Medicines Agency.…”

Section: Introductionmentioning

confidence: 99%

Influence of Solid Oral Dosage Form Characteristics on Swallowability, Visual Perception, and Handling in Older Adults

et al. 2023

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Swallowability, visual perception, and any handling to be conducted prior to use are all influence factors on the acceptability of an oral dosage form by the patient. Knowing the dosage form preferences of older adults, as the major group of medication end users, is needed for patient-centric drug development. This study aimed at evaluating the ability of older adults to handle tablets as well as to assess the anticipated swallowability of tablets, capsules, and mini tablets based on visual perception. The randomized intervention study included 52 older adults (65 to 94 years) and 52 younger adults (19 to 36 years). Within the tested tablets, ranging from 125 mg up to 1000 mg in weight and being of different shapes, handling was not seen as the limiting factor for the decision on appropriate tablet size. However, the smallest sized tablets were rated worst. According to visual perception, the limit of acceptable tablet size was reached at around 250 mg for older adults. For younger adults, this limit was shifted to higher weights and was dependent on the tablet shape. Differences in anticipated swallowability with respect to tablet shapes were most pronounced for tablets of 500 mg and 750 mg in weight, independent of the age category. Capsules performed worse compared to tablets, while mini tablets appeared as a possible alternative dosage form to tablets of higher weight. Within the deglutition part of this study, swallowability capabilities of the same populations were assessed and have been reported previously. Comparing the present results with the swallowing capabilities of the same populations with respect to tablets, it shows adults’ clear self-underestimation of their ability to swallow tablets independent of their age.

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“…With a growing pharmaceutical industry and regulatory agency focus on the development of patient-centric medicines and ever-increasing pressure on drug development cycle time, , the early selection of an optimal formulation for clinical studies becomes critically important. To expedite drug product design and development processes, innovative in vitro and in silico tools are necessary − which can be used to reliably predict and rapidly assess critical bioavailability attributes.…”

Section: Introductionmentioning

confidence: 99%

Digitalizing the TIM-1 Model using Computational Approaches–Part One: TIM-1 Data Explorer

Sarcevica,

Hens,

Tomaszewska

et al. 2023

Mol. Pharmaceutics

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The TIM-1 gastrointestinal model is one of the most advanced in vitro systems currently available for biorelevant dissolution testing. This technology, the initial version of which was developed nearly 30 years ago and has been subject to a number of significant updates over this period, simulates the dynamic environment of the human gastrointestinal tract, including pH, transfer times, secretion of bile, enzymes, and electrolytes. In the pharmaceutical industry, the TIM-1 system is used to support drug product design and provide a biopredictive assessment of drug product performance. Typically, the bioaccessibility data sets generated by TIM-1 experiments are used to qualitatively compare formulation performance, and the use of bioaccessibility data as inputs for physiologically based pharmacokinetic (PBPK) modeling for quantitative predictions is limited. To expand the utility of the TIM-1 model beyond standard bioaccessibility measurements (which define the fraction available for absorption), we have developed a computational tool, TIM-1 Data Explorer, to describe the fluid and mass balance within the TIM-1 system. The use of this tool allows a detailed inspection and in-depth interpretation of the experimental data. In addition to mass balance calculation, this model also can be used to describe the critical processes a drug substance would undergo during a TIM-1 experiment, such as dissolution, precipitation on transfer from the stomach to duodenum, and redissolution. The TIM-1 Data Explorer was validated in two case studies. In the first case study with paracetamol, we have shown the ability of the simulator to adequately describe mass transfer events within the TIM-1 system, and in the second study with a weakly basic in-house compound, PF-07059013, the TIM-1 Data Explorer was successfully used to describe dissolution and precipitation processes.

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Rational and practical considerations to guide a target product profile for patient-centric drug product development with measurable patient outcomes – A proposed roadmap

Cited by 16 publications

References 26 publications

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

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

Influence of Solid Oral Dosage Form Characteristics on Swallowability, Visual Perception, and Handling in Older Adults

Digitalizing the TIM-1 Model using Computational Approaches–Part One: TIM-1 Data Explorer

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