Patient-centric drug product development: Acceptability across patient populations – Science and evidence

Stegemann, Sven; Klingmann, Viviane; Reidemeister, Sibylle; Breitkreutz, Jörg

doi:10.1016/j.ejpb.2023.04.017

Cited by 2 publications

(2 citation statements)

References 24 publications

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“…Integration of RWE is crucial, offering insights into broader clinical scenarios and assessing the real-world effectiveness of polysaccharide-based BDSs [314]. Incorporating patient feedback in patient-centric trials facilitates comprehensive assessments of biocompatibility and efficacy [336]. However, the employment of such innovative designs involves complexities; they require sophisticated statistical methodologies, con-tinuous monitoring, and transparent, ethical decision-making.…”

Section: Efficacy and Safetymentioning

confidence: 99%

Translational Challenges and Prospective Solutions in the Implementation of Biomimetic Delivery Systems

Wang,

et al. 2023

Pharmaceutics

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Biomimetic delivery systems (BDSs), inspired by the intricate designs of biological systems, have emerged as a groundbreaking paradigm in nanomedicine, offering unparalleled advantages in therapeutic delivery. These systems, encompassing platforms such as liposomes, protein-based nanoparticles, extracellular vesicles, and polysaccharides, are lauded for their targeted delivery, minimized side effects, and enhanced therapeutic outcomes. However, the translation of BDSs from research settings to clinical applications is fraught with challenges, including reproducibility concerns, physiological stability, and rigorous efficacy and safety evaluations. Furthermore, the innovative nature of BDSs demands the reevaluation and evolution of existing regulatory and ethical frameworks. This review provides an overview of BDSs and delves into the multifaceted translational challenges and present emerging solutions, underscored by real-world case studies. Emphasizing the potential of BDSs to redefine healthcare, we advocate for sustained interdisciplinary collaboration and research. As our understanding of biological systems deepens, the future of BDSs in clinical translation appears promising, with a focus on personalized medicine and refined patient-specific delivery systems.

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Section: Efficacy and Safetymentioning

confidence: 99%

Translational Challenges and Prospective Solutions in the Implementation of Biomimetic Delivery Systems

Wang,

et al. 2023

Pharmaceutics

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“…Although progress has been achieved in drug formulation for the pediatric population, some problems remain to be solved. The design of pediatric drug formulation needs to be based on the patient-centric drug product design process (PCDPD), namely patient, drug, and drug product characteristics, that are translated into a Quality Target Product Profile (QTPP) to drive the pharmaceutical product design process [108,109]. Moreover, assessing the safety of excipients used in the formulation of pediatric pharmacotherapy is crucial.…”

Section: Administration Route and Pharmaceutical Dosage Forms In Pedi...mentioning

confidence: 99%

Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies

Domingues,

Jarak,

Veiga

et al. 2023

Pharmaceutics

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The paradigm of pediatric drug development has been evolving in a “carrot-and-stick”-based tactic to address population-specific issues. However, the off-label prescription of adult medicines to pediatric patients remains a feature of clinical practice, which may compromise the age-appropriate evaluation of treatments. Therefore, the United States and the European Pediatric Formulation Initiative have recommended applying nanotechnology-based delivery systems to tackle some of these challenges, particularly applying inorganic, polymeric, and lipid-based nanoparticles. Connected with these, advanced therapy medicinal products (ATMPs) have also been highlighted, with optimistic perspectives for the pediatric population. Despite the results achieved using these innovative therapies, a workforce that congregates pediatric patients and/or caregivers, healthcare stakeholders, drug developers, and physicians continues to be of utmost relevance to promote standardized guidelines for pediatric drug development, enabling a fast lab-to-clinical translation. Therefore, taking into consideration the significance of this topic, this work aims to compile the current landscape of pediatric drug development by (1) outlining the historic regulatory panorama, (2) summarizing the challenges in the development of pediatric drug formulation, and (3) delineating the advantages/disadvantages of using innovative approaches, such as nanomedicines and ATMPs in pediatrics. Moreover, some attention will be given to the role of pharmaceutical technologists and developers in conceiving pediatric medicines.

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Patient-centric drug product development: Acceptability across patient populations – Science and evidence

Cited by 2 publications

References 24 publications

Translational Challenges and Prospective Solutions in the Implementation of Biomimetic Delivery Systems

Translational Challenges and Prospective Solutions in the Implementation of Biomimetic Delivery Systems

Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies

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