{"title":"Informed Participation and Patient Empowerment: A Patient- Centered Approach to Improve Pediatric Clinical Research","authors":"Mariangela Lupo, A. Intini, Doriana Filannino","doi":"10.5772/INTECHOPEN.74528","DOIUrl":"https://doi.org/10.5772/INTECHOPEN.74528","url":null,"abstract":"Over the last years, a Europe-wide trend toward a patient-focused approach is develop- ing and is influencing the decision-making process related to the clinical research. This new vision aims to draw on patient knowledge and experience in order to deliver ben - efits for all stakeholders of the drug development process, optimizing the clinical study design. In this context, the “patient empowerment” concept has been developed as an approach encouraging the active participation and self-determination of the patients in the caring procedure. For this reason, in 2016, European Patients’ Academy (EUPATI) launched a public consultation that ended in September 2016 with the release of the EUPATI guidance for patient involvement in the medicine research and development process. Likewise, the recommendations on the “Summaries of Clinical Trial Results for Laypersons” for the Implementation of Regulation (EU) No 536/2014 recommended a clear and comprehensible communication of the clinical trial results to the patients. However, rarely, all these attempts for the patient involvement pay attention to the pedi - atric population needs. An innovative approach for the patients’ involvement in pediatric clinical research is represented by the Young Persons Advisory Groups , an organization composed of youths, patients, and carers, actively participating in clinical research and advising researchers and their teams.","PeriodicalId":410164,"journal":{"name":"The Management of Clinical Trials","volume":"50 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2018-06-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"124588687","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
I. Schulman, W. Balkan, R. Saltzman, DanielDaFonseca, Lina V. Caceres, Cindy Delgado, Marietsy V. Pujol, K. Ramdas, J. Tovar, Mayra Vidro-Casiano, J. Hare
{"title":"Unique Aspects of the Design of Phase I/II Clinical Trials of Stem Cell Therapy","authors":"I. Schulman, W. Balkan, R. Saltzman, DanielDaFonseca, Lina V. Caceres, Cindy Delgado, Marietsy V. Pujol, K. Ramdas, J. Tovar, Mayra Vidro-Casiano, J. Hare","doi":"10.5772/INTECHOPEN.72949","DOIUrl":"https://doi.org/10.5772/INTECHOPEN.72949","url":null,"abstract":"This chapter will review the unique aspects and limitations of the design of phase I/II (safety and efficacy) clinical trials of stem cell therapy. Although the classical pharmacologic principles applicable to drugs are not applicable to biologic (live cell) therapeutic agents, an important stage in the development of any new therapeutic agent is the establishment of an optimal dosage and delivery route. This can be particularly challenging when the treatment is a biologic agent, such as stem cells, that may exert its therapeutic effects via complex or poorly understood mechanisms. To date, clinical studies have shown inconsistent findings regarding the relationship between cell dose and clinical outcomes. This can be at least partially attributed to variations in donor cell type, source, characteristics, dosing/concentration, delivery route, underlying mechanisms of action, and efficacy endpoints tested. The current recommendations will be reviewed herein to give new investigators a general understanding of the unique issues that need to be considered and addressed when designing a stem cell therapy phase I/II clinical trial.","PeriodicalId":410164,"journal":{"name":"The Management of Clinical Trials","volume":"22 24","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2018-06-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"120932171","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Challenges in Paediatric Clinical Trials: How to Make It Feasible","authors":"G. Chiaruttini, M. Felisi, D. Bonifazi","doi":"10.5772/INTECHOPEN.72950","DOIUrl":"https://doi.org/10.5772/INTECHOPEN.72950","url":null,"abstract":"The number of paediatric clinical trials in EU has remarkably increased in the last decade in response to the implementation of the new Paediatric Regulation and incentives aiming to define the need of child-specific drug development. Nevertheless, the gap between the number of paediatric and adult-randomised controlled trials is still substantial in almost every major clinical specialty. Economic, ethical, technological, geographical and cultural factors can influence the paediatric drug development and can represent the challenges to be faced for a smooth conduction of a paediatric clinical trial. The need for trials and paediatric patient’s engagement to commensurate with the approved paediatric investigation plans is so high that it is crucial to correctly address key factors. Particular care should be taken to develop well-designed studies, with efficient man agement plans, experienced administrative and healthcare personnel, awareness of socio-cultural features of the geographic areas involved and good communication with patients and their families in order to ensure ‘trial preparedness’. A case study on a mul - tinational paediatric clinical trial, presented within the recently ended research project ‘DEferiprone Evaluation in Paediatrics (DEEP)’, was reported to exemplify some of the challenges encountered by the authors and the actions taken to overcome them.","PeriodicalId":410164,"journal":{"name":"The Management of Clinical Trials","volume":"199 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2018-06-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"124469041","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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