N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine—A Review

Pediatric oncology is a critical area where the more efficient development of new treatments is urgently needed. The speed of approval of new drugs is still limited by regulatory requirements and a lack of innovative designs appropriate for trials in children. Childhood cancers meet the criteria of...

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Autores principales:	Michal Kyr, Adam Svobodnik, Radka Stepanova, Renata Hejnova
Formato:	article
Lenguaje:	EN
Publicado:	MDPI AG 2021
Materias:	small samples N-of-1 rare diseases personalized treatment pediatric oncology design Neoplasms. Tumors. Oncology. Including cancer and carcinogens RC254-282
Acceso en línea:	https://doaj.org/article/e362beda12c3412aac9b32d23e3902c8
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spelling	oai:doaj.org-article:e362beda12c3412aac9b32d23e3902c82021-11-11T15:31:15ZN-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine—A Review10.3390/cancers132154282072-6694https://doaj.org/article/e362beda12c3412aac9b32d23e3902c82021-10-01T00:00:00Zhttps://www.mdpi.com/2072-6694/13/21/5428https://doaj.org/toc/2072-6694Pediatric oncology is a critical area where the more efficient development of new treatments is urgently needed. The speed of approval of new drugs is still limited by regulatory requirements and a lack of innovative designs appropriate for trials in children. Childhood cancers meet the criteria of rare diseases. Personalized medicine brings it even closer to the horizon of individual cases. Thus, not all the traditional research tools, such as large-scale RCTs, are always suitable or even applicable, mainly due to limited sample sizes. Small samples and traditional versus subject-specific evidence are both distinctive issues in personalized pediatric oncology. Modern analytical approaches and adaptations of the paradigms of evidence are warranted. We have reviewed innovative trial designs and analytical methods developed for small populations, together with individualized approaches, given their applicability to pediatric oncology. We discuss traditional population-based and individualized perspectives of inferences and evidence, and explain the possibilities of using various methods in pediatric personalized oncology. We find that specific derivatives of the original N-of-1 trial design adapted for pediatric personalized oncology may represent an optimal analytical tool for this area of medicine. We conclude that no particular N-of-1 strategy can provide a solution. Rather, a whole range of approaches is needed to satisfy the new inferential and analytical paradigms of modern medicine. We reveal a new view of cancer as continuum model and discuss the “evidence puzzle”.Michal KyrAdam SvobodnikRadka StepanovaRenata HejnovaMDPI AGarticlesmall samplesN-of-1rare diseasespersonalized treatmentpediatric oncologydesignNeoplasms. Tumors. Oncology. Including cancer and carcinogensRC254-282ENCancers, Vol 13, Iss 5428, p 5428 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	small samples N-of-1 rare diseases personalized treatment pediatric oncology design Neoplasms. Tumors. Oncology. Including cancer and carcinogens RC254-282
spellingShingle	small samples N-of-1 rare diseases personalized treatment pediatric oncology design Neoplasms. Tumors. Oncology. Including cancer and carcinogens RC254-282 Michal Kyr Adam Svobodnik Radka Stepanova Renata Hejnova N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine—A Review
description	Pediatric oncology is a critical area where the more efficient development of new treatments is urgently needed. The speed of approval of new drugs is still limited by regulatory requirements and a lack of innovative designs appropriate for trials in children. Childhood cancers meet the criteria of rare diseases. Personalized medicine brings it even closer to the horizon of individual cases. Thus, not all the traditional research tools, such as large-scale RCTs, are always suitable or even applicable, mainly due to limited sample sizes. Small samples and traditional versus subject-specific evidence are both distinctive issues in personalized pediatric oncology. Modern analytical approaches and adaptations of the paradigms of evidence are warranted. We have reviewed innovative trial designs and analytical methods developed for small populations, together with individualized approaches, given their applicability to pediatric oncology. We discuss traditional population-based and individualized perspectives of inferences and evidence, and explain the possibilities of using various methods in pediatric personalized oncology. We find that specific derivatives of the original N-of-1 trial design adapted for pediatric personalized oncology may represent an optimal analytical tool for this area of medicine. We conclude that no particular N-of-1 strategy can provide a solution. Rather, a whole range of approaches is needed to satisfy the new inferential and analytical paradigms of modern medicine. We reveal a new view of cancer as continuum model and discuss the “evidence puzzle”.
format	article
author	Michal Kyr Adam Svobodnik Radka Stepanova Renata Hejnova
author_facet	Michal Kyr Adam Svobodnik Radka Stepanova Renata Hejnova
author_sort	Michal Kyr
title	N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine—A Review
title_short	N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine—A Review
title_full	N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine—A Review
title_fullStr	N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine—A Review
title_full_unstemmed	N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine—A Review
title_sort	n-of-1 trials in pediatric oncology: from a population-based approach to personalized medicine—a review
publisher	MDPI AG
publishDate	2021
url	https://doaj.org/article/e362beda12c3412aac9b32d23e3902c8
work_keys_str_mv	AT michalkyr nof1trialsinpediatriconcologyfromapopulationbasedapproachtopersonalizedmedicineareview AT adamsvobodnik nof1trialsinpediatriconcologyfromapopulationbasedapproachtopersonalizedmedicineareview AT radkastepanova nof1trialsinpediatriconcologyfromapopulationbasedapproachtopersonalizedmedicineareview AT renatahejnova nof1trialsinpediatriconcologyfromapopulationbasedapproachtopersonalizedmedicineareview
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N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine—A Review

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