Outcomes and endpoints in clinical trials supporting the marketing authorisation of treatments in paediatric acute lymphoblastic leukaemia

Benjamin Micallef; Robert Nistico; Ole Weis Bjerrum; Sinan Bardakci Sarac; Dianne Butler; Anthony Serracino-Inglott; John Joseph Borg

doi:10.1016/j.drudis.2022.05.015

Outcomes and endpoints in clinical trials supporting the marketing authorisation of treatments in paediatric acute lymphoblastic leukaemia

Benjamin Micallef, Robert Nistico, Ole Weis Bjerrum, Sinan Bardakci Sarac, Dianne Butler, Anthony Serracino-Inglott, John Joseph Borg^*

^*Kontaktforfatter

KI, OUH, Forskningsenhed for Hæmatologi (Odense)

Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › peer review

Abstract

The improvement in acute lymphoblastic leukaemia (ALL) treatment has led research efforts to focus on the unmet medical needs of an increasingly smaller patient cohort with resistant leukaemia and to develop more-targeted agents. Survival and response rates remain the most-prevalent endpoints in paediatric ALL research, but other intermediate clinical endpoints and molecular biomarkers for efficacy and mid- and long-term safety endpoints are also being investigated. The success of current ALL treatment appears to be driving new paradigms to optimise clinical drug development, while at the same time, regulatory tools in place are supporting meaningful drug development in the area.

Originalsprog	Engelsk
Tidsskrift	Drug Discovery Today
Vol/bind	27
Udgave nummer	9
Sider (fra-til)	2440-2466
ISSN	1359-6446
DOI	https://doi.org/10.1016/j.drudis.2022.05.015
Status	Udgivet - sep. 2022

Bibliografisk note

Funding Information:
We thank Francesco Pignatti, Julio Delgado, and Ralf Herold for helpful discussions.

Dokumenter og links

10.1016/j.drudis.2022.05.015

Citationsformater

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title = "Outcomes and endpoints in clinical trials supporting the marketing authorisation of treatments in paediatric acute lymphoblastic leukaemia",

abstract = "The improvement in acute lymphoblastic leukaemia (ALL) treatment has led research efforts to focus on the unmet medical needs of an increasingly smaller patient cohort with resistant leukaemia and to develop more-targeted agents. Survival and response rates remain the most-prevalent endpoints in paediatric ALL research, but other intermediate clinical endpoints and molecular biomarkers for efficacy and mid- and long-term safety endpoints are also being investigated. The success of current ALL treatment appears to be driving new paradigms to optimise clinical drug development, while at the same time, regulatory tools in place are supporting meaningful drug development in the area.",

keywords = "Childhood acute lymphoblastic leukaemia, Clinical drug development, Clinical trials, Endpoints in oncology, EU regulation, Humans, Antineoplastic Agents, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Child, Cohort Studies, Marketing",

author = "Benjamin Micallef and Robert Nistico and Bjerrum, {Ole Weis} and Sarac, {Sinan Bardakci} and Dianne Butler and Anthony Serracino-Inglott and Borg, {John Joseph}",

note = "Funding Information: We thank Francesco Pignatti, Julio Delgado, and Ralf Herold for helpful discussions.",

year = "2022",

month = sep,

doi = "10.1016/j.drudis.2022.05.015",

language = "English",

volume = "27",

pages = "2440--2466",

journal = "Drug Discovery Today",

issn = "1359-6446",

publisher = "Elsevier",

number = "9",

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TY - JOUR

T1 - Outcomes and endpoints in clinical trials supporting the marketing authorisation of treatments in paediatric acute lymphoblastic leukaemia

AU - Micallef, Benjamin

AU - Nistico, Robert

AU - Bjerrum, Ole Weis

AU - Sarac, Sinan Bardakci

AU - Butler, Dianne

AU - Serracino-Inglott, Anthony

AU - Borg, John Joseph

N1 - Funding Information: We thank Francesco Pignatti, Julio Delgado, and Ralf Herold for helpful discussions.

PY - 2022/9

Y1 - 2022/9

N2 - The improvement in acute lymphoblastic leukaemia (ALL) treatment has led research efforts to focus on the unmet medical needs of an increasingly smaller patient cohort with resistant leukaemia and to develop more-targeted agents. Survival and response rates remain the most-prevalent endpoints in paediatric ALL research, but other intermediate clinical endpoints and molecular biomarkers for efficacy and mid- and long-term safety endpoints are also being investigated. The success of current ALL treatment appears to be driving new paradigms to optimise clinical drug development, while at the same time, regulatory tools in place are supporting meaningful drug development in the area.

AB - The improvement in acute lymphoblastic leukaemia (ALL) treatment has led research efforts to focus on the unmet medical needs of an increasingly smaller patient cohort with resistant leukaemia and to develop more-targeted agents. Survival and response rates remain the most-prevalent endpoints in paediatric ALL research, but other intermediate clinical endpoints and molecular biomarkers for efficacy and mid- and long-term safety endpoints are also being investigated. The success of current ALL treatment appears to be driving new paradigms to optimise clinical drug development, while at the same time, regulatory tools in place are supporting meaningful drug development in the area.

KW - Childhood acute lymphoblastic leukaemia

KW - Clinical drug development

KW - Clinical trials

KW - Endpoints in oncology

KW - EU regulation

KW - Humans

KW - Antineoplastic Agents

KW - Precursor Cell Lymphoblastic Leukemia-Lymphoma

KW - Child

KW - Cohort Studies

KW - Marketing

U2 - 10.1016/j.drudis.2022.05.015

DO - 10.1016/j.drudis.2022.05.015

M3 - Journal article

C2 - 35597514

AN - SCOPUS:85130912522

SN - 1359-6446

VL - 27

SP - 2440

EP - 2466

JO - Drug Discovery Today

JF - Drug Discovery Today

IS - 9

ER -

Outcomes and endpoints in clinical trials supporting the marketing authorisation of treatments in paediatric acute lymphoblastic leukaemia

Abstract

Bibliografisk note

Dokumenter og links

Fingeraftryk

Citationsformater