TY - JOUR
T1 - Developing therapies for rare tumors
T2 - Opportunities, challenges and progress
AU - Bradford, Diana
AU - Reilly, Karlyne M.
AU - Widemann, Brigitte C.
AU - Sandler, Abby
AU - Kummar, Shivaani
N1 - Copyright:
Copyright 2016 Elsevier B.V., All rights reserved.
PY - 2016/1/2
Y1 - 2016/1/2
N2 - Introduction: Rare tumors account for one fourth of adult tumors; in children, rare tumors represent approximately 15-20% of childhood malignancies, thus accounting for a significant burden of disease. The rarity of these individual diseases creates many challenges, from developing a thorough understanding of the disease pathophysiology, clinical characterization, to the conduct of meaningful clinical trials and eventually the development of effective therapies.Areas covered: Despite these challenges, substantial advances have been made in recent years including the development of novel clinical trial designs and endpoints including molecularly driven treatment trials that have resulted in approval of novel therapies for rare diseases. Collaboration amongst basic and clinical researchers, patient advocacy groups, industry and regulatory agencies has proven successful in select cases and holds promise for future progress in the treatment of rare tumors. In this review, we will highlight several examples of trials for rare tumors, with a focus on examples from pediatric oncology, where strong, nationwide collaborative groups have existed for many years.Expert opinion: Future progress in developing therapies for rare tumors will depend not only on continued scientific advances, but also on collaboration between investigators from various disciplines, institutions, regulatory agencies and patient advocacy groups.
AB - Introduction: Rare tumors account for one fourth of adult tumors; in children, rare tumors represent approximately 15-20% of childhood malignancies, thus accounting for a significant burden of disease. The rarity of these individual diseases creates many challenges, from developing a thorough understanding of the disease pathophysiology, clinical characterization, to the conduct of meaningful clinical trials and eventually the development of effective therapies.Areas covered: Despite these challenges, substantial advances have been made in recent years including the development of novel clinical trial designs and endpoints including molecularly driven treatment trials that have resulted in approval of novel therapies for rare diseases. Collaboration amongst basic and clinical researchers, patient advocacy groups, industry and regulatory agencies has proven successful in select cases and holds promise for future progress in the treatment of rare tumors. In this review, we will highlight several examples of trials for rare tumors, with a focus on examples from pediatric oncology, where strong, nationwide collaborative groups have existed for many years.Expert opinion: Future progress in developing therapies for rare tumors will depend not only on continued scientific advances, but also on collaboration between investigators from various disciplines, institutions, regulatory agencies and patient advocacy groups.
KW - BASKET trials
KW - molecular subtypes
KW - orphan drugs
KW - umbrella trials
UR - http://www.scopus.com/inward/record.url?scp=84952983602&partnerID=8YFLogxK
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U2 - 10.1517/21678707.2016.1120663
DO - 10.1517/21678707.2016.1120663
M3 - Review article
AN - SCOPUS:84952983602
SN - 2167-8707
VL - 4
SP - 93
EP - 103
JO - Expert Opinion on Orphan Drugs
JF - Expert Opinion on Orphan Drugs
IS - 1
ER -