TY - JOUR
T1 - Towards early inclusion of children in tuberculosis drugs trials
T2 - A consensus statement
AU - Nachman, Sharon
AU - Ahmed, Amina
AU - Amanullah, Farhana
AU - Becerra, Mercedes C.
AU - Botgros, Radu
AU - Brigden, Grania
AU - Browning, Renee
AU - Gardiner, Elizabeth
AU - Hafner, Richard
AU - Hesseling, Anneke
AU - How, Cleotilde
AU - Jean-Philippe, Patrick
AU - Lessem, Erica
AU - Makhene, Mamodikoe
AU - Mbelle, Nontombi
AU - Marais, Ben
AU - McIlleron, Helen
AU - McNeeley, David F.
AU - Mendel, Carl
AU - Murray, Stephen
AU - Navarro, Eileen
AU - Anyalechi, E. Gloria
AU - Porcalla, Ariel R.
AU - Powell, Clydette
AU - Powell, Mair
AU - Rigaud, Mona
AU - Rouzier, Vanessa
AU - Samson, Pearl
AU - Schaaf, H. Simon
AU - Shah, Seema Kirti
AU - Starke, Jeff
AU - Swaminathan, Soumya
AU - Wobudeya, Eric
AU - Worrell, Carol
N1 - Funding Information:
EL's employer, the Treatment Action Group, receives non-commercial support from the Bill & Melinda Gates Foundation, the TB Alliance, and receives funding from the US Department of Veteran Affairs to coordinate community engagement for the US Centers for Disease Control Tuberculosis Trials Consortium. DFM is employed by Novartis. JS is a member of the Data and Safety Monitoring Board for paediatric trials of delamanid. EG, CM and SM are employed by the TB Alliance. The other authors declare no competing interests.
Funding Information:
We thank the following individuals for assistance in the planning and conduct of the workshop or drafting of the manuscript, or both: Sheryl Zwerski, Sarah Read, Larry Fox, Devasena Gnanashanmugam, Judi Miller, Ellen O'Gara, Paul Sato, Peter Kim, Tyseaia Squirewell McFarlane, Rahel Abebe, and Andrea Williams. This work was funded by National Institute of Allergy and Infectious Diseases and National Institute of Health. This project has also been funded in part with federal funds from the Department of Health and Human Services. The views expressed in written conference materials or publications and by speakers and moderators at HHS-sponsored conferences, do not necessarily show the official policies of the Department of Health and Human Services (DHHS) or individual DHHS or other US government agencies including the National Institute of Health, the Center for Disease Control and Prevention, the Food and Drug Administration or the US Agency for International Development; nor does mention of trade names, commercial practices, or organisations imply endorsement by the US Government. The views expressed in this consensus statement are the personal views of the authors and cannot be understood or quoted as made on behalf of the position of the European Medicines Agency, or one of its committees or working parties.
Publisher Copyright:
© 2015 Elsevier Ltd.
PY - 2015/6/1
Y1 - 2015/6/1
N2 - Children younger than 18 years account for a substantial proportion of patients with tuberculosis worldwide. Available treatments for paediatric drug-susceptible and drug-resistant tuberculosis, albeit generally effective, are hampered by high pill burden, long duration of treatment, coexistent toxic effects, and an overall scarcity of suitable child-friendly formulations. Several new drugs and regimens with promising activity against both drug-susceptible and drug-resistant strains have entered clinical development and are either in various phases of clinical investigation or have received marketing authorisation for adults; however, none have data on their use in children. This consensus statement, generated from an international panel of opinion leaders on childhood tuberculosis and incorporating reviews of published literature from January, 2004, to May, 2014, addressed four key questions: what drugs or regimens should be prioritised for clinical trials in children? Which populations of children are high priorities for study? When can phase 1 or 2 studies be initiated in children? What are the relevant elements of clinical trial design? The consensus panel found that children can be included in studies at the early phases of drug development and should be an integral part of the clinical development plan, rather than studied after regulatory approval in adults is obtained.
AB - Children younger than 18 years account for a substantial proportion of patients with tuberculosis worldwide. Available treatments for paediatric drug-susceptible and drug-resistant tuberculosis, albeit generally effective, are hampered by high pill burden, long duration of treatment, coexistent toxic effects, and an overall scarcity of suitable child-friendly formulations. Several new drugs and regimens with promising activity against both drug-susceptible and drug-resistant strains have entered clinical development and are either in various phases of clinical investigation or have received marketing authorisation for adults; however, none have data on their use in children. This consensus statement, generated from an international panel of opinion leaders on childhood tuberculosis and incorporating reviews of published literature from January, 2004, to May, 2014, addressed four key questions: what drugs or regimens should be prioritised for clinical trials in children? Which populations of children are high priorities for study? When can phase 1 or 2 studies be initiated in children? What are the relevant elements of clinical trial design? The consensus panel found that children can be included in studies at the early phases of drug development and should be an integral part of the clinical development plan, rather than studied after regulatory approval in adults is obtained.
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U2 - 10.1016/S1473-3099(15)00007-9
DO - 10.1016/S1473-3099(15)00007-9
M3 - Review article
C2 - 25957923
AN - SCOPUS:84929511425
SN - 1473-3099
VL - 15
SP - 711
EP - 720
JO - The Lancet Infectious Diseases
JF - The Lancet Infectious Diseases
IS - 6
ER -
