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Expert Opinion on Orphan Drugs Volume 2, 2014 - Issue 8
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Editorials

Coping with small populations of patients in clinical trials

Daniel J O’ConnorMedicines and Healthcare Products Regulatory Agency, 151 Buckingham Palace Road, Victoria, London SW1W 9SZ, UK+020 3080 6305; Daniel.O’[email protected]
, BSc MB ChB MSc PhD MFPM (Expert Medical Assessor and UK Representative on COMP) &
Robert James HemmingsStatistics and Pharmacokinetics Unit, Medicines and Healthcare Products Regulatory Agency, 151 Buckingham Palace Road, Victoria, London SW1W 9SZ, UK;Medicines and Healthcare Products Regulatory Agency, 151 Buckingham Palace Road, Victoria, London SW1W 9SZ, UK
, BSc MSc CStat (Unit Manager) (CHMP Member and Chair of SAWP) (Unit Manager) (CHMP Member and Chair of SAWP)
Pages 765-768 | Published online: 05 Jul 2014

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Daniel J. O’Connor, Jenny Buckland, Neil Almond, Jennifer Boyle, Carmen Coxon, Eleni Gaki, Javier Martin, Giada Mattiuzzo, Clive Metcalfe, Mark Page, Nicola Rose, Begona Valdazo-Gonzalez, Yuan Zhao & Christian K. Schneider. (2019) Commonly setting biological standards in rare diseases. Expert Opinion on Orphan Drugs 7:7-8, pages 305-314.
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Daniel J O’Connor. (2018) Editor’s foreword: evolving the rare cancer field. Expert Opinion on Orphan Drugs 6:9, pages 507-508.
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Articles from other publishers (6)

Saeko Kutsunugi, Kumiko Tsujino, Kyoko Murakami, Kazuko Iida, Tsugiko Gima, Yumiko Endoh, Yoko Tamashiro, Teresa E. Stone & Jun Kobayashi. (2020) Mothers' experiences of parenting a child with chromosomal structural abnormalities: The journey to acceptance. Japan Journal of Nursing Science 18:2.
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Diogo A. Fonseca, Inês Amaral, Ana Catarina Pinto & Maria Dulce Cotrim. (2019) Orphan drugs: major development challenges at the clinical stage. Drug Discovery Today 24:3, pages 867-872.
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Simon Day, Anneliene Hechtelt Jonker, Lilian Pek Lian Lau, Ralf-Dieter Hilgers, Ilan Irony, Kristina Larsson, Kit CB Roes & Nigel Stallard. (2018) Recommendations for the design of small population clinical trials. Orphanet Journal of Rare Diseases 13:1.
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Kit C. B. Roes. (2016) A framework: make it useful to guide and improve practice of clinical trial design in smaller populations. BMC Medicine 14:1.
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Lemuel J. Pelentsov, Andrea L. Fielder, Thomas A. Laws & Adrian J. Esterman. (2016) The supportive care needs of parents with a child with a rare disease: results of an online survey. BMC Family Practice 17:1.
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Lemuel J. Pelentsov, Andrea L. Fielder & Adrian J. Esterman. (2016) The Supportive Care Needs of Parents With a Child With a Rare Disease: A Qualitative Descriptive Study. Journal of Pediatric Nursing 31:3, pages e207-e218.
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