Characteristics of non-randomised studies using comparisons with external controls submitted for regulatory approval in the USA and Europe: a systematic review

BMJ Open
Sarah GoringNick Freemantle

Abstract

Non-randomised clinical trial designs involving comparisons against external controls or specific standards can be used to support regulatory submissions for indications in diseases that are rare, with high unmet need, without approved therapies and/or where placebo is considered unethical. The objective of this review was to summarise the characteristics of non-randomised trials submitted to the European Medicines Agency (EMA) or Food and Drug Administration (FDA) for indications in haematological cancers, haematological non-malignant conditions, stem cell transplants or rare metabolic diseases. We conducted systematic searches of EMA databases of conditional approvals, exceptional circumstances, or orphan drug designations and FDA inventories of orphan drug designations, accelerated approvals, breakthrough therapy, fast-track and priority approvals. Products were included if reviewed by at least one agency between 2005 and 2017, the primary evidence base was non-randomised trial(s) and the indication was for haematological cancers, stem cell transplantation, haematological conditions or rare metabolic conditions. We identified 43 eligible indication-specific products using non-randomised study designs involving comparisons wi...Continue Reading

Citations

Feb 16, 2019·Drugs·Kate McKeage
Nov 30, 2019·International Journal of Technology Assessment in Health Care·Tatiane Bomfim RibeiroMoacyr Roberto Cuce Nobre
Feb 16, 2021·Journal of Orthopaedic Trauma·Robert ZuraJohn I Mackowiak
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