Trials are needed before new devices are used in routine practice in EuropeBMJ 2013; 346 doi: https://doi.org/10.1136/bmj.f1646 (Published 18 March 2013) Cite this as: BMJ 2013;346:f1646
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I was surprised that the Edwards-SAPIEN aortic valve trial by Makkar and colleagues was shortlisted as one of the six papers for the “Research Paper of the Year Award 2013” (BMJ Feature Article, 24 April 2013) [1,2] as a recent BMJ article has highlighted how the investigators excluded 20% of the patients randomly allocated into this trial.
The shortlisted paper by Makkar and colleagues reports on the two year mortality outcome of Edwards-SAPIEN valve insertion versus usual medical care among 358 patients with severe and inoperable aortic valve stenosis. It is an update of a previous paper by the same investigators and both papers report the superiority of Edwards-SAPIEN valve insertion.[2,4] Unfortunately neither of these papers disclose the existence of a “randomised continued access protocol” to this trial which randomised a further 41 patients to Edwards-SAPIEN valve insertion and 50 patients to usual medical care over a 7-month period from March 2009. The data relating to these 91 patients has not been published, but are contained in a limited format within a briefing document produced for the US Food and Drug Administration (FDA) in July 2011.
Among these undisclosed trial participants some 13 (32%) individuals randomised to Edwards-SAPIEN valve insertion died compared to 10 (20%) randomised to usual medical care. This equates to one additional death for every 8 patients receiving the Edwards-SAPIEN valve. The “continued access protocol” randomised patients up until the end of September 2009. At the time of publication of the shortlisted paper the Edwards-SAPIEN trial would have accumulated more than two and a half years of mortality data; it will have now accumulated more than three and a half years of data concerning deaths among these 91 patients
The BMJ selected this paper “because it provides patients and doctors with high quality, real life evidence to weigh up the benefits and risks of this procedure”. If patients, doctors and policymakers are to make truly informed decisions then full disclosure and analysis of all patients who consented to be randomised into the Edwards-SAPIEN valve trial are required.
 Groves T. Research Paper of the Year award 2013. BMJ 2013; 346
doi: http://dx.doi.org/10.1136/bmj.f2512 (Published 24 April 2013)
 Makkar RR, Fontana GP, Jilaihawi H, Kapadia S, Pichard AD, Douglas PS, et al. PARTNER Trial Investigators. Transcatheter aortic-valve replacement for inoperable severe aortic stenosis. N Engl J Med 2012;366:1696-704
 Van Brabandt H, Neyt M, Hulstaert F. Transcatheter aortic valve implantation (TAVI): risky and costly. BMJ 2012;345:e4710. doi: 10.1136/bmj.e4710.
 Leon MB, Smith CR, Mack M, Miller DC, Moses JW, Svensson LG, et al.
PARTNER Trial Investigators. Transcatheter aortic-valve implantation for aortic stenosis in patients who cannot undergo surgery. N Engl J Med 2010;363:1597-607.
 Food and Drug Administration (FDA). The Edwards SAPIEN® THV Transcatheter Heart Valve System for Patients With Severe Aortic Stenosis Who Are Not Candidates for Conventional Open-Heart Aortic Valve Replacement Surgery. Briefing Document for the Circulatory Systems Device Panel Advisory Committee. Other Experience in Inoperbale Patients. Randomized, Continued-Access Patients. FDA (20 July) 2011: 95-99.
Competing interests: I have previously advised NHS Grampian Health Board concerning the provision of trans-aortic valve insertion (TAVI) and I have also been involved in assessing whether individual patients with inoperable aortic stenosis should be referred for TAVI based on the data published by Makkar and colleagues.