Rethinking the Food and Drug Administration's 2013 guidance on developing drugs for early‐stage Alzheimer's disease

Alzheimer's & Dementia - Tập 10 - Trang 247-250 - 2014
Lon S. Schneider1
1Keck School of Medicine, University of Southern California, Los Angeles, CA USA

Tóm tắt

AbstractThe February 2013 Food and Drug Administration (FDA) draft guidance for developing drugs for early‐stage Alzheimer's disease (AD) creates certain challenges as they guide toward the use of one cognitive outcome to gain accelerated marketing approval for preclinical AD drugs, and a composite clinical scale – the Clinical Dementia Rating Scale in particular – for the primary outcome for prodromal AD clinical trials.In light of the developing knowledge regarding early stage diagnoses and clinical trials outcomes, we recommend that FDA describe its requirements for validating preclinical AD diagnoses for drug development purposes, maintain the principle for requiring coprimary outcomes, and encourage the advancement of outcomes for early stage AD trials. The principles for drug development for early stage AD should not differ from those for clinical AD, especially as the diagnoses of prodromal and early AD impinge on each other. The FDA should not recommend that a composite scale be used as a sole primary efficacy outcome to support a marketing claim unless it requires that the cognitive and functional components of such a scale are demonstrated to be individually meaningful. The current draft guidelines may inadvertently constrain efforts to better assess the clinical effects of new drugs and inhibit innovation in an area where evidence‐based clinical research practices are still evolving.

Tài liệu tham khảo

U.S. Food and Drug Administration.Guidance for industry Alzheimer's disease: developing drugs for the treatment of early stage disease (FDA‐2013‐D‐0077) DRAFT.February 2013 [accessed 2013 June 30]. Available from:http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM338287.pdf. 10.1016/j.jalz.2011.03.008 10.1016/S1474-4422(10)70223-4 Draft guidance for industry on Alzheimer's disease: developing drugs for the treatment of early stage disease [transcript from FDA Webinar].March 28 2013. [accessed 2013 October 20]. Available from:http://www.fda.gov/training/guidancewebinars/ucm367786.htm. 10.1212/01.wnl.0000219668.47116.e6 10.1056/NEJMoa050151 10.1212/WNL.0b013e3181cb3e25 10.1016/j.jalz.2011.05.1966 J.C.Morris.Revised criteria for mild cognitive impairment may compromise the diagnosis of Alzheimer disease dementia.Arch Neurol 2012 10.1016/j.jalz.2011.03.005 10.1056/NEJMp1302513 10.1212/WNL.43.11.2412-a 10.1016/S1474-4422(08)70090-5
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Alzheimer's & Dementia

Tập 10

247-250

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