Biomarkers in clinical trials for neurodegenerative diseases: Regulatory perspectives and requirements

Progress in Neurobiology

Volumn 95, Issue 4, 2011, Pages 498-500

  Broich, Karl ^a   Weiergräber, Marco ^a   Hampel, Harald ^b  

^a Federal Institute for Drugs and Medical Devices BfArM   (Germany)

^b GOETHE UNIVERSITY   (Germany)

Author keywords

Alzheimer's disease; Biological markers; Biomarkers; Clinical trials; CSF; EMA; FDA; Neurodegenerative diseases; Neuroimaging; Regulatory authorities; Validation

Indexed keywords

AMYLOID PROTEIN; BIOLOGICAL MARKER; TAU PROTEIN;

ALZHEIMER DISEASE; BIOLOGICAL TRAIT; DEGENERATIVE DISEASE; DEMENTIA; HIGH RISK POPULATION; HUMAN; NOTE; OUTCOME ASSESSMENT; PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROTEIN PHOSPHORYLATION; VALIDATION PROCESS;

BIOLOGICAL MARKERS; CLINICAL TRIALS AS TOPIC; DRUG APPROVAL; HUMANS; NEURODEGENERATIVE DISEASES; OUTCOME ASSESSMENT (HEALTH CARE); RESEARCH DESIGN;

EID: 82755197628     PISSN: 03010082     EISSN: 18735118     Source Type: Journal    
DOI: 10.1016/j.pneurobio.2011.09.004     Document Type: Note

References (27)

1. Baker S.G. Surrogate endpoints: wishful thinking or reality?. J. Natl. Cancer Inst. 2006, 98:502-503.
2. Baker S.G., Kramer B.S. A perfect correlate does not a surrogate make. BMC Med. Res. Methodol. 2003, 3:16.
3. Biomarkers and surrogate endpoints: preferred definition and conceptual framework. Clin. Pharmacol. Ther. 2001, 69:89-95. Biomarkers Definition Working Group.
4. Blennow K. Biomarkers in Alzheimer's disease drug development. Nat. Med. 2010, 16:1218-1222.
5. Blennow K., Hampel H., Weiner M., Zetterberg H. Cerebrospinal fluid and plasma biomarkers in Alzheimer disease. Nat. Rev. Neurol. 2010, 6:131-144.
6. Broich K. Outcome measures in clinical trials on medicinal products for the treatment of dementia: a European regulatory perspective. Int. Psychogeriatr. 2007, 19:509-524.
7. Bucher H.C., Guyatt G.H., Cook D.J., Holbrook A., McAlister F.A. Users' guides to the medical literature: XIX. Applying clinical trial results. A. How to use an article measuring the effect of an intervention on surrogate end points. JAMA 1999, 282:771-778. Evidence-Based Medicine Working Group.
8. Committee for Human Medicinal Products, 2008. Guideline on medicinal products for the treatment of Alzheimer's disease and other dementias.
9. Committee for Human Medicinal Products, 2011. Qualification opinion of novel methodologies in the predementia stage of Alzheimer's disease: cerebrospinal fluid related biomarkers for drugs affecting amyloid burden.
10. Council of the European Union, 2008. Council Regulation (EC) No 73/2008 of 20 December 2007 setting up the Joint Undertaking for the implementation of the Joint Technology Initiative on Innovative Medicines. Official Journal of the European Union, L30/38-L30/51.
11. Cummings J.L. Integrating ADNI results into Alzheimer's disease drug development programs. Neurobiol. Aging 2010, 31:1481-1492.
12. Cummings J.L., Doody R., Clark C. Disease-modifying therapies for Alzheimer disease: challenges to early intervention. Neurology 2007, 69:1622-1634.
13. European Medicines Agency, 2008. Biomarkers Qualification: Guidance to applicants.
14. Fleming T.R., DeMets D.L. Surrogate end points in clinical trials: are we being misled?. Ann. Intern. Med. 1996, 125:605-613.
15. Fox N.C., Black R.S., Gilman S., Rossor M.N., Griffith S.G., Jenkins L., Koller M. Effects of Abeta immunization (AN1792) on MRI measures of cerebral volume in Alzheimer disease. Neurology 2005, 64:1563-1572.
16. Frank R., Hargreaves R. Clinical biomarkers in drug discovery and development. Nat. Rev. Drug Discov. 2003, 2:566-580.
17. Gispen-de Wied C.C., Kritsidima M., Elferink A.J. The validity of biomarkers as surrogate endpoints in Alzheimer's disease by means of the Quantitative Surrogate Validation Level of Evidence Scheme (QSVLES). J. Nutr. Health Aging 2009, 13:376-387.
18. Hampel H., Burger K., Teipel S.J., Bokde A.L., Zetterberg H., Blennow K. Core candidate neurochemical and imaging biomarkers of Alzheimer's disease. Alzheimers Dement. 2008, 4:38-48.
19. Hampel H., Frank R., Broich K., Teipel S.J., Katz R.G., Hardy J., Herholz K., Bokde A.L., Jessen F., Hoessler Y.C., Sanhai W.R., Zetterberg H., Woodcock J., Blennow K. Biomarkers for Alzheimer's disease: academic, industry and regulatory perspectives. Nat. Rev. Drug Discov. 2010, 9:560-574.
20. Hampel H., Wilcock G., Andrieu S., Aisen P., Blennow K., Broich K., Carrillo M., Fox N.C., Frisoni G.B., Isaac M., Lovestone S., Nordberg A., Prvulovic D., Sampaio C., Scheltens P., Weiner M., Winblad B., Coley N., Vellas B. Biomarkers for Alzheimer's disease therapeutic trials. Prog. Neurobiol. 2010, 95:579-593.
21. Jack C.R., Vemuri P., Wiste H.J., Weigand S.D., Aisen P.S., Trojanowski J.Q., Shaw L.M., Bernstein M.A., Petersen R.C., Weiner M.W., Knopman D.S. Evidence for ordering of Alzheimer disease biomarkers. Arch. Neurol. 2011, (epub online).
22. Katz R. Biomarkers and surrogate markers: an FDA perspective. NeuroRx 2004, 1:189-195.
23. Mani R.B. The evaluation of disease modifying therapies in Alzheimer's disease: a regulatory viewpoint. Stat. Med. 2004, 23:305-314.
24. Mattsson N., Blennow K., Zetterberg H. CSF biomarkers: pinpointing Alzheimer pathogenesis. Ann. N.Y. Acad. Sci. 2009, 1180:28-35.
25. Trojanowski J.Q., Vandeerstichele H., Korecka M., Clark C.M., Aisen P.S., Petersen R.C., Blennow K., Soares H., Simon A., Lewczuk P., Dean R., Siemers E., Potter W.Z., Weiner M.W., Jack C.R., Jagust W., Toga A.W., Lee V.M., Shaw L.M. Update on the biomarker core of the Alzheimer's Disease Neuroimaging Initiative subjects. Alzheimers Dement. 2010, 6:230-238.
26. Vellas B., Coley N., Andrieu S. Disease modifying trials in Alzheimer's disease: perspectives for the future. J. Alzheimers Dis. 2008, 15:289-301.
27. Woodcock J., Woosley R. The FDA critical path initiative and its influence on new drug development. Annu. Rev. Med. 2008, 59:1-12.