Novel methods and technologies for 21st-century clinical trials a review

JAMA Neurology

Volumn 72, Issue 5, 2015, Pages 582-588

  Dorsey, E Ray ^a   Venuto, Charles ^a   Venkataraman, Vinayak ^b   Harris, Denzil A ^a   Kieburtz, Karl ^a  

^a UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

^b DUKE UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GLUCOSAMINE; KAVA; LITHIUM; PLACEBO; TADALAFIL; TOLTERODINE; VALERIAN;

ALZHEIMER DISEASE; AMYOTROPHIC LATERAL SCLEROSIS; CEREBROVASCULAR ACCIDENT; CLINICAL TRIAL (TOPIC); COST CONTROL; DISEASE MODEL; DRUG EFFICACY; DRUG RESPONSE; ERECTILE DYSFUNCTION; HUMAN; INSOMNIA; INTERNET; KNEE OSTEOARTHRITIS; MOBILE APPLICATION; NEUROLOGIC DISEASE; OVERACTIVE BLADDER; PARKINSON DISEASE; PATIENT PARTICIPATION; PRIORITY JOURNAL; REMOTE SENSING; RESEARCH SUBJECT; REVIEW; SELF REPORT; SIMULATION; STUDY DESIGN; VIRTUAL REALITY; DEVICES; METHODOLOGY; NERVOUS SYSTEM DISEASES; NEUROLOGY; PROCEDURES; TRENDS;

CLINICAL TRIALS AS TOPIC; HUMANS; NERVOUS SYSTEM DISEASES; NEUROLOGY; RESEARCH DESIGN;

EID: 84938539878     PISSN: 21686149     EISSN: None     Source Type: Journal    
DOI: 10.1001/jamaneurol.2014.4524     Document Type: Review

References (74)

1. DiMasi JA, Grabowski HG. The cost of biopharmaceutical R&D: is biotech different? Manage Decis Econ. 2007;28(4-5):469-479.
2. Paul SM, Mytelka DS, Dunwiddie CT, et al. How to improve R&D productivity: the pharmaceutical industry's grand challenge. Nat Rev Drug Discov. 2010;9(3):203-214.
3. DiMasi JA, Hansen RW, Grabowski HG. The price of innovation: new estimates of drug development costs.JHealth Econ. 2003;22(2):151-185.
4. US Food and Drug Administration. 2013 Novel New Drugs Summary. Silver Spring, MD: US Food and Drug Administration; 2014.
5. Booth B, Zemmel R. Prospects for productivity. Nat Rev Drug Discov. 2004;3(5):451-456.
6. Moore GE. Cramming more components onto integrated circuits.Electronics. 1965;38(8):114-117.
7. Milligan PA, Brown MJ, Marchant B, et al. Model-based drug development: a rational approach to efficiently accelerate drug development. Clin Pharmacol Ther. 2013;93(6): 502-514.
8. Holford NH, Nutt JG. Interpreting the results of Parkinson's disease clinical trials: time for a change. Mov Disord. 2011;26(4):569-577.
9. Mould DR. Models for disease progression: new approaches and uses. Clin Pharmacol Ther.2012;92 (1):125-131.
10. Gomeni R, Fava M; Pooled Resource Open-Access ALS Clinical Trials Consortium. Amyotrophic lateral sclerosis disease progression model. Amyotroph Lateral Scler Frontotemporal Degener. 2014;15(1-2):119-129.
11. US Food and Drug Administration. Disease Specific Model Library. http://www.fda.gov /AboutFDA/CentersOffices /OfficeofMedicalProductsandTobacco/CDER /ucm180485.htm. Accessed June 24, 2014.
12. Sheiner LB. Learning versus confirming in clinical drug development. Clin Pharmacol Ther. 1997;61(3):275-291.
13. Chataway J, Nicholas R, Todd S, et al. A novel adaptive design strategy increases the efficiency of clinical trials in secondary progressive multiple sclerosis.Mult Scler. 2011;17(1):81-88.
14. Kaufmann P, Thompson JL, Levy G, et al; QALS Study Group. Phase II trial of CoQ10 for ALS finds insufficient evidence to justify phase III. Ann Neurol. 2009;66(2):235-244.
15. O'Quigley J, Pepe M, Fisher L. Continual reassessment method: a practical design for phase 1 clinical trials in cancer. Biometrics. 1990;46(1):33-48.
16. Iasonos A, Wilton AS, Reidel ER, Seshan VE, Spriggs DR. A comprehensive comparison of the continual reassessment method to the standard 3 + 3 dose escalation scheme in Phase I dose-finding studies. Clin Trials. 2008;5(5):465-477.
17. I-SPY 2 Trial. http://ispy2.org/. Accessed June 10, 2014.
18. Adams CP, Brantner VV. Estimating the cost of new drug development: is it really 802 million dollars? Health Aff (Millwood). 2006;25(2):420-428.
19. Huntington Study Group. Announcement of 2CARE early study closure [press release]. 2014. http://www.huntington-study-group.org/LinkClick .aspx?fileticket=LurnlLVT3rw%3d&tabid=95. Accessed August 22, 2014.
20. Huntington Study Group. Announcement of CREST-E early study closure [press release]. 2014. http://www.huntington-study-group.org /CurrentClinicalTrials/CRESTE /CRESTEOctober2014PressRelease/tabid/316 /Default.aspx. Accessed November 19, 2014.
21. Statement on the termination of NET-PD LS-1 study. 2013. http://parkinsontrial.ninds.nih.gov /netpd-LS1-study-termination.htm. Accessed June 25, 2014.
22. Elias T, Gordian M, Singh N, Zemmel R. Why products fail in phase III. In Vivo. 2006;24:49-54.
23. Fahn S, Elton R; Members of the UPDRS Development Committee. Unified Parkinson's Disease Rating Scale. In: Fahn S, Marsden C, Caine D, Goldstein M, eds. Recent Developments in Parkinson's Disease. Vol 2. Florham Park, NJ: Macmillan Health Care Information; 1987:153-163.
24. Reilmann R, Bohlen S, Kirsten F, Ringelstein EB, Lange HW. Assessment of involuntary choreatic movements in Huntington's disease: toward objective and quantitative measures. Mov Disord. 2011;26(12):2267-2273.
25. Heldman DA, Espay AJ, LeWitt PA, Giuffrida JP. Clinician versus machine: reliability and responsiveness of motor endpoints in Parkinson's disease. Parkinsonism Relat Disord. 2014;20(6): 590-595.
26. Andres PL, English R, Mendoza M, et al. Developing normalized strength scores for neuromuscular research. Muscle Nerve. 2013;47(2): 177-182.
27. Tabrizi SJ, Langbehn DR, Leavitt BR, et al; TRACK-HD investigators. Biological and clinical manifestations of Huntington's disease in the longitudinal TRACK-HD study: cross-sectional analysis of baseline data. Lancet Neurol. 2009;8(9): 791-801.
28. Tabrizi SJ, Scahill RI, Owen G, et al; TRACK-HD Investigators. Predictors of phenotypic progression and disease onset in premanifest and early-stage Huntington's disease in the TRACK-HD study: analysis of 36-month observational data. Lancet Neurol. 2013;12(7):637-649.
29. Tsanas A, Little MA, McSharry PE, Ramig LO. Accurate telemonitoring of Parkinson's disease progression by noninvasive speech tests. IEEE Trans Biomed Eng. 2010;57(4):884-893.
30. Fisher RS, Blum DE, DiVentura B, et al. Seizure diaries for clinical research and practice: limitations and future prospects. Epilepsy Behav. 2012;24(3): 304-310.
31. Cook MJ, O'Brien TJ, Berkovic SF, et al. Prediction of seizure likelihood with a long-term, implanted seizure advisory system in patients with drug-resistant epilepsy: a first-in-man study. Lancet Neurol. 2013;12(6):563-571.
32. Holmes D. Can technology reveal the natural history of epilepsy? Lancet Neurol. 2014;13(4):347-348.
33. Weikert M, Suh Y, Lane A, et al. Accelerometry is associated with walking mobility, not physical activity, in persons with multiple sclerosis. Med Eng Phys. 2012;34(5):590-597.
34. Pulliam CL, Eichenseer SR, Goetz CG, et al. Continuous in-home monitoring of essential tremor. Parkinsonism Relat Disord. 2014;20(1):37-40.
35. Kaye J, Mattek N, Dodge HH, et al. Unobtrusive measurement of daily computer use to detect mild cognitive impairment. Alzheimers Dement.2014;10 (1):10-17.
36. Grajales FJ III, Sheps S, Ho K, Novak-Lauscher H, Eysenbach G. Social media: a review and tutorial of applications in medicine and health care. J Med Internet Res. 2014;16(2):e13. doi:10.2196/jmir.2912.
37. The Michael J. Fox Foundation and Intel join forces to improve Parkinson's disease monitoring and treatment through advanced technologies [press release]. https://www.michaeljfox.org /foundation/publication-detail.html?id=555& category=7. Accessed August 13, 2014.
38. Crossley GH, Boyle A, Vitense H, Chang Y, Mead RH; CONNECT Investigators. The CONNECT (Clinical Evaluation of Remote Notification to Reduce Time to Clinical Decision) trial: the value of wireless remote monitoring with automatic clinician alerts.JAm Coll Cardiol. 2011;57(10):1181-1189.
39. Varma N, Michalski J, Stambler B, Pavri BB; TRUST Investigators. Superiority of automatic remote monitoring compared with in-person evaluation for scheduled ICD follow-up in the TRUST trial: testing execution of the recommendations. Eur Heart J. 2014;35(20):1345-1352.
40. Mabo P, Victor F, Bazin P, et al; COMPAS Trial Investigators. A randomized trial of long-term remote monitoring of pacemaker recipients (the COMPAS trial).Eur Heart J. 2012;33(9):1105-1111.
41. Reiffel J, Verma A, Halperin JL, et al. Rationale and design of REVEAL AF: a prospective study of previously undiagnosed atrial fibrillation as documented by an insertable cardiac monitor in high-risk patients.Am Heart J. 2014;167(1):22-27.
42. Ezekowitz MD, Nagarakanti R, Lubinski A, et al; PASCAL Investigators. A randomized trial of budiodarone in paroxysmal atrial fibrillation. J Interv Card Electrophysiol. 2012;34(1):1-9.
43. Saposnik G, Reeves MJ, Johnston SC, Bath PM, Ovbiagele B; VISTA Collaboration. Predicting clinical outcomes after thrombolysis using the iScore: results from the Virtual International Stroke Trials Archive.Stroke. 2013;44(10):2755-2759.
44. Center for Connected Health Care Policy. Telehealth policy. 2014; http://cchpca.org /jurisdiction/12. Accessed November 19, 2014.
45. Ross S, Grant A, Counsell C, Gillespie W, Russell I, Prescott R. Barriers to participation in randomised controlled trials: a systematic review. J Clin Epidemiol. 1999;52(12):1143-1156.
46. Alfredo Caceres J, Greer DM, Goldstein JN, et al. Enrollment of research subjects through telemedicine networks in a multicenter acute intracerebral hemorrhage clinical trial: design and methods. J Vasc Interv Neurol. 2014;7(3):34-40.
47. Silva GS, Schwamm LH. Use of telemedicine and other strategies to increase the number of patients that may be treated with intravenous thrombolysis. Curr Neurol Neurosci Rep. 2012;12(1): 10-16.
48. Karlawish J, Cary MS, Rubright J, Tenhave T. How redesigning AD clinical trials might increase study partners' willingness to participate. Neurology. 2008;71(23):1883-1888.
49. Tanner CM, Meng CC, Ravina B, et al. A practical approach to remote longitudinal follow-up of Parkinson's disease: the FOUND study. Mov Disord. 2014;29(6):743-749.
50. Achey M, Aldred JL, Aljehani N, et al; International Parkinson and Movement Disorder Society Telemedicine Task Force. The past, present, and future of telemedicine for Parkinson's disease. Mov Disord. 2014;29(7):871-883.
51. Cialone J, Augustine EF, Newhouse N, Vierhile A, Marshall FJ, Mink JW. Quantitative telemedicine ratings in Batten disease: implications for rare disease research. Neurology. 2011;77(20):1808-1811.
52. Kobak KA, Leuchter A, DeBrota D, et al. Site versus centralized raters in a clinical depression trial: impact on patient selection and placebo response. J Clin Psychopharmacol. 2010;30(2):193-197.
53. Partridge AH, Winer EP. Informing clinical trial participants about study results. JAMA. 2002;288 (3):363-365.
54. Dorsey ER, Beck CA, Adams M, et al; Huntington Study Group TREND-HD Investigators. Communicating clinical trial results to research participants. Arch Neurol. 2008;65(12):1590-1595.
55. Berenson A. After a trial, silence. New York Times. November 21, 2007. http://query.nytimes.com/gst /fullpage.html?res= 950CE6DC1330F932A15752C1A9619C8B63& smid=pl-share. Accessed June 25, 2014.
56. PROMIS: Dynamic Tools to Measure Health Outcomes From the Patient Perspective. Home page. http://www.nihpromis.org /?AspxAutoDetectCookieSupport=1#4. Published 2014. Accessed August 7, 2014.
57. Neuro-QoL: Quality of Life in Neurological Disorders. 2014. http://www.neuroqol.org/Pages /default.aspx. Accessed August 7, 2014.
58. US Department of Health and Human Services Food and Drug Administration. Guidance for Industry-Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. http://www.fda.gov/downloads /Drugs/Guidances/UCM193282.pdf. Published December 2009. Accessed June 3, 2014.
59. Patrick DL, Burke LB, Powers JH, et al. Patient-reported outcomes to support medical product labeling claims: FDA perspective. Value Health. 2007;10(suppl 2):S125-S137.
60. Landro L. Services match patients with clinical trials. Wall Street Journal. April 23,2012.http: //online.wsj.com/news/articles /SB10001424052702303459004577361972881249382. Accessed June 3, 2014.
61. Marcus AD. Frustrated ALS patients concoct their own drug. Wall Street Journal. April 15, 2012. http://www.wsj.com/news/articles /SB1000142405270230481840457734595394348 4054?mg=reno64-ws. Accessed June 25, 2014.
62. Parent Project Muscular Dystrophy. The Duchenne community imperatives for the guidance for industry on Duchenne muscular dystrophy: developing drugs for treatment over the spectrum of disease. http://www.parentprojectmd.org/site /PageServer?pagename=Advocate-fdaguidance. Published June 25, 2014. Accessed August 22, 2014.
63. Selby JV, Beal AC, Frank L. The Patient-Centered Outcomes Research Institute (PCORI) national priorities for research and initial research agenda. JAMA. 2012;307(15):1583-1584.
64. Riggare S. Sara: Not Patient but Im-Patient [blog]. http://www.riggare.se/. Accessed June 3, 2014.
65. Helfand WH, Lazarus J, Theerman P. "...So that others may walk": the March of Dimes. Am J Public Health. 2001;91(8):1190.
66. De Cock KM, Jaffe HW, Curran JW. Reflections on 30 years of AIDS. Emerg Infect Dis. 2011;17(6): 1044-1048.
67. Research America: An Alliance for Discoveries in Health. America Speaks. 2014;14. http://www .researchamerica.org/uploads/AmericaSpeaksV14 .pdf. Accessed June 25, 2014.
68. McAlindon T. Why are clinical trials of glucosamine no longer uniformly positive? Rheum Dis Clin North Am. 2003;29(4):789-801.
69. Eilenberg KL, Hoover AM, Rutherford ML, Melfi CA, Segal S. From informed consent through database lock: an interactive clinical trial conducted using the Internet. Drug Inf J. 2004;38(3):239-251.
70. Jacobs BP, Bent S, Tice JA, Blackwell T, Cummings SR. An internet-based randomized, placebo-controlled trial of kava and valerian for anxiety and insomnia. Medicine (Baltimore). 2005; 84(4):197-207.
71. Wicks P, Vaughan TE, Massagli MP, Heywood J. Accelerated clinical discovery using self-reported patient data collected online and a patient-matching algorithm. Nat Biotechnol. 2011; 29(5):411-414.
72. Orri M, Lipset CH, Jacobs BP, Costello AJ, Cummings SR. Web-based trial to evaluate the efficacy and safety of tolterodine ER 4 mg in participants with overactive bladder: REMOTE trial. Contemp Clin Trials. 2014;38(2):190-197.
73. McAlindon T, Formica M, Kabbara K, LaValley M, Lehmer M. Conducting clinical trials over the internet: feasibility study. BMJ. 2003;327(7413): 484-487.
74. Christensen CM. The Innovator's Dilemma: When New Technologies Cause Great Firms to Fail. Boston, MA: Harvard Business School Press; 1997.