0
We're unable to sign you in at this time. Please try again in a few minutes.
Retry
We were able to sign you in, but your subscription(s) could not be found. Please try again in a few minutes.
Retry
There may be a problem with your account. Please contact the AMA Service Center to resolve this issue.
Contact the AMA Service Center:
Telephone: 1 (800) 262-2350 or 1 (312) 670-7827  *   Email: subscriptions@jamanetwork.com
Error Message ......
Review |

Novel Methods and Technologies for 21st-Century Clinical Trials A Review

E. Ray Dorsey, MD, MBA1,2; Charles Venuto, PharmD1,2; Vinayak Venkataraman, BSE3; Denzil A. Harris, BA2; Karl Kieburtz, MD, MPH1,2,4
[+] Author Affiliations
1Department of Neurology, University of Rochester Medical Center, Rochester, New York
2Center for Human Experimental Therapeutics, University of Rochester Medical Center, Rochester, New York
3currently a medical student at Duke University School of Medicine, Durham, North Carolina
4Clinical and Translational Sciences Institute, University of Rochester Medical Center, Rochester, New York
JAMA Neurol. 2015;72(5):582-588. doi:10.1001/jamaneurol.2014.4524.
Text Size: A A A
Published online

Importance  New technologies are rapidly reshaping health care. However, their effect on drug development to date generally has been limited.

Objectives  To evaluate disease modeling and simulation, alternative study design, novel objective measures, virtual research visits, and enhanced participant engagement and to examine their potential effects as methods and tools on clinical trials.

Evidence Review  We conducted a systematic search of relevant terms on PubMed (disease modeling and clinical trials; adaptive design, clinical trials, and neurology; Internet, clinical trials, and neurology; and telemedicine, clinical trials, and neurology), references of previous publications, and our files. The search encompassed articles published from January 1, 2000, through November 30, 2014, and produced 7976 articles, of which 22 were determined to be relevant and are included in this review.

Findings  Few of these new methods and technologies have been applied to neurology clinical trials. Clinical outcomes, including cognitive and stroke outcomes, increasingly are captured remotely. Other therapeutic areas have successfully implemented many of these tools and technologies, including web-enabled clinical trials.

Conclusions and Relevance  Increased use of new tools and approaches in future clinical trials can enhance the design, improve the assessment, and engage participants in the evaluation of novel therapies for neurologic disorders.

Figures in this Article

Sign in

Purchase Options

• Buy this article
• Subscribe to the journal
• Rent this article ?

Figures

Place holder to copy figure label and caption
Figure 1.
Preclinical and Clinical Drug Development Costs

The total cost of research and development cost (in 2000 US dollars) per approved new drug has increased dramatically during the past 3 decades, with the most notable cost increases occurring in the clinical phases. Data are obtained from DiMasi et al.3

Graphic Jump Location
Place holder to copy figure label and caption
Figure 2.
Disease-Drug Trial Models

Clinical trial modeling and simulation help to optimize trial designs by investigating assumptions and variability of different factors that affect trial performance. Disease progression models quantify changes in disease status over time, typically in the untreated state. Pharmacokinetic models represent the time course of drug disposition in the body. Pharmacodynamic models characterize the exposure-response relationship of the drug. Together, these models are integrated and applied to simulate different trial scenarios (eg, power calculations, dosing strategies, and trial execution) for predictive purposes. Curves represent interaction between both variables.

Graphic Jump Location

Tables

References

Correspondence

CME
Also Meets CME requirements for:
Browse CME for all U.S. States
Accreditation Information
The American Medical Association is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians. The AMA designates this journal-based CME activity for a maximum of 1 AMA PRA Category 1 CreditTM per course. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Physicians who complete the CME course and score at least 80% correct on the quiz are eligible for AMA PRA Category 1 CreditTM.
Note: You must get at least of the answers correct to pass this quiz.
Please click the checkbox indicating that you have read the full article in order to submit your answers.
Your answers have been saved for later.
You have not filled in all the answers to complete this quiz
The following questions were not answered:
Sorry, you have unsuccessfully completed this CME quiz with a score of
The following questions were not answered correctly:
Commitment to Change (optional):
Indicate what change(s) you will implement in your practice, if any, based on this CME course.
Your quiz results:
The filled radio buttons indicate your responses. The preferred responses are highlighted
For CME Course: A Proposed Model for Initial Assessment and Management of Acute Heart Failure Syndromes
Indicate what changes(s) you will implement in your practice, if any, based on this CME course.

Multimedia

Some tools below are only available to our subscribers or users with an online account.

2,571 Views
8 Citations
×

Sign in

Purchase Options

• Buy this article
• Subscribe to the journal
• Rent this article ?

Related Content

Customize your page view by dragging & repositioning the boxes below.

Articles Related By Topic
Related Collections
PubMed Articles
Jobs
JAMAevidence.com

The Rational Clinical Examination: Evidence-Based Clinical Diagnosis
Stroke

The Rational Clinical Examination: Evidence-Based Clinical Diagnosis
Stroke, Hemorrhagic

brightcove.createExperiences();