Featured Research

from universities, journals, and other organizations

New Generation Clinical Trials Could Save Time And Money, Improve Patient Care

Date:
January 6, 2006
Source:
University of Texas M. D. Anderson Cancer Center
Summary:
As we enter the era of personalized medicine, it is time to take a fresh look at how we evaluate new medicines and treatments for cancer, according to Donald Berry, Ph.D., professor and chair of the Department of Biostatistics and Applied Mathematics at The University of Texas M. D. Anderson Cancer Center.

As we enter the era of personalized medicine, it is time to take a fresh look at how we evaluate new medicines and treatments for cancer, according to Donald Berry, Ph.D., professor and chair of the Department of Biostatistics and Applied Mathematics at The University of Texas M. D. Anderson Cancer Center.

"We need to rethink how we design and conduct clinical trials in the United States," says Berry. "Our current system has served us well for the past 50 years, but the demands of 21st century medicine are beginning to put a strain on the current system, and we believe we have something to relieve that strain."

Berry outlines his approach to conducting clinical trials in the January 2006 issue of Nature Reviews Drug Discovery. In the article, he advocates turning the statistical method used to evaluate new drugs on its head. He states that the statistical method used nearly exclusively to design and monitor clinical trials today, a method called frequentist or Neyman-Pearson (for the statisticians who advocated its use), is so narrowly focused and rigorous in its requirements that it limits innovation and learning.

His solution, which he has advocated for more than 30 years, is to adopt a system called the Bayesian method, a statistical approach he says is more in line with how science works. He sites examples of Bayesian approaches being used routinely in physics, geology and other sciences. And he is putting his approach to the test at M. D. Anderson, where more than 100 cancer-related phase I and II clinical trials are being planned or carried out using the Bayesian approach. The main difference between the Bayesian approach and the frequentist approach to clinical trials has to do with how each method deals with uncertainty, an inescapable component of any clinical trial. Unlike frequentist methods, explains Berry, Bayesian methods assign anything unknown a probability using information from previous experiments. In other words, Bayesian methods make use of the results of previous experiments, whereas frequentist approaches assume we have no prior results.

"Using the Bayesian approach, it is natural to do continuous updating as information accrues," says Berry. "This characteristic makes it possible for us to build adaptive designs in clinical trials."

He argues that the Bayesian approach is better for doctors, patients who participate in clinical trials and for patients who are waiting for new treatments to become available.

"Doctors want to be able to design trials to look at multiple potential treatment combinations and use biomarkers to determine who is responding to what medication," says Berry. "At the end of the day, when they enroll the last patient in the study they want to be able to treat that patient optimally depending on the patient's disease characteristics. Using a Bayesian approach, the trial design exploits the results as the trial is ongoing and adapts based on these interim results. That kind of thing is an anathema in the standard approach."

However, Berry argues, such flexibility is crucial to clinical trials in the 21st century. "The advances of the 20th century have taught researchers that cancer is a diverse disease, and what works to treat one person's disease may not work for another," he says. "In order to have the kind of personalized medicine the 21st century will demand, it will be necessary to be more flexible in how we evaluate potential new treatments."

Of course, the most important factor in whether the Bayesian approach will gain acceptance in clinical trials reporting is whether the U. S. Food and Drug Administration will accept Bayesian approaches in making determination of safety and efficacy of new treatments. Berry says progress is being made both at pharmaceutical companies and at the FDA in bringing regulators up to speed on the Bayesian approach. "Our biggest challenge is to convince the regulators that we are not throwing the baby out with the bathwater by using a Bayesian approach," says Berry. "It is rigorous and we are not losing science by using it." For example, the FDA has approved the Bristol-Myers Squibb drug Pravigard Pac for prevention of secondary cardiac events based on data evaluated using the Bayesian approach.

In addition, Berry says, it is possible to reduce the exposure of patients in trials to ineffective therapy using the Bayesian approach.

For example, in adaptive clinical trials, if interim results indicate that patients with a certain genetic makeup respond better to a specific treatment, it is possible to recruit more of those patients to that arm of the study without compromising the overall conclusions. Moreover, using the Bayesian approach may make it possible to reduce the number of patients required for a trial by as much as 30 percent, thereby reducing the risk to patients and the cost and time required to develop therapeutic strategies.



Story Source:

The above story is based on materials provided by University of Texas M. D. Anderson Cancer Center. Note: Materials may be edited for content and length.


Cite This Page:

University of Texas M. D. Anderson Cancer Center. "New Generation Clinical Trials Could Save Time And Money, Improve Patient Care." ScienceDaily. ScienceDaily, 6 January 2006. <www.sciencedaily.com/releases/2006/01/060106125901.htm>.
University of Texas M. D. Anderson Cancer Center. (2006, January 6). New Generation Clinical Trials Could Save Time And Money, Improve Patient Care. ScienceDaily. Retrieved July 31, 2014 from www.sciencedaily.com/releases/2006/01/060106125901.htm
University of Texas M. D. Anderson Cancer Center. "New Generation Clinical Trials Could Save Time And Money, Improve Patient Care." ScienceDaily. www.sciencedaily.com/releases/2006/01/060106125901.htm (accessed July 31, 2014).

Share This




More Health & Medicine News

Thursday, July 31, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

House Republicans Vote to Sue Obama Over Healthcare Law

House Republicans Vote to Sue Obama Over Healthcare Law

Reuters - US Online Video (July 31, 2014) The Republican-led House of Representatives votes to sue President Obama, accusing him of overstepping his executive authority in making changes to the Affordable Care Act. Mana Rabiee reports. Video provided by Reuters
Powered by NewsLook.com
Despite Health Questions, E-Cigs Are Beneficial: Study

Despite Health Questions, E-Cigs Are Beneficial: Study

Newsy (July 31, 2014) Citing 81 previous studies, new research out of London suggests the benefits of smoking e-cigarettes instead of regular ones outweighs the risks. Video provided by Newsy
Powered by NewsLook.com
Dangerous Bacteria Kills One in Florida

Dangerous Bacteria Kills One in Florida

AP (July 31, 2014) Sarasota County, Florida health officials have issued a warning against eating raw oysters and exposing open wounds to coastal and inland waters after a dangerous bacteria killed one person and made another sick. (July 31) Video provided by AP
Powered by NewsLook.com
Health Insurers' Profits Slide

Health Insurers' Profits Slide

Reuters - Business Video Online (July 30, 2014) Obamacare-related costs were said to be behind the profit plunge at Wellpoint and Humana, but Wellpoint sees the new exchanges boosting its earnings for the full year. Fred Katayama reports. Video provided by Reuters
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins