Navigation Links
Assessing positive outcomes of phase III trials
Date:12/3/2010

Randomized phase III studies should be designed to find out whether a new drug or treatment makes a meaningful difference in patients' survival or quality of life, according to a commentary published online December 3rd in The Journal of the National Cancer Institute. Instead, most trials now are designed to detect a statistically significant difference between treatment and control groups, which may not be clinically meaningful, write Alberto Ocana, M.D., Ph.D. and Ian F. Tannock, M.D., Ph.D., of Princess Margaret Hospital in Toronto.

Regulatory agencies such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMEA) approve drugs usually based on statistically significant results of randomized phase III trials comparing a new, investigational drug with standard treatment. Ocana and Tannock note that pharmaceutical companies have typically sponsored clinical trials that are large enough to detect statistically significant differences in survival. But these differences are often trivial, they say. For instance, the trial that led to approval of erlotinib (Tarceva) for pancreatic cancer found that patients who took the drug had a median survival just 10 days longer than patients in the control group. However, the difference was statistically significant, and the drug was approved.

The authors write that pharmaceutical companies look for a difference in survival outcome between two groups of a trial that is "not usually the minimal difference in overall survival or progression-free survival that is clinically important, but more likely the minimal difference that is feasible to detect, considering the limits on the sample size and hence the cost of the trial."

The authors argue for another approach: "Ideally, trials should be designed [to detect] the minimum clinically important difference, taking into account the tolerability and toxicity of the new treatment, that would persuade oncologists to adopt the news treatment in place of the standard treatment" and investigators should try to reach at least a clinically important difference that was specified in the protocol.

They also suggest that the FDA and EMEA "should define what constitute a positive trial based on the concept of establishing a meaningful clinical benefit for patients similar to those included in any given trial."

In an accompanying editorial, J. Jack Lee, Ph.D., of the M.D. Anderson Cancer Center, writes that this "excellent commentary" calls for a new drug approval paradigm and challenges both the medical and statistical communities to find a better way to assessing whether a drug really works.

Lee goes on to argue for the adoption of the Bayesian approach in contrast to the more conventional frequentist approach. "Statistics in medicine has passed through its infancy and childhood. As it moves into its adolescence, the growing pains of reconciling frequentist and Bayesian views continue," he writes. In his view, though, the "roadblocks" of the Bayesian approach, namely the notion of subjectivity and computation difficulty, have been overcome.

"The Bayesian approach is complementary to and can provide a superior alternative to the frequentist paradigm," Lee writes. "I encourage medical researchers to have an open mind, learn more about Bayesian methods, and apply them to provide a more accurate statistical assessment of the results in clinical trials."


'/>"/>

Contact: Kristine Crane
jncimedia@oxfordjournals.org
301-841-1285
Journal of the National Cancer Institute
Source:Eurekalert

Related medicine news :

1. Researchers validate a new test for assessing childrens and teenagers fitness to prevent morbidity
2. Assessing the science behind health claims
3. For HIV-positive patients, delayed treatment a costly decision
4. Positive Attitude, Planning May Help Women Through Menopause
5. Positive psychological changes from meditation training linked to cellular health
6. Positive social traits trump bad health habits
7. Targeted therapy promising for HER2-positive metastatic breast cancer
8. New study finds positive return on investment for states that invest in quit smoking treatments
9. Study shows tranquil scenes have positive impact on brain
10. Survey of American women finds STD vaccine viewed positively
11. Penns Positive Psychology Center awards $2.9 million for research
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/25/2016)... ... 25, 2016 , ... Austin residents seeking Mohs surgery services, can now turn ... to Dr. Russell Peckham for medical and surgical dermatology. , Dr. Dorsey brings specialization ... selective fellowship in Mohs Micrographic Surgery completed by Dr. Dorsey was under the direction ...
(Date:6/25/2016)... TX (PRWEB) , ... June 25, 2016 , ... ... the United States, named Dr. Sesan Ogunleye, as the Medical Director of its new ... the facility Medical Director of our new Mesquite location,” said Dr. James M. Muzzarelli, ...
(Date:6/25/2016)... ... 25, 2016 , ... On Friday, June 10, Van Mitchell, Secretary of the ... to iHire in recognition of their exemplary accomplishments in worksite health promotion. , The ... Health & Wellness Symposium at the BWI Marriott in Linthicum Heights. iHire was one ...
(Date:6/24/2016)... ... June 24, 2016 , ... Those who have experienced ... feelings, many turn to unhealthy avenues, such as drug or alcohol abuse, as a ... has released tools for healthy coping following a traumatic event. , Trauma sufferers tend ...
(Date:6/24/2016)... ... June 24, 2016 , ... The Pulmonary Hypertension Association ... it will receive two significant new grants to support its work to advance ... 25th anniversary by recognizing patients, medical professionals and scientists for their work in ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)... 24, 2016 ... the addition of the " Global Markets for ... This report focuses on ... updated review, including its applications in various applications. The ... which includes three main industries: pharmaceutical and biotechnology, food ...
(Date:6/24/2016)... CLEMENTE, Calif. , June 24, 2016  American Respiratory Labs ... company, is now able to perform sophisticated lung assessments in patients, ... Technologies , Inc. Patients are no longer limited ... ndd,s EasyOne PRO ® , ARL patients like Jeanne R. of ... done in the comfort of her own home. ...
(Date:6/24/2016)... Belgium , June 24, 2016 /PRNewswire/ ... announced the appointment of Dr. Edward Futcher ... a Non-Executive Director, effective June 23, 2016.Dr. Futcher ... and Nominations and Governance Committees.  As a non-executive ... provide independent expertise and strategic counsel to VolitionRx ...
Breaking Medicine Technology: