[R] OT: Is randomization for targeted cancer therapies ethical?

[Marc Schwartz]

On Sep 21, 2010, at 6:52 AM, Liaw, Andy wrote:

>> From: JLucke at ria.buffalo.edu
>> 
>> Clearly inferior treatments are unethical. 
> 
> The Big Question is:  What constitute "clearly"?  Who or How to decide
> what is "clearly"?  I'm sure there are plenty of people who don't
> understand much Statistics and are perfectly willing to say the results
> on the two cousins show the conventional treatment is "clearly
> inferior".  Sure, on these two cousins we can say so, but what about
> others?
> 
>> Donald Berry at MD Anderson in Houston TX  and Jay Kadane at Carnegie 
>> Mellon have been working on more ethical designs within the Bayesian 
>> framework.  In particular, response adaptive designs reduce 
>> the assignment 
>> to and continuation of patients on inferior treatments.
> 
> I've heard LJ Wei talked about this kinds of designs (don't remember if
> they are Bayesian) more than a dozen years ago.  Don't know how common
> they are in use. 
> 
> Andy 
> 


They are becoming more popular as the FDA itself becomes more comfortable with both Bayesian approaches and adaptive designs.

The FDA released draft guidance earlier this year on adaptive designs for drugs and biologics:

  http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM201790.pdf

and final guidance for Bayesian approaches in device trials:

  http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm071121.pdf


There are also several presentations at this week's 2010 FDA Workshop in DC:

  http://www.amstat.org/meetings/fdaworkshop/index.cfm?fuseaction=onlineprogram


Beyond the ethical issues raised, I believe that industry sees these techniques as opportunities to streamline phased trial design, resulting in a compression of the overall timeline to make decisions on the viability of products in the development pipeline. 

HTH,

Marc Schwartz


>> 
>> 
>> Bert Gunter <gunter.berton at gene.com> 
>> Sent by: r-help-bounces at r-project.org
>> 09/20/2010 01:31 PM
>> 
>> To
>> r-help at r-project.org
>> cc
>> 
>> Subject
>> [R] OT: Is randomization for targeted cancer therapies ethical?
>> 
>> 
>> 
>> 
>> 
>> 
>> Hi Folks:
>> 
>> **Off Topic**
>> 
>> Those interested in clinical trials may find the following of 
>> interest:
>> 
>> http://www.nytimes.com/2010/09/19/health/research/19trial.html
>> 
>> It concerns the ethicality of randomizing those with life-threatening
>> disease to relatively ineffective SOC when new "biologically targeted"
>> therapies "appear" to be more effective. While the context may be new,
>> the debate, itself, is not: Tukey wrote (or maybe it was talked -- I
>> can't remember for sure) about this about 30 years ago. I'm sure many
>> other also have done so.
>> 
>> Cheers,
>> 
>> Bert