What's next? Clinical Trial vs. Off-Protocol Therapy?

June 13, 2009

Greetings,

Off-Protocol Therapies (OPT) are protocols that the physician selects or

modifies based on " conditions on the ground " . It could be a mix and match of

agents (standard, off-label, and sometimes investigational), chosen to meet the

unique clinical circumstances of the patient and treatment history.

One advantage of OPT is greater flexibility. The disadvantage is that the

results are not easily interpreted or useful as evidence that will guide

practice for others.

The focus of OPT is on using the best available information and tools to deal

with a non-standard or urgent clinical situation. ... The patient has already

tried this, so we will substitute that, or alter the dose, timing or sequence of

approved therapies, or include an investigational agent (under compassionate

use) that may overcome treatment resistance.

It seems that use of OPT is quite common. See for example:

What's next?

When standard therapy is not adequate, we can consider with the help of experts

clinical trials, but also OPT. It seems that use of OPT is more common than

referrals to clinical trials, probably because of the flexibility it provides,

but also because trial enrollment criteria often makes many patients,

particularly when not responding to standard protocols, ineligible for studies.

Important, I think, to seek guidance from qualified experts who have the most

knowledge of standard, off-label, and investigational agents, and also the most

experience with the medical condition. Already seeing an expert? Get a second

perspective from another.

-- The expert can then recommend an appropriate clinical trial or OPT, based on

first-hand information about your circumstances and treatment history - and

knowledge and access to the full range of appropriate therapeutic options (RIT,

investigational agents, SCT, etc).

Be sure to ask the expert specifically about OPT and clinical trials ... and

make sure that you are on the same page regarding the goal of therapy (curative

intent vs. management for example.)

If the expert seems reluctant to recommend any therapy, you might express your

interest in trying one of the many targeted agents as an alternative to the

natural course of the disease. (An oral drug, such as Enzastaurin, for example,

or ABT-263). While this would be a long shot, there have been instances in

research history of targeted therapies making a profound difference in late

stage disease, such as Gleevec for CML.

~ Karl

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