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Cll Friends,

We're all in the same and it seems all of us agree that compassionate

programs need to be expanded. IMO hit the nail on the head pointing out

how many patients died waiting for Gleveec's approval. How many years of life

were lost for CLL patients waiting for Rituximab?

I agree with 's assessment about Bureaucracies. And I have a feeling

that even Karl would agree in that sense to some degree. I agree that Karl

is a great asset for our community.

Consider the following overly brief parable. ( I really did try to keep

it brief but failed)

As I see things, Imagine being on top a tall mountain looking to the South

where I see a forest below. There I see a large Forest that seems to be

doing well. However I do see one section that is brown and has many dying

trees. Looking out on this vast forest, we all agree that for the most part,

it is doing well. This to me represents the FDA today. With my binoculars I

see Karl, doing well in his part of the forest genuinely serving as best

as he can. His efforts deserve our respect and appreciation. Yet, IMO I'd

like to suggest that he's seeing the individual trees..... yet not seeing the

entire forest and especially the area where the forest is having major

issues. In this picture, that troubled part of the forest represents how the

FDA is managing patients with no health to protect. Those who are told to go

home to die.

To the North I see what looks like it could be a mirage, not sure, but it

apparently looks like a small robust forest where the trees grow rapidly,

are very green, and producing many other trees much quicker than the south

forest. It looks like a small ecosystem that is doing well that to me

represents what the FDA could be doing when it comes to lethal disease. In this

North forest the costs of developing new technology is vastly cheaper than

in the South Forest. This not only saves lives but keeps our pharmaceuticals

here in our own Country rather than moving to other Countries like

manufacturing has for the last 50 years.

There is a distinct recognition that WHERE THERE IS NO HEALTH TO PROTECT,

THERE IS LESS REGULATION between the Doctors, legitimate research

institutions, and their Patients. The only regulation is that indeed, these

patients are very close to death and that what ever research is done with them

has

strong, open to the public reporting and humane treatment restrictions.

There is an understanding that these patients have the right to their own

well informed decisions in the spirit or respect of what we all hold dearly.

That is, the right to pursue LIFE, liberty, and the pursuit of happiness.

THESE PATIENTS in this North forest HAVE BEEN GIVEN MUCH GREATER FREE

RIEGN TO DIG DEEP INTO LEGITIMATE DEVELOPING TECHNOLOGY.

There is respect to the fact that when dealing with patients with no health

to protect, policy that encourages cheaper drug development saves lives.

In this regard, there are fewer animal study requirements as these take more

time, add expense, and actually impede good drugs from making it into

humans. Disease is complicated, and testing with other genetic packages such as

mice or even chimpanzees is inefficient and destined to fail in many

cases.

In this forest, large powerful groups like the lymphoma society, the breast

cancer groups, and others understand that investment is needed in the

process itself and not just in a specific treatment strategy.

The challenge is to understand that the system can be improved to make it

happen. I believe the Abigail Alliance would be a big step in the right

direction. Givepatientsafightingchance.com also brings up ideas in how this

hypothetical forest could be.

the fact that we're all discussing this is truly wonderful and yes..... in

one way or another...We are all on the same team. Its not about this person

or that person being wrong or disingenuous but what makes sense in moving

forward. I believe that if we step back, look from far away where we see

the entire forest, on the mountain top so to speak, we can see a much better

way of being much more efficient in how we manage lethal disease. That

will save years and years of life and endear the true spirit of the pursuit

of Life, liberty, and the pursuit of happiness.

Many other arguments but too long already. Sorry about my passion in this

regard.

Regards,

Leo

In a message dated 1/11/2011 7:02:18 P.M. Pacific Standard Time,

lbrown08540@... writes:

Genasense failed to achieve the primary goal(s)/ endpoints the researchers

(and drug company) set for themselves ahead of the clinical trial. By

slicing and dicing the data after the fact, in a small subset of the patient

cohort, they drew statistically iffy conclusions that served their argument.

As some wag once put it, after the fact sub-set analysis is like shooting

at a barn, then painting bulls-eye around the bullet hole, where ever it

happened to be. Hardly compelling " proof " .

Careful patient advocacy requires that we be able to tell the difference

between marketing hype of companies with clear financial interests and cases

where regulatory hurdles are keeping back a truly valuable drug from the

patient community. Genasense is hardly a good example for getting the

patient community mobilized.

: I agree with you that compassionate use programs need to be

expanded. I STRONGLY disagree with you that Karl is some kind of a FDA stooge

because he has been a patient advocate representing our needs and viewpoints on

FDA panels. He is perhaps the single voice of reason that has knowledge,

credibility and lack of vitriol in his rhetoric going for him - this patient

community is lucky to have him as our advocate.

>

> Sure Karl, I understand the point you make about bias.

>

> Irregardless, we don't have approval of Genasense after an inordinate

length of time.

>

> ~chris

>

>

>

> -> Hi Be aware that the link is to a business perspective, which

could well be biased -

>

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Hi,

Regarding the review time for imatinib (Gleevec), See Priority Review

Established by FDA to Expedite Patient Access to Medications: Priority Review

http://www.medscape.com/viewarticle/449551_5

Copying:

" Priority review was requested for imatinib at the time of NDA submission on

February 27, 2002.[8] Although FDA had 6 months to review the imatinib

application, the review was completed in 2.5 months.

.... This short review and approval time was in large part due to the efficacy

and safety demonstrated by imatinib. In addition, the fact that the imatinib

development objectives closely matched FDA's definition for unmet need may have

contributed to the rapid review.[8] "

I don't know off-hand how much time the sponsor needed following approval to set

up good manufacturing standards, necessary also prior to approval See

http://www.fda.gov/Drugs/DevelopmentApprovalProcess/Manufacturing/default.htm

All the best,,

Karl

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Very articulate and well thought out point of view.

My wife has CLL/SLL with 17p deletion. Not sure we have time to be 'saved' by

the FDA's process. It seems to me that they are the gatekeepers holding the key

to the gate in the high stone wall that separates the two forests.

Brett

On Jan 12, 2011, at 2:14 PM, sacramentodds@... wrote:

> Cll Friends,

> We're all in the same and it seems all of us agree that compassionate

> programs need to be expanded. IMO hit the nail on the head pointing out

> how many patients died waiting for Gleveec's approval. How many years of life

> were lost for CLL patients waiting for Rituximab?

> I agree with 's assessment about Bureaucracies. And I have a feeling

> that even Karl would agree in that sense to some degree. I agree that Karl

> is a great asset for our community.

> Consider the following overly brief parable.

<snipped by moderator>

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Novartis is already selling the successor to Gleevec, TASIGNA, which is for CML

patients who are refractory or non-responsive to Gleevec, and no doubt the firm

is looking ahead to the time Gleevec goes off patent. A senior Novartis

executive told me the firm started working on this drug ten years ago.

Development cycles for these drugs are very long. Capacity to manufacture the

drug might be allocated in parallel with other activities.

Adam

>

> Hi,

>

> Regarding the review time for imatinib (Gleevec), See Priority Review

> Established by FDA to Expedite Patient Access to Medications: Priority Review

http://www.medscape.com/viewarticle/449551_5

>

> Copying:

>

> " Priority review was requested for imatinib at the time of NDA submission on

February 27, 2002.[8] Although FDA had 6 months to review the imatinib

application, the review was completed in 2.5 months.

>

> ... This short review and approval time was in large part due to the efficacy

and safety demonstrated by imatinib. In addition, the fact that the imatinib

development objectives closely matched FDA's definition for unmet need may have

contributed to the rapid review.[8] "

>

> I don't know off-hand how much time the sponsor needed following approval to

set up good manufacturing standards, necessary also prior to approval See

http://www.fda.gov/Drugs/DevelopmentApprovalProcess/Manufacturing/default.htm

>

> All the best,,

>

> Karl

>

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