Re: The 'Go Slower' FDA

[Guest guest]

Excuse me Lynn,

I for one stand solidly in agreement of 's comments. Our government

( those making the rules) are not in the shoes that we're in. If they were,

I believe things would be different. Anyone analyzing the current system

will recognize that the system is overly expensive and overly delays the

advancement of medicine. A bit of tinkering can and will make the system more

efficient, lower costs, and save years of life. Some of these arguments are

explored at givepatientsafightingchance.com

I have heard enough that I believe he understands that no one

government employee means to harm or is bad. It is simply the culture that

exists

there and it is evident in things they say, the regulation that exists

today, and the direction that they're headed.

You don't have to be too smart to recognize that if new drugs get into the

correct humans quicker all mankind will benefit. You don't have to be too

smart to recognize that if new drugs get into the correct humans quicker,

the development costs will drop. You don't have to be too smart to recognize

our end of life options are part of what is bankrupting our country. The

fact that my last choices when facing death will be eating up hundreds of

thousands of tax dollars through medicare or going home to die is truly sad.

When those last days are at hand, How I wish that my government would allow

me the educated decision and ability to die with dignity fighting my

disease/cancer with a treatment that is still far from being approved for a

clinical trial.

1.2 billion for each new drug is unacceptable! We can do better!!

Come on Lynn, I believe that is just trying to

improve/influence/educate our group in ways to improve the system and using your

own words,

being a positive contributor. If it takes a bit of criticism on occasion, so be

it.

Keep it up . And yes Lynn, you are definitely right about signing up

for Clinical trials and making us available for testing with the current,

slow, expensive system that currently is in place.

With passion and desire to bring positive change,

Leo

In a message dated 3/14/2011 5:26:25 P.M. Pacific Daylight Time,

pualynn@... writes:

Well .. very confrontational posts from both and . ,

your communication style may be why you received NO responses. , really

unproductive generalization about government employees.

I think there is some very exciting research going on, especially in CLL

right now. If you are fortunate to be close to a research center, then make

yourself available, donate your blood, and become a positive contributor.

Lynn

[Guest guest]

,

Have you taken HDMP + Humax CD20? Dr. Kipps is thinking of using this for my

wife, Freddie. As you may remember, she has taken

HDMP + R a couple of times with OK results and little problems, but needs

something else. She has some nodes larger than he feels

that would benefit from Campath and wants to use this regimine.

Hope all is well with you,

Dan and Freddie Hill

_____

From: [mailto: ] On Behalf Of

S.

Sent: Monday, March 14, 2011 1:05 PM

Subject: The 'Go Slower' FDA

Employees at the FDA are government employees. The default position of any

government worker is to 'cover your rear-end'. (I know, I

was once a state employee.)

The FDA is striving for perfection. Adding random trials adds costs to often

small companies that can ill-afford clinical trials in

the first place.

Meanwhile, patients die.

But, who cares!

As long as you avoid the sensational headlines in the media blaming you and the

FDA. I wish both entities would grow up.

How many CML patients died waiting for Gleevic to be approved? Gleevic has saved

many lives since it was approved, but not those who

died while the FDA dawdled.

We ain't dealing with hair transplants here, folks. But I guess we're just

numbers to the bureaucrats.

*************************************

FDA panel wants stricter standards on cancer drugs

Posted by: " cllcanada "

cllcanada@... <mailto:cllcanada%40>

cllcanada

Tue Feb 8, 2011 4:23 pm (PST)

If you think CLL drug approval is slow now... it looks like it is going to get

slower...

Drug companies are in some cases failing to do the required post-approval

(fast-track) trials...

quote:

Randomized, controlled trials should be the default position for all trials, "

said panelist Lyman, MD, PhD, director of the

Duke Comprehensive Cancer Center in Durham, N.C. " We're not doing anyone a

service doing single-arm trials when a trial with a

control could easily be done. "

We frequently get sponsors [drug companies] who say 'What's the lowest number of

patients and response rates you'll take?' " said

Pazdur, MD, the director of the FDA's Office of Oncology Drug Products.

[Guest guest]

I did some research for a person who had two children with

cystic fibrosis. I contacted a number of well respected

researchers, 4-5 to be exact, and got NO replies. NO does

not mean nitric oxide, either. There is a huge climate of

fear in the research business, and researchers don't want to

say anything that could jeopardize their research and

implicate them should a patient opt to start taking even

natural medicaments that are " not fully tested " and perhaps

have a reaction to the materials that are often available in

nature food stores.

Plus, the researchers want credit for their work, it's the

way they get acclaim and promotions.

In the meantime, even if a material should do very well in

Stage 2 testing and appear to have a very bright future, the

researchers will very often refuse to say anything at all.

In the meantime, Rome burns and the patient regresses and

then needs a lung transplant. We can see the point of the

researcher, can we see the point of the afflicted? The

answer is very, very obvious. No! None of these researchers

gave me the time of day. Nothing, nada, zip.

In this case, the children afflicted with CF have to either

do their own research or find somebody else who can read the

papers and make germane suggestions. Ironically, despite the

penchant of the profession to eschew " natural products in

favor of synthesized molecules " , many of the new testing

protocols are using things like flavonols from soybeans,

resveratrol from grape skins, NAC obtainable from nature

food stores, turmeric as a source of curcumin, etc. Mein

gott, imagine MD's using natural products, things they very

often pooh pooh as being phoney baloney and bogus!

The medical practitioner taking care of these two now-adult

children has taken the " safe " road. If it is not FDA tested

all the way, regardless of whether or not stage 2 testing is

look very good or not, this doc refused to deviate from the

well worn path. It's fine and dandy to stick to the well

worn path, but now one of the children, age 23, is told he

needs a transplant. And, some of the testing has gone on for

several years, often many years, but the practitioner did

not appear to be aware of testing or uninterested in trying

to help the kids with materials being shown to be very safe

from prior usage in many societies throughout the world.

Mere medical hubris, that's all.

[Guest guest]

Well .. very confrontational posts from both and . , your

communication style may be why you received NO responses. , really

unproductive generalization about government employees.

I think there is some very exciting research going on, especially in CLL right

now. If you are fortunate to be close to a research center, then make yourself

available, donate your blood, and become a positive contributor.

Lynn

[Guest guest]

I appreciate the frustration.

Anecdotally, I've found the FDA government employees to be dedicated and highly

trained ... and importantly without financial conflict of interest in their

deliberations. They want to get the decision right, and they have a short time

to make the evaluations.

.... Reviewers also get cancers as do their loved ones, to suggest that they

don't care is possible only if you don't have experience with the people in the

agency and are unfamiliar with the history of drug review *

Regarding review turnarounds: The Prescription Drug User Fee Act (PDUFA)

mandates an expedited review of data (months) submitted to FDA for marketing

approval in exchange for user fees.

It's true that doing clinical research requires a lot of money, but the

alternative is no standards for drug approval - clear evidence that it helps and

doesn't harm. Without such standards, approximately 9 in 10 drugs proven

ineffective (harmful) would be on the market today.

Regarding Gleevec's review time, you can't assume it's effective prior to

testing it ... as most drugs are not. And for the atypical highly promising

drug that addresses an urgent unmet need, the company can set up an Expanded

Access program during the review time (and also recover costs now).

More on PDUFA and turnarounds - Copying from FDA:

" Fast Track, Accelerated Approval and Priority Review are approaches that are

intended to make therapeutically important drugs available at an earlier time.

They do not compromise the standards for the safety and effectiveness of the

drugs that become available through this process.

These revitalized FDA drug review approaches have yielded tangible results in

bringing safe and effective drugs to patients with serious diseases more

quickly. For example, since 1996, 68 drugs for cancer therapies have received

priority review and approval.

FDA reviewed Gleevec, a treatment for chronic myeloid leukemia, in * four

months.* Shortened review times have also brought promising treatments to

patients with HIV/AIDS more quickly. Kaletra for the treatment of HIV/AIDS was

reviewed and approved in 3.5 months. Pegasys, a combination product for the

treatment of Hepatitis C was approved for marketing in 4 months. "

Regarding the views of individual panelists, Lyman ... these vary. My

understanding being that the type of study needed is determined case by case.

All the best,

Karl

From: On Behalf Of

Sent: Monday, March 14, 2011 1:05 PM

> Employees at the FDA are government employees. The default position of any

government worker is to 'cover your rear-end'. (I know, I was once a state

employee.)

[Guest guest]

We see this plea, enter trials.... First on that subject.... In CLL, as Lynn

points out, one must be 'near' a center. Then the process just begins.

no easy task, especially for some who may not be in very good shape, have

families, jobs, or just no one to help. Not to mention, participation is NOT

FREE ! there are many attendant costs... tests that are not covered, just to

get into the trial... then in some cases drugs that are not necessarily the

'experiment' drug, but are part of the protocol, OR part of the treatment for

adverse events.... not covered in all cases at all... the travel even to close

by trials and the time away from work or family which puts a burden on those who

are not the patient. contemplating a trial alone is a daunting task. And

that's before one considers the potential risks.

We have to remember that CLL is a disease that we are discouraged from treating

until we MUST... since there is no telling what that time will bring, or how

many 'events' a patient may experience prior to when that " time to treat' will

come, and then will the trial they COULD manage to get to the one that they

would be accepted for is it any wonder that 'more participation is not seen?

Dr. Furman worries that single arm trials are possibly on the chopping block at

the FDA.... that would indeed be very sad since no drug company is going to keep

trying to make drugs for oncology, or even inroads, if they can't get them into

trials that are perhaps simpler, less costly, have fewer 'ethical' constraints,

and broader patient bases. rolling out trials is costly. (this is not a plea

for sympathy for drug companies, just a fact) so why would they go thru MORE of

that if it takes longer, costs more and it would be even harder to get

participants? Without single arm trials we won't have any new drugs.

Thus far.for CLL, MOST of the trials have been for pre treated patients.....

since most are discouraged from 'early' or pre emptive treating, again, who,

what and where? CLL kills. disables and discourages..... IT IS CANCER. just

like other cancers, perhaps worse in many ways since there is not rush to

treat.... aside from the psychological issues that brings with it, it means that

by the time patients get to 'treatment' many are not in shape to get the most

benefit... as brought out in another post today.

I've been on these lists long enough to be lucky enough to meet many of the

members. I've been trusted with off list posts which I will never share, but I

feel I've seen things that people are experiencing that they are fearful of

saying on lists. Trials are tribulations.. no doubt. financial hardships,

time away etc. Unlike other cancer patients, by the time we can even consider a

trial, our support is tired of the wait... exhausted from our slower pace, our

inability to participate, and endless appointments months apart when we again

come home with, nothing yet...... and then we are to ask them to bear with us

with the same furvor they MIGHT have had at the time of diagnosis when they

thought there was 'hope' of fixing this... and it's a trial, not a treatment per

se and it's going to cost MORE money and the best we can hope for is slowing

this down...... The frustration is out there,,,, it's just not seen on the

lists. believe me.

Perhaps ONCOLOGY drug approval needs to be thought of a little differently than

approval for drugs for nose bleeds. Those drugs are intended to solve a symptom

or problem for folks who presumably have a normal life expectancy.... We

don't... no matter what stage we're at.... we have that sword over our head...

I think Scot is just showing some frustration, which can read to some as anger,

which I'm guilty of myself, and who can blame us. Every few weeks we lose

another member, a friend we've come to know, we've watched their struggle...

should we be less than frustrated?

Perhaps CLL needs a new image.... or a new treatment protocol, but it seems the

whole package needs to be reviewed in light of what we know now....One thing is

clear, if all the people factors involved don't come to the table and throw it

all on there we may lose all trials, and all hope of new drugs. So if it takes

some anger and frustration, maybe that's ok too.

wishing all the best. beth fillman

[Guest guest]

>>>Perhaps CLL needs a new image.... or a new treatment protocol, but it seems

the

>>>whole package needs to be reviewed in light of what we know now....

Right on Beth!

It is called Personalized Medicine and it needs a paradigm shift

from the regulator bodies to make it happen...

Read more Here:

http://www.personalizedmedicinecoalition.org/sites/default/files/TheCaseforPerso\

nalizedMedicine_5_5_09.pdf

~chris

[Guest guest]

Yes, yes and yes. I, too, agree with Beth. Well said. But again, I was only

trying to encourage those who are (justifiably) frustrated and angry at the

disease to look within and be selective where, about what and to whom they vent.

And, I was trying to encourage those who can, to build a relationship with a

research center, both for their own health and for all who come after us with

CLL.

So what do we do? Anger and frustration can fuel the engines of change or they

can eat you up on the inside. I'm worried, as has been pointed out, that the

trial and approval paradigm will not work for the new types of treatment drugs.

I'm also concerned that we can share with each other what we already know, be

nasty to each other (thanks for the private message calling me pathetic and a

blathering idiot, whoever sent it) and not move anything forward. We have to

find ways of being more productive and, at the least, support each other with

compassion.

A number of you are very articulate in your writing. We could really use a set

of spokespeople to deliver your message(s). with his Patient Power? It

looks like the FDA-savvy amongst us have some ideas .. What about LRF and LLS

... the lymphoma / leukemia advocates? If only someone with the megaphone of

Wayne Dyer would get on board with these concerns. (Did he ever acknowledge the

seriousness of his CLL?)

So please let us know if you are already doing something or plan to. I'm going

to check in with my local LRF group in San Francisco etc. As says, " We

are all in this together " . I'd rather be marching forward than fighting like

snakes in a bag.

[Guest guest]

Great email Beth...but there are other very important cll

specifics overlooked and under or never stated by

researchers, doctors and patients...a cll pr campaign is

sorely needed which should be targeted to general

public....FDA....media, et. al. A celebrity who either

knows someone with cll or has it themselves would be great

in making this disease more " public " to say nothing of

getting more interest and money for research et. al.

It seems to me one of the biggest problems is there is no or

little old and new information getting out to the media,

general public, and the FDA...and i don't mean necessarily

doing the same speeches (usually paid for by the drug

companies) to the patients......in other words, we need to

educate those who don't know what is going on with cll,

explain what it is, especially now with all the new

scientific information being uncovered..and it should start

with doctors and researchers involved. Rather than giving

the same basic talk to the patients about the same generic

topics a great many of us already are aware of , educate the

" other " public....many CLL patients are able to educate

ourselves through lists and groups like this...yes, the

basics are helpful, but how many times can we go to these

lectures and hear much of the same fundamentals over and

over...

Tell it like it is the frustration involved with w and

w...no immediate steps are taken as with other cancers to

get the cancer out of your body...it isn't leaving...and

there is no cure yet...more and more toxic treatments...but

each one taking more and more out of you...being afraid to

go into a room with children or grandchildren if they have

colds...the knowledge cll patients live with that the cll

can turn into a more deadly form of leukemia...or that other

that they are more prone to other cancers....the list is

much longer ...but you get the idea...

***Stop calling cll the " best cancer " to get...or saying,

" you don't die from cll " . or " the tx you get is " light

chemo " ...it makes cll sound like a no big deal cancer to

others not afflicted with it...and thus...why do

research...why do trials, why give $$$$ to find a cure since

there are treatments available...it's a cancer of the older

person......etc...etc..

..I do hope you can all see where i am going with these

thoughts...

IMHO

Adrienne

[Guest guest]

Adrienne,

Very nicely put. I especially like your last two paragraphs, about the

fears and frustrations one faces with

W & W, being around children, not only with colds, but with chickenpox,

with live vaccinations, and with the " best cancer " mention. I've used that

myself, and then thought I might have minimized myself to my family and

friends, because I'm not " sick " right now, since I've been in basically good

health since my six weekly infusions of Rituxan which were completed just

exactly five years ago. We don't need to be selfish, but we don't want to

play down the potential for what might be in our future.

Norma Oxley, age 73, dx 12/05; tx 2/06 to 3/06.

In a message dated 3/16/2011 3:46:55 P.M. US Mountain Standard Time,

ajunior923@... writes:

Great email Beth...but there are other very important cll

specifics overlooked and under or never stated by

researchers, doctors and patients...a cll pr campaign is

sorely needed which should be targeted to general

public....FDA....media, et. al. A celebrity who either

knows someone with cll or has it themselves would be great

in making this disease more " public " to say nothing of

getting more interest and money for research et. al.

It seems to me one of the biggest problems is there is no or

little old and new information getting out to the media,

general public, and the FDA...and i don't mean necessarily

doing the same speeches (usually paid for by the drug

companies) to the patients......in other words, we need to

educate those who don't know what is going on with cll,

explain what it is, especially now with all the new

scientific information being uncovered..and it should start

with doctors and researchers involved. Rather than giving

the same basic talk to the patients about the same generic

topics a great many of us already are aware of , educate the

" other " public....many CLL patients are able to educate

ourselves through lists and groups like this...yes, the

basics are helpful, but how many times can we go to these

lectures and hear much of the same fundamentals over and

over...

Tell it like it is the frustration involved with w and

w...no immediate steps are taken as with other cancers to

get the cancer out of your body...it isn't leaving...and

there is no cure yet...more and more toxic treatments...but

each one taking more and more out of you...being afraid to

go into a room with children or grandchildren if they have

colds...the knowledge cll patients live with that the cll

can turn into a more deadly form of leukemia...or that other

that they are more prone to other cancers....the list is

much longer ...but you get the idea...

***Stop calling cll the " best cancer " to get...or saying,

" you don't die from cll " . or " the tx you get is " light

chemo " ...it makes cll sound like a no big deal cancer to

others not afflicted with it...and thus...why do

research...why do trials, why give $$$$ to find a cure since

there are treatments available...it's a cancer of the older

person......etc...etc..

..I do hope you can all see where i am going with these

thoughts...

IMHO

Adrienne