Re: clinical research: safety & end points and the roads to get there

[Guest guest]

The road to....... is paved with good intentions... we all

agree. I don't think it's 'research' we have problems

with, unless it's lack of funding for more of it, but rather

TIALS, but some of the same issues prevail in both arenas.

These two may be related, but are not necessarily the same

beast. Both are subject to the same human frailties, and

foibles.

While I don't disagree that there should be greater thought

to how and when the " new " treatments we hope for are

approved, however: here are some thoughts on this subject

that in my opinion, after giving this great thought, I feel

we need to be aware of when we plead for new treatments,

faster.

all the legislation, regulation change etc cannot ever force

some researchers to behave in an honorable and responsible

fashion. All professionals arrive at junctions of judgement

on a regular basis. Many times they cross over some lines

and we hope they do so in OUR favor. However, once they do,

the next cross becomes easier. The next time it could be

the other side they go with. After all, if they cross a

line in one direction, the human mind can easily trip over

to, " well, er, maybe I should give back to the other guy

now " . Being human they may feel they stretched a bit but if

they go the other way, just this once, it will make up for

the other stretch. things will then be even??

there are major stumbling blocks faced by both the medical

world and the commercial world of drug invention and

manufacture. Sadly, money is central to both factions.

Where there is money involved there are additional

influences that sneak into the formulas of what might be ok

or not ok. We all like to think that the medical team is

out only for our well being, while the " nasty " drug

companies are only interested in money. And of course, the

'government' is out to cheat us..... This may or may not be

entirely true. At least not all the time.

What happens, for example, when a particular trial

participant suffers severe events due to a drug trial? We

have had these events and most of the time, statistically at

least, they are resolved without fatalities. However,

there was one such event recently with one of the new

hopeful drugs and the trial team failed to respond at all.

This event has caused me to re think some of what I thought

was an easy position, that of " of course, speed it up, what

are they waiting for? " The patient was left to fend on

their own, and as a result of lack of care is now

permanently disabled. Others who have had the same

reaction, who WERE followed, were able to continue with the

trial, with a hiatus, and seem to be doing fine. This is

not an isolated case. Which patients will be listed as an

AE on the reports? Neither died. do we want to know about

both? do we now? NO.

this event posed some serious questions in regard to

regulations and the process of getting to approval of the

drug. First, does this patient go back to that center for

follow up NOW? Do they allow further testing at this point?

If the trial center now wants tests, for whom are they being

done? Who pays for these services, or for the weeks in

intensive care he endured? Remember please that others who

have had the same reaction were seen promptly, removed from

the drug, and continued later. (this I suppose is what we'd

call acceptable risk, NOT responding to a patient's needs is

NOT acceptable risk) AND does this EVENT ever get onto the

trial report of that trial center?

The state of affairs, at least in the US at this time is

that some states mandate insurance to pay for any drugs,

that are included in a trial, other than the presumably

'free' trial drug. and in some states, treatements

resulting from trials, even in some states, costs associated

with a trial, such as travel.....Others do not. Most don't

at present or do some very limited version, it's very

uneven. While this is a proposal, it is not in place at

this time. Would it be appropriate for the drug companies

to use some of their lobbying sway to push for universal

legislation to allow this? Might that not allow more people

to participate if that were the case? MIght it not allow US

to more safely enter trials that we might not be able to

otherwise? Perhaps we should start there? Might this be a

way to get new drugs to us sooner?

The costs for that one event have risen to over $100,000,

not covered by their insurance. In fact, their insurance

would not pay to fly him back to the trial center, but did

pay to get him to a critical care center, equal distance

from their home, in their own state. (not a trial center,

but thankfully one that had CLL doctors) Of course the

trial hospital had not contacted them, or the new doctors,

but even if they had, would they even want to go back there?

But suppose they had behaved properly and wanted him back?

should the trial pay for that so that they could get the

information they needed and record it correctly? OR even

help the patient.

A fear now exists in that family as to coming out with this

information. A fear that the patient will be denied

treatment elsewhere.. this should not even be an issue, but

I'm afraid I can't say it's entirely unreasonable. Might

this patient then be denied other trial entries, assuming

they would qualify medically.

As for CT's, I don't share the panic that many seem to have

over the CT's required by trials to evaluate not just the

progress or lack thereof, but also the presence of NEW

cancers that arise during the course of treatment. this can

be the case, whether these are due to the new drug or were

there all along. Might the drug companies fare better if

these were never known? I don't think we can assume that

the CT's are only for THEIR benefit, and only serve to

damage US. Those scans can and do discover other problems,

not only cancer, that may arise from the new drugs. Don't

WE want to know that? While " they " might be only too

willing to 'not know' what isn't looked for.

Age at exposure is known to increase risks of radiation

exposure. Trials are age specific and the ones we are

concerned with are mostly for patients that are already over

about 40 or 50. Prior to age 8 or 10, when the immune

system is immature, radiation is many many times more

damaging. Those bodies are also considerably smaller. The

patients who enter trials already have cancer, perhaps more

than the one they are in the trial for.

Do I believe that random CT's regardless are a great idea?

NO. And patients entering a trial SHOULD be told how many

and when CT's etc will be required. However there MAY be

events that arise during a trial that require further

scanning. (such as the case I just mentioned) However I do

think it would be a mistake to cut out most of the scanning

to speed up the arrival of new drugs to the market. (I

don't think the speed of approval would be affected due to

scans in any event)

This 'fear' of radiation is new. The US and other countries

have had a long love affair with radiation. Several

countries, especially the US carried on major radiation

experiments, NOT trials, using various forms of radiation

until the mid 1970's ! Doses were far greater than any of

the average cancer patients now get, unless perhaps they are

given radiation as part of their treatment. Many of those

now proven to be carcinogenic in themselves. Trial patients

already have cancer. The courses of their diseases are

largely unknown. Risks are part of any disease whether

treated or NOT. I find it ironic that the warnings are now

coming out at the same time that the government is pressed

to provide equal or more equal medical care for all

regardless of ability to pay. At the same time they are

warning us to avoid radiation, they are awarding hospitals

premiums if they cut back their use of scanning tests to

some 'acceptable' level. In fact, US hospitals are now

under pressure to comply with these new guidelines OR lose

their medicare/medicaid reimbursements ! Do we want our

doctors to even think that if they might find a benefit to

scan us that they may also be scrutinized for going over

some 'acceptable' administrative level?

I can see a day when radiation tests will be rationed and

decisions as to who should get them determined by forces we

may never know. I don't think we want that to happen

either.

Just some thoughts when advocating for faster approval of

new drugs. How fast, how to get there, who pays, who is

driving the bus.... but mostly where does the buck stop?

be well, Beth Fillman

[Guest guest]

When I was a child they did experimental radiation on me a

number of times to get rid of plantar warts it did get rid

of them but I wished my Dr didn't send me there! I do wonder

if my CLL could be from that!