Sunday, December 7, 2014

Patient rights in a clinical trial. An oxymoron???? Redux...

This post is a bit of a re-hash from a recent conversation on one of the melanoma forums.  Forgive me if you've already seen it, but I keep thinking about it I thought it was worth a post for those of you who did not.

It began with 'M' speaking out about her frustrations when dealing with Big Pharma and the way clinical trials operate.  Her husband has been through a lot.  Most recently he joined an ipi/nivo trial which miraculously rendered 8 lung nodules and 5 lymph nodes shrinking or inactive on subsequent scans. However, he developed hemolytic anemia and did continue to have 1 or 2 pesky lesions in his lungs that did not respond.  So, M was very frustrated that though the hemolytic anemia was resolved in February, BMS removed her husband from the trial, and even more disturbing, was the fact that they had no interest in an examination of the tissue that will be available after his upcoming lung surgery to remove the remaining active nodule.

An 'anonymous' Pharma/Clinical Trial defender replied {CAPS = theirs}:   
    "If he is still on a trial, BMS has their hands tied.  There are criteria for staying on a trial DEFINED BY THE FDA and if those criteria aren't met, the patient is dropped.  These criteria are setup PRIOR to the clinical trial starting and only people who fit this criteria can be used in the final drug approval.  The only thing you can do is ask if he could be treated "off protocol" - basically someone who doesn't fit the criteria needed for approval.  The reason you signed so many papers is to protect you and the company, and those papers clearly define the protocol for staying on the trial.  FDA requires this and it is the nature of clinical trials."

I really don't get these folks.  Are they simply completely taken in by the Big Pharma propaganda of 'poor pitiful us, just struggling to make a living and help the world' that their PR departments spew?  Do they just speak out of turn because they have no idea what it is REALLY like to be on a clinical trial?  Or, are they afraid that somehow Big Brother (I mean Big Pharma) is watching....and they don't want to be caught criticizing lest they somehow lose out on treatment when THEY decide they need it???  I really don't know.'s what I said:

Oh, M!  Sorry you are having to deal with all this.  When I was a little girl Marie Curie was my hero.  She still is.  Her work in radioactivity paved the way for the radiographic treatments and diagnostics that exist today.  But perhaps something she contributed apart from her work made an even greater difference.  She refused to patent her processes!!!!  Thus, allowing many other researchers to work on radiation and her ideas simultaneously...moving science forward exponentially....rather than waiting for only those researchers and institutions who could afford it to participate; to build on something that, in her view, belonged to humanity.  Marie Curie's today are few and far between.  Certainly BMS, Merck, et al. operate in a manner that seems to be lacking such a creed.

To the Pharma Defender's point. Yes, that assessment is pretty accurate regarding clinical trial protocols.  However, it is not so clear cut and easy as all that.  Additionally, the protocol is not as much in favor of the patient as is implied.  I have signed many such documents.  They all basically say:  "You will be getting this, for this long.  Here's how you qualify.  Here's how you get kicked out. If you live, die, grow three purple be it.  The company, researcher and the research institution will bear no responsibility....ethically or financially.  Costs will be paid by you for this, this and this....and for all expenses related to any side effects that may develop."

Now, as a patient...of course...I agreed.  I life and my rights away.  Patients are desperate.  No one signs up for a clinical trial when there are better options available.  And while various protocols are more fair to the patient than others, they are a legal document designed much more to protect the providers than the rights of those on the receiving end.  The best we can hope for as patients is that we will be giving "informed" consent.  Additionally, researchers, institutions and Pharma change the protocol ALL THE TIME!!!!  I have signed innumerable iterations of my ONE trial!!!  Never once has a patient been able to exact a change in such a document!

Additionally, for those who like to tout the lack of control Pharma, the FDA, and researchers have - I beg to differ.  Exceptions can be made any time they choose to make them.  They just don't often choose to do so.  For instance:  Take the case of Ralph M. Steinman.  Don't know Ralph?  Well, he was an amazing researcher in the area of the immune system and cancer....very smart....was awarded the Nobel Prize just three days after his death in 2011...and apparently, a really great guy.  Well.  Ralph got pancreatic cancer.  Perhaps the only cancer along with renal cell carcinoma that gives melanoma a good name.  So what happened?  His research buddies, folks at the FDA and various review boards pulled every string in the book.  "In addition to standard treatment, Steinman ended up enrolled - under a special patent provision - in four ongoing clinical trials of various dendritic cell-based cancer treatments, most of which were not even being tested for pancreatic cancer, along with several other experimental immunotherapy and chemo treatments.  Schlesinger, a member of the Rockefeller Institutional Review Board, steered his treatment through all the necessary IRB and FDA channels..."  [The Patient Scientist, Scientific American,  Harmon, Jan 2012]  Hmmm....  So, did Ralph deserve that special treatment?  Treatment that kept him alive years longer than most folks with his disease...and without said special treatment?  Sure!!!  I think so!  But!  Don't we all????  Must we all be brilliant, well connected scientists in order to be granted such care?

Finally, while it is obvious that patients cannot continue to take meds/treatments that result in life threatening side effects like hemolytic anemia....I think perhaps M's ultimate point was this....  If Big Pharma is really, truly interested in saving lives...not just making money....wouldn't they want to know not just what makes their med work, but what makes it fail?  What in those two last sucky little tumors in M's husband protected them while his other tumors responded to his treatment?  Wouldn't you want to know that?  Now, Pharma defenders will say, "Well, the FDA and pharmaceutical industry is not set up to do things like that when they run a trial."  And, those folks are right.  BUT....WHY NOT???????   Why aren't those in the drug industry REALLY set up to do REAL research?  Not just keep a tally of those who rise and fall? Achieve something more than a count of side effects?  Why ISN'T a research team set up to evaluate tissue samples and patients, de rigueur? 

'M', I wish you and your husband my best...with his upcoming surgery and whatever treatment path you seek next.  While there is much that is good in our system of clinical trials, there is much that is broken.  For what it's worth, this is one rattie who does not plan to stop squeaking anytime soon.
Yours, Celeste

M's response says it all:   
"Patients deserve to have some rights in clinical trials!!!  Unless you are a stage 4 patient that has been on a clinical trial, you have not a clue how powerless you are compared to the companies that make the drug."

Sending my very best to every rattie (plus a mouse or two) and their families - c

1 comment:

  1. Clinical Trials are definitely not what I thought they were prior to your experiences! I have been taken aback many times during your treatments with the various expectations they had of you and also with the fact that many of our expectations that were unfulfilled. I guess we did think it would be more like Marie Curie, but sadly it is more like big business.