Monday, December 8, 2014


Research saves lives.

Research ideas begin with a thought.

Beyond the thought, it becomes an idea, a concept, a design.  Tissue studies, animal studies and if the thought seems to hold up throughout the process, ultimately, the life saving research requires human clinical trials.

The process from the thought to the patient can be decades.  Since that's not the purpose of this post, I'm not going to seek out the necessary information to back that length of time statement.  I'll just say, "It takes a damn long time" and leave it there.

All Trials is an initiative I've supported since I learned about its existence.  It irks me in ways that I can't describe to know that study results are not always reported. is the repository for trial information.  It's cumbersome to use but it's there.  My aside comment:  Maybe the government should consider an overhaul of the site including some real, "normal" patients on a committee and I'd like to toss my name out for consideration.

And, as much as I'd love to be involved in a project like this, the unfortunate thing is that patients might be included as tokens or as unpaid consultants and frankly, my time and my efforts can't always be pro-bono.  The experts get paid and the patients, the real experts, should simply bask in the honor of being asked.  I'd rather bask in the glow of holding the hand of someone in need, in choosing small projects to help organizations whose efforts align with my beliefs.  I will gladly be first in line to volunteer my time in those capacities but frankly, when others are being paid for input, it's a tad insulting to be seated at the same table at my own expense.  HUGE digression that has nothing to do with this post either.  I think I just had to get that off my chest.

I need your help.  We need *all* study results to be reported.  Right now, the so-called negative findings are considered failures and are not reported in trial results.  From where I sit, there is no such thing as a failure when it comes to research.  A failure to meet a trial endpoint means we LEARNED something.  I don't see that as a negative anything.  We've ruled out one more needle in the proverbial haystack.  We've rid the haystack of one more "barking up the wrong tree" idea.  That's good.

These results are not reported.  Not only is that a disgrace to the taxpayers whose dollars fund this research, it is absolutely disgusting to the patient population that chose to enlist in one of these "failed" trials.  What?  Their enrollment meant nothing? They contributed to advance the science and their efforts are in a paper shredder.  That is the epitome of indifference by those who withhold evidence from those who put themselves out to participate in accumulating that evidence.

It's just wrong.

Currently, there are two complementary initiatives to change this paradigm and I support each of them.  Bear with me as I make a run at explaining why there is a need for two separate proposals and why it is imperative that each of us add our voices to support the changes.

The National Institute of Health aka The NIH, under the leadership of Francis Collins has a pending proposal that would require certain clinical trials to report ALL outcomes for which it has provided any source of funding.  Currently, according to the announcement, "scientists fulfill their obligation to contribute to the general body of knowledge through peer reviewed journal publications."

The entire peer review process can be a problem for a number of reasons.  Journals, customarily, are not available to the general public.  If a study is rejected for inclusion, it is a small number of people making the decision that the results of any given study are not important enough for their journal.  Not only is that an elitist stance, it removes the patient population from access to seeing the study information that is published unless we are willing to pay upwards of $35.00 to access.  Last I checked, there are no patients on the panels that determine what is considered publication worthy.  I believe there are studies that would be of tremendous interest to specific patient populations but without a patient voice on those review panels, that whole system reeks of paternalism.  Sorry... digression.  Huge but important digression as this all goes to patient centered, patient powered, patient whatever.....

Less than half of the clinical trials funded by the NIH were published within 30 months of trial completion according a recent study referenced in the announcement.  Concerns about "selective publication of the results of some trials and not others -- or publication of incomplete or partial findings...... can lead to inappropriate conclusions about the usefulness of particular therapies."  I do believe we refer to that as publication bias but don't quote me on that.

The new criteria would prevent duplication of efforts.  This is particularly useful when the duplication might involve something that was already studied and determined to be unsafe and unsuccessful.  That's not rocket science, that's simply common sense.  Then, there is the matter of those individuals who show up.  The volunteers who participate in a study assume risks. As previously mentioned, it is the epitome of "you don't matter, regardless of the risk you took," to withhold study results because they don't meet journal criteria or they are considered a failure.  The patient didn't fail anything.  The study didn't meet it's endpoint.  Or, particularly with phase I studies which determine safety, the intervention was found to be UNSAFE.

At the heart of this proposal is the need to find other ways for results to be shared with researchers, clinicians and patient communities.  It's not about the journals, it's not about presentations at a science meeting, it's about having the information, all of the information, in a place that is accessible to all, namely clinical  You can read the red tape version, NIH Request for Public Comments on the Draft NIH Policy on Dissemination of NIH-Funded Clinical Trial Information here. Comments will be accepted through February 19, 2015 and may be submitted via email, snail mail or fax.

Fax:      301-496-9839
Snail:    Office of Clinical Research & Bioethics Policy, Office of Science Policy, NIH
             6705 Rockledge Drive, Suite 750, Bethesda, MD   20892

As for the second complimentary proposal, online comments will be accepted in an open forum on  Clicking on the Comment Now button will open up a new page where you can submit your comments.  They may be viewed by others (although the name field is required, "Anonymous Anonymous" is acceptable field input).  You may read some of the previously submitted comments here.

The second proposal involves changes suggested to clinical trials reporting which expands upon a previous FDA amendment. The document is over 100 pages in length.  Again, bear with me as I make yet another run at an explanation the rest of us might understand....

The key changes are in the PDF which is available for download located here.  There are two pertinent elements that jump out at me from a patient perspective.  Determination of what constitutes an applicable clinical trial (ACT) would be removed from the hands of the submitting researcher.  Instead, the FDA will implement the use of an algorithm to determine which trials fall under the new reporting measures.  Presently, the application includes submission by a responsible party to indicate whether the trial is an ACT.  New data information would be required within the application to assist in making the determination via the algorithm.

Also, it appears there will be rules in place for evaluating the use of already approved drugs for other purposes.  What we know as "off-labeling" would be incorporated into the changes being sought by the FDA.  Adverse event reporting requirements will be expanded.  Whether anticipated or not, adverse events that occur in more than 5% of the study population would have to be included in a second table.  From the way this is written, it appears this is presently not required.

This second proposal is much more specific in its goals and as a result, seeks more detailed public commentary.  Specifically, they are looking for comments regarding the advantages and disadvantages of including technical and non-technical summaries in reporting.  Additionally, opinions and remarks regarding information that might assist in the understanding of adverse event information is being sought from those who are providing commentary.

Of note, this is not cancer specific.  This applies to all clinical trials funded by the NIH and all trials involving FDA regulated products.  Also noteworthy, comments are being accepted from anyone.  In other words, you do not have to be a US citizen to weigh in.

I encourage everyone interested in advancing research at a quicker pace to support these changes by raising their voice by the February 19, 2015 deadline.  If petitions are drafted or sample letters are proposed by any organizations that help add the patient voice to this discussion, I will be sure to update here.  The reporting part is one piece of the puzzle I am only superficially familiar with.  I can review a grant application, I can help write a lay abstract, I can decipher published study results and translate them into lay language.  The reporting process and journal inclusion is the big abyss and it would appear, for the moment, it's by design.  That design must change.

If my brain allows me to clear itself enough from the mountain of personal medical stuff that continues to swirl, I will take a stab at drafting something on my own.  Right now, I'm still trying to learn about hypertriglyceridemia (which apparently could well have been fatal, but while I was still in emergency mode fighting over proper patient care, I had no time to learn and wasn't being included in any medical discussions).  Throw in an attempt to navigate the mental illness maze when someone is too sick for some treatment programs and not sick enough for others.  Add a dash of being stuck in the middle of that mess leaving one feeling both helpless and hopeless and I know something MUST change.  Lives are being sacrificed.  Yes, there is much in front of me and despite the ACA's promise of mental health parity, it doesn't exist.  It's all about the haves v. the have nots, particularly as it applies to the treatment of those with mental illnesses and I defy anyone to show me differently.  In this country, at this point in time, that is simply not acceptable.

I have much to do.  But this clinical trial change is an opportunity with a ticking clock and it's one I could not afford to share, despite the fact, I have much to do.

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  1. I just read your link to AllTrials. Thanks for posting, AnneMarie. This does seem very important and I'm surprised that reporting all results, at least in some fashion, isn't done as a matter of course. I'm so glad to know about this. As usual, this is a terrific post. Thinking of you and all else going on in your life. xx Bud

  2. AnneMarie, also, I know that privately funded drug companies are not required to report negative data and can kill a trial at any point for any reason. But I believe if a company or institution takes federal money, they do have to report the results whether good or bad but there is no time limit to do so. So those trials may be reported as "pending" and could be maintained that way for years even though development has stopped. There should be a time limit with all results reported.

  3. dear Annemarie,

    I have read this post twice, and in some paragraphs several more times. my brain just seems unable to maintain the cohesive thread I know you have worked so hard to provide, Still, I do perceive there are matters that simply don't make sense, and fail to flesh out the whole story, and agree those instances are decidedly wrong. so - I will go to the links you have provided and do what I am able to raise my voice. great post, valued and appreciated your strong, savvy advocacy. and I am wishing you success and resolution for all you are carrying upon your shoulders with multiple fiascos re: mental health, and your husband's illness. wish I could be your helper, but know that even though I can't always be as enlightened as I wish, I send you my admiration, my thanks and big hope for tackling all of it.

    much love,

    Karen xoxoxox

  4. okay - I'm back and I did it - went to ALLTRIALS, provided my information, wrote a comment, and signed the petition. it was easy, and the main issues laid out to the left of the page were very helpful to succinctly and adamantly state my reasons. I will give my brain a rest, then go to the other sites and share my input. thanks Annemarie for this important and timely issue you have presented, and for empowering me to participate. and I completely support the other issue you raised about patients at the table, who are the real experts receive no compensation. when you get further into that, let me know what I can contribute to help.


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