How to Work with Patient Advocacy Groups & Patients

It’s been awhile since I posted anything – sorry about that. Between excessive travel, new projects with new partners, and unexpected foot surgery, time marched on without me! I actually wrote 2 guest posts that I haven’t mentioned, though. Here is the first one:

Get Real with Patients and Their Advocacy Groups at Clinical Trials Arena. It summarizes a talk I gave at their April 2016 Clinical Operations in Oncology Conference.

People ask me how to work with patients and patient advocacy groups all the time.

Short answer: it depends on the disease, and what type of input is desired at different times. The post listed above offers some ideas.

Two more opportunities are also coming up quickly with eyeforpharma:

  1. Join Jamie Roberts from CTTI, Victoria Dibiaso from Genzyme and I on August 4th at 11a EST as we discuss how to Drive Value and make the Patient-Centric Trial a Reality. It’s free!
  2. I’ll also be speaking with many other patient advocates and companies at the Patient-Centered Clinical Trials conference October 10-11th. Be sure to say hi if you see me there.

There are more happenings in this area – look for future posts here on this topic!

Happy Summer…

A Clinical Trial Failed Patients

Something horrible happened – a clinical trial failed, causing one unexpected death and seriously injuring 5 people. And instead of dealing with a dysfunctional research system, “experts” are spouting off on their own. The few articles written to date focus on the drug (aka the money), not on what people want to know.

My Initial Thoughts

high wire climbing in the wood

ClinicalTrialsArena asked me to comment, due to my unique expertise that straddles the patient and research worlds. After researching the topic,* here are my concerns – most of which have not been discussed yet.

This poses a much larger question – why am I the one bringing these up?

Fact 1: Something really bad happened – the first 6 people to get “repeated higher doses” died or were seriously damaged. This is a clear sign of system failure (e.g. approval, protocol, procedures, formulation, PharmcoDynamics).

Fact 2: A phase 1 clinical trial means it is the first time a drug is studied this way in people. 127 people in the trial is far larger than classic phase 1, and most don’t have placebos. Isn’t this more like a phase 2, or the new-fangled phase 1-2 trial? If so, what happened to the rules?

Fact 3: The drug showed activity in laboratory dishes and animals first. We don’t know what mouse-realtesting was done, or for how long. How realistic are the animal models, and how closely do they relate to humans?

Fact 4: Existing articles stress the need for more ‘transparency’ to share the drug’s molecular structure. Patients want honesty first, which in this case may mean, who screwed up?

Fact 5: I’m a big believer (and trail blazer) in presenting trial results, but that’s when we have results. NOT during an ongoing, active clinical trial. Why aren’t we calling for the protocol to be publicly published immediately?

medical_1000006456-120613intFact 6: Healthy volunteers joined this trial after reading an ‘informed consent’ form about its procedures and risks, and they were paid well. Why isn’t the consent form (for every trial) publicly available once the trial is approved?

Fact 7: In the U.S., the Institutional Review Board (IRB) would be shut down and investigated before any new trials could be opened.

Fact 8: A thorough investigation on all parts of this system failure is obvious. This, too, should be open and as public as soon as possible, and at all times.

Fact 9: Risk is an inherent part of clinical trials because risk is part of everyday life. Even when rules are followed, bad things can still happen. Patients aren’t stupid, so there is no need to shut down other clinical trials.

A Setback for Other Clinical Trials?

Amazing we even have to ask this question, right? Of course it will!

Only other researchers may think their trial will be ok because, oh let’s see, because that trial was different – yes, that’s it! That trial (take your pick):

  •  was in a different disease
  •  was in a different country
  •  was run by a company I don’t know
  •  had a different study design
  •  was a horrible accident that would never happen to me
  •  add your own excuse

But patients won’t care about any of that. They’ll steer away from danger or discomfort. And let’s face it, anything called a “clinical trial” isn’t comforting. And as far as whether the rule were (or weren’t) followed – that doesn’t matter either. The rules in the case of a clinical trial include the protocol, of course.

I am firmly in the patient/participant camp. Some may call this a bias, but I consider it the only worthwhile endpoint. I know patients want better answers NOW, and I know people in research deal daily with regulations and hubris.

There was clearly a system failure here, and people needlessly paid too high a price. Let’s find out why and make the pieces work together so people won’t worry about needlessly putting their lives on the line.

* Online information (as of 1/21/16) about the French clinical trial from a Portuguese company is located on Facebook, at #clinicaltrials on Twitter, and is listed in articles from:

All content © 2016 by Deborah Collyar unless otherwise specified. All rights reserved. Permission is granted to use short quotes provided a link back to this page and proper attribution is given to me as the original author.

When Clinical Trials Don’t Match Their Data

dart off-centerClinical trials (research studies in people) can help find better treatment, care and prevention for patients. When done well, they answer important questions posed in the protocol (study plan) before the trial starts. Unfortunately, those answers are often not published. Some also don’t have the right study designs, which means the studies won’t tell us what we truly want to know.

The Problem?

…”the international Cochrane Collaboration commonly exclude from evidence 50% to 75% of published studies…”

– Soumerai, Starr, and Majumdar, The Health Care Blog

Why? They don’t meet the basic research design standards that Cochrane relies on to produce reliable results. 1/2 to 3/4 of our clinical trials? Big problem! But it’s only one of the issues.

Why Does This Happen?

Many misalignments help foster these problems. In their article, Soumerai et al show how clinical trialists start with errors that get multiplied as they gather steam, especially when those who will profit spin stories that get picked up by the news media.

“We will show how single, flawed studies, combined with widespread news media attention and advocacy by special interests, can lead to ineffective or unsafe policies.”

– Soumerai, Starr, and Majumdar

John Ioannidis at Stanford has written about this for years, and was recently interviewed by Gary Schwitzer from (podcast available). John published a seminal paper in 2005 called “Why Most Published Research Findings Are False.” Sorry to say that 10 years later, it is still true and will be until incentives change.

The COMPARE Project has taken on a simple (but not easy) goal – to find out which clinical trials reported on the outcomes (endpoints) that they stated in the protocol (study plan). As of 12/10/2015, the 58 trials they checked had these depressing results:

  • 8 trials (14%) reported the stated outcomes and they didn’t add later outcomes.
    That means they did what they said they would do. Thank you.
  • In the 50 trials (86%) that did NOT report on the stated outcomes,
    • They did NOT report on 306 of the original outcomes.
    • They added 304 different outcomes after they saw the data.
Photo credit: stevendepolo via / CC BY

Photo credit: stevendepolo via / CC BY

That’s like waiting until everyone turns over their cards before you decide how you want to play! There can be valid reasons for some, but certainly not all, of these changes. Retraction Watch has a good post about this.

To follow up, Ben Goldacre and his team send letters to journals who published the trial results to alert them to errors so they can issue a correction.

…”we entrust journals with an incredibly important job, a huge portion of the knowledge management in medicine and science.  We should be able to expect that journals routinely police something as unambiguous as outcome switching, during the peer review and editing process. Where there are slip-ups, we should be able to expect that those errors are corrected swiftly.”

– Ben Goldacre

Why Does This Matter?

Patients rely on doctors for accurate information that can be used to make critical decisions. Physicians also want this, and often base their decisions that treatments off of these clinical trials. If the evidence isn’t real, it could actually lead to wrong decisions that might actually create harm to patients. And no one wants that, do they?

What are publishers doing about this?

The COMPARE Project is relatively recent, so maybe it just takes publishers longer than expected to publish the team’s letters and then issue corrections. Right?

Old letters in string - deskI mean, it only makes sense that such esteemed journals as the Journal of the American Medical Association (JAMA), The Lancet, the Annals of Internal Medicine (Annals), and the British Medical Journal (BMJ) would take this seriously and correct errors quickly. And they would commit to checking something simple like this for each clinical trial BEFORE it is published. Right?

Millions of patients who count on the accuracy of their publications certainly hope so.

All content © 2015 by Deborah Collyar unless otherwise specified. All rights reserved. Permission is granted to use short quotes provided a link back to this page and proper attribution is given to me as the original author.