Patient Thoughts on Medical Tests for Research

Here is a guest post I wrote earlier this year for METAvivor called “How Do People Feel about Bone Marrow Exams?” It was based on a study with Bonnie King from Stanford. While this may be one of the more gruesome-sounding medical procedures that some patients go through, it is not necessarily unique.

Too often, researchers think about all of the cool data they can collect during a clinical trial or research study, without thinking about what is would be like to experience all of those test procedures. Well, patients think about it, and often wonder what is wrong with the researchers!

I’m always perplexed when I hear about “adherence” issues in clinical trials. It used to be called “compliance” but that wasn’t as accurate, and brought up more negative connotations for the research community.

The fact is, for patients, endurance is the best term.

– Deborah Collyar

Staying in a clinical trial, or on any prolonged treatment plan, is an endurance test. There are many unpleasant, and sometimes risky, things that you have to do but hopefully you will get something out of it at the end. Patients hope for positive responses to treatment, or even remission, but that is not always possible.

x-ray film of the brain computed tomography

This is one of many reasons why it is important for those of us who represent patients to be involved in research discussions, from conceptual design to trial completion. We ask questions, such as, “Why do you need x number of these tests? Are they absolutely necessary to answer the questions in this study? What else could be done? Have you thought about asking the patient?”

Let’s work together to make the experience of participating in clinical trials as smooth as possible. Trial participants contribute so much already – they deserve to respect and consideration when we ask them to do things for research.

 

How Do We Get Better Research Results?

We learn about diseases and illnesses from research studies. Some focus on treatment (clinical trials), while others study groups of people (observational studies). Unfortunately, many studies are unclear, wrong, or can’t be easily transferred into everyday medical practice.

Study designs actually matter. They determine whether a research study helps real patients or just asks esoteric questions to further careers or make profits.

There are many ways to improve research studies. Let’s start with observational studies.

Observational Studies

There is one very simple concept to remember about observational studies:

different-paths_M15wj8_dCorrelation does not mean causation!

In other words, just because A and B fit together (are related, associated, or correlated with each other), it doesn’t mean that A caused B to happen.

HealthNewReview.org posted a great article about this, called Observational Studies – Does The Language Fit The Evidence? – Association Versus Causation.

There are several types of observational studies. This chart explains the pros and cons of each type.

F. Perry Wilson suggested a (better?) way to design observational studies in a recent MedPage Today post. A regular problem with these studies is that real life includes many things (factors) that researchers don’t include in studies:

“There are always other confounding factors that we didn’t think about, or we didn’t measure.” – F. Perry Wilson, MD

He suggests a method that helps build in factors that traditional study designs often overlook. Wilson did a great job of explaining both of these concepts in his article:

  • Randomized clinical trials (RCTs), known as the gold standard of clinical research, and
  • Instrumental variable analysis (IVs), which has not been popular, even though it has been around for over 80 years.

Study designs need to answer questions that apply to real people. This is why some of us work with study sponsors to infuse the patient perspective into study design and implementation. In case you are interested, here are some examples in articles and presentations. Just let me know if you’d like to join us.

There are lots of issues in trial design – this is just one. Future post material – aren’t you excited?! Please share your thoughts or add resources on observational studies in the comments. Thanks!

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.

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.

Clinical Trial Patient Summaries Win!

Congratulations to the MRCT Center of Brigham and Women’s Hospital and Harvard and team for winning the 2015 Award for Excellence in Human Research Protection from the Health Improvement Institute! The winning project recommends ways to write public summaries after a clinical trial is over.

Why Is This Important?

  • People join clinical trials (research studies) so researchers can learn if new treatments, tests, procedures, or other things work better than what is now offered to patients.
  • People who join the trial are research participants, and contribute a great deal – sometimes their very lives.
  • Participants are told they’ll learn important things about the study, but they rarely receive a summary of what happened during the trial. Until now (hopefully).

Thanks, Europe!

The European Medical Agency (EMA) created a new rule that says ALL clinical trial sponsors must create a public summary within 1 year after a clinical trial is closed (6 months for trials with children). This rule scared sponsors (aka drug companies, government, and private funders), so the MRCT Center assembled a Return of Results (ROR) Working Group (WG) to help.

Why did it take a government regulation to make this happen, you might ask?
Oh, they must have been busy with things more important than telling people what happened (sigh).

What is the Return of Results (ROR) Project?

sign direction expect-result made in 2d softwareThe MRCT Center ROR project focuses on patient needs (for a change!), instead of just the research system. This means including principles about plain language, health literacy, and how to explain numbers (numeracy) – making sense of clinical trial results for normal people.

The ROR project includes:

  • A Guidance Document: how to set up a process to create patient summaries.
  • A Toolkit: with helpful templates, non-promotional language, and checklists.

We studied a few groups who actually create clinical trial result summaries, like CISCRP and the Alliance for Clinical Trial in Oncology (disclosure – I run the summary project there).

The Award

“The award program recognizes submissions that our judging panel determines have demonstrated excellence in promoting the well-being of human research participants.”

– Dr. Peter G. Goldschmidt, President and Founder of Health Improvement Institute

Quite a mouthful, but nice recognition! They agree the ROR Project puts patients first.

Thanks to All

Thank you

Thank you

As Co-Chair of the MRCT Center ROR Working Group, along with Laurie Myers from Merck, we thank all 53 team members (pp. 2-4) for creating ‘how to’ information for all clinical trial sponsors to create plain language study result summaries for real people. Thanks also to Barbara Bierer, Rebecca Li, and the MRCT staff. We’ll continue to update as new information becomes available.

A Plea to Sponsors

The tools exist, so let’s get started NOW! Don’t wait for the EMA ruling or for the U.S. Food & Drug Administration (FDA) to follow suit. Patients, trial participants and the PUBLIC want these NOW. And some of us can help. Really. Contact me!

After all, it’s the right thing to do AND you really need some goodwill, but that’s another story…

What YOU Can Do

Please ask for a public summary of any clinical trial you want to know about. You can find most trials listed at https://clinicaltrials.gov/. And ask them to stop calling them “lay” summaries (what does that really mean, anyway?).

More information on clinical trial result summaries is in this post.

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.

Last Chance to Say Your Word on NPRM

I posted “Informed about Changes to Research Consents?” on 11/15/15. You now have until 1/6/2016 at 11:58pm EST to send your comments about the changes that will affect hundreds of thousands of patients, researchers and institutions (at least). PLEASE send in your comments!

Here’s how:

  1. Read my blog post on 11/15/15 to refresh yourself, and feel free to use any part for your comments.
  2. Look at the summary of changes, according to the US Office of Human Research Protections (OHRP).
  3. Read some of the public comments that have already been posted (so far, there are 957 comments). Here is an example of one of them from the Genetic Alliance – feel free to use any part for your comments.
  4. Click if you want Tips for Submitting Comments (go to bottom of page and click on .pdf file).
  5. Post YOUR Comments at this link!
  6. Feel free to share your comments below on this post if you want to discuss them.

I’m finishing my comments now and will post them… but don’t hold your breath – create your own comments NOW! GO FOR IT!