Clinical Trials: How Can Patients Benefit More?

Sharing of data from clinical trials could help accelerate progress against cancer.These days the media hype around cancer “breakthroughs” seems to have reached a new high. There have been advances, but there is still such a long way to go before we can say we have real breakthroughs that are changing the outlook for most patients.

There are likely many reasons why progress is so slow. But one thing that would almost certainly make a big difference is if there were true collaboration among researchers conducting clinical trials.

Clinical trials are expensive and time consuming. Patients in clinical trials have chosen to participate not just for their own benefit but also to make a contribution for the greater good. And yet, the knowledge we as a society draw from many clinical trials is often incomplete or even nonexistent.

I wrote recently about highlights from the 2015 San Antonio Breast Cancer Symposium. One of the most interesting and eye-opening presentations was by Dr. Martine Piccart on lessons learned in the search for new treatments for HER2-positive breast cancer. The drug Herceptin, which has been available since the 1990s, has made a big difference for many patients. But it is not a cure and some patients develop resistance to it as well as the handful of newer medicines that also target HER2. Researchers are trying to find new targeted therapies that will help patients but progress is very slow.

Dr. Piccart said that current research efforts into HER2-positive breast cancer are failing to address the needs of patients. And the reason that this is happening is that researchers  are not collaborating and are not sharing data from their research.

Patients with advanced HER2-positive breast cancer want to know, among other things, “can I be sure that the chosen therapy will truly help me?” Patients with early stage disease also want to be sure that any aggressive treatment they receive will help them, and to know that if they receive a shorter or simpler treatment, it will work just as well.

There are lots of leads that researchers can explore to begin obtaining answers to these questions, Dr. Piccart explained. A common theme is finding biomarkers, or genetic signatures beyond HER2, that will help to identify patients who will or will not benefit from specific treatments or combination of treatments.

In other words, it’s all about precision medicine. But there are so many avenues to explore to get the answers we need that we can’t afford to have researchers working in silos, unaware of findings from other studies, duplicating efforts and going down dead-end paths.

What Happens to Clinical Trial Results Now?

Researchers in the United States who conduct randomized clinical trials are required under U.S. law to register their trials and report the results of their studies within 1 year on ClinicalTrials.gov. But this requirement is not strictly enforced and compliance is uneven. In fact, a recent study published in the medical journal BMJ found that two-thirds of clinical trials led by scientists at academic institutions didn’t share their results publicly within 2 years of the study’s completion.

As bad as that is, the problem goes beyond the failure to share the overall trial results. Clinical trial results are usually presented as averages. They may show, for example, how much longer a patient with advanced HER2-positive breast cancer, on average, went without a recurrence receiving the medicine being tested compared with standard treatment. But these averages may obscure wide variations in patient experience, and we need to understand what factors are driving the different experiences.

And that gets back to what Dr. Piccart said. Helping more patients is going to require that we customize treatment to the needs of the individual patient. To do that, researchers will need to be able to take the information from clinical trials broken down by patient (minus any information that would identify the person) and subject that to careful analysis.

Initiatives to Increase Sharing of Clinical Trial Data

Dr. Piccart spoke of her dream that “one day we will agree to put all these data [from clinical trials] in a single platform. After all, these data belong to our patients.” There are in fact some initiatives already underway that can help directly or indirectly to make this “dream”, as Dr. Piccart expressed it, a reality.

In 2015, the Institute of Medicine issued guiding principles and a framework for the responsible sharing of clinical trial data. One of the report’s key recommendations is that sponsors of clinical trials should share the data that supports a published study’s results within 6 months of publication.

This January, an international group of medical journal editors proposed that, as a condition of publication, authors share with others the de-identified patient data underlying their study no later than 6 months after publication.  The journal editors’ proposal included many elements of the Institute of Medicine framework, one of which was that authors must include a plan for data sharing in their clinical trial registration.

The group encouraged feedback on their proposal, which can be provided any time up until April 18, 2016 at http://www.icmje.org. Here’s a comment that I posted:

I’m a patient advocate who was diagnosed with breast cancer at age 35 with no family history of the disease. Those of us who have dealt with cancer often wonder why progress against this set of diseases is as slow as it is. It seems that one of the likely reasons is the limited collaboration that takes place among researchers today.

There really is no excuse for failure to cooperate when it comes to data from clinical trials. Patients are acting beyond only their own self-interest when they choose to participate in clinical trials. The data from trials really belongs to the patients who participate and by extension to us all.

Making the de-identified data from clinical trials available benefits society in many ways, including by enabling the results to be fully analyzed and understood, to allow for the conduct of further studies using the data, and to avoid duplication. There needs to be a mechanism to ensure the regular sharing of data from clinical trials. I support the proposed requirements as a way to help make that happen.

The AllTrials Initiative

Another initiative to be aware of and support is AllTrials. AllTrials is an international initiative that is calling for all clinical trials–past and present–to be registered and their full methods and summary results reported.

The AllTrials campaign is not calling for individual patient data to be made publicly available. But still, registering and summary reporting for all trials would be an important and necessary start toward shedding light on all of the research that has been and is being conducted with patients. And public sharing of results could lead to offline requests for appropriate sharing in a nonpublic way of individual de-identified patient data to facilitate new research work. The organizations’s website notes that there are now initiatives in many countries to work out how individual patient data can be shared with other researchers.

The AllTrials petition has been signed by over 87,000 individuals and more than 600 organizations. Take a moment and add your name to the petition.

Have you participated in a clinical trial?

Should de-identified patient data from clinical trials be shared with other researchers?

Related Posts
San Antonio Breast Cancer Symposium 2015: “These Data Belong to Our Patients”
Breast Cancer: How Much Progress Have We Made?
The I-SPY 2 Breast Cancer Trial: How Clinical Trials May Be Changing

Photo Credit: Scott Rothstein via Shutterstock

4 responses

  1. Lisa, once again, your blog is spot on. I signed the petition. Are you going to SF next month?

    • Thanks so much, Eunice. I appreciate your leaving me a comment. I won’t be there next month unfortunately, but hopefully will be able to make it next time.

  2. Yes! One of many things that could be done to improve the pace of research results. I also think we need to know about research ‘failures’ as well as successes. We can all learn a lot from what goes wrong, sometimes more from what doesn’t work that from what does. Thank you, Lisa. Have shared this. Kathi

    • That is a great point, Kathi. So much research is ‘wasted’ because the results were not what was expected or hoped for, and so the findings are never published. Thanks for your comments and for sharing this!

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