University of Antioquia | Leaders in Pharmaceutical Business Intelligence (LPBI) Group

Posts Tagged ‘University of Antioquia’

Reboot evidence-based medicine and reconsider the randomized, placebo-controlled clinical trial

Posted in Computational Biology/Systems and Bioinformatics, FDA Regulatory Affairs, Genome Biology, Health Economics and Outcomes Research, Health Law & Patient Safety, HealthCare IT, Medical Devices R&D Investment, Personalized and Precision Medicine & Genomic Research, Proteomics, Regulated Clinical Trials: Design, Methods, Components and IRB related issues, Statistical Methods for Research Evaluation, tagged BRAF, Clinical trial, Editor-in-chief, Eric Topol, evidence based medicine, Melanoma, National Institutes of Health, University of Antioquia on November 4, 2012| 5 Comments »

Larry H Bernstein, MD, FACP, Reporter

Clinical Trials
Dr. Eric Topol, Director of the Scripps Translational Science Institute and Editor-in-Chief of Medscape Genomic Medicine and the heart.org.
In our series The Creative Destruction of Medicine, I’m trying to get into critical aspects of how we can Schumpeter or reboot the future of healthcare by leveraging the big innovations that are occurring in the digital world, including digital medicine.

But one of the things that has been missed along the way is that how we do clinical research will be radically affected as well. We have this big thing about evidence-based medicine and, of course, the sanctimonious randomized, placebo-controlled clinical trial. Well, that’s great if one can do that, but often we’re talking about needing thousands, if not tens of thousands, of patients for these types of clinical trials. And things are changing so fast with respect to medicine and, for example, genomically guided interventions that it’s going to become increasingly difficult to justify these very large clinical trials.

For example, there was a drug trial for melanoma and the mutation of BRAF, which is the gene that is found in about 60% of people with malignant melanoma. When that trial was done, there was a placebo control, and there was a big ethical charge asking whether it is justifiable to have a body count. This was a matched drug for the biology underpinning metastatic melanoma, which is essentially a fatal condition within 1 year, and researchers were giving some individuals a placebo.

Would we even do that kind of trial in the future when we now have such elegant matching of the biological defect and the specific drug intervention? A remarkable example of a trial of the future was announced in May.[1] For this trial, the National Institutes of Health is working with [Banner Alzheimer’s Institute] in Arizona, the University of Antioquia in Colombia, and Genentech to have a specific mutation studied in a large extended family living in the country of Colombia in South America. There is a family of 8000 individuals who have the so-called Paisa mutation, a presenilin gene mutation, which results in every member of this family developing dementia in their 40s.

Clinical Trials (journal) (Photo credit: Wikipedia)

Consumer-Driven Healthcare Is an Uncomfortable Concept per Eric J. Topol, MD (pharmaceuticalintelligence.com)
Eric Topol: Information into Action at Wired Health (fora.tv)
http://pharmaceuticalintelligence.com/2012/10/18/modernizing-drug-and-device
-approvals-speeding-the-process/
Qualcomm Foundation Awards Scripps Health $3.75M for Digital Health (xconomy.com)
Scripps Physician-Scientist Dr. Eric Topol Outlines Top Five Areas for Genomic Medicine Advances in 2011 (prweb.com)

Read Full Post »

Get Rid of the Randomized Trial; Here’s a Better Way

Posted in Alzheimer's Disease, FDA Regulatory Affairs, Regulated Clinical Trials: Design, Methods, Components and IRB related issues, Statistical Methods for Research Evaluation, tagged Alzheimers Disease, BRAF, Clinical trial, Eric Topol, National Institutes of Health, University of Antioquia on August 23, 2012| 13 Comments »

Reporter: Aviva Lev-Ari, PhD, RN

Eric Topol: Get Rid of the Randomized Trial; Here’s a Better Way

Eric J. Topol, MD

Posted: 08/20/2012

WATCH VIDEO

768635?src=ptalk

Hi. I’m Dr. Eric Topol, Director of the Scripps Translational Science Institute and Editor-in-Chief of Medscape Genomic Medicine and theheart.org. In our series The Creative Destruction of Medicine, I’m trying to get into critical aspects of how we can Schumpeter or reboot the future of healthcare by leveraging the big innovations that are occurring in the digital world, including digital medicine.

For example, there was a drug trial for melanoma and the mutation of BRAF, which is the gene that is found in about 60% of people with malignant melanoma. When that trial was done, there was a placebo control, and there was a big ethical charge asking whether it is justifiable to have a body count. This was a matched drug for the biology underpinning metastatic melanoma, which is essentially a fatal condition within 1 year, and researchers were giving some individuals a placebo.

Would we even do that kind of trial in the future when we now have such elegant matching of the biological defect and the specific drug intervention? A remarkable example of a trial of the future was announced in May.^[1] For this trial, the National Institutes of Health is working with [Banner Alzheimer’s Institute] in Arizona, the University of Antioquia in Colombia, and Genentech to have a specific mutation studied in a large extended family living in the country of Colombia in South America. There is a family of 8000 individuals who have the so-called Paisa mutation, a presenilin gene mutation, which results in every member of this family developing dementia in their 40s.

Researchers will be testing a drug that binds amyloid, a monoclonal antibody, in just [300]^[1] family members. They’re not following these patients out to the point of where they get dementia. Instead, they are using surrogate markers to see whether or not the process of developing Alzheimer’s can be blocked using this drug. This is an exciting way in which we can study treatments that can potentially prevent Alzheimer’s in a very well-demarcated, very restricted population with a genetic defect, and then branch out to a much broader population of people who are at risk for Alzheimer’s. These are the types of trials of the future and, in fact, it would be great if we could get rid of the randomization and the placebo-controlled era going forward.

One of things that I’ve been trying to push is that we need a different position at the FDA. Now, we can find great efficacy, but the problem is that establishing safety often also requires thousands, or tens of thousands, of patients. That is not going to happen in the contrived clinical trial world. We need to get to the real world and into this digital world where we would have electronic surveillance of every single patient who is admitted and enrolled in a trial. Why can’t we do that? Why can’t we have conditional approval for a new drug or device or even a diagnostic test, and then monitor that very carefully. Then we can grant, if the data are supported, final approval.

I hope that we can finally get an innovative spirit, a whole new way of a conditional and then final approval in phases in the real world, rather than continuing in this contrived clinical trial environment. These are some things that can change in the rebooting or in the creative destruction, or reconstruction, of medicine going forward.

Thanks so much for your attention and your continued support of The Creative Destruction of Medicine series on Medscape.