A Better Way To Bring Medicines To Market

  • by: |
  • 05/22/2014
Recently I was honored to co-author a letter  to the editor of JAMA with Eric Topol, MD and Stuart Kauffman, PhD,MD in response to an article calling for more randomized trials.   The letter was to have been an article and had other co-authors, but JAMA requested a response which by their editorial guidelines understandably limits co-authorship.    Here is the response to the article (below) and a link to the article itself.   

Study Design and the Drug Development Process
To the Editor

A Viewpoint by Dr Djulbegovic and colleagues1 claimed not only that randomized clinical trials (RCTs) aremore ethical but that greater use of randomized designs throughout the drug development process would “improve the efficiency, ie, enable faster development ofnew, successful treatments.”  However,RCTs are outdated for several reasons. First,RCTs are inadequate to evaluate cancer therapies. Genomic analysis is uncovering the tremendous heterogeneity of what previously were considered single diseases. Genomic analysis of cancers of individual patients is disclosing the large number of mutations, and thus targets, within one person. Developing RCTs for targeted therapies would be difficult and timeconsuming, prolonging the wait for effective treatments.
Second, RCTs are a less efficient and accurate method of establishing which treatments work.

A recent study demonstrated that RCTs fail to predict or improve outcomes when evaluating multicausal diseasenetworks or treatments orwhen assessing which particular interactions are relevant.2 Rather than increase the use of RCTs, we propose an increase in the use of N-of-1 studies, which are based on simulations that rapidly sort through billions of possible interactions
at the clinical, genomic, and biological levels to arrive at predictive models of multicausality. This approach has identified candidate markers, which have been successfully used in clinical trials.3 These studies also have predicted the most effective treatments for patients based on a particular genotype
and medical history when RCTs have not.4 Increasing the use of RCTs would add to the cost and time required to developand use new products. Other analytic methods more quickly and precisely match patients to treatment.

1. Djulbegovic B, Hozo I, Ioannidis JPA. Improving the drug development
process: more not less randomized trials.JAMA. 2014;311(4):355-356.
2. Eppstein MJ, Horbar JD, Buzas JS, Kauffman SA. Searching the clinical fitness
landscape.PLoS One. 2012;7(11):e49901.
3. Wu C-C, D’Argenio D, Asgharzadeh S, Triche T. TARGETgene: a tool for
identification of potential therapeutic targets in cancer.PLoS One.
4. Garnett MJ, Edelman EJ, Heidorn SJ, et al. Systematic identification of
genomic markers of drug sensitivity in cancer cells.Nature.

Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

Blog Roll

Alliance for Patient Access Alternative Health Practice
Better Health
Biotech Blog
CA Medicine man
Cafe Pharma
Campaign for Modern Medicines
Carlat Psychiatry Blog
Clinical Psychology and Psychiatry: A Closer Look
Conservative's Forum
Club For Growth
Diabetes Mine
Disruptive Women
Doctors For Patient Care
Dr. Gov
Drug Channels
DTC Perspectives
Envisioning 2.0
FDA Law Blog
Fierce Pharma
Fresh Air Fund
Furious Seasons
Gel Health News
Hands Off My Health
Health Business Blog
Health Care BS
Health Care for All
Healthy Skepticism
Hooked: Ethics, Medicine, and Pharma
Hugh Hewitt
In the Pipeline
In Vivo
Internet Drug News
Jaz'd Healthcare
Jaz'd Pharmaceutical Industry
Jim Edwards' NRx
Kaus Files
Laffer Health Care Report
Little Green Footballs
Med Buzz
Media Research Center
More than Medicine
National Review
Neuroethics & Law
Nurses For Reform
Nurses For Reform Blog
Opinion Journal
Orange Book
Peter Rost
Pharm Aid
Pharma Blog Review
Pharma Blogsphere
Pharma Marketing Blog
Pharmacology Corner
Pharmaceutical Business Review
Piper Report
Prescription for a Cure
Public Plan Facts
Real Clear Politics
Shark Report
Shearlings Got Plowed
Taking Back America
Terra Sigillata
The Cycle
The Catalyst
The Lonely Conservative
Town Hall
Washington Monthly
World of DTC Marketing
WSJ Health Blog