What We Learn From Clinical Trials

What We Learn From Clinical Trials

04.04.13 | By Stephanie Fischer

A recent op-ed in Forbes offers a welcome rebuttal to misguided criticism of the conduct of clinical trials by the biopharmaceutical industry by refuting the notion that "medicine is broken" partly due to the hiding of "negative" clinical trial results. First, it explains how even clinical trials that do not validate the premise that they were intended to evaluate are not necessarily "negative": Clinical studies that yield unexpected or unwanted results are seldom negative in the sense of revealing actual net harm to patients from the drug or medical device being tested; far more often and for a variety of reasons, a "negative" study simply fails to show that it's effective for certain indications, or uses. The author, Dr. Henry Miller, would know. He spent 15 years at the Food and Drug Administration (FDA) as a medical reviewer and founder of the Office of Biotechnology. He explains that potential new medicines are often tested in parallel for different indications such as for different types of cancer or infections. Clinical trials may fail for various reasons such as having too few participants to be statistically significant or focusing on an inappropriate route, dose or frequency of administration.

Learning from Setbacks

The drug discovery process is long and difficult, costing more than $1 billion over 10-15 years. Only one out of 10,000 potential new medicines makes it through pre-clinical and clinical trials and is approved by the FDA to be used by patients. Fortunately, we learn from the many failures as well as the rare successes. Dr. Miller explains how even unsuccessful or inconclusive clinical trials can contribute to science: If the methodology and execution are sound, even data from unsuccessful studies can provide important leads - a reminder that the nuances of clinical trials are complex and that understanding their limitations is essential to our discovering which therapies work and which do not.

Sharing Data from Successful -and Unsuccessful- Trials

The column dismisses criticism of a lack of transparency in clinical trials by explaining how the clinical trials of any potential medicines that would seek FDA approval are registered and the results - positive or negative - shared with regulators. Clinical trials are also registered on a public database maintained by the National Institutes of Health (NIH). ClinicalTrials.gov currently has information on more than 140,000 clinical trials in all 50 states and 183 countries. The NIH reports that the website receives more than 95 million page views per month and more than 60,000 unique visitors daily. In addition, PhRMA member companies have voluntarily committed to share summary results of all clinical trials in patients for discontinued research programs. The biopharmaceutical research sector is proud to be part of a complex clinical trial ecosystem that develops and evaluates potential new medicines in order to make safe and effective new therapies available to the patients who need them. In addition to learning from individual setbacks, the industry continues to look for ways to improve the clinical trials process in order to speed the delivery of safe and effective new medicines to patients, and to encourage innovation.

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