Roll the Dice: Playing the Clinical Trial Lottery

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Clinical Trials are key stepping stones in the drug development process. Companies spend billions of dollars on drug testing each year – and yet, for a surprising number of medicines, we still don’t know if they’re really safe or effective.

A recent New York Times article probes the question, “Do Clinical Trials Work?” In the article, Avastin, a drug being developed as a treatment for an aggressive form of brain cancer, was introduced as a hot topic at this year’s American Society of Clinical Oncology meeting. The drug, created by Genentech, a division of the pharmaceutical giant Roche, was heavily tested by Dr. Mark R. Gilbert in a study involving 600 patients. In the study, half of the participants were given placebo drugs, while the other half were given Avastin. Unfortunately, there was no difference in survival found between those who took the Avastin pill and those who took the placebo pill.

Hopes were high for Phase 3 of drug development, because in earlier, smaller studies by Dr. Gilbert, some participants saw shrinkage in the size of their tumors. Although hopes were squashed during Phase 3, doctors are still excited by a small victory gained from the study. Doctors have now recognized that using a more controlled, randomized study is a smarter way to approach drug development studies. Prior to this study, no comparison groups had been used. Using comparison groups, combined with the identification of key target populations for studies have been identified as critical for success in each of the phases of clinical trials.

Best Practices’ backed up this theory in a benchmarking report, “Success Factors and Failure Points in Oncology Product Launches”. The report explains that it is absolutely essential to clearly define the target patient population when developing a drug. A clearly defined target patient population or subpopulation has a high impact on successful market entry & potential product differentiation. In order to accurately identify patient populations, respondents ranked identifying patient diagnostic type by name or segment as highly effective. Identifying patient function and emotional needs was also ranked as a highly effective way to identify patients that are well suited for therapy.

The high failure rate in drug development is likely due to the fact that developers often miss variation in how different people – with different genotypes— respond differently to drugs. This ultimately creates problems for the FDA, Pharma, payers and patients. Accurately defining key target publics can lead to a more successful drug development process. While the question, “do clinical trials really work?” still has no definite answer, Pharma companies can ultimately benefit from a strategic and well-planned drug development process.

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