The pharma industry’s gold standard- the large, multi-center, randomized, controlled clinical trial is failing.
About 95% of drugs entering the 4 phase clinical trial process fail. Many of these drugs are effective in small trials but fail in the large multi-center trials.
Something is wrong? Maybe the large trials select more of the wrong patients for the wrong drugs rather than the right patients for the right drugs.
Instead of randomly trying drugs on anyone with a specific disease, what is needed is a way of intelligently determining which patients will do well with any one target drug.
Individualized psychology and physiology are remarkable. Everyone is different. Everyone responds differently to drugs. This may account for the variability in the response to new drugs and the failure of the traditional large clinical trial.
On a genetic level, a patient with pancreatic cancer will not be identical to other patients with pancreatic cancer.
The solution seems to be to design “small” clinical trials and enroll only those who have the appropriate genetic or molecular signatures.
Genentech, the pharma company, did this with the breast cancer drug Herceptin. This drug targets tumour cells that have an abundance of the protein HER2.
Many new drugs are being produced with companion diagnostic agents. This is to identify the molecular signature of the patient’s disease. This allows for an intelligent matching of the right drug to the right patient. Success rates are likely to be much higher. This will be important for patients because there will be a larger selection of drugs with better clinical outcomes.
As with the Herceptin model, patients in new smaller clinical trials are being matched with experimental drugs that are designed to target a specific molecular sub-type of breast cancer. In these trials, researchers are testing up to a dozen drugs from multiple drug companies and are phasing out those drugs that don’t appear to be working and substituting others without stopping the clinical trial.
These new smaller clinical trials with the intelligent matching of the patient’s molecular signature with the new drug will prove to be a very important next step in helping patients with complex health problems.
Today, Rupert Case Management’s CANCER ASSIST program provides patients with the option of molecular profile testing to intelligently match the patient’s disease with effective new drugs. See the video on CANCER ASSIST at www.rupertcasemanagement.com under services.
( reference- New York Times, Sunday Review, page 1, July 14 2013)