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Safety Monitoring Boards and Clinical Trials


A good example of when no news is simply no news

When a new drug is tested on human subjects, safety is the number one concern. During unblinded trials, the investigators (doctors) dosing the drug and the company are capable of monitoring the safety of the drug. During blinded, randomized trials, however, neither the investigators nor the company knows who is receiving the drug.

Data Safety Monitoring Boards (DSMBs, also called Independent Data Review Boards) are used to ensure safety of patients in blinded clinical trials. A DSMB is comprised of doctors who are usually not investigators and rarely have any connection to the company. Their job is to examine the unblinded data for any trends suggesting safety problems.

Occasionally, you'll see a press release from a biotech company touting the fact the DSMB has not indicated there are any safety problems. In my experience, any company who does this trends to the over-promotional side and/or is too desperate for good news. By the time a drug gets to randomized trials, it is very unusual for the DSMB to note any problem. If problems do crop up, then someone in management almost always screwed up.

DSMBs also have the power to halt a study for good reasons. If the data coming from the trial is so obviously good that it is unethical not to give the drug to the other arm in the randomized trial, the DSMB is authorized to halt the trial. Before you get your hopes up, this happens very rarely - likely in less than 1% of all randomized trials. The medical condition being treated has to be very severe, the quality of the treatment in the arm of the trial not receiving the study drug has to be poor, and the study drug has to work in a very, very large percentage of patients who receive it in order for the DSMB to halt the trial. Each is an unusual circumstance in itself, but having the three together in one trial is very unusual.

The reason I bring this up today is Titan Pharmaceuticals (TTP:AMEX) announced their DSMB halted a randomized Phase IIb trial of the cancer drug Pivanex for unexpected toxicity. We have some familiarity with Pivanex as we used to follow Titan until we uncovered the fact that the CEO and CFO entered into a financial arrangement that allowed them to profit from a steep decline in Titan's stock price. In Titan's release this morning, they said the DSMB had recommended additional dosing of Pivanex in combination with the chemotherapy drug Taxotere be halted until the cause behind the side effects is uncovered.

This is really one of the worst-case scenarios in biotechnology. Even if it turns out the side effects seen were bad luck (like a series of people with heart trouble ending up on the study arm and dying prematurely), the trial almost always has to be done over again because the blinded aspect has to be broken to determine the root cause of the side effects. Furthermore, even if the cause of the side effects are determined to not be associated with the study drug, the drug has a "stain" on it that will cause a high degree of skepticism among doctors, patients, Wall Street, and especially the FDA.

Most of the time, DSMBs are no factor in clinical trials. If you are investing with the hope a DSMB will halt a trial early, you are likely going to be burned. A DSMB halting a trial on good data is extremely rare. A DSMB halting a trial for safety issues is not quite as rare, but still rare enough to make an investment based upon such an event to be a long shot.

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