Envisioning 2.0

Prologue: The Tale of the Over-Hyped Drug

Excitement is high around the latest potential blockbuster product from a major pharmaceutical company. Physicians and company spokespersons describe the new medication in breathless prose. “This drug has the potential to transform the way we treat this illness,” they proclaim. The firm fires up its PR engines and glowing stories about the medication begin to appear in medical and business press.

Despite the frenzy around the new product, a few suggest patience. They note that the company has only completed preliminary clinical studies and it will take a lot more testing before the full potential of the medication is realized. However, their warnings are ignored.

Then, disaster strikes. Phase III clinical trials reveal that certain patients taking the medication suffer from severe side effects. To make matters worse, the medication is not as efficacious as previously believed. The company’s stock price plummets and investors, patients and others demand answers from the suddenly beleaguered firm.

“The Tale of the Over-hyped Drug” is a familiar story to anyone following the pharmaceutical and biotech industries. The field is littered with the carcasses of once-promising medications that failed to meet expectations. Vanlev, Exanta and Iressa are just a few of the medications that have either under-performed or failed to win FDA approval.

Despite these object lessons pharma and biotech firms continue to hype unproven medications. One example is the much-heralded experimental hepatitis C drug, VX-950. In May, the Hepatitis C Association asserted that the medication “had the most powerful punch yet seen against this major cause of liver cancer.” (The New York Times published a report on the medication today.)

Some of the excitement around VX-950 is well-founded. Hepatitis C is a devastating disease. However, there are precious few effective treatment options for the illness. VX-950 appears to pack a powerful punch. When combined with interferon, a common therapy, VX-950 reduced blood levels of hepatitis C to undetectable levels, according to a press release issued today by Vertex Pharmaceuticals, makers of the drug.

However there is a catch to this exciting news. First, the Vertex study was a Phase II trial. Much larger Phase III studies will be required before the true effectiveness of the medication can be evaluated. In addition, the trial enrolled only 12 patients. While VX-950 demonstrated great efficacy in this group, it will be years before we know whether this treatment is what it seems.

According to the Times, the preliminary results of VX-950 clinical trials have not stopped Joshua Boger, the head of Vertex, from boasting about the drug. According to the Times: “Mr. Boger began his talk [at a J.P. Morgan health care conference last month] by showing a picture of the Apple iPod. ‘Every so often,’ he said, ‘there is a game-changing product — one that transforms a product category, one that transforms a company and one that transforms an industry.”

Strong words for a medication that one of Vertex’s own investigators is urging caution over. In a report by the Hepatitis C Association, Dr. Henk W. Reesink of Academic Medical Center in Amsterdam, Netherlands, warned that “nobody knows if [VX-950] will prove safe over time. Nobody knows whether its effects will continue long enough to cure hepatitis C virus infection.”

Given Vertex’s financial situation (it has lost nearly $1 billion over 16 years) it is not surprising that Boger is hyping the drug. VX-950 could reverse the firm’s fortunes. More importantly, VX-950 is a promising medication that has the potential to significantly improve the lives of those suffering from hepatitis C. Given this, I would hate to see this medication not live up to expectations.

I recommend caution. Under-promise and over-deliver. It’s not the most exciting of strategies, but in the volatile and unpredictable biotech industry, it’s a good one. I’ll be ready to believe the hype when the drug delivers. Until then, it’s wait and see for me.