The Next Breakthrough

By Rob Wright, Chief Editor, Life Science Leader
Follow Me On Twitter @RfwrightLSL

By  Rob Wright

I recently read a story about Biogen Idec and Abbott Labs releasing information about a Phase 2b study of daclizumab, touting some promising results for the once-monthly treatment of multiple sclerosis (MS). Apparently, the 150-mg dose arm demonstrated a 54% reduction in the annualized relapse rate and "highly statistically significant" data recorded on secondary endpoints. Perhaps you are wondering what this means. Are we possibly seeing the next breakthrough?

Stats 101
MS is a potentially debilitating disease in which your body’s immune system eats away at the protective sheath that covers your nerves. So if we do have a breakthrough for these patients it would certainly be great news. But, let me explain a little bit about statistics so we don’t get overly excited and start day trading the above mentioned company stocks. For those of you who are not fluent in stats, here is the cliff notes version. “Significant” in stats means probably true — as in not due to chance. It is worth noting that a research finding may be true without being important. So, when statisticians say a result is "highly significant" they mean it is very probably true, not necessarily highly important. I guess we have to take their word that the above announcement is not only true, but important, since the companies decided to release the information. I recall a comment from one of my stats professors which may put this in perspective — “If you torture the data long enough, it will confess.” Which makes me wonder, with the market bouncing around like a super ball tossed in a crowded shopping mall, how much of the bravado behind these types of announcements is catering to the analysts? The end of Q3 is rapidly approaching.

Breakthroughs Not Blockbusters
At the R&D Leadership Summit this past year, Fred Hassan warned about the problems when leaders manage to analysts’ expectations and not their commonsense. Perhaps this is why the life sciences industry went through the blockbuster phase. Now the buzz is all around breakthroughs. I have had several executives use this word with me when describing their approach to drug development. Bernard Munos, who I interviewed for an article in the May issue of Life Science Leader, “Three Keys to Innovation,”  believes the real innovators are people who see things other people don’t. Munos was interviewed in the August issue of Forbes on the same subject. To be sure, Forbes has a larger circulation than Life Science Leader. Perhaps now we will really see a tipping point regarding his message of breakthroughs versus blockbusters and transformational change in drug discovery.

Seeing Things Differently
So, who will make the first breakthrough? This remains to be seen. I am placing my money on whoever the Gates Foundation decides to back. Melinda Gates seems to see things differently and has been instrumental in revolutionizing drug discovery through the foundation’s “Grand Challenges.” The initiative uses an agile, accelerated grant-making process. The process is open to anyone from any discipline. Simply fill out a short two-page application online, no preliminary data required. Winning grants are chosen approximately four months from the submission deadline. Initial grants of $100,000 are awarded two times a year. Successful projects have the opportunity to receive a follow-on grant of up to $1 million. What can Big Pharma learn from this? Look outside your own four walls for innovation and be willing to put a little money behind it to see where it leads. Most importantly, simplify the process.