Luke Timmerman writes about one of the big changes that is takig place in the development of clinical trials:
Amgen had an epiphany of sorts a little more than four years ago. The world’s largest biotech company decided clinical trials had become too much of a crapshoot, and it needed to shake up its way of developing new drugs. This really isn’t unique to Amgen, it’s more like an industry emergency. Drug companies spent $44.5 billion on research and development last year, yet last year they managed to produce the fewest number of new medicines in 24 years, a big reason why companies are scrambling for a better way.
One of Amgen’s strategies is to gather samples from patients in early-stage clinical trials, and study them for biomarkers, or signature proteins, that can offer clues about whether the drug’s working (or not). Amgen now has a team of 240 people doing work in this area, which it calls medical sciences. Company scientists in Seattle with expertise in clinical immunology and computational biology are teaming up with molecular biology experts in Cambridge, MA, and people with other skills in the San Francisco Bay Area, as well as at headquarters in Thousand Oaks, CA.
This is a beginning of personalized medicine. The companies will get a much better idea of why and how some drugs work for some people. This has sometimes been done but usually AFTER a trial is complete, as the company tries to find some sub-population the drug works in. This sounds much more directed.
Their job is to run a gauntlet of questions about drugs in the early phases of development, says Scott Patterson, Amgen’s executive director of medical sciences. Is the drug hitting its intended target on human cells? What affect is it really having on the biological pathway envisioned in the lab? Can the samples offer insight into the best possible dose? Perhaps most importantly, can the samples show Amgen how to stratify patients based on who’s likely to benefit from the drug, and who isn’t?
“This gives us greater confidence in our drugs as they move through the pipeline,” Patterson says. “You’ve got to have confidence to make the investments to take them through development.”
Now Amgen is doing this because they sort of dug themselves into a deep hole when they bought Abgenix to acquire a drug for colorectal cancer.
Amgen paid a fortune—$2.2 billion—in 2006 to acquire Abgenix to get full control over Vectibix. Yet the drug has not emerged as the kind of $2 billion-a-year commercial success originally predicted by analysts. That’s partly because a study in March 2007 was halted when it showed Vectibix raised the risk of death in a clinical trial when used in tandem with Genentech’s Avastin, another colorectal cancer drug that works differently.
But they went back to the samples and found out that a particular gene allele may be responsible for their failure.
About 17 percent of patients with normal KRAS genes had tumors shrink after they took the drug, while none of those with mutated KRAS genes saw that benefit, according to research published in March in the Journal of Clinical Oncology. About 43 percent of patients have the mutated KRAS, and suddenly looked like they should avoid the drug, which is high-priced, and causes a significant rash as a side effect.
So, ignore those with the ‘wrong’ allele and your clinical trial will look much better. You can more accurately gauge the market for the drug and, hopefully, run cheaper trials.
Now Luke does something you can expect in blogs but not often in ‘real’ journalism articles. He adds some perspective and context by providing a personal viewpoint. I really like this.*
What does it mean for Amgen as a company? Like a lot of people, I wonder if the company is really keen on finding out when its drug doesn’t work for more than 40 percent of patients, and that it therefore shouldn’t be given to them. It’s possible that by narrowing the pool of customers, but raising the overall odds of success among those who do take it, Amgen could sell even more of the drug. After all, Genentech’s breast cancer treatment, Herceptin, which is approved for about one-fourth of breast cancer patients with a certain gene mutation, still generated $1.3 billion in U.S. sales last year.
To which I respond, if I were a company, I would love this. Not only do I have a much better much better medical case for doctors to use MY company’s drug but if I can find a drug that works with those 40%, I now has TWO drugs to offer the doctor for two different populations. Better medicine and better commercial products. Done with cheaper clinical trials. What’s not to like?
Many times a clinical trial has to enroll a large number of people in order to see real efficacy. Here, I could do the trial with 40% less people the first time than in the ‘old’ days and then I could do a focussed trial on just the other 40%.
At the end of the day, I would have 2 drugs that cover the entire population for the same cost as a trial that only gets me one drug that covers 60%. WHich would you rather have?
*The reason this is so great is that it is how conversations are started. I could not have been a part of this conversation as easily with a normal newspaper article. The style is just a monolog. But by adding his own perspective, Luke invites me to add mine. Cool.