Ed. note: This morning at 8:15 EDT, Isaac Kohane, MD, PhD, will tell the audience at TEDMED 2013 about his goal of using every clinical visit to advance medical science.
To preview his talk, we’ve updated a past Vector story about SHRINE, a system Kohane helped develop to allow scientists to use clinical data from multiple hospitals for research.
Clinical research really comes down to a numbers game. And those numbers can be the bane of the clinical researcher. If there aren’t enough patients in a study, its results could be statistically meaningless. But getting enough patients for a study, particularly for rare diseases, can be a daunting challenge.
We’re pretty focused on the safety of the things around us. Our drinking water gets checked for chemicals, bacteria and other things that could make us sick. Kids’ car seats are tested to make sure they’ll keep children safe in an accident.
But there’s one surprising arena where this focus on safety and testing often falls short: the medications we give our children. Not just in the United States, but globally.
There are lots of reasons why fewer drugs get tested for safety and efficacy in children than in adults. It’s time-consuming, expensive and, frankly, risky. The ethics of testing new medications in children are pretty thorny.
And, overall, the market for pediatric drugs is much, much smaller than that for drugs for adults, since children fortunately don’t get sick as often as us grown-ups.
But for some diseases like asthma and diarrheal diseases, children bear a greater burden than adults—one that’s not matched by the amount of research done on drugs for kids. …
A few months ago, my mother had a skin cancer removed from her nose. To the best of our knowledge, the tumor tissue was discarded in a biohazard bag, never to be seen again.
Often, though, bits of tumor like that removed from Mom become the raw material that keeps the engine of research running. And every once in a while, a patient’s tissues lead to a real blockbuster, like a cell line or a protein with uniquely valuable biological properties or a lucrative drug that benefits millions of people.
Which brings us (literally) to the $64,000 question: Should the patient who donated those tissues reap some of the rewards? …
When you get down to it, science, particularly in the clinical realm, is something of a numbers game. An experiment or study’s weight depends greatly on its size (how many patients took part, how many times the experiment was repeated, etc.). For any number of reasons, though, researchers may only be able to bring a few people into a study and collect limited data, restricting both the answers it can provide and the impact of those answers on the field. Such has been the case with autism, for example, where studies tend to be small and patient populations haven’t always been well defined.
But what if one could compare apples to oranges – or, at least, Golden Delicious to Cortlands – by creating one large “uberstudy,” merging the results of many small studies in ways that would allow comparisons among them to generate some level of consensus about a treatment or discovery? …