“An Experimental Drug’s Bitter End (Business Day, June 7) is but one example of many of what is wrong with the drug industry’s research and development model.
Clinical trials include hundreds of patients with a disease that may manifest differently in each. While sheer participant number provides statistical strength, it hides the positive effects of drugs on some patients.
We are left with a sad situation: patients who had a successful response lose access to that drug because it fails to work in most. This will continue unless we can account for subtle differences among patients.
Now, we can derive stem cells from patients’ skin cells and test drugs on the actual cells affected by a disease before clinical trials.
We can see what is likely to benefit different patient subgroups, to finally address the needs of all, which will mean that a drug that is effective for some people but not for most will not have to be denied to those it can help.
That is why the New York Stem Cell Foundation, a nonprofit organization, built a system to do exactly this: the New York Stem Cell Foundation Global Stem Cell Array, which contains stem cells representing the full range of human genetics, allowing us safe and rapid tests of an unlimited number of potential drugs.”
–Suasan L. Solomon, Chief Executive, New York Stem Cell Foundation, New York, June 7, 2013
“The fact that some medicines help only a small subset of patients is one of the central premises behind personalized medicine. When that subset is too small for the medicine to be commercially viable, we shouldn’t turn our backs on that problem; we should fix it.
There are laws in place to encourage the development and marketing of medicines for rare diseases, the accommodations justified by the commercial hurdles associated with producing treatments for small populations.
While there will be cost of production and intellectual property hurdles, it may be possible to empower an industry to arise for the purpose of manufacturing even smaller batch medicines.
The bottom line is that in some way policy makers should address situations in which clinical trials affirm the safety and effectiveness of a treatment for small subpopulations of those with a given disease.
People who agree to participate in a clinical trial— or loved ones placed in the position of making that decision for them— should not help prove that there is hope, only to have it dashed.
If our country goes down the path of personalized medicine, we must adjust our laws and regulations to do so in an ethical manner.
–Eleanor Dehoney, Vice President of Policy and Advocacy, Research!America, Virgina, June 7, 2013
Read more at the New York Times.