Obama’s Precision Medicine Initiative: Paying For Precision Drugs Is The Challenge
By David Kroll (Forbes) – Earlier this afternoon, I had read that in tonight’s State of the Union address, President Obama was to discuss the fruits of the Human Genome Project in the context of what’s become known as precision medicine.
Precision medicine has evolved from the concept of personalized medicine in that our focus in disease treatment and prevention is not semantically correct, i.e., focused solely on the individual. Instead, medicines are emerging that are targeted precisely to a subset of molecular problems shared by small groups within the larger population with a given disease.
Precision medicines are those directed toward a precise defect in the primary cause of a disease to either inhibit a malfunction or, even better, restore a normal function.
The goal of precision medicine is to give the treatment to the patients most likely to benefit because we now know the precise cause, at the genetic level, for this disease in a group of people.
Inherent in this definition is that the drug is not given to people with the disease who do not share the same precise defect, thereby avoiding waste of a drug on someone not only won’t benefit, but would only experience side effects.
The precision comes from advances in genetics and protein biochemistry that allow diseases to be identified and then broken down into subsets based on precise defects that cause the disease. But the other side of precision medicine is knowing how to develop drugs that work in these cases of precise defects.
Let’s take cystic fibrosis, for example. Sitting tonight with the First Lady was Stanford University graduate and Wright State University medical student, Bill Elder. The 27-year-old doctor-to-be was born with a disease we call cystic fibrosis. The disease results when a protein pump controlling chloride in organs like the lungs, pancreas, and intestines, doesn’t perform up to snuff.
In the lungs especially, the malfunctioning chloride pump causes thin secretions that protect the tissues to become thick and difficult to expel, allowing persistent bacterial infections and tissue damage where gas-exchanging lung tissue is replaced by non-functional, non-pliable fibrous tissue.
While cystic fibrosis is considered one disease, many molecular paths exist to get there. Bill Elder has one of those paths shared by 4% or 5% of the 70,000 cystic fibrosis patients worldwide. Scientists were able to identify the molecular change in chloride pump that causes it to malfunction, then start working on drugs that could restore its normal function.