The Patient Equation

8Castleman Disease—Not One Rare Disease with No Treatments, But Three Rare Diseases…with Hope, Thanks to Data

Let's talk about the typical model of drug development. Life sciences companies are normally looking for the blockbuster, a treatment for the masses that can cover as many patients as possible. Of course effectiveness is important, but we only need to be more effective than the current treatment in order to go to market, and if we can potentially meet the needs of millions, even if not absolutely everyone who tries the drug will wind up benefitting…well, that's just the reality of drug development.

Precision medicine turns that paradigm on its head. As we're able to get more precise with treatments, the number of patients for whom those treatments are relevant will necessarily decrease. This creates a huge shift in the kinds of business decisions a life sciences company will choose to make. It's a different goal, about taking all of the information we can generate and activating it to be something that really makes a difference for each and every patient.

We saw that in the previous chapter with immunotherapy for cancer and phage therapy for bacterial infections, and it's just as salient a point here, with Castleman disease. To go from treating a rare disease with one relatively low-effectiveness treatment to figuring out that the condition may actually be three distinct diseases, each of which may need a different treatment…we're talking about affecting lives deeply, ...