What risk would you take with your child’s life? What if the treatment had a 25% chance of death or serious injury, would you risk it?
The willingness of patients and caregivers to accept a level of risk when enrolling in a clinical trial or otherwise taking an investigational drug is different for every patient or family. However, there are some common factors that can be identified to create a model for how patients and caregivers often assess their risk tolerance.
I’ve developed a model for analyzing risk tolerance based on my discussions with families with different rare diseases and whose children are in different stages of those rare diseases. Hopefully this model, or something like it, could be useful by the FDA or others in analyzing the risk tolerance of patients and caregivers in enrolling in clinical trials, accessing investigational drugs via compassionate access (individual INDs or expanded access programs), or approving drugs for rare diseases.
For a disease that dramatically shortens a child’s life span, significantly impacts their quality of life, and for whom the continuation and perhaps increased intensity of these effects is fairly certain, when the current disease burden is significant, and when the child has progressed such in the disease that their lifespan is dwindling, the tolerance of risk in using an investigational drug is substantial.
Note that certain factors are disease specific, based on diagnosis alone, but they are affected and modified by the current effects and course of the disease on a particular patient.
What do you think? What would you add? What factors affect the risks you’d take to save your child?