“Physicians of all types need to be involved with (clinical trial research) because the biotechnology is advancing so rapidly right now,” said Neal Weinreb, MD, a leading genetics and lysosomal storage disease researcher located in Coral Springs, Florida.

Dr. Weinreb is referring to the fact that as we learn more about disease and their subtypes, it is becoming common for clinical trials to be more focused on small subsets of a disease to better target the therapy.

“The science of biotechnology reaching the point where it’s individualized and targeted therapies are probably going to be so rapidly produced that they’re going to change the way medicine is being practiced,” noted Dr, Weinreb, adding, “our whole approach to disease is going to be based on individual approaches and to some extent almost every disease then is going to become like a rare disease.”

And that future is happening now. “In terms of leukemia treatment, it’s now become standard practice to do the genomic profile of leukemic cells which of course has both prognostic implications but there may be a relatively small number of patients with acute myeloid leukemia for example who have a specific genomic profile for whom a drug could be life-saving, even curative,” stated Dr Weinreb.

Dr. Weinreb further stated that as we develop these more targeted therapies, we also have to rethink how we design clinical trials – including how we do the statistical analysis, what are the appropriate outcome measures, as well as how we approve these targeted medicines for rare diseases.