Day two of Global Gene’s 2017 Patient Advocacy Summit started out with a fire side chat with Christopher Austin, MD, the Director of the NIH’s National Center for Advancing Translational Sciences (NCATS).
Austin went to Harvard Med, and while we all know Harvard is the party school of the Ivy League, he worked hard and eventually made his way into clinical neurology. But it’s not just his hard work that makes him our ally…
His compassion for the patient experience and suffering shined through almost immediately, when told a heartbreaking story of his experience with an ALS patient who wanted to be unplugged from his ventilator. Dr. Austin’s job was to sit in a room with that patient for three hours, as the patient lost his life.
In that moment he thought “There has got to be a better way.”
He then found his way to the NIH under the National Human Genome Research Institute (NHGRI) after the completion of the Human Genome Project. It was here that he wanted to figure out how to come up with meaningful therapies from the results of this project. While here, he founded FOUR rare disease-focused initiatives, centers and projects. And when NCATS was born six years ago, he quickly became the director within the first year or two of it’s launch.
When he spoke, his passion for finding and developing medicines was more than apparent. But almost as equally impressive was his willingness eagerness to admit that our “street cred” as patients is critical in helping researchers connect the dots to creating those therapies.
Throughout his talk, his consideration and recognition for the patient experience was palpable. He has made himself so available to the rare community and the rare research community, that he casually directed the audience to “give [him] a call”.
Once we attended our break-out sessions, he spoke in the Drug Development Track on the subject of therapies getting from the lab to the clinic, faster.
He pointed out that over the last 60 years, there has been an overwhelming decline in productivity in therapy development. It hasn’t been positively effected by ethical changes, computer advancements, cat scans, MRIs, the Human Genome Project, gene therapy/editing, etc. as one might expect. If anything, all of these advancements have created more information, more “noise”, that may confuse the people and processes involved in developing treatments.
He shares our frustration in seeing this trend going the wrong way. He recognizes that officially, unless something changes, the cost of a new drug will be infinity in just a few decades. It’s already kind of infinity. Can you think of any other sector or industry that in over 60 years, the efficiency of making a product has gone down?
This is the very problem that NCATs was started to solve, and something he strives to fix. He admitted that these are complex problems that won’t simply be solved by optimization consultants for process improvements… we need exponential improvements. We need completely different ways of thinking about this process. He reiterated: NCATs is all about new ways of doing things to get more treatments to patients, more quickly.
In the end of both of his talks, which were filled with refreshing self-deprecating humor and incredible self-awareness, we were left with this question:
How do we take the potential for treatment and make it “real” for patients?
