Patient Worthy attended the NIH‘s National Center for Advancing Translational Sciences NCATS meeting and in a word: WOW!
Start with the patient– the end user, the source of the aberrant biology and chemistry, the one for whom the stakes are greatest: what a novel idea! As a clinician, let the patient define what a significant treatment is, what risks they are willing to take, what elements of a complex disease are most troubling.
In all of the meetings, conferences and symposiums that this reporter has attended, never has the patient and the patient advocate had such a voice.
Throughout the day, the researchers, physicians, staff and the fellows, all listened to those who are living with rare diseases. And on a Friday before a major holiday weekend!
Consistently, NCATS Director, Dr. Chris Austin, reminded all of the NCATS staff to listen, not talk.
According to NCAT’s Strategic Plan, there is more than a willingness, to collaborate with patients; there is an actionable plan which includes:
- Pre-clinical innovation such as the Tissue Chip Initiative Project
- Recognition that there are social, regulatory and legal roadblocks as much as there are scientific discovery delays, and that new approaches are needed i.e. Fair Data principals in Force 11 which specifies that data should be findable, accessible, interoperable and re-usable.
- Collaboration, even with intellectual property licensing, so that commercialization can put medicine where it can work.
- The Trnds and BrlDGs programs to define meaningful endpoints, understand natural history, recruit clinical trial participants faster and use collaborative means to raise research funds.
- NTU – New Therapeutic Uses Program– innovative ideas to use current, known assets for new uses
- Biomedical Data Translation– more use of patient registries, better documentation of clinical symptoms, access to samples, and the sharing of data between diseases which have similar biology
- GARD- Genetics and Rare Diseases Information Center and the Rare disease Clinical research Network Program
What I heard from NCATS is: Let’s use technology, novel partnerships between industry and scientists, new models for gathering patient data faster and smarter, and a bit more flexibility and imagination to make progress in our lifetime, and let the needs of the patients be central to this process.
Where to start: NCATS has developed a toolkit for Patient-Focused Therapy Development. This toolkit will serve to guide patient groups through the process of working with NCATS.
If you want to learn about this toolkit in person, a demonstration will be given on Friday, Sept 8th at the Natcher Conference Center at NIH. Click here for more information.
Collaboration: Collaboration is key for progress. A case study and hopefully just one model of collaboration was presented on The Alpha-1 Project, Inc., a for-profit, collaborative effort. While funded by the Alpha-1 Foundation, it utilized a post doc dedicated fellow working with access to the resources of the NIH, to discover and hopefully bring to market, an effective treatment for Alpha- 1.
Pre-clinical innovation: From listening to a presentation on the Tissue Chip Project, I gathered it is for testing the effect of potential compounds on human tissue, without the risks entailed in Phase I clinical trials. These chips may also be combined to see how a substance might work in a human system. Find more information, click here.
These developments and presentations were informative and exciting! And they reaffirmed my hope for progress in the rare disease realm.
“The most difficult thing is the decision to act, the rest is merely tenacity. The fears are paper tigers. You can do anything you decide to do. You can act to change and control your life; and the procedure, the process is its own reward.” – Amelia Earhart
