Throughout the week of August 7th, I have visits lining up in D.C. with top policymakers and regulators, politicians, stem cell industry leaders, researchers and CBER officials.
I admit, I’m not your typical patient advocate. Accordingly, I’ve begun shedding that term for one I believe is more accurate - ambassador.
If you are a cell therapy provider (especially if you’re considering an FDA RMAT clinical trial), listen up. If you are a patient, I’m all about you. I’m still a patient myself. Please message me personally any way you’d like. Tell me your story, what you need. I’ll pass it on to the right people if I can.
The continued dialog and information-sharing I have enjoyed with top-level officials over the last couple of years has been a true privilege, and quite humbling. The interchange is based upon respect, appropriate boundaries, and what I believe to be the expression of mutual hope for all of us and the people we love, who will all become patients one day.
Such access is impressive, but, aside from that, for me what has always been most exciting about those conversations is that they are so genuine, mutually helpful, and meaningful. My dream and passion as a patient who has had a remarkable outcome from cell therapy is to bring people together solidly in the system that has now been created with legislation and the FDA's new reforms and initiatives.
To accomplish that, I have established a non-profit which will help bring patients into FDA RMAT trials by providing direct grants for ancillary expenses associated with participation. It will also support providers by incentivizing them to train themselves in ethics, patient continuum of care, informed consent, and other patient-centric skills. Finally, it will conduct behavioral research and offer behavioral co-studies with those conducting RMAT clinical trials, to understand what motivates patients in their decision-making and the behaviors necessary to optimizing their treatment outcomes.
Another part of the effort is consulting, established for the purpose of guiding providers wishing to conduct RMAT-designated trials through the process as policy emerges and solidifies here in the U.S. Providing these services to cell therapy providers is specialized, and very expensive. We are making these services available for another purpose other than business: to bring providers into the pipeline of FDA approval as soon as possible, so patients can access care. Without help, providers will not be prepared to verify and predict for patients the effectiveness or safety of their research / treatments.
Because patients need to access clinical trials, and providers need to conduct clinical trials, it is my opinion that a typical patient advocate is not enough to break the inertia. An ambassador, however, could be the answer. As an ambassador, then, I am able to communicate the common interests we all share as stakeholders in the cell therapy movement. Because of my pledge to help relay interests and needs of patients and cell therapy providers alike, I am consistently invited to, in a sense, “get up from the children’s table” - to outgrow the decades-old “patient pigeonhole” and to join the dialog where the real decisions are made.
This time around in DC, my focus will be communicating the needs of patients, and interests of cell therapy providers, which have emerged concurrently as themes in my conversations, and suggest ways to bring them together. Then, I’ll be back, bringing new insights, fresh ideas, and regulator's general sentiments (which often translate to goals). The information is general, and not designed to give anybody an advantage over anybody else. I will also suggest ways we as patients or cell therapy providers can HELP the folks in Washington make their jobs easier, collaborate with us to smooth the pavement for providers navigating the new RMAT pathway, or help patients join a clinical trial. These individuals give up their time to meet with me, for the goal of helping patients access this new system. I share my thoughts, ideas, strategies and questions with them.
My itinerary is firming up, but for now, here is a partial list of the people I’ll be visiting with. :
Dr. Francis Collins - Director, NIH
(RMAT ) Designation reviewers at FDA CBER
Dr. Celia Witten - CBER
Dr. Peter Marks - CBER
Jonathan Jagoda - Director, Federation of State Medical Boards*
Tissue Regeneration Company/Researcher(s)
Various Pharmaceutical Federal Policy Representatives
Various Others (TBA)
I leave you with a question only an ambassador can ask: “If you were talking one-on-one with decision makers in the stem cell therapy arena, what would you say? What questions would you ask?”
If you are a patient, please know you may also message me, or contact me any way you’d like. Tell me what you need, and I’ll try to pass it on to the right people if I can. If you are a provider, feel free to contact me.
*The FSMB recently appointed me to their Stem Call policy and practice workgroup, which is now poised to start helping state medical boards to begin self-regulating cell therapy practice
*Regenerative Medicine Approved Therapy (new FDA pathway for accelerated approval of cell therapy products and devices)