Recently, I taught a module on regulatory trends and RMAT strategy at a hands-on weekend clinical training event in Austin, one of those events at which clinicians planning to integrate cell-based therapy within their current practices learn the basic theory, principles, protocols and technologies available to get them started, or enhance their knowledge and skills.
Although I’m of the opinion that, if done with high standards and credentialed faculty, (like I believe the event I attended last week in Austin was), some of these weekend seminars can serve a valuable function, they remain controversial to many, who claim that a “weekend course” is inadequate preparation for any responsible practitioner considering entering the regenerative medicine space.
Regardless of one’s stance on the matter, I have learned since I first began attending them as an inspiring “patient face” to raise awareness, that these events serve as an accurate barometer for detecting the prevailing sentiment of the cell therapy community on many issues.
These forums are an impressive example of a peer culture, complete with generalized beliefs, values consensus, and even dogma. I have observed each of these evolve since the beginning of my involvement in reaching out to this community, and have noticed over the last few I have attended that there has been a new emphasis on accountability and ethical responsibility, originating from within the participant population (the faculty and programming has remained essentially the same).
It is a clear cultural shift in preparation for strengthening regulatory trends, including a likely rollout of state legislative initiatives to join Texas and Arkansas, and self-regulation policies by state medical boards over the next year or so.
The initial excitement among participants in the early days workshops I attended was palpable. However, as time has passed, and as the regulatory environment is shaped and clarified under current FDA leadership, the allure of novelty has notably diminished. The proliferation of knowledge, commentary and analysis out there is beginning to demystify the novelty and dim the satisfaction of out-smarting regulations. A consensus is growing, slowly, that it’s time to plan for regulatory oversight, from many different players.
The customary “revolutionary” tone of a “Wild West” scenario that has been the hallmark stereotype (sometimes deserved) of independent innovators in the field to date, is becoming more subdued, and is being challenged finally, by participants with the willingness to ask the right questions.
Although some players will always wrap a tight-fisted grip around deeply-held biases and experiences that spawned the culture to question regulatory authority, challenge its scope and relevance for stem cells, and press the envelope, forcing the issue, raising the bar, and modeling decentralization of innovation, is again redefining itself.
In Austin, there was outward, vocal approval, and expressions of relief at two recent FDA actions against “peers” known to the field, and in many cases personally and for a long time.
I’ve seen this coming for some time, and have been communicating my observations to top regulators and policy makers, but Austin has cemented my belief that the stem cell clinic industry, in general, is now beginning to incubate more than feverish forward thinking. The “go to” self-made innovators in the field challenged other presenters on their assumptions of safety, appropriateness of claims, and level of specialty knowledge. They warned each other not to simply listen to peers’ advice when determining their regulatory responsibilities. Not everyone present was appreciative, but the trend and message were both clear.
For my part, I tried to do my job as an “Ambassador Bridge”, communicating as responsibly as I could the knowledge gained from the open communication afforded me by NIH and FDA: assume no radical departures from regulatory positions established over the last several months. Science hasn’t changed, and what is not known about safety still remains largely unknown, and must be established through sound and rigorous trials.
And, that Federal regulators are willing to provide heavy support to help, and we patients can expect their commitment to getting there as quickly and efficiently as possible, most probably though offering clinic-based researchers and small business labs access to the careful and creative use of the new RMAT designation for clinical trials.
And, especially the new “reality mix” they haven’t been expecting.
I presented a slide entitled “The FDA Is No Longer Alone”, depicting the several newcomers to the regulatory landscape, all of whom will soon show their face and claim their space in the oversight matrix that is evolving: State medical boards, professional organizations, national clinical registry project initiatives, private certification organizations, IRB reform efforts, and, most interestingly, state counter-legislative and regulation activity.
The presentation was incredibly well received, for which I am grateful. Not surprisingly, though, the news of another shift in power and control was a little unsettling.
How should weekend education providers respond and support their participants’ need for regulatory preparation?
As a patient, I feel the first step should be an ethics “reboot”, and a commitment to first understand the themselves the upcoming trends, and then to enforce a corresponding set of policies and standards within their curriculum, with their sponsors, and for faculty relationships.
It’s also time for them to vet their sponsors and speakers against the prevailing ethical standards, including scrutiny of what the participants are signaling needs attention: claims, assumptions, patient ethics and informed consent, specialty knowledge, and engagement of regulatory authority.