In case you haven’t heard, last December, with the passage of the 21st Century Cures Act, Congress provided an avenue for FDA reform that has opened an opportunity to talented, research-oriented cell therapy providers to become investigators and conduct clinical trials on the compounds they are using to treat patients.
It’s a conceptual “rework” of the REGROW act, which proposed a “second pathway” for regenerative medicine cellular and tissue product approvals, based on a separate-but-equal “non drug” concept, similar to the model used in Japan that has been largely responsible for a boom in innovation and investment overseas.
It’s complex, but suffice it to say that the instrument through which the 21st Century Cures Act mandates cellular and tissue “products” be trialed for safety and efficacy, concurrent with immediate patient access, is called the Regenerative Medicine Advanced Therapy (RMAT) designation, and FDA is currently accepting applications.
The door is open, and, if you’re a provider doing good work helping patients, it’s an exciting opportunity to validate your efforts, not to mention, attract investors.
But, there are few resources to the kind of specialized knowledge, information, context and resources needed to guide interested providers carefully through the new nooks and crannies in achieving the designation.
Here are five (5) great reasons to consider hiring a consultant to help you with obtaining the RMAT designation:
Time and the unknown: There are likely to be more than the average number of glitches in the early implementation of the RMAT, on both the applicant and regulatory sides. You may want to continue your practice and let somebody else handle the bumps in the road.
Awareness of regulatory sentiment: It’s important to understand that being in tune with not just the letter of the law, so-to-speak, but the spirit of it, The added value a well-informed consultant can bring to your application comes from guiding both your strategic planning and sometimes, even the indication you choose. This gains a regulator’s attention and reflects a convenient and meaningful “fit” as the regulator reviews it.
Collaborative connection: We’re in interesting times when it comes to attitudes about regulation and public policy. It is my belief that collaboration with FDA actually has a chance to work right now, perhaps in part to a shared anticipation among providers and regulators of the unpredictable impact from political and economic forces.
The “Serious Condition” provision: This is a relatively new idea for the routine mainstream of IND’s, although it is in use for compassionate care-based approvals. Also, the qualification(s) for RMAT designated studies are not purely market-driven. They are quality-of-life driven as well. A consultant can bring strategic nuance and creative thinking to strengthen your RMAT application and justification. It is still uncertain which conditions will be considered “serious”, but now is the time to influence the list.
Cost: There are many clear reasons for investing on the front end in planning and executing the steps to an RMAT trial. Among them are: avoiding holds, withdrawals, and sub-optimal IRB matches. Failure to mitigate against these risks exposed by lack of planning and guidance will almost certainly result in losing “considerable coin”.
Message me if you’d like to know more about how a uniquely positioned consultant can help.