A look at distributed manufacturing for advanced therapies
In October 2022, the United States FDA announced it was seeking feedback on Distributed and Point-of-Care Drug Manufacturing.
For those of us in the development, production, or delivery of cell, gene, or tissue-based advanced therapies, it was easy to ignore. It was issued by the FDA Center for Drug Evaluation and Research (CDER) and, on the face of it, looked to be about 'drugs' and manufacturing technologies, including devices in development for the decentralized or point-of-care manufacture of such drugs. On a closer read of the 'scope', however, it was evident this was relevant to #advancedtherapies in a very real way based on the following quote:
"While the initial analysis focused on products regulated by CDER, FDA’s Center for Biologics Evaluation and Research (CBER) also expects the development of advanced manufacturing technologies associated with DM and POC manufacturing for products that it regulates. As such, we invite feedback on the discussion questions related to products regulated by CDER and CBER."
With the rapid evolution of technologies in development to enable the decentralized manufacturing of advanced therapies - from 3D printers to cleanrooms-in-a-box, to closed and automated manufacturing systems, to mobile manufacturing units - there is clearly a technology push to enable distributed and at or near point of care manufacturing or at least for certain production units of the manufacturing workflow to be done outside of highly centralized facilities. For some, this is seen to be a key to lowering the costs of at least certain types of cell and gene therapies, particularly autologous treatments. Combine that with the high manufacturing costs associated with today's approved cell and gene therapies (which according to the Institute for Clinical and Economic Review (ICER) currently average $1-2M per treatment) and there is certainly an associate market pull to explore avenues to lower costs.
All of this is why at least the US FDA and the UK's MHRA are beginning to actively explore how to design regulation for a future that will incorporate different manufacturing models including those which enable decentralization of at least certain segments of drug and biotherapeutic production.
In conjunction with October's announcement, the FDA issued a discussion paper with some of their very preliminary thoughts and a call for feedback - all of which can be read here.
To be clear, Blood Centers of America 's network of centers is engaged in its mission to support saving patients' lives and empowering patient health by enabling the availability and use of human blood, cells, and tissues. We remain agnostic or supportive of many drug, biotherapeutic, healthcare, manufacturing, or business models. However, with its national footprint of facilities across the United States and a history of biologic supply to community-based healthcare providers, the independent blood centers certainly envision a role in supporting donor and patient biological collections, testing, storage, and delivery of advanced therapies to support both centralized and decentralized production models.
In keeping with its mission, BCA participated in the call for feedback from the FDA to ensure the voice, capabilities, and infrastructure of the nation's independent blood centers was heard and has a seat at the table for this critically important discussion. We are proud to be among the first tier of stakeholders to engage the FDA on this important subject including other submissions from BIO, PhRMA, ISCT, AABB, ISPE, Miltenyi, Kite, and others. Each of these submissions can be viewed here:
The Advanced Therapies Network of Blood Centers of America looks forward to being an active part of the many ways we all can contribute to bringing advanced therapies to every patient who needs them. For both centralized and decentralized manufacturing models, this may include leveraging the network's apheresis collection sites across the United States, providing access to umbilical cord blood or birth tissue, testing services, near point-of-care unit processing or full-scale manufacturing, cryopreservation or storage near the patient or hospital, and biorepository and distribution to healthcare facilities in real-time, on-demand.
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