Roundtable Part 4: What can industry, regulators, and tech providers do to make the implementation of cell and gene therapies more efficient?

Continue to better broker the free exchange of ideas and knowledge gained from lab bench and manufacturing suite, to the clinic.

All parties involved should continue the collaboration to define this set of platform assays/methodologies which innovators, contractors, and regulators could be familiarized with.

Regulatory approaches, published methods, and reference materials are all well recognized as areas where more support and guidance are critical to the future of CGT. In addition, we need well-trained scientists knowledgeable in the techniques and concepts around CGT. Partnerships and engagement with educators is an area where we, the active members of this community, can make a certain difference in the future of CGT. Courses around the mechanisms of therapies, the equipment utilized for testing safety/potency/purity, and understanding the fundamentals of a regulatory career in this field will ensure that the excitement and skill set are ready for the necessary development and commercialization of the next generation of cell and gene therapies.

Partnering with your vendor is critical. It’s important to work through the engineering aspects to ensure you can be as closed as possible. We hear in the industry about 100% functionally closed versus 100% closed. Here at Matica Bio, we are moving to 100% closed because we think that it is critical and it also de-risks our process and increases potential for safety of the product itself. It also ensures that what we deliver at the end is safe, sterile and effective.

Thank you for this question.

FDA is steadfastly committed to working with stakeholders, including other government agencies, academia, industry, and product manufacturers, to foster beneficial innovation in this field and make the development and approval of innovative cell and gene therapies more efficient.

As we continue to develop an evidence-based framework for these products, FDA understands that we may need to re-evaluate and modernize our approach to the unique challenges of these products while also ensuring the resulting therapies are both safe and effective.

There are indeed steps that could be taken toward more efficient gene therapy product development. The sharing of best manufacturing practices among academic and industrial developers could result in advances associated with better product quality, including consistency and yield, along with reduced costs. Additionally, while countries around the world have their own regulatory authorities, there are not uniform global quality safety standard for the evaluation and regulation of cell and gene therapy products. FDA supports work toward regulatory convergence and, ultimately, global harmonization of regulations for these products. FDA is pursuing this goal with international partners, global regulators, and the World Health Organization (WHO).

The key to improving the implementation of these therapies is understanding how new technology, regulations, and economics will evolve in the future to improve the efficiency of manufacturing, standardization, adoption and reimbursement. Tech providers need to be “ahead of the curve” to ensure therapy developers have the right tools to scale their manufacturing processes, the regulators need to be sufficiently resourced to review and approve new therapies at scale, clinical staff need to be suitably educated to administer these drugs and support patients through the critical early phases, and insurance providers and healthcare services need to have streamlined systems to ensure efficient reimbursement. We are a long way from this right now – but steady progress is being made in all these areas.