Making advanced therapies takes industrializing personalization – STAT

Whats the best way to measure the real rate of progress in personalized cell therapies, gene therapies, and other advanced therapies?

Ive been tracking the ever-growing flow of reports about these therapies in scientific journals and press releases for 15 years, ever since I co-led the passage of Californias $3 billion Stem Cell Research and Cures Act in 2004.

But to truly gauge who will benefit from todays innovations, Ive learned I also need to study the stream of business and technology announcements that runs in parallel. That might seem more mundane but to veterans of advanced therapies, making the science work actually signals success for these gene-, tissue-, and cell-based advanced therapies.

The reason is simple. My experience working with advanced therapies has taught me, time and again, that true next-generation medicine requires the industrialization of personalization. That sounds like an oxymoron, but it isnt. To create individualized therapeutics in a sustainable way, we need to deliver even if it seems counterintuitive mass customization.

Breakthroughs such as CAR-T cell therapies are inspiring. They are also unsustainably expensive, difficult to manufacture, and complicated to deliver. We can change this by creating a more focused cross-collaborative production and delivery ecosystem.

The Food and Drug Administration anticipates that it will approve 10 to 20 advanced therapies a year beginning in 2025. It also expects to receive up to 200 clinical trial applications for cell and gene therapies per year, starting now. The more than 1,000 advanced therapy clinical trials now underway worldwide could enroll almost 60,000 patients, according to the Alliance for Regenerative Medicine. That pace wont be possible without new systems and networks that reduce cost, simplify manufacturing, and streamline delivery.

I can see some of these on the horizon when I read the biotech and pharma partnerships reported in BioSpace and BioCentury. Of the 100 most recent, almost 10% were dedicated to cell- and gene-therapy companies and organizations. These partnership announcements are typically viewed as opportunities to highlight new business deals or contract wins. But they are also daily snapshots of the infrastructure of an evolving next-generation health care system forming from within. Here are just a few examples from 2019:

Its encouraging to see biopharma manufacturing, logistics, transport, and other partners in the cell- and gene-therapy ecosystem coming together in new ways to ensure the successful and reliable delivery of advanced therapies for individual patients. But much more evolution is needed to provide sustainable patient access to advanced therapies.

We need even more industry collaboration to overhaul and connect existing health care systems, so production and delivery of cell- and gene-based therapies can be more automated and affordable. According to estimates from credible industry colleagues and leaders, end-to-end automation can shave costs by at least 20% to 30%, and at the same time greatly improve predictability and patient safety.

We must also make this new world simpler for health care providers. Doctors and nurses must not only understand how advanced therapies work medically, but be able to order and deliver them safely with a minimum of delay or hassle. As noted in the New Yorker, CAR-T requires bringing a manufacturing lens to medicine. Supporting health care providers means creating true collaboration between digital technology providers, hospitals, logistics providers, biotech and pharma companies, and manufacturing, like the Boston initiative I described earlier.

Standardization is often decried as cookie-cutter medicine. In this space, however, it is the wave of the future.

While patient biology is unique, and each patients cells may produce a one-of-a-kind manufacturing batch, essential parts of the production and delivery process should be as predictable and easy as possible. One key place to start is in-process drug labeling. When patients cells become the raw material for advanced therapies, these labels become more complex and more necessary: When a patient is about to receive a cell therapy infusion, its essential that the name on the bag of genetically re-engineered cells is his or hers. The Standards Coordinating Body, an FDA-funded but independent nonprofit, is now leading an industry-wide labeling initiative for cell and gene therapies.

There are other clear signs that the advanced therapies field gets it when it comes to infrastructure needs, such as the inclusion of digital health and handling of patient data as categories of focus in the federal Cures 2.0 initiative currently circulating in Washington. But much remains to be done.

In centers caring for individuals with cancer and rare diseases, thousands of patients are today receiving advanced therapies that are transforming their lives. We need to make that possible for many, many more by working together to industrialize and personalize in parallel.

Amy DuRoss is the CEO and co-founder of Vineti, a digital technology company that provides next-generation software platforms for advanced therapies. Before that she was managing director for new business creation for GE Ventures, chief business officer at Navigenics, the co-founder and executive director of Proposition 71, Californias $3 billion stem cell research initiative that passed in 2004, and chief of staff at the resulting California Institute for Regenerative Medicine.