2018 has been a year of advancement for the cell therapy industry. With the first two approved CAR-T cell therapies now on the market, organizations are shifting their focus from commercialization to industrialization through well-designed supply chains, standardized quality assurance practices and long-term outcomes tracking to support current and future cell therapy products.
Let’s take a look at the key challenges, advancements and trends that are shaping our industry.
The need for industry standardization
Developing uniform standards for the collection, manufacturing, shipping, distribution and reporting of cell therapies has been an area of focus this year.
“The topic of standardization is certainly gaining traction in the field, with an increasing number of conference presentations and strategic discussions devoted to this key subject,” said Mark Flower, Vice President of Business Development and Strategic Partnerships at Be The Match BioTherapies®. “In order for therapies to continue to advance and be available to patients in need, the current burden on the infrastructure, primarily in apheresis and collection facilities, needs to be reduced. There is a huge opportunity to leverage our expertise in incorporating standardized protocols and licensed audits, to ensure that the industry continues to move forward.”
This effort requires collaboration across the industry, from apheresis centers to cell therapy developers, and increasingly requires international coordination. Novartis and Gilead are moving quickly to market their CAR-T therapies in Europe, establishing manufacturing and supply chains there.
While this process can be challenging, progress has been made in the last six months, including a Standardization Summit hosted by Be The Match BioTherapies that brought together key industry leaders, collection center professionals, standards coordinating bodies and transplant physicians.
Decentralized manufacturing models
With the maturity of the cell therapy industry, the topic of decentralizing manufacturing continues to arise. Recently these conversations have shifted, yet still rely heavily on improvements in technology that enable manufacturing of cell therapies at the point of care. This approach can simplify supply chains, allow for more flexibility and perhaps make personalized treatments much easier.
At the leading edge of this trend is Miltenyi Biotec. The company’s CliniMACS Prodigy device is an all-in-one automated manufacturing system with the ability to make various cell types. Earlier this year the Prodigy was for the first time approved as the manufacturing platform for a commercial drug, Zalmoxis, by the EMA.
The rise of China as a cell therapy development hub
This year has seen growth in the Chinese cell therapy industry, a trend that seems likely to continue. At the start of the year, China had 153 CAR-T studies in progress, just behind 186 in the U.S., Bloomberg reported. One of the most prominent Chinese cell therapy companies is Legend Biotech, based in Nanjing, which reported impressive data from its anti-BCMA CAR-T therapy at ASCO last year and went on to sign a $350 million deal for the treatment with Johnson & Johnson at the end of 2017. In March, the Chinese FDA approved the company’s clinical trial application, making LCAR-B38M the first cellular therapy to enter clinical trials in China.
Continuing debate about reimbursement environment
As payers have begun to set policies around coverage of CAR-T therapies, the reimbursement debate persists. Earlier this year the Centers for Medicare and Medicaid Services agreed to reimburse Yescarta and Kymriah via the Medicare Part B program, though the decision raised further debate about whether CAR-T infusions should take place in an inpatient or outpatient setting. In May the agency went a step further, initiating a National Coverage Analysis for CAR-T therapies. That process, expected to take a full year, will determine whether CAR-T treatments are nationally reimbursed by Medicare, and will serve as a key benchmark as commercial insurers formalize their policies. Our Public and Payer Policy colleagues, including Susan Leppke, Director of Public and Payer Policy at National Marrow Donor Program® (NMDP) /Be The Match®, are leading the charge to generate support and raise awareness of the need to bring more therapies to patients in need.