This has been a landmark year for the cell and gene therapy industry. It’s also been an exciting year for Be The Match BioTherapies®, as we’ve forged new partnerships and expanded our international presence. We highlight a few key efforts below as we look forward to the year ahead.
The push toward cell and gene therapy standardization
Standardization became a buzzword this year with the demand for cell and gene therapies on the rise. Industry needs, audit fatigue at apheresis centers and cell therapy labs, and other issues strained the growing cell therapy infrastructure, raising questions about capacity and efficiency. Amy Ronneberg, president of Be The Match BioTherapies, spoke to reporters at STAT and MedCity News about the concerns in CAR-T therapies in particular.
Throughout 2018, Be The Match BioTherapies worked with cell and gene therapy companies, apheresis centers and others to improve standardization and remove hurdles to cell therapy expansion, including hosting a summit with industry stakeholders and, more recently, launching the Quality System Audit Program.
We look forward to continuing this collaborative work in 2019, working closely with key industry leaders and the Standards Coordinating Body, to move us closer to our common goal—saving more lives through cell and gene therapy.
Globalization of cell and gene therapy
The cell and gene therapy industry continues to expand throughout the world, rapidly advancing through key markets including the E.U., U.K., China and Australia. The strength that comes from a global presence and the support of global partners is continually showcased across the industry.
To support the global growth of the industry, Be The Match BioTherapies has also continued to expand internationally. In September, Be The Match BioTherapies announced a collaboration with Cellex Cell Professionals, a German-based full service provider, and welcomed an international collection network manager, Petr Machalik. In December, we also announced a collaboration with the National Health Service Blood and Transplant of the United Kingdom.
China’s rise as a biotech giant continued throughout this past year. According to STAT, China’s “Pharma Valley” is now home to over 500 biotech companies, while several U.S.-based companies have expanded their presence there. Immunotech, a medical startup, recently launched China’s largest cell therapy trial.
And China is not alone in supporting the booming biotech industry. Landmark approvals in Europe have expanded patient access to cell and gene therapy and broadened the conversation about the future of medicine.
Outcomes and reimbursement in CAR-T
The 2017 approvals of Kymriah® and Yescarta®, which spurred growth of a robust pipeline of CAR-T therapies, represented a new era for patients with blood cancers. Throughout 2018, CAR-T research has continued to grow, but challenges regarding manufacturing, cost and reimbursement have created roadblocks for patients.
As Susan Leppke, director of Health and Public Policy at the National Marrow Donor Program®/Be The Match®, explained in a previous Cell Lines blog post, “Most manufacturers are used to the world of drugs. A CAR-T therapy is a treatment – yes, it’s a drug, but there’s a whole mechanism involved that’s not the drug.” That mechanism has additional costs, which create reimbursement challenges that are causing long waitlists for some patients. Despite the challenges, Leppke and her colleagues are making progress in expanding access so patients can get the life-saving therapies they need.
In the meantime, research on CAR-T therapies continues to advance. Researchers are beginning to track long-term patient outcomes through controlled clinical trials and data collection.
Companies across the biotech space are also pursuing therapies that go beyond autologous treatments like Kymriah and Yescarta, which require cells to be isolated from a patient’s blood before they can be manufactured for treatment. Allogeneic cell therapies, now in development across the biotech space, could give patients a cost-saving, “off-the-shelf” option.
Progress in hematology/oncology research
At the American Society of Hematology (ASH) annual meeting, held this year in San Diego, “CAR-T and gene therapies affirmed their emerging place in the treatment of blood illnesses” as Jacob Bell wrote for BioPharma Dive.
An allogeneic cell therapy for acute lymphoblastic leukemia from California-based Allogene made a positive splash, while bluebird bio’s gene therapy to treat transfusion-dependent β-Thalassemia and others, such as Spark’s gene therapy to treat hemophilia were subjected to scrutiny. Both Spark and bluebird bio have been recognized as leaders in the field of gene therapy and are being closely watched as their gene therapy treatments evolve.
Regulation and pricing of gene therapy
In July 2018, the FDA issued six draft guidance documents related to gene therapy development for rare diseases including hemophilia, retinal disorders and rare disorders. The guidance documents offer a look at what the FDA expects from a growing industry and also present a roadmap to the many companies with gene therapies currently in development, as Zachary Brennan explained in Endpoints News.
Ann Kemp, senior manager of Regulatory Compliance for Be The Match BioTherapies, provided her perspective in a previous Cell Lines blog post saying, “The updates to the manufacturing documents provide needed clarity for the industry. A major highlight of these guidances is an update to the definition of gene therapy, which hadn’t been changed in over a decade.”
Pricing of these transformative therapies continues to be a question, especially after Novartis announced in November that a single dose of their new gene therapy to treat spinal muscular atrophy could be valued at more than $4 million. (The company hasn’t yet set an actual price for the drug.)
Such sums raise inevitable and important questions about access, but it’s also crucial to remember that when these therapies finally do reach patients – Spark’s Luxturna, for example – the impact on quality of life and long-term health outlook can be enormous.
It’s been an eventful year in the industry, and we look forward to seeing the advancements that 2019 brings.
Whether your therapy is in research, pre-clinical or clinical studies, or commercially available, Be The Match BioTherapies has the unparalleled industry expertise in designing valuable solutions to advance your therapy.