Cell therapy offers a treatment option for some advanced blood cancers, but the manufacturing process is lengthy and complex. The biopharmaceutical industry is working on various technologies that could make manufacturing faster and more efficient, or perhaps supplant the current process altogether.
The company said the study of Kymriah in follicular lymphoma met its primary endpoint, though it did not disclose the trial data. It plans to submit approval applications to the FDA and EMA next year.
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
Data on multiple CAR-Ts presented at the recent ASCO meeting have raised the prospect of outpatient use of the therapies, which is favorable for reimbursement as well as for patients. But experts pointed to significant infrastructure challenges that centers face.
A team at UPenn led by CAR-T pioneer Carl June used CRISPR/Cas9 to engineer T cells for treating multiple myeloma and sarcoma. Data on three patients were published in Science. All the patients' cancers ultimately progressed, though none had cytokine release syndrome.
More than years after its initial approval, Kymriah has shown comparable efficacy and an apparently better safety profile than it demonstrated in the studies that led to its leukemia and lymphoma approvals. Another therapy, made by BMS, is a potential competitor.
The Medicare coverage decision, issued Wednesday evening after a nearly three-month delay, removes several requirements that some had feared would limit access to the cell therapies, while also allowing for some off-label use.
At the Abarca Forward conference earlier this year, George Van Antwerp, managing director at Deloitte, discussed how social determinants of health and a personalized member experience can improve medication adherence and health outcomes.
In a statement, the agency said that a decision on whether to have Medicare cover the cell therapies would not be issued Friday, but would be forthcoming.
A spokesperson for the company noted that patients in the Phase II L-MIND trial of the CD19-targeting drug tafasitamab would be too old and frail to be candidates for CAR-T therapies.
The agency also proposed increasing the maximum new technology add-on payment from 50 percent to 65 percent for fiscal year 2020.
Allogeneic CAR-Ts could serve an important niche - particularly for patients who can't receive autologous CAR-Ts - but the earliness of the data makes conclusions tough to draw.
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The lack of an overarching CMS reimbursement policy on CAR T-Cell therapy has meant that coverage decisions have generally landed on the desk of local Medicare Administrative Contractors.
NICE recommends Kymriah to the Cancer Drugs Fund due to remaining questions about evidence to support it, but believes those questions will be resolved.
Along with Boston Scientific's Watchman in the technology category, Novartis and Gilead's Kite shared the biotechnology award for CAR-T therapy, while Celgene/Agios's Idhifa won the pharmaceutical prize.
Still, despite costs largely in line with the therapy's benefits, the study's author and another expert said reimbursement models have not kept up and need improvement.
A manufacturing error during a clinical trial of Kymriah, now marketed by Novartis, is less likely today, but could be an issue as CAR-Ts move into earlier therapy lines.