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Latest & Greatest CMS Proposes Higher Reimbursement Rate for CAR T-Cell Therapies The Centers for Medicare and Medicaid Services (CMS) has proposed a new system for calculating reimbursement rates for hospitals administering chimeric antigen receptor (CAR) T-cell therapies. The system was released as part of the proposed 2021 Inpatient Prospective Payment System (IPPS), which determines Medicare payment policies. Under the new approach, CMS would compensate health-care systems that administer CAR T-cell treatments based on the average price of the two therapies currently on the market: Gilead’s axicabtagene ciloleucel and Novartis’ tisagenlecleucel. Currently, Medicare reimbursement rates for CAR T-cell therapies are based on the average cost of administering a bone marrow transplant, which a 2017 study found has a median cost between $150,000 and $300,000, depending on the patients’ diagnosis. CAR T-cell treatments, however, have list prices more than $450,000 per patient. The previous reimbursement system may have forced hospitals to choose between taking a financial loss or declining to offer the treatment. The new rules would affect only patients who receive coverage through Medicare, but private insurance providers may use federal guidelines as a model for setting their own payment rates. CMS is expected to confirm the IPPS rulings in the fall after accepting comments from the public and industry and patient groups. Sources: STAT, May 12, 2020; American Health & Drug Benefits, October 2017. FDA Approves Tazemetostat for EZH2-Mutated Follicular Lymphoma Tazemetostat, an enhancer of zeste homolog 2 (EZH2) gene inhibitor, was granted accelerated approval for adult patients with relapsed or refractory follicular lymphoma (FL) and an EZH2 mutation who have received at least 2 prior therapies or have no satisfactory alternative treatment. The agency simultaneously approved the cobas EZH2 Mutation Test as a companion diagnostic for tazemetostat. Approval for tazemetostat was supported by results from two open-label, single-arm cohorts of a multicenter trial that included 95 adults with histologically confirmed, previously treated EZH2 mutant FL. Patients received tazemetostat 800 mg orally twice daily until disease progression or unacceptable toxicity. The trial’s primary endpoints were overall response rate (ORR) and duration of response (DOR). The ORR was 69% for 42 patients with EZH2- mutation FL, with 12% achieving complete response (CR) and 57% achieving partial response (PR). Median DOR in these patients was 10.9 months (95% CI 7.2 to not estimable). In 53 patients with EZH2 wild-type FL, the ORR was 34%, with CR and PR rates of 4% and 30%, respectively. The median DOR for this group was 13 months (95% CI 5.6 to not estimable). The most common adverse events associated with tazemetostat (occurring in ≥20% of patients) included fatigue, upper respiratory tract infection, musculoskeletal pain, nausea, and abdominal pain, with serious adverse reactions in 30% of patients. The approved starting dose is 800 mg taken orally twice daily with or without food. Sources: FDA press release, June 18, 2020; Roche Diagnostics press release, June 19, 2020. PTG-300 Receives Orphan Drug Designation for Treatment of Polycythemia Vera The FDA granted orphan drug designation to PTG- 300 for the treatment of phlebotomy-requiring polycythemia vera (PV). PTG-300 is an injectable synthetic hepcidin mimetic in development for the treatment of PV and hereditary hemochromatosis. The agency’s decision was based on data from a phase II, open-label, dose escalation study of adult patients with PV who had received at least three phlebotomies within a previous 24-week period. Preliminary results showed that treatment with PTG-300, at doses ranging from 10 to 80 mg for up to 28 weeks, led to dose-related control of hematocrit levels and eliminated the need for phlebotomy in all 6 patients who received per-protocol treatment. The drug’s manufacturer reported that a seventh patient had an unintended dose interruption at 12 weeks, received a single phlebotomy, and resumed participation. Injection-site reactions and bruising were reported as the most common adverse events, and the drug’s safety profile was found to be similar to that observed in prior studies. Enrollment in the study is ongoing and a total of 8 patients have enrolled to date. Sources: Protagonist Therapeutics press release, June 17, 2020; Protagonist Therapeutics press release, May 7, 2020; ClinicalTrials.gov, December 20, 2019. FDA Partners With NY Health Tech Firm to Collect Real-world COVID-19 Data The FDA announced a new research project to collect real-world data from insurance companies and electronic health records on the use of diagnostic tests and medications for COVID-19. The agency is partnering with New York–based health tech company Aetion for the project. Together, the FDA and Aetion hope to use advanced analytic techniques to understand how COVID-19 presents and is treated among different patient populations, identify risk factors for coronavirus-related complications, and evaluate the outcomes of potential interventions. The collaboration will make use of the Aetion Evidence Platform, which will make it possible to facilitate sharing and reproduction of findings. “The urgency of addressing the COVID-19 pandemic has demanded that we expand our work to identify, access, and analyze new data sets to widen the breadth of the information available,” said FDA Principal Deputy Commissioner Amy Abernethy MD, PhD, in a statement. The partnership was a result of the FDA’s participation in the COVID-19 Evidence Accelerator, an initiative of the agency’s Reagan-Udall Foundation and the Friends of Cancer Research. The Accelerator brings together experts from government, academia, and industry to collaborate on gathering and sharing real-world evidence on the pandemic. The Evidence Accelerator initiative has two components: the Therapeutic Evidence Accelerator (launched in April) and the Diagnostics Evidence Accelerator (launched in June). Sources: STAT, May 19, 2020; FDA press release, May 19, 2020; Aetion press release, May 19, 2020; FDA press release, June 18, 2020. Check out online exclusives at www.ashclinicalnews.org 12 ASH Clinical News July 2020 Bonus Mid-Year Edition