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.
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12 ASH Clinical News July 2020 Bonus Mid-Year Edition