VALUE IN HEALTH
-
MAY 2020
S45
vs. 10.7 months). Baseline characteristics were well-balanced. During 1L OCM
episode and observed 1L duration, ibrutinib patients had significantly fewer monthly
days with outpatient services compared to CIT patients (1L OCM: RR= 0.74,
P,0.0001; 1L duration: RR= 0.86, P,0.0001). Ibrutinib patients incurred significantly
higher monthly pharmacy costs (1L OCM: MMCD= $4,878, P,0.0001; 1L duration:
MMCD= $4,892, P,0.0001) that were fully offset by lower monthly medical costs (1L
OCM: MMCD= -$8,289, P,0.0001; 1L duration: MMCD= -$5,888, P,0.0001), yielding
a monthly total cost reduction (1L OCM: MMCD= -$3,411, P,0.0001; 1L duration:
MMCD= -$996, P,0.0001) versus CIT patients. Conclusions: During 1L OCM epi-
sodes and observed 1L duration, continuous ibrutinib treatment was associated
with lower outpatient HRU and total cost reduction compared to CIT in Medicare
patients with CLL/SLL. The total cost reduction was driven by fewer outpatient
days.
PCN127
COMPARISON OF PARTITIONED SURVIVAL VERSUS
MARKOV COHORT MODELING APPROACHES IN THE
EVALUATION OF COST-EFFECTIVENESS OF
BLINATUMOMAB VERSUS CHEMOTHERAPY IN ADULT
PATIENTS WITH ACUTE LYMPHOBLASTIC LEUKEMIA IN
FIRST HEMATOLOGICAL COMPLETE REMISSION WITH
MINIMAL RESIDUAL DISEASE
1
2
2
3
2
2
Delea TE, Despiegel N, Boyko D, Dirnberger F, Tiwana S, Sapra S
1
2
Policy Analysis Inc., Brookline, MA, USA, Amgen Inc., Thousand Oaks, CA, USA,
Amgen GmbH, Munich, Germany
3
Objectives: For rare diseases like acute lymphoblastic leukemia (ALL), trials
frequently present limitations including small samples and single arm design. Mar-
kov cohort models (MCM) can include intermediate endpoints affecting survival but
are data intensive. In this context, we propose to assess the value of using an MCM
instead of a partitioned survival model (PSM) that only relies on relapse-free survival
PCN125
NOVEL ANTI-SICKLING AGENTS AND HYDROXYUREA
VERSUS CHRONIC TRANSFUSIONS IN SICKLE CELL
DISEASE: A COST-EFFECTIVENESS ANALYSIS
(RFS) and overall survival (OS) in evaluating cost effectiveness of blinatumomab
versus chemotherapy. Methods: In the PSM, RFS and OS were based on parametric
distributions fit to patient failure-time data from the BLAST trial and a historical
comparator study. To validate PSM findings, an MCM, which explicitly estimates the
contribution of minimal residual disease (MRD) and hematopoietic stem-cell
transplant (HSCT) on survival, was developed. Transition probabilities for HSCT,
relapse, and death, were estimated using the same data. We evaluated the sensitivity
of both models around parameters and assumptions. Results: Both models led to
similar incremental cost-effectiveness ratios for blinatumomab versus chemo-
therapy: $102,016/QALY in the PSM and $118,659/QALY in the MCM. Incremental
costs (PSM: $261,876; MCM: $242,940) and QALYs (PSM: 2.57; MCM: 2.05) were
comparable. The lower QALY difference in the MCM (versus the PSM) is due to lower
estimations of post-relapse survival for blinatumomab. The lower incremental cost in
the MCM is due to the explicit allocation of MRD-related costs, leading to higher cost
offsets. Probabilistic sensitivity analyses showed little variability due to parameter
uncertainty in both models. Conclusions: Using both models, blinatumomab is cost
effective versus chemotherapy in ALL patients with MRD for US healthcare payers.
The PSM structure cannot explicitly account for treatment effects on survival
mediated by MRD response or HSCT. However, the MCM requires more assumptions
due to limited data for estimating transition probabilities. When data is limited, a
PSM is recommended as the primary approach.
1
2
2
Onasanya O, Park JE, Zafari Z
1
2
University of Maryland, Baltimore, Baltimore, MD, USA, University of
Maryland School of Pharmacy, Baltimore, MD, USA
Objectives: Novel therapies for managing vaso-occlusive crises in pediatric sickle cell
disease (SCD) have emerged in recent years. However, evidence of their cost-effec-
tiveness is lacking. This study performed a cost-effectiveness analysis of off-label
hydroxyurea, hydroxyurea (Siklos), L-glutamine, crizanlizumab and voxelotor, as
compared to chronic blood transfusions as standard of care for managing SCD in
adolescents. Methods: Applying a decision tree model, we compared economic and
clinical outcomes of 4 novel FDA-approved products including off-label hydroxyurea
over one-year time horizon in a hypothetical cohort of adolescent SCD patients from
a US payer perspective. The main outcome is incremental cost per SCD-related
hospitalizations averted (ICER). Model inputs were derived from drug clinical trials,
published literature and average wholesale prices (AWP) from Wolters Kluwer Medi-
Ò
Span . Costs were converted to 2019 US dollars using the consumer price index.
Results: The incremental costs and incremental SCD-related hospitalizations averted
were –($7,951) and 0.4/year for hydroxyurea, $23,793 and 0.4/year for L-glutamine,
$35,986 and 0.2/year Siklos, $107,283 and 0.3/year crizanlizumab, and $141,883 and
0.5/year for Voxelotor. Generic hydroxyurea dominated standard of care, Siklos and
Crizanlizumab. The ICER of L-glutamine and Voxelotor, when compared to generic
hydroxyurea was $1,368,627 and $1,615,456 respectively per SCD hospitalization
averted. L-glutamine dominated Siklos and Crizanlizumab, but not standard of care.
PCN128
COST-EFFECTIVENESS OF RIBOCILIB PLUS FULVESTRANT
(R+F) VS FULVESTRANT (FUL) IN HORMONE RECEPTOR–
POSITIVE, HUMAN EPIDERMAL GROWTH FACTOR
RECEPTOR 2–NEGATIVE (HR+/HER2-) ADVANCED BREAST
CANCER (ABC): A CANADIAN HEALTHCARE PERSPECTIVE
At
a willingness-to-pay threshold of $100,000 per SCD-related hospitalization
averted, only L-glutamine was deemed cost-effective compared to standard of care
with ICER of $55,816 per averted hospitalization. Conclusions: Generic hydroxyurea
and L-glutamine may be cost-effective interventions for preventing hospitalizations
in adolescents with sickle cell disease.
1
2
3
3
4
Stellato D, Thabane M, Chandiwana D, Lanoue B, Delea TE
1
2
Policy Analysis Inc. (PAI), Brookline, MA, USA, Novartis Oncology, Mississauga,
3
ON, Canada, Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA,
PCN126
4
Policy Analysis Inc., Brookline, MA, USA
COST-EFFECTIVENESS OF SECOND GENERATION TKIS
COMPARED WITH IMATINIB FOR CHRONIC MYELOID
LEUKAEMIA: IMPLICATIONS OF TREATMENT-FREE
REMISSION
Objectives: The MONALEESA-3 trial demonstrated the efficacy and safety of R+F vs.
placebo plus FUL for patients with HR+/HER2- ABC. This analysis evaluated the cost-
effectiveness of R+F vs. FUL in patients with HR+/HER2- ABC from a Canadian
healthcare payer perspective. Methods: The incremental cost-effectiveness ratio
1
2
2
DA Costa M, Schaffel R, Lira R
(ICER) expressed as incremental costs per quality-adjusted life-year (QALY) gained)
1
2
FEDRAL UNIVERSITY OF RIO DE JANEIRO, RIO DE JANEIRO, RJ, Brazil, FEDERAL
UNIVERSITY OF RIO DE JANEIRO, RIO DE JANEIRO, Brazil
for R+F vs. FUL, was estimated using a semi-Markov cohort model developed in
Microsoft Excel with states for progression-free (PF), post-progression (PP), and
dead. A 15-year time horizon was used. Survival distributions for PFS, PPS and time to
discontinuation (TTD) were based on parametric survival distribution fit to data from
MONALEESA-3. Health-state utilities were estimated using EQ-5D index values
collected in MONALEESA-3. Direct costs of ABC treatment (medication and admin-
istration costs, follow-up and monitoring, adverse events, subsequent treatments)
were based on Canadian-specific values from published sources. Costs ($ CAN) and
QALYs were discounted at 1.5% annually. Results: In the base case, R+F was esti-
mated to result in gains of 1.20 life years and 0.98 QALYs vs. FUL, at an incremental
cost of $152,757. The ICER of R+F vs. FUL was $156,638 per QALY gained based on
deterministic analyses and $157,293 based on the mean of probabilistic analyses.
Results were sensitive to parametric distributions used for projecting long-term TTD,
PFS and PPS. Conclusions: For patients with HR+/HER2- ABC, R+F is projected to
result in substantial gains in QALYs compared with FUL. At its current list price,
Ribociclib used in combination with FUL is cost-effective in these patients at a
threshold ICER of $156,638. These results may be useful in deliberations regarding
reimbursement and access to this treatment.
Objectives: Although second generation Tyrosine kinase inhibitor (TKIs) nilotinib
and dasatinib are associated with higher rates of sustained deep molecular response
(
SDMR) compared to imatinib, the later remains as frontline therapy for chronic
myeloid leukaemia (CML) in Brazil due to lower prices of generic formulations.
However, as treatment free remission (TFR) became feasible for CML patients, second
generation TKIs might be a valuable option for frontline therapy since SDMR is the
main eligibility criterion for treatment suspension. This study compared the cost-
effectiveness of first and second generation TKIs as frontline CML treatment
considering TRF as a possibility. Methods: We constructed a Markov model to
compare the ten year cost-effectiveness of imatinib frontline therapy versus nilotinib
or dasatinib from a Brazilian third payer perspective, assuming 5% annual dis-
counting. The model included six health states: first line treatment, second line
treatment, TFR, treatment resume, disease progression and death. Transition prob-
abilities were obtained from eligible studies. Only direct medical costs were
considered including those related to drugs acquisition, medical visits and laboratory
tests (exchange rate 1 BRL = 4 USD). The main outcome was progression-free life-
years (PF-LY). We assessed different price scenarios for second generation TKIs.
Results: Nilotinib-first strategy offered 0.85 additional PF-LY with an ICER of US$
29,541.98/PF-LY compared to imatinib. Dasatinib frontline was the most costly
PCN129
strategy with an ICER of US$ 81,355.07/PF-LY. These results were robust to proba-
bilistic sensitivity analysis. The scenarios analysis showed that a 25% reduction in
nilotinib price would make it the dominant strategy. Conclusions: Although TFR was
included as a health state in the model, imatinib remained as the most economically
advantageous frontline CML treatment, mainly because of the low price of its generic
formulations. However, a decrease in the price of nilotinib could significantly change
this scenario.
A COST-UTILITY AND BUDGET IMPACT ANALYSIS OF
ENZALUTAMIDE FOR THE TREATMENT OF
NONMETASTATIC CASTRATION-RESISTANT PROSTATE
CANCER (NMCRPC) IN MEXICO
1
1
2
3
Toro W, Braun S, Sanchez LA, Anaya P
1
2
Astellas Pharma Inc., Northbrook, IL, USA, IQVIA, Mexico City, DF, Mexico,
IQVIA, Falls Church, VA, USA
3
Degirmenci, Volkan
