Journal of Medical Economics

ISSN: 1369-6998 (Print) 1941-837X (Online) Journal homepage: https://www.tandfonline.com/loi/ijme20

Cost-effectiveness analysis of telotristat ethyl

for treatment of carcinoid syndrome diarrhea
inadequately controlled with somatostatin
analogs

V. N. Joish, F. Frech & P. Lapuerta

To cite this article: V. N. Joish, F. Frech & P. Lapuerta (2018) Cost-effectiveness

analysis of telotristat ethyl for treatment of carcinoid syndrome diarrhea inadequately
controlled with somatostatin analogs, Journal of Medical Economics, 21:2, 182-188, DOI:
10.1080/13696998.2017.1387120

To link to this article: https://doi.org/10.1080/13696998.2017.1387120

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JOURNAL OF MEDICAL ECONOMICS, 2018
VOL. 21, NO. 2, 182–188
https://doi.org/10.1080/13696998.2017.1387120
Article 0156-FT.R1/1387120
All rights reserved: reproduction in whole or part not permitted

ORIGINAL RESEARCH

Cost-effectiveness analysis of telotristat ethyl for treatment of carcinoid

syndrome diarrhea inadequately controlled with somatostatin analogs
V. N. Joisha, F. Frechb and P. Lapuertaa
a
Lexicon Pharmaceuticals, Inc., Basking Ridge, NJ, USA; bFormer Employee of Lexicon Pharmaceuticals, Basking Ridge, NJ, USA

ABSTRACT ARTICLE HISTORY

Aims: This study evaluated the cost-effectiveness of telotristat ethyl (TE) added to somatostatin analog Received 22 August 2017
octreotide (SSA þ TE) compared to octreotide alone (SSA) in patients with carcinoid syndrome diarrhea Revised 20 September 2017
(CSD) whose symptoms remain uncontrolled with SSA alone. Accepted 27 September 2017
Materials and methods: A deterministic Markov model evaluated the costs and quality-adjusted life-
KEYWORDS
years (QALY) gained with SSA þ TE vs SSA per a third-party US payer perspective. The model reflected Carcinoid syndrome; cost-
clinical practice and resource use estimates based on current standards of care, with utility estimates effectiveness; telotristat
based on similar symptoms from ulcerative colitis. Treatment efficacy was based on the phase III clin- ethyl; neuroendocrine
ical trial of SSA þ TE vs SSA alone [TELESTAR, NCT01677910]. According to TELESTAR, 44% of SSA þ TE tumors; diarrhea;
and 20% of SSA patients responded to therapy after 12 weeks. At each 4-week assessment period, SSA somatostatin analogs
patients not adequately controlled received increasing doses of SSA and SSA þ TE patients discontin-
ued TE and moved to SSA only. Drug costs for adequately and not adequately controlled patients
were $4,291.75 and $5,890.57 for SSA, respectively, and $9,456.07 and $5,890.57 for SSA þ TE,
respectively.
Results: The base-case analysis demonstrated lifetime QALYs of 1.67 at a cost of $495,125 for the SSA
cohort and 2.33 ($590,087) for SSA þ TE with an incremental QALY for SSA þ TE of 0.66 for an add-
itional $94,962. The incremental cost per QALY gained was $142,545. Sensitivity analyses demonstrated
high probability (>99%) of SSA þ TE being cost-effective at thresholds for rare diseases and orphan
drugs of $300,000–$450,000.
Limitations: The recent availability of TE precluded the incorporation of clinical and economic inputs
based on real-world practice patterns. The scarcity of epidemiology and utility information for this rare
condition required the use of some proxy estimates.
Conclusions: This analysis demonstrated TE is a cost-effective treatment option when used on top of
standard of care in CSD patients.

Introduction with CSD require more hospitalizations, office visits, and

Carcinoid syndrome (CS) is known to develop in patients
with neuroendocrine tumors (NETs), caused by tumoral secre-
tion of vasoactive amines and peptides, and is associated
with diarrhea, abdominal pain, vasomotor symptoms, bron-
choconstriction, and complications such as carcinoid heart
disease1. The prevalence of NETs in the US is 103,0002, of
whom 63,000 (61%) are estimated to have gastroentero-
pancreatic tumors3. Approximately 10–20% of these patients
have been reported to have functioning tumors, often lead-
ing to CS4,5. The US FDA defines rare disease as having a
prevalence <200,0006. Despite the limited availability of pre-
cise epidemiological data for CS diarrhea (CSD), the extrapo-
lated prevalence of CSD must be far smaller than this
threshold. The age-adjusted incidence of carcinoid tumors is
estimated to be 1.9 per 100,000 persons in the US, for both
men and women7, representing 0.5% of newly-diagnosed
tumors8. This places the incidence of CS well below 1.9 per
100,000 persons in the US, with specific complications such
as CSD in an even smaller sub-set of the population. Patients
higher healthcare costs than those without diarrhea, high-
lighting the deleterious effects of this rare condition9.
The National Comprehensive Cancer Network guidelines
recommends the somatostatin analogs (SSA) octreotide and
lanreotide for treatment of CSD10. Daily subcutaneous injec-
tions of octreotide (100–600 mcg, mean daily dosage
300 mcg) are approved for use in the US in 2–4 divided
doses for 2 weeks, which may be followed by the long-acting
release (LAR) suspension formulation (20 mg every 4
weeks)11,12. The SSAs were designed to block the release of
vasoactive peptides and amines, but patients may develop
reduced responsiveness to SSA therapy and experience sub-
sequent breakthrough symptoms13,14.
Telotristat ethyl (XermeloTM, Lexicon Pharmaceuticals,
Basking Ridge, NJ) is a novel oral small-molecule tryptophan
hydroxylase (TPH) inhibitor with a high molecular weight and
acidic moieties that keep it from crossing the blood–brain
barrier that is approved in the US for the treatment of CSD
in combination with SSA therapy in adults inadequately

CONTACT V. N. Joish vjoish@lexpharma.com Lexicon Pharmaceuticals, Inc., 110 Allen Road, Basking Ridge, NJ 07920, USA
ß 2017 Informa UK Limited, trading as Taylor & Francis Group
www.informahealthcare.com/jme

controlled by SSA therapy15–17. The TELESTAR phase III
randomized, controlled clinical trial (ClinicalTrials.gov number
NCT01677910) demonstrated significant reductions in bowel
movement (BM) frequency and urinary 5-hydroxyindole acetic
acid (u5-HIAA) among patients with CSD not adequately con-
trolled by SSA therapy alone18. Telotristat ethyl, in addition
to SSA therapy, was shown to be generally safe and well-tol-
erated through an open-label extension study showing a sus-
tained effect on bowel movement control18.
Advancements in the management of diseases and associ-
ated complications have seen closer discrimination and inter-
pretation of value in therapeutic decisions. Leading oncology
centers, in particular, have adopted value frameworks to sup-
port evidence-based coverage decisions. More than 70% of
US-based managed care medical and pharmacy directors
recently indicated the use of value frameworks to inform
decision-making related to oncology therapeutic manage-
ment19. Rigorous assessment of cost-effectiveness using such
value frameworks is central to understanding the most effi-
cient use of new health technologies. Rare diseases with
orphan drug treatment options are known to carry special
considerations in health technology assessment and cost-
effectiveness analysis20,21.
The National Institute for Health and Clinical Excellence
(NICE) Highly Specialized Technologies (HST) program evalu-
ates new technologies for rare diseases, and has adapted
some of its cost-effectiveness calculation assumptions for
orphan drug treatments21. The Institute for Clinical and
Economic Review (ICER) recently proposed an assessment
framework for “ultra-rare” conditions with 10,000 patients
per year, or 3.0 per 100,00022. ICER maintains the evidence
standard for ultra-rare conditions, but acknowledges the chal-
lenge of evidence generation in this population, and extends
the upper bound of the willingness-to-pay (WTP) threshold
to $500,000 per quality-adjusted life-year (QALY)22.
The objective of this study was to evaluate the cost-effect-
iveness of telotristat ethyl with SSA (SSA þ TE) compared to
SSA alone among patients with CSD whose symptoms
remain uncontrolled with SSA therapy alone.
Methods
Model overview
A deterministic Markov model was constructed to evaluate
the costs and QALY gains associated with SSA þ TE vs SSA
treatment in patients with CSD uncontrolled with initial SSA
therapy over a lifetime time horizon according to a third-
party US payer perspective.
The model included three health states: Adequate Control;
Control Not Adequate; and Dead. The model simulation
extrapolated costs and patient outcomes from health states
observed in the TELESTAR trial. The model utilized the pri-
mary end-point definition of treatment response from the
TELESTAR trial of 30% reduction in daily BM for 50% of
the time over 12 weeks18.
Patients maintaining Adequate Control on SSA þ TE
throughout the simulation continued therapy until study end
or death. Patients receiving SSA who did not achieve
COST-EFFECTIVENESS ANALYSIS OF TE WITH SSA 183
treatment response were assumed to receive increasing doses
of octreotide until loss of response and treatment discontinu-
ation or death. SSA þ TE patients who did not achieve
Adequate Control were considered to discontinue TE treat-
ment and receive SSA therapy only with increased dosing,
mirroring the increased dosing of non-responders in the SSA
cohort. As a rule, drug therapy for symptomatic conditions
such as carcinoid syndrome is continued in patients who con-
tinue to derive tangible benefit, and otherwise discontinued
when no benefit is evident or there are toxicity concerns. This
assumption is consistent with health economic analyses in
immunology, and has been demonstrated in the case of
ulcerative colitis23. A schematic overview of the included
health states and transitions is presented in Figure 1.
The structure of the model was designed to reflect current
clinical practice for patients receiving SSA therapy and uti-
lized SSA þ TE treatment efficacy based on the TELESTAR
trial. Patients with adequate response to initial SSA þ TE
treatment remained in this state through the simulation or
until death, according to the durability of SSA þ TE treatment
response demonstrated in the open-label extension of the
TELESTAR trial18. A proportion of patients receiving SSA with
an initially adequate response to treatment experienced a
loss of response to octreotide, as reflected in clinical experi-
ence such as that reported by Toumpanakis et al.24. Patients
experiencing inadequate control with SSA received dose
escalations in line with standard clinical practice to maintain
SSA therapy beyond recommended dosing levels, despite
non-response25. Approximately 40% of carcinoid patients are
reported to receive SSA above the indicated dosing fre-
quency or 30 mg maximum dose in efforts to manage uncon-
trolled symptoms, tumor progression, elevated u5-HIAA
levels, or other factors26.
Transition probabilities
The base case analysis placed 44% of SSA þ TE and 20% of
SSA patients in the Adequate Control state according to the
12-week TELESTAR responder analysis18. Patients in the SSA
cohort were assumed to move into the Not Adequate
Control state at a constant rate of 0.01 every 4 weeks, based
on Toumpanakis et al.’s24 report of 3-month probability of
inadequate control with octreotide therapy.
The probability of death in this cohort was calculated
using u5-HIAA as a predictor of mortality, based on an ana-
lysis in patients with mid-gut carcinoid tumors27. This regres-
sion model was used to predict the probability of death for
each cycle, health state, and comparator, assumed to esti-
mate mortality rates for an individual with age and u5-HIAA
level equivalent to the mean values reported in TELESTAR.
Age-specific mortality rates were estimated and averaged
according to the age distribution of TELESTAR patients. The
12-week TELESTAR u5-HIAA levels were applied to model
cohorts where SSA patients were assigned a mean of
54.83 mmol/mol creatinine and SSA þ TE patients a mean of
36.27 mmol/mol18. Since serious adverse events and related
discontinuations were similar among TELESTAR treatment
groups and were not expected to significantly impact
184 V. N. JOISH ET AL.

Figure 1. Markov model health states and patient transitions. Abbreviations. SSA, somatostatin analog; TE, telotristat ethyl.

Table 1. Base-case resource utilization and treatment input costs.

Intervention Adequate control cost Not adequate control cost
Resource utilization
Outpatient visit $650.39 $1,128.19
Outpatient pharmacy $176.92 $597.25
Emergency department visit $11.68 $13.85
Inpatient stay $887.49 $1,464.32
Other medical encounters $74.38 $156.95
Total resource use costs $1,800.86 $3,360.56
Treatments
SSA, octreotide (24.32 mg average monthly dose) $4,291.75 $5,890.57
Telotristat ethyl (21,000 mg average monthly dose) $5,164.32 $5,164.32
SSA þ TE $9,456.07 $5,890.57 (discontinued TE, SSA only)
Outpatient visit cost does not include SSA physician service and drug costs in order to avoid duplication of SSA costs in the analysis.

quality-of-life or healthcare costs, adverse event-related
health states and transitions were not included in this
analysis.
Costs
All costs were based on an analysis of administrative claims
from a large US health insurance plan including members
with carcinoid syndrome from both commercial and
Medicare Advantage plans28. Evidence of SSA dose escalation
was used to determine SSA treatment responsiveness and
subsequent healthcare resource utilization according to the
Burton and Lapuerta28 analysis. Resource use estimates,
excluding drug costs, were assumed to be identical for
SSA þ TE and SSA patients with adequate or inadequate
response to treatment (Table 1).
Drug costs for SSA (octreotide LAR) were based on the
average monthly dose identified in the analysis conducted
by Burton and Lapuerta28 for adequately controlled
(24.32 mg) and not adequately controlled (33.38 mg) patients.
In the absence of information on the impact of telotristat
ethyl on octreotide dosing, it was estimated that SSA dosing
was equivalent for all adequately controlled SSA and
SSA þ TE patients. The recommended dose for telotristat
ethyl is 250 mg, three times daily17. Patients continuing
SSA þ TE treatment were assumed to remain persistent and,
thus, require the full cost of therapy in that cycle. The unit
cost of octreotide was $176.47, based on the lowest calcu-
lated price per milligram, according to the published whole-
sale acquisition cost (Optum, EncoderPro, February 2007).
The cost of telotristat ethyl was $61.48 per 250-mg unit, or
$5,164 per 4-week cycle.
Utility
Overall, the frequency of CSD-related BM and number of
flushing episodes have been associated with lower health-
related quality-of-life among patients with NETs29; however,
utility estimates based on controlled vs uncontrolled symp-
toms have not been published. This analysis used ulcerative
colitis as a proxy for estimates of active disease and remis-
sion states with controlled or uncontrolled CSD. Utility esti-
mates for these states were based on a time trade-off study
of ulcerative colitis patients in the US yielding proxy values
of 0.32 and 0.79 for uncontrolled and controlled carcinoid
syndrome, respectively30. Similar to that of CSD, the
 COST-EFFECTIVENESS ANALYSIS OF TE WITH SSA 185

Table 2. Sensitivity analysis parameters for the cost-effectiveness analysis.

Parameter Base case value Low value High value Probabilistic sensitivity analysis
Distribution SD of the distribution
Costs
Ambulatory visit costs (AC) $650.39 $520.31 $780.47 Gamma $44.45
Emergency department visits (AC) $11.68 $9.34 $14.02 Gamma $3.38
Inpatient stays (AC) $887.49 $709.99 $1,064.99 Gamma $130.12
Other medical (AC) $74.38 $59.50 $89.26 Gamma $25.80
Outpatient pharmacy (AC) $176.92 $141.54 $212.30 Gamma $36.82
Ambulatory visit costs (CNA) $1,128.19 $902.55 $1,353.83 Gamma $47.76
Emergency department visits (CNA) $13.85 $11.08 $16.62 Gamma $2.11
Inpatient stays (CNA) $1,464.32 $1,171.46 $1,757.18 Gamma $296.66
Other medical (CNA) $156.95 $125.56 $188.34 Gamma $63.70
Outpatient pharmacy (CNA) $597.25 $477.80 $716.70 Gamma $73.26
SSA, octreotide LAR depot $176.47 $141.18 $211.76 Gamma $17.65
Average SSA dose
SSA only group (AC) 24.32 19.5 29.2 Normal 2.4
SSA only group (CNA) 33.38 26.7 40.1 Normal 3.3
SSA þ TE group (AC) 24.32 19.5 29.2 Normal 2.4
SSA þ TE group (CNA) 33.38 26.7 40.1 Normal 3.3
Treatment efficacy (12 weeks)
SSA patients with AC at end of trial 20.0% 16.0% 24.0% Beta 0.02
SSA þ TE patients with AC at end of trial 44.0% 35.2% 52.8% Beta 0.04
Transition probability (4 weeks) from AC to CNA 1.0% 0.8% 1.2% Beta 0.1%
Long-Term probability of mortality (every 4 weeks) 0.013 0.010 0.015 – –
Utility
Utility (AC) 0.79 0.63 0.95 Beta 0.08
Utility (CNA) 0.32 0.24 0.36 Beta 0.03
Abbreviations. AC, adequate control; CNA, control not adequate; SSA, octreotide; SSA þ TE, octreotide and telotristat ethyl.

symptomatology of ulcerative colitis includes diarrhea,
abdominal pain, urgency, weight loss, fever, and pain, and
treatment goals are to relieve symptom severity in the short-
term and avoid symptom flares in the long-term31. The US
Food and Drug Administration’s guidance on clinical trial
endpoints for ulcerative colitis emphasizes the value of
patient-reported daily bowel movement frequency31, as was
the primary efficacy endpoint of the TELESTAR trial.
Untreated patients in the TELESTAR trial had 5.2–6.1 mean
daily bowel movements at baseline, similar to stool fre-
quency reported for untreated ulcerative colitis patients of
4–5 per day32, or where >90% have had one or more bowel
movements than normal per day33. The severity of ulcerative
colitis related to bowel movement frequency includes a simi-
lar range, where mild disease may be indicated by <4/day,
4–6/day moderate disease, and >6/day severe disease, based
on the presence of other symptoms34. The similarity of symp-
tomatology and expected response to treatment in the form
of reduced bowel movement frequency between ulcerative
colitis and CSD offered the closest proxy condition for this
analysis.
Cost-effectiveness analysis
This analysis was conducted for patients using a lifetime time
horizon to evaluate LY gains, lifetime costs, QALY gains, and
incremental cost effectiveness ratios based on costs per
QALY gained. A 3% discount rate was applied to costs and
outcomes occurring beyond 1 year, based on common health
economic analysis practices in the US35. A WTP threshold of
$150,000 per QALY gained was used as a benchmark for
cost-effectiveness, based on the most recent ICER Value
Assessment Framework cost-effectiveness threshold range36.
Probabilistic sensitivity analyses (PSA) were conducted with
WTP thresholds more closely aligned with value frameworks
used with orphan drugs for rare and ultra-rare condi-
tions21,22. Regarding the distributional assumptions of varia-
bles that entered the PSA, the gamma distribution was used
for all cost-related inputs that tended to be right-skewed.
The average SSA doses in the SSA and SSA þ TE arms were
assumed to follow a normal distribution. The beta distribu-
tion was used for those model inputs whose values were lim-
ited to a range of 0–1, such as probability and utility inputs.
These analyses considered thresholds 2–3-times higher than
the conventional value, in the range of $300,000–$450,000
per QALY gained. To assess the impact of base-case model
assumptions, further sensitivity analyses were conducted
related to resource utilization costs, treatment dosing and
costs, and utility estimates (Table 2).
Results
Cost-effectiveness analysis
The base case analysis according to a conventional disease-
intervention value framework provided lifetime QALYs of 1.67
at a cost of $495,125 for the SSA cohort and 2.33 ($590,087)
for SSA þ TE, with an incremental QALY for SSA þ TE of 0.66
at an additional cost of $94,962. The incremental cost per
QALY gained was $142,545, making the telotristat ethyl-con-
taining regimen cost-effective within the conventional value
framework WTP threshold of $150,000 per QALY.
Sensitivity analyses
When considering WTP thresholds more appropriate to a rare
or ultra-rare disease value framework, the probability of
186 V. N. JOISH ET AL.
Figure 2. Cost-effectiveness acceptability curve. Abbreviations. SSA, somatostatin analog; TE, telotristat ethyl; WTP, willingness to pay.
SSA þ TE being cost-effective was high (Figure 2)21,22. At a
2-fold WTP threshold of $300,000, the probability of SSA þ TE
being cost-effective was 99.1%, and at a 3-fold threshold of
$450,000 it was 99.9%, compared to 57.2% at the conven-
tional disease-intervention threshold of $150,000. One-way
sensitivity analyses, which rank model inputs by their impact
on the incremental cost-effectiveness ratio, showed the
model was most sensitive to the utility value of patients
achieving adequate control (Figure 3) and, to a lesser degree,
to the utility value assigned to inadequate control. The incre-
mental cost-effectiveness ratio was also sensitive to the num-
ber of SSA units administered for patients not achieving
adequate control, SSA þ TE units administered, and patient
responsiveness to treatment (probability of achieving
adequate control for each regimen).
Discussion
The TPH inhibitor telotristat ethyl has demonstrated clinically
meaningful reductions in BM frequency and u5-HIAA in
patients with CSD not achieving adequate response to SSA
therapy alone18. The efficacy of telotristat ethyl in reducing
daily BM was sustained with no new safety events in an
open-label extension trial18. In the reality of limited resources
and high demand for quality care with meaningful health
outcomes, healthcare decision-makers must contemplate the
efficiency of health technology innovations in addition to
their clinical benefits and risks. The prevalence and impact of
disease are central to such assessments. This analysis demon-
strated the cost-effectiveness of telotristat ethyl for patients
with carcinoid syndrome not adequately controlled with SSA
therapy alone according to both conventional and rare
disease value assessment frameworks. The cost-effectiveness
of the SSA þ TE regimen was largely driven by improved
symptom reduction and quality-of-life demonstrated in the
phase III clinical trial program.
The prevalence of NETs is 100,000 in the US2.
Approximately 19% of patients with NETs have CS, and a
small fraction of CS patients have associated diarrhea5.
Although rare, the occurrence of CS continues to increase1,2,5.
The occurrence of diarrhea, as measured by daily bowel
movements for patients with carcinoid syndrome, is
prominent and costly, as patients tend to be of working age
(mean ¼ 51.5 years) with comorbidities9. CS patients with
diarrhea have a nearly 50% greater odds of any hospitaliza-
tion and 112% greater odds of carcinoid syndrome-related
hospitalization, incurring $27,334 more in medical costs than
matched counterparts without diarrhea9. Unfortunately,
nearly half (40%) of the patients given the historical standard
of care for CSD do not achieve adequate response, leaving
significant medical and health economic unmet needs for
this rare condition28.
The interpretation of value in rare conditions with fre-
quently occurring, expensive complications requires consider-
ation of the impact on patients’ quality-of-life and survival, in
addition to the efficiency of such meaningful improvements.
The base-case incremental cost per QALY gained with telotri-
stat ethyl of $142,545 was within the ICER threshold of
$150,000 for conventional disease assessments, with a 57%
probability of cost-effectiveness at this threshold. This ana-
lysis further demonstrated a very high probability (>99%) of
cost-effectiveness with telotristat ethyl treatment within WTP
thresholds more suited to value frameworks associated
with rare and ultra-rare conditions of $300,000–$450,000.

Figure 3. One-way sensitivity analysis results. Abbreviations. AC, adequate control; CNA, control not adequate; SSA, somatostatin analog; TE, telotristat ethyl.
Since the model was most sensitive to the utility of patients
achieving adequate control, which was derived from the pub-
lished literature for ulcerative colitis, we varied this estimate
in the sensitivity analyses and in threshold analyses. This
allowed us to determine the minimum incremental utility
required for TE to be a cost-effective treatment option, in
addition to standard of care SSA therapy. The incremental
utility values of >0.44, > 0.21, or >0.14 were required for the
SSA þ TE to be cost-effective at $150,000, $300,000, and
$450,000 thresholds.
This cost-effectiveness analysis must be interpreted with
certain considerations. It should be noted that the recent
availability of telotristat ethyl has precluded a longer histor-
ical calculation of real-world practice patterns. The exact
impact on healthcare resource utilization of telotristat ethyl,
which may differ from that of SSA, cannot yet be quantified.
The lack of published utility estimates in this rare condition
required the use of proxy estimates from the case of patients
with ulcerative colitis, which held notable influence on the
sensitivity of results. Even though the disease symptomatol-
ogy, burden, and treatment expectations are similar, further
research on the utility associated with CSD specifically is
needed. The treatment response threshold of 30% reduc-
tion in daily BM frequency was determined to be clinically
significant from interviews with TELESTAR participants37,38;
however, we were not able to test other response thresholds
in this analysis, which may be considered in future research.
Furthermore, the present analysis is US-specific and, given
the variability in practice patterns, SSA dosing, and drug
acquisition costs, the findings should not be generalized to
healthcare settings in other countries. Local adaptations of
this model may be an interesting topic for future research.
This cost-effectiveness analysis was based on the results
of the first and only randomized controlled double-blind
COST-EFFECTIVENESS ANALYSIS OF TE WITH SSA 187
clinical trial for CSD, in which telotristat ethyl demonstrated
clinically meaningful improvements in key markers of disease.
Response to therapy for CSD can be subjective and defined
in several ways; however, this analysis applied the pre-speci-
fied definition of disease response used in the telotristat
ethyl clinical trial program.
Conclusions
This analysis demonstrated that the addition of telotristat
ethyl is cost-effective when given to patients with CSD not
adequately controlled with somatostatin analogs.
Transparency
Declaration of funding
This study was sponsored by Lexicon Pharmaceuticals, manufacturer of
telotristat ethyl, which is approved for the treatment of carcinoid syn-
drome diarrhea.
Declaration of financial/other interests
VNJ and PL are employees of Lexicon Pharmaceuticals. JF provided writ-
ing support, which was funded by Lexicon Pharmaceuticals. MK and MM
are employees of Optum and were hired as consultants by Lexicon
Pharmaceuticals.
Acknowledgments
The authors wish to acknowledge Jeff Frimpter, MPH, and
Communications Symmetry for medical writing support, which was
funded by Lexicon Pharmaceuticals. The authors also wish to acknow-
ledge Michele Kohli and Michael Maschio for their technical input on
this economic model.
188 V. N. JOISH ET AL.
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