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Table 1 Model input parameters and data sources*

From: Cost-utility analysis of pralatrexate for relapsed or refractory peripheral T-cell lymphoma based on a case-matched historical control study along with single arm clinical trial

Parameters

Base-case values

Data source

Number of treatment cycles

 Initial treatment: pralatrexate

2 cycles with a 7-week cycle

PROPEL study [6]

 Initial tx.: CC

4.67 cycles with a 3-week cycle

Clinician panel survey

 Subsequent tx.: CC

4.33 cycles with a 3-week cycle

Clinician panel survey

Transition probability

Type of distribution used to extrapolate survival curves

 Pralatrexate

Generalized gamma

O’Connor et al. [7]

 CC

Gompertz

O’Connor et al. [7]

Response rate

 Initial treatment: pralatrexate

 

PROPEL study [6]

 CR

0.126

 PR

0.211

 SD

0.221

 PD

0.442

 Initial tx.: CC

 

Estimation based on

 CR

0.198

Korean subjects [7]

 PR

0.214

 SD

0.032

 PD

0.556

 Subsequent tx.: CC

 

Estimation based on

 CR

0.123

Korean subjects [7]

 PR

0.158

 SD

0.035

 PD

0.684

SCT rate following initial tx.

0.529

Clinician panel survey

SCT success rate following initial tx.

0.663

Kim et al. [9]

SCT rate following subsequent tx.

0.458

Clinician panel survey

SCT success rate following subseq. tx.

0.663

Kim et al. [9]

Probability of AE

 Mucositis (ICD-10 codes: K123)

 

PROPEL study [6]

 Initial tx.: pralatrexate

0.0172

Crump et al. [10];

 Initial tx.: CC

0.0365

Jerkeman et al. [11];

 Subsequent tx.: CC

0.0393

Wang et al. [12];

 Thrombocytopenia (D695)

 

Velasquez et al. [13];

 Initial tx.: pralatrexate

0.0267

Ezzat et al. [14];

 Initial tx.: CC

0.1122

Press et al. [15]

 Subsequent tx.: CC

0.0839

 

 Anemia (D611, D630)

 

 Initial tx.: pralatrexate

0.0118

 Initial tx.: CC

0.0257

 Subsequent tx.: CC

0.0279

 Neutropenia (D70)

 

 Initial tx.: pralatrexate

0.0172

 Initial tx.: conventional chemo.

0.1250

 Subsequent tx.: conventional chemo.

0.1011

 Nausea/vomiting (R11)

 

 Initial tx.: pralatrexate

0.0026

 Initial tx.: conventional chemo.

0.0053

 Subsequent tx.: conventional chemo.

0.0070

 Peripheral neuropathy (G900)

 

 Initial tx.: pralatrexate

0.0000

 Initial tx.: conventional chemo.

0.0063

 Subsequent tx.: conventional chemo.

0.0053

Costs1 (US dollars)

 Medication cost2

 Initial tx.: pralatrexate

$2465

Manufacturer’s suggesting price

 Initial tx.: CC

$240

Maximum Payable Amount Table of

 Subseq. tx.: CC

$238

Concomitant drug cost

 

Korean NHI, Clinician panel survey

 Initial tx.: pralatrexate

 

 First cycle, last cycle

$4, $3

Initial tx.: CC

$207

 

Subseq. tx.: CC

$198

 

SCT cost

$27,343

HIRA-NPS data (2011–2016), KHSCTA, KMDP

Routine monitoring test cost

 

Maximum Payable Amount Table of

 Initial tx.: pralatrexate

 

 First cycle, other cycles

$106, $17

Korean NHI, Clinician panel survey

 Initial tx.: CC

 

 First cycle, other cycles

$129, $40

 Subseq. tx.: CC

 

 First cycle, other cycles

$123, $38

Costs to treat AE3

 

Korean NHI healthcare

statistics

 Mucositis

$1288

 Thrombocytopenia

$2859

 Anemia

$2877

 Neutropenia

$2760

 Nausea/vomiting

$707

 Peripheral neuropathy

$643

Utility (disutility)

 CR

0.885

Kang et al. [16]

 PR

0.784

 SD

0.746

 PD

0.567

SCT success4: 45–49, 50–54, 55–59, 60–64 years old

0.976, 0.971, 0.966, 0.936

KNHANES (2014)

Mucositis

(−0.075)

Kang et al. [16]

Thrombocytopenia

(−0.095)

Kang et al. [16]

Anemia

(−0.085)

Kang et al. [16]

Neutropenia

(− 0.107)

Kang et al. [16]

Nausea/vomiting

(− 0.059)

Nafees et al. [17]

Peripheral neuropathy

(−0.42)

Swinburn et al. [18]

  1. AE Adverse event; CC Conventional chemotherapy; CR Complete response; HIRA-NPS Health Insurance Review and Assessment Service-National Patients Sample; ICD-10 code International Classification of Disease code 10th edition; KHSCTA Korean Hematopoietic Stem Cell Transplantation Association; KMDP Korea Marrow Donor Program; KNHANES Korea National Health and Nutrition Examination Survey; NHI National Health Insurance; PD Progressive disease; PR Partial response; SD Stable disease; SCT Stem cell transplantation
  2. 1. All costs are presented as weekly costs in 2019 US dollars and were estimated from a societal perspective, including medical costs, transportation costs to visit health care institutions, and care giver’s costs for hospitalization. 2. Medication costs include drug costs and drug administration costs, such as costs of outpatient visits, hospitalization, medication management, aseptic preparation, and injections. 3. Since all the adverse events (AEs) included are grade 3 or higher and require hospitalization, the cost to treat each AE was measured as the average cost per hospitalization with the condition. 4. Utility during the post-SCT health states (i.e., SCT success state) was assumed to be the same as that of the general population, which is the average utility value for the general population aged 45 to 65 years obtained from 2014 KNHANES data