Romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura

Graham Mowatt, Charles Boachie, Mark Crowther, Cynthia Mary Fraser, Rodolfo Andres Hernandez, Xueli Jia, Laura Ternent

Research output: Contribution to journalArticle

5 Citations (Scopus)
4 Downloads (Pure)

Abstract

This paper presents a summary of the evidence review group (ERG) report into the clinical and cost-effectiveness of romiplostim for the treatment of adults with chronic immune or idiopathic thrombocytopenic purpura (ITP) based upon a review of the manufacturer's submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The Submission's evidence came from two relatively high-quality randomised controlled trials (RCTs). The ERG found no evidence that any important data were missed or that data extraction was inaccurate. In both RCTs more patients in the romiplostim than in the placebo group achieved a durable platelet response [non-splenectomised patients: romiplostim 25/41 (61%), placebo 1/21 (5%), odds ratio (OR) 24.45, 95% confidence interval (CI) 3.34 to 179.18; splenectomised patients: romiplostim 16/42 (38%), placebo 0/21 (0%), OR 8.5 (95% CI 1.15 to 372)] and an overall platelet response [non-splenectomised patients: romiplostim 36/41 (88%), placebo 3/21 (14%), OR 34.74, 95% CI 7.77 to 1.55.38; splenectomised patients: romiplostim 33/42 (79%), placebo 0/21 (0%), OR 16.6 (95% CI 2.37 to 706]. The difference in mean period with a platelet response was 13.9 weeks (95% CI 10.5 to 17.4) in favour of romiplostim in the RCT of non-splectomised patients and 12.1 weeks (95% CI 8.7 to 15.6) in favour of romiplostim in the RCT of splectomised patients. The manufacturer's economic model evaluated the cost-effectiveness of romiplostim compared with standard care. The ERG had concerns about the way the decision problem was addressed in the economic model and about the non-adjustment of findings for confounding factors. In non-splenectomised patients, using romiplostim as a first option treatment, the base-case incremental cost-effectiveness ratio (ICER) was 14,840 pound per quality-adjusted life-year (QALY). In splenectomised patients the ICER was 14,655 pound per QALY. Additional sensitivity analyses performed by the ERG identified two issues of importance: whether individuals entered the model on watch and rescue or on active therapy in the comparator arm (ICER 21,674 pound per QALY for non-splenectomised patients, 29,771 pound per QALY for splenectomised patients); whether it was assumed that any unused medicine would be wasted. Combining all of the separate sensitivity analyses, and assuming that watch and rescue was not the first-line treatment, increased the ICERs further (non-splenectomised 37,290 pound per QALY, splenectomised 131,017 pound per QALY). In conclusion, the manufacturer's submission and additional work conducted by the ERG suggest that romiplostim has short-term efficacy for the treatment of ITP, but there is no robust evidence on long-term effectiveness or cost-effectiveness of romiplostim compared with relevant comparators.

Original languageEnglish
Pages (from-to)63-68
Number of pages6
JournalHealth Technology Assessment
Volume13
Issue numberSuppl 2
DOIs
Publication statusPublished - Sep 2009

Cite this

Mowatt, G., Boachie, C., Crowther, M., Fraser, C. M., Hernandez, R. A., Jia, X., & Ternent, L. (2009). Romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura. Health Technology Assessment, 13(Suppl 2), 63-68. https://doi.org/10.3310/hta13suppl2/09

Romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura. / Mowatt, Graham; Boachie, Charles; Crowther, Mark; Fraser, Cynthia Mary; Hernandez, Rodolfo Andres; Jia, Xueli; Ternent, Laura.

In: Health Technology Assessment, Vol. 13, No. Suppl 2, 09.2009, p. 63-68.

Research output: Contribution to journalArticle

Mowatt, G, Boachie, C, Crowther, M, Fraser, CM, Hernandez, RA, Jia, X & Ternent, L 2009, 'Romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura', Health Technology Assessment, vol. 13, no. Suppl 2, pp. 63-68. https://doi.org/10.3310/hta13suppl2/09
Mowatt G, Boachie C, Crowther M, Fraser CM, Hernandez RA, Jia X et al. Romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura. Health Technology Assessment. 2009 Sep;13(Suppl 2):63-68. https://doi.org/10.3310/hta13suppl2/09
Mowatt, Graham ; Boachie, Charles ; Crowther, Mark ; Fraser, Cynthia Mary ; Hernandez, Rodolfo Andres ; Jia, Xueli ; Ternent, Laura. / Romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura. In: Health Technology Assessment. 2009 ; Vol. 13, No. Suppl 2. pp. 63-68.
@article{00048cbfd45348e29a577fbf91a436e9,
title = "Romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura",
abstract = "This paper presents a summary of the evidence review group (ERG) report into the clinical and cost-effectiveness of romiplostim for the treatment of adults with chronic immune or idiopathic thrombocytopenic purpura (ITP) based upon a review of the manufacturer's submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The Submission's evidence came from two relatively high-quality randomised controlled trials (RCTs). The ERG found no evidence that any important data were missed or that data extraction was inaccurate. In both RCTs more patients in the romiplostim than in the placebo group achieved a durable platelet response [non-splenectomised patients: romiplostim 25/41 (61{\%}), placebo 1/21 (5{\%}), odds ratio (OR) 24.45, 95{\%} confidence interval (CI) 3.34 to 179.18; splenectomised patients: romiplostim 16/42 (38{\%}), placebo 0/21 (0{\%}), OR 8.5 (95{\%} CI 1.15 to 372)] and an overall platelet response [non-splenectomised patients: romiplostim 36/41 (88{\%}), placebo 3/21 (14{\%}), OR 34.74, 95{\%} CI 7.77 to 1.55.38; splenectomised patients: romiplostim 33/42 (79{\%}), placebo 0/21 (0{\%}), OR 16.6 (95{\%} CI 2.37 to 706]. The difference in mean period with a platelet response was 13.9 weeks (95{\%} CI 10.5 to 17.4) in favour of romiplostim in the RCT of non-splectomised patients and 12.1 weeks (95{\%} CI 8.7 to 15.6) in favour of romiplostim in the RCT of splectomised patients. The manufacturer's economic model evaluated the cost-effectiveness of romiplostim compared with standard care. The ERG had concerns about the way the decision problem was addressed in the economic model and about the non-adjustment of findings for confounding factors. In non-splenectomised patients, using romiplostim as a first option treatment, the base-case incremental cost-effectiveness ratio (ICER) was 14,840 pound per quality-adjusted life-year (QALY). In splenectomised patients the ICER was 14,655 pound per QALY. Additional sensitivity analyses performed by the ERG identified two issues of importance: whether individuals entered the model on watch and rescue or on active therapy in the comparator arm (ICER 21,674 pound per QALY for non-splenectomised patients, 29,771 pound per QALY for splenectomised patients); whether it was assumed that any unused medicine would be wasted. Combining all of the separate sensitivity analyses, and assuming that watch and rescue was not the first-line treatment, increased the ICERs further (non-splenectomised 37,290 pound per QALY, splenectomised 131,017 pound per QALY). In conclusion, the manufacturer's submission and additional work conducted by the ERG suggest that romiplostim has short-term efficacy for the treatment of ITP, but there is no robust evidence on long-term effectiveness or cost-effectiveness of romiplostim compared with relevant comparators.",
author = "Graham Mowatt and Charles Boachie and Mark Crowther and Fraser, {Cynthia Mary} and Hernandez, {Rodolfo Andres} and Xueli Jia and Laura Ternent",
year = "2009",
month = "9",
doi = "10.3310/hta13suppl2/09",
language = "English",
volume = "13",
pages = "63--68",
journal = "Health Technology Assessment",
issn = "1366-5278",
publisher = "National Co-ordinating Centre for HTA",
number = "Suppl 2",

}

TY - JOUR

T1 - Romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura

AU - Mowatt, Graham

AU - Boachie, Charles

AU - Crowther, Mark

AU - Fraser, Cynthia Mary

AU - Hernandez, Rodolfo Andres

AU - Jia, Xueli

AU - Ternent, Laura

PY - 2009/9

Y1 - 2009/9

N2 - This paper presents a summary of the evidence review group (ERG) report into the clinical and cost-effectiveness of romiplostim for the treatment of adults with chronic immune or idiopathic thrombocytopenic purpura (ITP) based upon a review of the manufacturer's submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The Submission's evidence came from two relatively high-quality randomised controlled trials (RCTs). The ERG found no evidence that any important data were missed or that data extraction was inaccurate. In both RCTs more patients in the romiplostim than in the placebo group achieved a durable platelet response [non-splenectomised patients: romiplostim 25/41 (61%), placebo 1/21 (5%), odds ratio (OR) 24.45, 95% confidence interval (CI) 3.34 to 179.18; splenectomised patients: romiplostim 16/42 (38%), placebo 0/21 (0%), OR 8.5 (95% CI 1.15 to 372)] and an overall platelet response [non-splenectomised patients: romiplostim 36/41 (88%), placebo 3/21 (14%), OR 34.74, 95% CI 7.77 to 1.55.38; splenectomised patients: romiplostim 33/42 (79%), placebo 0/21 (0%), OR 16.6 (95% CI 2.37 to 706]. The difference in mean period with a platelet response was 13.9 weeks (95% CI 10.5 to 17.4) in favour of romiplostim in the RCT of non-splectomised patients and 12.1 weeks (95% CI 8.7 to 15.6) in favour of romiplostim in the RCT of splectomised patients. The manufacturer's economic model evaluated the cost-effectiveness of romiplostim compared with standard care. The ERG had concerns about the way the decision problem was addressed in the economic model and about the non-adjustment of findings for confounding factors. In non-splenectomised patients, using romiplostim as a first option treatment, the base-case incremental cost-effectiveness ratio (ICER) was 14,840 pound per quality-adjusted life-year (QALY). In splenectomised patients the ICER was 14,655 pound per QALY. Additional sensitivity analyses performed by the ERG identified two issues of importance: whether individuals entered the model on watch and rescue or on active therapy in the comparator arm (ICER 21,674 pound per QALY for non-splenectomised patients, 29,771 pound per QALY for splenectomised patients); whether it was assumed that any unused medicine would be wasted. Combining all of the separate sensitivity analyses, and assuming that watch and rescue was not the first-line treatment, increased the ICERs further (non-splenectomised 37,290 pound per QALY, splenectomised 131,017 pound per QALY). In conclusion, the manufacturer's submission and additional work conducted by the ERG suggest that romiplostim has short-term efficacy for the treatment of ITP, but there is no robust evidence on long-term effectiveness or cost-effectiveness of romiplostim compared with relevant comparators.

AB - This paper presents a summary of the evidence review group (ERG) report into the clinical and cost-effectiveness of romiplostim for the treatment of adults with chronic immune or idiopathic thrombocytopenic purpura (ITP) based upon a review of the manufacturer's submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The Submission's evidence came from two relatively high-quality randomised controlled trials (RCTs). The ERG found no evidence that any important data were missed or that data extraction was inaccurate. In both RCTs more patients in the romiplostim than in the placebo group achieved a durable platelet response [non-splenectomised patients: romiplostim 25/41 (61%), placebo 1/21 (5%), odds ratio (OR) 24.45, 95% confidence interval (CI) 3.34 to 179.18; splenectomised patients: romiplostim 16/42 (38%), placebo 0/21 (0%), OR 8.5 (95% CI 1.15 to 372)] and an overall platelet response [non-splenectomised patients: romiplostim 36/41 (88%), placebo 3/21 (14%), OR 34.74, 95% CI 7.77 to 1.55.38; splenectomised patients: romiplostim 33/42 (79%), placebo 0/21 (0%), OR 16.6 (95% CI 2.37 to 706]. The difference in mean period with a platelet response was 13.9 weeks (95% CI 10.5 to 17.4) in favour of romiplostim in the RCT of non-splectomised patients and 12.1 weeks (95% CI 8.7 to 15.6) in favour of romiplostim in the RCT of splectomised patients. The manufacturer's economic model evaluated the cost-effectiveness of romiplostim compared with standard care. The ERG had concerns about the way the decision problem was addressed in the economic model and about the non-adjustment of findings for confounding factors. In non-splenectomised patients, using romiplostim as a first option treatment, the base-case incremental cost-effectiveness ratio (ICER) was 14,840 pound per quality-adjusted life-year (QALY). In splenectomised patients the ICER was 14,655 pound per QALY. Additional sensitivity analyses performed by the ERG identified two issues of importance: whether individuals entered the model on watch and rescue or on active therapy in the comparator arm (ICER 21,674 pound per QALY for non-splenectomised patients, 29,771 pound per QALY for splenectomised patients); whether it was assumed that any unused medicine would be wasted. Combining all of the separate sensitivity analyses, and assuming that watch and rescue was not the first-line treatment, increased the ICERs further (non-splenectomised 37,290 pound per QALY, splenectomised 131,017 pound per QALY). In conclusion, the manufacturer's submission and additional work conducted by the ERG suggest that romiplostim has short-term efficacy for the treatment of ITP, but there is no robust evidence on long-term effectiveness or cost-effectiveness of romiplostim compared with relevant comparators.

U2 - 10.3310/hta13suppl2/09

DO - 10.3310/hta13suppl2/09

M3 - Article

VL - 13

SP - 63

EP - 68

JO - Health Technology Assessment

JF - Health Technology Assessment

SN - 1366-5278

IS - Suppl 2

ER -