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Lessons learned using real-world data to emulate randomized trials-a case study of treatment effectiveness for newly diagnosed immune thrombocytopenia

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Lessons learned using real-world data to emulate randomized trials-a case study of treatment effectiveness for newly diagnosed immune thrombocytopenia. / McGrath, Leah J; Nielson, Carrie; Saul, Bradley; Breskin, Alexander; Yu, Ying; Nicolaisen, Sia K; Kilpatrick, Karynsa; Ghanima, Waleed; Christiansen, Christian F; Bahmanyar, Shahram; Linder, Marie; Eisen, Melissa; Wasser, Jeffrey; Altomare, Ivy; Kuter, David; Sørensen, Henrik T; Kelsh, Michael; Brookhart, M Alan.

I: Clinical Pharmacology and Therapeutics, Bind 110, Nr. 6, 12.2021, s. 1570-1578.

Publikation: Bidrag til tidsskrift/Konferencebidrag i tidsskrift /Bidrag til avisTidsskriftartikelForskningpeer review

Harvard

McGrath, LJ, Nielson, C, Saul, B, Breskin, A, Yu, Y, Nicolaisen, SK, Kilpatrick, K, Ghanima, W, Christiansen, CF, Bahmanyar, S, Linder, M, Eisen, M, Wasser, J, Altomare, I, Kuter, D, Sørensen, HT, Kelsh, M & Brookhart, MA 2021, 'Lessons learned using real-world data to emulate randomized trials-a case study of treatment effectiveness for newly diagnosed immune thrombocytopenia', Clinical Pharmacology and Therapeutics, bind 110, nr. 6, s. 1570-1578. https://doi.org/10.1002/cpt.2399

APA

McGrath, L. J., Nielson, C., Saul, B., Breskin, A., Yu, Y., Nicolaisen, S. K., Kilpatrick, K., Ghanima, W., Christiansen, C. F., Bahmanyar, S., Linder, M., Eisen, M., Wasser, J., Altomare, I., Kuter, D., Sørensen, H. T., Kelsh, M., & Brookhart, M. A. (2021). Lessons learned using real-world data to emulate randomized trials-a case study of treatment effectiveness for newly diagnosed immune thrombocytopenia. Clinical Pharmacology and Therapeutics, 110(6), 1570-1578. https://doi.org/10.1002/cpt.2399

CBE

McGrath LJ, Nielson C, Saul B, Breskin A, Yu Y, Nicolaisen SK, Kilpatrick K, Ghanima W, Christiansen CF, Bahmanyar S, Linder M, Eisen M, Wasser J, Altomare I, Kuter D, Sørensen HT, Kelsh M, Brookhart MA. 2021. Lessons learned using real-world data to emulate randomized trials-a case study of treatment effectiveness for newly diagnosed immune thrombocytopenia. Clinical Pharmacology and Therapeutics. 110(6):1570-1578. https://doi.org/10.1002/cpt.2399

MLA

Vancouver

Author

McGrath, Leah J ; Nielson, Carrie ; Saul, Bradley ; Breskin, Alexander ; Yu, Ying ; Nicolaisen, Sia K ; Kilpatrick, Karynsa ; Ghanima, Waleed ; Christiansen, Christian F ; Bahmanyar, Shahram ; Linder, Marie ; Eisen, Melissa ; Wasser, Jeffrey ; Altomare, Ivy ; Kuter, David ; Sørensen, Henrik T ; Kelsh, Michael ; Brookhart, M Alan. / Lessons learned using real-world data to emulate randomized trials-a case study of treatment effectiveness for newly diagnosed immune thrombocytopenia. I: Clinical Pharmacology and Therapeutics. 2021 ; Bind 110, Nr. 6. s. 1570-1578.

Bibtex

@article{3bbea33f4efd4f09afe9684ac3ec0347,
title = "Lessons learned using real-world data to emulate randomized trials-a case study of treatment effectiveness for newly diagnosed immune thrombocytopenia",
abstract = "Regulatory agencies are increasingly considering real-world evidence (RWE) to support label expansions of approved medicines. We conducted a comparative effectiveness study to emulate a proposed randomized trial of romiplostim versus standard-of-care (SOC) therapy among patients with recently diagnosed (≤ 12 months) immune thrombocytopenia (ITP), that could support expansion of the romiplostim label. We discuss challenges that we encountered and solutions that were developed to address those challenges. Study size was a primary concern, particularly for romiplostim initiators, given the rarity of ITP and the stringent trial eligibility criteria. For this reason, we leveraged multiple data sources (Nordic Country Patient Registry for Romiplostim; chart review study of romiplostim initiators in Europe; Flatiron Health EMR linked with MarketScan claims). Additionally, unlike the strictly controlled clinical trial setting, platelet counts were not measured at regular intervals in the observational data sources, and therefore the endpoint of durable platelet response often used in trials could not be reliably measured. Instead, the median platelet count was chosen as the primary endpoint. Ultimately, while we observed a slightly higher median platelet count in the romiplostim group versus SOC, precision was limited because of small study size (median difference was 11 x 109 /L (CI: -59, 81)). We underscore the importance of conducting comprehensive feasibility assessments to identify fit-for-purpose data sources with sufficient sample size, data elements, and follow-up. Beyond technical challenges, we also discuss approaches to increase the credibility of RWE, including systematic incorporation of clinical expertise into study design decisions, and separation between decision-makers and the data.",
keywords = "ADULTS, CARE, DENMARK, GUIDELINES, PURPURA, REGRESSION, ROMIPLOSTIM, THROMBOPOIETIN-RECEPTOR AGONISTS",
author = "McGrath, {Leah J} and Carrie Nielson and Bradley Saul and Alexander Breskin and Ying Yu and Nicolaisen, {Sia K} and Karynsa Kilpatrick and Waleed Ghanima and Christiansen, {Christian F} and Shahram Bahmanyar and Marie Linder and Melissa Eisen and Jeffrey Wasser and Ivy Altomare and David Kuter and S{\o}rensen, {Henrik T} and Michael Kelsh and Brookhart, {M Alan}",
year = "2021",
month = dec,
doi = "10.1002/cpt.2399",
language = "English",
volume = "110",
pages = "1570--1578",
journal = "Clinical Pharmacology and Therapeutics",
issn = "0009-9236",
publisher = "JohnWiley & Sons, Inc.",
number = "6",

}

RIS

TY - JOUR

T1 - Lessons learned using real-world data to emulate randomized trials-a case study of treatment effectiveness for newly diagnosed immune thrombocytopenia

AU - McGrath, Leah J

AU - Nielson, Carrie

AU - Saul, Bradley

AU - Breskin, Alexander

AU - Yu, Ying

AU - Nicolaisen, Sia K

AU - Kilpatrick, Karynsa

AU - Ghanima, Waleed

AU - Christiansen, Christian F

AU - Bahmanyar, Shahram

AU - Linder, Marie

AU - Eisen, Melissa

AU - Wasser, Jeffrey

AU - Altomare, Ivy

AU - Kuter, David

AU - Sørensen, Henrik T

AU - Kelsh, Michael

AU - Brookhart, M Alan

PY - 2021/12

Y1 - 2021/12

N2 - Regulatory agencies are increasingly considering real-world evidence (RWE) to support label expansions of approved medicines. We conducted a comparative effectiveness study to emulate a proposed randomized trial of romiplostim versus standard-of-care (SOC) therapy among patients with recently diagnosed (≤ 12 months) immune thrombocytopenia (ITP), that could support expansion of the romiplostim label. We discuss challenges that we encountered and solutions that were developed to address those challenges. Study size was a primary concern, particularly for romiplostim initiators, given the rarity of ITP and the stringent trial eligibility criteria. For this reason, we leveraged multiple data sources (Nordic Country Patient Registry for Romiplostim; chart review study of romiplostim initiators in Europe; Flatiron Health EMR linked with MarketScan claims). Additionally, unlike the strictly controlled clinical trial setting, platelet counts were not measured at regular intervals in the observational data sources, and therefore the endpoint of durable platelet response often used in trials could not be reliably measured. Instead, the median platelet count was chosen as the primary endpoint. Ultimately, while we observed a slightly higher median platelet count in the romiplostim group versus SOC, precision was limited because of small study size (median difference was 11 x 109 /L (CI: -59, 81)). We underscore the importance of conducting comprehensive feasibility assessments to identify fit-for-purpose data sources with sufficient sample size, data elements, and follow-up. Beyond technical challenges, we also discuss approaches to increase the credibility of RWE, including systematic incorporation of clinical expertise into study design decisions, and separation between decision-makers and the data.

AB - Regulatory agencies are increasingly considering real-world evidence (RWE) to support label expansions of approved medicines. We conducted a comparative effectiveness study to emulate a proposed randomized trial of romiplostim versus standard-of-care (SOC) therapy among patients with recently diagnosed (≤ 12 months) immune thrombocytopenia (ITP), that could support expansion of the romiplostim label. We discuss challenges that we encountered and solutions that were developed to address those challenges. Study size was a primary concern, particularly for romiplostim initiators, given the rarity of ITP and the stringent trial eligibility criteria. For this reason, we leveraged multiple data sources (Nordic Country Patient Registry for Romiplostim; chart review study of romiplostim initiators in Europe; Flatiron Health EMR linked with MarketScan claims). Additionally, unlike the strictly controlled clinical trial setting, platelet counts were not measured at regular intervals in the observational data sources, and therefore the endpoint of durable platelet response often used in trials could not be reliably measured. Instead, the median platelet count was chosen as the primary endpoint. Ultimately, while we observed a slightly higher median platelet count in the romiplostim group versus SOC, precision was limited because of small study size (median difference was 11 x 109 /L (CI: -59, 81)). We underscore the importance of conducting comprehensive feasibility assessments to identify fit-for-purpose data sources with sufficient sample size, data elements, and follow-up. Beyond technical challenges, we also discuss approaches to increase the credibility of RWE, including systematic incorporation of clinical expertise into study design decisions, and separation between decision-makers and the data.

KW - ADULTS

KW - CARE

KW - DENMARK

KW - GUIDELINES

KW - PURPURA

KW - REGRESSION

KW - ROMIPLOSTIM

KW - THROMBOPOIETIN-RECEPTOR AGONISTS

UR - http://www.scopus.com/inward/record.url?scp=85114888903&partnerID=8YFLogxK

U2 - 10.1002/cpt.2399

DO - 10.1002/cpt.2399

M3 - Journal article

C2 - 34416023

VL - 110

SP - 1570

EP - 1578

JO - Clinical Pharmacology and Therapeutics

JF - Clinical Pharmacology and Therapeutics

SN - 0009-9236

IS - 6

ER -