Publications 2023

The cost-effectiveness of pegcetacoplan in complement treatment-naïve adults with paroxysmal nocturnal hemoglobinuria in the USA.

Fishman J, Wilson K, Drzewiecka A, Pochopień M, Dingli D.

J Comp Eff Res. 2023 Oct;12(10):e230055. doi: 10.57264/cer-2023-0055. Epub 2023 Sep 1.

PMID: 37655691 Free PMC article.


Cost-effectiveness of finerenone in chronic kidney disease associated with type 2 diabetes in The Netherlands.

Quist SW, van Schoonhoven AV, Bakker SJL, Pochopień M, Postma MJ, van Loon JMT, Paulissen JHJ.

Cardiovasc Diabetol. 2023 Nov 28;22(1):328. doi: 10.1186/s12933-023-02053-6.

PMID: 38017448 Free PMC article.


The impact of amortization of gene therapies funding on the results and conclusions of CEMs and BIMs.

Polek H, Janik J, Paterak E, Dabbous M, Pochopień M, Toumi M.

J Mark Access Health Policy. 2023 Jul 10;11(1):2232648. doi: 10.1080/20016689.2023.2232648. eCollection 2023.
PMID: 37440980 Free PMC article. 


Cost-effectiveness of recombinant factor VIII Fc versus emicizumab for prophylaxis in adults and adolescents with haemophilia A without inhibitors in the UK.

Kragh N, Tytula A, Pochopien M, Aballéa S, Toumi M, Hakimi Z, Nazir J, Bystrická L, Fatoye F.

Eur J Haematol. 2023 Mar;110(3):262-270. doi: 10.1111/ejh.13901. Epub 2022 Dec 11.

PMID: 36398467 Free PMC article.

Publications 2022

Challenges in the market access of regenerative medicines, and implications for manufacturers and decision-makers: a systematic review.

Qiu T, Pochopień M, Hanna E, Liang S, Wang Y, Han R, Toumi M, Aballéa S.Regen Med.
2022 Mar;17(3):119-139. doi: 10.2217/rme-2021-0083. Epub 2022 Jan 19.PMID: 35042424 Review.


Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment.

Qiu T, Pochopien M, Liang S, Saal G, Paterak E, Janik J, Toumi M.Front Public Health.
2022 Feb 9;10:773629. doi: 10.3389/fpubh.2022.773629. eCollection 2022.PMID: 35223725 Free PMC article. Review.


The cost-effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting.

Hakimi Z, Wilson K, McAughey E, Pochopien M, Wojciechowski P, Toumi M, Knight C,
Sarda SP, Patel N, Wiseman C, de Castro NP, Nazir J, Kelly RJ.J Comp Eff Res. 2022
Sep;11(13):969-985. doi: 10.2217/cer-2022-0076. Epub 2022 Jul 7.PMID: 35796199 Free


Cost-minimization analysis of recombinant factor VIII Fc versus emicizumab for treating patients with hemophilia A without inhibitors in Europe.

Mancuso ME, Castaman G, Pochopien M, Aballéa S, Drzewiecka A, Hakimi Z, Nazir J,
Fatoye F.J Med Econ. 2022 Aug 22:1-25. doi: 10.1080/13696998.2022.2115777. Online
ahead of print.PMID: 35993970


Capturing the value of vaccination within health technology assessment and health economics: Literature review and novel conceptual framework.

Beck E, Biundo E, Devlin N, Doherty TM, Garcia-Ruiz AJ, Postma M, Sheikh S, Smela B,
Toumi M, Wasem J, Nolan T, Salisbury D. Vaccine. 2022 Jun 26;40(30):4008-4016. doi: 10.1016/j.vaccine.2022.04.050. Epub 2022 May 23. PMID: 35618559.


Impact of the COVID-19 pandemic on the conduct of clinical trials: a quantitative analysis

Margas W, Wojciechowski P, Toumi M.J Mark Access Health Policy. 2022 Aug 9;10(1):2106627. doi: 10.1080/20016689.2022.2106627. eCollection 2022.PMID: 35968522 Free PMC article.


Effectiveness and Safety of Nonvitamin K Oral Anticoagulants Rivaroxaban and Apixaban in Patients with Venous Thromboembolism: A Meta-Analysis of Real-World Studies

Wu O, Morris S, Larsen TB, Skjøth F, Evans A, Bowrin K, Wojciechowski P, Margas W, Huelsebeck M.Cardiovasc Ther. 2022 Jun 9;2022:2756682. doi: 10.1155/2022/2756682. eCollection 2022.PMID: 35801133 Free PMC article.

Publications 2021

Efficacy and Safety of Avatrombopag in Patients with Chronic Immune Thrombocytopenia: A Systematic Literature Review and Network Meta-Analysis.

Wojciechowski P, Wilson K, Nazir J, Pustułka I, Tytuła A, Smela B, Pochopien M, Vredenburg M, McCrae KR, Jurczak W.Adv Ther. 2021 Jun;38(6):3113-3128. doi: 10.1007/s12325-021-01752-4. Epub 2021 May 1.PMID: 33934279


An overview of health technology assessments of gene therapies with the focus on cost- effectiveness models.

Pochopień M, Paterak E, Clay E, Janik J, Aballea S, Biernikiewicz M, Toumi M.J Mark
Access Health Policy. 2021 Nov 13;9(1):2002006. doi: 10.1080/20016689.2021.2002006.
eCollection 2021.PMID: 34790341 Free PMC article. Review.


Considering potential solutions for limitations and challenges in the health economic evaluation of gene therapies.

Pochopień M, Qiu T, Aballea S, Clay E, Toumi M.Expert Rev Pharmacoecon Outcomes
Res. 2021 Dec;21(6):1145-1158. doi: 10.1080/14737167.2021.1969229. Epub 2021 Aug
30.PMID: 34407704


FINE-CKD model to evaluate economic value of finerenone in patients with chronic kidney disease and type 2 diabetes.

Pochopień MT, Cherney DZI, Folkerts K, Levy P, Millier A, Morris S, Roy-Chaudhury P,
Sullivan SD, Mernagh P.Am J Manag Care. 2021 Dec;27(20 Suppl):S375-S382. doi:
10.37765/ajmc.2021.88808.PMID: 34878755 Free article.


Relationship between FEV 1 decline and mortality in patients with bronchiolitis obliterans syndrome-a systematic literature review

Kemp R, Pustulka I, Boerner G, Smela B, Hofstetter E, Sabeva Y, François C. Respir Med. Respir Med. 2021 Nov;188:106608. doi: 10.1016/j.rmed.2021.106608. Epub 2021 Sep 10. PMID: 34517199.


Targeted literature review of the burden of illness in patients with chronic kidney disease and type 2 diabetes

Fried LF, Folkerts K, Smela B, Bowrin KD, Mernagh P, Millier A, Kovesdy CP. Am J Manag Care. 2021 May;27(8 Suppl):S168-S177. doi: 10.37765/ajmc.2021.88660. PMID: 34019359.


Efficacy and Safety of Rivaroxaban Compared with Other Therapies Used in Patients with Peripheral Artery Disease Undergoing Peripheral Revascularization: A Systematic Literature Review and Network Meta-Analysis

Bauersachs R, Wu O, Hawkins N, Bowrin K, Wojciechowski P, Clay E, Huelsebeck M.Cardiovasc Ther. 2021 Aug 24;2021:8561350. doi: 10.1155/2021/8561350. eCollection 2021.PMID: 34497668 Free PMC article. Review.


Efficacy of rFIXFc versus rIX-FP for the Treatment of Patients with Hemophilia B: Matching-Adjusted Indirect Comparison of B-LONG and PROLONG-9FP Trials

Astermark J, Wojciechowski P, Aballéa S, Hakimi Z, Nazir J, Klamroth R.J Blood Med. 2021 Jul 14;12:613-621. doi: 10.2147/JBM.S312885. eCollection 2021.PMID: 34285621 Free PMC article.


Comparative Efficacy and Safety of Peficitinib Versus Tofacitinib and Baricitinib for Treatment of Rheumatoid Arthritis: A Systematic Review and Network Meta-Analysis

Tanaka Y, Okumura H, Kim S, Dorey J, Wojciechowski P, Chorąży J, Kato D, Schultz NM.Rheumatol Ther. 2021 Jun;8(2):729-750. doi: 10.1007/s40744-021-00284-1. Epub 2021 Mar 16.PMID: 33725321 Free PMC article.


Efficacy of rFVIIIFc versus Emicizumab for the Treatment of Patients with Hemophilia A without Inhibitors: Matching-Adjusted Indirect Comparison of A-LONG and HAVEN Trials

Klamroth R, Wojciechowski P, Aballéa S, Diamand F, Hakimi Z, Nazir J, Abad-Franch L, Lethagen S, Santagostino E, Tarantino MD.J Blood Med. 2021 Feb 25;12:115-122. doi: 10.2147/JBM.S288283. eCollection 2021.PMID: 33664606 Free PMC article.


Efficacy and safety of baloxavir marboxil versus neuraminidase inhibitors in the treatment of influenza virus infection in high-risk and uncomplicated patients - a Bayesian network meta-analysis

Taieb V, Ikeoka H, Wojciechowski P, Jablonska K, Aballea S, Hill M, Hirotsu N.Curr Med Res Opin. 2021 Feb;37(2):225-244. doi: 10.1080/03007995.2020.1839400. Epub 2020 Nov 9.PMID: 33079575

Publications 2020

Managed Entry Agreements: Policy Analysis From the European Perspective

Dabbous M,Chachoua L, Caban A, Toumi M. Value Health. 2020 Apr;23(4):425-433. doi:
10.1016/j.jval.2019.12.008. PMID: 32327159 Free article.


Efficacy and Treatment Burden of Intravitreal Aflibercept Versus Intravitreal Ranibizumab Treat-and-Extend Regimens at 2 Years: Network Meta-Analysis Incorporating Individual Patient Data Meta-Regression and Matching-Adjusted Indirect Comparison

Ohji M, Lanzetta P, Korobelnik JF, Wojciechowski P, Taieb V, Deschaseaux C, Janer D, Tuckmantel C.Adv Ther. 2020 May;37(5):2184-2198. doi: 10.1007/s12325-020-01298-x. Epub 2020 Mar 28.PMID: 32222903 Free PMC article.

Publications 2019

Oncology from an HTA and Health Economic Perspective.

Francois C, Zhou J, Pochopien M, Achour L, Toumi M.Recent Results Cancer Res.
2019;213:25-38. doi: 10.1007/978-3-030-01207-6_3.PMID: 30543005


Cost-effectiveness of fluocinolone acetonide implant (ILUVIEN®) in UK patients with chronic diabetic macular oedema considered insufficiently responsive to available therapies.

Pochopien M, Beiderbeck A, McEwan P, Zur R, Toumi M, Aballéa S.BMC Health Serv Res.
2019 Jan 9;19(1):22. doi: 10.1186/s12913-018-3804-4.PMID: 30626376 Free PMC article.


Number needed to treat based on real-world evidence for non-vitamin K antagonist oral anticoagulants versus vitamin K antagonist oral anticoagulants in stroke prevention in patients with non-valvular atrial fibrillation

Briere JB, Bowrin K, Millier A, Toumi M, Wojciechowski P, Taieb V.J Med Econ. 2019 Aug;22(8):760-765. doi: 10.1080/13696998.2019.1606001. Epub 2019 May 3.PMID: 30969801 Free article.

Publications 2017

Proposed changes to the reimbursement of pharmaceuticals and medical devices in Poland and their impact on market access and the pharmaceutical industry

Badora K, Caban A, Rémuzat C, Dussart C, Toumi M. J Mark Access Health Policy. 2017 Sep 25;5(1):1381544. doi: 10.1080/20016689.2017.1381544. eCollection 2017. PMID: 29081924 Free PMC article.


Supply-side and demand-side policies for biosimilars: an overview in 10 European member states

Rémuzat C, Kapuśniak A, Caban A, Ionescu D, Radière G, Mendoza C, Toumi M. J
Mark Access Health Policy. 2017 Apr 28;5(1):1307315. doi: 10.1080/20016689.2017.1307315.eCollection 2017. PMID: 28740617 Free PMC article


Filling the gap in CNS drug development: evaluation of the role of drug repurposing

A Caban , K Pisarczyk , K Kopacz , A Kapuśniak , M Toumi , C Rémuzat , A Kornfeld 

J Mark Access Health Policy,2017 Apr 10;5(1):1299833.doi: 10.1080/20016689.2017.1299833. eCollection 2017.

Publications 2016

Effectiveness and safety of vedolizumab for treatment of Crohn's disease: a systematic review and meta-analysis

Moćko P, Kawalec P, Smela-Lipińska B, Pilc A. Arch Med Sci. 2016 Oct 1;12(5):1088-1096.doi:10.5114/aoms.2016.61915. Epub 2016 Aug 24. PMID:
27695501; PMCID: PMC5016590.


Leki swoiste stosowane w leczeniu wirusowego zapalenia wątroby typu C. Lek W Polsce

Smela-Lipińska, B., Moćko, P., Kawalec, P., & Pilc, A. (2016). 26(1), 43–51.


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