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2025

Are There Specificities for Assessing Quality of Life and Utilities in Rare Diseases for Economic Evaluation in France: A Case Study of Published CEESP (Health Economic and Public Health Committee) Opinions

Value & Outcomes Spotlight (ISPOR)

Authors: Sarah Sadeuk-Benabbas, Erwan Autin, Anne-Line Couillerot, Valérie Clément

Risk of Exacerbation and Level of Healthcare Resource Utilization in Myasthenia Gravis Assessed by Myasthenia Gravis Activities of Daily Living Score

Neurology and Therapy

Authors: Angela Ting, Minjee Park, Oshin Sangha, Mohita Kumar, Jean-François Ricci, Edward Lee & Richard J. Nowak

Implementing Digital Pathology: Qualitative and Financial Insights From Eight Leading European Laboratories

Virchows Archiv

Authors: Xavier Matias-Guiu, Jordi Temprana-Salvador, Pablo Garcia Lopez, Solene-Florence Kammerer-Jacquet, Nathalie Rioux-Leclercq, David Clark, Christian M. Schürch, Falko Fend, Sven Mattern, David Snead, Nicola Fusco, Elena Guerini-Rocco, Federico Rojo, Marie Brevet, Manuel Salto Tellez, Angelo Dei Tos, Thomas di Maio, Silvia Ramírez-Peinado, Elizabeth Sheppard, Huw Bannister, Anastasios Gkiokas, Mario Arpaia, Ons Ben Dhia & Nazario Martino

Impact on Beclometasone Dipropionate Pharmacokinetics When Switching to a Low Global Warming Potential Propellant in a Pressurised Metered-dose Inhaler

Pulmonary Pharmacology & Therapeutics

Authors: François Rony, Maria Gloria Pittelli, Cristina Contursi, Ilaria Pacchetti, Emanuele Rocco Calabrò, Luca Vittorio Viganò, Kusum S Mathews Gianluigi Poli, Katrin Van Leuven, Matteo Martini

Evaluating Extracorporeal Renal Replacement Therapies: Balancing Budgetary Considerations With Ethical Implications

Blood Purification

Authors: Gonzalo Ramírez-Guerrero; Fiammetta Zanetti; Akash Nayak Karopadi; Claudio Ronco

The Low Global Warming Potential Propellant HFA-152a Does Not Induce Bronchoconstriction or Impair Mucociliary Clearance

Pulmonary Pharmacology & Therapeutics

Authors: Michela Salvadori, Dave Singh, Kusum Mathews, Luca Girardello, Mauro Cortellini, Aida Emirova, Ilaria Pacchetti, Martina Foti, Veronica Puviani, Gianluigi Poli, François Rony

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2024

Patient Motivations, Concerns, and Perspectives on Sharing Their Electronic Medical Records (EMRs) using Digital Health Applications: A Preliminary Study

ISPOR Europe 2024

Authors: George O, Willmon R, Ricci JF, Perez-Guagnelli E

Development of Patient-Centric Digital Engagement Methods to Address Low Adoption & Adherence to Digital Health Tools: A Novel Conceptual Framework

ISPOR Europe 2024

Authors: Doherty J, Willmon R, Ricci JF, Perez-Guagnelli E

Methods to Include the Organizational Impacts in Health-Economic Evaluations: Experience From CAR-T in France

ISPOR Europe 2024

Authors: Fernandes Soares J, Pozzar M, Autin E, Couillerot A

Global Dynamics of Drug Pricing: Analyzing the Influence of Emerging Countries on Pricing Strategies in Developed Nations

ISPOR Europe 2024

Authors: Bordoy Comas-Cros C, Zhu X, Stasse C, Vidiella E, Lizano-Díez I, Blanqué-Catalina N

Epidemiology and Hospital Management of Patients With PROS in France

ISPOR Europe 2024

Authors: Canaud G, Sano B, Beziz D, Petrica N, Benkanoun L, Pouriel M, Moutie A, Couray-Targe S

Budget Impact Analysis of Ublituximab as aMaintenance Treatment for Relapsing Multiple Sclerosis (RMS) in Spain

ISPOR Europe 2024

Authors: Conde Galan C, Le Rouzo G, Castillo C, Aguado M, Hernaez Colque M

Integrated Care Process and Pharmacological Prescription for Patients With Hypercholesterolemia or Mixed Dyslipidaemia at High or Very High Cardiovascular Risk in Catalonia

ISPOR Europe 2024

Authors: Guiu Segura J, García V, De la Paz I, Marzo I, Salvador N, Carod C, Zamora A, Yago G

Understanding the Pricing and Reimbursement Outcomes of Advanced Therapy Medicinal Products in Spain

ISPOR Europe 2024

Authors: Vico T, Águila M, Hernaez Colque M, De la Paz I, Castillo C

Incremental Burden Attributable to Exposure to Socio-Environmental Factors on Patients With Lung Cancer in France

ISPOR Europe 2024

Authors: Youinou M, Rosé M, Batourou Sano B, Ricci JF, Petrica N

Typology of Patients With Lung Cancer According to Demographics and Exposure to Environmental Factors in France: A Real-World Study

ISPOR Europe 2024

Authors: Youinou M, Rosé M, Sano B, Ricci JF, Petrica N

Psychological Burden in People Living With Myasthenia Gravis: Prevalence and Associated Risk Factors of Depression and Anxiety From the MGFA Global MG Patient Registry (MGFAPR) in the US (2017-2022)

ISPOR 2024

Authors: Park M, Sangha O, Gwathmey K, Ricci JF, Willmon R

Patterns of Glucocorticoid Use Among Persons With Systemic Lupus Erythematosus (SLE) Over Fifteen Years

ISPOR 2024

Authors: Katz P, Pedro S, Park J, Park M, Michaud K

Sex Differences on Limitations in Physical Functioning and Overall Quality of Life of People Living With Myasthenia Gravis From the MGFA Global MG Patient Registry (MGFAPR) in the US

ISPOR 2024

Authors: Sangha O, Park M, Nowak R,
Ricci JF

Understanding the Impact of Physical Exercise in People Living With Myasthenia Gravis From the MGFA Global MG Patient Registry (MGFAPR) in the US (2013-2023)

ISPOR 2024

Authors: Zurinaga A, Nowak R, Ricci JF,
Park M

Prevalence of Comorbidities and Hospitalization in the 65 and over SLE (SYSTEMIC LUPUS ERYTHEMATOSUS) Patient Population: A Comparative Analysis of the FORWARD Lupus Registry and Medicare Data in the US (2017-2021)

ISPOR 2024

Authors: Park M, Nair A, Cortinez V, Buser H, Katz P, Pedro S

Characterization of SLE Patient Population From the FORWARD Lupus Registry (FLR) in the US

ISPOR 2024

Authors: Park M, Pedro S, Lannon J, Calado F, Michaud K, Katz P

An International Consensus Panel on the Potential Value of Digital Surgery

BMJ Open

Authors: Jamie Erskine, Payam Abrishami, Jean-Christophe Bernhard, Richard Charter, Richard Culbertson, Jo Carol Hiatt, Ataru Igarashi, Gretchen Purcell Jackson, Matthew Lien, Guy Maddern, Joseph Soon Yau Ng, Anita Patel, Koon Ho Rha, Prasanna Sooriakumaran, Scott Tackett, Giuseppe Turchetti, Anastasia Chalkidou

Effect of Telemonitoring on the Rate of Dropout During Home Non-Invasive Ventilation: A Retrospective Study Using a Home Care Provider Database

BMJ Open

Authors: Raphael Le Mao, Christophe Gut Gobert, Joelle B Texereau, Frédérique Kremer, Marion Goret, Aurélie Chekroun Martinot, Mathieu Rosé, Wojciech Trzepizur, Frédéric Gagnadoux

Long-Term Inhaled Corticosteroid Treatment in Patients With Chronic Obstructive Pulmonary Disease, Cardiovascular Disease, and a Recent Hospitalised Exacerbation: The ICSLIFE Pragmatic, Randomised Controlled Study

European Journal of Internal Medicine

Authors: Papi, A., Forini, G., Maniscalco, M., Bargagli, E., Crimi, C., Santus, P., Molino, A., Bandiera, V., Baraldi, F., D’Anna, S. E., Carone, M., Marvisi, M., Pelaia, C., Scioscia, G., Patella, V., Aliani, M., Fabbri, L. M., & ICSLIFE Study Group (2024)

Relationship Between Patient-Reported Outcomes to Hospitalization in Patients Living With SLE: A Retrospective Cohort Study Utilizing the FORWARD Lupus Registry

ACR Convergence 2024

Authors: Minjee Park, Sofia Pedro, Jean-François Ricci, Patti Katz

Costs and Healthcare Resource Utilization Associated With Inpatient and Day Hospitalizations of Patients With Lupus and Psychiatric Manifestations in France

ACR Convergence 2024

Authors: Nicoleta Petrica, Nathan Cohen, Maria Pini, Minjee Park, Mathieu Rosé, Jean-François Ricci

An International Consensus Panel on the Potential Value of Digital Surgery

BMJ Open

Authors: Jamie Erskine, Payam Abrishami, Jean-Christophe Bernhard, Richard Charter, Richard Culbertson, Jo Carol Hiatt, Ataru Igarashi, Gretchen Purcell Jackson, Matthew Lien, Guy Maddern, Joseph Soon Yau Ng, Anita Patel, Koon Ho Rha1, Prasanna Sooriakumaran, Scott Tackett, Giuseppe Turchetti, Anastasia Chalkidou

Long-term Inhaled Corticosteroid Treatment in Patients With Chronic Obstructive Pulmonary Disease, Cardiovascular Disease, and a r\Recent Hospitalised Exacerbation: The ICSLIFE Pragmatic, Randomised Controlled Study

European Journal of Internal Medicine

Authors: Alberto Papia, Giacomo Forini, Mauro Maniscalco, Elena Bargagli, Claudia Crimi, Pierachille Santus, Antonio Molino, Valeria Bandiera, Federico Baraldi, Silvestro Ennio D’Anna, Mauro Carone, Maurizio Marvisi, Corrado Pelaia, Giulia Scioscia, Vincenzo Patella, Maria Aliani, Leonardo M Fabbria on behalf of the ICSLIFE Study Group

Safety and Efficacy of Givinostat in Boys With Duchenne Muscular Dystrophy (EPIDYS): a Multicentre, Randomised, Double-blind, Placebo-controlled, Phase 3 Trial

The Lancet, Neurology

Authors: Eugenio Mercuri, Juan J Vilchez, Odile Boespflug-Tanguy, Craig M Zaidman, Jean K Mah, Nathalie Goemans, Wolfgang Müller-Felber, Erik H Niks, Ulrike Schara-Schmidt, Enrico Bertini, Giacomo P Comi, Katherine D Mathews, Laurent Servais, Krista Vandenborne, Jessika Johannsen, Sonia Messina, Stefan Spinty, Laura McAdam, Kathryn Selby, Barry Byrne, Chamindra G Laverty, Kevin Carroll 22, Giulia Zardi 23, Sara Cazzaniga, Nicoletta Coceani, Paolo Bettica, Craig M McDonald

Epidemiology, Survival, and Healthcare Resource Use of Patients With Pulmonary Hypertension Associated With Lung Disease and/or Hypoxia in the UK

ISPOR 2024

Authors: Kiely, D., Wort, S., Funes, D., Petrica, N., Proenca, C., Pini, M., Fernández Delgado, M., Bacchini Jeanneret, G.

Adaptation and Validation of a Bidirectional Matching Algorithm to Enrich Hospital Data With Information From the French National Death Registry: A Case Study in Patients With Lung or Prostate Cancer (MO-REAL)

ISPOR 2024

Authors: Sanchez, S., Lehmann, E., Corneloup Savignol, H., Baradji, A., Ricci, J.F., Nogues, R., Waegeneire, C., Lebas, B.

Assessing the Value of Real-World Evidence for HTA Decision-Making

ISPOR 2024

Authors: Angeli, E., Sabbar, W., Nascimento, J., Badinedi, R., Proenca, C., Godbole, M.

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2023

Harnessing the Potential of natural-Language Processing and Interconnected Data Streams for Complex Diseases in the Hospital Setting: Lupus Case Study in France (Lupus REAL)

ISPOR 2023

Authors: Marc Jouve, Eymeric Lagonotte, Marion Valette, Batourou Sano, Jean-François Ricci, Pr Vincent Vuiblet

Best Practice Considerations on the Assessment of Robotic Assisted Surgical Systems: Results From an International Consensus Expert Panel

Cambridge University Press

Authors: Jamie Erskine, Payam Abrishami,
Richard Charter, Americo Cicchetti, Richard Culbertson, Eliney Faria, Jo Carol Hiatt, Jim Khan, Guy Maddern, Anita Patel, Koon Ho Rha, Paresh Shah, Prasanna Sooriakumaran, Scott Tackett, Giuseppe Turchetti, Anastasia Chalkidou

Pressure Ulcer Prevention Devices in the Management of Older Patients at Risk After Hospital Discharge: an SNDS Study

Journal of Wound Care

Authors: Marion Lartigau, Martine Barateau, Mathieu Rosé, Nicoleta Petricã, Nathalie Salles

Essential Elements of Technology Transfer

BioProcess Online

Authors: Peter Alexander and Phillip Ramsey

Manufacturing Challenges of Therapeutic Antibody-Drug Conjugates

BioProcess International

Authors: Peter Alexander, Kelly Weitzel, Angela Linderholm, William E. Haskins, Sanjeevani Ghone, Steven M. Chamow

Support of RWE in the Price and Reimbursement Decision-Making Process for Rare Diseases in Spain

ISPOR 2023

Authors: Saborido CM, Calleja MA, Martínez Sesmero JM, Piñol FS, Fontanet M, Castillo C, de la Paz Cañizares I, Hernaez Colque M, Asensio I, Ferrández O

Exploring the Value of RWD Collected From Early Access Programs to Support HTA Decision-Making

ISPOR 2023

Authors: Catia Proenca, Rithvik Badinedi, Mrunmayee Godbole, Nicoleta Petrica, Minjee Park, Batourou Sano, Maria Pini

The Comparative Analysis of Accelerated Coverage Pathways for Innovation (ACPI) for Digital Technologies

ISPOR 2023

Authors: Anjali Ramkeesoon, Ons Ben Dhia

A Tutorial on Modern Bayesian Methods in Clinical Trials

Therapeutic Innovation & Regulatory Science

Authors: Muehlemann, N., Zhou, T., Mukherjee, R. et al.

Emotional Journey of Patients With Eosinophilic Esophagitis

Advances in Therapy

Authors: Helen Larsson, Mary J Strobel, Eduardo Perez-Guagnelli

Optimizing Healthcare Outcomes: Identifying the Conditions for Successful Value-based Contracts

ISPOR Europe 2023

Authors: Bento G, Sathi C, Dumoulin O, Grandy A, Mestre-Ferrandiz J, Pascual Argente N, Chouman S, Towle P, Bechara A

Qualitative Analysis of Value-based Contract Experience, Perception, and Future Adoption Across Geographies

ISPOR Europe 2023

Authors: Sathi C, Dumoulin O, Bento G, Grandy A, Mestre-Ferrandiz J, Pascual-Argente N, Chouman S, Towle P, Bechara A

Value-Based Contracts Types Across Global Healthcare Systems: Comparing Perceptions Between Finance-Based and Outcomes-Based Agreements

ISPOR Europe 2023

Authors: Pascual-Argente N, Mestre-Ferrandiz
J, Bento G, Sathi C, Dumoulin O, Grandy A, Chouman S, Towle P, Bechara A

Characterization of the Lupus Patient Experience Based on Longitudinal Patient Generated Real World Data From a Digital Health APP (HEALTH STORYLINE)

ISPOR Europe 2023

Authors: Willmon R, Vivaldi V, Cohen N, Petrica N, Ricci JF

Single-arm Studies in Rare Diseases and Statistical Methods to Mitigate Lack of Comparative Data: What Kind of Assessment Is More Successful for the French HTA Body?

ISPOR Europe 2023

Authors: Belamri M, Pozzar M, Yahia A, Couillerot A

Handling Uncertainty in Health Economic Assessments: Experience From COVID-19 in France

ISPOR Europe 2023

Authors: Autin E, Pozzar M, Couillerot A

Long-term Safety and Efficacy of Velmanase Alfa Treatment in Children Under 6 Years of Age With Alpha-mannosidosis: A Phase 2, Open Label, Multicenter Study

Journal of Inherited Metabolic Disease

Authors: Nathalie Guffon, Vassiliki Konstantopoulou, Julia B. Hennermann, Nicole Muschol, Irene Bruno, Albina Tummolo, Ferdinando Ceravolo, Giulia Zardi, Andrea Ballabeni, Allan Lund

Access and Reimbursement Pathways for Digital Health Solutions and In Vitro Diagnostic Devices: Current Scenario and Challenges

Frontiers in Medical Technology

Authors: Andrea Mantovani, Claudia Leopaldi, Cassandra Maria Nighswander, Rossella Di Bidino

Burden of the Disease and Management in COVID-19 Hospitalized Patients in MSO and Home Care in France: COVID Hospital Study (2020-2022)

ISPOR 2023

Authors: Pozzar, M., Petrica, N., Ben Romdhane, H., Mongazon-Cazavet, M.

New Pathway in Anti-HER2 Treatments Administration in Breast Cancer in France: Which Impacts of the Involvement of an Advanced Practice Nurse in Hospital at Home Program?

ISPOR 2023

Authors: Pozzar, M., Couillerot, A., Gherardi, A., Marchand, L., Aurélie de Lehvenfehlt, Le Lay, K., Pallaro, S., Bigas, M., Maillan, G., Deluche, E.

Incorporating Environmental Sustainability Considerations into National Health Technology Assessments: A Landscape Review

ISPOR 2023

Author: Stelina Tsantila

Cost-Effectiveness of the PPARγ Modulator N-acetyl-GED-0507-34-LEVO (NAC-GED 5%) Versus Benzoyl Peroxide-Adapalene for Moderate-to-Severe Acne

ISPOR 2023

Authors: Matos, T., Godbole, M., Grand, A., Demont, E., Viti, F., Bellinvia, S., Zambeletti, L., Sebaratnam, D.

New Pathway in Anti-HER2 Treatments Administration in Breast Cancer in France: Which Impacts of the Involvement of an Advanced Practice Nurse in Hospital at Home Program?

ISPOR 2023

Authors: Pozzar, M., Couillerot, A., Gherardi, A., Marchand, L., Aurélie de Lehvenfehlt, Le Lay, K., Pallaro, S., Bigas, M., Maillan, G., Deluche, E.

Factors Having an Impact on the Prescription of Cancer Treatments in Hospital at Home Program in Bronchopulmonary Cancer: A French Claims Database Analysis (2016-2021)

ISPOR 2023

Authors: Pozzar, M., Couillerot, A., Rose, M., Sano, B., Petrica, N.

Exploring the Value of RWD Collected from Early Access Programs to Support HTA Decision-Making

ISPOR 2023

Authors: Proenca, C., Badinedi, R., Godbole, M., Petrica, N., Park, M., Sano, B., Pini, M.

Treatment Approvals in Oncology and Relationship with Improvement in 5-Year Relative Survival for Cancer Patients Europe

ISPOR 2023

Authors: Gulotta, G., Reinert, M., Cruz Arrioja, J., Zaniboni, B., Naser, R., Mos, J.

Resource Use Associated With Alpelisib for the Treatment of Patients With PROS: A Modeling Approach

ISPOR 2023

Authors: Petrica, N., Yasar, N., Foss, P., Palladino, S., Ricci, J.F.

Exploring the Perceived Value of Fixed-Dose Combinations in Type 2 Diabetes Mellitus in the EU4 and the UK

ISPOR 2023

Authors: Gulotta, G., Reniert, M., Cruz Arrioja, J., Zaniboni, B., Naser, R., Mos, J.

Treatment Approvals in Oncology and Relationship with Improvement in 5-Year Relative Survival for Cancer Patients Europe

ISPOR 2023

Authors: Gulotta, G., Reinert, M., Cruz Arrioja, J., Zaniboni, B., Naser, R., Mos, J.

Resource Use Associated With Alpelisib for the Treatment of Patients With PROS: A Modeling Approach

ISPOR 2023

Author: Petrica, N., Yasar, N., Foss, P., Palladino, S., Ricci, J.F.

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2022

The Future of 3D Printed Biofilms for In Vitro and In Vivo Wound Infection Models

Wound Masterclass

Authors: Mitch Sanders, Mia Hanna, Vanessa Vu, Lindsay Poland

Alligator-Derived Hyaluronic Acid: Bacteriostatic and Fungastatic Properties Against Pathogens

Wound Masterclass

Authors: Mitch Sanders, Mia Hanna, Vanessa Vu, Lindsay Poland

Considerations for Analyzing and Interpreting Data From Biometric Monitoring Technologies in Clinical Trials

Digital Biomarkers

Authors: Bohdana Ratitch, Isaac R. Rodriguez-Chavez, Abhishek Dabral, Ariano Fontanari, Julio Vega, Francesco Onorati, Benjamin Vandendriessche, Stuart Morton, Yasaman Damestani

GMP Manufacturing and IND-Enabling Studies of a Recombinant Hyperimmune Globulin Targeting SARS-CoV-2

Pathogens

Authors: Mizrahi, R.A.; Lin, W.Y.; Gras, A.; Niedecken, A.R.; Wagner, E.K.; Keating, S.M.; Ikon, N.; Manickam, V.A.; Asensio, M.A.; Leong, J.; et al.

Impact of Publication of the French Commission for Economic and Public Health Evaluation (CEESP) Doctrine on the Conclusions of Its Economic Opinions

ISPOR Europe 2022

Authors: Meyer M, Belamri M, Autin E, Couillerot A

Are There Specificities for Assessing Quality of Life and Utilities in Rare Diseases for Economic Evaluation in France: A Case Study of Published CEESP Opinions

ISPOR Europe 2022

Authors: Sadeuk-Benabbas S, Autin E, Couillerot A, Clément V

Relationship Between MAN2B1 Genotype/Subcellular Localization Subgroups, Antidrug Antibody Detection, and Long-term Velmanase Alfa Treatment Outcomes in Patients with Alpha-Mannosidosis

JIMD Reports

Authors: Line Gutte Borgwardt, Ferdinando Ceravolo, Giulia Zardi, Andrea Ballabeni, Allan Meldgaard Lund

Evolution of the Number of Metastatic Lung Cancer Patients Before and During the COVID 19 Pandemic: A French National Hospital Database Analysis

ISPOR 2022

Authors: Sano, B., Petrica, N., Finas, R., Rose, M.

Are There Specificities for Assessing Quality of Life and Utilities in Rare Diseases for Economic Evaluation in France: A Case Study of Published CEESP Opinions

ISPOR Europe 2022

Authors: Sadeuk-Benabbas S, Autin E, Couillerot A, Clément V

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2021

Generation of Recombinant Hyperimmune Globulins From Diverse B-Cell Repertoires

Nature Biotechnology

Authors: Keating, S.M., Mizrahi, R.A., Adams, M.S. et al.

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2020

Randomized Controlled Trial Evidence of Cost-Effectiveness of a Multifaceted AKI Intervention Approach

Kidney International Reports

Authors: Nicholas M. Selby, Luís Korrodi-Gregório, Anna Casula, Nitin V. Kolhe, Daniel Ribes Arbonés, Katelyn D. Bukieda, Deepak Sahu, Chris Rao, Giacomo Basadonna