Article
Pharmacology & Pharmacy
Noa Rosenberg, Sibren van den Berg, Nina N. Stolwijk, Bart A. W. Jacobs, Hendrika C. Post, Anna M. G. Pasmooij, Saco J. de Visser, Carla E. M. Hollak
Summary: This paper provides an easily applicable roadmap for academic researchers to make rare disease medicines available for patients. It emphasizes the importance of interaction with regulators and highlights alternative routes for timely access to medicines. Aligning academic studies with regulatory and reimbursement requirements is crucial for improving access to rare disease medicines.
FRONTIERS IN PHARMACOLOGY
(2023)
Article
Pharmacology & Pharmacy
Elisabeth Bakker, Kelly Plueschke, Carla J. Jonker, Xavier Kurz, Viktoriia Starokozhko, Peter G. M. Mol
Summary: Real-world data/evidence (RWD/RWE) can provide insightful information on medicines' clinical effects and guide regulatory decisions. However, further evaluation is needed to use RWD/RWE to demonstrate the efficacy of medicines. This study characterized RWD/RWE presented by applicants and analyzed its contribution to regulatory decisions on the benefit-risk profile of medicines. Common limitations of submitted RWD/RWE included missing data, lack of representativeness, and bias risks.
CLINICAL PHARMACOLOGY & THERAPEUTICS
(2023)
Article
Pharmacology & Pharmacy
C. Mircea S. Tesileanu, Francesco Pignatti, Enrico Tognana, Anthony Humphreys
Summary: The European Medicines Agency (EMA) provides guidance and support to pharmaceutical companies through business pipeline meetings (BPMs). An analysis of oncology BPMs over a 5-year period revealed common topics such as regulatory pathways/guidelines and trial design. Targeted therapy, companion diagnostics, institutional collaboration, trial design, and regulatory pathways/guidelines were the most discussed topics in these meetings, with molecular developments being the common denominator.
CLINICAL PHARMACOLOGY & THERAPEUTICS
(2023)
Article
Clinical Neurology
Oriana Ciani, Michela Meregaglia, Mario Alberto Battaglia, Gianpaolo Brichetto, Antonella Conte, Claudio Gasperini, Valeria Sansone
Summary: A study was conducted to investigate the use of patient-reported outcome measures (PROMs) in the regulatory approval of drugs for neurological conditions in Europe between 2017 and 2022. It was found that 57% of the evaluation reports included PROMs as secondary endpoints, with the most common ones being EQ-5D, SF-36, and PedsQL. The study concluded that while neurology utilizes PROMs more frequently, there is a need for better harmonization of the instruments used.
NEUROLOGICAL SCIENCES
(2023)
Article
Anesthesiology
Erik Florvaag, Gunnar Johansson
Summary: Two recent case-control studies published in the British Journal of Anaesthesia have revealed that the intake of pholcodine-containing cough medicines prior to general anesthesia significantly increases the risk of anaphylaxis caused by neuromuscular blocking agents. These findings from a French multicentre study and a single-centre study from Western Australia strongly support the pholcodine hypothesis for IgE-sensitisation to neuromuscular blocking agents. The European Medicines Agency, which faced criticism for its previous inaction, has finally recommended the cessation of sales for all pholcodine-containing medicines in the EU by December 1, 2022. Only time will tell whether this measure will effectively reduce the incidence of perioperative anaphylaxis in the EU, similar to the results seen in Scandinavia.
BRITISH JOURNAL OF ANAESTHESIA
(2023)
Review
Hematology
Herbert Afegenwi Mbunkah, Jens Reinhardt, Chancelar Kafere, Heiner Scheiblauer, Irena Prat, Claudius Micha Nuebling
Summary: Blood transfusion is a routine life-saving medical procedure that emphasizes the importance of blood compatibility between donors and recipients. High-quality, safe, and well-performing in vitro diagnostic medical devices play a crucial role in transfusion medicine.
Letter
Medicine, General & Internal
Mark P. Lythgoe, Paul Middleton
Summary: This qualitative improvement study examines COVID-19 vaccine approvals by medicine regulatory agencies in the US, EU, and Canada, comparing regulatory review times and analyzing clinical evidence supporting authorization.
Review
Economics
Ari Gnanasakthy, Courtney Levy, Lindsey Norcross, Lynda Doward, Randall Winnette
Summary: A review of oncology drug labeling in Europe from 2017-2021 showed that 20% of new drug approvals included patient-reported outcome (PRO)-related language in the labeling compared to 33% from 2012-2016. The decline in PRO-related labeling may be due to an increase in open-label studies during the same period.
Review
Health Care Sciences & Services
Giovanni Tafuri, Andrea Bracco, Jens Grueger
Summary: The EU Orphan Regulation has led to an increase in the development of medicines for rare diseases. However, access to these treatments varies greatly across EU member states. This article provides an overview of the current situation, discusses policy landscapes, and makes recommendations for improvement to ensure patient access to orphan drugs.
EXPERT REVIEW OF PHARMACOECONOMICS & OUTCOMES RESEARCH
(2022)
Article
Medicine, General & Internal
Marc Maliepaard, Priscilla Nibi, Gabriella Nibi, Anna M. G. Pasmooij
Summary: This paper reports on the experience of the EMA in dealing with CDx before the implementation of the new IVDR. The analysis shows that the EMA has focused on the clinical performance and analytical performance of CDx, which can be valuable for future assessments.
FRONTIERS IN MEDICINE
(2022)
Review
Medicine, General & Internal
Marieke J. H. J. Dekker, Pieter Stolk, Anna M. G. Pasmooij
Summary: This study evaluated the qualification opinions and scientific advices of the Committee for Medicinal Products for Human Use on the use of novel remote monitoring technologies in clinical trials. Mainly focusing on accelerometers, mobile applications, and glucose monitoring devices, the recommendations of the CHMP mainly addressed relevance of outcome measures, validation, precision, compliance, and data handling. Although there is an increasing trend in the use of RMTs in registration trials, it is still relatively rare.
FRONTIERS IN MEDICINE
(2021)
Article
Pharmacology & Pharmacy
Efthymios Manolis, Alfredo Garcia-Arieta, Anders Lindahl, Evangelos Kotzagiorgis, Jobst Limberg, Oyvind Holte, Paulo Paixao, Carolien Versantvoort, Flora Musuamba Tshinanu, Kevin Blake, Michiel Van den Heuvel
Summary: Model-informed drug development approaches are widely accepted by the EMA for new drug development, but there is no consensus on their use for generic drugs. This commentary proposes conditions for the use of mechanistic models to support or potentially waive clinical bioequivalence/bioavailability studies, expanding on the existing EMA regulatory framework for bioequivalence and PBPK modeling.
CPT-PHARMACOMETRICS & SYSTEMS PHARMACOLOGY
(2023)
Article
Clinical Neurology
Stina Saunders, Sarah Gregory, Matthew H. S. Clement, Cindy Birck, Serge van der Geyten, Craig W. Ritchie
Summary: The EPAD project is a large-scale research program that aims to facilitate early intervention and prevention of Alzheimer's disease. With over 2,000 research participants and collaboration with 39 partners, the EPAD project has generated valuable data and samples that are freely accessible. The project's assets will be maintained and contribute to future research in the field.
FRONTIERS IN NEUROLOGY
(2022)
Article
Infectious Diseases
Nicolas Vignier, Olivier Bouchaud, Andrea Angheben, Emmanuel Bottieau, Guido Calleri, Joaquin Salas-Coronas, Charlotte Martin, Jose Manuel Ramos, Matthieu Mechain, Christophe Rapp, Hans-Dieter Nothdurft, Maria Velasco, Azucena Bardaji, Gerardo Rojo-Marcos, Leo G. Visser, Christoph Hatz, Zeno Bisoffi, Tomas Jelinek, Stephan Duparc, Yann Bourhis, Silva Tommasini, Maurizio Iannucelli, Antonella Bacchieri, Giovan Giuseppe Mattera, Emilio Merlo Pich, Ronald H. Behrens
Summary: The study recruited 294 participants from European countries, showing that artenimol-piperaquine is effective and well-tolerated for malaria treatment in this population. Adverse events and QT prolongation rates were similar to those seen in larger cohorts from endemic regions.
Review
Pharmacology & Pharmacy
Frauke Naumann-Winter, Franziska Wolter, Ulrike Hermes, Eva Malikova, Nils Lilienthal, Tania Meier, Maria Elisabeth Kalland, Armando Magrelli
Summary: This study analyzes regulatory documents on orphan designated medicinal products in the EU and finds that real-world data is more commonly referenced in marketing authorization applications for rare diseases. The contribution of external data to efficacy aspects can be evaluated in terms of clinical development, regulatory decision making, and post-approval data collection. In the sample analyzed, the majority of medicinal products for rare diseases involve external data on efficacy, while purely administrative data does not play a role in these applications.
FRONTIERS IN PHARMACOLOGY
(2022)
Article
Infectious Diseases
S. Murray, L. McKenna, E. Pelfrene, R. Botgros
INTERNATIONAL JOURNAL OF TUBERCULOSIS AND LUNG DISEASE
(2015)
Article
Infectious Diseases
Jonathan Cohen, Jean-Louis Vincent, Neill K. J. Adhikari, Flavia R. Machado, Derek C. Angus, Thierry Calandra, Katia Jaton, Stefano Giulieri, Julie Delaloye, Steven Opal, Kevin Tracey, Tom van der Poll, Eric Pelfrene
LANCET INFECTIOUS DISEASES
(2015)
Article
Immunology
Charles Knirsch, Demissie Alemayehu, Radu Botgros, Sabrina Comic-Savic, David Friedland, Thomas L. Holland, Kunal Merchant, Gary J. Noel, Eric Pelfrene, Christina Reith, Jonas Santiago, Rosemary Tiernan, Pamela Tenearts, Jennifer C. Goldsack, Vance G. Fowler
CLINICAL INFECTIOUS DISEASES
(2016)
Article
Immunology
Helen Donnelly, Demissie Alemayehu, Radu Botgros, Sabrina Comic-Savic, Barry Eisenstein, Benjamin Lorenz, Kunal Merchant, Eric Pelfrene, Christina Reith, Jonas Santiago, Rosemary Tiernan, Richard Wunderink, Pamela Tenaerts, Charles Knirsch
CLINICAL INFECTIOUS DISEASES
(2016)
Review
Infectious Diseases
Eric Pelfrene, Elsa Willebrand, Ana Cavaleiro Sanches, Zigmars Sebris, Marco Cavaleri
JOURNAL OF ANTIMICROBIAL CHEMOTHERAPY
(2016)
Article
Public, Environmental & Occupational Health
Harish Nair, Octavio Ramilo, Irmgard Eichler, Eric Pelfrene, Asuncion Mejias, Fernando P. Polack, Koen B. Pouwels, Joanne M. Langley, Marta Nunes, Nicoline van der Maas, Leyla Kragten-Tabatabaie, Eugenio Baraldi, Terho Heikkinen, Brigitte Fauroux, Mike Sharland, Cyrus Park, Paolo Manzoni, Nikolaos G. Papadopoulos, Federico Martinon-Torres, Renato Stein, Louis Bont
JOURNAL OF GLOBAL HEALTH
(2016)
Review
Infectious Diseases
E. Pelfrene, M. Mura, A. Cavaleiro Sanches, M. Cavaleri
CLINICAL MICROBIOLOGY AND INFECTION
(2019)
Article
Infectious Diseases
Eric Pelfrene, Martin Harvey Allchurch, Nsengi Ntamabyaliro, Victoria Nambasa, Fatima V. Ventura, Nithyanandan Nagercoil, Marco Cavaleri
PLOS NEGLECTED TROPICAL DISEASES
(2019)
Article
Virology
Eric Pelfrene, Zigmars Sebris, Marco Cavaleri
Editorial Material
Public, Environmental & Occupational Health
Eric Pelfrene, Radu Botgros, Marco Cavaleri
Summary: The paper highlights the global issue of antimicrobial resistance and the potential exacerbation due to the ongoing COVID-19 pandemic. It stresses the need for collaboration to address the problem and emphasizes the importance of an integrated strategy to tackle this public health peril.
ANTIMICROBIAL RESISTANCE AND INFECTION CONTROL
(2021)
