Patient Registries: An Underused Resource for Medicines Evaluation : Operational proposals for increasing the use of patient registries in regulatory assessments.
Sign Up to like & getrecommendations! Published in 2019 at "Drug Safety"
DOI: 10.1007/s40264-019-00862-x
Abstract: IntroductionPatient registries, 'organised systems that use observational methods to collect uniform data on a population defined by a particular disease, condition, or exposure, and that is followed over time', are potentially valuable sources of data… read more here.
Keywords: regulatory assessments; increasing use; patient registries; registry ... See more keywords
Factors Influencing the Generation of Evidence from Simple Data Held in International Rare Disease Patient Registries
Sign Up to like & getrecommendations! Published in 2019 at "Pharmaceutical Medicine"
DOI: 10.1007/s40290-019-00316-w
Abstract: Background Rare diseases (defined as affecting  read more here.
Keywords: patient registries; rare disease; disease; evidence ... See more keywords
Rare Disease Registries Are Key to Evidence-Based Personalized Medicine: Highlighting the European Experience
Sign Up to like & getrecommendations! Published in 2022 at "Frontiers in Endocrinology"
DOI: 10.3389/fendo.2022.832063
Abstract: Rare diseases, such as inherited metabolic diseases, have been identified as a health priority within the European Union more than 20 years ago and have become an integral part of EU health programs and European… read more here.
Keywords: patient registries; rare disease; disease; evidence ... See more keywords
Contribution of patient registries to regulatory decision making on rare diseases medicinal products in Europe
Sign Up to like & getrecommendations! Published in 2022 at "Frontiers in Pharmacology"
DOI: 10.3389/fphar.2022.924648
Abstract: Between 2000 and 2021, the European Medicines Agency (EMA) assigned the orphan designation to over 1,900 medicines. Due to their small target populations, leading to challenges regarding clinical trial recruitment, study design and little knowledge… read more here.
Keywords: patient registries; registries regulatory; medicinal products; contribution patient ... See more keywords