Imposed registries within the European postmarketing surveillance system: Extended analysis and lessons learned for regulators

Păcurariu, Alexandra; Plueschke, Kelly; Olmo, Carla Alonso; Kurz, Xavier

doi:10.1002/pds.4449

Cited by 8 publications

(8 citation statements)

References 3 publications

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Section: Introductionmentioning

confidence: 99%

“…Notwithstanding such potential, the under-use of patient registries in the regulatory context is striking [10, 11]. Of 335 products recommended for approval between 2005 and 2013 by the main scientific committee of the European Medicines Agency (EMA), the Committee for Medicinal Products for Human Use (CHMP), 31 registries were requested to fulfil a condition of the marketing authorisation, but by December 2017, just ten had been completed [11]. Potentially, registries could provide data permitting comparisons of outcomes from different treatments across different countries and healthcare settings over time as well as assessment of the impact of measures taken to minimise risks of medicinal products [12, 13].…”

Section: Introductionmentioning

confidence: 99%

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Patient Registries: An Underused Resource for Medicines Evaluation

et al. 2019

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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 for supporting regulatory decision-making, especially for products to treat rare diseases. Nevertheless, patient registries are greatly underused in regulatory assessments. Reasons include heterogeneity in registry design and in the data collected, even across registries for the same disease, as well as unreliable data quality and data sharing impediments. The Patient Registries Initiative was established by the European Medicines Agency in 2015 to support registries in collecting data suitable to contribute to regulatory assessments, especially post-authorisation safety and effectiveness studies.MethodsWe conducted a qualitative synthesis of the published observations and recommendations from an initiative-led multi-stakeholder consultation and four disease-specific patient registry workshops. We identified the primary factors facilitating the use of registry data in regulatory assessments. We generated proposals on operational measures needed from stakeholders including registry holders, patients, healthcare professionals, regulators, marketing authorisation applicants and holders, and health technology assessment bodies for implementing these.ResultsTen factors were identified as facilitating registry use for supporting regulatory assessments of medicinal products. Proposals on operational measures needed for implementation were categorised according to three themes: (1) nature of the data collected and registry quality assurance processes; (2) registry governance, informed consent, data protection and sharing; and (3) stakeholder communication and planning of benefit-risk assessments.ConclusionsThese are the first explicit proposals, from a regulatory perspective, on operational methods for increasing the use of patient registries in medicines regulation. They apply to registry holders, patients, regulators, marketing authorisation holders/applicants and healthcare stakeholders broadly, and their implementation would greatly facilitate the use of these valuable data sources in regulatory decision-making.Electronic supplementary materialThe online version of this article (10.1007/s40264-019-00848-9) contains supplementary material, which is available to authorized users.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Patient Registries: An Underused Resource for Medicines Evaluation

et al. 2019

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“…A predecessor to this study by Bouvy et al found that for 392 products that received a positive CHMP opinion from 2005-2013, there were 31 registries that were requested for 30 products (7.7%) in total (65% were product registries, 35% were disease registries) with 71% having a primary safety objective. 15,16 This frequent use of product registries led to the establishment of the EMA Patient Registry initiative supporting the use of existing patient registries for the postauthorization benefit-risk monitoring of medicinal products. 17 This was subsequently followed up with proposals for operational methods for increasing the use of patient registries in medicines regulation by addressing: the nature of the data collected and registry quality assurance processes; registry governance, informed consent, data protection, and sharing; and stakeholder communication and planning of benefit-risk assessments.…”

Section: Discussionmentioning

confidence: 99%

Marketing Authorization Applications Made to the European Medicines Agency in 2018–2019: What was the Contribution of Real‐World Evidence?

Flynn

Plueschke

Quinten

et al. 2021

Clin Pharma and Therapeutics

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108

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Information derived from routinely collected real‐world data has for a long time been used to support regulatory decision making on the safety of drugs and has more recently been used to support marketing authorization submissions to regulators. There is a lack of detailed information on the use and types of this real‐world evidence (RWE) as submitted to regulators. We used resources held by the European Medicines Agency (EMA) to describe the characteristics of RWE included in new marketing authorization applications (MAAs) and extensions of indication (EOIs) for already authorized products submitted to the EMA in 2018 and 2019. For MAAs, 63 of 158 products (39.9%) contained RWE with a total of 117 studies. For 31.7% of these products, the RWE submitted was derived from data collected before the planned authorization. The most common data sources were registries (60.3%) followed by hospital data (31.7%). RWE was mainly included to support safety (87.3%) and efficacy (49.2%) with cohort studies being the most frequently used study design (88.9%). For EOIs, 28 of 153 products (18.3%) contained RWE with a total of 36 studies. For 57.1% of these products, studies were conducted prior to the EOIs. RWE sources were mainly registries (35.6%) and hospital data (27.0%). RWE was typically used to support safety (82.1%) and efficacy (53.6%). Cohort studies were the most commonly used study design (87.6%). We conclude that there is widespread use of RWE to support evaluation of MAAs and EOIs submitted to the EMA and identify areas where further research is required.

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“…EU analyses of postauthorisation safety studies, and the use of registries are also available 21–25 . A review of EU centrally approved products between 2005–2013 indicated that EMA imposed PLEG through a registry in 4 and 29% of nonorphan and orphan medicinal products, respectively, and in 12 and 67% of products licensed under conditional or exceptional routes, respectively 21 .…”

Section: Pleg Characteristicsmentioning

confidence: 99%

Regulatory and health technology assessment advice on postlicensing and postlaunch evidence generation is a foundation for lifecycle data collection for medicines

Moseley

Vamvakas

Berntgen

et al. 2020

Brit J Clinical Pharma

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The understanding of the benefit risk profile, and relative effectiveness of a new medicinal product, are initially established in a circumscribed patient population through clinical trials. There may be uncertainties associated with the new medicinal product that cannot be, or do not need to be resolved before launch. Postlicensing or postlaunch evidence generation (PLEG) is a term for evidence generated after the licensure or launch of a medicinal product to address these remaining uncertainties. PLEG is thus part of the continuum of evidence development for a medicinal product, complementing earlier evidence, facilitating further elucidation of a product's benefit/risk profile, value proposition, and/or exploring broader aspects of disease management and provision of healthcare. PLEG plays a role in regulatory decision making, not only in the European Union but also in other jurisdictions including the USA and Japan. PLEG is also relevant for downstream decision‐making by health technology assessment bodies and payers. PLEG comprises studies of different designs, based on data collected in observational or experimental settings. Experience to date in the European Union has indicated a need for improvements in PLEG. Improvements in design and research efficiency of PLEG could be addressed through more systematic pursuance of Scientific Advice on PLEG with single or multiple decision makers. To date, limited information has been available on the rationale, process or timing for seeking PLEG advice from regulators or health technology assessment bodies. This article sets out to address these issues and to encourage further uptake of PLEG advice.

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Imposed registries within the European postmarketing surveillance system: Extended analysis and lessons learned for regulators

Cited by 8 publications

References 3 publications

Patient Registries: An Underused Resource for Medicines Evaluation

Patient Registries: An Underused Resource for Medicines Evaluation

Marketing Authorization Applications Made to the European Medicines Agency in 2018–2019: What was the Contribution of Real‐World Evidence?

Regulatory and health technology assessment advice on postlicensing and postlaunch evidence generation is a foundation for lifecycle data collection for medicines

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