Dataset Information

Use of real-world evidence in postmarketing medicines regulation in the European Union: a systematic assessment of European Medicines Agency referrals 2013-2017.

ABSTRACT:

Objectives

To assess the use, and evaluate the usefulness, of non-interventional studies and routinely collected healthcare data in postmarketing assessments conducted by the European Medicines Agency (EMA).

Design

We reviewed and systematically assessed all referrals to the EMA made due to safety or efficacy concerns that were evaluated between 1 January 2013 and 30 June 2017. We extracted information from the assessment report and the referral notification. Two reviewers independently assessed the contribution of non-interventional evidence to decision-making.

Results

The preliminary evidence leading to the assessment in 52 eligible referrals was mostly from spontaneous reports (cited in 26 of 52 referrals) and randomised trials (22/52). In contrast, many evidence types were used for the full assessment. Non-interventional studies were frequently used in the full assessment for the evaluation of product safety (31/52) and product efficacy (18/52). In particular, non-interventional studies were relied on for the evaluation of safety and efficacy in subgroups, the evaluation of safety relating to a rare adverse event, understanding product usage and misuse and for evaluation of the effectiveness of risk minimisation measures. The most common recommendations were changes to product information (43/52) and marketing authorisation withdrawal or suspension (12/52). In the majority of referrals, non-interventional evidence was judged to contribute to the decision made (30/52) and in three referrals it was the primary source of evidence.

Conclusions

European regulatory decision-making relies on multiple evidence types, particularly randomised trials, spontaneous reports and non-interventional studies. Non-interventional studies had an important role particularly for the characterisation and quantification of adverse events, the evaluation of product usage and for evaluating the effectiveness of regulatory action to minimise risk.

SUBMITTER: Brown JP

PROVIDER: S-EPMC6830614 | biostudies-literature | 2019 Oct

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Use of real-world evidence in postmarketing medicines regulation in the European Union: a systematic assessment of European Medicines Agency referrals 2013-2017.

Brown Jeremy Philip JP Wing Kevin K Evans Stephen J SJ Bhaskaran Krishnan K Smeeth Liam L Douglas Ian J IJ

BMJ open 20191028 10

<h4>Objectives</h4>To assess the use, and evaluate the usefulness, of non-interventional studies and routinely collected healthcare data in postmarketing assessments conducted by the European Medicines Agency (EMA).<h4>Design</h4>We reviewed and systematically assessed all referrals to the EMA made due to safety or efficacy concerns that were evaluated between 1 January 2013 and 30 June 2017. We extracted information from the assessment report and the referral notification. Two reviewers indepen ...[more]

PMID: 31662354

Similar Datasets

Project description:BackgroundMedications and their prices are key issues for healthcare. Although access to medicines at affordable prices had been specified as a key objective of the European Health Policy, it seems that these goals have not been achieved. Therefore, we attempted an evaluation of affordability of selected medicines at full prices.MethodsThe analysis concerned 2012 and was conducted between 2013 and 2015 in all the European Union (EU) countries divided into 3 groups depending on the date of their accession to the EU. Finally, we considered 9 originators used in the treatment of schizophrenia and multiple sclerosis. Information on drug prices were collected from pharmacies. Participation in the study was voluntary and anonymous in order to avoid accusations of advertising. To evaluate affordability, several factors were used (e.g. minimum earnings and Gini coefficient). Due to unavailability in some countries, the exact number of analyzed medicines varies.ResultsDrug prices vary significantly between EU Member States. Almost eleven fold difference was observed between Germany (EUR 1451.17) and Croatia (EUR 132.77) in relation to Interferone beta-1a 22 μg. Generally, prices were the highest in Germany. The cheapest drugs were found in various countries but never in the poorest ones like Bulgaria or Romania. Discrepancies in wages were observed too (the smallest minimum wage was EUR 138.00 in Bulgaria and the highest EUR 1801.00 in Luxembourg). Full price of olanzapine 5mg, however, was higher in Bulgaria (EUR 64.53) than, for instance, in Belgium (EUR 37.26).ConclusionsAnalyzed medications are still unaffordable for many citizens of the EU. Besides, access to medicines is also impaired e.g. due to parallel trade. Unaffordability of medications may lead to the patients' non-compliance and therefore to increased direct and indirect costs of treatment. Common European solutions are needed to achieve a real affordability and accessibility of medications.

Project description:ObjectivesStratified medicine is often heralded as the future of clinical practice. Key part of stratified medicine is the use of predictive biomarkers, which identify patient subgroups most likely to benefit (or least likely to experience harm) from an intervention. We investigated how many and what predictive biomarkers are currently included in European Medicines Agency (EMA) licensing.SettingEMA licensing.ParticipantsIndications and contraindications of all drugs considered by the EMA and published in 883 European Public Assessment Reports and Pending Decisions.Primary and secondary outcome measuresData were collected on: the type of the biomarker, whether it selected a subgroup of patients based on efficacy or toxicity, therapeutic area, marketing status, date of licensing decision, date of inclusion of the biomarker in the indication or contraindication and on orphan designation.Results49 biomarker-indication-drug (B-I-D) combinations were identified over 16 years, which included 37 biomarkers and 41 different drugs. All identified biomarkers were molecular. Six drugs (relating to 10 B-I-D combinations) had an orphan designation at the time of licensing. The identified B-I-D combinations were mainly used in cancer and HIV treatment, and also in hepatitis C and three other indications (cystic fibrosis, hyperlipoproteinaemia type I and methemoglobinaemia). In 45 B-I-D combinations, biomarkers were used as predictive of drug efficacy and in four of drug toxicity. It appeared that there was an increase in the number of B-I-D combinations introduced each year; however, the numbers were too small to identify any trends.ConclusionsGiven the large body of literature documenting research into potential predictive biomarkers and extensive investment into stratified medicine, we identified relatively few predictive biomarkers included in licensing. These were also limited to a small number of clinical areas. This might suggest a need for improvement in methods of translation from laboratory findings to clinical practice.

Dataset Information

Use of real-world evidence in postmarketing medicines regulation in the European Union: a systematic assessment of European Medicines Agency referrals 2013-2017.

Objectives

Design

Results

Conclusions

Publications

Use of real-world evidence in postmarketing medicines regulation in the European Union: a systematic assessment of European Medicines Agency referrals 2013-2017.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure