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RWE Guest Post – Germany – From Concept to Evaluation: The Journey of RWE requests by the G-BA

RWE 201 – RWE Guest Post – Germany – From Concept to Evaluation: The Journey of RWE requests by the G-BA

 

Guest: Anja Pownell

German Market Access – Simplified: https://germanmarketaccesssimplified.com/

Introduction

Since 2020, the German Federal Joint Committee (G-BA) can request real-world evidence (RWE) to enhance its decision-making, as a crucial complement to traditional clinical trial data, for drugs that received regulatory approval under special statuses.

The process of requested RWE collection is meticulous and involves several key steps:

  1. Plenum Determination: The G-BA plenum decides whether RWE collection is necessary for a specific drug.
  2. Draft Concept Development: Within the next six months, the G-BA or IQWiG develops a draft concept that outlines the data collection’s specifics, including type, duration, extent, key questions, data collection methods, and data evaluation.
  3. External Expert Input: The G-BA shares the draft concept with external experts, including the Federal Institute for Drugs and Medical Devices (BfArM), the Paul-Ehrlich-Institute (PEI), medical societies, the Drug Commission of the German Medical Association (AkdÄ), and the manufacturer, for their input.
  4. Finalization of the Concept Plan: The G-BA plenum finalizes the concept plan within the next 6 months, ensuring that it covers all aspects of data collection. In some cases, the G-BA may restrict reimbursement to clinicians participating in the data collection, ensuring comprehensive data coverage.
  5. Data Collection and Evaluation: The manufacturer is then responsible for conducting the study, which can involve setting up new studies or utilizing existing registries to capture the required data. The manufacturer evaluates the collected data and shares the analyses with G-BA.
  6. Periodic Assessments: Every 1.5 years, the G-BA assesses the progress of data collection, evaluating whether the data collection is proceeding as planned, whether the data is sufficient for a benefit re-assessment, and whether any amendments to the concept plan are needed to enhance data meaningfulness.

The Role of IQWiG

With the introduction of the RWE collection requests, in 2020, the Institute for Quality and Efficiency in Health Care (IQWiG) developed criteria and detailed recommendations for manufacturers and registries on how to supplement the trial data with RWE (German: “Versorgungsnahe Daten”), such as registry data. If the RWE is of high quality, the IQWiG and G-BA can use it to determine the additional benefit of an intervention outside of a clinical trial.

According to IQWiG, “manufacturers don’t interpret the detailed requirements on RWE collection how we wish they did. Overall, the problems often were missing key information and differences between patient populations. It was not possible to conduct a meaningful benefit assessment with the data. Manufacturers often did identify the problems themselves but did not draw the necessary consequences.”

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RWE Guest Post – Germany – From Concept to Evaluation: The Journey of RWE requests by the G-BA2023-11-05T12:37:59+00:00

Spain – Championing the Ethical and Responsible Use of Real World Data

RWE 201 – Spain – Championing the Ethical and Responsible Use of Real World Data

 

Farmindustria Code of Conduct: https://codigoprotecciondatos.farmaindustria.org/sites/medicamentosinnovadores/docs/PRODF484450.pdf

In 2022, the Spanish Data Protection Agency (AEPD) gave its stamp of approval to the Farmaindustria “Code of Conduct regulating the processing of personal data in the field of clinical trials and other clinical research and pharmacovigilance.” These self-regulation standards focus on clinical studies involving medicines and are designed to help study sponsors understand the appropriate application of data protection regulations.

Support for Real-World Data and Evidence

This code of conduct acknowledges the significance of real-world data (RWD), which covers a broad spectrum of patient health information sourced from routine clinical practice, patients’ records, and even data acquired from wearable devices. Real-world evidence derived from this data is pivotal for observational studies with medicinal products. Such studies are instrumental in identifying the therapeutic effects of drugs, understanding adverse reactions, and gaining insights into medicine usage patterns without disrupting conventional clinical procedures.

Key Provisions from the Code of Conduct:

  1. Data Protection Impact Assessment (DPIA): Before the commencement of any clinical research, a thorough DPIA must be undertaken. This ensures that the data processing activities associated with the research are assessed for potential risks to participants’ rights and freedoms. If the assessment indicates high, unmitigated risks, relevant data protection authorities must be consulted prior to starting the research.
  1. Consent Stipulations: For any intended reuse of a participant’s data, a legitimate basis in line with data protection laws is mandatory. If the reuse involves coded data, it might not necessitate new consent, provided that certain regulatory stipulations are met, including robust security measures and a commitment to prevent re-identification.
  2. Broad Consent: Given the dynamic nature of clinical research, where full identification of data processing purposes might be challenging, participants can be approached for broad consent. This allows their data to be used for broader areas of investigation related to the original study. However, it’s imperative to keep participants informed, respect data protection principles, and secure necessary authorizations.

The comprehensive code of conduct provides clarity on various terminologies, from “patient’s records” to “trusted third party” and underscores the obligatory nature of the code for entities that choose to adhere. By fostering a standardized approach to data protection in clinical research, the code champions the ethical and responsible use of RWD, promoting transparency, trust, and advancing the field of medicine.

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Spain – Championing the Ethical and Responsible Use of Real World Data2023-11-05T12:33:16+00:00

EU – RWD/RWE is Embedded into the New EU Medicines Regulations

RWE 201 – EU – RWD/RWE is Embedded into the New EU Medicines Regulations

 

Coming Soon…New EU Medicines Regulations: https://health.ec.europa.eu/medicinal-products/pharmaceutical-strategy-europe/reform-eu-pharmaceutical-legislation_en

In 2023, the European Commission undertook an ambitious overhaul of its pharmaceutical regulations. This revision addresses foundational pharmaceutical legislation, specifically Regulation 726/2004, Directive 2001/83/EC, and rules for medicines tailored for children and rare diseases, namely Regulation 1901/2006 and Regulation 141/2000/EC.

Primary Aims:

– Ensure all EU patients access safe, effective, and affordable medicines promptly and fairly.

– Bolster medicine supply security across the EU.

– Propagate an innovation-centric environment for medicine R&D in Europe.

– Pivot towards environmentally sustainable medicines.

– Confront antimicrobial resistance and environmental pharmaceutical contamination through a holistic One Health approach.

Key Points of the Revision:

  1. Individual Patient Data: Regulators can now request structured individual patient data from clinical studies, promoting data-driven benefit-risk assessments for medicines throughout their life cycle (Recital 63 of MP-R).
  2. Transparency of Public Support: There’s now a mandate to disclose any direct financial backing received from public authorities for medicine R&D, fostering accountability and transparency (Recital 131 of MP-D).
  3. Patient Representation: The CHMP (Committee for Medicinal Products for Human Use) and PRAC (Pharmacovigilance Risk Assessment Committee) now include patient representatives, enriching patient voices in decisions.
  4. Real World Data (RWD): The revision endorses the use of health data, particularly RWD, for regulatory decision-making. Through systems like the DARWIN and European Health Data Space infrastructure, the agency can harness supercomputing, AI, and big data without jeopardizing privacy (Recital 60 of MP-R).
  5. Regulatory Sandbox: This introduces a controlled setting wherein innovative regulatory solutions can be tested, cultivated, and authenticated under scrutiny (Articles 2(12), 113-155 of MP-R).
  6. Compassionate Use Programs: Provisions for these programs, which offer early medicinal product access, have been strengthened. It’s vital to collect data from these programs to evaluate the benefit-risk ratio of medicines (Recital 57 & Article 26(4) of MP-R).
  7. Comparative Effectiveness: By repurposing medicines and leveraging comparative trial data, patients will have expedited access to novel treatments. Such data assists authorities in ascertaining a medicine’s cost-effectiveness.
  8. Relative Effectiveness: The EU has devised a scientific, evidence-based methodology to gauge the relative effectiveness of medicinal products. This focuses on a medicine’s added value against other health technologies but doesn’t extend to its marketing authorization (Recital 130 of MP-R).

In essence, this legislative revamp by the European Commission fosters a more patient-centric, transparent, and data-driven approach in the EU pharmaceutical landscape.

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EU – RWD/RWE is Embedded into the New EU Medicines Regulations2023-11-05T12:28:42+00:00

EU – EU’s Action Plan for Real-World Data (RWD) & RWE

RWE 201 – EU – EU’s Action Plan for Real-World Data (RWD) & RWE

 

The European Union has embarked on an ambitious journey to weave Real World Evidence (RWE) into the fabric of its healthcare system. The strategy, underscored by a comprehensive multi-year plan, focuses on harnessing the power of real-world data (RWD) through collaboration, standardization, and innovation. The infrastructure for this integration outlines seven pivotal components: Terminology, Findability, Access, Quality, Advanced Analytics, Use, and Use Cases.

 

RWE 2020 – The Foundation: The EU emphasizes on the importance of RWD Collection and RWE Generation. Key pillars of this foundational year include:

– Enhancing collaboration nationally and across borders.

– Creating a robust framework with effective methodologies.

– Recognizing the role of RWE in supplementing clinical trials.

– Aiming to support decisions by regulators, HTA, and payers.

– Advocating support for healthcare providers.

 

2021:

– Focuses on access to RWD and the establishment of the EU4Health Program.

– Introduction of new legislations: Artificial Intelligence Regulation, Health Technology Assessment Regulation, and laws on RWE Value and RWD Access.

– Notably, a step towards centralized data governance with the European Health Data Space Regulation.

 

2022:

– Expands on the RWE Use Cases with guidelines and draft laws.

– Emphasis on medicinal product regulations.

– The Data Governance Act aims to support data access and streamline data utilization.

 

2023:

– Proliferation of RWE use cases continues.

– Medicinal Products Regulation is in the limelight alongside explorations into single-arm trials by EMA.

– The year marks a notable focus on AI with the EMA’s guidance on Artificial Intelligence.2024:

– The direction is set towards advanced RWD analytics and a broader scope of RWE Use Cases.

– Introduces a Catalogue of NIS using RWD Sources and a focus on RWD findability with source catalogues = The replacement for the EU PAS Register

 

This roadmap not only outlines the EU’s commitment to innovative healthcare solutions but also sets a precedent for global health systems to leverage real-world data and evidence for enhanced patient care.

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EU – EU’s Action Plan for Real-World Data (RWD) & RWE2023-11-05T12:24:00+00:00

EU – EMA RWE Framework to Support Regulatory Decision Making

RWE 201 – EU – EMA RWE Framework to Support Regulatory Decision Making

 

EMA RWE Framework 2023: https://www.ema.europa.eu/en/documents/report/real-world-evidence-framework-support-eu-regulatory-decision-making-report-experience-gained_en.pdf

The European Medicine Agency (EMA) is actively working on creating a framework that will facilitate the use and establishes the value of RWE in decision-making throughout the entire drug lifecycle.

The current framework includes 3 evidence generation pathways: (1) In-house database e.g., The Health Improvement Network (THIN®), (2) DARWIN-EU, and (3) ‘Traditional’ primary data studies.

80% of the database studies, also referred to as ‘rapid data analytics’ , were delivered in less than 90 days.  Whereas delivery of the DARWIN-EU studies averaged 215 days. The majority of the studies focused on safety, rare diseases, and paediatrics. 

Key Learning

  1. Suitability of RWD Sources:

– RWD aids in enhancing evidence from clinical trials, aiding EU regulatory decisions.

– Most suitable studies addressed primary care scenarios due to available databases.

  1. Regulatory Context & Timelines:

– Comprehending regulatory nuances and evidence gaps is crucial for apt data selection and study design.

– In-house studies, given their speed, fit well with research questions having tight timelines.

  1. Building Capability & Capacity:

– Familiarity with RWD concepts, methodologies, and terms is essential for RWE acceptance.

  1. Usefulness for Decision-making:

– RWD studies bolster scientific evaluations in multiple regulatory situations.

– Understanding data source attributes aids in grasping study strengths, limitations, and RWE’s value.

  1. Other Process-related Aspects:

– Standardizing RWD study approaches, through agreed processes and templates, enhances RWE efficiency.

Recommendations

  1. RWD Source Expansion: Access more diverse data sources for better representation. Retain all three RWE pathways and collaborate with NCAs to maximize RWE generation.
  2. Enhance RWE Timeliness: Adopt proactive RWD study identification methods and increase RWE generation speed, possibly via tools like DARWIN EU.
  3. Boosting Capability & Capacity: Implement the Big Data Steering Group’s curriculum specifically for regulatory stakeholders.
  4. Improving Decision-making Utility: Detail strengths and limitations in future reports for improved interpretation.
  5. Process Refinement: Encourage reflections on RWE’s potential to bridge knowledge gaps and further unify processes.

Conclusions

Over the report’s timeframe, 27 regulator-led RWD studies were finalized, aiding various regulatory assessments like PRAC and SAWP. These studies spanned across safety, drug use, disease epidemiology, and more, primarily providing descriptive analyses. Some comparative analyses were also done. The range and design of these studies highlight the vast potential of the existing RWE resources and the Agency’s adaptability in addressing diverse research inquiries. However, further enhancements can be made to fully harness the EMA RWD study framework’s potential.

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EU – EMA RWE Framework to Support Regulatory Decision Making2023-11-05T12:17:32+00:00

EU – EMA’s 2025 Vision for RWE

RWE 201 – EU – EMA’s 2025 Vision for RWE

 

EMA 2025 Vision: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9299492/

EMA RWE Framework Report: https://www.ema.europa.eu/en/documents/report/real-world-evidence-framework-support-eu-regulatory-decision-making-report-experience-gained_en.pdf

The European Medicines Agency (EMA) isn’t simply relying on RWE submissions from companies. They’ve proactively embarked on developing the DARWIN EU framework, providing access to real-world data (RWD), enabling them to validate study designs, confirm disease prevalence for paediatric waivers, orphan drug designations, and more.

By 2025, EMA aims to:

  1. Fully integrate the use of RWE.
  2. Establish its value across all regulatory domains.

RWE and RWD are not novelties in the EU’s medicinal regulation. While their roles in safety monitoring and disease epidemiology are well-established, their evidentiary worth, especially for efficacy demonstration, is under continued assessment.

Key Developments:

[1] DARWIN EU: Launched in 2022, funded by the EU4Health program, it’s an EU-wide RWD network. It facilitates high-quality studies pivotal for regulatory decisions…by and for the EMA.

[2] RWE Framework Report (June 2023): The EMA detailed how RWE aids in:

– Designing and gauging the feasibility of upcoming studies.

– Ensuring representativeness and validity of completed research.

– Gauging disease prevalence and incidence.

– Understanding clinical management practices.

– Monitoring drug utilization in real-world scenarios.

– Evaluating the efficacy and safety of medical measures.

– Measuring the effectiveness of risk minimisation measure.

Significance: The COVID-19 pandemic highlighted the importance of RWE, as regulators utilized RWD to monitor the safety and efficacy of treatments. This emphasizes the regulator’s role, not just as a gatekeeper but as an adept analyst of RWE. The European Medicine Regulators Network (EMRN) recognizes this, investing in enhancing the skills of its workforce through recruitment, dedicated training, and promoting best practices.

In summary, RWE (generated from RWD) provides the EMA with a practical perspective on medical interventions in real-life scenarios, becoming indispensable in its regulatory role.

Given the EMA’s proactive stance on integrating RWE into their regulatory processes, how is your organization adapting its drug development strategies to ensure you’re not only keeping pace but also leveraging the full potential of real-world evidence?

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EU – EMA’s 2025 Vision for RWE2023-11-05T12:19:04+00:00

EU – EMA Big Data Steering Group Updates Its Workplan to Accelerate Transformation to Data-Driven Medicines Regulation

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EU – EMA Big Data Steering Group Updates Its Workplan to Accelerate Transformation to Data-Driven Medicines Regulation2023-11-05T11:05:33+00:00
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