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#12 Practical RWE – Training: Study Team Training

#12 Practical RWE – Training: Study Team Training

 

Study team training is crucial in both Real-World Evidence (RWE) studies and clinical trials, but the importance and focus of the training can vary significantly due to the different natures and objectives of these two types of studies.

  1. Nature of Data Collection:

– In clinical trials, data is collected under controlled conditions with predefined protocols. Training emphasizes adherence to these protocols, ensuring uniformity and minimizing bias.

– RWE studies, on the other hand, use data from sources like electronic health records, insurance claims, or patient registries. Training here focuses on understanding these data sources, identifying relevant data, and ensuring its quality and integrity.

  1. Regulatory Compliance and Guidelines:

– Clinical trials are highly regulated with strict guidelines for conduct. Training ensures compliance with these regulations, including patient safety and reporting requirements.

– RWE studies, while still subject to regulatory considerations, have different compliance requirements. Training helps in understanding these nuances and applying appropriate methodologies.

  1. Study Design and Methodology:

– Training for clinical trials includes detailed study design, placebo controls, blinding methods, and other experimental procedures.

– In RWE studies, training focuses more on observational study designs, statistical methods for handling real-world data, and techniques for reducing bias in non-randomized settings.

  1. Patient Interaction and Consent:

– Clinical trials usually require direct patient interaction, informed consent, and continuous monitoring. Training includes effective communication with participants and managing adverse events.

– RWE studies often use existing data where direct patient interaction is minimal. Training focuses more on data privacy, patient consent in the context of data use, and ethical considerations.

  1. Data Analysis and Interpretation:

– Clinical trial training includes specific methods for analyzing clinical data and interpreting results according to clinical endpoints.

– In RWE studies, training often covers a broader range of data analysis skills, including dealing with large datasets, diverse data types, handling missing data, and applying real-world evidence to clinical or policy decisions.

  1. Collaboration and Multidisciplinary Understanding:

– Both study types require understanding roles across disciplines. However, RWE studies often involve more diverse teams (e.g., data scientists, health economists, healthcare providers, policy makers) and thus require training in effective cross-disciplinary collaboration.

In summary, while study team training is fundamental in both clinical trials and RWE studies for ensuring quality, compliance, and reliability of results, the specific focus of the training differs to cater to the unique challenges and objectives of each study type.

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#12 Practical RWE – Training: Study Team Training2024-02-01T15:50:04+00:00

Real World Evidence (RWE) 201 – China’s Emerging Trend: Embracing Real-World Evidence in Drug Development and Regulation

RWE 201 – China’s Emerging Trend: Embracing Real-World Evidence in Drug Development and Regulation

–  Zhong et al., Advancing the Development of Real-World Data for Healthcare Research in – – – China: https://bmjopen.bmj.com/content/12/7/e063139

– NMPA Guidelines on the Use of RWE to Support Drug Development: https://redica.com/wp-content/uploads/NMPA_-Attachment_-_Guiding-Principles-of-Real-World-Data-Used-to-Generate-Real-World-Evidence-Trial_.pdf

– Hainan Boao Lecheng International Medical Tourism Pilot Zone: https://www.cde.org.cn/main/news/viewInfoCommon/e3cd052e638876697399de962b7354a4

China is increasingly embracing the use of real-world evidence (RWE) in research and drug development. The systematic use of RWE to support drug regulatory decision-making is still in its infancy in China, but national drug regulatory authorities have begun applying RWE in the review and approval process. The application and development of real-world data (RWD) and RWE in drug regulatory decision-making have been vigorously promoted in China.

One of the ways China is using RWE is to support decisions about whether a drug should be included in the reimbursement list and whether it should be renewed. The Hainan Boao Lecheng international medical tourism pilot zone, known as the Hainan model, has been used to generate RWE to support regulatory approval without conducting a trial in China. This type of evidence could also be used to support reimbursement decisions to minimize delay to market entry.

However, there are challenges to the wider adoption of RWE in China. These include limited research capacity and the need for more researchers with in-depth understanding of methodologies, data, and healthcare settings in China. There are also regulatory hurdles, such as the need for approval from the Human Genetic Resource Administration of China (HGRAC) for studies using healthcare RWD, and cross border data transfer security assessments and licences.

Despite these challenges, there are signs of progress. For example, the province of Hainan has pioneered the use of RWE, including for drugs and medical devices that are new to China. The National Medical Products Administration (NMPA) of China has also published guidelines on the use of RWE in supporting drug review, indication expansion, post-approval evaluation, and research and development of traditional Chinese medicine.

In conclusion, while the use of RWE in China is still developing, it is gaining recognition and is being increasingly used in drug development research and regulatory decision-making. This trend is likely to continue as China seeks to improve its healthcare system and drug development processes.

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Real World Evidence (RWE) 201 – China’s Emerging Trend: Embracing Real-World Evidence in Drug Development and Regulation2023-12-08T14:17:40+00:00

Poland – The Journey towards Real World Evidence and Digital Transformation

RWE 201 – Poland – The Journey towards Real World Evidence and Digital Transformation

 

Real World Evidence (RWE) and Real World Data (RWD) initiatives in Poland are still in the early stages of development, but there is a growing interest and recognition of their potential benefits. Key stakeholders in Poland have not yet fully embraced RWE as an evidence source, but there is a growing need for more evidence to allocate scarce resources. There is significant interest among physicians in practical evidence and a broad spectrum of possible actions that can be undertaken to improve the formal use of RWE in Poland. However, creating databases (registers) requires considerable financial and organisational effort, which creates a significant impediment to the effective development of RWE in Poland.

In terms of the implementation of the European Health Data Space (EHDS), Poland has shown support for the proposal, emphasizing that EHDS will contribute to the digitization of the healthcare sector in Poland. The patients will gain more rights when it comes to the control of their health data. Poland’s experience after engaging in the cross-border exchange of e-prescriptions and creating the COVID-19 certificate is very positive, giving prospects for the development of EHDS.

Poland has also adopted SNOMED CT as part of its digital health transformation. The country’s e-Health Centre, which implements key IT projects for the healthcare system, will represent Poland at SNOMED International. The renewed adoption of SNOMED CT in Poland will support the realization of another key element of the country’s strategy – ensuring that medical professionals are able to electronically document and easily share patient data.

However, there are challenges to overcome in obtaining RWD. These include the multitude of data sources, the need for a different process for RWD compared to clinical trials, ensuring the study is representative of real-life, managing research-naïve sites, and using the right tools.

In summary, while there are challenges to overcome, Poland is making strides in implementing RWE and RWD initiatives to support the implementation of the EHDS. The country is taking steps to digitize its healthcare sector, improve patient rights to health data, and facilitate the sharing of patient data among medical professionals. These initiatives are expected to contribute to the improvement of healthcare outcomes and the efficient allocation of resources in the country.

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Poland – The Journey towards Real World Evidence and Digital Transformation2023-11-05T13:02:30+00:00

RWE Guest Post – Does Real-World Evidence Play a Role in G-BA’s Benefit Assessments in Germany?

RWE 201 – RWE Guest Post – Does real-world evidence play a role in G-BA’s benefit assessments in Germany?

 

Guest: Anja Pownell

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

Introduction

The German Federal Joint Committee (G-BA), responsible for assessing the additional benefits of healthcare interventions, theoretically, has a relatively new tool at its disposal to make more informed decisions. Since 2020, G-BA has had the authority to request real-world evidence (RWE) data collection for certain products, providing a valuable addition to the traditional clinical trial data used in benefit assessments.

Real-World Evidence Data Collection requested by the G-BA

Some drugs, such as those with conditional approval, those authorized under exceptional circumstances, or orphan drugs, receive regulatory approval under special statuses. These approvals aim to make these treatments available to patients quickly, often leading to a lack of mature or sufficient data for conventional benefit assessments.

In these cases, the G-BA can request that manufacturers collect RWE on their use in routine clinical practice, a process known as “Anwendungsbegleitende Datenerhebung.” The G-BA can request this data collection from the manufacturer immediately upon market entry of a new drug or at a later time point. This data collection ensures better, more relevant information becomes available as patients receive the product in daily German clinical practice.

After several years, the G-BA re-evaluates the drug, incorporating the collected RWE alongside data from clinical trials. This comprehensive evaluation is intended to help the G-BA make more informed decisions regarding the additional benefit. Currently, RWE collection is underway for only two products, highlighting the novelty of this approach:

– Zolgensma in spinal muscle atrophy, final data expected by 2027.

– Tecartus in relapsed/refractory mantle cell lymphoma, final data expected in 2028.

The need and feasibility of RWE collection are being assessed for another 8 products.

Thus, it is too early to fully understand the role RWE will play in G-BA’s decision-making. These first re-assessments after RWE collection in 2027 or 2028 will undoubtedly provide valuable insights into how the G-BA will deal with the non-comparative RWE.

The Future of RWE in G-BA’s Decision-Making

As we move forward, it will be interesting to see how the utilization of RWE in benefit assessments evolves and expands, offering more insights and maintaining a high level of transparency in the decision-making process within the German healthcare system.

[Source: G-BA – Anwendungsbegleitende Datenerhebung] (https://www.g-ba.de/themen/arzneimittel/arzneimittel-richtlinie-anlagen/anwendungsbegleitende-datenerhebung/)

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RWE Guest Post – Does Real-World Evidence Play a Role in G-BA’s Benefit Assessments in Germany?2023-11-05T12:43:00+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 – 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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