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Real World Evidence (RWE) 201 – Canada – Methods and Guidelines for Reporting Real World Evidence

RWE 201 – Canada – Methods and Guidelines for Reporting Real World Evidence

 

Guidance for Reporting Real-World Evidence (RWE) [published May 2023] is a comprehensive document developed by the Canadian Agency for Drugs and Technologies in Health (CADTH) and Health Canada to provide clear guidance on the conduct, reporting, and appraisal of RWE studies for regulatory approval and health technology assessment (HTA) in Canada.

Guidance Objectives:

  1. Ensure regulators and HTA agencies have ample information for evaluating a study’s appropriateness for decision-making.
  2. Establish core RWE study reporting standards that align globally.
  3. Prioritize transparent reporting while acknowledging RWD and RWE challenges.

Implementation: Given the diverse sources and uses of RWD, this guidance offers flexibility for various RWE applications. Some RWE studies might not need all components described here. The guidance anticipates updates as the RWE field evolves.

RWE Utility:

While randomized controlled trials (RCTs) are the gold standard for establishing the safety and efficacy of health technologies, they often lack wider applicability due to specific target populations. RWE, derived from RWD, has benefits like the inclusion of under-represented populations in trials, such as children, older adults, and diverse ethnic groups. Its expanded sample sizes and longer follow-ups offer a richer dataset than clinical trials, providing insights into healthcare provider and patient perspectives about accessibility and usability of health technologies. For instance, RWE is instrumental in pharmacovigilance and monitoring adverse events.

RWE Limitations: Concerns include biases, confounding factors, and the lack of rigorous outcome ascertainment as seen in clinical trials. Clear standards are essential for transparent reporting of RWE.

RWE Guidance Structure: Guidance is provided for the reporting of 12 study components as follows:

  1. Research Questions and Study Design
  2. Setting and Context
  3. Data Specifications, Access, Cleaning Methods, and Linkage
  4. Data Sources, Data Dictionary, and Variables
  5. Participants
  6. Exposure Definitions and Comparators
  7. Outcomes
  8. Bias, Confounding, and Effect Modifiers or Subgroup Effects
  9. Statistical Methods
  10. Study Findings
  11. Interpretation and Generalizability
  12. Limitations

In summary, this recent guidance aims to harmonize global and Canadian reporting standards for RWE through expert consensus. It includes a Recommendations Checklist to ensure compliance and is designed to be adaptable across different types of RWE studies, laying the groundwork for best practices and methodological rigor in using RWE for regulatory and HTA purposes.

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NORWAIT – A Cautionary Tale

October 14, 2023|2023, RWE 201|

RWE 201 - NORWAIT – A Cautionary Tale Norwait Study: https://rwr-regs.com/norway-norwait-study-deterioration-for-several-participants-in-a-controversial-observational-cancer-study/Observational studies, as the term implies, typically involve low to no risk for patients. Their main purpose is to [...]

Sweden – Pioneering the Use of RWD

October 14, 2023|2023, RWE 201|

RWE 201 - Sweden – Pioneering the Use of RWD Cancer Registry: https://www.socialstyrelsen.se/statistik-och-data/register/cancerregistret/Sweden has been a pioneer in several real-world data (RWD) and real-world evidence (RWE) initiatives, particularly in [...]

Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research

October 14, 2023|2023, RWE 201|

RWE 201 - Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research  CCNCE Reflection Paper (Apr 2023): https://www.aifa.gov.it/documents/20142/1808580/Criticita_etiche_ricerca_osservazionale_06.04.2023.pdfThe Italian National Coordination [...]

Real World Evidence (RWE) 201 – Canada – Methods and Guidelines for Reporting Real World Evidence2023-08-30T08:52:31+00:00

Real World Evidence (RWE) 201 – Health Canada’s 15 Key Elements for Protocol Development

RWE 201 – Health Canada’s 15 Key Elements for Protocol Development

 

Health Canada acknowledges that RWE can be particularly useful in areas where conducting controlled clinical trials is challenging or not feasible. By leveraging RWE, regulatory authorities can gain additional insights into drug safety and efficacy, ultimately improving access to safe and effective drugs for patients.

Protocol development is a critical step in ensuring the quality and validity of RWE studies. When designing a research protocol, several key elements should be considered:

[1] Research Question: Define the objectives, aims, and rationale, supplemented by a literature review.

[2] Milestones: Include timelines for data collection, progress reports, and study registration.

[3] Research Design: Clearly specify data type, measures of occurrence, and adverse events reporting.

[4] Study Populations: Define the study group and discuss its generalizability.

[5 & 6] Exposure & Outcome Measurement: Define, measure, and validate the exposure and outcomes pertinent to the study.

[7] Bias Mitigation: Identify potential biases and strategies to counter them.

[8] Effect Measure Modification: The protocol should address the collection of items that could modify the effect being studied and explain how they are included in the analysis.

[9] Data Sources: Describe sources and methods of data collection, including coding systems.

[10] Statistical Analysis: Detail statistical approaches, handling of missing data, and sensitivity analyses.

[11] Data Management: Outline storage, software, and quality control mechanisms.

[12] Feasibility & Limitations: Discuss the study’s viability and prospective mitigation strategies.

[13] Ethics & Data Protection: Emphasize participant protection, confidentiality, and ethical compliance.

[14] Amendments & Deviations: Document any changes to the original protocol.

[15] Communication Plan: Address how study results will be communicated and ensure transparency.

Data Quality in RWE:

[1] Prospective Data: Ensure transparency, defined procedures, and documentation for high-quality data.

[2] Retrospective Data: Despite its challenges, maintain high standards and traceability to the source.

In conclusion, these key elements of protocol development provide a framework for conducting high-quality RWE studies. Protocol development and data quality are crucial factors in ensuring the validity and reliability of RWE studies. By following good research practices and addressing elements such as research question, milestones, study design, data sources, statistical analysis, and data management, stakeholders can generate high-quality evidence to support regulatory submissions. Incorporating RWE into the drug product life cycle improves access to safe and efficacious drugs for patients, ultimately benefiting public health [1].

 

References:

[1] Health Canada – Elements of Real World Data/Evidence Quality throughout the Prescription Drug Product Life Cycle (March 2019)

Link: https://www.canada.ca/en/services/health/publications/drugs-health-products/real-world-data-evidence-drug-lifecycle-report.html

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NORWAIT – A Cautionary Tale

October 14, 2023|2023, RWE 201|

RWE 201 - NORWAIT – A Cautionary Tale Norwait Study: https://rwr-regs.com/norway-norwait-study-deterioration-for-several-participants-in-a-controversial-observational-cancer-study/Observational studies, as the term implies, typically involve low to no risk for patients. Their main purpose is to [...]

Sweden – Pioneering the Use of RWD

October 14, 2023|2023, RWE 201|

RWE 201 - Sweden – Pioneering the Use of RWD Cancer Registry: https://www.socialstyrelsen.se/statistik-och-data/register/cancerregistret/Sweden has been a pioneer in several real-world data (RWD) and real-world evidence (RWE) initiatives, particularly in [...]

Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research

October 14, 2023|2023, RWE 201|

RWE 201 - Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research  CCNCE Reflection Paper (Apr 2023): https://www.aifa.gov.it/documents/20142/1808580/Criticita_etiche_ricerca_osservazionale_06.04.2023.pdfThe Italian National Coordination [...]

Real World Evidence (RWE) 201 – Health Canada’s 15 Key Elements for Protocol Development2023-08-20T18:01:03+00:00

Real World Evidence (RWE) 201 – Beyond Clinical Trials: Health Canada’s Commitment to Real World Evidence

RWE 201 – Beyond Clinical Trials: Health Canada’s Commitment to Real World Evidence

 

Health Canada is dedicated to enhancing drug accessibility, affordability, and correct usage within the country. To achieve this, it’s maximizing (optimizing) the use of real world evidence (RWE) for its regulatory decisions. RWE is generated from real world data (RWD) and offers an alternative source for assessing drug safety, efficacy, and effectiveness. This is especially crucial when conventional clinical trials are not suitable [1].

While clinical trials are often deemed the gold standard for drug evaluation, they might not always be feasible or representative of the broader population. The rising availability of RWD globally can provide deeper insights, particularly for specific patient groups, potentially leading to faster and broader drug access.

Incorporating RWE into the drug regulation process is a priority for Health Canada, and they’re continually refining their expertise in this realm. To solidify the role of RWE, they are collaborating with key stakeholders like industry representatives and health technology assessment entities. A recent joint document with Canada’s Drug and Health technology Agency (published May 2023) provides guidance on the effective use of RWE (Guidance for Reporting Real-World Evidence). Insights from this collaborative venture will be explored more in this series [2] [3].

Quality assurance of RWE is paramount. Factors to determine its quality encompass data reliability, study design, statistical analysis, and results interpretation. Protocol development is a cornerstone of high-quality RWE. Health Canada has pinpointed fifteen essential aspects to address during this phase, from research design to study results communication. Especially with retrospective data, maintaining RWE quality demands rigorous research questions, validated data elements, and transparency [4].

For drug submissions utilizing RWE, Health Canada sets forth stringent review requirements. Sponsors must transparently present their use of RWE, give reasons for its choice over traditional methods, detail RWE collection and analysis processes, and tackle potential biases. Pre-submission consultations with Health Canada are also advisable.

In conclusion, Health Canada’s proactive stance on RWE underpins its commitment to drug accessibility and affordability. By harnessing RWE, they aim to cater to groups often overlooked in clinical trials, address certain patient or disease challenges, and amplify drug accessibility rates. Emphasizing collaboration, protocol development, and data quality, Health Canada is setting a robust, transparent standard for RWE utilization throughout the drug’s life cycle.

 

References:

[1] Health Canada – Optimizing the Use of Real World Evidence to Inform Regulatory Decision-Making (April 2019)

Link: https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/announcements/optimizing-real-world-evidence-regulatory-decisions.html

[2] Canada’s Drug and Health technology Agency (CDHTA) – Real-World Evidence for Decision-Making (August 2023)

Link: https://www.cadth.ca/real-world-evidence-decision-making

[3] Canada’s Drug and Health technology Agency (CDHTA) – Guidance for Reporting Real-World Evidence (May 2023)

Link: https://www.cadth.ca/guidance-reporting-real-world-evidence

[4] Health Canada – Elements of Real World Data/Evidence Quality throughout the Prescription Drug Product Life Cycle (March 2019)

Link: https://www.canada.ca/en/services/health/publications/drugs-health-products/real-world-data-evidence-drug-lifecycle-report.html

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NORWAIT – A Cautionary Tale

October 14, 2023|2023, RWE 201|

RWE 201 - NORWAIT – A Cautionary Tale Norwait Study: https://rwr-regs.com/norway-norwait-study-deterioration-for-several-participants-in-a-controversial-observational-cancer-study/Observational studies, as the term implies, typically involve low to no risk for patients. Their main purpose is to [...]

Sweden – Pioneering the Use of RWD

October 14, 2023|2023, RWE 201|

RWE 201 - Sweden – Pioneering the Use of RWD Cancer Registry: https://www.socialstyrelsen.se/statistik-och-data/register/cancerregistret/Sweden has been a pioneer in several real-world data (RWD) and real-world evidence (RWE) initiatives, particularly in [...]

Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research

October 14, 2023|2023, RWE 201|

RWE 201 - Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research  CCNCE Reflection Paper (Apr 2023): https://www.aifa.gov.it/documents/20142/1808580/Criticita_etiche_ricerca_osservazionale_06.04.2023.pdfThe Italian National Coordination [...]

Real World Evidence (RWE) 201 – Beyond Clinical Trials: Health Canada’s Commitment to Real World Evidence2023-08-20T17:12:57+00:00

Real World Evidence (RWE) 201 – A Tale of Two Regulatory Paths: Non-Interventional Studies in the USA

RWE 201 – A Tale of Two Regulatory Paths: Non-Interventional Studies in the USA

 

Non-interventional studies are different from randomized controlled trials (RCTs). Instead of actively intervening in a patient’s healthcare, researchers just observe and record what happens naturally during the patients normal (routine) healthcare journey. The data from these studies is useful because it helps generate ideas for further research, flag potential safety concerns, or even provide additional evidence for regulatory decisions, like updating product labels or expanding the use of approved medical products.

Now, if you’re thinking of submitting this data to the FDA, there are certain regulations (rules) you must follow. In the U.S., there are two main pathways:

[1] For studies that will be submitted to the FDA: There’s a ‘checklist’ to follow, such as complying with certain sections of the Code of Federal Regulations (21 CFR 11, 21 CFR 50, 21 CFR 56, etc.) and listing your study on public databases like clinicaltrials.gov.

[2] For studies that won’t be submitted to the FDA: The requirements are a bit different, focusing more on the Common Rule (45 CFR 46), but still following similar safety rules, such as 21 CFR 314 (drugs) and 21 CFR 600 (biologics).

Despite the differences, there are common themes between the two. Both need ethical green lights in the form of Institutional Review Board (IRB) approvals, and participant informed consent is a must. With health data involved, ensuring privacy is non-negotiable, meaning regulations like HIPAA come into play. And regardless of the regulatory pathway, the study should be well-designed and use rigorous methodology to ensure data quality, integrity, and reliability. This includes clear definitions of exposures, outcomes, confounders, and other variables.

Because it’s hard to predict if non-interventional study’s results (RWE) might be submitted to the FDA, it’s a good idea to play it safe and align with FDA regulatory requirements and guidelines from the start.

In both cases, it’s essential to follow good epidemiological and statistical practices to ensure that the findings are robust, valid, and interpretable. Non-interventional studies, by their nature, are more susceptible to confounding and bias than RCTs, so it’s crucial to apply methods that address or mitigate these potential pitfalls.

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NORWAIT – A Cautionary Tale

October 14, 2023|2023, RWE 201|

RWE 201 - NORWAIT – A Cautionary Tale Norwait Study: https://rwr-regs.com/norway-norwait-study-deterioration-for-several-participants-in-a-controversial-observational-cancer-study/Observational studies, as the term implies, typically involve low to no risk for patients. Their main purpose is to [...]

Sweden – Pioneering the Use of RWD

October 14, 2023|2023, RWE 201|

RWE 201 - Sweden – Pioneering the Use of RWD Cancer Registry: https://www.socialstyrelsen.se/statistik-och-data/register/cancerregistret/Sweden has been a pioneer in several real-world data (RWD) and real-world evidence (RWE) initiatives, particularly in [...]

Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research

October 14, 2023|2023, RWE 201|

RWE 201 - Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research  CCNCE Reflection Paper (Apr 2023): https://www.aifa.gov.it/documents/20142/1808580/Criticita_etiche_ricerca_osservazionale_06.04.2023.pdfThe Italian National Coordination [...]

Real World Evidence (RWE) 201 – A Tale of Two Regulatory Paths: Non-Interventional Studies in the USA2023-08-20T15:40:46+00:00

Real World Evidence (RWE) 201 – FDA’s Advancing RWE Program

RWE 201 – FDA’s Advancing RWE Program

Real-world evidence is transforming the regulatory landscape, enabling the FDA to make informed decisions based on robust data from real-world settings. In line with this, the FDA has launched the Advancing Real-World Evidence (RWE) Program, aiming to harness the benefits of RWD to support new labelling claims, expand indications for approved medical products, and fulfil post-approval study requirements.

Goals of the Program:

  1. Identify strategies for creating RWE that satisfies regulatory needs for labelling effectiveness or post-approval study obligations.
  2. Establish agency protocols for consistent RWE decision-making and shared knowledge.
  3. Encourage public understanding of RWE’s attributes for informed regulatory choices through public discussions.

Eligibility:

  1. Sponsors must have a pre-IND or IND number for their medical product
  2. The RWE proposal should aim at supporting labelling for effectiveness or meeting post-approval study demands
  3. Both FDA and sponsor must consent to publicly disclose the study design

Selection:

  1. The FDA welcomes all fitting RWE proposals. Selection is based on data’s suitability, study design quality, regulatory conduct, and diverse data sources and methodologies

Submission Process:

  1. Semi-annual submission deadlines: 31 March and 30 September, continuing until 31 March 2027
  2. Sponsors will be told whether their meeting request has been accepted within 45 days post-deadline. Successful applicants can apply for an additional 3 follow-up meetings.

Initial Meeting Request Content – Sponsors must provide:

  1. Product name
  2. IND number
  3. Study purpose (new labelling/post-approval study)
  4. Proposed indication
  5. Product development history
  6. Rationale for RWE approach
  7. Study design overview
  8. Non-disclosable design elements and reasons for their omission
  9. Study design details: objectives, design, eligibility, endpoints, treatment, comparator, concomitant therapies
  10. Data sources: category, description, validation, linkage, additional data collection.
  11. Analysis plan: sample size, analytic plan, confounding factors, follow-up period, data handling
  12. Other factors: study pre-specification, patient data availability, human subject protection

Disclosure Agreement:

Before initial meetings, FDA and sponsors decide on public data disclosure. Certain details, like sponsor name and patient data, are excluded from disclosure. However, unique disclosure considerations are allowed. Participation is voluntary.

In conclusion, the FDA’s Advancing RWE Program offers a structured approach for sponsors to collaborate with the FDA and harness the potential of RWE in label extensions and post market studies.

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NORWAIT – A Cautionary Tale

October 14, 2023|2023, RWE 201|

RWE 201 - NORWAIT – A Cautionary Tale Norwait Study: https://rwr-regs.com/norway-norwait-study-deterioration-for-several-participants-in-a-controversial-observational-cancer-study/Observational studies, as the term implies, typically involve low to no risk for patients. Their main purpose is to [...]

Sweden – Pioneering the Use of RWD

October 14, 2023|2023, RWE 201|

RWE 201 - Sweden – Pioneering the Use of RWD Cancer Registry: https://www.socialstyrelsen.se/statistik-och-data/register/cancerregistret/Sweden has been a pioneer in several real-world data (RWD) and real-world evidence (RWE) initiatives, particularly in [...]

Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research

October 14, 2023|2023, RWE 201|

RWE 201 - Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research  CCNCE Reflection Paper (Apr 2023): https://www.aifa.gov.it/documents/20142/1808580/Criticita_etiche_ricerca_osservazionale_06.04.2023.pdfThe Italian National Coordination [...]

Real World Evidence (RWE) 201 – FDA’s Advancing RWE Program2023-08-21T11:58:11+00:00

Real World Evidence (RWE) 201 – FDA’s RWE Considerations Draft Guidance

RWE 201 – FDA’s RWE Considerations Draft Guidance

The 21st Century Cures Act, signed into law in 2016, mandated the FDA to establish a framework for the evaluation of RWE in regulatory decision-making. This draft FDA guidance document forms part of the FDAs RWE program and addresses several key regulatory considerations for the use of RWE in regulatory decision-making. Let’s explore each of these considerations in more detail:

FDA Draft Guidance – Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products (Dec 2021): https://www.fda.gov/media/154714/download

[1] Applicability of 21 CFR Part 312: Interventional studies involving drugs generally meet the definition of a clinical investigation (clinical trial) and are subject to FDA regulations under 21 CFR Part 312. On the other hand, non-interventional studies analyse data reflecting the use of a marketed drug in routine medical practice, as such non-interventional studies are not clinical investigations as defined under 21 CFR 312.3 and do not require an investigational new drug application (IND).

[2] Early Engagement with the FDA: Transparency is essential in non-interventional studies to ensure the reliability of the data collected. Sponsors should engage with the FDA in the early stages of study design, provide draft versions of protocols and statistical analysis plans for review, and finalise these documents before conducting the study analyses.

[3] RWD Data Access: Sponsors should discuss with the FDA their expectations regarding access to RWD for their development program. They should ensure that patient-level data from RWD can be submitted to the FDA when required and have agreements in place with third-party data owners to provide relevant data for inspection.

[4] Study Monitoring: Study monitoring in non-interventional studies focuses on maintaining the reliability of RWD and data integrity. Sponsors should ensure that applicable human subject protection requirements are met (IRB approval and informed consent – 21 CFR 50 and 21 CFR 56) and consider a risk-based approach to study oversight.

[5] Safety Reporting: Adverse events identified during non-interventional studies must be reported to the FDA in accordance with postmarketing safety reporting regulations (21 CFR 314.80).

[6] Other Sponsor Responsibilities: Sponsors are responsible for various activities related to the design, conduct, and oversight of non-interventional studies. These activities include selecting qualified researchers, ensuring compliance with protocols and regulations (e.g., 21 CFR 11), maintaining study records, registering the study on clinicaltrials.gov, and providing access to relevant records upon request.

In summary, this FDA guidance document provides valuable insights into the considerations for leveraging RWE to support regulatory decisions. By adhering to the guidance and engaging with the FDA early in the study design process, industry stakeholders can effectively utilise RWE to enhance the safety and effectiveness evaluation of drugs and biological products.

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NORWAIT – A Cautionary Tale

October 14, 2023|2023, RWE 201|

RWE 201 - NORWAIT – A Cautionary Tale Norwait Study: https://rwr-regs.com/norway-norwait-study-deterioration-for-several-participants-in-a-controversial-observational-cancer-study/Observational studies, as the term implies, typically involve low to no risk for patients. Their main purpose is to [...]

Sweden – Pioneering the Use of RWD

October 14, 2023|2023, RWE 201|

RWE 201 - Sweden – Pioneering the Use of RWD Cancer Registry: https://www.socialstyrelsen.se/statistik-och-data/register/cancerregistret/Sweden has been a pioneer in several real-world data (RWD) and real-world evidence (RWE) initiatives, particularly in [...]

Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research

October 14, 2023|2023, RWE 201|

RWE 201 - Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research  CCNCE Reflection Paper (Apr 2023): https://www.aifa.gov.it/documents/20142/1808580/Criticita_etiche_ricerca_osservazionale_06.04.2023.pdfThe Italian National Coordination [...]

Real World Evidence (RWE) 201 – FDA’s RWE Considerations Draft Guidance2023-08-21T16:03:17+00:00

Real World Evidence 201 – FDAs RWE Framework

RWE 201 – FDAs RWE Framework

Real World Evidence (RWE) 201 – FDAs RWE Framework

RWE 201: https://rwr-regs.com/rwe-201/

The FDA’s Real-World Evidence (RWE) Program framework, established under the 21st Century Cures Act, focuses on several key aspects:

[1] Scope of RWE Program Under 21st Century Cures Act: The program aims to evaluate the potential use of RWE to support the approval of a new indication for a drug already approved under section 505(c) of the FD&C Act or to help support or satisfy drug post-approval study requirements (e.g., FDAs Advancing RWE Program).

[2] Current Use of Real-World Data (RWD) for Evidence Generation: RWD sources, such as electronic health records, medical claims, and billing data, can be used for data collection and to develop analysis infrastructure to support various study designs, including randomized clinical trials and observational studies.

Current Uses of RWE:

  • Complementing traditional clinical trial data.
  • Supporting post-market commitments or requirements.
  • Label expansions (e.g., adding new indications for approved drugs).
  • Observational studies to understand product safety and effectiveness.

[3] Generating Evidence Regarding Safety and Effectiveness: Historically, there has been limited use of RWE to support effectiveness. Advances in the availability and analysis of RWD have increased the potential for generating robust RWE to support FDA regulatory decisions.  However, the value of using RWE to support regulatory decisions on drug efficacy and effectiveness has yet to be established.  To be explored further when we move to the EU and the EMAs RWE 2025 Vision.

[4] Framework for Evaluating RWD/RWE for Use in Regulatory Decisions: The FDA’s framework for evaluating RWD and RWE involves assessing the fitness of RWD (sources) for use in regulatory decisions, potential for study designs using RWD to support effectiveness, and regulatory considerations for study designs using RWD.

Data Quality and Reliability:

  • Not all RWD is fit for regulatory decisions. The FDA assesses the relevance and reliability of RWD.
  • Sources of RWD must be transparent, and any transformations to derive RWE must be robust and valid.

Methodological Challenges:

  • There are inherent biases in RWD because they are observational in nature
  • FDA focuses on minimising biases and promoting the use of appropriate statistical methods.

The framework aims to facilitate the acceleration of medical product development and the delivery of medical innovations for patients in need. It also distinguishes between the sources of RWD and the evidence derived from that data, as evaluating RWE in the context of regulatory decision-making depends on the evaluation of the methodologies used to generate the evidence and the reliability and relevance of the underlying RWD.

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NORWAIT – A Cautionary Tale

October 14, 2023|2023, RWE 201|

RWE 201 - NORWAIT – A Cautionary Tale Norwait Study: https://rwr-regs.com/norway-norwait-study-deterioration-for-several-participants-in-a-controversial-observational-cancer-study/Observational studies, as the term implies, typically involve low to no risk for patients. Their main purpose is to [...]

Sweden – Pioneering the Use of RWD

October 14, 2023|2023, RWE 201|

RWE 201 - Sweden – Pioneering the Use of RWD Cancer Registry: https://www.socialstyrelsen.se/statistik-och-data/register/cancerregistret/Sweden has been a pioneer in several real-world data (RWD) and real-world evidence (RWE) initiatives, particularly in [...]

Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research

October 14, 2023|2023, RWE 201|

RWE 201 - Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research  CCNCE Reflection Paper (Apr 2023): https://www.aifa.gov.it/documents/20142/1808580/Criticita_etiche_ricerca_osservazionale_06.04.2023.pdfThe Italian National Coordination [...]

Real World Evidence 201 – FDAs RWE Framework2023-08-16T15:06:53+00:00

Real World Evidence 201 – The 21st Century Cures Act

RWE 201 – The 21st Century Cures Act

The 21st Century Cures Act (CURES 1.0), signed into law in the U.S. in December 2016, aimed to accelerate medical product development and foster innovation and advances in patient care. One of the key aspects of the Act is its focus on utilizing Real-World Evidence (RWE), transforming the way healthcare providers approach patient care and medical research.

RWE refers to data collected from various sources outside of traditional clinical trials, such as Electronic Health Records (EHR), claims and billing data, patient registries, and patient-generated data. The 21st Century Cures Act recognizes the importance of RWE in improving the efficiency of the drug development process by supporting its use in the approval of new indications for approved drugs, as well as satisfying post-approval study requirements (i.e., PMCs and PMRs).

By leveraging RWE, the FDA can make regulatory decisions using more diverse and inclusive patient data, reflecting a broader patient population and potentially leading to more personalized and effective treatments. It also has the potential to reduce costs and time needed for traditional trials, accelerating the availability of life-saving drugs.

Critics of using RWE in regulatory decisions cite concerns about data quality, reliability, and potential biases. To address these concerns, the FDA has been working to develop guidance and standards for collecting and evaluating RWE, which we’ll explore later.

The 21st Century Cures Act encourages the use of real-world evidence (RWE) in regulatory decision-making in several ways:

[1] Developing an RWE Framework and Guidance: The Cures Act required the FDA to develop a framework and guidance for evaluating RWE in the context of regulating drugs and biologics. This helps support approvals of new indications for previously approved drugs and fulfil post-approval study requirements.

[2] Promoting the Use of RWE in Regulatory Decisions: The Act places additional focus on using RWE to support regulatory decision-making, such as approving new indications for medical devices without requiring new clinical trials.

[3] Modernizing Clinical Trial Designs: The Cures Act supports the modernization of clinical trial designs, including the use of RWE and clinical outcome assessments, which can lead to more efficient clinical trials and faster access to new treatments for patients.

In summary, the 21st Century Cures Act’s emphasis on RWE signifies a shift towards a more modern and flexible approach to medical product development. It fosters collaboration between regulatory agencies, healthcare providers, and industry to utilize real-world data in improving patient outcomes, speeding up innovation, and enhancing the efficiency of healthcare delivery.

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NORWAIT – A Cautionary Tale

October 14, 2023|2023, RWE 201|

RWE 201 - NORWAIT – A Cautionary Tale Norwait Study: https://rwr-regs.com/norway-norwait-study-deterioration-for-several-participants-in-a-controversial-observational-cancer-study/Observational studies, as the term implies, typically involve low to no risk for patients. Their main purpose is to [...]

Sweden – Pioneering the Use of RWD

October 14, 2023|2023, RWE 201|

RWE 201 - Sweden – Pioneering the Use of RWD Cancer Registry: https://www.socialstyrelsen.se/statistik-och-data/register/cancerregistret/Sweden has been a pioneer in several real-world data (RWD) and real-world evidence (RWE) initiatives, particularly in [...]

Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research

October 14, 2023|2023, RWE 201|

RWE 201 - Italy – CCNCE – Ethical and Regulatory Issues in the Processing of Patient Data in Observational Research  CCNCE Reflection Paper (Apr 2023): https://www.aifa.gov.it/documents/20142/1808580/Criticita_etiche_ricerca_osservazionale_06.04.2023.pdfThe Italian National Coordination [...]

Real World Evidence 201 – The 21st Century Cures Act2023-08-16T14:54:57+00:00
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