USA | Draft FDA Non-Interventional Study Guidance
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USA | FDA Finalises Rare Diseases Considerations Guidance
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USA | New Draft FDA Guidance: Collection of Race and Ethnicity Data in Clinical Trials and Clinical Studies for FDA-Regulated Medical Products
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USA | The Importance of Clinical Study Transparency
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- USA | Draft FDA Guidance – Postmarketing Approaches to Obtain Data on Populations Underrepresented in Clinical Trials for Drugs and Biological Products Gallery
USA | Draft FDA Guidance – Postmarketing Approaches to Obtain Data on Populations Underrepresented in Clinical Trials for Drugs and Biological Products
USA | Draft FDA Guidance – Postmarketing Approaches to Obtain Data on Populations Underrepresented in Clinical Trials for Drugs and Biological Products
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USA | Advancing Real-World Evidence Program Submission Deadline and New FAQs
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USA | Final FDA RWE Considerations Guidance Published
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Real World Evidence (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.
Real World Evidence (RWE) 201…THE END – A Landscape Analysis of Regional RWE Frameworks – The European Health Data Space and DARWIN-EU
stuart.mccully2023-12-08T14:58:47+00:00December 8, 2023|2023, RWE 201|
RWE 201 - THE END - A Landscape Analysis of Regional RWE Frameworks - The European Health Data Space and DARWIN-EU In our real world evidence (RWE) 201 series [...]
Real World Evidence (RWE) – Event Horizon
stuart.mccully2023-12-08T14:54:21+00:00December 8, 2023|2022, RWE 201|
RWE 201 - RWE Event Horizon The term "event horizon" in the context of pharmaceuticals, particularly when discussing the shift towards real-world evidence (RWE) in regulatory submissions, is metaphorically [...]
Real World Evidence (RWE) 201 – Advancing Healthcare in Australia: Embracing Real-World Evidence and Data in Medicine Regulation and Development
stuart.mccully2023-12-08T14:49:27+00:00December 8, 2023|2023, RWE 201|
RWE 201 - Advancing Healthcare in Australia: Embracing Real-World Evidence and Data in Medicine Regulation and Development Australia has several real-world evidence (RWE) initiatives to support healthcare [...]
Real World Evidence (RWE) 201 – South Korea’s Pioneering Approach: Integrating Real-World Evidence in Healthcare and Drug Development
stuart.mccully2023-12-08T14:39:24+00:00December 8, 2023|2023, RWE 201|
RWE 201 - South Korea's Pioneering Approach: Integrating Real-World Evidence in Healthcare and Drug Development South Korea has several initiatives to support healthcare and drug development using [...]
Real World Evidence (RWE) 201 – Taiwan: Integrating Real-World Evidence for Drug Development and Policy Making
stuart.mccully2023-12-08T14:34:38+00:00December 8, 2023|2023, RWE 201|
RWE 201 - Taiwan: Integrating Real-World Evidence for Drug Development and Policy Making Taiwan has several real-world evidence (RWE) initiatives to support healthcare and drug development. Over the past [...]
Real World Evidence (RWE) 201 – Revolutionizing Healthcare in Japan: The Rise of Real-World Evidence in Drug Development
stuart.mccully2023-12-08T14:31:09+00:00December 8, 2023|2023, RWE 201|
RWE 201 - Revolutionizing Healthcare in Japan: The Rise of Real-World Evidence in Drug Development Japan has several initiatives to support healthcare and drug development using real-world evidence (RWE). [...]
Real World Evidence (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:
- Identify strategies for creating RWE that satisfies regulatory needs for labelling effectiveness or post-approval study obligations.
- Establish agency protocols for consistent RWE decision-making and shared knowledge.
- Encourage public understanding of RWE’s attributes for informed regulatory choices through public discussions.
Eligibility:
- Sponsors must have a pre-IND or IND number for their medical product
- The RWE proposal should aim at supporting labelling for effectiveness or meeting post-approval study demands
- Both FDA and sponsor must consent to publicly disclose the study design
Selection:
- 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:
- Semi-annual submission deadlines: 31 March and 30 September, continuing until 31 March 2027
- 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:
- Product name
- IND number
- Study purpose (new labelling/post-approval study)
- Proposed indication
- Product development history
- Rationale for RWE approach
- Study design overview
- Non-disclosable design elements and reasons for their omission
- Study design details: objectives, design, eligibility, endpoints, treatment, comparator, concomitant therapies
- Data sources: category, description, validation, linkage, additional data collection.
- Analysis plan: sample size, analytic plan, confounding factors, follow-up period, data handling
- 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.
Real World Evidence (RWE) 201…THE END – A Landscape Analysis of Regional RWE Frameworks – The European Health Data Space and DARWIN-EU
stuart.mccully2023-12-08T14:58:47+00:00December 8, 2023|2023, RWE 201|
RWE 201 - THE END - A Landscape Analysis of Regional RWE Frameworks - The European Health Data Space and DARWIN-EU In our real world evidence (RWE) 201 series [...]
Real World Evidence (RWE) – Event Horizon
stuart.mccully2023-12-08T14:54:21+00:00December 8, 2023|2022, RWE 201|
RWE 201 - RWE Event Horizon The term "event horizon" in the context of pharmaceuticals, particularly when discussing the shift towards real-world evidence (RWE) in regulatory submissions, is metaphorically [...]
Real World Evidence (RWE) 201 – Advancing Healthcare in Australia: Embracing Real-World Evidence and Data in Medicine Regulation and Development
stuart.mccully2023-12-08T14:49:27+00:00December 8, 2023|2023, RWE 201|
RWE 201 - Advancing Healthcare in Australia: Embracing Real-World Evidence and Data in Medicine Regulation and Development Australia has several real-world evidence (RWE) initiatives to support healthcare [...]
Real World Evidence (RWE) 201 – South Korea’s Pioneering Approach: Integrating Real-World Evidence in Healthcare and Drug Development
stuart.mccully2023-12-08T14:39:24+00:00December 8, 2023|2023, RWE 201|
RWE 201 - South Korea's Pioneering Approach: Integrating Real-World Evidence in Healthcare and Drug Development South Korea has several initiatives to support healthcare and drug development using [...]
Real World Evidence (RWE) 201 – Taiwan: Integrating Real-World Evidence for Drug Development and Policy Making
stuart.mccully2023-12-08T14:34:38+00:00December 8, 2023|2023, RWE 201|
RWE 201 - Taiwan: Integrating Real-World Evidence for Drug Development and Policy Making Taiwan has several real-world evidence (RWE) initiatives to support healthcare and drug development. Over the past [...]
Real World Evidence (RWE) 201 – Revolutionizing Healthcare in Japan: The Rise of Real-World Evidence in Drug Development
stuart.mccully2023-12-08T14:31:09+00:00December 8, 2023|2023, RWE 201|
RWE 201 - Revolutionizing Healthcare in Japan: The Rise of Real-World Evidence in Drug Development Japan has several initiatives to support healthcare and drug development using real-world evidence (RWE). [...]