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USA | FDA Finalises RWD Guidance on Assessing Registries To Support Regulatory Decision-Making for Drug and Biological Products

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USA | FDA Finalises RWD Guidance on Assessing Registries To Support Regulatory Decision-Making for Drug and Biological Products2024-01-09T14:36:31+00:00

USA | FDA Finalises Guidance on “Data Standards for Drug and Biological Product Submissions Containing Real-World Data

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USA | FDA Finalises Guidance on “Data Standards for Drug and Biological Product Submissions Containing Real-World Data2024-01-09T14:24:58+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

Norway – Real World Data Initiatives

RWE 201 – Norway – Real World Data Initiatives

Norway has made significant strides in real-world data (RWD) and real-world evidence (RWE) initiatives, particularly within the healthcare sector. This is consistent with the global trend of employing RWD and RWE to complement traditional clinical trial data in order to understand the use and potential benefits or risks of a product under actual conditions of use.

[1] The Norwegian Prescription Database (NorPD): This database contains information about all prescription drugs dispensed at pharmacies to individual patients in outpatient care in Norway. It does not include drugs dispensed in hospitals or non-prescription drugs. The database is particularly useful for studies on drug utilization, adherence, and safety.

>> Link: https://www.fhi.no/en/he/norpd/norwegian-prescription-database/

[2] The Cancer Registry of Norway: The Cancer Registry is an essential component for cancer research in Norway. It provides a comprehensive picture of cancer incidence in the Norwegian population and is a source of real-world data on cancer.

>> Link: The Cancer Registry of Norway

[3] The Norwegian Patient Registry (NPR): This registry contains data about all patients who receive treatment in the specialist health service in Norway. The registry can be used to create statistics and for research purposes.

>> Link: https://www.helsedirektoratet.no/tema/statistikk-registre-og-rapporter/helsedata-og-helseregistre/norsk-pasientregister-npr

[4] Biobanks: Norway has several biobanks, which store biological samples used for medical research. These biobanks, coupled with health registries, are valuable resources for RWE initiatives.

>> Link: https://www.fhi.no/en/hd/biobanks/

[5] The Cohort of Norway (CONOR): CONOR is a collection of health data and blood samples from 200,000 Norwegians. This collaborative health survey can be used for a wide range of epidemiological studies based on RWD.

>> Link: https://www.fhi.no/en/hs/conor/

[6] Electronic Health Records (EHR): Norway has adopted EHR systems, which can be utilized for RWD extraction, albeit with the appropriate permissions and adhering to privacy regulations.

With databases like the NorPD tracking outpatient prescriptions, the Cancer Registry focusing on cancer incidences, and the Norwegian Patient Registry capturing specialist health services, Norway boasts a broad spectrum of health data. The Cohort of Norway (CONOR) further provides extensive epidemiological data. These initiatives, combined with biobanks and electronic health records, ensure a rich, multidimensional data environment. Such extensive and diverse data repositories enable deep insights into drug utilization, disease patterns, and patient outcomes, making Norway a forerunner in leveraging real-world data for healthcare research and improvements.

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Norway – Real World Data Initiatives2023-10-14T09:09:39+00:00

Sweden – Pioneering the Use of RWD

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 the healthcare sector. RWD and RWE refer to data and evidence obtained outside of traditional randomized controlled trials, usually from electronic health records, registries, and other observational sources. Some notable initiatives and resources in Sweden include:

[1] Swedish National Patient Register (NPR): Managed by the Swedish National Board of Health and Welfare, the NPR contains detailed patient-level information on all hospital admissions and outpatient visits in Sweden.

[2] Swedish Prescribed Drug Register: This is a national database that contains information on all prescriptions dispensed to patients in Sweden.

[3] Swedish Cancer Registry: Initiated in 1958, this registry has near-complete coverage of all cancer diagnoses in Sweden.

[4] Quality Registries: Sweden has more than 100 national quality registries that contain individual-level data on patient problems, medical interventions, and outcomes after treatment.

[5] Biobanks: Sweden has a long tradition of collecting tissue samples, which are stored in biobanks. These samples can be used for a variety of RWE studies, including genetic research.

[6] SWEDEHEART: This is a national registry for cardiac care. It’s an example of one of Sweden’s more specific health registries that provides valuable RWD for various cardiac conditions and their treatments.

[7] Swedish Rheumatology Quality Register (SRQ): This registry provides RWD on rheumatic diseases and their treatments.

[8] Stockholm Science City: This initiative aims to make Stockholm and Sweden a platform for health outcomes and real-world evidence in Europe. It leverages the country’s legacy health data, academic excellence, and digital start-ups to create a strong innovation ecosystem.

Sweden’s robust system of registries and biobanks, combined with its universal healthcare system, provides a unique opportunity for generating RWE. This has been critical for drug and device monitoring post-market, epidemiological studies, health economic evaluations, and many other types of research.

The Swedish government and various institutions in the country have recognized the potential of using RWD and RWE for improving patient outcomes, shaping health policies, and guiding clinical decisions. Consequently, they have invested in infrastructure, governance, and collaboration between various stakeholders, including healthcare providers, academia, and the pharmaceutical industry, to ensure the effective use of RWD and RWE.

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Sweden – Pioneering the Use of RWD2023-10-14T09:03:34+00:00

UK – MHRA – Randomised Controlled Trials Using Real World Data

RWE 201 – UK – MHRA – Randomised Controlled Trials Using Real World Data

 

MHRA RWD Guidance: https://www.gov.uk/government/publications/mhra-guidance-on-the-use-of-real-world-data-in-clinical-studies-to-support-regulatory-decisions/mhra-guideline-on-randomised-controlled-trials-using-real-world-data-to-support-regulatory-decisions

The MHRA guideline focuses on sponsors planning to conduct randomised, controlled clinical trials (RCTs) primarily using Real-World Data (RWD) for regulatory decisions concerning medicinal products.

Scope

The guideline covers:

  1. Clinical trial authorisation in the UK.
  2. Trial design, including endpoint selection and safety requirements.

It excludes discussions on observational studies, and clinical trials using RWD as a control arm.

Definition and Types of RWD Trials

RWD is health data obtained outside clinical studies and can include electronic healthcare records, disease registries, and patient-reported outcomes. In a simple RWD-based RCT, patients are randomized to receive standard clinical care alone or an added intervention. Data quality and study design need to be as rigorous as traditional RCTs for the results to be valid for regulatory decisions.

Protocol Requirements

The protocol should pre-specify objectives, data collection methods, and primary and secondary endpoints. Consent is required before enrolment, and in most real-world settings, patients are not blinded to treatment allocation.

Regulatory Acceptability

From a regulatory standpoint, the source of the data (RWD, hybrid, or traditional) is irrelevant as long as the trial answers the necessary regulatory questions. The need for blinding should be considered, especially if the primary endpoints are not sufficiently objective.

Examples and Practical Considerations

The guideline suggests that RWD-based trials can be effective when dealing with an established EHR database and objective endpoints like all-cause mortality. Scenarios where the intervention is an existing licensed product with a well-known safety profile are particularly suited for RWD trials. It’s critical not to assume the completeness of potential endpoints in EHRs. Sponsors are advised to conduct a feasibility study to assess the reliability of the data capture methods.

Data Gaps and Hybrid Trials

If the RWD source does not cover all required endpoints, a hybrid trial can supplement RWD with specific additional data. These could be additional clinical assessments, which might even be carried out remotely.

In summary, the MHRA guideline serves as a comprehensive framework for sponsors interested in leveraging RWD for RCTs aimed at supporting regulatory decisions for medicinal products. While RWD-based trials offer advantages in reducing patient and healthcare burdens, they must be designed and executed with rigor comparable to traditional RCTs to be deemed acceptable for regulatory purposes.

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UK – MHRA – Randomised Controlled Trials Using Real World Data2023-10-14T08:31:42+00:00

Real World Evidence (RWE) 201 – France – Haute Autorité de Santé (HAS) – Real World Study Guide

RWE 201 – France – Haute Autorité de Santé (HAS) – Real World Study Guide

 

HAS Real World Stidy Guide: https://www.has-sante.fr/upload/docs/application/pdf/2021-06/real-world_studies_for_the_assessment_of_medicinal_products_and_medical_devices.pdf

The Haute Autorité de Santé (HAS), France’s National Authority for Health, is responsible for scientifically evaluating medicinal products and medical devices in terms of their medical, economic, and public health value. While clinical trials have traditionally been the foundation for these assessments, HAS is increasingly incorporating “real-world data” (RWE) into their evaluations.

Real-world data comes from sources other than conventional clinical trials and includes observational data and data on the use, efficacy, and safety of health products. This kind of data is particularly useful because clinical trials often have limitations; their controlled conditions can differ significantly from real-life medical practice, and their results might not be applicable in all cases. For example, new therapies involving gene technologies or machine learning bring uncertainties that can only be fully understood in real-world conditions.

Real-world studies offer advantages like understanding a product’s performance in actual clinical settings, identifying the patients most likely to benefit, and foreseeing potential risks. They also allow for the inclusion of patient perspectives, which are becoming increasingly important in the evaluation process.

HAS has begun updating its methodological guide on real-world studies to provide practical guidelines for such evaluations. This is due to increased access to health data and the growing recognition of the importance of real-world data to complement clinical trials. These guidelines aim to aid all stakeholders involved in the health product evaluation process, including manufacturers, research companies, and academic teams, in the design and implementation of real-world studies.

In summary, HAS is expanding its reliance on real-world data to enhance the quality and applicability of its health product assessments. This shift is aimed at capturing a more comprehensive picture of product effectiveness and safety, which ultimately benefits patient care.

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Real World Evidence (RWE) 201 – France – Haute Autorité de Santé (HAS) – Real World Study Guide2023-09-04T08:30:12+00:00
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