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Real World Evidence (RWE) 101 – STaRT-RWE

RWE 101 – STaRT-RWE

START-RWE (Structured Template for Planning and Reporting on the Implementation of Real World Evidence Studies) was developed to address the need for improved transparency and reproducibility in real-world evidence (RWE) studies.
 
Link: https://lnkd.in/dGp3auQ
 
The template includes sections on study design, data collection, data analysis, and reporting. The study design section includes questions about the study’s objectives, population, intervention, comparator, outcomes, and study period. The data collection section includes questions about the data sources, data collection methods, and data quality assurance procedures. The data analysis section includes questions about the statistical methods that will be used to analyze the data. The reporting section includes questions about the study’s findings, limitations, and implications.
 
The template is intended to be used by researchers and clinicians to develop and implement RWE studies in a rigorous and transparent manner. The template can also be used by funders, regulators, and other stakeholders to assess the quality of RWE studies. The publication includes a number of case studies that illustrate how the STaRT-RWE template has been used to develop and implement RWE studies.
 
In the context of RWE transparency and reproducibility, START-RWE is important for the following reasons:
 
1. Transparency: The comprehensive reporting facilitated by START-RWE allows for complete transparency in the methods and findings of RWE studies. It ensures that all important details regarding the study design, data sources, analysis methods, results, and interpretations are fully disclosed and clearly communicated. This openness supports better interpretation and use of study findings.
2. Reproducibility: Transparency leads to reproducibility. By providing a comprehensive and detailed account of the study methods, START-RWE enables other researchers to replicate the study, which is a cornerstone of scientific validation. Reproducibility also allows for the results of RWE studies to be confirmed and refined in different populations and settings, enhancing their generalizability and impact.
 
The STaRT-RWE template is a valuable resource for researchers and clinicians who are planning or conducting RWE studies. The template can help to ensure that RWE studies are conducted in a rigorous and transparent manner, which can improve the quality of the evidence and ultimately lead to better healthcare decision-making.

 

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Real World Evidence (RWE) 101 – STaRT-RWE2023-08-07T17:12:23+00:00

Real World Evidence (RWE) 101 – Protocol Design and ISPE GPP

RWE 101 – Protocol Design and ISPE GPP

The International Society for Pharmacoepidemiology (ISPE) Guidelines for Good Pharmacoepidemiology Practices (GPP) are a set of best practices for the conduct of pharmacoepidemiologic research, which includes the creation and analysis of real-world evidence (RWE).
 
Link: https://lnkd.in/eHSZVunX
 
ISPE GPP provides a benchmark for quality in pharmacoepidemiology studies, covering all aspects of the research process from study design and conduct to communication of study results. They provide guidance on maintaining ethical standards, ensuring data quality and integrity, managing study data and documents, managing and reporting adverse events, and many other facets of study conduct. Regulatory bodies in the USA, Canada, EU and Germany recommend that ISPE should be considered by marketing authorisation holders and investigators for the development of study protocols, the conduct of studies and the writing of study reports.
 
In the context of RWE and RWE study protocols, ISPE GPP is particularly important for the following reasons:
 
1. Enhanced Credibility and Quality of Studies: Adhering to ISPE GPP can help to ensure that RWE studies are designed and conducted to a high standard, which enhances their credibility and the reliability of their results.
 
2. Consistency and Comparability: ISPE GPP provide a consistent framework for RWE studies, which can facilitate comparison between studies and encourage uniformity in research practices. For example:
– Section II of ISPE GPP provides detailed guidance on protocol design and context
– Section IV covers study conduct and study reporting
 
3. Ethical Conduct: The guidelines provide recommendations for maintaining ethical standards throughout the research process, including the protection of study participants and the use of their data.
 
4. Regulatory Compliance: RWE is being increasingly used by regulatory agencies to inform decisions about the safety and effectiveness of treatments. Adhering to ISPE GPP can help to ensure that studies meet the standards expected by these agencies.
 
5. Transparency: The guidelines promote transparency in the reporting of study methods and results, which is crucial for the interpretation and validation of RWE.
 
6. Archiving: In the absence of national requirements or guidance, ISPE GPP recommends that study-related materials be maintained for at least five years after final report or first publication of study results.
 
In essence, following ISPE GPP can ensure that RWE studies are conducted to a high scientific and ethical standard, increasing their value to healthcare providers, regulators, and patients.

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Real World Evidence (RWE) 101 – Protocol Design and ISPE GPP2023-08-07T17:03:05+00:00

Real World Evidence (RWE) 101 – Protocol Design and Scientific Best Practices

RWE 101 – Protocol Design and Scientific Best Practices

 

Designing a robust Real-World Evidence (RWE) study is crucial for generating reliable and valid insights that are acceptable to regulators. Here are some scientific best practices to consider according to regional drug regulatory authorities:
 
EUROPEAN UNION – According to the European Medicines Agency (EMA), relevant scientific guidance should be considered by marketing authorisation holders and investigators for the development of study protocols, the conduct of studies and the writing of study reports…These scientific guidelines include the:
1. ENCePP Guide on Methodological Standards in Pharmacoepidemiology
2. ENCePP Checklist for Study Protocols, and
3. Guidelines for Good Pharmacoepidemiology Practices of the International Society of Pharmacoepidemiology (ISPE GPP)
Ref: Section VIII.B.1. of GVP Module VIII
 
GERMANY – The observation plan is to be drawn up according to recognized recommendations of scientific or regulatory guidelines…for example:
1.  Guidelines for Good Pharmacoepidemiology Practices of the International Society for Pharmacoepidemiology (ISPE GPP),
2. Recommendations of the ” ENCePP Guide on Methodological Standards in Pharmacoepidemiology”, and
3. Guidelines for Good Epidemiological Practice (GEP) of the German Society for
Epidemiology (DGEpi)
Ref:  Section 2.7.1 of the BfArM/PEI Recommendations – December 2022
 
CANADA – All research protocols should be well-designed and include a comprehensive assessment of good research principles. There are fifteen key elements that should be considered for each protocol, and are reflective of the:
1. European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) Protocol Checklist, and
2. The Guidelines for Good Pharmacoepidemiology Practices (GPP).
Both prospective and retrospective designs should attempt to address each element, or provide justification why it may not be applicable to the specific study.
Ref: Health Canada – Elements of Real World Data/Evidence Quality throughout the Prescription Drug Product Life Cycle, March 2019
 
USA – According to the FDA’s “Best Practices for Conducting and Reporting Pharmacoepidemiologic Safety Studies Using Electronic Healthcare Data” of May 2013, the following provide general guidance applicable to all pharmacoepidemiologic safety studies:
1. ISPE guidelines (ISPE GPP),
2. STROBE reporting framework, and
3. The ENCePP methods checklist
Ref: FDA Guidance – Best Practices for Conducting and Reporting Pharmacoepidemiologic Safety Studies Using Electronic Healthcare Data
 
These scientific best practices will help ensure that the RWE study is robust, credible, and provides meaningful information to patients, healthcare providers, and policy makers.

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Real World Evidence (RWE) 101 – Protocol Design and Scientific Best Practices2023-08-07T16:51:55+00:00

Real World Evidence (RWE) 101 – Study Designs

RWE 101 – Study Designs

Real-world evidence (RWE) studies are becoming increasingly important in healthcare decision-making. There are various study designs used to generate RWE, each with their unique benefits:
 
[1] Cohort Studies: Cohort studies are observational in nature, where two or more groups (cohorts) distinguished by their exposure to a certain factor (like a medical treatment or lifestyle choice) are followed and assessed to determine the effect of that exposure. These studies are advantageous in studying rare outcomes, multiple outcomes, or outcomes that take a long time to develop.
 
[2] Case-Control Studies: These studies start with the outcome and then look backward to find the exposure. They’re efficient for studying rare diseases or diseases with a long latency period, as well as multiple exposures. However, they may not be as reliable as cohort studies due to potential recall bias.
 
[3] Cross-Sectional Studies (Prevalence Studies): These studies observe a defined population at a single point in time or time interval. They’re useful in understanding the burden of a disease in a population, assessing public health needs, and planning healthcare services.
 
[4] Registry-Based Studies: These are observational studies where data are collected prospectively or retrospectively for patients with a particular condition or who are undergoing a specific procedure. Registry-based studies provide long-term, real-life data about prognosis, adverse events, quality of life, and cost-effectiveness.
 
[5] Pragmatic Clinical Trials (PCTs): Unlike traditional randomized clinical trials, PCTs are designed to determine the effectiveness of interventions in real-world routine practice conditions. They often include a more diverse patient population, multiple care settings, and less rigid protocols.
 
[6] Retrospective Studies: This involves analyzing existing datasets (like EHRs or insurance claims databases) to find associations or trends. This is also known as secondary use of existing data. While these are generally quicker and less expensive, they are subject to the limitations of the existing data, which may not have been collected for research purposes.
 
[7] Prospective Observational Studies: In these studies, subjects are followed over time with data collected about various factors that might influence the outcome of interest. These studies are useful in understanding the natural history of disease and the effectiveness of different treatments in the real world. These studies usually include both secondary data (data collected for a different purpose) and primary data (data collected specifically for the purposes of the study).

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Real World Evidence (RWE) 101 – Study Designs2023-08-07T16:44:52+00:00

Real World Evidence (RWE) 101 – Protocol Considerations

RWE 101 – Protocol Considerations

Real-world evidence (RWE) study protocols and clinical trial protocols both outline the design and conduct of a study. However, they are distinctly different in several ways given the differences in objectives, methodologies, settings, and populations involved in clinical trials versus RWE studies.
 
[1] Objectives: The main objective of a clinical trial is to evaluate the efficacy and safety of a medical intervention in a controlled environment, usually by comparing it to a placebo or standard treatment. On the other hand, RWE studies typically aim to understand how an intervention works in routine clinical practice, often focusing on outcomes such as long-term effectiveness, side-effects, quality of life, and cost-effectiveness.
 
[2] Study Design and Methodology: Clinical trials, especially phase III, are predominantly randomized controlled trials (RCTs) where subjects are randomly assigned to the intervention or control group to minimize bias. They follow a pre-specified protocol and are conducted under tightly controlled conditions. RWE studies, on the other hand, are typically observational in nature and analyze data from sources like electronic health records (EHRs), claims databases, or patient registries.
 
[3] Setting: Clinical trials are conducted in specific, controlled environments and follow a strict protocol. RWE studies are conducted in routine clinical practice settings, making them more representative of ‘real-world’ conditions.
 
[4] Population: Clinical trials often have strict inclusion and exclusion criteria, resulting in a relatively homogeneous group of participants. This can limit the generalizability of the results. RWE studies, in contrast, involve broader, more diverse populations (including those often excluded from trials like the elderly, people with multiple co-morbidities, etc.), making the findings more generalizable to everyday practice.
 
[5] Data Collection: In clinical trials, data collection is rigorous, detailed, and specific to the trial endpoints. Adverse events are actively sought and documented. RWE studies primarily rely on existing data sources such as EHRs, patient registries, or insurance claims data. This can potentially lead to incomplete or inaccurate data.
 
[6] Intervention: In clinical trials, the intervention (dosage, frequency, duration, etc.) is pre-specified and strictly monitored. In RWE studies, interventions reflect routine clinical practice and may vary widely.
 
[7] Follow-up: Clinical trials have a defined follow-up period while RWE studies can often provide information on long-term outcomes, given they use data from routine clinical practice over longer periods.
 
While clinical trials provide the highest level of evidence for determining a treatment’s efficacy, RWE studies complement this by providing evidence on real-world effectiveness and long-term safety.

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Real World Evidence (RWE) 101 – Protocol Considerations2023-08-07T16:38:31+00:00

Real World Evidence (RWE) 101 – PASS Approval Requirements

RWE 101 – PASS Approval Requirements

 

Post-authorisation safety studies (PASS) are studies conducted after a medicine has been authorized (licensed) with the goal of acquiring more data about the medicine’s safety, or to measure the effectiveness of risk-management measures. These studies play a vital role in understanding the safety and effectiveness of a drug in a real-world context.
 
While specific requirements can vary by country, they often include:
 
[1] Ethics Committee Approval: Any study involving human subjects generally needs to be approved by an Ethics Committee (EC) or Institutional Review Board (IRB). This body ensures that the study is designed and implemented in a manner that respects the rights, safety, and well-being of the participants (i.e., compliance with the Declaration of Helsinki).
 
[2] Regulatory Approval: Depending on the country and type of PASS (e.g., Category 1, 2, 3, or voluntary PASS) additional regulatory approval may be needed. For instance, in the European Union, any category 1 or 2 PASS protocol of a medicine authorised through the Centralised Procedure has to be approved by the European Medicines Agency (EMA).
 
[3] Data Protection Laws Compliance: Studies must comply with all relevant data protection laws (e.g., GDPR). This includes regulations on the use and sharing of personal data, as well as provisions for protecting the privacy of participants.
 
[4] Study Registration: Most categories of PASS must be registered in a public database (i.e., EU PAS Register). This increases transparency and allows for the tracking and sharing of results.
 
[5] Adherence to Good Pharmacovigilance Practices (GVP): This is especially pertinent in the EU, where PASS must adhere to Module VIII of the GVP.
 
[6] Provision of Adequate Information to Participants: There’s usually a requirement that all participants must be given adequate information about the study, and that informed consent must be obtained.
 
[7] Reporting Requirements: There will be requirements to report adverse events, progress, and the final results to the respective regulatory bodies (i.e., ethics committees and national competent authorities).
 
However, these requirements can be subject to change over time and may vary in specifics from one country to another. Therefore, it’s crucial to consult with the appropriate regulatory body or a regulatory affairs professional familiar with these regulations when planning a post-authorisation safety study.

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Real World Evidence (RWE) 101 – PASS Approval Requirements2023-08-07T16:30:41+00:00

Real World Evidence (RWE) 101 – Categories of PASS

RWE 101 – Categories of PASS

Real-world evidence (RWE) is the clinical evidence about the usage and potential benefits or risks of a product derived from the analysis of real-world data (RWD). RWD and RWE are playing an increasingly important role in healthcare decisions.
 
In the context of pharmacovigilance, post-authorization safety studies (PASS) are studies performed after a medicine has been approved and is available on the market. The purpose of these studies is to obtain further information on a medicine’s safety, or to measure the effectiveness of risk-management measures.
 
Post-authorization safety studies (PASS) can be interventional (clinical trials) or non-interventional. As per EMA’s GVP Module V, there are 3 categories of PASS:
 
Category 1 PASS (Imposed PASS) – These are studies required by a regulatory authority as a condition of a (normal) marketing authorization. These studies are often required to clarify the safety profile of the drug, or to assess risks that were identified in pre-approval stages, but could not be fully characterized due to limitations like sample size of clinical trials or underrepresentation of certain populations. These studies are listed in Section II.C of the risk management plan (RMP) Summary.
 
Category 2 PASS (Specific Obligations) – These are studies that are specific obligations imposed when a medicine has received a conditional marketing authorisation. This applies to medicines that target conditions with an unmet medical need – for which no treatment option exists, or where they can offer a major therapeutic advantage over existing treatments. These studies are often required because the medicines approval has been accelerated to ensure timely access by patients, limiting the amount of safety data available at the time of approval. These studies are listed as “Specific Obligations” in Section II.C of the EPAR RMP Summary
 
Category 3 PASS (Other PASS) – These are studies might be required (as opposed to being imposed) by the EMA or a national competent authority in the RMP to investigate a safety concern or to evaluate the effectiveness of risk minimisation activities.  These studies are listed as “Other Studies” in Section II.C of the RMP Summary.
 
Category 1 and 2 PASS are supervised under Articles 107m, 107n-q of Directive 2001/83/EC, meaning the protocol and study report must comply with Annex III of Regulation 520/2012/EU and EMA’s GVP Module VIII. The protocol must be approved (endorsed) by the EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) (medicines approved through the Centralised Procedure).
 
Category 3 PASS are supervised under Article 107m of Directive 2001/83/EC, which requires, for example, that studies are not promotional, healthcare professional renumeration is equivalent to fair market value, and final study reports should be send to the national competent authorities where the study is conducted.

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Real World Evidence (RWE) 101 – Categories of PASS2023-08-07T16:14:33+00:00

Real World Evidence (RWE) 101 – Safety Monitoring

RWE 101 – Safety Monitoring

Real World Evidence (RWE) complements clinical trials and provides additional insights that are difficult to achieve in controlled environments. Here’s why:

[1] Sample Size and Diversity: Clinical trials often involve a relatively small and selected population, while RWE studies involve larger and more diverse populations. This allows for a better understanding of the safety profile of a drug among different demographics, including age, race, gender, and individuals with different comorbidities.

[2] Long-term Follow-up: Clinical trials usually have a limited duration, while RWE can provide long-term safety data, including rare side effects that may only become apparent over time.

[3] Real-World Setting: Clinical trials are conducted in controlled settings and follow strict protocols. The patients who participate are often healthier and more adherent to treatment than average. On the other hand, RWE reflects the real-world setting, capturing the effects of the drug when used in routine clinical practice, which can differ substantially from trial conditions.

[4] Poly Pharmacology (Concomitant Medications): In the real world, patients often receive combinations of treatments, and the effectiveness and safety of these combinations can be different than individual treatments. RWE provides information about these combinations, something that is difficult to study in clinical trials.

[5] Post-Marketing Surveillance: Once a drug is approved and in use, RWE provides a mechanism to monitor its safety in the larger population. Post-marketing surveillance can help to identify rare adverse events that were not detected in clinical trials due to smaller sample size.

However, it’s important to note that RWE and clinical trials each have their strengths and weaknesses. Clinical trials remain the gold standard for demonstrating efficacy and obtaining regulatory approval because they can establish causality via randomization. RWE, while providing valuable insights on effectiveness and safety, often comes from observational studies, where it can be harder to determine cause-and-effect relationships because of potential confounding factors. Therefore, both are needed and (traditionally) used at different stages of the drug development and monitoring process.

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Real World Evidence (RWE) 101 – Safety Monitoring2023-08-07T16:06:48+00:00

Real World Evidence (RWE) 101 – How RWE is Being Used to Support the Treatment of Cancer

RWE 101 – How RWE is Being Used to Support the Treatment of Cancer

Real-world evidence (RWE) enhances cancer treatment through providing more comprehensive and personalized patient data. Here are summarized examples:

[1] Targeted Therapies for Lung Cancer: RWE aids in identifying actionable mutations in non-small-cell lung carcinoma (NSCLC), facilitating targeted treatments.
[2] Immuno-Oncology Treatments: Through studying melanoma patient responses to immune checkpoint inhibitors, RWE informs long-term safety, efficacy (effectiveness), and optimal usage.
[3] Treatment Adherence and Persistence: RWE helps determine factors affecting adherence to oral chemotherapies, enabling effective patient management.
[4] Clinical Trial Design: Real-world data can ensure clinical trial populations better reflect the real-world population’s diversity, increasing the trials’ relevance.
[5] Post-Market Surveillance: RWE allows monitoring of approved cancer drugs’ safety and effectiveness, identifying previously unknown adverse effects and comparing different treatments.
[6] Comparative Effectiveness Research: RWE can evaluate the benefits and harms of different treatments, such as drug classes or treatment methods, enhancing decision-making.
[7] Pediatric Oncology: RWE offers valuable insights into pediatric cancer treatment patterns and outcomes, critical given the ethical concerns with trials in pediatric populations.
[8] Health Economics and Outcomes Research: RWE assesses the cost-effectiveness of cancer treatments, informing coverage and reimbursement decisions.
[9] Precision Medicine: Real-world genomic data, combined with clinical outcomes, helps create more personalized treatment strategies, like using PARP inhibitors for BRCA-mutated ovarian cancer.
[10] Population Health Management: RWE can expose disparities in cancer care, prompting efforts to address these gaps.

In summary, RWE significantly contributes to cancer treatment. It complements clinical trials, supports personalized care, highlights patient voice, and improves overall cancer care quality. Despite data standardization and quality challenges, RWE’s potential to enhance cancer treatment remains vast.

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Real World Evidence (RWE) 101 – How RWE is Being Used to Support the Treatment of Cancer2023-08-07T15:57:12+00:00

Real World Evidence (RWE) 101 – Parkinson’s Disease

RWE 101 – Parkinson’s Disease

Real-world evidence (RWE) refers to clinical evidence regarding the usage and potential benefits or risks of a treatment derived from analysis of real-world data (RWD). RWD can include data from electronic health records (EHRs), claims and billing activities, product and disease registries, patient-generated data, and data gathered from other sources that reflect routine clinical practice.

In the context of Parkinson’s disease (PD), RWE can provide valuable insights into disease prevalence, real-world effectiveness of treatments, adherence to medication, quality of life, and more.

PD affects nearly 1% of individuals aged 60 and over. Real-world data, like those derived from large epidemiological studies or health databases, can help to reveal the true prevalence and incidence of PD in different populations. This information can assist in understanding the disease burden, aid healthcare planning, and inform research directions.

Treatment options for PD, as per conventional evidence, primarily consist of levodopa, dopamine agonists, and MAO-B inhibitors, along with non-pharmacological interventions like physical and occupational therapy, and in some cases, deep brain stimulation.

However, RWE provides additional context to these treatments. For example, RWE studies can demonstrate how these treatments are used in routine clinical practice, outside the controlled environment of clinical trials. They can highlight issues such as medication adherence, side-effects in real-world populations, long-term effectiveness, and the use of combination therapies.

RWE can also be used to understand disparities in treatment access and outcomes in different population subgroups. For instance, it might show that certain racial or socioeconomic groups have poorer access to PD treatments or worse outcomes, indicating the need for targeted interventions.

Moreover, RWE is crucial in identifying unmet needs and directing research towards novel treatments. For example, real-world data might reveal a significant number of PD patients not responding adequately to existing treatments, leading to the exploration of new therapeutic approaches like stem cell or gene therapy.

However, RWE has limitations, such as potential biases related to data collection and confounding factors, which should be taken into account when interpreting results.

In conclusion, RWE has a valuable role in understanding the real-world implications of PD, enhancing our knowledge about the disease, its treatment, and ultimately, improving patient care.

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Real World Evidence (RWE) 101 – Parkinson’s Disease2023-08-07T14:39:03+00:00
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