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Real World Evidence (RWE) 101 – Benefits of RWE in the Context of Rare Diseases

RWE 101 – Benefits of RWE in the Context of Rare Diseases

 

Real-world evidence (RWE) refers to information on health care that comes from real-world settings, such as electronic health records (EHRs), insurance claims, product and disease registries, patient-generated data, and emerging sources like wearables and social media.

RWE is becoming increasingly important in the management of rare diseases like Addison’s disease, which is characterized by insufficient hormone production from the adrenal glands. Here are some benefits RWE can provide:

[1] Improved Understanding of Natural Disease Progression: Due to the low prevalence of rare diseases, our understanding of them often lacks detail. Real-world data can help fill these knowledge gaps by offering insights into the disease’s natural history and progression.

[2] Efficacy and Safety of Treatments: Clinical trials are the gold standard for assessing the efficacy and safety of new treatments, but they may not represent the broader patient population due to their strict inclusion and exclusion criteria. RWE, being derived from a diverse population, can provide insight into how a treatment works in a broader range of patients.

[3] Patient-focused Drug Development: With RWE, researchers can understand the burden of disease and the benefits patients value most, which can guide patient-focused drug development.

[4] Health Economics and Outcomes Research (HEOR): RWE can provide critical data for health economic evaluations, cost-effectiveness studies, and budget impact analyses.

[5] Early Identification and Diagnosis: Since many rare diseases often go undiagnosed due to their rarity and a lack of awareness among healthcare providers, RWE can help in early identification and diagnosis by revealing patterns or correlations that might not be apparent in smaller datasets.

[6] Post-marketing Surveillance: After a drug has been approved, RWE can be used to monitor its long-term effectiveness and side effects in a larger population.

[7] Guiding Clinical Practice: By understanding how treatments work in the real world, doctors can be better informed about the likely benefits and risks for individual patients, thus guiding personalized treatment.

However, it’s important to note that while RWE provides numerous benefits, it also comes with challenges, including data quality and integrity, privacy and security concerns, and the need for sophisticated analytical methods to derive meaningful conclusions from complex and heterogenous datasets.

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Real World Evidence (RWE) 101 – Benefits of RWE in the Context of Rare Diseases2023-08-07T14:32:56+00:00

Real World Evidence (RWE) 101 – Disease Prevalence vs Incidence

RWE 101 – Disease Prevalence vs Incidence

Real-world evidence (RWE), derived from real-world data (RWD), allows us to gain insights into disease prevalence and incidence outside of controlled clinical trial settings. This information plays a crucial role in shaping healthcare policies, identifying healthcare needs, directing research efforts, and improving patient care.

Disease prevalence refers to the total number of individuals in a population who have a specific disease at a given time. This includes both new (incidence) and pre-existing cases. RWE can offer a more comprehensive picture of disease prevalence by incorporating data from various sources such as electronic health records (EHRs), insurance claims data, patient registries, and more. This data is especially valuable in understanding chronic diseases, such as diabetes or heart disease, where prevalence data can reveal the burden of disease in a population, help to identify risk factors, and guide resource allocation.

Disease incidence, on the other hand, refers to the number of new cases of a disease that develop in a specific time period. Incidence data is particularly valuable in understanding infectious diseases or diseases linked to specific exposure or risk factors, like cancer or HIV/AIDS. Incidence data derived from RWD can help track disease outbreaks, identify populations at risk, and assess the effectiveness of preventative measures.

For example, using EHRs and public health databases, RWE can offer insights into the incidence of a new disease like COVID-19, track its spread, and monitor the effectiveness of public health measures in real-time.
RWE also plays a critical role in post-market surveillance of drugs and medical devices. By monitoring incidence rates of adverse events in the general population, it is possible to detect safety issues that may not have appeared during clinical trials, due to their limited size or exclusion of certain patient groups.

However, there are potential limitations to RWE including data quality, missing data, and bias in data collection. These factors should be considered while interpreting RWE for disease prevalence and incidence.

In conclusion, RWE provides a comprehensive and real-time view of disease prevalence and incidence in real-world settings, thereby informing health policy decisions, guiding research, and improving overall patient care.

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Real World Evidence (RWE) 101 – Disease Prevalence vs Incidence2023-08-07T14:27:06+00:00

Real World Evidence (RWE) 101 – Principles for Ensuring that Research Results are Reliable, Valid, and Objective

RWE 101 – Principles for Ensuring that Research Results are Reliable, Valid, and Objective

Ensuring that research results are reliable, valid, and objective requires careful planning, execution, and evaluation. Here are several key factors to consider:

1. Research Design: The study design should be robust and suitable for answering the research questions. It should incorporate procedures for controlling variables, randomizing assignments (if applicable), and testing hypotheses. The design should also minimize the potential for bias.

2. Sampling: The selection of participants or samples should be appropriate and unbiased. Larger sample sizes generally increase reliability and can improve validity. The sample should be representative of the population to which you want to generalize your findings.

3. Data Collection: The methods for collecting data should be consistent and standardized. Any measurement tools used should be reliable (provide consistent results over time) and valid (accurately measure what they’re supposed to measure).

4. Data Analysis: Statistical analysis should be appropriate for the type of data collected and the research questions. This includes correctly handling missing data and making proper inferences. Be cautious of multiple testing issues that can lead to false positives.

5. Replication: A study’s results are more reliable if they can be replicated by other researchers. To facilitate replication, provide a clear and thorough description of your methods (e.g., use the STaRT-RWE structured template for planning and reporting of real world evidence studies).

6. Peer Review: Have your research reviewed by others in your field before publishing. They can provide valuable feedback and catch any errors or inconsistencies you may have missed.

7. Transparency and Openness: Be open and honest about your methodology, data, results, and any potential conflicts of interest. This can include sharing your raw data and analysis scripts if possible.

8. Addressing Confounding Factors: Identify and control for potential confounding factors – variables that could cause both the independent and dependent variables to change, thereby creating a false impression of a cause-effect relationship.

9. Interpretation: Be careful not to overstate your findings. Make sure your conclusions are supported by your data, and acknowledge any limitations of your study.

10. Ethical Considerations: Ensure that your study complies with ethical guidelines. This includes respecting participants’ rights and privacy, obtaining informed consent, and avoiding harm.

By adhering to these principles, researchers can increase the chances that their findings will be reliable, valid, and objective. However, it’s also important to recognize that no study is perfect, and all research comes with some degree of uncertainty. The goal is to minimize this uncertainty as much as possible.

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Real World Evidence (RWE) 101 – Principles for Ensuring that Research Results are Reliable, Valid, and Objective2023-08-07T14:20:58+00:00

Real World Evidence (RWE) 101 – Why Should Non-Interventional Studies NOT be Promotional?

RWE 101 – Why Should Non-Interventional Studies NOT be Promotional?

Non-interventional studies (NIS) are designed to observe and analyze data from real-world clinical settings without intervening or manipulating any variables. These studies play a crucial role in generating real-world evidence (RWE) and are important for understanding the effectiveness, safety, and outcomes associated with various healthcare interventions. Here are a few reasons why NIS should not be promotional:

1. Scientific integrity: The primary purpose of NIS is to gather unbiased and objective data to answer research questions or explore hypotheses. If these studies are conducted with a promotional intent, it can compromise the scientific integrity of the research. The results may be influenced or biased, leading to misleading interpretations and potentially affecting patient care.

2. Patient safety: NIS involve collecting data from patients in routine clinical practice. If these studies are conducted with a promotional motive, there is a risk of prioritizing the promotion of a product over ensuring patient safety. Patient well-being should always be the foremost concern in research, and any attempt to manipulate or skew the data for promotional purposes undermines this ethical principle.

3. Transparency and trust: NIS should be conducted with transparency and should provide reliable and unbiased evidence. When these studies are used as promotional tools, it can erode trust in the research process and industry. Maintaining public trust is crucial for the advancement of healthcare and the ethical conduct of research.

4. Conflicts of interest: If non-interventional studies are conducted for promotional purposes, it raises concerns about potential conflicts of interest. Researchers or organizations conducting the study may have financial or other vested interests in promoting a particular product. Such conflicts can compromise the objectivity and independence of the research and may lead to biased reporting and selective publication of results.

5. Regulatory considerations: Regulatory authorities often require non-interventional studies to provide robust and unbiased data to inform decision-making about the safety and effectiveness of medical products. If these studies are promotional in nature, it can undermine the credibility of the regulatory process and lead to inappropriate approvals or recommendations based on biased evidence.

To ensure the ethical conduct of NIS, it is essential to separate research activities from promotional activities. Clear guidelines and regulations are in place to prevent the misuse of research studies for promotional purposes and to maintain the integrity and scientific rigor of non-interventional research. The primary focus should be on generating reliable evidence, improving patient care, and advancing scientific knowledge in an unbiased and ethical manner.

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Real World Evidence (RWE) 101 – Why Should Non-Interventional Studies NOT be Promotional?2023-08-07T14:15:07+00:00

Real World Evidence (RWE) 101 – Seeding Studies

RWE 101 – Seeding Studies

Seeding studies, in the context of real-world evidence (RWE), refer to studies that were conducted by pharmaceutical or medical device companies after a product’s approval or market introduction. These studies aimed to promote/ increase the market presence of the approved drug. Regulators like the FDA and EMA view seeding studies as unethical for several reasons.

Link: https://lnkd.in/e9vP4KxA

1.  Misleading intent: Seeding studies were primarily conducted to influence prescribing habits, promote a specific product, and create a favourable impression among healthcare providers. This promotional intent conflicts with the primary objective of research, which should be to generate unbiased and reliable evidence.

2.  Methodological flaws: Seeding studies often lacked rigorous scientific methodology. Some were observational in nature and lacked control groups or blinding, making it difficult to draw valid conclusions about product safety and efficacy. The data collected was of poor quality, limiting its utility for meaningful analysis and decision-making.

3. Transparency and bias: Seeding studies were sponsored by the manufacturers of the products being studied. This created potential conflicts of interest and raised concerns about transparency and data integrity. The financial relationships between study sponsors and participating healthcare professionals potentially biased the study results and compromised the independence and objectivity of the research.

4. Publication bias: Seeding studies were susceptible to publication bias, where positive or favourable results were more likely to be published, while negative or unfavourable findings were suppressed or unreported. This selective reporting distorted the overall evidence base and misled healthcare providers and regulators in their decision-making.

5. Ethical considerations: Seeding studies raised ethical concerns regarding patient safety and informed consent. Participants were not adequately informed about the purpose, risks, and benefits of these studies.

Regulatory bodies, including the FDA and EMA, strive to protect patient welfare and ensure the integrity of the research process. They require studies to be conducted with scientific rigor, unbiased intent, and adherence to ethical principles. In the EU, it is a legal requirement that non-interventional studies are not promotional (Article 107m(3) of Directive 2001/83/EC).

It is important for researchers, industry sponsors, and regulators to maintain transparency, adhere to ethical guidelines, and prioritize patient welfare in the pursuit of real-world evidence. By doing so, the integrity of RWE can be preserved, and reliable evidence can guide healthcare decisions and promote the well-being of patients.

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Real World Evidence (RWE) 101 – Seeding Studies2023-08-07T14:09:15+00:00

Real World Evidence (RWE) 101 – HIPAA

RWE 101 – HIPAA

HIPAA (Health Insurance Portability and Accountability Act), enacted in 1996, is a federal law in the United States that establishes regulations for the protection of individuals’ health information and safeguards their privacy and confidentiality.

In the context of RWE, HIPAA applies to the collection, use, and disclosure of protected health information (PHI) obtained from patients’ medical records, claims data, or other sources. Here’s an overview of HIPAA’s impact on RWE:

Privacy Rule: The HIPAA Privacy Rule sets standards for the protection of individuals’ PHI. It outlines the permissible uses and disclosures of PHI by covered entities, such as healthcare providers, health plans, and healthcare clearinghouses. Researchers utilizing RWE must adhere to these privacy regulations when accessing and handling PHI.

De-identification: HIPAA provides guidelines for de-identifying PHI, allowing researchers to use data without requiring patient consent.

De-identified data is stripped of direct identifiers (e.g., names, addresses) and must have a low risk of re-identification. Researchers utilizing de-identified data are exempt from certain HIPAA requirements but must still handle data responsibly and protect against re-identification risks.

Limited Data Set: HIPAA allows the use and disclosure of a limited data set without patient authorization. A limited data set contains PHI with certain direct identifiers removed, but it may still include information such as dates and geographic data. Researchers must enter into a data use agreement with the covered entity providing the limited data set, ensuring compliance with HIPAA regulations.

Research Authorization: In some cases, researchers may seek individual authorization from patients to access their PHI for RWE studies. HIPAA specifies the required elements for a valid authorization, including a clear description of the information to be disclosed, the purpose of the disclosure, and the rights of the individual regarding their PHI.

Security Rule: The HIPAA Security Rule mandates safeguards to protect the confidentiality, integrity, and availability of electronic PHI (ePHI). It requires covered entities and their business associates to implement administrative, physical, and technical safeguards to secure ePHI against unauthorized access, use, or disclosure.

Penalties and Enforcement: HIPAA violations can lead to severe penalties, including civil and criminal sanctions.

In summary, HIPAA plays a critical role in protecting individuals’ health information in the context of RWE. Researchers must understand and adhere to HIPAA regulations when handling PHI, ensuring privacy and confidentiality while conducting valuable RWE studies. Compliance with HIPAA requirements safeguards patient rights, fosters trust, and promotes the responsible use of health data for research purposes.

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Real World Evidence (RWE) 101 – HIPAA2023-08-07T14:04:10+00:00

Real World Evidence (RWE) 101 – The Common Rule

RWE 101 – The Common Rule

The Common Rule plays a significant role in the governance of observational studies. The Common Rule refers to a set of regulations and ethical principles designed to protect human subjects participating in research studies conducted or supported by federal agencies in the United States. It sets the standards for the ethical conduct of research and ensures the protection of individuals involved in research studies.

Observational studies, which are a type of study design used in RWE research, involve the collection and analysis of data from participants in their real-world settings. These studies aim to assess outcomes and associations between various factors, such as treatments, interventions, or exposure to certain conditions, without any intervention or manipulation by the researcher.

The Common Rule establishes the following key principles and requirements:

Informed Consent: The Common Rule emphasizes the importance of obtaining informed consent from individuals participating in research studies. Researchers must provide participants with clear and understandable information about the study, its purpose, risks, potential benefits, and any other relevant details. Informed consent ensures that participants have the necessary information to make an informed decision about their participation.

Institutional Review Board (IRB) Oversight: The Common Rule requires that all research involving human subjects undergo review by an IRB. The IRB evaluates the study’s ethical implications, safeguards the rights and welfare of participants, and ensures that the research adheres to the Common Rule’s principles. IRBs play a critical role in assessing the risks and benefits of observational studies and determining whether they meet ethical standards.

Privacy and Confidentiality: Observational studies often involve the collection of sensitive data from participants, such as medical records or personal information. The Common Rule mandates that researchers take appropriate measures to protect the privacy and confidentiality of individuals involved in the study. This includes implementing safeguards to prevent unauthorized access, use, or disclosure of participant data.

Balancing Risks and Benefits: The Common Rule requires researchers to conduct a thorough risk-benefit analysis of the observational study. They must assess the potential risks associated with data collection and analysis and weigh them against the potential benefits of the research. This analysis helps ensure that the benefits of the study justify any potential risks to the participants.

By adhering to the Common Rule, researchers can ensure that observational studies in RWE are conducted ethically, with proper safeguards for participant rights, welfare, and privacy. The Common Rule helps maintain the integrity of research and promotes public trust in the scientific community’s ability to generate reliable and valuable real-world evidence.

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Real World Evidence (RWE) 101 – The Common Rule2023-08-07T13:58:31+00:00

Real World Evidence (RWE) 101 – The Tuskegee Syphilis Study (the reason we have the Belmont Report and the Common Rule)

RWE 101 – The Tuskegee Syphilis Study (the reason we have the Belmont Report and the Common Rule)

The Tuskegee Syphilis Study, conducted from 1932 to 1972, stands as a notorious example of unethical human research. Its fallout had substantial implications on the regulation of observational studies:

Ethical Guidelines: The Tuskegee study expedited the development of ethical standards, including the Belmont Report (1979). The report emphasized respect for persons, beneficence (acting in a way that brings about positive outcomes and promotes the welfare of others), and justice (the principle of treating people fairly and equitably), all crucial in research ethics.

Informed Consent: The Tuskegee study underscored the importance of informed consent. It is now required for researchers to provide comprehensive information about the study and its potential risks and benefits.

Protection for Vulnerable Populations: The study highlighted the need for protections for vulnerable populations. Additional safeguards have since been implemented to prevent similar abuses.

Institutional Review Boards (IRBs): Post-Tuskegee, the requirement for IRBs became more prevalent. IRBs review and monitor research involving humans to ensure ethical standards.

Transparency and Accountability: The study led to regulations mandating transparency, data sharing, and mechanisms for accountability in the case of ethical breaches.

Public Trust and Participation: The Tuskegee study damaged public trust, particularly among African Americans. This underlines the importance of building and maintaining public trust for research participation.

Cultural Competency: The racial implications of the Tuskegee study highlighted the importance of cultural competency in research. Training in cultural competency has since become a norm.

Training in Research Ethics: The study led to mandatory training in research ethics for investigators conducting human subject research.

International Impact: The study had a global impact on observational study regulation. It influenced updates to the Declaration of Helsinki, an international set of ethical principles regarding human experimentation.

For more details about the Tuskegee Syphilis Study see the RWE 101 Supplement I published today…

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Real World Evidence (RWE) 101 – The Tuskegee Syphilis Study (the reason we have the Belmont Report and the Common Rule)2023-08-07T13:45:24+00:00

Real World Evidence (RWE) 101 – Observational Study vs Non-Interventional Study

RWE 101 – Observational Study vs Non-Interventional Study

In the context of real-world evidence (RWE), the terms “observational study” and “non-interventional study” are often used interchangeably to refer to studies that collect data outside the controlled environment of a clinical trial. However, it’s worth noting that some subtle differences can exist based on the specific context or regulatory guidelines. Here’s an overview:

[1] Observational Study: An observational study is a research design where researchers observe and collect data on participants without intervening or administering any specific treatment. Observational studies aim to analyze associations, correlations, or patterns in real-world settings. They can be prospective (following participants over time) or retrospective (analyzing existing data or medical records).

[2] Non-interventional Study: A non-interventional study is a study type (EU and US regulatory definition)  that does not involve any healthcare or treatment interventions imposed by researchers. It is often used as an umbrella term for studies that collect data in real-world settings, without manipulating variables. Non-interventional studies are primarily focused on describing, analyzing, or assessing outcomes, exposure, or associations.

It’s important to note that regulatory guidelines and definitions may vary across different regions and agencies. For instance, the US FDA’s guidance on RWE refers to “real-world studies”, “observational Studies”, and “non-interventional (observational) studies”, while the European Union Clinical Trials Regulation (Regulation EU/536/2014) uses the term “non-interventional studies.” However, in practice, the intent of these studies—collecting data without actively intervening—is often similar.

In the context of RWE, both observational studies and non-interventional studies typically leverage real-world data sources such as electronic health records, claims databases, registries, surveys, or patient-reported outcomes. They aim to generate evidence (real world evidence) on treatment outcomes, comparative effectiveness, safety profiles, and other healthcare-related factors.

Ultimately, the precise terminology used may vary, but the fundamental principle is that observational studies and non-interventional studies within the context of RWE both involve the collection and analysis of real-world data without actively imposing healthcare or treatment interventions on participants.

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Real World Evidence (RWE) 101 – Observational Study vs Non-Interventional Study2023-08-07T13:29:05+00:00

Real World Evidence (RWE) 101 – Are Non-Interventional Studies Regulated?

RWE 101 – Are Non-Interventional Studies Regulated?

Yes, non-interventional studies (NIS) are regulated. While the specific regulations and requirements may vary by country, there are generally guidelines and provisions in place to ensure the conduct and reporting of non-interventional studies meet certain standards.

Here are some key points related to the regulation of non-interventional studies:

Ethical Considerations: Non-interventional studies involving human participants must adhere to ethical principles and guidelines (i.e., Declaration of Helsinki) and be approved by an ethics committe (IRB/REC). These guidelines often cover aspects such as informed consent, confidentiality, privacy protection, and the rights and welfare of study participants.

Data Protection: Regulations related to data protection and privacy, such as the EU General Data Protection Regulation (GDPR), are applicable to non-interventional studies. Researchers must ensure that the collection, storage, and processing of personal data comply with these regulations.

Regulatory Oversight: Regulatory authorities may have oversight over non-interventional studies, particularly when the studies are the results of a regulatory commitment (e.g., PMRs and PASS). In the EU, for example, the EMA provides guidance on non-interventional post-authorisation safety studies (PASS) (GVP Module VIII).

Good Pharmacovigilance Practices (EU): Non-interventional studies focused on post-authorization safety assessments of medicinal products are subject to good pharmacovigilance practices. These practices include the collection, analysis, and reporting of adverse drug reactions and safety data.

Reporting Requirements: Non-interventional studies may have reporting requirements (e.g., EU PAS Register) to ensure transparency and accountability. This may include the submission of study protocols, study results, safety updates, or other relevant data to regulatory authorities or ethics committees.

It’s important to note that the specific regulations and requirements for non-interventional studies can vary between countries and regions. Researchers conducting non-interventional studies should be familiar with the applicable regulations in their jurisdiction and seek guidance from regulatory authorities, ethics committees, or relevant professional organizations to ensure compliance with the required standards.

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Real World Evidence (RWE) 101 – Are Non-Interventional Studies Regulated?2023-08-07T13:12:49+00:00
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