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Real World Evidence (RWE) 101 – Ethical Imperatives (CIOMS 2023)

RWE 101 – Ethical Imperatives (CIOMS 2023)

Informed decision making with patients typically relies on evidence from clinical trials that describe the likely benefits and toxicities. However, patients treated in everyday practice tend to be older and more frail, to have poorer function and performance status, and to have more comorbidities and less social support than those selected to participate in clinical trials. Thus, generalisability to typical patient populations treated in daily practice is often limited. Kennedy-Martin et al explored the generalisability of RCTs in cardiology, mental health, and oncology by assessing studies comparing participants in such trials with those in everyday clinical practice. Patients treated in everyday clinical practice tended to be older, were more often women, and had more comorbidities; 71% of studies concluded explicitly that RCTs were not broadly representative of real-world patients, in particular, pregnant and lactating women are a very large population that is often entirely unrepresented in clinical trials. Furthermore, patients enrolled in trials were treated according to guidelines more often and received more in-hospital procedures. Strict selection criteria for RCTs meant that participants were at a much lower risk of adverse events compared with patients treated in clinical practice.
If the efficacy-effectiveness gap means that patients are being given inaccurate information about the potential benefits and risks of treatments, then decisions made using that information may be being made without valid informed consent, disrespecting patient’s autonomy and putting them at risk of avoidable harm. The efficacy-effectiveness gap also raises important issues regarding justice; if resource allocation decisions, including which treatments are funded, are made using evidence that is biased by the efficacy-effectiveness gap, then those decisions will also be flawed, with potentially wide implications for patients. Ultimately, the efficacy-effectiveness gap undermines the gold standard status of RCTs, and actually suggests that it would be unethical to continue with such a flawed representation of real-world effects on patients. Increasing use of RWE is one important way to fill the efficacy-effectiveness gap and augment the evidence from RCTs. This should not be seen as dropping the gold standard, or diminishing the standard of evidence required; rather, enhancing and supplanting RCT evidence with RWD can instead be viewed as reinforcing the gold standard with platinum plating.
Source: Section 4.2 (Ethical arguments for incorporating more RWE) of the CIOMS Draft Guidance – Real-World Data and Real-World Evidence in Regulatory Decision Making (6 June 2023)

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Real World Evidence (RWE) 101 – Ethical Imperatives (CIOMS 2023)2023-08-07T21:46:07+00:00

Real World Evidence (RWE) 101 – Ethical Imperatives

RWE 101 – Ethical Imperatives

Real-world evidence (RWE) refers to the clinical evidence regarding the usage and potential benefits or risks of a product derived from analysis of real-world data (RWD). These data are routinely collected from a variety of sources, such as electronic health records, claims and billing data, patient registries, and data gathered through personal devices. RWE is increasingly being used in clinical trials and marketing authorisations.
The ethical imperative of incorporating RWE into clinical trials intended to support marketing authorizations arises from several factors:
[1] Patient-centric Approach: RWE provides a more holistic and accurate view of the patient experience as it considers a wide range of factors such as comorbidities, patient behaviour, social determinants of health, and the full array of interventions. This allows for a patient-centric approach in medical research, which respects the dignity, rights, and welfare of individuals.
[2] Generalizability: RWE is collected from diverse patient populations and settings and can therefore inform on how a drug or intervention works in the ‘real world,’ beyond the controlled environment of randomized controlled trials (RCTs). This increases the generalizability of research findings and supports the ethical principle of justice, ensuring that the benefits of research are applicable and beneficial to all groups.
[3] Safety and Effectiveness: RWE can provide additional information on a drug’s safety and effectiveness in broader populations and over longer periods, beyond what can be feasibly assessed in RCTs. This can provide early signals of potential harm and help prevent unnecessary patient exposure to harmful interventions.
[4] Efficiency: Incorporating RWE can make research more efficient, reducing the time and cost of bringing a new product to market. This is ethically significant as it can accelerate patient access to beneficial new treatments.
[5] Informed Decision Making: RWE helps physicians, patients, and policy-makers make informed decisions. Better evidence can improve the quality of care, patient outcomes, and health system efficiency.
[6] Inclusivity: Traditional clinical trials often exclude certain patient groups, such as the elderly or those with multiple health conditions. RWE includes data from these often under-represented groups, ensuring that the findings are applicable to them.
The importance of RWE lies in its ability to provide an enhanced understanding of a drug’s safety, effectiveness, and value in routine clinical practice. It can capture the wide heterogeneity of patients, healthcare providers, and treatment settings, providing a much-needed complement to traditional RCTs. By integrating RWE into the process of marketing authorizations, regulators can make more informed decisions, ensuring that only safe and effective drugs reach the market, ultimately improving patient health outcomes.

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Real World Evidence (RWE) 101 – Ethical Imperatives2023-08-07T21:29:44+00:00
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