In clinical trials, primary outcome measures play a pivotal role in determining the effectiveness of treatments and interventions. However, understanding primary outcome measures can be challenging for both patients and advocacy groups. In this article, we will be demystifying primary outcome measures and highlighting the significant role patient advocacy groups can play in informing them.
What are Primary Outcome Measures?
Primary outcome measures are measured by researchers to assess the effect of an intervention/treatment in a clinical trial. These measures are carefully selected based on several factors, including:
- The phase of the study. For example, the primary outcome measure in a phase 2 clinical study is usually a measurement of safety (e.g., side effects, tolerability) whereas in a phase 3 clinical trial it is usually a measurement of efficacy.
- The study’s objectives. If the study wants to determine if a medication can reduce seizures, a likely primary outcome measure would be the number of seizures per week.
- Which outcomes are most clinically relevant (i.e., what elements of the targeted disease are the most debilitating for patients.)
- What regulatory bodies look for. Most regulatory bodies prefer standardized and validated measures, so that data from one clinical trial can be more easily compared to data from other, similar trials.
Common primary outcome measures include survival rates, disease progression, symptom improvement, or quality of life assessments.
Differences in perspectives between researchers, clinicians, regulators, and patients can make it quite difficult to reach a consensus on the most appropriate primary outcome measure in a clinical trial. For this reason, secondary outcome measures are often included in trials, which allows additional clinical parameters or patient-reported outcomes to be considered. This provides a more comprehensive understanding of treatment efficacy and safety. With that said, if a study does not show that its primary outcome measure is statistically significant, or if a presentation of data focuses more on impressive secondary outcomes than the primary outcome, the odds of that intervention proceeding to the next phase of a clinical trial or getting approved by a regulatory agency are low.
How Patient Advocacy Groups Can Inform Primary Outcome Measures:
Patient advocacy groups play a vital role in bridging the gap between researchers and patients. These groups represent the voices and interests of patients affected by a particular disease or condition. By actively engaging with researchers and clinical trial sponsors, patient advocacy groups can provide valuable insights into the lived experiences and priorities of patients.
Patient advocacy groups can inform outcome measures in a number of ways:
- By providing patient input. Patient advocacy groups can facilitate patient engagement activities, such as focus groups, surveys, or interviews, to gather input on potential outcome measures. This direct feedback from patients can inform researchers about the most important aspects of the disease or treatment outcomes from a patient’s perspective.
- By advocating for patient-centred outcomes. Patient advocacy groups can advocate for the inclusion of outcomes that matter most to patients. By sharing their experiences and preferences, they can influence the selection of primary outcome measures that capture the impact of the disease and its treatment on patients’ lives. Patient advocacy groups can also advocate for the inclusion of patient-reported outcomes (PROs) as primary outcome measures, as they offer valuable insights into the subjective experiences and perceptions of patients.
- By participating in study designs. Patient advocacy groups can actively participate in the design phase of clinical trials, including the selection of primary outcome measures. By collaborating with researchers, they can ensure that study endpoints are relevant, meaningful, and reflective of patients’ needs and priorities.
By actively engaging with researchers and clinical trial sponsors, patient advocacy groups can ensure that primary outcome measures are patient-centred, meaningful, and reflective of patients’ needs and preferences. Through collaborative efforts, researchers, clinicians, and patient advocacy groups can work together to improve the relevance and effectiveness of clinical research outcomes, ultimately benefiting patients and advancing medical knowledge and care.
