For a greater chance of success, pharmaceutical companies must emphasize clinical trial validity to ensure that the outcomes are relevant and applicable to patients in real-world clinical settings and that the results can withstand increased scrutiny from the Food and Drug Administration (FDA).
“By saying your clinical trial is valid, you’re saying that the results are meaningful and interpretable,” explains Paul Williams, Psy.D., senior pharma account manager for Pearson Assessments. “To make this claim, every aspect of the study must be held to very high standards.”
However, failure to demonstrate efficacy, safety concerns, issues with inclusion and exclusion criteria, and patient recruitment are among the most common reasons clinical trials fail. Intensifying scrutiny from the FDA has also led to dramatic increases in the number of warning letters issued to drug manufacturers that threaten the commercialization of new therapeutics.
Following these five steps can increase the odds of clinical trial success.
Choosing the best study design.
In 2022, 14.5% of interventional clinical trials were terminated before completion. Optimizing the design of a clinical trial can have a significant impact on its success and provides the foundation for better outcomes. In contrast, a flawed study design can affect whether a clinical trial demonstrates efficacy.
Successful clinical trials consider inclusion/exclusion criteria and site selection as well as patient recruitment, retention and burden to improve outcomes. With proper oversight, artificial intelligence can be used to conduct literature reviews, assess the suitability of eligibility criteria, predict patient dropout rates and compare statistical methods with successful outcomes to help researchers choose the best study design.
Selecting the right endpoints
Selecting endpoints is complex, and making the wrong choice can jeopardize the trial. In 2021, a high-profile clinical trial for an HIV vaccine failed to meet the endpoint of reducing HIV transmission by 50% compared to placebo, leading to the discontinuation of the trial.
In drug development, clinical trials often contain multiple endpoints. While increasing the number of endpoints allows researchers to better assess the impact of the drug on multiple characteristics of a disease, it also increases the odds of making false conclusions about those endpoints.
Using valid clinical assessments to measure outcomes can ensure the validity of clinical findings.
“Establishing reliability is essential to ensure that changes over time in a patient’s performance are the result of disease progression and not poor test items or administration and scoring errors,” says Lynsey Psimas, Ph.D., Pearson’s director of sales, pharma services.
Deploying the right assessment tools
Valid assessments are an integral part of clinical trial success. Research showed that 61% of the measurement tools used in trials had no evidence for measurement properties. The reliability and the remaining tools were rated “sufficient” for validity.
Choosing clinical outcome assessments (COAs) to generate outcomes that are meaningful to patients is essential, Williams says.
“To help ensure your trial is valid, it’s necessary to choose valid and reliable instruments,” he says. “Shortcuts lead to reducing validity in ways that are not obvious or totally understood resulting in messy data and faulty conclusions.”
Developing COAs for a clinical trial isn’t just time-consuming; it’s expensive and requires expertise. It also has the potential to affect the outcomes. Most Pearson COAs can be adapted for electronic administration, which helps with patient compliance, virtual data collection, and reduced errors in administration and scoring while streamlining efficiencies and saving time.
Recruiting study participants
Recruitment failure can be linked to a host of issues from mistrust in research and lack of logistical support to unrealistic estimates and lack of patient engagement in trial design.
Technology can aid in clinical trial recruitment. Using AI to analyze medical records and social media content to identify suitable participants cuts down on the time and resources required to meet recruitment goals.
Ensuring data quality and validity
The success of a clinical trial relies on collecting high-quality data. Not all studies follow guidelines to ensure data integrity, which affects the generalizability of research findings.
“The quality of data directly impacts the usefulness of the data in human clinical trials,” Psimas says. “Using tools developed in a psychometrically sound way and demonstrating reliability and validity is critical to making sound medical decisions about new therapies as these therapies are dependent upon reliable data from clinical trials.”
Providing education to promote standardized practices, monitoring data quality, establishing a data management plan and prioritizing real-time data collection helps improve data quality and reduce the volume of missing data.
“The term ‘clinical’ means you are dealing with the health and wellbeing of others,” Williams says. “Those involved at every level of clinical trial research must adhere to the highest standards possible.”
For more information about how Pearson Assessments’ Pharma Research Services division helps to ensure clinical trial data is reliable, valid and can withstand rigorous FDA scrutiny, contact Pearson Assessments’ Pharma Research Services for a consultation.