实现双赢：通过战略规划设计更具代表性的试验

Table of contentsIntroductionHow does prioritizing representativeness benefit my trial?Scientific benefitsOperational benefitsRegulatory benefitsCommercial benefitsHow should I approach planning for a representative trial?Characterize differences within the patient populationGenerate distribution estimatesSet strategic goalsLeverage insights to craft successful operational strategiesWhat should I look for in a strategic partner?What’s next?About IQVIAAbout the authors 3444556677789910 In the world of clinical trials, the ultimate goal is to execute a well-designedtrial that yields high-quality, robust evidence that sufficiently characterizes anddemonstrates the therapy’s efficacy and safety on an efficient timeline. En routeto this goal, sponsors should not underestimate the importance of planning forrepresentative enrollment, a process that aims to study populations and generatedata representative of the real-world patient population. The FDA reinforcedthe importance of planning for representative enrollment in 2022 and againin 2024 when they issued draft guidance on how to effectively develop andsubmit a Diversity Action Plan (DAP) for clinical studies.As stated by the FDA in its guidance, “actions to improverepresentativeness in clinical studies can support moreequitable and timely access to medical discoveries andinnovations, improve the generalizability of resultsacross the intended patient populations, improve ourunderstanding of the disease and/or medical productunder study, and inform the safe and effective use ofthe medical product for all patients.1” In addition tothe understanding and insight provided by achievingrepresentativeness, the FDA-outlined approach for DAPsallows for accelerated enrollment by expanding a trial’sreach and enabling more tailored recruitment.IntroductionUnderstanding the demographic makeup of a program’sintended use population and tailoring operational planswith that knowledge improves a trial’s ability to achieverepresentative enrollment and generalizable data.1.FDA (2024, June). Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies Guidance for Industry.U.S. Food and Drug Administration. https://www.fda.gov/media/179593/download. iqvia.com | 3While there is currently uncertainty around whetherthe FDA’s expectations for submitting DAPs willcontinue, what remains certain is that understandingthe demographic makeup of a program’s intendeduse population and tailoring operational plans withthat knowledge improves a trial’s ability to achieverepresentative enrollment and generalizable data,which is an enduring regulatory, scientific, medical,and commercial concept. In the following text, we explorethe benefits we have seen from work on DAPs and howsponsors should pursue planning for representativenessto gain those advantages. 4 | Achieving a Win-Win: Designing More Representative Trials Through Strategic PlanningII. Operational benefitsUnderstanding the intended use population early on canhelp inform operational activities of the trial, allowingsponsors to fine-tune eligibility criteria, statistical analysis,site strategy and selection, investigator training, andrecruitment strategy. A sponsor can accelerate andenhance study recruitment by strategically selecting siteswhere patients are more likely to qualify for the trial andtailor local outreach accordingly. In one case, we saw thatwhile real-world data showed low prevalence among thebroader Hispanic population, a literature review uncovereda particularly high propensity to have the condition amongthose of Mexican descent. This insight fueled more precisesite selection, educated investigators, and guided patientoutreach, leading to above-goal enrollment ahead ofschedule4. When combined, these subpopulation insightscan help rapidly identify eligible patients, accelerateenrollment, and increase the speed to market. How does prioritizingrepresentativeness benefitmy trial?Designing more representative trials has already provenitself as a valuable investment. Scientific, operational,regulatory, and commercial advantages will continueto reveal themselves as the effort to make trials morerepresentative expands.I. Scientific benefitsHistorically, clinical development has started with theassumption that all patients are the same, missingthe opportunity to consider how differences acrossdemographic subgroups could impact outcomes.However, many drugs exhibit variability in effect acrossdifferent demographic groups (e.g., beta-blockersand ACE inhibitors have been demonstrated to havedifferences in effect in Black and White populationswhen used to treat hypertension2,3). As such, it is criticalto gather thorough data to understand variability acrossthe intended use population. Ensuring representativeenrollment contributes to data generalizable to thefull patient population and enables earlier detectionof potential variability in effect and safety.In addition, building a ro