Real-World Evidence at Every Stage – How Aetion Powers the Drug Development Lifecycle

Natalie Schibell, MPH and Ashley Jaksa, MPH

Drug development is driven by high-stakes decisions across research, clinical design, regulatory strategy, and commercial execution. Each decision plays a defining role in a therapy's success, yet it is often made without a clear understanding of what is happening in the real world.

Aetion® was built to change that.

We equip life sciences teams with the tools, technology, and scientific expertise to generate decision-grade real-world evidence (RWE). Powered by Aetion Evidence Platform® (AEP), our integrated solutions support the production of scientifically credible, regulatory-aligned insights, reducing risk, informing strategy, and aligning stakeholders across the product lifecycle.

Building a Strong Foundation for Development

Decisions must be made before a trial begins, before a market is entered. Which populations are most underserved? Where is there room to differentiate? What does success look like, not in theory but in clinical reality? Aetion helps teams answer these questions early and clearly, creating a more grounded foundation for development. 

How Aetion Supports the Critical Decisions that Shape Early Development

Strengthening Clinical and Real-World Relevance

Designing trials that reflect real-world populations—and ultimately developing therapies that succeed in practice—requires more than traditional methods. It demands evidence grounded in care, cost, and patient experience. Aetion provides both: AEP enables the rapid generation of scientifically credible RWE, while our scientific experts help sponsors interpret results and act confidently. Here’s how that combination translates into value across core evidence planning domains:

Natural History and Standard of Care Characterization

• What it enables: Understanding how diseases progress and how care is delivered in practice—essential inputs for endpoint selection, comparator choice, and trial feasibility.
• In action: Aetion used real-world data (RWD) to evaluate the eligibility criteria for a proposed late-line oncology trial. The original inclusion/exclusion criteria—particularly strict definitions of prior treatment failure and performance status—excluded most real-world patients who would otherwise be clinically appropriate for enrollment. Feasibility modeling showed that, under those parameters, recruitment timelines would be severely constrained. Using AEP, our team collaborated with the sponsor to adjust the criteria, preserving scientific rigor while enabling more representative and achievable recruitment.
• Platform + People: AEP supports descriptive study design by enabling comprehensive cohort characterization, treatment pathway mapping, and longitudinal follow-up—key elements of natural history analyses. Our scientific team collaborates with sponsors to translate these findings into actionable inputs for endpoint selection, comparator choice, and trial feasibility.

Generalizability Assessment

• What it enables: Evaluating whether clinical trial populations accurately represent real-world patient demographics and care environments, ensuring that study findings apply to everyday clinical practice.
• In action: Aetion's collaboration with COTA enhanced access to diverse real-world oncology data. This enabled comprehensive evaluations of whether trial populations reflected the broader cancer population, supporting regulatory discussions and strengthening confidence in the real-world applicability of trial findings.
• Platform + People: AEP supports transparent cohort analyses, allowing sponsors to assess representativeness early and often. Aetion’s scientific experts help interpret findings and integrate generalizability assessments into trial design, regulatory strategy, and external communication.

Burden of Disease and Resource Utilization Analysis

• What it enables: Quantifying costs, understanding utilization patterns, and assessing the broader impact of diseases. This information is essential for prioritizing healthcare initiatives, setting pricing strategies, and effectively communicating value to stakeholders.
• In action: Aetion conducted a retrospective claims analysis focusing on patients with psoriasis and concomitant joint disease. The study revealed that these patients incurred greater healthcare resource utilization and higher costs than those with psoriasis alone. These insights demonstrated the importance of early diagnosis and treatment of joint symptoms to improve patient outcomes and reduce economic burden.
• Platform + People: AEP is built to analyze large-scale claims and EMR datasets, enabling precise measurement of cost outcomes, care utilization patterns, and disease burden. Aetion’s scientists partner across functions to convert these data into evidence that supports pricing strategy, market access planning, and value communication.

Humanistic Burden and Quality of Life Insights

• What it enables: Identifying the patient populations where a therapy can deliver the greatest value, based on real-world disease trajectories, progression risk, and quality-of-life impact. These insights support precision development, benefit-risk assessments, health technology assessment (HTA) submissions, and long-term access strategies by ensuring that therapies are targeted to those who benefit most.
• In action: Aetion collaborated with Cognito Therapeutics to develop a validated, claims-based model to identify patients at highest risk of rapid progression from mild cognitive impairment (MCI) to Alzheimer’s disease. Drawing on RWD from over five million Medicare and commercially insured patients, the model supports early identification of high-risk individuals for clinical trial enrichment and treatment targeting. These insights inform precision development strategies, enable burden-of-illness and cost analyses, and strengthen future HTA submissions by capturing population-level risk and unmet need in neurodegenerative disease.
• Platform + People: AEP enables the integration of diverse real-world data sources—including claims, electronic medical records, and disease registries—to generate high-resolution, patient-centered insights. These sources often include patient-reported outcomes that help quantify quality of life, symptom progression, and functional status. Aetion’s scientific experts work cross-functionally with clinical, HEOR, and access teams to translate these insights into meaningful evidence for HTA submissions, access strategy, and stakeholder communication.

Enabling Access, Launch, and Lifecycle Value

As therapies approach launch, the pressure to deliver timely, credible, and actionable evidence grows. Payers, regulators, and internal stakeholders demand proof beyond trials—evidence that drives strategy, supports access, and sustains long-term value. This is where Aetion excels: translating RWD into insights that power confident decisions across commercialization.

Here’s how Aetion helps sponsors sustain that momentum:

• Generate payer-relevant evidence: Aetion helps teams produce RWE that demonstrates real-world value—linking clinical benefit, economic impact, and quality of care. These insights support global HTA submissions, pricing negotiations, and coverage decisions.
• Support global market access: Aetion tailors analyses to regional data, treatment patterns, and payer needs—equipping sponsors with localized evidence strategies that scale globally and improve positioning with decision-makers.
• Monitor real-world safety and value: Aetion enables ongoing assessment of therapeutic performance post-launch—capturing safety signals, real-world effectiveness, and utilization trends. These insights support regulatory compliance, risk management, and lifecycle value demonstration.
• Accelerate asset integration: Aetion delivers rapid insight into disease burden, treatment pathways, and evidence gaps—enabling faster portfolio alignment, evidence planning, and value maximization for new assets.
• Drive commercial precision: Aetion equips commercial and medical affairs teams with insights into real-world prescribing, provider behavior, and care dynamics—informing segmentation, messaging, and field strategy to optimize uptake.

The Aetion Difference

The Aetion Evidence Platform® (AEP) is purpose-built to power applications that generate decision-grade real-world evidence—anchored in validated methods, transparent study design, and global regulatory standards. From data curation to regulatory submission, AEP supports the end-to-end lifecycle of evidence generation.

AEP powers three integrated applications, each serving a distinct role in the evidence journey:

• Aetion® Discover enables teams to explore population characteristics, care patterns, and treatment pathways, helping to identify the right data, refine study design, and inform cohort selection.
• Aetion® Generate drives secure, scalable evidence generation through three integrated modules: Protect ensures data privacy and regulatory compliance; Replicate supports compliant data sharing with high-fidelity synthetic datasets; and Enhance applies generative modeling to fill data gaps and strengthen analyses in complex or underrepresented populations.
• Aetion® Substantiate ensures methodological transparency and reproducibility, aligning studies with regulatory and HTA requirements. It provides full traceability and documentation for internal governance and external review.

Aetion delivers evidence for exploration and high-stakes decision-making—whether for regulatory submission, payer engagement, or internal strategy. Outputs are built to meet the standards of reviewers, clinicians, and health economists, with traceability and consistency across R&D, HEOR, medical, and market access.

Others summarize data. Aetion delivers evidence that stands up in the room—and we’ll stand there with you. Defensible, actionable, and built for decisions that matter.

Partner with Aetion to Elevate Evidence-Driven Development

Contact us to explore how Aetion® can help your team build and execute a timely, relevant, and scientifically sound evidence strategy—no matter where you are in the development journey.

In environments where uncertainty can stall progress, trusted evidence accelerates it. Aetion delivers the clarity to act and the credibility to move forward with confidence.