Pragmatic Trials: Targeting evidence generation to inform market access and meet payers’ needs

The drug development community has achieved extraordinary success in recent years, from the rapid rollout of vaccines to breakthroughs in the efficacy of treatments for rare diseases. However, as treatments become more specialized, reimbursement and market access are more critical to sponsors’ success than ever before. New therapies are groundbreaking in terms of impact and effect, but today’s pricing models are unsustainable for these new and innovative therapies.

For payment systems to evolve to deal with these challenges, payers are taking a new look at evidence generation and data usage. Medicines are so specialized today that it is incumbent on sponsors to create insights and evidence targeted to payers’ needs: a pragmatic, cost-efficient approach.

The trend toward low-intervention studies

Historically, generating evidence to demonstrate efficacy in late-phase trials relied on interventional studies. Retrospective data were considered less credible. What was needed was a scientifically robust and rigorous model that is less complex and burdensome than a fully interventional study. Thus, we have seen a strong trend away from late-phase interventional studies and an explosion in the application of real-world studies and data.

These real-world studies, also known as pragmatic trials, aim to capture a patient’s care path in a structured research model with minimum intervention. The research is typically conducted in real-world clinical practice settings, including a hospital, physician’s office, clinic, or the patient’s or caregiver’s home.

The value of leveraging existing data to fill evidence gaps

Essentially, the data generated are a byproduct of the patient’s experience during their journey through the disease. Establishing mechanisms for collecting these data that are robust and credible can broaden the acceptability of this approach. These mechanisms might use the full apparatus of a health system, including electronic health records, telemedicine, claims data, product and disease registries, patient-reported outcomes, and health applications such as social media and wearables. The real-world data generated becomes the source for deriving insights – real-world evidence, or RWE – about the product’s effectiveness.

More focused data, more cost-efficiency

Pragmatic trials can represent the minimally interventional mechanism to collect more focused data that can feasibly detect patterns and provide insights that can be used to fill gaps in evidence. This approach can demonstrate effectiveness for endpoints required by payers that are not otherwise covered. And crucially, it enables controlling for other variables, leading to a more predictable outcome and a better understanding of how a specific population will respond.

However, the key to success is understanding all stakeholder perspectives, including payers. This understanding will inform the trial’s design and how to conduct the research to generate evidence to fill gaps, establish effectiveness in specific patient populations and inform pricing decisions.

Parexel has long been a leader in innovative thinking in late-phase study design to help reduce the overall cost of gathering evidence and solve market access challenges. Parexel can support payer submissions using RWE by identifying the right endpoints and operationalizing real-world and pragmatic trial designs. Our experience in studies generating real-world evidence has allowed us to establish a deep understanding of stakeholders’ evidence needs in the context of the market landscape, the current standard of care, patient profiles, insights, and evidence gaps.

To further explore the topics of pragmatic trials and RWE, please get in touch – [email protected]

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