Evidence at the Forefront: Why Integrated Evidence Planning is Essential for Biopharma Innovation?

The Importance of Integrated Evidence Planning

Integrated evidence planning (IEP) is increasingly recognized as a critical necessity in the biopharmaceutical industry due to its ability to streamline and synergize evidence generation across various stages of drug development. With evidence required at every level, IEP facilitates a cohesive approach that addresses the diverse needs of stakeholders, ultimately enhancing the success of new therapies in the market.

Synergistic Role of Evidence Components

The IEP framework harmonizes various evidence-generating activities, empowering biopharma companies to efficiently address the distinct needs of multiple stakeholders, including:

Each stakeholder group poses unique questions regarding a product's value, effectiveness, and safety. IEP enables biopharma companies to generate and coordinate the necessary evidence to address these diverse inquiries, ensuring:

• Comprehensive evidence packages: Meeting the requirements of each stakeholder group
• Efficient resource allocation: Minimizing duplication of efforts and reducing costs
• Enhanced stakeholder engagement: Facilitating effective communication and addressing specific concerns
• Improved product adoption: Supporting informed decisions and driving market access

Though usually only considered at larger pharmaceutical companies, IEP’s focus on an efficient allocation of resources means it is most important in smaller companies, which often operate under limited funds. If the budget prevents a smaller company from investing the upfront resources necessary to set up an IEP, it can consider contracting through third parties already skilled in the subject.

Axtria’s Client-Focused Integrated Evidence Plan

A critical aspect of strategic product launch planning is developing evidence related to the burden of illness and the product’s economic value. Even before the readout of pivotal trial results, a substantial amount of information is often accessible from existing literature and real-world data (RWD) to support these efforts, even in the case of rare or under-researched conditions. In one case, the Axtria RWE/HEOR/ES team applied the IEP approach for a small pharmaceutical client before the launch of a medical device designed to prevent surgery-related complications.¹

Axtria’s first initiative was to conduct an extensive systematic literature review (SLR) to assess the clinical burden associated with surgeries, identify relevant patient populations for the client, and quantify the clinical burden.

This facilitated the development of an early economic model, demonstrating significant cost savings as the preventative device averted costly post-surgical complications. However, due to the limited availability of data in the literature, the full burden of disease could not be comprehensively captured.

A study utilizing RWD was conducted to address this evidence gap, comparing patients with surgical complications to those without over a period of 3.5 years. This analysis revealed additional costs incurred by patients with complications and provided real-world validation of the outcomes identified through the SLR. To further enhance the credibility and visibility of these findings, they were disseminated through key health economic and clinical journals and conferences.

Building upon the extensive peer-reviewed evidence from RWD analyses, the initial economic model is now being revisited to see whether it can be refined into a flexible tool for communication with payers and other key stakeholders. Through this iterative process of evidence generation and re-evaluation, Axtria has demonstrated the full impact of an integrated approach to evidence generation and meticulous execution of critical projects. This has positioned our small pharmaceutical client for a successful product launch and effective reimbursement negotiations.  

¹ Axtria takes the same approach to implementing IEP for all medical innovations, although this particular case involves a medical device.

Systematic Literature Review (SLR) and Meta-analyses:

The initial phase of executing an SLR ensures that all relevant existing research is identified and synthesized. This foundational step provides a comprehensive overview of the current evidence landscape, highlighting gaps and informing subsequent analyses.

Following the SLR, conducting meta-analyses allows for a more nuanced understanding of the data by aggregating results from multiple studies. This enhances the reliability of findings, and the inputs obtained from the current body of literature support the development of strong economic models that predict the potential outcomes of the client’s product over the next five-to-ten-year horizon.

Economic Modeling: Demonstrating Cost Savings Through Preventive Measures

An early economic model was developed, including cost-effectiveness (CE) and budget impact analyses. Early economic models can be leveraged in several ways to support the client's evidence needs and product positioning, empowering clients to:

• Demonstrate product value to payers
• Inform pricing strategies
• Shape market access
• Support product differentiation
• Guide clinical development
• Facilitate lifecycle management

The original version of Axtria’s economic model for our small pharma client was based on a meta-analysis of existing literature, consisting of a cost-effectiveness (CE) model with a decision tree design. This was further expanded to integrate a budget impact model based on epidemiological information in the literature. By harnessing the budget impact and CE models, we enabled our client to:

• Showcase clinical and economic benefits to payers
• Optimize pricing and value-based arrangements
• Tailor market access strategies for diverse payer segments
• Differentiate their therapy through robust value evidence
• Inform clinical development and future trials
• Adapt to evolving market conditions through continuous model updates 

These capabilities enable clients to navigate complex healthcare landscapes, drive business success, and ultimately deliver valuable treatments to patients. Axtria’s economic model was designed in consultation with subject matter experts who have many years of experience in the field, focusing on the flexibility to adapt to ever-changing input parameter values.

Axtria also has a strong expertise in modeling complex diseases. As described in a recent whitepaper, “Modeling the ‘Difficult to Model’ Disease,” Axtria adopts unique approaches to building economic models for rare diseases. These economic models utilize insights gained from early literature review sources and meta-analysis. Biopharma companies can use them to plan according to the financial implications of their medical devices/products. These models are vital for demonstrating value to payers and healthcare providers, thereby facilitating market access and reimbursement discussions.

Validation of Burden of Illness Through Real-World Data

The next phase of our enacted IEP involved RWD analyses to validate the findings from the previously conducted SLR and meta-analyses, providing a crucial layer of evidence. Real-world evidence (RWE), the result of RWD analyses, offers a unique lens into the clinical effectiveness of therapies in practice, revealing how they perform in real-world settings, often differing from controlled trial environments. By integrating RWE into economic models, biopharma companies can:

• Refine product projections: Ensure forecasts are anchored in actual patient experiences and outcomes
• Enhance accuracy: Validate burden of illness estimates with real-world data for both clinical and economic outcomes, along with safety and efficacy
• Inform decision-making: Leverage RWD insights to guide development, market access, and commercial strategies
• Bridge evidence gaps: Supplement controlled trial data with real-world evidence, providing a more comprehensive understanding and strong evidence for regulatory bodies and payers

Axtria has extensive expertise in the RWE and HEOR fields, as demonstrated in a recent whitepaper, “Seeing the Whole Elephant: Integrated Advanced Data Analytics in Support of RWE.” This paper further illustrates how integrating all methods and data can guide a holistic, analytic approach and decision-making. RWD analysis offers a powerful tool for biopharma companies to validate and refine their understanding of the burden of illness, ultimately leading to more informed decisions and effective therapies that meet real-world patient needs.

Publish or Perish: The Importance of Evidence Communication

After each phase of evidence generation, results were published for more effective evidence communication. Axtria disseminated four manuscripts in high-impact economic and clinical journals, covering the SLR, economic model, and RWE. In addition to these publications, Axtria is actively involved in developing and delivering conference presentations and other valuable communication materials. For example, the SLR, economic model, and RWE results of our small pharma clients' IEP projects were each presented at prestigious economic and clinical congresses. In particular, Axtria was a sponsor at ISPOR 2024, wherein our team showcased numerous presentations, including the case study discussed here.

As exemplified in this case study, Axtria deeply understands the importance of IEP implementation. Our recent whitepaper, The Evolution and Future of Integrated Evidence Planning, further demonstrates this. Through effective integrating evidence generation under the IEP framework, Axtria helps biopharma companies navigate the complex landscape of stakeholder requirements, ultimately bringing valuable treatments to patients more efficiently.