Bolster your investor narrative with RWE

To successfully compete for outside funding, your biotech must offer investors a compelling value story. The most successful stories include a robust clinical development plan, and depending on how close to commercial launch, a comprehensive launch plan. These are augmented by an integrated evidence generation plan (IEGP), a comprehensive strategy for delivering evidence that will demonstrate your product’s clinical and commercial potential and is foundational to the value story. 

During a recent webinar, we discussed how creating an IEGP is the best way to avoid common evidence gaps — deficits that can cost you time, capital, and credibility. An IEGP allows for avoidance of duplicity in evidence generation activities and more efficient consolidation of evidence needs (e.g. taking multiple local affiliate evidence needs and consolidating into a centralized evidence program).   

When developing your IEGP, we strongly recommend building in approaches for generating and using real-world evidence (RWE). While randomized clinical trials (RCTs) will be the foundation of your clinical development program, RWE can further strengthen your efforts and your value story, particularly for products that treat rare diseases or are on accelerated approval pathways. Leveraging RWE can also help you optimize your resources and improve the probability of success when negotiating favorable reimbursement rates, both of which will be important to potential investors.   

The outlook for RWE 

Collecting and analyzing data from real-world settings can support evidence generation across functions. With the growing availability of data from high-quality real-world sources — including claims databases, electronic medical records, patient registries, and more — regulatory authorities are increasingly considering RWE alongside RCT data to support claims of product safety and effectiveness. Research indicates that regulatory authorities are approving an increasing number of new product applications and label expansions that include RWE approaches and designs.^i,ii At the same time, we’re seeing growing maturity in guidance from regulators regarding the collection, analysis, and incorporation of RWE in drug development programs.ⁱⁱⁱ 

In addition to helping demonstrate a product’s safety and effectiveness, RWE can be a key tool to solidify market access and to gain market share through differentiation. Evidence generation that allows developers to show predictable and durable outcomes over time, can be used to support value-based agreements, and well-designed postmarketing evidence generation strategies can further improve the likelihood of optimal reimbursement at market entry. 

The role of RWE in your development plan 

As early as the preclinical stage, you can use RWE to better understand clinical unmet need, clinical practices, the effectiveness of the current standard of care and gaps in the care continuum. The evidence you generate can help you make informed choices about pursuing and allocating outside capital. 

RWE can also support effective clinical study design through a deeper understanding of patient characteristics, patient dosing preferences, clinician- and patient-preferred endpoints, patient journey and other factors. During reimbursement negotiations, RWE can further contextualize value. For example, you might use comparator/current standard of care data to demonstrate clinical and economic superiority to an on-the-market product.  

Additionally, patient-reported outcomes and clinical outcome assessments can help all stakeholders better understand quality-of-life measures and other endpoints important to patients in a real-world setting. 

Figure 1. RWE provides value throughout the product lifecycle.  

Integrating RWE for maximum impact 

RWE can be incredibly helpful for filling evidence gaps but no team can fill every gap at once. Biotech companies typically operate under unique scientific, financial, regulatory, and commercial pressures, and so their strategic prerogatives can differ from those of a more mature pharma company.  

When developing your IEGP, focus on the areas in which RWE can create the greatest possible value in relation to your current strategic prerogatives — this can be a fine balance between broader strategy and capital allocation. This will require your team to agree on your objectives. For example, if your immediate success depends on fast study startup, focus on RWE that will support identification of patients and predictable patient enrollment. If you need to contextualize ongoing RCT results, generate RWE based on clinical relevance to better contextualize go/no-go decision making. In addition to development goals, you should also consider your short- and long-term funding objectives, as the evidence to support seed funding may differ from the evidence needed for project launch funding or an initial public offering (IPO). 

We also recommend that you plan early for RWE, as meeting the high standards of stakeholders can be a complex undertaking. Your team will need to define a data strategy to execute on evidence generation activities. This may include identification and priming of multiple data sources, as well as a data harmonization strategy (e.g. linking disparate sources through a common data model and establishing standard definitions of populations and outcomes consistent with those studied in RCTs). You should address these considerations as early as possible in development to maximize the impact of RWD to generate RWE. 

In considering stakeholder standards, it’s critical to account for the full range of stakeholder needs. Earning regulatory approval is a critical step in drug development but addressing market access requirements is equally important. Without payer buy-in, your product will never effectively reach patients, so plan to generate RWE to demonstrate not only safety and efficacy but cost-effectiveness and comparative clinical benefit as well. As payers have also pivoted to value-based contracts that reimburse based on performance, developers should enter negotiations with the best possible understanding of their product’s probable outcomes and a robust evidence strategy to demonstrate this. We encourage developers to identify the RWD sources that can provide the best insight into long-term durability of response. This is particularly important for precision medicine products, which are often costly and largely unproven in broader real-world clinical practice. 

RWE is critical to creating a compelling investor narrative. During clinical development, you will generate an enormous amount of evidence — including RWE — from a huge number of sources. You will want to shape this data into a dynamic investor narrative that is comprehensive but also easily communicated and understood. To deal with the complexity of multiple data and evidence strands, a common trend is to leverage a dynamic evidence technology platform. Such platforms can be developed to obtain dynamic signals and insights from disparate data and evidence strands and can be used to generate evidence and insights on competitor products, generate real-time health economic signals, and monitor evolving treatment landscapes and patterns. To do this in a manually curated way can be both time-consuming and cost-prohibitive. 

In the increasingly complex and competitive biotech space, RWE will play a growing role in your success. Parexel’s experts can help you unlock its full potential to help secure critical funding and reach your goals faster. We’re always available for a conversation. 

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