Leveraging Real-World Evidence for Indication Expansion

Regulatory Bodies Increasingly Recognize the Value of RWE in Decision-making Processes

Real-World Evidence (RWE) plays an increasing role in recent regulatory decision-making. Instead of conducting time-consuming and costly clinical trials, RWE can offer comprehensive insights from diverse patient populations to potentially accelerate regulatory approvals, providing a cost-effective indication expansion option that must be considered. RWE can also be a valuable pairing to insufficient RCT outcomes.

BSP excels in leveraging RWE, including for this purpose. We have extensive experience evaluating and utilizing a wide range of real-world data sources, including clinical trials, claims, electronic health records, registries and published studies. Our diverse, capable team has a proven track record of supporting agency communications to meet our clients’ regulatory objectives.

BSP is actively pursuing label expansion initiatives for existing treatments using real-world evidence (RWE) in both rare and chronic disease spaces

BSP's framework for evaluating RWE is a three-pronged approach, which enables us to ensure we have the right data to fit the regulatory needs

For instance, with the FDA:

BSP’s Workflow:

Kickoff/Planning

Review current evidence and client regulatory strategy options
Conduct stakeholder/KOL interviews
Formulate regulatory strategies and likelihood of success
Align chosen strategy with internal objectives

Study plan development

Develop study protocol framework
Identify potential datasets that support study design
Evaluate study feasibility for robust cohorts/required data fields
Draft statistical analysis plan (SAP)

Propose plans to Regulatory Agency

Prepare briefing documents
Schedule and attend meetings with the Agency
Answer Agency queries or counter proposals

Implementation

Finalize RWE study protocol/SAP
Execute RWE analysis
Conduct regular progress meetings with clients

Regulatory submission

Compile study findings
Develop submission package for Agency
Answer Agency queries or counter proposals

Relevant Experience and Past Work

Case 1. Our client sought to achieve indication extension for their plasma-derived products by leveraging RWE

BSP’s Approach:

Identify, critique, and estimate the likelihood of regulatory success for each potential strategy to establish a rank order of the options
Conduct feasibility assessment of potential RWE source data
Make a successful proposal to FDA to achieve the agreed research plan for each prioritized initiative
Conduct any necessary agreed analyses

Case 2. Our client conducted a pivotal clinical trial using a biologic in the neurosurgical space, the outcomes of which met primary safety endpoints but missed the primary efficacy endpoint. Therefore, they wished to explore the feasibility of label expansion harnessing RWE, among other possible regulatory strategies

BSP’s Approach:

Review the totality of all data available for the product, RWD analytics conducted to date, and patient exposures
Conduct a targeted literature search for all published evidence that may be used to support a regulatory strategy and FDA filing
Formulate potential strategies informed by available evidence; critique and estimate the likelihood of regulatory success of each one, to create a rank order of options
Align with client on selected strategy
Construct a proposal to FDA/ meeting packet to support the prioritized strategy
As needed, conduct any necessary agreed analyses post meeting to support filing

Contact BSP today to explore how our unparalleled expertise in RWE can support your indication expansion program

BSP is skilled in utilizing RWE to drive indication expansion. Our comprehensive approach integrates cutting-edge data analytics, clinical expertise, and regulatory insights to maximize the potential of existing therapies. In addition to supporting over 85 programs across various disease areas and drugs/medical devices, we have successfully demonstrated creative solutions in situations with minimal precedence.

Partner with us to unlock new therapeutic potential and streamline regulatory pathways through the power of RWE.

Boston Strategic Partners