Across the pharma product lifecycle, factors like the regulatory environment and business pressures on R&D and market access teams drive a strong imperative to further incorporate real-world evidence (RWE) into business processes. In parallel, to support biopharma’s increased use of RWE, we’ve also seen an emergence of new analytic methodologies to help mitigate analytic quality and validity concerns, particularly given the move to personalized medicine and research on sub-populations.
Given this context, RWE has critical applications across drug development. For instance, in early development, RWE supports understanding the standard of care, unmet need, burden of disease, and disease natural history. RWE can be leveraged further to optimize clinical trial design, for instance by identifying optimal subgroups for Phase III trials. Within the realm of HEOR and market access, RWE can further inform budget impact and cost-effectiveness models for relevant populations. Finally, the postmarketing applications of RWE are numerous, and span safety (e.g., pharmacovigilance), commercial (e.g., head-to-head effectiveness and safety studies, monitoring safe and effective off-label use), and beyond.
As large and mid-sized pharma manufacturers invest in RWE-related assets and capabilities, they organize their resources in varied ways. One analysis as of March 2020 found that six of the top 10 pharma companies had some form of “Center of Excellence” (CoE) focused on RWE.* These CoEs vary in remit and degree of utilization. Other companies choose a federated model, allowing different functional groups to choose the best RWE assets (i.e., datasets, software tools) and capabilities to suit their needs. Read on for an explanation of these two illustrative models, including the advantages and challenges, and success enablers for both.
Centralized RWE organizational structure
- Description: Across large and mid-sized pharma, CoEs’ scope can include: centralized procurement of data assets and analytical software, centralized people resources (e.g., epidemiologists, health economists, data scientists) to run analytics in support of varied functional groups, including Safety & Regulatory, Clinical Development, Medical Affairs, Commercial, and Market Access. CoEs can report into and hold budget from different parts of the organizations, for example the Chief Digital Officer or the Head of R&D.
- Advantages: This structure sets an opportunity to set evidence generation priorities centrally. These priorities can be continuous across the product lifecycle and portfolio and supported by centralized budget and investment. Such prioritization requires more explicit organizational buy-in and executive leadership for RWE-related topics, and can lead to increased coordination and alignment from stakeholders in their evaluation and procurement of tools and resources.
- Watch-outs: Particularly in large organizations, organizational silos can challenge the execution of coherent, continuous use of RWE across the enterprise. In many instances, the CoE and its remit may not be widely known in the organization, challenging the ability to set RWE strategy and drive the agenda. Based on its remit, a CoE may be compelled to optimize for which data assets or tools work best across multiple functions, at the expense of what is preferred for an individual function. In the absence of a CoE, functions have more flexibility to optimize real-world data (RWD) assets or tools for their purposes—for instance, use of open claims data may be helpful for Commercial or Business Analytics questions on market uptake of a drug, but would not be appropriate for a regulatory-grade safety analysis.
Success enablers include:
- Empowered leadership and sufficient budget to have clout in the organization;
- Top-down (i.e., by CEO, executive leadership) recognition and messaging about CoE’s role and remit;
- Governance structure that allows for CoE to be close enough to R&D, brand teams’ priorities, to be able to draft and execute on a relevant RWE strategy; and
- Sufficient flexibility to accommodate different functional and portfolio needs, while centrally coordinating common requirements as possible.
Federated RWE organizational structure
- Description: In a federated arrangement there is no CoE, and functional teams (i.e., Safety & Regulatory, Clinical Development, Medical Affairs, Commercial and Market Access) set strategy for and resource their evidence generation separately from other functions. Resourcing includes selecting and purchasing RWD assets and capabilities. There may be an effective RWE owner by function, for example, those who focus on observational research within a Safety or Epidemiology group, or a Center for Trial Design within R&D.
- Advantages: There is an opportunity to fully optimize for the RWE needs specific to a functional group, and to more nimbly pursue acquisition of those data assets, software, or individual skill sets.
- Watch-outs: Lack of central investment in RWE challenges an organization’s ability to set a cross-lifecycle, cross-portfolio evidence generation strategy, and execute over time. A lack of coordination can result in significant under-realization of RWE potential cross-functionally. Furthermore, siloed analytic planning and purchasing can result in duplication of effort and reduce benefits from economies of scale
Success enablers include:
- Sufficient cross-functional forums, or committees for cross-functional teams to align on strategy, evidence generation priorities, and resourcing plans;
- Frequent formal and informal touchpoints among “RWE owners” in various functional groups; and
- Effective mechanism for knowledge sharing and pooling of enterprise-wide resources (e.g., data assets, etc.) for visibility across functions.
Of course, hybrid versions of the archetypes above are also pervasive. While any set of organizational design choices comes with tradeoffs, multiple approaches can be effective depending on the size, culture and context of the organization. Regardless of design choices, thoughtful governance and strong execution are critical to ensuring that organizations optimize their use of RWE enterprise-wide.
* Primary interviews and analysis; companies in scope all $15B and above in annual revenue.