Real-world data (RWD) is patient-level data not collected in conventional randomized controlled trials. Examples include electronic medical records, claims data, mortality data, consumer data, registries, data collected in observational studies, chart review, and pragmatic clinical trials. Real World Evidence (RWE) is the evidence derived from the analysis and/or synthesis of RWD. RWE has long been associated with post-marketing research activities, to understand a treatment’s safety, effectiveness and use in the real-world. Increasingly, we are seeing the value of RWE in medicines development using population-based databases to characterise frequency and distribution of disease, identifying the population to be treated, understanding whether a disease affects high risk populations, identifying unmet medical needs, determining disease prevalence, characterizing standard of care, and understanding healthcare system budget impact.

Gathering RWE from the outset of research can help biopharma companies make better early decisions about product viability, and quantify the value of a potential product to investors and other key stakeholders -- long before it comes to market. Having this data can be particularly useful for emerging biopharma companies that focus on niche and specialty areas where there may be little historic data on disease impact, demand for treatments, product pricing and risk scenarios. By gathering RWE from the outset of their development process, they can begin to fill in some of these gaps, delineating precise patient populations for a specific treatment, quantifying the unmet medical need to define potential market demand, and describing the benefit proposition the product will bring to patients as a way to support reimbursement decisions. All of this information can help developers make more informed decisions throughout the development lifecycle, while providing stakeholders with a holistic view of the benefits and risks of the product.

Whether that stakeholder is an internal leader attempting to gain an unbiased view of their portfolio’s value, or an external investor determining whether to acquire or invest in a firm, this data can help level-set expectations, and prevent either group from over- or under-investing in an asset.

Asking for it

Investors and external stakeholders increasingly expect emerging biopharma companies to provide this data as part of the assessment process, and industry groups are actively exploring how to better incorporate real-world evidence earlier and throughout the drug development process. One notable example is the GetReal project, a three year initiative launched in 2013, by the Innovative Medicines Initiative (IMI), an EU public-private consortium of pharmaceutical companies, academia, advocate groups, HTA agencies and regulators. The goal of the project is to show how new methods of real-world evidence collection and synthesis could be adopted earlier in pharmaceutical R&D and the healthcare decision-making process to benefit all relevant stakeholders. Bodies such as the European Network of Centres for Pharmacepidemiology and Pharmacovigilance (ENCePP), a network coordinated by the European Medicines Agency (EMA), understand that real-world data plays a critical role during the life cycle of a medicinal product and that planning data collection and integrating knowledge starts in early development and is life-long. ENCePP is looking at how it can support collaborations for improved access to and use of RWD. In addition, the demand for early access to treatment has prompted initiatives such as the EMA’s Medicines Adaptive Pathways to Patients (MAPPs) and the Massachusetts Institute of Technology (MIT) Center for Biomedical Innovation’s New Drug Development Paragdims (NEWDIGs). A key component of these projects is a life cycle approach to evidence development that includes RWE. 

Yet the drug development paradigm is not up to speed with stakeholder demand for this data. RWE is still often viewed as something that only adds value after approval, as a way to validate impact and support pricing. But waiting until the end of the development process to gather real-world evidence is a wasted opportunity for emerging biopharma companies that can directly impact the viability and financial success of their product and company.

My colleagues, Laura Marquis, Dean Summerfield, Jeremy Broadis and I, will be addressing this issue at the BioEurope conference, November 7-9 in Cologne, Germany. In our panel session - Incorporating real-world evidence and data throughout the clinical development plan to show value to investors of emerging biopharma companies (EBPs) - we will define real-world data and real-world evidence and discuss what healthcare stakeholders are asking for and why. We will look at how emerging biopharma companies can incorporate real-world evidence throughout the clinical development plan, and why taking such steps will help them build their economic business case and provide stakeholders and investors with a more definitive picture of the product’s value.

We hope this session, and initiatives like GetReal will help the industry gain greater insight into the value of gathering real-world data sooner, and help companies figure out how to use this vital research to add clinical and economic value to their drug development lifecycle.

Topics in this blog post: Biopharma, Clinical Trials, Evidence, Recruiting, Value