Drug development is moving beyond traditional endpoints as patient-centricity becomes the new standard. Together, patient-centric evidence like patient-reported outcomes (PROs) and real-world evidence (RWE) give researchers visibility into patients’ unique needs, preferences, and experiences so they can better understand the tangible impact of various drugs and therapies.

As patient-centric evidence continues to shape the future of drug development, challenges around increased complexity and regulatory scrutiny must be addressed. Statistical computing environments (SCEs) are vital for overcoming these hurdles by centralizing and governing access to diverse datasets, enabling researchers to conduct reproducible and auditable analyses.

PROs + RWE = A deeper understanding of the patient experience

When analyzed in conjunction, PROs and RWE provide a holistic view of the patient experience—all the way from the controlled environment of a trial to the realities of everyday patient life.

PROs offer a window into how treatments actually impact a patient’s quality of life. This is particularly useful in areas like cancer research, since oncology drugs can be especially hard on the body. PROs help determine whether certain drugs are generally more tolerable than others so regulators and payers can give them preference over their “harsher” counterparts—as long as their traditional endpoints (i.e., survival and progression) are comparable.

RWE provides crucial insights about efficacy by giving researchers access to more longitudinal data about the patient experience, including after trials end. For example, RWE can reveal how oncology drugs perform outside of tightly controlled trial settings to determine whether patients experience the same survival benefits in everyday use, or if efficacy drops due to real-world factors like adherence, comorbidities, etc.

These data sources are essential for supporting better patient outcomes, but more data means more challenges.

The challenges of working with patient-centric evidence

Despite the benefits they unlock, PROs and RWE introduce a higher level of complexity and regulatory scrutiny into the drug development process.

PROs can carry cultural biases. For example, in some cultures people are encouraged not to complain about pain, which could lead to inaccurate data. Furthermore, PROs have traditionally had low compliance rates; life gets busy and people forget to or stop reporting their outcomes.

RWE can be biased too since it isn’t random and therefore may not be based on representative data. For instance, the data from electronic health records (EHRs) used in RWE studies disproportionately reflects patients who have consistent access to healthcare, which skews results away from underserved populations.

Data heterogeneity and reproducibility quickly become a challenge when diverse, patient-centric datasets—from wearables to EHRs to survey instruments—are combined without consistent standards or governance.

SCEs: The bridge between patient-centric evidence and improved patient outcomes

SCEs serve as the linchpin that enables PROs and RWE to pave the way to better patient outcomes. By providing a controlled environment where researchers can access and analyze data from multiple sources, SCEs support transparency, lineage, and reproducibility while also improving collaboration.

For example, regulators and pharmaceutical companies might work together in a SCE to ensure PRO instruments/surveys are closely aligned with the therapeutic objectives of a given trial. Additionally, biostatisticians and study teams can use the technology to run mock analyses to assist with protocol simulation, determine required sample sizes and control methods, and perform endpoint feasibility around RWE and PRO data.

SCEs can also help pharmaceutical teams determine how RWE and PROs will fit into their statistical analysis plan (SAP) earlier in the process so that biostatisticians and medical teams can align on how a drug’s efficacy and safety will be evaluated. And importantly, regulators can see exactly what data contributed to a given analysis and how the analysis was completed through version-controlled code, immutable datasets, and documented data lineage.

As PROs and RWE continue to influence drug development, the number of data sources researchers have to analyze will expand. One thing that will remain constant, however, is the need for a well-governed environment for analysis—and SCEs will always serve as the trusted foundation. Only SCEs can provide the control, auditability, and reproducibility pharmaceutical companies need to deliver therapies that reflect real patient impact, turning today's data into tomorrow’s medical breakthroughs.