What are clinical outcomes assessments?

Patient-centered outcomes assessment starts with an understanding of the impact of a disease and its treatment on patients, and what patients value from a treatment perspective. It measures things that are important to patients, such as how a treatment will affect symptoms and their day-to-day functioning.

Such patient-focused approaches use clinical outcomes assessments (COAs), in which information is gathered on the impact of treatment on the person and can markedly enhance the understanding of the patient experience in cancer clinical trials.

COAs can broadly be classified into four distinct groups based on how data is collected:

• PROs – patient-reported outcomes
• ClinROs - information gleaned from clinical observation
• ObsROS - other observer reported outcomes (ObsROs),
• PerfOs - and data from performance on a task or test (PerfOs)

In cancer-related clinical trials, regulatory agencies and advocacy groups increasingly encourage using PROs to describe the clinical benefit of a therapeutic regimen.

This includes using information on disease-associated symptoms or functions to help determine treatment efficacy and understand treatment-associated side effects.  These measures, which collect information directly from the patient, without interpretation by somebody else, improve researchers’ understanding of the patient perspective on the treatment’s impact.

Why are patient-centered outcomes important?

Regulators, clinicians and patients need meaningful information about a treatment’s benefits and risks in order to make important treatment decisions.

Additionally, these measures also provide valuable insights about the impact of the disease and its treatment that help improve patient care even when the treatment does not turn out to have sufficient activity against the cancer.

Why are patient-centered outcomes important in early-phase trials?

There has been a shift in the clinical trial landscape towards early-phase studies, where data from well-designed and pivotal phase I/II trials increasingly leads to accelerated drug approval. 

Early-phase clinical trials primarily investigate toxicity and tolerability. Standard toxicity assessments provide only limited or inadequate information.  Patient-focused approaches using COAs to gather information about the impact of treatment on the person, not only the tumor, would markedly enhance the understanding of the patient experience in these trials.

Standardizing and incorporating PROs into earlier phase trials would allow them to be optimized, improve study design and allow more rigorous and systematic assessment of PROs in later-phase trials.

These more systematic PRO designs may then increase the chance of a PRO being incorporated into a product’s label. That could ultimately empower patients, caregivers, and healthcare providers to make more informed treatment decisions leading to better outcomes and better quality of life for patients.