Successful COA selection paves the path to drug development

Last year, the U.S. Food and Drug Administration approved 55 new drugs. Choosing the right clinical outcome assessments (COAs) is an important part of successfully advancing drug development and securing regulatory approval.

COAs are critical to all phases of drug development, from collecting patient data to submission for regulatory approval. Their use throughout these phases ensures that the outcomes being measured are meaningful to patients and capture the therapeutic impact of a drug.

Successful COA selection matters

Selecting the appropriate COA for use in a clinical trial is essential for evaluating the benefits and risks of a treatment and influences how treatment claims are labeled.

The information gathered from COAs isn’t critical for individual trials only; it can also be used to compare results across different trials. It’s not surprising, then, that COAs have a growing role in establishing the clinical benefit of novel therapies and in regulatory decision-making.

Choosing the right COA for a clinical trial is a complex and critical process. COAs should:

• Be relevant to the patient’s experience.
• Capture outcomes that are meaningful to patients.
• Align with patients’ health concerns and treatment goals.

An effective COA is psychometrically sound, with established reliability, validity and sensitivity to detect meaningful changes in the condition being treated. Additionally, COAs must meet standards set forth by regulatory agencies, be consistent with trial objectives, and consider patient burden.

“Selecting an inappropriate COA carries significant risks,” says J. Lynsey Psimas PhD, director of business development at Pearson Assessments US. “It can produce misleading results regarding the efficacy or safety of a treatment, potentially leading to incorrect conclusions about its clinical benefits and increasing the risk of regulatory delays or rejection.”

Psimas adds that a poorly chosen COA may also impose an undue burden on patients, which could negatively impact their participation in clinical trials and lead to higher dropout rates.

From scale selection to drug development

The selection of the right COA is fundamental to demonstrating the efficacy and safety of a treatment and ensuring that clinical outcomes reflect real patient experiences. Pearson assessments are backed by over 80 years of test development. These well-validated scales have been widely used in research and clinical trials across various therapeutic areas. They are particularly well-suited for central nervous system (CNS) conditions such as Alzheimer’s disease, schizophrenia, rare genetic diseases and neurodevelopmental disorders.

Recent examples of Pearson assessments use in successful drug development include:

Chronic schizophrenia: An international pharmaceutical company, Newron Pharmaceuticals, used the Positive and Negative Syndrome Scale (PANSS) as a primary endpoint in a clinical trial evaluating the safety, tolerability and efficacy of a novel drug for chronic schizophrenia. The PANSS is widely recognized for use in clinical trials and has long been the gold standard for schizophrenia trials.

The four-week, international, randomized, double-blind and placebo-controlled add-on Phase II/III study was conducted in 45 centers in 11 countries across Europe, Asia and Latin America. The results substantiate the role of glutamate as a new therapeutic option for treatment resistant schizophrenia (TRS) patients.

Pediatric spinal muscular atrophy: Researchers from Novartis are using the Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III), in a pivotal Phase 3 study. This open-label, single arm, multicenter clinical trial evaluates the safety, tolerability and efficacy of an intravenous drug for pediatric patients with spinal muscular atrophy (SMA).

The Bayley was used to measure key developmental motor milestones and their change from baseline at 6 and 12 months post-dose in participants with SMA.

Hunter Syndrome (MPS II): REGENXBIO announced positive long-term results from its pivotal trial of RGX-121 for MPS II (Hunter syndrome). Data showed an 85 percent median reduction in cerebrospinal fluid (CSF) levels of heparan sulfate, a key biomarker, sustained for up to two years. Additionally, 80 percent of patients discontinued enzyme replacement therapy. This Phase 2/3 multicenter, open-label study used the Bayley-III as a primary endpoint and the Vineland-II as a secondary endpoint.

The selection of a well-validated COA plays an integral role in advancing drug development and securing regulatory approval. Choosing the right COA enhances the impact of trial findings, facilitates cross-study comparisons, and streamlines the process of bringing new therapies to market.

Appropriate COAs not only minimize the need for trial redesign but also keep timelines and budgets on track, contributing to the success of clinical trials and the development of innovative treatments.

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