Enhancing clinical trials: The vital role of Clinical Outcome Assessments

17th July 2024

Discover why Clinical Outcome Assessments (COAs) are vital in clinical trials for measuring patient outcomes and ensuring reliable data for regulatory approval and improved patient care.

Clinical Outcome Assessments (COAs) are essential tools in clinical trials, offering a standardised method to measure patient outcomes directly from the patients themselves or through observation. These assessments include patient-reported outcomes (PROs), clinician-reported outcomes (ClinROs), observer-reported outcomes (ObsROs), and performance outcomes (PerfOs). By capturing meaningful data on how patients feel and function, COAs provide critical insights into the efficacy and safety of new treatments.

The FDA recognises the value of COAs in the regulatory approval process. According to their guidance on COAs, these instruments are fundamental in demonstrating a treatment’s benefit from the patient’s perspective. The FDA emphasise the need for COAs to be reliable, valid, and responsive to changes in a patient’s health status. This alignment with regulatory standards ensures that the data collected is credible and can support labelling claims, ultimately facilitating the approval process for new therapies.

Our tools have been integral in numerous clinical trials, providing reliable and validated measures to capture patient-centred outcomes across diverse therapeutic areas. Over the years, our instruments have consistently demonstrated their value in enhancing the quality and robustness of clinical data, supporting the development of new therapies and improving patient care globally.

It’s important to note that not all clinical trials include or use COAs. While COAs are invaluable for understanding the patient perspective, some trials may rely solely on clinical or observational means or refer to biomarker data for example. Utilising study data obtained through GlobalData and Clinical Trials Gov, we are able to depict use/planned use of some of our instruments in clinical trials since 2015.

• The Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ), which is specifically used to measure the subjective well-being of patients with amyotrophic lateral sclerosis (ALS, commonly referred to as Lou Gehrig’s Disease) and the occasional condition of motor neurone disease (MND), has been listed on 14% of all clinical trials for ALS, either as part of a primary objective or a secondary objective. One such use, was in Mitsubishi Tanabe Pharma Corporation’s study for the efficacy and safety of orally receiving Edaravone (Radicava), which is a U.S. FDA-Regulated Drug Product.
• The Endometriosis Health Profile (EHP), used to measure the wide range of effects that endometriosis can have on women’s lives, has been used in 11% of all listed clinical trials specific to Endometriosis. In 2022, Myovant Sciences and Pfizer were granted U.S FDA approval of MYFEMBREE® following the success of two clinical trials (SPIRIT 1, SPIRIT2); both of which utilised the EHP as secondary outcome measures to measure pain.
• The Parkinson’s Disease Questionnaire (PDQ), which is designed to address aspects of functioning and well-being for those affected by Parkinson’s disease, has been used in 18% of all listed trials since 2015, again either to assess a primary objective or a secondary objective. The PDQ was for example deployed during the INTREPID randomized controlled trial, during which patients were implanted bilaterally with Boston Scientific’s FDA-approved Vercise Genus Deep Brain Stimulation system.

At Oxford University Innovation (OUI), our Clinical Outcomes team is dedicated to providing high-quality clinical outcome assessment (COA) instruments and comprehensive support for their use in clinical trials and clinical practice. We understand that speed and careful planning are critical for implementation in clinical trials, and our streamlined processes reflects that.

Efficient Licensing

Our quick and efficient licensing process minimises delays, allowing your research to proceed smoothly. We prioritise rapid responses, provide clear guidelines, and offer dedicated support to ensure a hassle-free experience.

Translation Services

Use of accurately translated COA instruments is essential for trials being conducted both on a global scale and those which are capturing the voices of patients who speak multiple languages. We ensure translations are conducted by qualified professionals, maintaining the integrity and cultural relevance of the instruments and conforming to translation and linguistic validation methodology recommended by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and approved by regulatory agencies such as the Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

eCOA Services

We work closely with licensees and eCOA vendors to ensure faithful digital migration of our COAs. By engaging early and providing detailed guidance, we help preserve the measurement properties of our instruments in electronic formats, again following industry best practices.

The Clinical Outcomes team are dedicated to supporting those wishing to use our COA instruments in all forms of research and clinical use. If you have any questions related to our COA Library or would like to find out more about how you can obtain a licence to use them within your study, you can contact the Clinical Outcomes team via healthoutcomes@innovation.ox.ac.uk.

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