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Outcome Measures

Driving forward patient-centered clinical trials

Pharmaceutical companies face increasing pressure to demonstrate not only the efficacy of their treatments, but also the impact of these treatments on patients' lives. This interest in and evolving regulatory requirement to put patient’s at the center of the drug development process means pharmaceutical sponsors must design clinical trials with patient-centered outcomes in mind. Patients are, as the FDA states, “experts in what it is like to live with their condition [and are] uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation.”1

To address the industry’s need to conduct more patient-centered clinical trials, ICON today announced the launch of its new Outcome Measures service line, a suite of end-to-end services to support sponsors in the identification, selection and implementation of measures that matter to patients. 

What are Outcome Measures?

Outcome Measures refers to a multi-faceted approach to identifying, selecting, and implementing evidence-based measures that matter to patients. Simply put, they are the various measures researchers can focus on to assess a treatment’s efficacy. While endpoints define the goals, such as specific events or outcomes that are used to determine the success of a trial, outcome measures provide the data to determine if those goals were met. 

ICON’s Outcome Measures service aims to drive forward patient-centered treatments while also helping sponsors increase the likelihood of regulatory approval. By focusing on outcomes that matter to patients and addressing real-world concerns, patient-centered research can produce more compelling evidence for regulators to consider when evaluating a new treatment. This approach not only increases the chances of approval but also helps ensure that approved treatments will provide meaningful benefits to patients in practice.

An industry evolution

The advent and ubiquity of Digital Health Technologies (DHTs) which remotely, passively, and continuously collect participant data, is a significant advancement for clinical trials and has been instrumental in the rise of patient-centered clinical trials. Wearable devices, mobile apps, and telemedicine platforms have significantly increased the amount of patient-generated data available to researchers. Analysing data from ClinicalTrials.gov, a study in npj Digital Medicine found a substantial increase (~34% compound annual growth rate) in the use of DHTs in clinical trials, highlighting the industry's rapid shift towards integrating these technologies into the clinical trial process.2 This growth highlights the imperative for pharmaceutical companies to leverage DHTs to collect outcome measures that reflect patients' daily experiences, thereby enhancing the relevance and accuracy of trial data. In fact, more than 130 pharma and biotech organisations have already relied on digital biomarkers and sensor-derived clinical outcome assessments (COAs) to capture patient-centered endpoints.3

As the use of DHTs in clinical trials has grown, so too has the use of Clinical Outcome Assessments (COAs) to inform patient-centered endpoint strategies. Specifically, Patient-Reported Outcomes (PROs), which are “assessments completed directly by patients, reflecting their perception of their health, quality of life, and the impact of disease and treatment.”4 PROs are particularly valuable for understanding the patient’s perspective on symptoms, side effects, and overall well-being while on treatment, providing insight into how a therapy affects patients' lives, beyond what can be measured through clinical tests and observations. By incorporating PROs into clinical trials, pharmaceutical companies can gain a deeper understanding of the benefits and potential side effects of a treatment from the patients' point of view, which is essential for developing therapies that truly meet patients' needs.

A growing imperative

With increasing emphasis on the patient voice in clinical research, there is growing demand from regulators and patients alike for pharmaceutical companies to show that new therapies meet clinical endpoints and also deliver improvements in patients' quality of life and lived experience. This industry shift presents a clear need for comprehensive, reliable outcome measures that can capture the full impact of treatments on patients. In fact, the FDA has released a four-part guidance series providing step by step recommendations on how to collect and submit patient experience data for regulatory decision making. The guidance includes advice on how to collect comprehensive and representative input; how to identify what is important to patients; how to select, develop or modify fit-for-purpose clinical outcome assessments; and finally, how to incorporate COAs into endpoints for regulatory decision making.5

As the first clinical research organisation to offer an end-to-end Outcome Measures service, ICON addresses this market demand by offering pharmaceutical sponsors a streamlined, end-to-end approach to meet the evolving expectations of patients and of regulators, who are increasingly prioritising patient-focused data in their evaluations of new therapies. And as differentiating treatments has become more difficult, sponsors are striving to differentiate their products based not only on efficacy but also on the value they bring to patients.6 In this environment, demonstrating meaningful patient outcomes is both a key differentiator as well as a regulatory imperative. 

From end to endpoints

Indeed, the growing adoption of DHTs and COAs underscores the need for a service that can seamlessly integrate these tools into clinical trials, ensuring that the data collected is both relevant and actionable. By launching this end-to-end Outcome Measures service, ICON positions itself as a leader in patient-centered research, providing sponsors with the expertise and resources needed to meet the rising market demand for treatments that truly resonate with patients.

At ICON, our belief is that the use of outcome measures that prioritise the patient experience will be key to driving innovation and achieving successful regulatory outcomes. ICON's new Outcome Measures service is uniquely positioned to support sponsors in this endeavor by providing comprehensive, end-to-end solutions that align with both regulatory expectations and the evolving needs of patients. By adopting this patient-centered approach, sponsors can not only enhance the likelihood of regulatory approval but also ensure that the treatments they develop truly meet the needs of those they are designed to help. As regulatory bodies such as the FDA and EMA increasingly emphasise the importance of patient-centered outcomes, partnering with ICON to avail of Outcome Measures capabilities can demonstrate meaningful treatment benefits to regulators and patients alike. 

We look forward to partnering with you to make a meaningful difference in patients' lives. 

1 The US Food and Drug Administration: CDER Patient-Focused Drug Development

2 Marra, C., Chen, J.L., Coravos, A. et al. Quantifying the use of connected digital products in clinical research. npj Digit. Med. 3, 50 (2020). https://doi.org/10.1038/s41746-020-0259-x

3 ICON Blog: Digital endpoints widely adopted in pharmaceutical and biotech-sponsored clinical trials

4 Mapi Research Trust: COAs 101: Going back to basics

5 The US Food and Drug Administration: FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making

6 ZS: The growing challenge of product differentiation
 

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