Transforming research to reflect the voice of the patient

We are witnessing an increasing momentum in the transformation of traditional drug development towards patient centricity. As patients and their caregivers have first-hand experience of living with a disease, experiencing its symptoms and impacts in everyday life- they are in a unique position to inform the drug development across the lifecycle. Patients’ voice is crucial, not just in understanding the disease, or optimizing the study design and delivery, but also in interpreting and meaningfully communicating the value of health interventions.

Written by Kristina Harris

At Galapagos, we strive to engage and partner with patients across the drug development lifecycle. Our science is focused on developing medicines with new modes of action, which not just treat the symptoms, but change the course of the disease. In this endeavor, we recognize that our research also needs to be designed around patients’ unmet needs, reflecting what is truly important to them- in a scientifically sound way.

How do we incorporate patient voice in a research design? Understanding the disease experience of our patients and/or their caregivers is a first step in successfully designing any patient-centered study. This information can be obtained by consulting the existing literature and interviewing or surveying subject matter experts, patients and their caregivers. Knowing patients’ experience of their disease and which symptoms and impacts matter to them the most, helps us to embark on choosing the best outcomes to focus on in our research.

Secondly, the measures we choose need to be scientifically robust and this is where the measurement science comes in. The outcome measures need to have good evidence of their validity (to capture well what it says it measure- e.g., pain), reliability (to be free from error), and responsiveness (to be able to detect change over time). Another important property is interpretability. Interpretability helps us to map a numerical score (or a change in the score) to its clinical importance. Such as: “Is the change of 12% in the pain score meaningful to patients?” or “What is the minimum patient detectable change for fatigue score?”.

We strive to use COAs with good evidence of their measurement properties, that meaningfully measure aspects of health which are important to patients. Such measures may be already in existence (i.e., “off the shelf”) or they can be in existence but in need of further validation/ adaptation. Sometimes, a good measure for a particular purpose may not exist. In this case it would need to be developed from scratch and in close partnership with patients, clinicians, and regulators.

In our research, we also endeavor to utilize the power of digital. Technologies like Bring Your Own Device (BYOD) strategies- where patients can use their own devices (e.g., smartphones) to collect the study data and the use of wearables (e.g., smartwatch) can help streamline and enrich the data collection, whilst minimizing patient burden and even driving patient engagement. As an agile company, we are increasingly exploring and utilizing electronic data collection methods to capture clinical endpoint data in our studies.

At Galapagos, we are dedicated to improving patients‘ lives through science. We recognize that bringing patient input into the drug development process is a critical part of developing medicines that have a meaningful benefit. Furthermore, integrating patients’ voice in our research enables us to meaningfully communicate the clinically relevant treatment benefits to all our stakeholders- such as regulators, payers, prescribers, and ultimately our patients, helping them make informed decisions about their care. I am proud to be a part of this journey and excited to develop and implement patient-centric research strategies- ensuring patients’ voice is at the heart of our research.

About Kristina Harris (Patient Centered Outcomes Research Leader at Galapagos)""Kristina Harris is passionate about patient centricity. She joined Galapagos in 2020 as a Patient Centered Outcomes Research (PCOR) Leader, with a mission to drive further the transformation of our clinical research, focusing on the things important to patients. Kristina has extensive experience in patient-focused research and health outcome measurement across academia, consultancy, and pharmaceutical industry. She is motivated by innovating and creating value for patients.