Decentralized Trials Can Be Commonplace for Rare Disease Patients

Decentralized clinical trials have proven to allow continued recruitment and retention of patients, keeping trials and patient’s wellbeing on track, but what about keeping daily life on track for a patient and their family?

For rare disease patients, a clinical trial may be the important next step in their healthcare journey, an important key that unlocks the next door of treatment, so it’s imperative for those trial schedules to be flexible and meet the life needs of the patient. We know that a patient is more than their health condition — they’re parents, children, they go to school and work, they have a life outside of clinical research, and trial designs don’t always consider that.

MRN has spent close to two decades advocating for a decentralized, patient-focused approach to clinical trials, bringing trial visits to the patient in their own community. While the industry in recent years has discussed various decentralized, patient-centric strategies, COVID-19 brought their implementation to the very forefront of clinical trial development. As an industry, we saw sites close and studies lose their trial patients. Because of this, eyes have been opened to all of the possibilities decentralized solutions can bring to a trial, but with implementation comes a learning curve.

During this webinar, featured speakers will give you insights into their experience running decentralized trials so you can be ahead of the curve. They will discuss when decentralized trial visits will work, how to make them work, the impact on rare disease patients and families and how you can support them through decentralizing trials with a look into the journey of a family navigating life with a rare disease.

Speakers

Caroline Potts, MSc Health Sciences, MSc Healthcare and Design, Senior Executive Director, Visit Management, Medical Research Network

Caroline has been instrumental in the delivery of decentralized clinical trials for over three years. In her current role as Senior Executive Director for Visit Management, she manages MRN’s global Project Management teams.

Before joining MRN, Caroline worked in the public sector for 15 years supporting research in hospitals and the primary care setting. She also provided support to research academics by writing funding applications, making submissions to regulatory authorities and running a large portfolio of studies to ICH-GCP requirements in clinical specialties such as stroke medicine, Parkinson’s disease, orthopaedics, gastrointestinal medicine and respiratory medicine. During this time, Caroline was one of the founding members of the UKRD group as well as an Executive member of the RD Forum providing R&D Directors and research staff working in the public sector with strategic guidance in supporting high-quality research in the NHS.

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Tracey Murray, RGN, DipEd Nursing, BA(Hons) Health Studies, Director, Rare Disease Nurse Network

Tracey is a registered nurse with over 22 years’ experience of leading change and working within a variety of healthcare settings and roles. After specialising in Gerontology, she introduced a new workforce to Addenbrookes Hospital: Physician’s Assistants. She then joined the Norfolk, Suffolk and Cambridgeshire Strategic Health Authority as part of a team looking at Modernising the Medical Workforce.

Currently she is Matron of Outpatients at Cambridge University Hospitals NHS Foundation Trust, an active advocate for Rare Diseases — especially Sotos Syndrome, which her daughter has — and a director of The Rare Disease Nurse Network. Tracey describes the journey of living with a child with a rare disease as that of a roller coaster, travelling through a mixture of emotions, experiencing highs and lows, never truly knowing what to expect but cherishing every moment.

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