Co-creation with patients is vital for effective clinical trial design. A study by Skovlund, PC, Nielsen, BK, Thaysen, HV et al. shows that, in clinical trials, “patient involvement can lead to improvements in design, selection of relevant outcome measures and effective recruitment of trial participants”.
For a medicine to be authorised, clinical efficacy and safety need to be established, usually through robust and relevant clinical trial data. There are several unique challenges in the development of orphan disease treatments. Low patient numbers, an incomplete understanding of the disease pathology, phenotypic heterogeneity and a lack of established endpoints are barriers to efficient and effective clinical trials.
A key aspect is the choice and selection of the primary and key secondary efficacy endpoints: a reliable, valid, sensitive, interpretable and meaningful endpoint is vital to a well-designed study. This is necessary both as the basis to demonstrate the clinical impact of any therapeutic intervention and to stand up to regulatory scrutiny.
In this webinar, attendees will learn how Aparito’s Patient Group Accelerator Programme enables patients to co-create alongside sponsors & CROs. The Accelerator is designed to understand and fulfil patients’ needs by working closely with patient organisations and finding new endpoints that are relevant to their specific conditions.
The featured speakers speak to a young patient to gain their perspective on being involved with clinical trial design and they demonstrate how this feedback can be taken into study design as they close out the session with a real-life example from Aparito’s Accelerator programme.
Register to learn the value of patient co-creation in clinical trial design and how patient involvement can improve trial design and clinical outcome measures.
Begonya Nafría, Patients Engagement in Research Coordinator, Sant Joan de Déu Children's Hospital Barcelona
Begonya Nafría is the Patients Engagement in Research Coordinator at Sant Joan de Déu Children’s Hospital (Spain). She has extensive experience in patient and family involvement research initiatives. She also has a personal story as a caregiver and patient advocate as the sister of a young adult with cerebral palsy.
Her areas of expertise are in paediatric patient involvement research, more specifically in the field of clinical trials and in health innovation. She is currently a PhD student in patient involvement in the paediatrics field, and pursuing a thesis project about the rights of children and young people participating in clinical trials.
- Fellow of EUPATI (first cohort) and member of its Steering Committee
- Coordinator and Founder Member of eYPAGnet (European Young Patients Advisory Group Network – www.eypagnet.eu)
- Coordinator of Kids Barcelona (YPAG of Sant Joan de Déu Children’s Hospital – www.kidsbarcelona.org)
- Co-chair of Children’s Medicines Working Party of EFPGCP
- Volunteer member of the Editorial Board of Center for Information & Study on Clinical Research Participation (CISCRP)
- Member of the Patients and Families working group
- Chair of the Cross-Border Access to Paediatric Clinical Trials of EnprEMA (European Network of Paediatric Research of EMA)
- Coordinator of the cross-cutting theme of patient’s involvement in Conect4Children project (pan-European paediatric clinical trials network)
Begonya’s experience and activities aim to achieve a better quality of life for the paediatric patients with the help of research and new therapies and ensuring their active involvement towards patient-centric clinical research and innovation.
Nora Navarro, Patient, Kids Barcelona
Nora Navarro is 19 years old and is pursuing a degree in criminology. A passionate advocate of science, Nora was a patient of Sant Joan de Déu Children’s Hospital since she was nine months old and since 2018 has been an active member of the Kids Barcelona Young Person’s Advisory Group.
Lucy Dixon, Chair, PCD Support UK
Lucy Dixon is the chair of PCD Support UK and is studying for her master’s in social research at the University of York.
Lucy has focused on addressing inequality in both education and health throughout her professional career with specialisms working with disadvantaged or marginalised groups, rare disease communities and voluntary sector organisations.
Lucy also mentors disadvantaged young people seeking to attend selective universities, including Oxford and Cambridge, as well as those applying to medicine (undergraduate).
Elisa Ferrer Mallol, Patient Advocacy Manager, Aparito
Dr. Elisa Ferrer Mallol is the Patient Advocacy Manager at Aparito.
Dr. Elisa Ferrer Mallol holds a pharmacy degree and a PhD in physiology. She has broad experience in different areas of the healthcare industry, such as drug safety, medical publishing and research project management. In the last few years, she has been working in the rare disease patient advocacy space, first at EURORDIS, the European umbrella of rare disease patient organisations and now at Aparito managing the Patient Group Accelerator, a programme to help co-design digital endpoints in partnership with patient groups.
Who Should Attend?
This webinar will benefit those working in Clinical Operations and Med Affairs in the following sectors:
- Emerging Biopharma
- CROs, especially those specialized in rare diseases
- Patient Advocacy
What You Will Learn
- How Aparito designed a study to evaluate engagement with physiotherapy using mobile health technology
- How to involve patients early in the design phase of clinical trials
- How attendees can apply learnings to their own studies
Patients living with life-limiting diseases need access to clinical trials and innovative treatments.
Aparito digitizes clinical trials to provide that access, wherever patients are.
We unlock real-world data through mobile apps, video assessments & wearable devices via Atom5(tm), our iOS & Android-compatible web and mobile app platform.
Available in 193 countries and 125 languages, the Atom5(tm) platform integrates clinical & regulatory expertise to capture patient data and develop digital endpoints for hybrid and decentralized clinical trials to streamline the drug development process.