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BIA and RNID webinar recording: Preparing for clinical trials in a novel therapeutic area
Watch BIA and RNID's webinar recording of preparing for clinical trials in a novel therapeutic area.
Completing a practical and meaningful clinical trial is essential to delivering on the promise of a new therapeutic. However, it can be a real challenge to do this, particularly for an emerging therapeutic area, where existing trial infrastructure may be limited.
As you may have seen remarkable progress is being made in the emerging therapeutic area of hearing loss with gene therapies showing real promise in phase 1/2 trials.
In this webinar, panel members share case studies of their experiences in novel hearing loss clinical trials, in order to help companies working in other emerging areas think about the obstacles and opportunities that exist in setting up and executing your trial, and what you can do to ensure it is as effective as possible.
Kate Blackstone
Kate joined the NIHR in 2024 after 13 years working in clinical research in Australia and the United Kingdom. Kate has held leadership roles in research ethics committees, research offices, study delivery teams, Clinical Research Facilities (CRF) and the UKCRF Network. Kate has overseen the NHS delivery of phase I to III clinical trials including novel and advanced therapies. Kate has supported MHRA phase I accreditation and the creation of quality management systems (QMS) intended to support the safe delivery of high-risk trials. Kate is now based in the NIHR’s External Affairs team linking together all the great work of the NIHR and charities. Kate's role offers charities a dedicated entry point to engage with the NIHR and similarly a resource for NIHR colleagues looking to work with charities.
Mehul Patel
Mehul Patel is a physician-scientist with board certification in Pulmonary Medicine and General Internal Medicine. He has over 20 years of clinical experience and is a passionate advocate for healthcare innovation and improvement. He complemented his medical training with an intercalated BSc in Pharmacological Sciences at the University of London, and with a PhD from Imperial College London where he investigated the systemic manifestations of chronic lung disease. He has led clinical trials and built collaborations across academia and industry that have resulted in international presentations, awards and publications. He was an investigator on global industry-sponsored clinical trials whilst working as a Consultant Respiratory Physician in the NHS before he transitioned to working in industry, initially within Discovery Medicine at GSK, before subsequently joining AstraZeneca where he is now the VP of Respiratory Clinical Development.
Anne Schilder
Anne GM Schilder is Professor of Otorhinolaryngology at University College London (UCL) Ear Institute and Director of Translational Hearing Research at the National Institute for Health Research UCL Hospitals Biomedical Research Centre. Her research focuses on translation of hearing discoveries into novel treatments for patients with hearing loss. It ranges from designing and delivering first-in-man trials, analysis of routine health data, to health economics and health policy to prepare healthcare systems for the arrival of drug, cell and gene therapies for hearing loss. Dr Schilder practices Paediatric ENT at the UCLH Royal National Ear Nose and Throat Hospital and holds an honorary chair at the University Medical Center Utrecht in the Netherlands where she works with primary care researchers to develop evidence-based interventions for otitis media in children.
Vassili Valayannopoulos
Dr Valayannopoulos, M.D., Ph.D., MBA, is a Rare Metabolic Disease expert and an experienced pharmaceutical industry executive with more than 20 years’ experience in the Rare Genetic Disease space. With training in pediatric metabolic medicine, clinical genetics and pediatric neurology and prior to joining the pharmaceutical industry, he served as Senior Faculty and Principal Investigator at the Department of Pediatric Inherited Metabolic Disorders and Pediatric Neurology at Necker-Enfants Malades University Hospital, Paris, France. Dr Valayannopoulos conducted clinical research in collaboration with IMAGINE Research Center at Necker with a special focus on neurodegenerative metabolic diseases, gene therapy and lysosomal storage disorders, and worked with expert networks for inherited Metabolic disorders (E-IMD, CETL). He has served as Principal Investigator and national coordinator in several clinical trials for lysosomal storage disease, organic acidemias, fatty acid oxidation defects and congenital glycosylation defects (academic and industry sponsored) and is an author or co-author on numerous peer-reviewed journal articles. He led for several years the annual Metabolic Training Course for clinicians and laboratory scientists sponsored by the Society for the Study of Inborn Errors of Metabolism (SSIEM). Dr Valayannopoulos served in various Clinical Development and Medical Affairs leadership roles in the past 10 years. At Sanofi Genzyme he developed a portfolio of phase 4 and real-world studies focusing on lysosomal storage disorders; at Ultragenyx he led clinical development focusing on AAV-based gene therapy for inherited metabolic disease and more recently at Decibel Therapeutics and currently at Regeneron he is leading the auditory sciences clinical programs and is also involved in Regeneron’s lysosomal storage disease programs. Dr Valayannopoulos earned his medical degree and completed his pediatric residency program at Paul Sabatier University Medical