Meeting the Needs of Rare Disease Patients in Clinical Trials During the COVID-19 Pandemic

Meeting the Needs of Rare Disease Patients in Clinical Trials During the COVID-19 Pandemic

A Discussion on Regulatory and Operational Considerations

This 1-hour webinar discusses how the COVID-19 pandemic has impacted clinical studies for rare diseases and how patient-centric trial design with regulatory consideration can be utilised to mitigate and manage potential delays and support patient recruitment in orphan and rare disease clinical trials.

About the webinar

COVID-19 is a serious, life-threatening and fast spreading viral infection that has had an unexpected global impact. As the world is learning to adapt to a ‘new normal’, so have clinical trials.

“Rare Diseases” is an emerging global public health priority. There are more than 7,000 identified and classified rare diseases, 71.9% of which are genetic and 69.9% which are exclusively paediatric onset. Upon the European definition of “rare disease” (5 per 10 000), it is estimated that there are globally >440 million individuals affected by a rare disease at any point in time, excluding rare cancers, infectious diseases, and poisonings. Rare diseases (RDs) are numerous, heterogeneous in nature, and geographically disparate. Few of them are preventable or curable, most are chronic, degenerative, and many leads to early death. The inherent challenges are derived from their low prevalence, a poor scientific knowledge, and the scarcity of expertise as well as their chronic, degenerative, and life-threatening nature. While healthcare services currently prioritize COVID-19 treatment, clinical trials for orphan drugs continue working in an unprecedented environment which has challenged traditional study design, clinical operations and driven innovation in patient-centric trials. The global COVID-19 pandemic disrupted everyone’s lives, exposing rare disease patients to yet another health threat and forcing them to potentially make unwilling sacrifices.

Patient groups, physicians and regulators are eager to support the delivery of rare and orphan clinical trials, allowing flexibility while maintaining the standards necessary to deliver high-quality data. “Orphan drugs” companies are looking for support and guidance in how to navigate this new challenging clinical landscape, and CROs are working to deliver solutions that meet the needs of patients and drug developers to achieve orphan drug designation.

Join Dr. Carlos Camozzi, Chair of the Simbec-Orion “Rare and Orphan Advisory Board”, and Dr. Chirag Patel, Director of Regulatory Affairs for a discussion around how the challenge of rare disease studies during COVID-19 could offer an unexpected opportunity for clinical trial innovation, followed by a Q&A session.

The webinar covers:

  • How the COVID-19 pandemic has driven clinical trials to adapt to a “new normal” and accelerated innovation in clinical trial design
  • How to adapt to the limitations and additional challenges for clinical trial design during the COVID-19 pandemic
  • Meeting the needs of vulnerable and paediatric patient populations at risk while ensuring high-quality clinical data
  • The importance of relationships with physicians, patient advocacy and support groups
  • How regulatory considerations within trial design can reduce the risk of delay, especially for international studies where local regulations will need to be considered
  • How a CRO can utilise experience and regulatory expertise as a clinical development partner to guide and facilitate rare disease and orphan drug trials in a pandemic


Dr. Carlos Camozzi, Chair, Simbec-Orion Rare and Orphan Advisory Board

Dr. Carlos Camozzi is an experienced Chief Executive and Corporate Governance in the Biopharmaceuticals and Health Technology with over 30 years C-level expertise/experience of the orphan drugs, gene-therapy, biopharma and medical devices industry.

Carlos has generated innovative solutions to the evaluation, design and the execution of non-clinical, translational, and clinical development projects for several orphan drugs, paediatric, and oncology clinical trials. He successfully led several clinical development programmes, regulatory interactions, consultations, submissions and approvals of Orphan Drug Applications (ODA), Paediatric Investigational Plans (PIP) and Marketing Authorisations Applications (MAA) by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA).


Dr Chirag Patel, Director of Regulatory Affairs, Simbec-Orion

Dr. Chirag Patel has over ten years’ regulatory affairs experience both within clinical research organisations (CROs) and the pharmaceutical industry. He has in-depth experience in managing submissions for multi-country studies across Phases I, II & III and provides expert knowledge of local national requirements for the EU and Latin America. He has managed the preparation of a Marketing Authorisation Application (MAA) via a decentralised procedure and filed US IND Applications.