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 will discuss how the COVID-19 pandemic has impacted clinical studies for rare diseases and how patient-centric trial design, with regulatory consideration, can be utilized to mitigate and manage potential delays and support patient recruitment in rare and orphan clinical trials.

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 are 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 pediatric onset. Following the European definition of the incidence of a “rare disease” (no more than 5 in 10,000 individuals), it is estimated that there are over 440 million individuals globally who are affected by a rare disease at any point in time, excluding rare cancers, infectious diseases and poisonings. Rare diseases are numerous, heterogeneous in nature and geographically disparate. Few of them are preventable or curable, most are chronic, degenerative and many lead 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 on how to navigate this new challenging clinical landscape, and CROs are working to deliver solutions that meet the needs of patients and drug developers.

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 will cover:

• 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 pediatric 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 utilize experience and regulatory expertise as a clinical development partner to guide and facilitate rare disease and orphan drug trials in a pandemic

Speakers

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, pediatric and oncology clinical trials. He successfully led several clinical development programs, regulatory interactions, consultations, submissions and approvals of Orphan Drug Applications (ODA), Pediatric Investigational Plans (PIP) and Marketing Authorizations Applications (MAA) by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA).

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

Dr. Chirag Patel has over ten years regulatory affairs experience both within clinical research organizations (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 Authorization Application (MAA) via a decentralized procedure and filed US IND Applications.

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