Orphan Drug Designation: Meeting the Unmet Need in Rare Diseases

Chirag Patel, Director of Regulatory Affairs & Technical Writing, Simbec-Orion

Chirag has ten years of 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.

In his academic career Chirag, a medicinal chemist, has conducted research into the synthesis and biochemical evaluation of inhibitors of estrone sulfatase as potential anti-tumour agents and completed his PhD at Kingston University.

Art Gertel, President and Principal Consultant, MedSciCom, LLC

Art has over 35 years of experience in increasingly senior management-level positions in the pharmaceutical industry. He has particular expertise in the preparation of large, complex corporate and regulatory (NDA/BLA/MAA) documents and thorough familiarity with relevant U.S., Canadian, EU, and ICH Guidelines. He is experienced in the initiation of departmental policies, procedures, and organization. Art’s responsibilities encompass clinical, preclinical, promotional, and medical educational aspects of all phases of product research, development, and registration.

Sadiq Lutfi, Regulatory Affairs Manager, Simbec-Orion Group

Currently, Sadiq is working as a Regulatory Affairs Manager. In this capacity, he coordinates the regulatory aspects of clinical studies throughout the entire lifecycle of a clinical trial by providing regulatory consultancy to study teams and establishing regulatory application, response and amendment strategies.

Sadiq is a leader established in multiple clinical trial settings including investigator sites, non-commercial sponsor management office and clinical research organisations, in addition to leading the pharmacy department through MHRA GCP inspections.

Professionally, he is a registered pharmacist with clinical pharmacy knowledge in all therapeutic indications and particular experience as hospital pharmacy lead in endocrinology and ophthalmology.