Orphan Drug Designation: Meeting the Unmet Need in Rare Diseases

Life Sciences, Pharmaceutical Regulation, Pharmaceutical, Drug Discovery & Development,
  • Thursday, April 26, 2018

Achieving Orphan Drug Designation is a significant step enabling your product to be licensed faster, in a cost-efficient manner, and achieve market exclusivity for 10 years.

Join this 1-hour webinar where the audience will gain a practical overview of the steps to achieve orphan status including:

  • Review of the Orphan Drug Designation eligibility criteria, including a registered medicine with a new ‘orphan indication’
  • An overview of Orphan Drug Designation incentives, such as tax, marketing approval and market exclusivity
  • Practical application, process and insights
  • EU & FDA requirements and assessing the differences


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.

Message Presenter

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.

Message Presenter

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.

Message Presenter

Who Should Attend?

Senior professionals from bio/pharmaceutical companies developing therapies for rare and orphan diseases, or a registered medicine with a new ‘orphan indication’.

What You Will Learn

  • What criteria make a drug eligible for Orphan Drug Designation
  • Which incentives make Orphan Drug Designation beneficial for biopharmaceutical companies
  • Insights into the process of applying for Orphan Drug Designation
  • The differences between EU & FDA requirements

Xtalks Partner

Simbec Orion Clinical

Orion Clinical is the full-service clinical development division of Simbec-Orion Group.

Founded 20 years ago as a full service CRO we have expanded, through consistent delivery of studies in complex areas. Our operations are based in the United Kingdom, France, USA, Germany and Italy. We have operational staff across mainland Europe (Western, Eastern & Central) and throughout North America.

With over 20 years of experience in the delivery and conduct of oncology clinical trials, including rare & orphan and paediatric studies, we use our skills elegantly to design, execute and deliver our clients’ clinical development needs.

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