A18. Rare disease therapies: strategies and considerations in developing an orphan medicine

Tracks
Prescription and Non-Prescription Medicine Regulation
Wednesday, June 12, 2024
2:45 PM - 3:45 PM
C2.2+C2.3

Chair & Speakers

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Dr Elaine Blair
Director of Regulatory Affairs APAC
PTC Therapeutics

Chair: Rare disease therapies: strategies and considerations in developing an orphan medicine

Abstract

Regulatory agencies worldwide have implemented orphan drug legislations to facilitate research, development, and marketing approval of drugs to treat rare diseases. Central to these regulations are the concepts of scientific rationale, prevalence of the medical condition, and clinical superiority/significant benefit to achieve orphan drug designation. In this session, we will delve into the current regulations, strategies and challenges in developing an orphan product. Recent updates to legal framework and orphan policies in EU and APAC countries will be presented. We will also navigate challenges and practical considerations when developing an orphan drug in clinical research.

Biography

Elaine Blair has over 25 years' experience in the pharmaceutical industry, with extensive knowledge in a wide range of therapeutics areas including oncology, rare diseases and gene therapy. Elaine is currently Director of Regulatory Affairs APAC at PTC Therapeutics and is responsible for regulatory matters and strategies for countries in the APAC region. PTC is a global biopharmaceutical company that delivers therapies for people living with rare diseases. Elaine holds a PhD in Pharmaceutics from the University of Sydney. She is passionate about supporting regulatory affairs and education and has been an active member of ARCS since 1999. Over the years, she has served as convenor of the regulatory education sub-committee, conference advisory committee member, and as a Chair/Speaker at ARCS conferences.
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Helene Sou
Global Regulatory Policy, Emerging Markets
Takeda

Rare disease therapies: strategies and considerations in developing an orphan medicine

Biography

Helene Sou is a Director in Global Regulatory Policy and Innovation, focused on the Growth and Emerging Markets region in Takeda and currently based in Singapore. Helene has 19 years of experience in international regulatory affairs gained through country, regional and global regulatory roles. In the early part of her career, Helene worked several years as an EU Regulatory Consultant at Voisin Consulting Life Sciences in Paris, France before expanding her focus to International Regulatory Affairs in Boston, USA. In Shire Pharmaceuticals, Helene was the Asia-Pacific Regional Regulatory Lead and Regulatory Head of Southeast Asia. In Takeda, Helene was the Global Regulatory Lead for plasma-derived therapies, then acted as the Regulatory Liaison for the Emerging Markets. Currently, she is an active member of the Global Regulatory Policy Network (formerly APEC Rare Disease Network), IFPMA, EFPIA-ASEAN and Korea-Taiwan network and DIA Singapore Program Committee. Helene holds a Master of Sciences degree in Biotechnology and Management from the ESIEE-Management school in Paris.
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Adele Hosseini
Director
HealthCare Excellence Pty Ltd

Rare disease therapies: strategies and considerations in developing an orphan medicine

Biography

Dr. Adele Hosseini, an accomplished executive in scientific and clinical affairs, with extensive experience in the pharmaceutical and biotech industry. She holds a PhD in Pharmacy from the University of Sydney and has contributed to drug development in various management and consultancy roles. Dr. Hosseini's experience in C-Suite roles and management positions in drug development and clinical research spans over three decades. Her expertise lies in developing comprehensive strategies that align with overarching business objectives. She is well-versed in drug development strategy and pathway, with a foundation of experience in leadership, global clinical research, regulatory affairs, and university lecturing. Her exposure across pharmaceuticals, clinical research organisations, medical devices, and academia has equipped her with the skills necessary to develop effective strategies and pathways for drug development.
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Dr Pedro Franco
Head Of Europe Global Regulatory And Scientific Policy
Merck Serono Limited

Rare disease therapies: strategies and considerations in developing an orphan medicine

Biography

Pedro Franco has an extensive educational background, holding a degree in Pharmaceutical Science (PharmD) from the University of Lisbon, a Master's in Organic Chemistry (M.Sc) from the New University of Lisbon in collaboration with Imperial College in London, a PhD in Orphan drugs and Rare diseases from the University of Sheffield, and an Executive MBA from Business Bayes School at City University in London. He also completed his music studies in Cello at Lisbon Conservatory. Currently, Pedro serves as the Head of Europe Global Regulatory and Scientific Policy at Merck KGaA, focusing primarily on regulatory intelligence, strategy, and policy. He also serves as the liaison for the China and Latam regions concerning regulatory and policy topics. Prior to his role at Merck, Pedro worked at the European Medicines Agency for 12 years in various roles and departments, with a focus on regulatory affairs, international collaboration, assessment, authorization of medicines, orphan drugs, pediatrics, peer review, quality, and strategy. Before that, he was employed at INFARMED, the Portuguese national competent authority, as a pharmaceutical assessor. Earlier in his career, he worked as a postgraduate research fellow in the area of toxicology, carcinogenic chemistry, and drug development.
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