Workshop Description:
The importance of orphan products to serve unmet clinical needs for rare diseases continues to increase, with considerable political support, as evidenced by approximately one-third of drugs approved by the FDA in 2012 having orphan drug designation.  With nearly 200 new orphan drugs entering the development process each year, these orphan products represent one of the fastest growing segments in the biopharmaceutical industry.  In Australia and in the major jurisdictions, orphan drug candidates arise from diverse technologies including new biological and small molecule drugs, and gene and cell therapies, all intended to address unmet needs of patients afflicted with rare diseases.
This workshop will cover regulatory strategy for orphan drug and device development from the global perspective and take into account the commercial perspectives.  It is designed to provide attendees with in-depth understanding of regulatory pathway, registration and marketing strategies, timelines and potential pitfalls in development and registration of medicines for rare conditions.  Selected case studies will be discussed in details to provide practical advice and example, as well as highlight key aspects for their success and failure.  Learn hands-on approaches on how to build your program and prepare Orphan Drug Applications.  Will be also discussed different regulatory strategies for orphan drug and device products versus non-orphan products, and how those are relevant to your product.

Workshop Outline:

• Are you an Orphan Drug?
• Financial benefits and market exclusivity
• How to determine which incentives applies to your program
• Associated challenges of orphan drug programs, how to predict and avoid them
• Case Study Approach (success and failure)
• Strategies behind successful programs
• Regulatory pathway and timelines
• How to write Orphan Drug Applications
• Regulatory options from the European, US and Australian perspectives
• Advantages and disadvantages of recent developments in the regulation of orphan drug approval in Australia, EU and US

Chris Holloway, BSc, Ph.D, Dr.rer.hum.biol.habil
Chief Scientific Officer at ERA Consulting Group
Dr. Chris Holloway has worked in regulatory affairs, specialising in biotech products and other biologics for nearly 30 years, since leaving academia, where he was Professor of Clinical Biochemistry at Hannover Medical School in Germany. Chris graduated in 1971 and obtained his Ph.D. in 1975, after which he held a Royal Society European Research Fellowship at the Max-Planck-Institute in Germany. He then moved into university, and was awarded the Venia Legendi in 1982.
Chris began consulting in the regulation of biologics in the 1980s. Subsequently, in 1987, he launched the regulatory arm of IBR, providing regulatory support for companies and European national agencies alike. At the vanguard of this field, Chris has personally been involved in the development of over 400 different products, contributing particularly towards approaches to manufacturing and control as well as early non-clinical and clinical development. His team has compiled complete dossiers, and Chris authors expert reports for European and international submissions. He has personally had oversight in the approval of biosimilar products in Europe and his team is currently working on around 10 different biosimilar products.
Of the many twists, turns and pitfalls of getting a biosimilar to market, Chris has interacted with EU member state agencies, the EMA and FDA. Coupled with a thorough knowledge of current regulatory guidelines and regulatory expectations in general, this has enabled clients of ERA to confidently navigate these challenges, thus expediting their application.

Dianne Jackson-Matthews, BSc, Ph.D, RAC
Deputy Group Director of Regulatory Affairs at the ERA Consulting Group
Dr. Jackson-Matthews is, and heads the ERA Consulting (Australia) Pty Ltd operation in Brisbane, Australia. Dianne has over 25 years of experience in pharmaceutical product development, spanning the areas of drugs, biotech/biologics/biosimilars and cell/gene therapies in the US, Europe and Australia. Dianne has experience in development of regulatory and technical strategies, conducts regulatory agency interactions worldwide, prepares regulatory documentation and submissions, and performs regulatory and technical due diligence assessments supporting funding and licensing opportunities for the investment community and the industry.
Prior to joining ERA in 2001 as the Director of the Washington DC office, Dianne was the Director of Regulatory Affairs at a biotech company in New Jersey, where she gained extensive experience with biotechnology products over a 12 year period, covering GMP production, GLP and GCP testing, and regulatory affairs. She also has 6 years’ experience with in vitro diagnostic device development in the US. Dianne has held an Adjunct Associate Professor position at the University of Queensland, Australia, since 2010.

Michael Flood, Principal at Locus Consulting Pty Ltd
Michael Flood has nearly forty years’ experience working in the medical devices industry. Joining the Therapeutic Goods Administration in 1993, he re-established and expanded the agency’s post-market programs for medical devices.  He has had extensive involvement with premarket assessment systems management and policy development in Australia, and was involved in the introduction of a Global Harmonisation Task Force (GHTF) based regulatory framework for medical devices in 2002.
After leaving the TGA in mid-2010, Michael Flood has established a consultancy practice in Australia – Locus Consulting, specializing in regulatory affairs, international regulatory training for economies introducing devices regulations, health technology assessment and biomedical engineering.
Michael is very active in a number of standards development committees for medical devices, both within Australia and internationally, and has held visiting appointments at several Australian Universities, including an appointment as adjunct lecturer in a biomedical engineering elective unit at the Australian National University.