Orphan Drug & Rare Disease Development: Understanding the European and US Regulatory Landscape

Life Sciences, Clinical Trials, Pharmaceutical Regulation, Patient Recruitment & Retention,
  • Tuesday, May 19, 2015

Today more than ever researchers are focused on providing care for diseases that in the past have remained neglected. The National Institute of Health (NIH) has identified around 7,000 rare diseases, many of which are of genetic origin. This presentation focuses on the regulatory aspects of bringing therapies for rare diseases to the market, specifically to identify how the regulatory pathways between Europe and US are similar and where there are differences.

Understanding these regulatory nuances can be critical for the development of such medicinal products for rare diseases, also known as orphan medicinal products. Our speakers will present an overview of current guidance documents, possible loopholes, and possible strategies to take into consideration when developing such products.

Furthermore, the presentation aims to provide the audience with an understanding of how certain legal tools and incentives have stimulated the growth of the drug products for rare diseases and what other things can be done in order to achieve a better impact. Also presented are potential challenges and obstacles developers can encounter when dealing with the regulatory agencies and best practice to overcome them.


Nach Dave, Director, Regulatory Affairs, Premier Research

Mr. Dave has been working for and consulting to the Pharmaceutical and medical device industry for 15 years. He started in Data Management with Sandoz Pharmaceuticals and has since worked in senior positions in clinical operations and regulatory affairs for companies such as Merck, Bristol Myers Squibb, and most recently Maxx Orthopaedics. In addition to industry appointments, he was previously an adjunct professor in the Drug Regulatory Program at LIU.

His industry experience is comprised of efforts on more than 15 multi-national clinical trials, presenting at FDA and EMEA meetings and speaking engagements at international industry conferences. Mr Dave has personally led the efforts for filing IDE, PMAs, 510k submissions as well as having worked on INDs and NDAs.

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Carol Huntington, Associate Director, Regulatory Affairs Europe, Premier Research

Carol Huntington joined Premier Research in December 2014, as an Associate Director in Global Regulatory Affairs, where she provides regulatory strategic and procedural advice. Carol has about 20 years of drug development experience gained in small, medium and large pharma companies working in Germany, the UK and France. She has worked as a regulatory project manager, and also has experience of Regulatory Writing, including compiling briefing documents for Regulatory Agency meetings, as well as writing Module 2 clinical summaries, and Module 1 documents for MAAs and NDAs.

She has managed large complex projects from first-in-man studies to putting together the dossier and obtaining the approval in the EU for a project with a failed pivotal study. Hertrack record includes successful IND/CTA/MAA submissions, with expertise in development projects.

Prior to joining the Pharmaceutical industry, Carol worked for several years as a clinical hospital pharmacist, providing advice to doctors and pharmacists on all aspects of the use of medicines. As she is a registered pharmacist in the UK, she undertakes regular Continual Professional Education.

Carol has a pharmacy degree from Kings College, London. She is also associate editor for the Regulatory Rapporteur Journal, which is published monthly by TOPRA in the UK. Her therapeutic experience includes: haematology, oncology, adrenal insufficiency, cardiovascular, neuropathic pain, severe pain, epilepsy, women’s health.

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Who Should Attend?

VPs, Directors, Managers, Department Heads, Scientists and Researchers working within:

  • Clinical Affairs
  • Clinical R&D
  • Clinical Research
  • Clinical Pharmacology
  • Clinical Operations
  • Project Management
  • Regulatory Affairs
  • Medical Affairs

for pharmaceutical, biotechnology and medical device companies

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Premier Research

Premier Research is a leading CRO serving highly innovative biotech, Pharmaceutical and medical device companies. The company has a wealth of experience in rare disease and pediatric research having managed about 100 projects in each area in the last five years alone. Its services include clinical research and regulatory outsourcing in the areas of analgesia; neurology; infectious, cardiovascular, and respiratory disease; dermatology; oncology; and medical devices.

Premier Research and operates in 50 countries. It employs 1,000+ clinical professionals dedicated first and foremost to fulfilling each customer’s requirements in a timely, accurate, and cost-effective manner. This includes a strong international network of monitors and project management professionals combined with regulatory, data management, statistical, scientific, and medical experts, and staff at its well-established network of dedicated clinical sites.

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