THE GLOBAL GUIDE
Finally, there is a single resource you can turn to that will provide the information required to initiate a compassionate use/expanded access program
The Global Guide to Compassionate Use Programs was developed over four years by industry, government, regulatory, operational and pharmacovigilance experts with decades of combined global experience.
This book will help you navigate the complex and often confusing individual country regulations and clearly set forth the formal guidance documents, submission requirements and applications – and clarify the roles and responsibilities of the key parties involved in compassionate use programs including sponsor (manufacturer) led and individual physician requests for access.
Using this comprehensive reference guide will save you valuable resources navigating through and translating various country-specific websites. This compendium of over 60 countries, where there is a validated pathway for compassionate use, contains specific detail on the various regulatory pathways as well as critical internet links that allow you to immediately access source regulation documents, including those dealing with pharmacovigilance and drug labelling requirements.
Regular updates are available that address new or revised legislation which will provide you with the most up-to-date information and ensure compliance for stakeholders that may be involved in compassionate use from industry sponsors, manufacturers, individuals and groups of physicians, specialty service providers and independent consultants.
Purchasing a subscription will give you immediate access to current content, with regular RSS feeds alerting you to updates on a per-country basis.
Who is the resource designed for?
- Biopharmaceutical Companies
- Regulatory Authorities
- Specialty Service Providers
- Global Program / Project Managers
- Medical Affairs Regulatory Professionals, Drug Safety, and Supply Chain
- Drug Distributors and Wholesalers
- Patient Advocacy Groups
- Non-Governmental Agencies (NGOs)
- Professional Societies
- Legal Firms
About the Authors
Sean D. Turbeville, PhD
Sean Turbeville, PhD, has 18 years of biotechnology experience and has worked extensively in Global Medical Affairs. His expertise is in orphan products (Onclogy, Genetics, Metabolic, and Neuromuscular) with small biopharmaceutical companies and creating international programs to support clinical and commercial therapies in the United States, Latin America, European Union, Asia Pacific, and the Middle East.
Sean has held a variety of roles in Medical Affairs from building medical affairs organizations, overseeing strategy and operations, creating medical information, and scientific communications to launching compassionate use/expanded access programs and medical research initiatives.
Carl S. Hornfeldt, PhD, RPh
Carl S. Hornfeldt, PhD, RPh is currently the president of Apothekon, Inc. In this capacity, he provides medical information and medical writing expertise with an emphasis on specialty diseases to numerous pharmaceutical, biotechnology and medical device companies. He has written or assisted with the publication of over 200 peer-reviewed manuscripts and hundreds of abstracts and standard response letters for client use in the fields of oncology, dermatology, toxicology and numerous rare diseases such as narcolepsy and liposomal storage disorders.
Richard Klein, BSA
Richard Klein is an internationally recognized expert in pre-approval, or expanded access to unapproved therapeutic agents. He currently serves as Director, Expanded Access Programs & Policy for the GE2P2 Global Foundation after more than a 41-year career with the Food and Drug Administration (FDA). During that time, he worked in various capacities at FDA providing him with a well-rounded understanding of the regulatory issues that affect patients. He helped develop the revised expanded access regulations and guidelines, led the creation of the FDA expanded access website, and played an active role in the development of the streamlined application for individual patient access, and the Reagan-Udall Foundation.
Connie Coulomb, MBA
Connie Coulomb, MBA is Managing Partner at Coulomb Strategy Consulting LLC, a firm dedicated to helping companies advance their strategy and turn exciting research into successful commercial products that benefit patients’ lives. With more than 20 years’ experience in biopharma, Mrs. Coulomb has held a variety of roles in the areas of strategy, business development, marketing, sales, market research and market access in large, medium and small biopharma companies, including Merck, Amgen, Biogen, Gilead, and Onyx. In her most recent corporate role, Connie Coulomb served as Divisional Vice-President and General Manager for Biogen, where she managed a $1.5B Multiple Sclerosis Business in the United States.
Karen Frascello, BSc
Karen has over 26 years of experience in the biopharma industry, in a variety of commercial and strategic consulting roles. She started her career as a pharmaceutical sales representative and was promoted into increasing levels of managerial responsibility eventually having oversight for large sales teams across the US for Pfizer and AstraZeneca. Karen moved into the Compassionate Use arena in 2010 when she joined Clinigen (formerly Idis, Inc.), a leader in the design, implementation and management of global compassionate use programs for pharma and biotechnology companies. In 2015, she was recruited by Caligor Coghlan to build a new compassionate use business unit and was responsible for all new business development and had full oversight of global operations and regulatory affairs. In 2019, she joined Alnylam Pharmaceuticals as Director, Global Medical Affairs, Early Access. In this role, she has oversight of all planning and implementation for global early access across the entire pipeline of rare disease products.
George van Baelen, MScPh
Georges van Baelen, MScPh is Founder and President of Arctiryon, Inc., headquartered in Chicago, USA. He studied Medical and Pharmaceutical Sciences at the Katholieke Universiteit Leuven, Belgium and did doctoral work in Toxicology and Advanced Pharmacology at the Joint Graduate Program at Rutgers University / Robert Wood Johnson Medical School in New Jersey, USA, before starting a career in Medical Information and Drug Safety in the pharmaceutical industry. He also obtained certification in Pharmacovigilance Practices from the University of Antioquia in Medellin, Colombia. He became a Director of Cardiology at United Health, participating in publication planning and authoring several articles on heart disease and its therapies. As Global Director of Pharmacovigilance at AXA, Mr. van Baelen instituted and built comprehensive pharmacovigilance systems while overseeing global Medical Information Services for various biotechnology and pharmaceutical companies.
Joanne Sparkes, MSc
Joanne Sparkes, MSc is the operational lead for this resource. Like all the team she has a scientific background having trained and worked in the UK National Health Service as a Medical Physicist. She now runs a data-driven digital marketing agency, Expedio.
Joanne excels at designing bespoke spreadsheets for the analysis and presentation of data. She also helps businesses use their data to make decisions and to connect the technical pieces of working online – from developing websites, online courses and self-publishing to social media campaigns and email marketing automation.