15 November 2016

Accelerating Commercialization of Rare Disease Treatments

Brussels, Belgium

Mapi is the market leader in all the disciplines needed to commercialize Orphan Drugs and manage Rare Disease programs

Real World Evidence: Industry leaders in real world research to support regulatory requirements, commercialization evidence, and engage treatment populations

Patient Insights: Services to increase recruitment and retention, and ensure the patient’s voice is heard throughout the lifecycle of drug discovery, from protocol development to post marketing

Direct-To-Patient: A dedicated global team and call center enabling patient enrollment, retention, and support throughout the study

Patient Reported Outcomes: World-renowned leadership with access to over 4,000 PRO measures and expertise in custom scale development

Language Services: Recognized leaders in PRO validation, patient support, and HCP materials

Strategic Regulatory Services: Offering consultation and experienced procurators for regulatory meetings

Pharmacovigilance & Risk Management: Experts in international pharmacovigilance and safety, with strategically placed Qualified PV contacts

Real World Strategy & Analytics: Global Health Economics team with expertise in Rare Disease research, including Meta Analysis, Cost Consequent Analysis, and Market Access to support payers and health technology assessments

Patient Advocacy: Deep relationships with both patient advocacy and patient community groups

Selected Presenters

  • Will Maier
  • Patricia Anderson
  • Céline Taveau

Dr. Will Maier

Mapi's Chief Scientific Officer, Dr. Will Maier provides scientific leadership to Mapi's research and consulting services across a range of scientific areas. Dr. Maier has more than 20 years of drug development and commercialization experience. He served as Senior Director of Epidemiology at GlaxoSmithKline and Elan Pharmaceuticals and led research groups conducting observational research to support reimbursement, marketing and drug safety investigations of pharmaceuticals. Dr. Maier is a member of the EMA's European Network of Centres for Pharmacoepidemiology and Pharmacovigilance.

Patricia Anderson

Patricia Anderson began her career at Novopharm in the micro lab and then at Bayer, where she moved into Regulatory Affairs. After a string of successful NDS's with Bayer, she moved to a start-up, where she achieved international approval of their lead product. Currently, Ms. Anderson is VP of Regulatory Services with Mapi and has assisted with numerous global filings. She is one of TOPRA's North American certified European Regulatory Affairs Instructors. She also is on RAPs Planning Committee for their Annual Meeting and is on the Board of Editors for RAPs Focus.

Céline Taveau

Céline Taveau has 8 years of experience in market access. Céline provides senior leadership for projects on strategic market access and HTA's for pharmaceutical, vaccine and medical device companies. Céline has extensive experience with orphan drugs and has worked on projects in oncology, immunology, cardiovascular and infectiology. Her experience is wide working on projects for market access strategies, value proposition development, French HTA submissions and payer advisory boards.

Discussion points include:

Methodologies in developing Real World Data: Engaging stakeholders in developing evidence strategies
  • How is expanded access used to provide treatment for patients with rare diseases?
  • Compassionate Use Programs in the window between submission and commercialization
  • What are the benefits of conducting clinical studies and registries for rare diseases?
  • What are the types of studies conducted during different phases of orphan drug development?
  • How are patient registries used to generate real-world data for rare diseases and orphan drugs?
  • Ethical Patient Warehousing – working with Advocacy to identify populations in advance of research
  • What are the unique challenges of conducting rare disease/orphan drug registries?
  • How can proactive study management strategies successfully address these challenges?
Market Access Challenges for Rare Diseases: Recent Developments in HTA and Reimbursement Decision-Making Considerations for Orphan Drugs in Europe
  • Opportunities for early engagement with stakeholders, including clinical and economic evidence generation advice
  • The current reimbursement environment in Europe
  • Country-specific orphan drug programs and information requirements
  • Existing challenges to reimbursement
  • Demonstrating cost-effectiveness/budget impact
  • Innovative schemes for reimbursement of orphan drugs
Global regulatory strategies: The emerging global Regulatory Pathway for Rare Disease treatments
  • Regulatory strategies for successful orphan drug R&D
  • Have regulations increased or shifted making designation or approval more difficult?
  • Perspectives form Europe and US priority review vouchers


Register to Attend Mapi’s Overcoming Challenges in Rare Disease Health Research & Orphan Drug Program Commercialization Workshop:

The Venue

sheratonSheraton Brussels Airport Hotel

Brussels National Airport
Brussels, 1930, Belgium

Tel: +32 2 710 80 00

Dining & Refreshment

Open for lunch and dinner Monday through Friday, Gullivers Restaurant welcomes you with light French cuisine, bu et breakfast, bu et lunch, and an à la carte menu o ering gourmet suggestions.

Our Chef invites you to discover a wide range of local Belgian tastes. Through the “100% Belge” initiative, his team creates dishes made with the best local products. Not only a positive project for the environment, but also a great opportunity to discover some of the best products of our region and learn more about all the men and women cultivating these products.

In The Area


One minute from the hotel you can nd the biggest shopping street of Brussels. This main street is lined up with shops (mainly clothes), all spread alongside the City 2 shopping mall. The City 2 shopping center also includes the upscale Inno Gallery department store. The other side of the streets will lead you to the city’s Stock Exchange and the Opéra house ‘La Monnaie’.


This square is the most important tourist destination and most memorable landmark in Brussels. It is also a UNESCO World Heritage Site. Next to the Grand-Place enjoy typical Belgian dishes at Rue du bouchers or ‘Brussels Belly’, one of the most characteristic pedestrian streets of Brussels.


The venue specializes in concerts and music festivals, as well as exhibitions of art and photography. It also o ers a panoramic view which is one of the most surprising of Brussels.


In the land of Smurfs and Tintin, experience a truly Belgian experience! Brussels is famed for its comic strip art so don’t miss out on this unique discovery. This building is also an Art Nouveau gem by Victor Horta.