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Orphan Drugs & Rare Diseases 2017 Americas Conference

September 12, 2017 @ 08:00 - September 14, 2017 @ 17:00 UTC+0


September 12-14, 2017


Crowne Plaza Hotel, San Francisco Airport, 1177 Airport Blvd., Burlingame, CA 94010

Event Overview:

The global orphan drugs market is expected to grow at a steady CAGR of 11.43% from 2016 to 2022. At the current forecasted growth rate, the orphan drugs market is expected to grow from USD 111.87 billion in 2016 and become worth USD 214.14 billion by 2022.

The growth of the global orphan drugs market is increasing due to the growing rare diseases. Orphan diseases are rare in nature which means its occurrence rate is very low, but its prevalence rate is expected to increase in the forecasted period thereby increasing the demand of global orphan diseases market. There are around 7000 rare types of rare diseases and disorders, these numbers are increasing day by day. 80% of rare diseases are genetically originated and rest are result of infections, environmental causes and allergies. Around 50% of genetic diseases are in children’s, 30 % of children with rare diseases cannot live for more than 5 years. Rare diseases are one of the life-threatening diseases that kills millions of lives worldwide. Orphan drugs are the pharmaceutical agent that is specially developed for rare diseases. The prevalence rate of rare diseases is growing at a faster rate, resuling in the growing need of drugs to cure that diseases.

Paradigm Global Events is again proud to present our bi-annual Orphan Drugs & Rare Diseases Global Congress 2017 Americas. The conference will provide a unique platform for the convergence of stakeholders in the orphan drugs industry to discuss and network with top tier government, hospitals, pharmaceuticals, biopharmaceuticals, non-profit organisations, orphan drugs developers as well as regional and local manufacturers. We are putting together an agenda that address the driving macroeconomic factors, policies and issues that will steer the development of orphan drugs globally including commercialisation, policies, reimbursement, pricing, access and more. We look forward to having you be part of the event! The two-day Congress will provide an interactive and intimate discussion and networking format led by key industry speakers with intimate knowledge in the industry. Gain practical strategies and best practices on challenges, innovations, technologies and concepts surrounding orphan drugs and rare diseases.

Attend this conference to learn about the following topics -

  • In-depth analysis of the orphan drugs markets
  • Focus on the existing and future trends of rare diseases & orphan drugs
  • Highlights on the key growth and investments
  • Insights on key technological trends impacting the orphan drugs market
  • Understanding the roles of the key players and stakeholders
  • Learn the key economic drivers in the orphan market
  • The Roles of Big Pharmas, Biotechs and Start-Ups
  • What Strategies Are Available to Ensure Partnership Success
  • Clinical Trials: From Early Drug Development to Patient Recruitment and More
  • Pricing Strategy: How to Price Correctly?

Who should attend?

Presidents, Heads/Chiefs, Directors, VPs and Managers of:

  • Research and Development
  • Regenerative Medicine
  • External R&D Innovation
  • Immunology
  • Clinical Scientist
  • Gene Therapy
  • Translational Science
  • Molecular Geneticist
  • Program Management
  • Patient Advocacy and Public Affairs
  • Medical Affairs
  • Regulatory Affairs
  • Clinical Field Specialist
  • Sales and Marketing
  • Outcomes & Evidence
  • Commercial Development
  • Product Specialist
  • Global Strategic Services
  • Business Planning and Operations

Mapi's Event Role:


Featured Speaker & Presentation:

Rory GrahamSenior Director, Regulatory Affairs, Europe & International Region, Mapi Group

Mr. Graham has facilitated numerous aspects of drug and medical device development during his 25 year career in the pharmaceutical, biotechnology and device industries. Mr. Graham has held senior positions in the area of regulatory affairs in companies in Europe and Asia-Pacific. He has arranged and directly participated in meetings with many global agencies including FDA, EMA, PMDA, TGA and European National Agencies. He has experience in various therapeutic areas including CNS, Oncology, Hematology, Immunology, Diabetes, Analgesia and Wound-Care. He has achieved orphan designations for pharmaceutical products in the EU, US and Australia. Mr. Graham leads a talented team of regulatory professionals that produce client-specific regulatory and development strategies, as well as solutions for global and local market needs

Day 2: September 13, 2017 at 12:10 - 12:40 // "The science of endpoints in the current regulatory & HTA environment – basic principles"

  • Introduction to the science of endpoint design in clinical trials in the current regulatory and HTA environment in the US and Europe.
  • The creation, categorization and hierarchy of endpoints will be described
  • A look at the varying approaches and requirements of different stakeholder groups, including: Regulatory Agencies, Clinicians, HTA bodies, Payors, and patient groups.
  • Real-life examples of endpoints used in selected therapeutic areas and Techniques for the development and validation of new endpoints.
  • Guidances and publications to assist newcomers to the area and Non-alignment between different agencies and geographical differences in approach.
  • Potential risk mitigation strategies and Alternative registration and agency interaction strategies

Click for additional information

Learn more:

Orphan Drugs & Rare Diseases 2017 Americas

Register to attend now:


Interested in attending this conference but haven't registered yet? Check out our pre-conference workshop first for discounted registration!

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September 12, 2017 @ 08:00
September 14, 2017 @ 17:00
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