Featuring

13th Orphan Drugs & Rare Diseases Global Congress 2020 Americas - East

Days
Hours
Minutes
Seconds

17th November 2020 - Pre-Conference Workshop
18th - 19th November - 2 Days Conference
Boston, MA, USA

Venue

Hilton Boston Back Bay

Address

40 Dalton St, Boston, MA 02115

Phone

(617) 236-1100

Pharma Conference US Boston

Orphan Drugs & Rare Diseases

Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases Global Congress 2020 Americas. It’s the 13th in the series of our Flagship tri-annual Orphan Drugs & Rare Diseases event. Due to the pandemic, we are looking to host a HYBRID Event, still the same congress but with a different set-up. This congress will provide you with a comprehensive overview of the critical issues shaping the future of Orphan Drugs.

The COVID-19 Pandemic has impacted the lives of just about everyone, patients receiving continuous treatment and care included. The Rare Disease community is already experiencing considerable challenges in achieving early and detailed diagnosis and having access to care and life-saving treatment, to chronic, highly complex, progressive and severely disabling nature of their diseases, which generate specific care needs. Undoubtedly, during this crisis, the rare diseases community is experiencing unprecedented overall disruption even more….

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Insights & Attendees

Featuring Key Industry Experts

Andrea C. Furia-Helms

M.P.H., Director, Patient Affairs Staff, Office of Clinical Policy and Programs, Office of the Commissioner, U.S. Food and Drug Administration

Alan J.Balch

PhD, CEO, National Patient Advocate Foundation

Kristin Smedley

President, Curing Retinal Blindness Foundation

David A Pearce

PhD, President of Innovation, Research, & World Clinic, Sanford Health

All Expert Speakers

From a patient advocacy organization perspective, was a very informative meeting. Learned a lot from presentations and networking that will inform how we can add value to the advocacy-patient-drug developer dialogue.

aftd
Dr. Nadine Tutton

Scientific Director, Research, Association for Frontotemporal Degeneration

I was very impressed with the format, the content was interesting and well done. I felt I learned a lot and was glad to be able to attend.

pharmolam
Senior Director

Business Development at Pharm-Olam, LLC

Introducing

2020 CONFERENCE FORMAT

The two-day Congress will provide an interactive, cutting edge and comprehensive discussion and networking format led by key experts with intimate knowledge in the industry. Gain practical strategies and best practices on challenges, innovations, technologies and concepts surrounding orphan drugs and rare diseases.

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Sponsor & Exhibit
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Dear Colleagues,

Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases Global Congress 2020 Americas. It’s the 13th in the series of our Flagship tri-annual Orphan Drugs & Rare Diseases event. Due to the pandemic, we are looking to host a HYBRID Event, still the same congress but with a different set-up. This congress will provide you with a comprehensive overview of the critical issues shaping the future of Orphan Drugs.

The COVID-19 Pandemic has impacted the lives of just about everyone, patients receiving continuous treatment and care included. The Rare Disease community is already experiencing considerable challenges in achieving early and detailed diagnosis and having access to care and life-saving treatment, to chronic, highly complex, progressive and severely disabling nature of their diseases, which generate specific care needs. Undoubtedly, during this crisis, the rare diseases community is experiencing unprecedented overall disruption even more….

According to a report by Fortune Business Insights, the Global Orphan Drugs Market size will emerge from COVID-19 crisis at a moderate growth during the forecast period of 2020 to 2026. The global orphan drugs market size is projected to reach USD 340.84 billion by the end of 2027. As stated, the market was worth USD 151.00 billion in 2019 and will exhibit a CAGR of 10.5% during the forecast period, 2020-2027.

The current regulatory climate increasing number of regulatory approvals, expanding numbers of successful clinical trials related to this drugs, tremendous investments in R & D, and also the interest and existence of a number of large scale manufacturers, will create opportunities and contribute exponentially on the overall market growth.

The two-day Congress will provide an interactive, cutting edge and comprehensive discussion and networking format led by key experts with intimate knowledge in the industry. Gain practical strategies and best practices on challenges, innovations, technologies and concepts surrounding orphan drugs and rare diseases. We look forward to welcoming you at the congress!

Sincerely yours,

Jocelyn Raguindin
Jocelyn Raguindin

Jocelyn is Conference Director at Paradigm Global Events (PGE). She has over 15+ years experience in organizing pharma-industry conferences and has been at the helm of every PGE conference since 2013. Jocelyn is based in London, UK.

Gain Latest Insights on:

  • Current Orphan Drugs landscape and implications of COVID 19
  • Major drivers in global rare disease market
  • Key developmental strategies carried out and Influencing factors that may affect market share to stand out in this industry
  • Current FDA thinking regarding common issues encountered in rare disease drug development
  • Finding innovative and alternative ways in funding the development of Orphan Drugs
  • Patients perspective: what really matters to rare disease patients and caregivers?
  • Key authorities facilitating development and approval of diagnostic products/services
  • Coming Together in Developing Orphan Drugs and Crossing Borders
  • Trends and evolution of Advance Therapy Medicinal Products
  • How Can the Developer and the Patient Assist in the Evolution and Development of Orphan Drugs to make it accessible to patient in shorter period of time?
  • Strategies to improve Access and Affordability
  • What Do Developers Look for When Looking for an Outsourcing Partner?
  • What Do Insurance Companies Think About Orphan Drugs? Will They Make Modifications to Their Policies to Support Patients with Rare Diseases?
  • Opportunity to network with Peers, potential Partners and Investors

Who Should Attend

  • This congress is specially created for valued stakeholders in the Rare Disease community: Presidents, Heads/Chiefs, VPs, Directors, and Managers in the area of:
  • Clinical Research Organizations
  • Research and Development
  • Personalised Medicine
  • Regenerative Medicine
  • External R&D Innovation
  • Innovative Medicine
  • Rare and Ultra-Rare Diseases
  • Cell and Gene Therapy
  • Translational Science
  • Molecular Geneticist
  • Program Management
  • Patient Advocacy Groups
  • Public Affairs
  • Medical Affairs
  • Regulatory Affairs
  • Market Access
  • Pricing and Reimbursement
  • Health Economics Outcomes Research
  • Commercial Development
  • Investments and Funding
  • Product Specialists
  • Global Strategic Services
  • Business Planning and Operations
  • Pharmacies
  • Academia

Andrea C. Furia-Helms

M.P.H., Director, Patient Affairs Staff, Office of Clinical Policy and Programs, Office of the Commissioner, U.S. Food and Drug Administration

Andrea Furia-Helms is the Director of the Patient Affairs Staff in the Office of Clinical Policy and Programs, Office of the Commissioner. In her role, she collaborates with patient communities, the FDA medical product Centers and other offices to incorporate patient and caregiver perspectives in cross-cutting regulatory meetings. Ms. Furia-Helms spent over ten years in the FDA’s Office of Health and Constituent Affairs where she directed the FDA Patient Representative Program and coordinated patient engagement activities for the agency.

Prior to FDA, Ms. Furia-Helms was Director of the Back to Sleep (now Safe to Sleep) campaign, a public-private partnership to educate communities on Sudden Infant Death Syndrome (SIDS), at the National Institutes of Health.  She developed SIDS outreach initiatives for African American, American Indian and Latino communities.

Ms. Furia-Helms has a B.A. in psychology from Framingham State University, a B.S. degree in community health education from University of Maryland, and a Master of Public Health degree from The George Washington University.

Alan J. Balch

PhD, CEO, National Patient Advocate Foundation

Dr. Balch has over fifteen years of executive leadership in the non-profit sector with an emphasis on consensus-building and collaboration.  He has led numerous federal advocacy efforts on a range of issues both at the legislative and regulatory level.  He became the CEO of both NPAF and PAF in 2013.  From 2006 – 2013, he served as the Vice President of the Preventive Health Partnership — a national health promotion collaboration between the American Cancer Society, American Diabetes Association, and American Heart Association. Prior to 2006 Dr. Balch was the Executive Director of Friends of Cancer Research. 

Dr. Balch has served on the Executive Board of the Patient Advocate Foundation and National Patient Advocate Foundation since 2007.   He also serves on numerous advisory boards and committees. 

He earned his PhD in environmental studies with a concentration in political economy in 2003 from the University of California, Santa Cruz; master’s degree in environmental sciences in 1997 from the University of Texas in San Antonio; his bachelor’s degree (cum laude) in biology in 1994 from Trinity University in San Antonio.

Kristin Smedley

President, Curing Retinal Blindness Foundation

Kristin Smedley’s two sons were diagnosed soon after birth with a rare blindness. Although Kristin was initially paralyzed with fear of raising two blind children, she launched an extraordinary mission to change their bleak future ahead.   She worked with education systems in multiple states in which she lived to get her sons the tools they needed to achieve in public schools.

Her children have become popular, accomplished athletes, high achieving students, talented musicians, and International Braille competition finalists. Her oldest son, Michael has achieved Dean’s List in his first two college semesters.

In 2011 Kristin founded the only patient organization in the world for her sons’ inherited retinal disease.  CRB1.org is a global leader in changing what a rare eye disease diagnosis means, and Kristin is a sought-after speaker and collaborator in the field. 

Kristin recently published her first book, Thriving Blind: Stories of Real People Succeeding Without Sight.  It hit #1 New Release in Kindle and Paperback on Amazon.com.

David A Pearce

PhD, President of Innovation, Research, & World Clinic, Sanford Health

David Pearce is President of Innovation, Research, & World Clinic for Sanford Health. He completed his undergraduate Bachelor of Science Degree with honors in biological sciences at Wolverhampton Polytechnic in 1986.  He gained his PhD in 1990 at the University of Bath, UK, and did postdoctoral training at the University of Rochester, U.S., and Oxford University, UK.

Dr. Pearce heads the leading lab in Juvenile Batten disease research.  He has been researching Juvenile Neuronal Ceroid Lipofuscinosis (Batten disease) since 1997. His research has led to the first clinical trial for Juvenile Batten disease.  He has published over 100 research papers on Batten disease. He also oversees a national registry for rare diseases known as the Coordination of Rare Diseases at Sanford (CoRDS).   He has served on numerous NIH review committees, has organized rare disease workshops for the National Institute for Neurological Disorders and Stroke (NINDS) arm of the National Institutes of Health (NIH) and is currently the vice chair of the consortium assembly for the International Rare Diseases Research Consortium (IRDiRC). 

As President of Innovation, Research, & World Clinic at Sanford, he oversees the development of research programs, including more than 450 researchers, eight research centers and more than 300 ongoing clinical trials.