Featuring

11th Orphan Drugs & Rare Diseases Global Congress 2019 Americas

Days
Hours
Minutes
Seconds

9th September 2019 - Pre-Conference Workshop
10th - 11th September 2019 - 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 2019 Americas. It’s the 11th in the series of our Flagship tri-annual Orphan Drugs and Rare Diseases event, this congress will provide you with current trends and innovations driving access to meet unmet medical needs of rare disease patients.

Introducing a new format this year, we are putting together an agenda that addresses “Current Trends and Innovations driving access to meet unmet medical needs of rare disease patients”.
We have gathered together 50+, high caliber expert speakers to participate and share their knowledge and expertise through Panel discussions, keynote presentations, round table discussions and 4 major agenda streams addressing all aspect of the industry.

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

NEW 2019 CONFERENCE FORMAT

New for Orphan Drugs & Rare Diseases Global Congress 2019 – choose your focus streams in the afternoon of both conference days by attending keynotes, presentations and group discussions that have the most relevance to you. Choose from..

Regulatory, Commercial & Current Trends

Click here for further details

Regulatory, Commercial & Current Trends

This focus stream covers improving the precision of precision medicines, understanding technical issues, novel approaches to regulation, the role of patient organizations in approval, pricing, reimbursement and more!

Access and Collaborations

Click here for further details

Access and Collaborations

This focus stream highlights global early access programs to support rare indications, the importance of Latin American sponsors with Orphan Disease Therapies, utilizing real-world evidence and more!

Innovations, Clinical Development & Advance Therapeutics

Click here for further details

Innovations, Clinical Development & Advance Therapeutics

This focus stream explores the rare disease final frontier and whether Asia is the place where innovation will be found in the 2020's. We also explore Hermansky-Pudlak Syndome, drugs development resources and much more.

Gold Sponsor

Gold Sponsor

Gold Sponsor

Silver Sponsor

Bronze Sponsor

Lead Exhibitor

Associate Sponsor

Scroll to Top

Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases Global Congress 2019 Americas. It’s the 11th in the series of our Flagship tri-annual Orphan Drugs and Rare Diseases event, this congress will provide you with current trends and innovations driving access to meet unmet medical needs of rare disease patients.

According to a recent report, North America continue to be anticipated to stay dominant in the global orphan drugs market between 2018 – 2025. According to the report, the global orphan drugs market was valued at US$ 125000 Mn in 2017 and is anticipated to reach US$ 294000 Mn by 2025. The report also stated that the market will exhibit a remarkable CAGR of 11.4% during the forecast period.As per Fortune Business Insights, the North America orphan drugs market was worth US$ 67,136.0 Mn in 2017 and will continue to rise at a steady pace until 2025.

The growth of the global orphan drugs market is increasing at a rapid pace due to the growing awareness of people concerning the use of novel drugs. Government in North Americas are also increasingly investing on the research and development and of orphan drugs and innovative technologies. With further growth anticipated to meet the high unmet demand for more efficacious drugs with very little side effects. Although, the number of people affected by rare diseases are considerably low, the return of investment is high due to the high cost of orphan drugs. Global collaboration is also likely to fuel growth. FDA recognizes the significance of orphan drugs in the treatment of rare debilitating, life-threatening diseases therefore supporting stakeholders to promote research and development in this area.

However, some factors such as high initial investment that leads to higher per patient treatment cost, reimbursement uncertainties and high cost of drug development are hindering the growth of orphan drugs market. North America registered significant growth for the market during the forecast period due to rising healthcare spending, constructive government initiatives, growing occurrence of chronic diseases and small timeline required for orphan drug development.

11th Orphan Drugs & Rare Diseases Global Congress 2019 Americas 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 organizations, orphandrugs developers as well as regional and local manufacturers.

Introducing a new format this year, we are putting together an agenda that addresses “Current Trends and Innovations driving access to meet unmet medical needs of rare disease patients”. We have gathered together 50+, high caliber expert speakers to participate and share their knowledge and expertise through Panel discussions, keynote presentations, round table discussions and 4 major agenda streams addressing all aspect of the industry.

We look forward to welcoming you at the congress!

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 major drivers in global raredisease market
  • Key developmental strategies carried out and Influencing factorsthat may affect market share to stand out in this industry
  • Current FDA thinking regarding common issues encountered in raredisease drug development
  • Finding innovative and alternative ways in funding thedevelopment of Orphan Drugs
  • Patients perspective: what really matters to rare disease patientsand caregivers?
  • Key authorities facilitating development and approval of diagnosticproducts/services
  • Coming Together in Developing Orphan Drugs and CrossingBorders
  • Trends and evolution of Advance Therapy Medicinal Products
  • How Can the Developer and the Patient Assist in the Evolution andDevelopment of Orphan Drugs to make it accessible to patient inshorter period of time?
  • Strategies to improve Access and Affordability
  • What Do Developers Look for When Looking for an OutsourcingPartner?
  • What Do Insurance Companies Think About Orphan Drugs? WillThey Make Modifications to Their Policies to Support Patients withRare Diseases?
  • Opportunity to network with Peers, potential Partners and Investors

You Will Meet:

  • Presidents, Heads/Chiefs, Directors, VPs and Managers in the are of:
  • 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
  • Clinical Research Organizations
  • 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

David Cory

President and CEO - Eiger BioPharmaceuticals

David Cory is an industry veteran with over 25 years of experience in large pharmaceuticals, emerging stage biotechnology, and specialty pharmaceutical organizations with an established track record of operational performance, including five successful Orphan Designation drug programs.

Cory is President and CEO, Director, and business founder of Eiger BioPharmaceuticals, leading the company since its founding in 2008 and responsible for negotiating the company’s multipletechnology and product licenses from Stanford University, Merck, Nippon Kayaku, Janssen, BMSand The Progeria Research Foundation. Prior to Eiger, Cory was President and Chief Operating Officer of Prestwick Pharmaceuticals, an orphan CNS specialty pharmaceutical company, which was acquired by Biovail for $160 million in 2007. Previously, Cory was a Co-Founder at CoTherix, an orphan pulmonary arterial hypertension company, which was acquired by Actelion for $425 millionin 2006. Cory began his biotech career at InterMune, focused on developing orphan products foridiopathic pulmonary fibrosis, where he was a Senior Vice President and a key executive in the company IPO, building and managing the company’s commercial organization, in-licensing 4 products and raising over $400 million in the capital markets. InterMune was ultimately acquired by Roche for $8 billion in 2013. Cory spent over a decade in large pharma in positions of increasing responsibilities in commercial operations at Glaxo, Glaxo Wellcome, and Glaxo Smith Kline where heled teams in oncology, critical care, CNS, infectious disease and respiratory therapeutic areas, and directed 10 newly approved pharmaceutical product launches.

Cory earned a Bachelor of Science in Pharmacy from the University of Cincinnati, College ofPharmacy, was board certified in Pharmacy in the state of Ohio, and holds a Master’s Degree inBusiness Administration from the University of Maryland.

Amit Rakhit

Chief Medical Officer and Head of Research & Development, Ovid Therapeutics

Amit Rakhit is Chief Medical Officer and Head of R&D at Ovid Therapeutics (NASDAQ: OVID), a publicly traded biotechnology company focused on developing medicines for people living with rare neurologic conditions.  In this role Amit oversees research, drug development and commercialization for the Ovid portfolio.  Amit has over 20 years’ experience including both clinical practice and pharmaceutical/biotechnology industry experience. Amit earned his B.A. in molecular biology from the University of California, Berkeley, his M.D. from Tufts University, his M.S. in clinical investigation from Vanderbilt University and MBA from Columbia University. He completed his fellowship in pediatric cardiology and was subsequently on staff at The Children’s Hospital, Boston affiliated with Harvard University.

Prior to Ovid, he was SVP and Head of Worldwide Medical at Biogen where he led the medical function in disease areas such as multiple sclerosis, neurodegenerative diseases, and hemophilia, and orphan diseases such as spinal muscular atrophy and amyotrophic lateral sclerosis.  Prior to Biogen, Amit was part of the R&D and medical organization at Bristol-Myers Squibb.

Amit is an Advisor Trustee for the Liberty Science Center in NJ as well as an Advisory member of the Healthcare Board of the Partnership Fund for New York City.  

Matthias Schoenermark

Managing Director, SKC Beratungsgesellscha

Prof. Matthias P. Schönermark, M.D., Ph.D., is a trained head & neck surgeon with a Ph.D. in molecular oncology. In 1998, he left his job as an Associate Professor at Hannover Medical School for a position as project leader & manager at The Boston Consulting Group, where he spent several years, supporting clients in the health care industry in Europe and overseas. In 2005, after a year as Associate Partner with A.T. Kearney, he founded the SKC Beratungsgesellschaft mbH (SKC). He was appointed as Professor of Health Care Management at Hannover Medical School in 2001. He spent several years at Columbia University in New York and at Dartmouth Medical School in Hanover, New Hampshire and lectures at universities & business schools in Europe and the U.S. on strategic issues in health care.

In his function as managing partner, he is a permanent consultant to numerous leadership personalities of international health insurance and provider organizations, as well as of medtech and pharmaceutical companies on strategic management, innovation management and change management issues. He holds an unrivalled track record of solving complex and demanding market access challenges, especially in the orphan drug and oncology sector and is one of the most experienced negotiation leaders in reimbursement and pricing procedures.

Alison Schecter

MD, FACC, FCCP, Global Program Head, Rare Disease (current), Sanofi Genzyme

Alison is currently the Global Program Head for the Rare Disease Development at Sanofi Genzyme. She is responsible for defining the strategic focus of Rare Disease development in collaboration with Head of Commercial. She works closely with external innovation and BD to make review and decide on opportunities to be brought forward for consideration including Bioverativ. In addition, Alison now leads the acid sphingomyelinase deficiency (ASMD) (formerly known as Niemann-Pick) Global Team. She coordinates the regulatory, clinical operations, medical affairs, legal and CMC to allow submission for the first in class therapy for this ultra-rare diseases with high mortality and morbidity and no known treatment.

Dr. Schecter is an internationally recognized physician scientist and board-certified cardiologist.  Her academic research focused on chemokine and cytokine signaling in cardiovascular and viral diseases focusing on vascular disease, lipids and heart failure with over 50 peer reviewed papers and multiple NIH RO1 grants that supported her independent laboratory. Dr. Schecter, as tenured Associate Professor at Mount Sinai School of Medicine, was Co-Director of the over one hundred person Mount Sinai Cardiovascular Research Center and sat on multiple NIH and AHA study sections and special panels.

Alison completed her Internal Medicine residency at the Johns Hopkins Hospital and her Cardiology fellowship at Massachusetts General Hospital with a research fellowship at Mount Sinai. She also co-founded the Women’s CARE (Cardiac Assessment and Risk Evaluation) Program to educate and treat under-served women in Spanish Harlem, NY and has appeared on national, local TV and radio as a medical expert on cardiovascular and women’s health.