Featuring

16th Orphan Drugs & Rare Diseases Global Congress 2021 Americas - East Coast

Addressing Scientific Challenges to Advance New Treatments for Rare Diseases

Days
Hours
Minutes
Seconds

18th October 2021 - Pre-Conference Workshops
19th - 20th October 2021 - 2 Days Conference

Exhibit & Sponsor Opportunities
Venue

Hilton Boston Back Bay

Address

40 Dalton St, Boston, MA 02115

Phone

(617) 236-1100

Orphan Drugs & Rare Diseases Conference - US Boston

Orphan Drugs & Rare Diseases

Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases Global Congress 2021 Americas. It’s the 16th in the series of our Flagship tri-annual Orphan Drugs & Rare Diseases event. 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

Barry Ticho, PhD

M.D., PhD., Chief Medical Officer, Stoke Therapeutics, Inc.

Rahul Ballal, PhD

President & CEO, Imara Inc.

Christopher Dandrea

Vice President, Global Program Management, uniQure

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

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

2021 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.

View Conference Agenda

Orphan Drugs & Rare Diseases 2021 Americas

Sponsors

Gold Sponsor

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Lead Exhibitor

Associate Sponsor

Associate Sponsor

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Dear Colleagues,

Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases
Global Congress 2021 Americas. It’s the 16th in the series of our Flagship tri-annual Orphan
Drugs & Rare Diseases event. This congress will provide you with a comprehensive overview of the critical issues shaping the future of Orphan Drugs.

The COVID-19 Pandemic has added urgency and profoundly impacted the lives of rare disease patients because they are amongst the most vulnerable to Covid-19. The 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 reports (Fortune Business Insights), the global orphan drugs market size was
valued at USD 151 billion in 2019 and is projected to reach 340.84 billion by 2027. The oncology therapy area possesses a substantially higher share in the global market as new cancer-related disease emerges and series of FDA approvals will result in increased commercialisation of these drugs. The growing demand for immunomodulators will also contribute to the orphan drugs market growth. Increasing R&D initiatives and investments by prominent players and patient advocacy involvement have resulted in major pharmaceutical breakthroughs, and the development of blockbuster drugs for the treatment of several rare diseases will create opportunities and contribute exponentially to the overall market growth.

However, the coronavirus pandemic is expected to negatively impact the global market. Another critical factor restricting the orphan drugs market is the high cost associated with this drug which can significantly limit the widespread adoption of this product, especially in emerging countries.

The two-day Congress aims to bring together stakeholders to discuss strategies and best
practices, innovations, technologies and concepts that can promote rare diseases product
development in an interactive, thought-provoking and uplifting manner, by way of a keynote
presentation, plenary sessions, panel discussions, roundtables and stream sessions.

We look forward to welcoming you to 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 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

Barry Ticho, M.D., PhD.,

Chief Medical Officer, Stoke Therapeutics, Inc.

As Chief Medical Officer Dr. Ticho is responsible for Stoke’s efforts to develop first-in-class RNA based disease-modifying medicines to treat severe neurologic genetic diseases. Prior to joining Stoke Barry was Head of R&D for Cardiovascular and Metabolic Diseases at Moderna Therapeutics.  He was previously Head of External R&D Innovation for Cardiovascular and Metabolic Diseases at Pfizer.  Prior to that he was Vice President of Clinical Development at Biogen where he led clinical development for the Tysabri program for MS and led the aducanumab program for Alzheimer’s Disease. Barry obtained his M.D. and Ph.D. degrees from the University of Chicago and completed Pediatrics training at Northwestern University and a Cardiology fellowship at Children’s Hospital in Boston. He was on clinical staff at Harvard Medical School and Massachusetts General Hospital. 

Rahul Ballal, PhD

President & CEO, Imara Inc.

Rahul has served as Imara’s President and Chief Executive Officer and as a member of the board of directors since June 2018. He has more than 15 years of life sciences experience in business development, strategic partnerships, venture capital, and company financing. Rahul joined Imara from Northern Biologics and Versant Ventures, where he served as Chief Business Officer and an Entrepreneur-in-Residence, respectively. At Northern Biologics, he built a first-in-class immuno-oncology portfolio, led a broad strategic partnership with Celgene, and managed several key functional roles at the executive level. At Versant, he negotiated foundational assets for existing portfolio and new companies, sourced several new deals, and participated in investment team activities.

Rahul was previously Vice President, Business Development at Flexion Therapeutics, where he led several business development transactions, supported multiple functions during Flexion’s initial public offering, and completed a key $175 million strategic partnership with Patheon UK. Prior to Flexion, he held business development and leadership positions at Novartis Venture Fund, Broad Institute of MIT and Harvard, and Redmind Inc. Rahul earned a B.S. in biology from Brown University, a M.S. in bioinformatics from Johns Hopkins University, and a Ph.D. in biochemistry from Georgetown University. He is also a Kauffman Fellow.

Christopher Dandrea

Vice President, Global Program Management, uniQure

Chris Dandrea is a 25 year veteran in the Biopharmaceutical Research, Development and Commercialization space.  His current role as uniQure’s Vice President of Global Program Management is focused on scale-up, acceleration and productivity of uniQure’s growing Research & Development Pipeline of CNS/Neuro and Rare Gene Therapy programs.  Prior to uniQure, Chris was Sanofi’s Head of Oncology Project Management tasked to build a next-generation project management organization to support rapid oncology portfolio growth.   Additional experience includes Rare Disease Program Leadership at Genzyme, Life Science Management Consulting, Medical Device Engineering and service as a US Naval Officer.  Education includes Engineering degrees from Duke University and an MBA from Carnegie Mellon University.  

Christine Von Raesfeld

Founder & CEO, People with Empathy

As Founder/CEO of People with Empathy, Christine Von Raesfeld is a leader in bringing a critically needed patient perspective to cutting-edge medical innovations. Committed to providing patients with chronic and rare diseases with the support they need, Christine works with patient advocacy organizations, industry representatives, and individual patients and their loved ones. Living with many rare and chronic diseases, she believes that the only way to ensure health equity is to leverage one’s own data. As a patient involved in Stanford’s Humanwide program, she speaks on the benefits and advantages of precision medicine, with a special interest in pharmacogenomics. 

Christine’s drive to make lives better for patients has been nationally recognized. In 2019, she spoke on stage at the Startup Health Festival as an invited guest of Sanguine Biosciences. In 2018, Christine served on the Team of Patient Advisors for PatientsLikeMe. She has been nationally recognized for her work in advocacy and when several awards including Silicon Valley Business Journal’s100 Women of Influence, 2019 and a 2020 Community Hero by Assemblyman Kansen Chu. In addition, Christine serves as a Virtual Advisor Team member for the All of Us research program, a Technical Expert Panelists for CMS and a patient advisor/consultant or multiple pharmaceuticals off branded campaigns and startup ventures.