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15th Orphan Drugs & Rare Diseases Global Congress 2021 Europe

Delivering Innovative Therapies to Patients with Rare Diseases

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13th - 15th September 2021 - Pre - Conference Workshop + 2 Days Conference
The Chelsea Harbour Hotel, Chelsea Harbour Dr, London SW10 0XG, UK

Pharma Conference London UK

15th Orphan Drugs & Rare Diseases Global Congress 2021

Paradigm Global Events is again proud to present our Orphan Drugs and Rare Diseases 2021 Europe. It’s the 15th in the series of our Flagship tri-annual Orphan Drugs and Rare Diseases event, this congress will provide you with a comprehensive overview of the critical issues shaping the future of Orphan Drugs.

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, pharmaceuticals, biotechs, non-profit organizations, orphan drugs developers, research organizations as well as patient groups and advocates. 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 access, commercialization, collaborations, policies, reimbursement, pricing, and more.

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Insights & Attendees

Previous Key Industry Experts Speakers

Doug Danison

Head of Europe, bluebird bio

Pedro Franco

Director for Global Regulatory & Scientific Policy (GRASP), Merck

Emily Crossley

Co-founder, Duchenne UK

Femida Gwadry-Sridhar

CEO and Founder, Pulse Infoframe Inc.

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

Orphan Drugs & Rare Diseases Global Congress 2021 – 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…

Commercialization, Current Trends and Collaborations

Commercialization, Current Trends and Collaborations

Access, Pricing and Reimbursement

Access, Pricing and Reimbursement

Innovations, Discovery and Clinical Development

Innovations, Discovery and Clinical Development

Advance Therapy Medicinal Products

Advance Therapy Medicinal Products

View Conference Agenda

Gold Sponsor

Gold Sponsor

Silver Sponsor

Lead Exhibitor

Associate Sponsor

Associate Sponsor

Sponsor & Exhibit
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Paradigm Global Events is again proud to present the  Orphan Drugs and Rare Diseases 2021 Europe. It’s the 15th in the series of our Flagship tri-annual Orphan Drugs and 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…

According to a report by Fortune Business Insights, the Global Orphan Drugs Market size will emerge from the 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 these 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 to 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 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 COVID19
  • 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
  • How the MHRA managing orphan medicinal products after Brexit?
  • 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 Advanced Therapy Medicinal Products
  • How Can the Developer and the Patient Assist in the Evolution and Development of Orphan Drugs to make it accessible to patients in a 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 Investorsm

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

Doug Danison

Head of Europe, bluebird bio

Doug Danison is the Head of Europe at bluebird bio since January 2021. He joined bluebird bio in July 2015 to establish the global pricing, market access, and health economics team known as Access Value & Evidence Strategy (AVES).

Doug has more than 20 years of Pricing & Market Access experience, including roles with Eli Lilly & Company, Amgen, Millennium/Takeda Oncology. In that time, he has led or overseen the preparation of market access strategies for products in early development, pivotal study planning, launch, and post-launch lifecycle management. While addressing market access challenges from a variety of vantage points including Global, European Regional, and U.S. roles he’s built teams, processes, and a knowledge base that enables the execution of market access strategies that anticipate and meet the needs of access decision-makers in an increasingly challenging market access environment.

Pedro Franco

Director for Global Regulatory & Scientific Policy (GRASP), Merck

Pedro Franco holds a degree in Pharmacy (PharmD) from the University of Lisbon, a Master in Organic Chemistry (M.Sc) from the New University of Lisbon with collaboration with Imperial College in London, and a Ph.D. in Orphan drugs and Rare diseases from the University of Sheffield. Pedro Franco completed also his music studies (Cello) in Lisbon.

Currently, Pedro is working as a Director for Global Regulatory & Scientific Policy (GRASP) at Merck, and he is mainly focused on regulatory intelligence, strategy, and policy. In addition, he is the liaison for the China and Latam region concerning regulatory and policy topics. Previously, He worked at the European Medicines Agency for 12 years in different roles and departments mainly focusing on regulatory affairs, international collaboration, assessment, authorisation of medicines, orphan drugs, paediatrics, peer review, quality, and strategy. Beforehand, he was working at INFARMED (the Portuguese national competent authority) as a pharmaceutical assessor. Before joining regulatory competent authorities, he worked as a post-graduate research fellow in the area of toxicology, carcinogenic chemistry, and drug development.

Emily Crossley

Co-founder, Duchenne UK

Emily Crossley is the co-founder and joint CEO of Duchenne UK. She graduated from Oxford University with a degree in modern history and became a reporter and anchor for Channel 4 News and CNN International. After her eldest son was diagnosed with Duchenne Muscular Dystrophy (DMD), she set up the Duchenne Children’s Trust. The charity joined forces with Joining Jack and is now Duchenne UK. In 7 years Duchenne UK has committed £14 million to funding treatments and a cure for DMD. Emily helped to create the Duchenne Dash, an iconic 24-hour bike ride from London to Paris to raise money and awareness for DMD. This year the Duchenne Dash raised over £1.2 million. Emily has advocated on behalf of patients around the world. She has represented the patient community in meetings at the EMA, the MHRA, the FDA, and NICE. She has spoken at Parliamentary meetings and was praised in the House of Commons for the charity’s work, by the Minister for Life Sciences, George Freeman MP. She is a member of the MHRA patient group consultative forum.

Femida Gwadry-Sridhar,

CEO and Founder, Pulse Infoframe Inc.

Femida is a pharmacist, clinical epidemiologist, and methodologist with over 25 years of experience in clinical trials, patient registries, knowledge translation, health analytics, and clinical disease outcomes. Femida has received more than $10 million in research funding as a principal investigator or co-investigator. She was awarded a career scientist award in 2006 from the Canadian Institute of Health Research and through her work founded the first knowledge translation health informatics lab in North America, creating a collaborative ecosystem for interdisciplinary research. Recently recognized as a lifetime fellow of CIHR for her contributions to global research with impact.

Femida has published over 200 manuscripts and abstracts in top tier journals and actively continues her professional interests in enabling technologies for better provider workflow and patient engagement. Femida is a founding member of the International Society for Pharmacoeconomics and Outcomes Research medication adherence group, which has provided an early glimpse into how to conduct research in adherence.

She is an expert reviewer in North America and Europe on large scale research network grants and a frequent international speaker on patient engagement, patient registries, innovation, health informatics and data visualization. The recipient of numerous nominations, awards and positive press, Femida has joined Springboard Enterprises as a female entrepreneur in digital health. She also holds her Ph.D. in research methodology and health economics from McMaster University.