2020 AGENDA

 

Live, 24 - 27 August 2020

Schedule

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Aug 1709:00
Conference pass

Messenger RNA therapy for inherited metabolic diseases

Next Generation Therapies
Paolo Martini, Chief Scientific Officer, Rare Diseases, Moderna Therapeutics
Aug 1709:20
Conference pass

Patient-customized oligonucleotide therapy for ultra-rare genetic diseases

Next Generation Therapies
Timothy Yu, Attending Physician, Investigator, Division Of Genetics And Genomics, Boston Children's Hospital
Aug 1709:40
Conference pass

Hope Accelerates Here: A Nonprofit Biotech Model to Accelerate Ultra-Rare Disease Programs

Next Generation Therapies
Laura Hameed, Executive Director, The Columbus Children’s Foundation
Aug 1710:00
Conference pass

AAVS3 gene therapy aimed at normalizing Factor IX levels in patients with severe or moderately severe hemophilia B and providing a platform to treat other rare diseases

Next Generation Therapies
Aug 1710:20
Conference pass

Modifier gene therapy platform for inherited retinal diseases: one therapeutic for multiple diseases.

Next Generation Therapies
Rasappa Arumugham, Chief Scientific Officer, Ocugen
Aug 1710:40
Conference pass

Engineered Red Cells to Treat Metabolic Disorders

Next Generation Therapies
Christina Coughlin, Chief Medical Officer, Rubius Therapeutics
Aug 1711:00
Conference pass

Using Directed Evolution to Create Improved Gene Therapy Vectors

Next Generation Therapies
Casey Case, Senior Vice President Of Research And Development, 4D Molecular Therapeutics
Aug 1711:20
Conference pass

Advances in RNAi Therapeutics for Rare Diseases

Next Generation Therapies
Patrick Haslett, Executive Director Of Clinical Research And Biology, Alnylam Pharmaceuticals
Aug 1711:40
Conference pass

ADX-2191: A New Approach for Rare Proliferative Ocular Diseases

Next Generation Therapies
Todd Brady, Chief Executive Officer, Aldeyra Therapeutics
Aug 1712:00
Conference pass

Pitch 1: Development of RLS-0071 in Hypoxic Ischemic Encephalopathy

Pitch and Partner
Ulrich Thienel, Chief Executive Officer, ReAlta Life Sciences
Aug 1713:00
Conference pass

Pitch 7: Phase IB trial of Carboxyamidotriazole Orotate (Pravitinib™) in glioblastoma patients

Pitch and Partner
Rashida Karmali, CEO, Tactical Therapeutics Inc
Aug 1713:10
Conference pass

Pitch 8: Multi Characteristic Opsin for vision restoration in patients with retinitis pigmentosa

Pitch and Partner
Samarendra Mohanty, Founder And President, NanoScope Technologies
Aug 1713:20
Conference pass

Pitch 9: Targeting pancreatic cancer with Caflanone

Pitch and Partner
Ngeh Toyang, Chief Executive Officer, Flavocure Biotech
Aug 1713:30
Conference pass

Pitch 10: Processa Pharmaceuticals, Inc. – combining High Growth with Lower Risk

Pitch and Partner
David Young, Chief Executive Officer, Processa Pharmaceuticals, Inc.
Aug 1713:40
Conference pass

Pitch 11: Sensorion Innovative therapies to treat, prevent and restore within the field of hearing loss disorders

Pitch and Partner
Nawal Ouzren, Chief Executive Officer, Sensorion Pharma
Aug 1713:50
Conference pass

Pitch 12: Grace Therapeutics: Innovative Products for Orphan CNS Conditions

Pitch and Partner
Prashant Kohli, Vice President Commercial Operations, Grace Therapeutics
Aug 1714:00
Conference pass

Pitch 13: NTP42, an antagonist of the thromboxane receptor, attenuates experimentally induced pulmonary arterial hypertension (PAH)

Pitch and Partner
Therese Kinsella, Founder And Chief Scientific Officer, ATXA Therapeutics
Aug 1714:10
Conference pass

Pitch 14: Crossborder Orphan Drug Development: Differences in China and the US

Pitch and Partner
William Xiang, Director, Strategy And Operations, XW Laboratories
Aug 1714:20
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Pitch 15: Reversing the Warburg Effect for Glioblastoma Treatment

Pitch and Partner
Alan Cash, Chief Executive Officer And Co-Founder, MetVital
Aug 1714:30
Conference pass

Pitch 16: BSN175 for the treatment of Prader-Willi Syndrome

Pitch and Partner
Robert Lodder, President, Biospherics
Aug 1714:40
Conference pass

Pitch 17: Using machine learning to discover Pyronaridine for the orphan disease Ebola

Pitch and Partner
Sean Ekins, Founder And Chief Executive Officer, Collaborations Pharmaceuticals
Aug 1714:50
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Pitch 18: CLR 131 a novel targeted radiotherapeutic for the treatment of multiple myeloma, lymphoplasmacytic lymphoma, and pediatric neuroendocrine tumors, sarcomas and brain tumors

Pitch and Partner
Jarrod Longcor, Chief Business Officer, Cellectar Biosciences Inc
Aug 1715:00
Conference pass

Pitch 19: Salinomycin QUATRAMER for Small Cell Lung Cancer (SCLC

Pitch and Partner
Randy Milby, Chief Executive Officer, Hillstream Biopharma
Aug 1715:20
Conference pass

Pitch 21: Elements of creativity in orphan drug development

Pitch and Partner
Alon Ben-Noon, CEO, NeuroSense Therapeutics

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Aug 2408:30
Conference pass

Breaking down silos to further education and understanding of pan-tumor therapies

Advanced Therapies Clinical + Rare Oncology
Pooja Merchant, Head Of Patient Partnership, Engagement And Oncology, Bayer US LLC
Susan S, Advocate, Patient Advocate
Aug 2408:30
Conference pass

Best practices for venture philanthropy and industry partnership

Rare Disease Advocacy World
Debra Miller, CEO & Founder, CureDuchenne
Aug 2408:30
Conference pass

Developing AI capabilities to rapidly screen complex EMR datasets for rare disease clinical insights and patient identification

Digital Health and AI
Ranjit Kumble, Global Data Science Lead, Customer Analytics And Insights, Pfizer
Aug 2408:50
Conference pass

Tumor-agnostic therapies, from clinical trials to drug registration: a case study

Advanced Therapies Clinical + Rare Oncology
Dmitry Petrov, Head Of Medical Writing And Pharmacovigilance, ICRC-Weyer GmbH
Aug 2408:50
Conference pass

Key Role for Patient Advocacy Groups in Rare Disease Drug Development: Making Better Use of This Valuable Resource

Rare Disease Advocacy World
Raymond Huml, Vice President of Medical and Scientific Strategy, Head, Health’s Rare Disease Consortium, Syneos Health
Aug 2408:50
Conference pass

AI in rare diseases and personalized medicines - how diagnostic signals identified from electronic health data can help patients receive earlier diagnosis and treatment

Digital Health and AI
Christopher Rudolf, Founder And Chief Executive Officer, volv global
Aug 2409:10
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PANEL: Continuing innovation and partnership for rare cancer drug development

Advanced Therapies Clinical + Rare Oncology
Moderator: Dmitry Petrov, Head Of Medical Writing And Pharmacovigilance, ICRC-Weyer GmbH
Jim Palma, Executive Director, TargetCancer Foundation
Kristin Anthony, President, Pten Hamartoma Tumor Syndrome Foundation
Anna Shurshalina, Global Safety Lead, Safety Physician, Oncology. Benefit Risk Management, Pharmacovigilance, Bayer Pharmaceuticals
Abby Sandler, Executive Director, Mypart, Center For Cancer Research,, National Institutes of Health
Aug 2409:10
Conference pass

PANEL: Stronger Together during COVID-19: Continuing industry and patient partnerships along the full continuum of development, from pre-clinical through commercialization

Rare Disease Advocacy World
Ron Bartek, President, Director, And Founder, Friedreich's Ataxia Research Alliance
Matt Flesch, Vice President Of Communications And Patient Advocacy, Horizon Therapeutics
Michael Hanley, Chief Commercial Officer, Aeglea BioTherapeutics
Kim Stephens, President, Project Alive
Debbie Drell, Director Of Membership, National Organization for Rare Disorders (NORD)
Jenn Mcnary, Founder, One Rare
Aug 2409:10
Conference pass

PANEL: Hospital and Industry innovation in patient Identification for rare diseases

Digital Health and AI
Marius George Linguraru, Principal Investigator at Children’s National Health System, Professor of Radiology, Pediatrics and Biomedical Engineering, The George Washington University
Younos Abdulsattar, US Vyndaqel Medical Team Lead, Rare Disease, Pfizer
Christopher Rudolf, Founder And Chief Executive Officer, volv global
Aug 2410:05
Conference pass

Chairperson’s opening remarks

Keynotes
Ellen Coleman, President And Chief Executive Officer, VOZ Advisors
Aug 2410:10
Conference pass

Keynote Address: Rare diseases in the time of Covid-19

Keynotes
Jeremy Levin, Chairman and Chief Executive Officer, Chairman, BIO, Ovid Therapeutics
Aug 2410:35
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Keynote Address: The future of rare diseases: developing and accessing the next generation of therapies

Keynotes
Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical
Aug 2410:55
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Keynote Panel: Patient involvement in drug development for rare diseases – examining how patients are driving meaningful development and what companies are doing to incorporate patient input throughou

Keynotes
Neena Nizar, President, The Jansens Foundation
Jessica Scott, Head of R&D Patient Engagement Office,, Takeda Pharmaceuticals
Amy Mckee, Vice President, Regulatory, Parexel Consulting, Parexel International
Dara Richardson-Heron, Chief Patient Officer, Pfizer
Yann Le Cam, Chief Executive Officer, EURORDIS
Aug 2412:30
Conference pass

FDA update: real world evidence in place of control arm studies for advanced therapies clinical trial designs

Advanced Therapies Clinical + Rare Oncology
Wilson Bryan, Director, Office Of Tissues And Advanced Therapies, Cber, FDA
Aug 2412:30
Conference pass

Non-profit drug discovery enabling platforms vs advocacy organizations - challenges in pharma perception of foundations

Rare Disease Advocacy World
Annette Bakker, President, Childrens Tumor Foundation
Aug 2412:30
Conference pass

Open Source Imaging Consortium against Fibrosing Interstitial Lung Diseases (ILD) - setting up business and legal framework for success

Digital Health and AI
Zheng Yang, Head Of Technology And Data Innovation, Customer Value, Boehringer Ingelheim
Aug 2412:50
Conference pass

Creating Commercial-ready & Scalable Solutions for Gene Therapy Development & Manufacturing

Advanced Therapies Clinical + Rare Oncology
Thomas VanCott, Chief Technology Strategy Officer at Catalent Biologics, Catalent Cell & Gene Therapy
Aug 2412:50
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Redefining Rare: Health Journeys for millions of rare disease patients to unlock medical discoveries

Rare Disease Advocacy World
Brian Loew, CEO, Inspire
Aug 2412:50
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Examining industry strategies for leveraging patient data and AI to advance rare disease patient identification, R&D, and commercialization

Digital Health and AI
Oodaye Shukla, Chief Data Officer, HVH Precision Analytics, now EVERSANA
Aug 2413:10
Conference pass

PANEL: Continuing clinical trials during COVID - rethinking trial designs, data collection and regulatory submissions

Advanced Therapies Clinical + Rare Oncology
Magali Taiel, CMO, GenSight Biologics
Jeffrey Terkowitz, VP, Product, Inspire
Tim Turnham, Vice President, VOZ Advisors
Genevieve Laforat, Vice President, Clinical Development, Aspa Therapeutics/BridgeBio Gene Therapy
Aug 2413:10
Conference pass

PANEL: Transforming rare disease drug discovery models through open science and artificial intelligence

Digital Health and AI
Moderator: Oodaye Shukla, Chief Data Officer, HVH Precision Analytics, now EVERSANA
Zheng Yang, Head Of Technology And Data Innovation, Customer Value, Boehringer Ingelheim
Hubert Misztela, Machine Learning Expert At Novartis, Lecturer At Dublin Business School, Novartis
Aug 2413:20
Conference pass

PANEL: Moving treatments from lab to trial – what are the current barriers and how are patient organizations overcoming these challenges and collaborating with partners

Rare Disease Advocacy World
Amy Gray, Chief Executive Officer, Charcot-Marie-Tooth Association
Sharon Hesterlee, Executive Vice President, Chief Research Officer, Muscular Dystrophy Association
Claudia Hirawat, Executive Chairman, VOZ Advisors
Lianna Orlando, Senior Director Of Research, CureDuchenne
Aug 2414:10
Conference pass

ROUNDTABLE 1: Balancing the Patient Experience & ROI – understanding when to incorporate patient accommodations into study programs and balancing short and long term financial goals

Keynotes
Amanda Finlayson, Therapeutic Strategy Lead, ClinEdge
Aug 2414:10
Conference pass

ROUNDTABLE 2: Forecasting & epidemiology - rare-disease epidemiology methods and resources to support commercial forecasts

Keynotes
David Lapidus, President, Lapidus Data
Aug 2414:10
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ROUNDTABLE 3: Expanded Access Programs- How EAPs can complement rare disease drug development and optimize commercial launch

Keynotes
Sam Lucas, SVP, Expanded Access Programs, Durbin
Julia Campbell, VP, Client Strategies and Services, Durbin
Todd Galles, VP, US Business Development, EAPs, Durbin
Aug 2414:10
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ROUNDTABLE 4: FDA updates - Office of Orphan Product Development

Keynotes
Janet Maynard, Director, Office Of Orphan Products Development, Food And Drug Administration, U.S. Food and Drug Administration
Lewis Fermaglich, Medical Advisor, Office of Orphan Products Development,, FDA
Aug 2415:20
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Translating gene therapies from academia to industry - The need to create a common standard for development, vernacular and data quality to ensure faster regulatory approvals

Advanced Therapies Clinical + Rare Oncology
Gavin Corcoran, Chief Research And Development Officer, Axovant Sciences
Aug 2415:20
Conference pass

Rare Disease International: Is it time for global access? If not now, when?

Rare Disease Advocacy World
Durhane Wong-Rieger, President And Chief Executive Officer, Canadian Organization For Rare Disorders
Aug 2415:20
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Leveraging AI for discovery and repurposing drugs— making data valuable through interdisciplinary collaboration and rethinking business models for rare diseases and cancer in the age of AI

Digital Health and AI
Panna Sharma, President And Chief Executive Officer, Lantern Pharma
Aug 2415:40
Conference pass

Regulatory considerations in getting the first RNAi therapy approved - case study of ONPATTRO

Advanced Therapies Clinical + Rare Oncology
Andrew Slugg, Senior Vice President Of Regulatory Affairs, Alnylam Pharmaceuticals
Aug 2415:40
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Payer engagement strategy – relationship building and bridging information gaps to ensure access for cystic fibrosis patients

Rare Disease Advocacy World
Jacqueline Erdo, Senior Manager, Policy & Advocacy,, Cystic Fibrosis Foundation
Aug 2416:00
Conference pass

In Clinic, At Home, and Are We Missing Something Else?

Advanced Therapies Clinical + Rare Oncology
Michael Murphy, Chief Medical & Scientific Officer, Worldwide Clinical Trials
Laurie Witherwax, Sr Vice President, Site Activation, Worldwide Clinical Trials
Aug 2416:00
Conference pass

The importance of patient engagement in the age of ICER

Rare Disease Advocacy World
Terry Wilcox, Co-Founder And Executive Director, Patients Rising & Patients Rising
Aug 2416:20
Conference pass

Feasibility of targeting multiple diseases in one clinical trial using a platform vector gene therapy approach

Advanced Therapies Clinical + Rare Oncology
Philip J Brooks, Program Director, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)
Aug 2416:20
Conference pass

The burden of access on patients and families

Rare Disease Advocacy World
Mindy Cameron, Advocacy Director, Little Hercules Foundation
Aug 2417:00
Conference pass

Keynote Panel: Examining industry strategies for leveraging patient data and AI to advance rare disease patient identification, R&D, and commercialization

Keynotes
Moderator: Oodaye Shukla, Chief Data Officer, HVH Precision Analytics, now EVERSANA
Thomas Abbott, Head, Real World Data And Evidence, Astellas Pharma
Sonalee Agarwal, Vice President Of Value And Evidence Strategy, Alnylam Pharmaceuticals
Sean Khozin, Global Head Of Data Strategy, Johnson & Johnson

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Aug 2508:30
Conference pass

The challenges of bringing gene therapy treatments for rare disease to clinical trial

Advanced Therapies - Manufacturing
Scott Dorfman, CEO, Odylia Therapeutics
Aug 2508:30
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Leverage digital tools to meet gaps in genetic workforce and supporting diagnosis in rare diseases

Diagnosis
Marshall Summar, Division Chief Of Genetics And Metabolism, Children's National Medical Center
Aug 2508:50
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Following patients with rare and orphan diseases from clinical trials to market

Clinical Development & Regulatory
Leanne Larson, Corporate Vice President and WW Head, Real-World Evidence, Parexel
Aug 2508:50
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Preparing for Success: Early Development Considerations for Orphan Drug Commercialization

Advanced Therapies - Manufacturing
Cornell Stamoran, Vice President of Corporate Strategy, Catalent Pharma Solutions
Aug 2508:50
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Role of biomarkers in the diagnosis of rare hereditary diseases

Diagnosis
Peter Bauer, Chief Genomic Officer, CENTOGENE US, LLC
Aug 2509:10
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PANEL: Science-driven and patient-focused: Rare disease patient and caregiver Inclusion in early therapeutic discovery

Clinical Development & Regulatory
Luke Rosen, Vice President, Accelerated Development & Patient Engagement, Ovid Therapeutics
Andrea Furia-Helms, Director, Patient Affairs Staff, U.S. Food and Drug Administration
Sarah Glass, Senior Director, Translational Medicine, Head of Rare Diseases, Parexel
Mary Mcgowan, Executive Director, The Myositis Association
Andrea Furia-Helms, Director, Patient Affairs Staff, U.S. Food and Drug Administration
Aug 2509:10
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AVS3 gene therapy aimed at normalizing Factor IX levels in patients with severe or moderately severe hemophilia B and providing a platform to treat other rare diseases

Advanced Therapies - Manufacturing
Aug 2509:10
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PANEL: Future outlook and initiatives in advancing Newborn Screening for rare diseases

Diagnosis
Melissa Wasserstein, Chief, Division Of Pediatric Genetic Medicine, Department Of Pediatrics,, Montefiore Medical Center
Dean Suhr, President, MLD Foundation
Allison May Rosen, Representative, Firefly Fund
Pam Crowley Andrews, Co-Founder And Executive Director, Firefly Fund
Pat Furlong, Founding President And Chief Executive Officer, Parent Project Muscular Dystrophy
Emily Fields, Senior Manager, Patient Advocacy, bluebird bio
Aug 2510:05
Conference pass

Chair available for sponsorship

Keynotes
If you are interested in sponsoring this session, contact Taylor Coate now at +1 (646) 619-1838 ortaylor.coate@terrapinn.com
Aug 2510:10
Conference pass

Keynote Fireside Chat: Gene therapy: the future is now

Keynotes
Moderator: Sarah Dash, Chief Executive Officer, Alliance For Health Policy
Bobby Gaspar, Chief Executive Officer, Orchard Therapeutics
Aug 2510:35
Conference pass

Keynotes Fireside Chat: What’s the takeaway - How can rare diseases benefit from the regulatory and scientific innovation seen in COVID-19 vaccine development?

Keynotes
Moderator: Christopher Austin, Director, National Center For Advancing Translational Sciences (Ncats), National Institutes of Health (NIH)
Peter Marks, Director, Center For Biologics Evaluation And Research (CBER), U.S. Food and Drug Administration
Debra Miller, CEO & Founder, CureDuchenne
Aug 2511:10
Conference pass

Keynote Address: KYMRIAH case study: what it takes to bring an organization from clinical to commercial scale for vector manufacture

Keynotes
Nick Page, Chief Operations Officer, Oxford Biomedica
Aug 2512:30
Conference pass

Using an integrated digital model for patient finding in rare disease trials

Clinical Development & Regulatory
Peter Payne, Vice President, Head of the Digital Research Network, Optum
Aug 2512:30
Conference pass

Pfizer’s global gene therapy business – a case study on building end-to-end capabilities and Scalable manufacturing capacity

Advanced Therapies - Manufacturing
Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer
Aug 2512:30
Conference pass

Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease -Progress Update

Diagnosis
Linn Parrish, Global Patient & Scientific Affairs, Chief Medical Office,, Takeda
Aug 2512:50
Conference pass

Next generation human enzymes: Innovative solutions for patients with rare diseases

Clinical Development & Regulatory
Anthony Quinn, President And Chief Executive Officer, Aeglea BioTherapeutics
Aug 2512:50
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Nucleic Acid Therapeutics: quality & regulatory considerations

Advanced Therapies - Manufacturing
Tracy Meffen, VP, Quality & Regulatory Affairs,, Genevant Sciences
Aug 2512:50
Conference pass

Progress in diagnosing rare disease patients leveraging natural language processing

Diagnosis
Tom Defay, Senior Director, Research And Development, Strategy And Alliance Management, Alexion Pharmaceuticals
Aug 2513:10
Conference pass

Rare Disease Recruitment: Addressing enrollment hurdles and differentiating your clinical trial in an increasingly competitive research environment

Clinical Development & Regulatory
Jonathan Kornstein, Executive Director, Program Strategy, Premier Research
Aug 2513:10
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Analytical requirements for investigational new drug (IND) and biologic license application (BLA) submissions

Advanced Therapies - Manufacturing
Joseph Lee, Executive Director, PTC Therapeutics
Aug 2513:10
Conference pass

Rare disease patient identification and diagnosis – accelerating the process

Diagnosis
Marisa Ricci, Vice President Patient Diagnosis Programs And U.S. Field Medical Engagement, Ultragenyx Pharmaceutical
Aug 2513:30
Conference pass

Updates from FDA on Rare Disease Clinical Trial Design and incorporation of patient voice

Clinical Development & Regulatory
Peter Stein, Director, Office Of New Drugs (Ond), Cder, Food and Drug Administration (FDA)
Aug 2513:30
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Rare disease patient identification and diagnosis – accelerating the process

Diagnosis
Marisa Ricci, Vice President Patient Diagnosis Programs And U.S. Field Medical Engagement, Ultragenyx Pharmaceutical
Aug 2514:10
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ROUNDTABLE 1: The patient journey - improving clinical trial performance and delivering better study outcomes through a personalized approach to the patient experience

Keynotes
Scott Gray, CEO, Clincierge
Bjorn Wester, Global Client Services Manager, Clincierge
Dana Schoknecht, Life Sciences Manager, MUV People
Aprel Gandy, Global Strategic Program Manager – Health & Life Science, Travel Leaders Corporate
Aug 2514:10
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ROUNDTABLE 2: Patient-Focused Drug Development – how to get started

Keynotes
Meagan Spychala, Assistant Vice President Of Patient Engagement And Program Strategy, Rho, Inc
Aug 2514:10
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ROUNDTABLE 3: Patient, advocate, caregiver (PAC) and industry collaboration: Going Beyond Clinical Trial Design

Keynotes
Laura Wuerth, Vice President, rareLife solutions
Terri Klein, President And Chief Executive Officer, National M.P.S. Society
Peter Snyder, Medical and Scientific Publications, rareLife solutions
Dakota Fisher-Vance, Patient Advocacy Manager, Horizon Therapeutics
Rob Colenso, Senior Director, Product Management, rareLife solutions
Mukund Nori, Director, Medical Affairs & Principal Med, rareLife solutions
Aug 2514:10
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ROUNDTABLE 4: Virtualization of Clinical Trials, Registries and Long-term Follow-up in Rare Diseases: Perspectives of Patients and Industry

Keynotes
Paulla Dennis, Director, Rare Disease and Pediatrics,, Covance
Frederick Derosier, Executive Director, Rare Diseases, Covance
Aug 2515:20
Conference pass

Working with regulators on Real World Evidence to support clinical trial development in the absence of control groups

Clinical Development & Regulatory
Mike Hale, Vice President And Chief Statistical Scientist, Takeda Pharmaceuticals
Aug 2515:20
Conference pass

Commercializing advanced therapies for a rare population – the bluebird bio experience

Advanced Therapies - Manufacturing
Alison Finger, Chief Commercial Officer, bluebird bio
Aug 2515:20
Conference pass

Newborn whole-genome sequencing: A potential solution to democratizing the diagnosis

Diagnosis
Khrystal K Davis, President, Texas Rare Alliance
Aug 2515:40
Conference pass

A new era of clinical trials informed by patient focused clinical reported outcome measures and Real World Evidence

Clinical Development & Regulatory
Moderator: Nach Dave’, Vice President, Development Strategy, Premier Research
Allyson Berent, Chief Operating Officer, Genetx Biotherapeutics, Chief Science Officer, Foundation for Angelman Syndrome Therapeutics
Melissa Hogan, Rare Disease Parent/Advocate, CEO, Doulots
Alison Skrinar, Vice President, Clinical Outcomes Research And Evaluation, Ultragenyx Pharmaceutical
Aug 2515:40
Conference pass

Succeeding with Star Wars medicine: translating the science and value of gene therapy to key stakeholders

Advanced Therapies - Manufacturing
Gavin Jones, Director of Rare Disease, OPEN Health
Emily Crossley, Co-Founder, Duchenne U.K.
Betsy Bogard, Programme Leader, Early Development, Avrobio
Aug 2515:40
Conference pass

Genomic Analysis in a Prenatal Setting

Diagnosis
Andrea Haworth, Head of Clinical Services, Congenica
Aug 2516:00
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PANEL: Shared biology: increasing access to approved therapies for rare disease patients with shared biology through accurate diagnosis, criteria for inclusion, and exploring a regulatory path for app

Diagnosis
Christina Waters, Chief Executive Officer And Founder, RARE Science
Sameer Zuberi, Consultant Paediatric Neurologist, President, European Paediatric Neurology Society, Royal Hospital for Children,, National Health Service
Nancy Spinner, Chief of the Division of Genomic Diagnostics, The Children's Hospital of Philadelphia
Allison Watson, Co founder/Secretary, Ring20 Research and Support UK
Aug 2516:20
Conference pass

Integrating a patient-first mindset in the development and commercialization of gene therapies

Advanced Therapies - Manufacturing
Parag Meswani, SVP, Commercial, Axovant Gene Therapies
Aug 2516:40
Conference pass

Ex-US commercial strategy for gene therapies – considerations for country specific regulation, supply chain & site set up

Advanced Therapies - Manufacturing
Anny Bedard, Head Of International Business, Commercial Operations, Sarepta Therapeutics
Aug 2516:50
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Keynote: Announcing the Winners of the 2020 NCATS Rare Diseases Are Not Rare! Challenge

Keynotes
Alice Chen Grady, Program Officer, Office of Rare Diseases Research, NIH/NCATS

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Aug 2608:30
Conference pass

Creating successful patient-centric rare disease global marketing & commercial teams

Commercial
Bert Bruce, Vice President, Rare Disease Commercial Development, Pfizer
Aug 2608:30
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European orphan regulation - awarding meaningful innovation for unmet medical needs

Payers Seminar
Evert Jan Van Lente, Director Eu-Affairs, AOK Bundesverdand
Aug 2608:30
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How regulations are helping to further liberate data to increase access for patients and developers

Patient Data Seminar
Avinash Shanbhag, Director, Nationwide Health Information Network Division, U.S. Department of Health & Human Services
Aug 2608:50
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Orphan drug launches: what are the key drivers for success?”

Commercial
Simone Seiter, Partner, Simon-Kucher & Partners
Aug 2608:50
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Pricing, profitability and patient access to rare disease medicines

Payers Seminar
Adam Hutchings, Director, Dolon
Aug 2608:50
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PANEL: The need for machine readable data, policies around data sharing, and a federated data system for rare diseases

Patient Data Seminar
Moderator: Christina Waters, Chief Executive Officer And Founder, RARE Science
Lynsey Chediak, Project Lead, Precision Medicine, World Economic Forum
Peter Goodhand, Chief Executive Officer, Global Alliance for Genomics & Health
Arthur Herman, Fellow, World Economic Forum, Precision Medicine Portfolio, Principal Strategic Consultant, Kaiser Permanente
Aug 2608:50
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Sponsored Session by Syneos Health

Patient Data Seminar
More information will be available soon
Aug 2609:10
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PANEL: Continuing commercial planning & operations during COVID-19

Commercial
Moderator: Simone Seiter, Partner, Simon-Kucher & Partners
Vikram Karnani, Executive Vice President, Chief Commercial Officer, Horizon Therapeutics
Rachelle Jacques, Chief Executive Officer, Enzyvant
Wolfram Lux, Senior Director, Simon-Kucher & Partners
Sujay Kango, Chief Commercial Officer, Acceleron Pharma
Aug 2609:10
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PANEL: Sustainability of orphan medicines in Europe: what does the rare disease payer landscape look like post-Covid19?

Payers Seminar
Moderator: Adam Hutchings, Director, Dolon
Francis Arickx, Head Of The Directorate Reimbursement Of Medicines And Pharmaceutical Policy, NIHDI - INAMI Belgium
Nuriya Musina, Head Of Development, Federal State Budgetary Institution, The Center For Healthcare Quality Assessme, Ministry of Health Russian Federation
Evert Jan Van Lente, Director Eu-Affairs, AOK Bundesverdand
Anna Bucsics, Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)
Aug 2610:05
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Keynote chairperson opening remarks

Keynotes
Scott Gray, CEO, Clincierge
Aug 2610:10
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Keynote Address: Pricing one-time treatments based on value – impact on patients, the health care system and society

Keynotes
David Lennon, President, AveXis
Aug 2610:35
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Keynote Fireside Chat: Industry perspective on value and access strategies – developing a cohesive agenda and unified framework for orphan drugs

Keynotes
Moderator: Jim Lang, Chief Executive Officer, EVERSANA
Bhash Parasuraman, Vice President Of Business Unit Lead, Rare Disease, Patient And Health Impact, Pfizer
Aug 2611:05
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Keynote Panel: Successfully navigating a new era: outlooks on future access to treatments, cures and clinical trials for rare disease patients

Keynotes
Moderator: Sophie Schmitz, Managing Partner, Partners4Access
Diane Berry, Vice President, Global Health Policy And Government Affair, Sarepta Therapeutics
Troyen Brennan, Executive Vice President And Chief Medical Officer, CVS Health
Sheela Upadhyaya, Rare Disease Expert, National Institute for Health and Care Excellence
John Crowley, Chief Executive Officer, Amicus Therapeutics Inc
Peter Saltonstall, Chief Executive Officer, National Organization for Rare Disorders (NORD)
Aug 2612:30
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The Promise of Genomic Medicines: The Path to the Patient

Commercial
Sandy Macrae, President And Chief Executive Officer, Sangamo Therapeutics
Aug 2612:30
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Navigating privacy in a data driven world: perspectives, studies, and future considerations among the rare disease community

Patient Data Seminar
Rachele Hendricks-Sturrup, Health Policy Lead, Future of Privacy Forum
Aug 2612:50
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Case Study: SPINRAZA in SMA - broadening access to new populations through real world evidence and data generation

Pricing and Reimbursement
Loukas Xaplanteris, Director Eu+ Value And Market Acces, Biogen
Eelko Den Breejen, Associate Director, Market Access, Biogen
Aug 2612:50
Conference pass

Reserved for Syncrogenix

Patient Data Seminar
More information will be available soon!
Aug 2613:10
Conference pass

Bringing gene therapies to patients through unique funding and unified mission

Commercial
Frederic Revah, Chief Executive Officer, Genethon
Aug 2613:10
Conference pass

PANEL: Bridging the evidence gap for approval - Examining recent cases of real world evidence for reimbursement

Pricing and Reimbursement
Moderator: Andrew Hobbs, Managing Director, Huron Consulting Group
Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
Shelby Corman, Executive Director and Lead, Real-World Evidence/Data Analytics Center of Excellence, OPEN Health
Brian Newkirk, Market Access Director, Alnylam Pharmaceuticals
Marianne Hamilton Lopez, Research Director, Duke Margolis Center for Health Policy
Francis Arickx, Head Of The Directorate Reimbursement Of Medicines And Pharmaceutical Policy, NIHDI - INAMI Belgium
Aug 2613:10
Conference pass

Having the end in mind when collecting data - considerations for working with your patient community, international data privacy laws and ultimately sharing with external stakeholders to further drug

Patient Data Seminar
Moderator: Monica Weldon, President/CEO, Bridge The Gap Syngap Education And Research Foundation
Pamela Mace, President, Fibromuscular Dysplasia Society of America
Rob Burley, Director Of Campaigns, Care And Support, The Muscular Dystrophy U.K.
Salvatore La Rosa, Chief Scientific Officer, Children's Tumor Foundation
Dimitar Yonchev, Patient Advocate Representative, Pro Retina Deutschland e.V.
Aug 2614:10
Conference pass

ROUNDTABLE 1: Guided by patients – how, when, and why to build authentic partnerships with patients and patient organizations

Keynotes
Scott Schliebner, SVP, Scientific Affairs, PRA Health Sciences
Aug 2614:10
Conference pass

ROUNDTABLE 2: Access in Europe – best practices for advanced therapies launch

Keynotes
Akshay Kumar, Partner, Partners4Access
Aug 2614:10
Conference pass

ROUNDTABLE 3: Physician profiling – identifying physicians and health care organizations who treat or (mis)diagnose rare diseases

Keynotes
Mark Kroes, Chief Executive Officer, Global Data Support
Aug 2614:10
Conference pass

ROUNDTABLE 4: Nonclinical toxicology and real-world epidemiology essential for a successful rare disease product launch

Keynotes
Jon Fryzek, Practice Director, Epidemiology, EpidStrategies, A Division of ToxStrategies
Lauren Bylsma, Senior Epidemiologist, EpidStrategies, A Division of ToxStrategies
Kirsten Mease, Managing Scientist, ToxStrategies
Marcie Wood, Senior Managing Scientist, Practice Director, Biopharmaceutical/Pharmaceutical, ToxStrategies
Aug 2614:10
Conference pass

ROUNDTABLE 5: Rare Diseases - Leveraging Data to Address Patient Scarcity in Clinical Trials

Keynotes
More information will be available soon.
Sheila Diamond, Scientific Business Development, Medidata, a Dassault Systèmes company
Alicia Staley, Senior Director, Patient Engagement, Medidata, a Dassault Systèmes company
Aug 2614:10
Conference pass

ROUNDTABLE 6: Patient and provider experiences – a tailored tech + talent approach to enhance the journey

Keynotes
Jan Nielsen, Division President, Patient Solutions, AssistRx Patient Solutions
Aug 2615:10
Conference pass

BREAK

Keynotes
BREAK
Aug 2615:40
Conference pass

Real World Evidence Generation in Orphan Drug Development

Commercial
Kui Huang, Rare Disease Epidemiology Lead Strategist, Pfizer
Aug 2615:40
Conference pass

PANEL: What is fair? A discussion on the perceptions of fairness in pricing for treatments of rare diseases and curative therapies

Pricing and Reimbursement
Moderator: Charles River Associates
Moderator: Andrew Parece, Vice President, Charles River Associates
Will Richmond, Lead, Value And Access, Sanofi Genzyme
Jose F. Gomez, SVP, Global Market Access & Value, Avrobio
Craig Wakeford, Director, Global Value And Access, Biogen
Aug 2615:40
Conference pass

Payment Models: Practical learnings & potential solutions from the launch of the first gene therapy in the US

Pricing and Reimbursement
Jay Newman, Senior Vice President and General Manager, Head Of Us Commercial, Spark Therapeutics
Aug 2615:40
Conference pass

Rare Disease Cures Accelerator (RDCA-DAP) – standardizing data quality and harmonizing with regulators

Patient Data Seminar
Vanessa Boulanger, Director Of Research Programs, NORD
Aug 2616:00
Conference pass

Novel applications of RWE in rare disease epidemiology

Commercial
Alexander Cole, Executive Director, Global Head Epidemiology, Alexion
Aug 2616:00
Conference pass

New Findings: U.S-based payer, physician and patient input on fair pricing

Pricing and Reimbursement
Andrew Parece, Vice President, Charles River Associates
Matthew Majewski, Vice President, Charles River Associates
Aug 2616:00
Conference pass

CureDuchenne Biobank - Integrating Patient Data Partnerships with Stakeholder Engagement

Patient Data Seminar
Romina Foster-Bonds, Programs Director, CureDuchenne
Aug 2616:20
Conference pass

Reserved for Cambrex

Patient Data Seminar
More information will be available soon
Aug 2616:40
Conference pass

What can industry do to further empower organizations to collect patient-centered data

Patient Data Seminar
Ali Mohamadi, Executive Director, BioMarin Pharmaceutical Inc.

Create your personal agenda –check the favourite icon

Aug 2709:00
Conference pass

MENA: Working with multiple stakeholders in the region to enable access to treatment for rare diseases patients

Global Market and Patient Access
Rania Ashraf, Associate Director Market Access, Janssen
Aug 2709:00
Conference pass

Addressing value and patient access for one-time potentially curative treatments

Pricing and Reimbursement
Francis Pang, Vice President Global Market Access, Orchard Therapeutics
Aug 2709:00
Conference pass

Panel: How are pharma and biotech companies evaluating new opportunities in the rare disease space – understanding the science, patient populations

Pitch and Partner
Weiyong Sun, Senior Director, Specialty Medicine Search, Evaluation And Global Business Development, Daiichi Sankyo Co Ltd
Daniel Curran, Head, Rare Diseases Therapeutic Area Unit, Takeda
Joana Granado, External Innovation Director, Actelion - A Janssen Pharmaceutical Company of Johnson&Johnson
Thomas Kassberg, Chief Business Officer And Executive Vice President, Ultragenyx Pharmaceutical
Aug 2709:20
Conference pass

Bringing advanced therapies to novel territories: expanding patient access in low to middle income countries

Global Market and Patient Access
Derek Ansel, Director, Rare, Orphan and Pediatric Diseases, Synteract
Aug 2709:20
Conference pass

Planning a successful cell & gene therapy launch in Europe in an ever-changing landscape for transformative therapies

Pricing and Reimbursement
Sophie Schmitz, Managing Partner, Partners4Access
Aug 2709:40
Conference pass

PANEL: How to further access to diagnostic programs and therapeutics in emerging rare disease markets

Global Market and Patient Access
Rania Ashraf, Associate Director Market Access, Janssen
Samuel Agyei Wiafe, Executive Director, Rare Disease Ghana Initiative
Nataliya Shandarovska, patient, patient advocate, senior staff nurse, National Alliance for Rare Diseases Support - Malta
Edmund Lim, Abc, We CARE Journey
Aug 2709:40
Conference pass

PANEL: - Title to be determined

Pricing and Reimbursement
Thayer Surette, Deputy Director, Partnership to Improve Patient Care (PIPC)
Martin Zagari, Vice President Global Health Economics, Amgen Inc
Francis Pang, Vice President Global Market Access, Orchard Therapeutics
Omar Dabbous, Vice President, Health Economics And Outcomes Research, AveXis
Aug 2710:50
Conference pass

Keynote Panel: Future of digital medicine & access to care – what has COVID taught us and what hurdles still need to be addressed?

Keynotes
Marshall Summar, Division Chief Of Genetics And Metabolism, Children's National Medical Center
Durhane Wong-Rieger, President And Chief Executive Officer, Canadian Organization For Rare Disorders
Aug 2712:20
Conference pass

Changes to ICER’s Value Framework for 2020, and Relevance for Cures and Treatments of Ultra-Rare Diseases

Pricing and Reimbursement
Rick Chapman, Director Of Health Economics, ICER
Aug 2712:20
Conference pass

Investors Panel: Evaluating investment opportunities in the rare disease & orphan space

Pitch and Partner
Moderator: Ken Kengatharan, Managing Partner, Atheneos Ventures And Chief Executive Officer, Atheneos Ventures
Anjan Aralihalli, Venture Partner, C.T.I. life Sciences Fund
Jeffrey Schwartz, Managing Director, Bain Capital Life Sciences
Daniel O'Mahony, Partner, Seroba Life Sciences
Art Pappas, Managing Partner, Pappas Capital
Aug 2712:40
Conference pass

EUROPE: Update on the current review of orphan drug incentives in Europe – what could change and how would these changes impact drug developers, patient access and overall business models

Global Market and Patient Access
Bernard Grimm, Director, EuropaBio
Aug 2712:40
Conference pass

Session will be hosted by EVERSANA

Pricing and Reimbursement
Speaker will be announced soon
Aug 2713:00
Conference pass

PANEL: Use of Real world evidence in HTA review of orphan products

Pricing and Reimbursement
Trevor Richter, Director, Common Drug Review And Optimal Use, CADTH
Jennifer Bright, Executive Director, Innovation And Value Initiative
Rick Chapman, Director Of Health Economics, ICER
Aug 2713:20
Conference pass

JAPAN: Update on rare disease opportunities and barriers in Japan

Global Market and Patient Access
Yukiko Nishimura, President, ASrid Japan
last published: 25/Aug/20 16:55 GMT

 

Get Involved At World Orphan Drug Congress USA

 

To Sponsor Or Exhibit

 
 

Justin Franks
justin.franks@terrapinn.com
t/ +1 914 819 3506

 

 

 

To Speak

 
 

Claire Murphy
claire.murphy@terrapinn.com
t/ +1 646 619 1784

 

 

 

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Shannon McGorry
shannon.mcgorry@terrapinn.com
t/ +1 646 619 1840