2019 Agenda

 

Oxon Hill, MD, 10 - 12 April 2019

Schedule

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Apr 1009:00
Conference pass

Keynote Address: How far innovation in rare diseases has come and considerations for future development

Next Generation Therapies Seminar
James Wilson, Director Of Gene Therapy Program, University of Pennsylvania School Of Medicine
Apr 1009:00
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Reimbursement of Spinraza - first of its kind, multi-country contract negotiation and procedures

Payers Seminar
Francis Arickx, Head Of The Directorate Reimbursement Of Medicines And Pharmaceutical Policy, National Institute for Health and Disability Insurance RIZIV-INAMI
Apr 1009:00
Conference pass

Implementing new technologies to create more effect cell and gene clinical trials and to generate real world evidence

Digital Health and Artificial Intelligence Seminar
Dan Tierno, Global Data Sciences And Analytics, Bayer
Apr 1009:00
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Workshop 1:  Hosted by rareLife solutions: Community stewardship - building a digital home

Workshops
Are free-to-use online “mass” communities meeting the needs of rare disease community members? People gathering online are seeking safe, knowledge-rich experiences dedicated to their rare disease. Leaders of advocacy organizations are being tasked with understanding online alternatives with an emphasis on stewardship, revenue generation, quality and creating a harmonious environment where information can be pre-vetted and shared with confidence. Building customized “Digital Homes” meets the emotional support, knowledge and clinical trial education needs of rare disease patients and families, while raising funds for the mission of the advocacy organization.
Apr 1009:00
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Workshop 2: Hosted by Engage Health: Identifying and engaging patients and clinicians in the rare disease space

Workshops
This three-hour workshop will introduce new tools to engage patients and clinicians at every stage of drug development. Together we can find those needles in that haystack and speed the development of life-improving and life-saving therapies. Together we can make a difference to the patients and families impacted by rare diseases.
Apr 1009:20
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Precision Genetic Medicine through Base Editing

Next Generation Therapies Seminar
John Evans, Chief Executive Officer, Beam Therapeutics
Apr 1009:20
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How to get a conversation going between payers, companies, regulators and HTA

Payers Seminar
Anna Bucsics, Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)
Apr 1009:20
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AI-assisted ultra-rapid whole genome sequencing for timely diagnosis in pediatric intensive care units

Digital Health and Artificial Intelligence Seminar
Ray Veeraraghavan, Director Of It And Informatics, Rady Children�s Institute For Genomic Medicine
Apr 1009:40
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Targeted augmentation of nuclear gene output (TANGO) as a novel treatment for rare genetic diseases

Next Generation Therapies Seminar
Barry Ticho, Chief Medical Officer, Stoke Therapeutics
Apr 1009:40
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Ensuring fair pricing of pharmaceuticals and looking into the Canadian landscape for highly priced medicines

Payers Seminar
Apr 1009:40
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Facilitating earlier diagnosis of rare disease patients through machine learning techniques: a case study

Digital Health and Artificial Intelligence Seminar
Jonathan Woodring, Executive Vice President and General Manager, IPM.ai
Stephen Lamb, Principal, IPM.ai
Apr 1010:30
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Development of an LNA-based Therapy for Angelman Syndrome

Next Generation Therapies Seminar
Michelle Krishnan, Translational Medicine Leader In Rare Diseases, Roche Pharma Research and Early Development
Apr 1010:30
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Patients, Pricing and Profits: Is the orphan drug business model fair and sustainable?

Payers Seminar
This session provides an opportunity to revisit our popular simulation exercise, expanding on it by bringing in the perspective of investment, and how risk affects these decisions.Building on from the successful Price Simulation Exercise of 2018, where participants were put in the shoes of a biotech CEO making a pricing decision for an innovative new rare disease medicine, Dolon will present an interactive session which further explores:
  • How biopharmaceutical executives make pricing decisions for orphan drugs?
  • Which factors are relevant to pricing decisions and which are not?
  • What is the relationship between R&D investment and price?
  • How can risk, cost and time of development be factored into these decisions?
  • What is the impact of policies (US ref pricing, EU patent duration) on innovation?
  • Understand price as an incentive for innovation.
Adam Hutchings, Managing Director, Dolon
Apr 1010:50
Conference pass

Development of an LNA-based Therapy for Angelman Syndrome

Next Generation Therapies Seminar
Michelle Krishnan, Translational Medicine Leader In Rare Diseases, Roche Pharma Research and Early Development
Apr 1010:50
Conference pass

Oligonucleotide Therapy for SCN2A Gain-of-Function Epilepsies

Next Generation Therapies Seminar
Steven Petrou, Chief Scientific Officer, RogCon
Apr 1011:00
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Technology at the service of the healthcare informatician - Using decision model and notation for clinical research and decision support

Digital Health and Artificial Intelligence Seminar
Vipul Kashyap, Chief Information Architect, Northwell Health
Apr 1011:10
Conference pass

Next-generation gene-edited cell therapies

Next Generation Therapies Seminar
Rachel Haurwitz, Chief Executive Officer, Caribou Biosciences
Apr 1011:20
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Application of supervised machine learning in rare disease drug development

Digital Health and Artificial Intelligence Seminar
Qing Liu, Statistical Science and Program Strategy, Amicus Therapeutics Inc
Apr 1011:30
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Imara-687, a novel Ph-2 therapy for treatment of sickle cell disease

Next Generation Therapies Seminar
Rahul Ballal, CEO, Imara
Apr 1011:30
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Computational facial phenotyping of genetic syndromes

Digital Health and Artificial Intelligence Seminar
Antonio Porras, Scientist, Children's National Health System
Apr 1011:40
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GOLD SPONSOR SLOT

Digital Health and Artificial Intelligence Seminar
If you are interested in sponsoring this session in the Digital Health & Artificial Intelligence Seminar, contact André Singer now at +1 646 619 1797 or andre.singer@ terrapinn.com
Apr 1013:00
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PKU Alliance on supporting new approaches to finding a therapy

Next Generation Therapies Seminar
Apr 1013:00
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Overview of states’ Medicaid reimbursement practices for orphan drug products

Payers Seminar
Cynthia Denemark, Pharmacy Director, Delaware Division of Medicaid & Medical Assistance
Apr 1013:00
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Platforms for patient-centric collaborative research in the era of precision medicine

Digital Health and Artificial Intelligence Seminar
Alex Sherman, Director, Center For Innovation And Bioinformatics, Massachusetts General Hospital Neurological Clinical Research Institute
Apr 1013:00
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Workshop A: Hosted by MME and LapidusData: Predicting and influencing the market potential of orphan drugs and gene therapies

Workshops
Embracing the interdependence of forecasts, pricing, reimbursement, and early clinical decisionsMarket forecasting presents special challenges for orphan drugs and gene therapies. This workshop will provide real-world examples of how epidemiology, pricing, and early clinical decisions can inform your forecast, and more importantly, how you can influence these factors to achieve commercial success. Our unparalleled experience with rare diseases allows us to share cases of winning—and losing—strategies for market sizing, pricing/reimbursement, trial design, and even product formulation.
Apr 1013:00
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Workshop B: Hosted by Clinigen Group - The expanded access to commercialization journey: interdependencies, operational considerations, strategy & assessment framework

Workshops
Quite often, organizations running an Expanded Access Program (EAP) fail to consider the practical or strategic considerations related to the transition from an EAP to commercial availability and access. Despite being intricately linked, many organizations view Expanded Access and commercial access as two independent mechanisms. This mentality can, at times, hinder patient access which can have a significant impact on rare disease patients, in particular.
Apr 1013:20
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Gene editing with CRISPR for Duchenne muscular dystrophy – novel pre-clinical design, data collection, and advantages of SingleCutCRISPR technology

Next Generation Therapies Seminar
Leonela Amoasii, Director, Gene Editing, Exonics Therapeutics
Apr 1013:20
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Brazil’s landscape for the recognition of rare diseases and the evaluation of orphan drugs to ensure access and continuous supply

Payers Seminar
Leandro Safatle, Secretário-Executivo, ANVISA
Apr 1013:40
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CRISPR technology to treat Sickle Cell Disease and β-Thalassemia

Next Generation Therapies Seminar
Jerome Rossert, Vice President Clinical Development, Vertex Pharmaceuticals
Apr 1013:40
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Assessment and reimbursement of drugs for rare diseases in Ireland and the efforts after joining BeNeLuxA

Payers Seminar
Michael Barry, Clinical Director, National Centre for Pharmacoeconomics
Apr 1013:40
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Cross functional utility of digital communication in research and development

Digital Health and Artificial Intelligence Seminar
Luke Rosen, Head Of Patient Engagement And Digital Health Experience, Ovid Therapeutics
Apr 1014:00
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Accelerating health with artificial intelligence and analytics

Digital Health and Artificial Intelligence Seminar
Molly Mccarthy, National Director, Us Provider Industry And Chief Nursing Officer, Microsoft
Apr 1015:00
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PHA121: a new oral small molecule for hereditary angioedema (HAE)

Next Generation Therapies Seminar
Morgan Conn, Chief Business Officer, Pharvaris
Apr 1015:00
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Future considerations for advanced therapies and ultra rare therapies and the role of the New Therapies Council

Payers Seminar
Luc Boileau, Président-Directeur General, Inesss
Apr 1015:00
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Reimagining Drug Discovery for Rare Disease Through AI

Digital Health and Artificial Intelligence Seminar
Ron Alfa, Senior Vice President, Translational Discovery, Recursion Pharmaceuticals
Apr 1015:20
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Combatting rare diseases with ex-vivo, lentiviral vector mediated gene therapy

Next Generation Therapies Seminar
Apr 1015:25
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Lessons learned from innovative reimbursement contracts for advanced therapies - further possibilities in risk sharing and pay for performance in Europe

Payers Seminar
Detlev Parow, Head Pharmaceutical Department, D.A.K.
Apr 1015:40
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Beyond one disease at a time: Platform approaches to rare disease therapeutics development

Next Generation Therapies Seminar
Philip Brooks, Program Director, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)
Apr 1015:50
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Recognizing the need for variety in and working effectively with different levels of evidence in orphan drug evaluations

Payers Seminar
Eugean Jiwanmall, Senior Research Analyst, Technology Evaluation And Medical Policy, Independence Blue Cross
Apr 1016:00
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AAVHSC vectors: A bimodal platform for in vivo, nuclease-free gene editing and gene therapy for rare genetic diseases

Next Generation Therapies Seminar
Albert Seymour, Chief Scientific Officer, Homology Medicines
Apr 1016:15
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Rare disease efforts at Aetna

Payers Seminar
Daniel Knecht, Vice President, Clinical Strategy And Policy, Aetna
Apr 1016:20
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Uncovering New Medical Utility with Next Generation Enzyme Therapeutics

Next Generation Therapies Seminar
Apr 1016:40
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Working with Employer groups to determine reimbursement of orphan

Payers Seminar
Jay Weaver, Divisional Vice President, Enterprise Pharmacy, Blue Cross Blue Shield, Health Care Service Corp.

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Apr 1108:25
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Chairperson's opening remarks

Keynotes
Ellen Coleman, President And Chief Executive Officer, VOZ Advisors
Apr 1108:30
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Keynote Address: Advancing RNAi therapeutics as a whole new class of medicines

Keynotes
John Maraganore, Chief Executive Officer, Alnylam Pharmaceuticals
Apr 1108:55
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Keynote Address: Re-thinking and reshaping “rare” – continuing the march towards curative therapies for the rare disease community

Keynotes
Christopher Austin, Director, National Center For Advancing Translational Sciences (Ncats), National Institutes of Health (NIH)
Apr 1109:20
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Keynote Address: Accelerating Orphan Drug development and improving outcomes, by unlocking genetic information

Keynotes
Arndt Rolfs, Chief Executive Officer, CENTOGENE AG
Apr 1109:40
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Keynote Panel: Advancing cell and gene therapy – considerations in manufacturing, R&D expertise, early payer engagement and continuous patient centricity

Keynotes
Moderator: Sophie Schmitz, Managing Partner, Partners4Access
Sandy Macrae, President And Chief Executive Officer, Sangamo Therapeutics
Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer
Matthew Patterson, President And Chief Executive Officer, Audentes Therapeutics
Mark Rothera, President And Chief Executive Officer, Orchard Therapeutics
Andre Choulika, Chairman And Chief Executive Officer, Cellectis
Samarth Kulkarni, Chief Executive Officer, CRISPR Therapeutics
Apr 1111:25
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Interdepartmental collaboration to ensure perspective and expertise of patient community in clinical development

Clinical Development & Regulatory
Michelle Berg, Vice President Patient Affairs, Abeona Therapeutics Inc.
Apr 1111:25
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An innovative business model: harnessing ‘the strength of small’ to advance orphan drugs & rare disease therapies through to commercialization

Commercial
Denise Scots Knight, Chief Executive Officer And Co-founder, Mereo BioPharma
Apr 1111:25
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RADAR, the NIH registry initiative – Clinical trial readiness through stronger natural history data

Rare Disease Advocacy World
Anne Pariser, Director, Office Of Rare Diseases Research, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)
Apr 1111:25
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ARGENTINA: Overall state of rare disease in Argentina: pathways and challenges for better access to therapies

Global Market and Patient Access
Natalia Fernandez Fruttero, Rare Diseases, Orphan Drugs & Gene Therapy, Experienced Consultant
Cecilia Arizcuren, Head of Patient Advocacy Argentina, Sanofi
Apr 1111:25
Conference pass

Potential solutions for assessment, reimbursement and affordability of advanced therapies in Europe

Pricing and Reimbursement
Annie Hubert, Senior Director, European Public Policy, Alliance for Regenerative Medicine
Apr 1111:25
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FDA tools to help advance clinical development of cell and gene therapies

Advanced Therapies - Clinical
Julie Tierney, Senior Policy Advisor For Strategic Planning And Legislation,Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA)
Apr 1111:25
Conference pass

One batch, one patient – can innovation in autologous therapies process development be radically accelerated to serve larger populations?

Advanced Therapies - Manufacturing
Derek Adams, Chief Technology And Manufacturing Officer, Bluebird Bio
Apr 1111:25
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Opportunities and challenges in developing medicines for rare cancers

Rare Oncology
Samit Hirawat, Executive Vice President, Head Of Oncology Global Development, Novartis
Apr 1111:25
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Investors Panel: Multi-stakeholder Investing in Cell therapy – evaluating modality risk, understanding the science

Pitch and Partner
Moderator: Brian Bronk, Head of Business Development, Rare Diseases & Rare Blood Disorders, Global Business Development & Licensing, Sanofi
Heather Behanna, Biotech Investor, Rare Diseases
Debra Miller, CEO, CureDuchenne
Walter Kowtoniuk, Principle, Third Rock Ventures, LLC
Apr 1111:50
Conference pass

Achieving clinical development by remaining laser focused on patients, developing the right partners and incorporating patient voice & value into a 360 R&D approach

Clinical Development & Regulatory
Ilan Ganot, President and Chief Executive Officer, Solid Biosciences
Apr 1111:50
Conference pass

Developing a commercial business model for ultra-rare disease

Commercial
Scott Dorfman, Chief Executive Officer, Odylia Therapeutics
Apr 1111:50
Conference pass

Patients Data Matters - key learnings from the first ever qualitative and quantitative study on data sharing for people living with a rare disease

Rare Disease Advocacy World
Yann Le Cam, Chief Executive Officer, EURORDIS
Apr 1111:50
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UAE: updates on market access regulation, rare disease clinical trial enrollment, and patient populations

Global Market and Patient Access
Amin Hussain Al Amiri, Assistant Undersecretary Of Public Health Policy And Licensing, United Arab Emirates Ministry of Health and Prevention
Apr 1111:50
Conference pass

Update on LUXTURNA implementation and outcomes-based efforts

Pricing and Reimbursement
Sarah Pitluck, Head, Global Pricing And Reimbursement, Spark Therapeutics
Apr 1111:50
Conference pass

Further defining the path to the clinic for in vivo gene editing

Advanced Therapies - Clinical
Charlie Albright, Chief Scientific Officer, Editas Medicine
Apr 1111:50
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Enabling product launch and reliable supply through an integrated manufacturing and supply chain strategy

Advanced Therapies - Manufacturing
Stewart Craig, Chief Manufacturing Officer, Orchard Therapeutics
Apr 1111:50
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National Cancer Institute initiatives to tackle rare cancer and foster drug development

Rare Oncology
Jerry Collins, Assoc Division Director, Developmental Therapeutic, National Cancer Institute
Apr 1112:15
Conference pass

From the end to the beginning: a patient-centric look at drug development and commercial access

Clinical Development & Regulatory
Juliet Moritz, Vice President, Patient & Stakeholder Engagement, Premier Research
Apr 1112:15
Conference pass

The Behind the Seizure program - an innovative, cross-company collaboration that aims to provide faster diagnosis for young children with epilepsy by making it easier to access genetic testing

Commercial
Moderator: Daniel Anderson, Head Of Commercial Partnerships, Invitae
Robin Sherrington, Senior Vice President Business and Corporate Development, Xenon Pharmaceuticals Inc
Barry Ticho, Chief Medical Officer, Stoke Therapeutics
Alexis Braun, Diagnostics Brand Lead, North America LSD, Biomarin Pharmaceuticals Inc.
Apr 1112:15
Conference pass

Owning your own data – lessons learned from establishing a direct-to-participant data platform

Rare Disease Advocacy World
Jennifer Thornton, Executive Director, A.T. Childrens Project
Apr 1112:15
Conference pass

GLOBAL: SPINRAZA Overview of global reimbursement landscape, key successes and learnings to date & Brazil case study

Global Market and Patient Access
Tom Rising, Director – Global Value & Access, Biogen
André Liamas, Director, Market Access and Government Affairs, Biogen
Apr 1112:15
Conference pass

Shifting mindsets to ensure cell and gene therapies are appropriately assessed – broadening the definition of value through evidence and data collection

Pricing and Reimbursement
Clark Paramore, Head Of Value Demonstration, Bluebird Bio
Apr 1112:15
Conference pass

Getting to the clinic through strategic partnership – combining scientific, clinical development, and manufacturing skills to develop new treatments for Cystic Fibrosis patients in Europe

Advanced Therapies - Clinical
Stephen Hyde, Associate Professor of Molecular Therapy at the University of Oxford, Principle Investigator, UK Cystic Fibrosis Gene Therapy Co, University of Oxford
Apr 1112:15
Conference pass

Cellectis' gene-editing enabled off-the-shelf CAR-T manufacturing take cell therapy to the next stage

Advanced Therapies - Manufacturing
David Sourdive, Executive Vice President Technical Operations, Cellectis
Apr 1112:15
Conference pass

Recent policy initiatives that have helped facilitate drug development for rare cancer population

Rare Oncology
Jeff Allen, President And Chief Executive Officer, Friends of Cancer Research
Apr 1112:15
Conference pass

How a venture-backed orphan drug accelerator is advancing promising therapies for rare diseases

Pitch and Partner
Imran Babar, Chief Business Officer, Cydan II, Inc.
Apr 1112:40
Conference pass

Patient-focused rare disease clinical trial protocols: patient-centered outcomes and beyond

Clinical Development & Regulatory
Margaret Vernon, Vice-President and General Manager, Patient-Centered Research, Evidera – Evidence, Value & Access by PPD
Kevin Marsh, Executive Director, Patient-Centered Research, Evidera – Evidence, Value & Access by PPD
Apr 1112:40
Conference pass

Integrating social determinants of health to increase adherence

Rare Disease Advocacy World
Oodaye Shukla, Chief Data & Analytics Officer, HVH Precision Analytics
Apr 1112:40
Conference pass

JAPAN: leveraging a comprehensive value creation platform to successfully enter and navigate the Japanese rare disease market

Global Market and Patient Access
Philippe Auvaro, Director and Senior Executive Officer, Supervisory Manager of Orphan Drug Business, CMIC HOLDINGS Co., Ltd.
Apr 1112:40
Conference pass

3 pillars of a sustainable CGT world – everything you need to know from a pricing, reimbursement and access perspective

Pricing and Reimbursement
Sophie Schmitz, Managing Partner, Partners4Access
Apr 1112:40
Conference pass

Use of real world data to support the path to approval for advanced therapies in rare diseases

Advanced Therapies - Clinical
Maryna Kolochavina, Director Client Engagement Solutions, Real World & Late Phase, Syneos Health
Apr 1112:40
Conference pass

Disruption in oncology clinical trial administration provides cost savings, efficiency and risk mitigation

Rare Oncology
Apr 1112:40
Conference pass

Pitch 1: Humanized monoclonal antibody targeting Interleukin-15 for the treatment of Eosinophilic Esophagitis

Pitch and Partner
Alain Vicari, Chief Executive Officer And Founder, Calypso Biotech
Apr 1112:50
Conference pass

Pitch 2: Anhydrous Enol-Oxaloacetate as a novel platform drug for CNS Intercellular Glutamate reduction and rare brain diseases

Pitch and Partner
Alan Cash, Chief Executive Officer And Co-Founder, MetVital
Apr 1113:00
Conference pass

When predictable execution trumps brilliant planning: the interplay of strategic and transactional relationships in orphan disease drug development

Advanced Therapies - Clinical
Michael Murphy, Chief Medical And Scientific Officer, Worldwide Clinical Trials
Apr 1113:00
Conference pass

Pitch 3: ONL1204, First-in-Class inhibitor of Fas (CD95) signaling to protect vision from retinal disease

Pitch and Partner
John Freshley, President And Chief Executive Officer, ONL Therapeutics
Apr 1114:30
Conference pass

ROUNDTABLE 1: Global expanded access – applying artificial intelligence and access innovation for expanded access programs

Keynotes
Anne Cropp, Chief Scientific Officer, Early Access Care
Apr 1114:30
Conference pass

ROUNDTABLE 10: Data ownership – addressing the need for increased data sharing while also considering company/patient data "ownership" through clinical trials and beyond

Keynotes
Tiina Urv, Program Director, Office Of Rare Disease Research, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)
Apr 1114:30
Conference pass

ROUNDTABLE 11: Educating HCPs – when considering the absence of newborn screening lessons learned from Metachromatic Leukodystrophy (MLD)

Keynotes
Tara Mathiesen, Director, Us Diagnostics, Orchard Therapeutics
Apr 1114:30
Conference pass

ROUNDTABLE 12: Chilling investment? – the potential implications of increased public scrutiny of rare disease drug prices in the US

Keynotes
Satyen Sanghavi, Chief Scientific Officer and Executive Director, Regrow Biosciences LLC , Texas - USA
Apr 1114:30
Conference pass

ROUNDTABLE 13: Cross-Ecosystem collaborations – mutual value propositions and collaborations for pharma, payers and providers to collaborate in the rare diseases space

Keynotes
Vipul Kashyap, Chief Information Architect, Northwell Health
Apr 1114:30
Conference pass

ROUNDTABLE 14: Bridging US and India - strategies to accelerate rare diseases clinical research and patient recruitment

Keynotes
Harsha Rajasimha, Co Founder, Organization for Rare Diseases India
Apr 1114:30
Conference pass

ROUNDTABLE 15: Federal Policy – update on rare disease federal policy and NORD initiatives to engage capital hill

Keynotes
Paul Melmeyer, Associate Director Of Public Policy, National Organization for Rare Disorders (NORD)
Apr 1114:30
Conference pass

ROUNDTABLE 16: Internal organization and policy - data sharing and ownership, internal structure and funding, scientific advisors and more

Keynotes
Neena Nizar, President, The Jansens Foundation
Denis Belyakov, Executive Director, Union of Patients' And Patients' Organizations With Rare Diseases
Terri Klein, President And Chief Executive Officer, National M.P.S. Society
Eric Hartman, Executive Director, Choroideremia Research Foundation Inc
Apr 1114:30
Conference pass

ROUNDTABLE 17: Patient Access in Europe & Beyond - Enabling access for rare disease therapies exploring alternative pathways for patients in need

Keynotes
Clive Whitcher, Vice President Head of Global Patient Access, Inceptua
Kelly Fearn, Principal Consultant, Inceptua
Apr 1114:30
Conference pass

ROUNDTABLE 18: Market Access & commercialization – how specialty services and options should vary for patient populations of 20, 2,000, and 20,000

Keynotes
Susan Robinson, Senior Vice President of Business Development, AnovoRx Group Llc
Apr 1114:30
Conference pass

ROUNDTABLE 19: Update on patient centricity in research – preliminary survey results from patient advocacy group leaders

Keynotes
Leone Atkinson, Executive Medical Director, Covance
Apr 1114:30
Conference pass

ROUNDTABLE 2: Recruitment and retention: innovative patient centric consultative strategies / services to enhance clinical trial recruitment efforts and to insure higher patient retention rates

Keynotes
Scott Gray, Chief Executive Officer, Clincierge
Greg Candelmo, Director Of Sales, Clincierge
Apr 1114:30
Conference pass

ROUNDTABLE 20: Combining imaging and genomics analysis – integration of multi-modal data analysis to accelerate and de-risk drug development in rare diseases

Keynotes
Matthew Silva, Executive Vice President, Scientific Applications, Invicro Llc
Brigette Tippin Davis, Senior Vice President, R&D, Ambry Genetics
Apr 1114:30
Conference pass

ROUNDTABLE 21: Inhouse versus outsource – determining the right orphan drug manufacturing fit for your organization

Keynotes
Apr 1114:30
Conference pass

ROUNDTABLE 3: Epidemiology & forecasting – value, challenges, and methods for rare diseases

Keynotes
David Lapidus, President, Lapidus Data
Apr 1114:30
Conference pass

ROUNDTABLE 4: BD & forecasting – how to build forecasts based on available epidemiological information and considerations for pricing and access

Keynotes
Joseph Musumeci, President, BluePrint Orphan
Apr 1114:30
Conference pass

ROUNDTABLE 5: Patient-focused research – new approaches to bring clinical trials directly to patients and change the patient-centric trial paradigm in rare diseases

Keynotes
Scott Schliebner, Vice President, Scientific Affairs, Rare Diseases, PRA Health Sciences
Apr 1114:30
Conference pass

ROUNDTABLE 6: Patient-centered Development: Early patient input, understanding patient goals in clinical trials, and furthering relationships between all stakeholders

Keynotes
Jamie Arnott, Senior Project Director, Rho, Inc
Apr 1114:30
Conference pass

ROUNDTABLE 7: Meaningful endpoints – navigating the differences in what it means to patients, caregivers, physicians and regulators during advanced therapies clinical development

Keynotes
Lisa Dilworth, Vice President Rare And Orphan Diseases, Synteract
Apr 1114:30
Conference pass

ROUNDTABLE 8: Driving diagnosis – steps industry can take to improve the diagnosis of rare disease

Keynotes
Kate Grady, Senior Director, Huron Life Sciences
Apr 1114:30
Conference pass

ROUNDTABLE 9: Planning for orphan drug supply – the importance of manufacturability

Keynotes
Cornell Stamoran, Vice President, Strategy & Gov’t Affairs, and Founder & Co-Chair, Catalent Applied Drug Delivery Institute, Catalent Pharma Solutions
Apr 1114:30
Conference pass

Pitch 4: Induction of tumor necrosis in hepatocellular carcinoma to enhance the efficacy of immune checkpoint inhibitors by combining a hypoxia-activation agent with embolization

Pitch and Partner
Ray Lee, Founder And Chief Medical Officer, Teclison Limited
Apr 1114:40
Conference pass

Pitch 5: Next generation of immunotherapeutic target discovery platform for rare diseases

Pitch and Partner
Maurizio Chiriva, Cheif Executive Officer, Kiromic Llc
Apr 1114:50
Conference pass

Pitch 6: CD24Fc for the prevention of GVHD and damage mediated inflammation

Pitch and Partner
Martin Devenport, Chief Operating Officer, OncoImmune Inc
Apr 1115:00
Conference pass

Pitch 7: FLAG-003 as an oral therapy for the treatment of glioma

Pitch and Partner
Frank Sorgi, President And Chief Executive Officer, FLAG Therapeutics
Apr 1115:10
Conference pass

Pitch 8: ST-001 for treatment of Cushing’s syndrome

Pitch and Partner
Manohar Katakam, Founder And Chief Executive Officer, SteroTherapeutics
Apr 1115:20
Conference pass

Pitch 9: Cibinetide for treatment of sarcoidosis-related small fiber neuropathy

Pitch and Partner
Joe Young, Chief Business Officer, Araim Pharmaceuticals Inc
Apr 1115:30
Conference pass

Pitch 10: AP101 for the treatment of Epidermolysis bullosa

Pitch and Partner
Apr 1115:40
Conference pass

Bringing broad stakeholder perspectives and end-to-end evidence into the clinical development and data generation framework

Clinical Development & Regulatory
Usman Iqbal, Vice President, Medical Affairs, Acer Therapeutics
Apr 1115:40
Conference pass

Commercial analytics – how to use data to overcome challenges in rare diseases

Commercial
Vikram Karnani, Chief Commercial Officer, Horizon Pharma
Apr 1115:40
Conference pass

Generating commercial insights and addressing data gaps across the patient journey using machine learning

Commercial
Chris Murphy, Group Vice President, Analytics, Horizon Pharma plc
Apr 1115:40
Conference pass

Decode Duchenne: how free genetic testing, counseling and a strong patient registry are supporting advances in Duchenne and Becker Muscular Dystrophy diagnosis, research and education

Rare Disease Advocacy World
Annie Kennedy, Senior Vice President Of Legislation And Public Policy, Parent Project Muscular Dystrophy
Apr 1115:40
Conference pass

SCOTLAND: Improved Access - allowing three years of patient access to medicines for ultra-rare conditions in NHS Scotland and creating the opportunity to collect real-world data for reimbursement

Global Market and Patient Access
Ailsa Brown, Lead Health Economist, Scottish Medicines Consortium
Apr 1115:40
Conference pass

Pay for performance – pilot contract models for cell and gene therapies one time treatment

Pricing and Reimbursement
Alexander Natz, Secretary General, EUCOPE
Apr 1115:40
Conference pass

Advancing the clinical program development of AAV-Based Gene therapies for rare diseases: a case study

Advanced Therapies - Clinical
Sal Rico, Vice President, Clinical Development, Audentes Therapeutics
Apr 1115:40
Conference pass

Development of commercial scale manufacturing of GalNAc conjugated siRNAs. analytical and process perspective

Advanced Therapies - Manufacturing
Alfred Boyle, Senior Vice President Technical Operations, Alnylam Pharmaceuticals
Apr 1115:40
Conference pass

Pitch 11: Accelerating retinal genetic therapies as a nonprofit biotech

Pitch and Partner
Lara Dorfman, Director of Business Development, Odylia Therapeutics
Apr 1115:40
Conference pass

Pitch 11: Accelerating retinal genetic therapies as a nonprofit biotech

Pitch and Partner
Lara Dorfman, Director of Business Development, Odylia Therapeutics
Apr 1115:50
Conference pass

Pitch 12: Nanoengineered chemotherapy patch for oral cavity cancer

Pitch and Partner
Manijeh Goldberg, Chief Executive Officer, Privo Technologies
Apr 1116:00
Conference pass

Best practices for patient group and industry interactions in clinical development and trials

Clinical Development & Regulatory
Ron Bartek, President, Director, And Founder, Friedreich's Ataxia Research Alliance
Jeffrey Sherman, Chief Medical Officer, Horizon Pharma
Apr 1116:00
Conference pass

Epidemiologic methods for rare diseases: Identifying patients using real-world data and literature

Commercial
Apr 1116:00
Conference pass

The role of genetic counselors in educating rare disease patients and families about genetic testing, data and diagnosis

Rare Disease Advocacy World
Kimberly Leblanc, Associate Director Of Research Operations Undiagnosed Diseases Network Coordinating Center, Harvard Medical School
Apr 1116:00
Conference pass

SPAIN: Opportunities to engage authorities, regional agencies and patient organizations to improve access in Spain

Global Market and Patient Access
Alba Ancochea Díaz, Directora Gerente, Spanish Federation of Rare Disease
Apr 1116:00
Conference pass

Advancing a national legislative framework for payment-over- for gene therapy products

Pricing and Reimbursement
Max Bronstein, Senior Director, Health Policy And Corporate Affairs, Audentes Therapeutics
Apr 1116:00
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Advancing the clinical program development of gene therapy for ultra-orphan disease: finding the patients, getting them enrolled

Advanced Therapies - Clinical
Barrett Katz, Chief Medical Officer, GenSight Biologics
Apr 1116:00
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Challenges in analytical development for gene therapy for rare diseases – Luxturna as a case study

Advanced Therapies - Manufacturing
Apr 1116:00
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Count Me In - accelerating research for rare cancers through patient-partnered genomic studies

Rare Oncology
Corrie Painter, Associate Director, Count Me In
Apr 1116:00
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Pitch 13: Treatment of diffuse large B-Cell Lymphoma (DLBCL) VAL001 - a phase IIb/III ready drug

Pitch and Partner
Johan Drott, Chief Executive Officer, Respiratorius
Apr 1116:10
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Pitch 14: TL-010 for treatment of schistosomiasis, foodborne trematodiases and taeniasis/neurocysticercosis

Pitch and Partner
Mingxin Qian, Chief Executive Officer, Tongli Biomedical
Apr 1116:20
Conference pass

Pediatric trial design - the right population, the right indication, the right design

Clinical Development & Regulatory
Susan Mccune, Director, Office Of Pediatric Therapeutics, Office Of The Commissioner, U.S. Food and Drug Administration
Apr 1116:20
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What’s next? New frontiers for rare disease epidemiology in big data, genomic sets, and FDA and EMA guidance

Commercial
Alexander Cole, Global Head, Epidemiology, Alexion Pharmaceuticals
Apr 1116:20
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Bringing an end to the diagnostic odyssey through genetic testing and partnership - Outcomes from the Global Commission partnership

Rare Disease Advocacy World
Linn Parrish, Head Of Corporate Responsibility, Shire
Apr 1116:20
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EU & CANADA: Working with international authorities on novel genetic medicines

Global Market and Patient Access
Anant Murthy, Vice President Of Market Access And Pricing, Alnylam
Apr 1116:20
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PANEL: Operationalizing value-based payments overtime - operational feasibility of longer term contracts and strategies to effectively collect long term data to ensure continued reimbursement

Pricing and Reimbursement
Moderator: Marianne Hamilton Lopez, Research Director, Duke Margolis Center for Health Policy
Timothy Hunt, Senior Vice President, Corporate Affairs, Editas Medicine
Gregory Daniel, Deputy Director, Duke Margolis Center for Health Policy
Apr 1116:20
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FDA gene therapy draft guidance documents – disease specific guidances and long-term follow up

Advanced Therapies - Clinical
Ilan Irony, Deputy Director Division Of Clinical Evaluation And Pharmacology, Center for Biologics Evaluation and Research (CBER), Food and Drug Administration (FDA)
Apr 1116:20
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Gene Therapy and AAV: Analytical Advancements Enabling Successful Product Development and Life-Cycle Management

Advanced Therapies - Manufacturing
Apr 1116:20
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PANEL: Barriers to clinical trials – need for increased coordination, clinical trial awareness, and changing the treatment paradigm

Rare Oncology
Jim Palma, Executive Director, TargetCancer Foundation
Melinda Bachini, Advocacy Coordinator, The Cholangiocarcinoma Foundation
John Hopper, President And Executive Director, Fibrolamellar Cancer Foundation
Josh Sommer, Co Founder And Executive Director, Chordoma Foundation
Corrie Painter, Associate Director, Count Me In
Apr 1116:20
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Pitch 15: Development of RNAi therapeutics for treatment of Cholangiocarcinoma (an orphan indication)

Pitch and Partner
Patrick Lu, President And Chief Executive Officer, Sirnaomics, Inc.
Apr 1116:30
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Pitch 16: First-in-class nicotine-blocker in development for the treatment of Buerger’s Disease: ATI-1013, a human anti-nicotine monoclonal antibody

Pitch and Partner
Apr 1116:40
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Innovations and strategies that accelerate rare and orphan drug development

Clinical Development & Regulatory
Changting C. Haudenschild, VP Technical, PAREXEL Consulting, PAREXEL International
Apr 1116:40
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Molecular methods to improve the selection of clinical trial participants

Rare Disease Advocacy World
Heather Marton, Reporting, Laboratory Liaison, MNG Laboratories
Apr 1116:40
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EUROPE: early access for orphan drugs: generating value for all stakeholders

Global Market and Patient Access
Stefan Walzer, General Manager And Founder, MArS Market Access & Pricing Strategy, MEDVANCE Germany
Dominique Amory, Member, Nextep, MEDVANCE
Mariangela Prada, Head Of Patient Access Unit, Intexo, MEDVANCE Italy
Max Brosa, Owner, Oblikue Consulting, MEDVANCE Spain
Apr 1116:40
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Pitch 17: Synthetic plasmalogen precursor PPI-1040 for the treatment of Rhizomelic Chondrodysplasia Punctata (RCDP) and other rare pediatric peroxisomal disorders

Pitch and Partner
Apr 1116:50
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Pitch 18: Lixivaptan for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Pitch and Partner
Apr 1117:40
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Keynote Panel: Patient Data – collection, ownership, and regulatory considerations

Keynotes
Moderator: Oodaye Shukla, Chief Data & Analytics Officer, HVH Precision Analytics
Simon Kos, Chief Medical Officer, Microsoft
James Greenwood, Chief Executive Officer, Biotechnology Industry Organization
Yann Le Cam, Chief Executive Officer, EURORDIS
Peter Saltonstall, President And Chief Executive Officer, National Organization for Rare Disorders (NORD)

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Apr 1208:45
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Chairperson’s opening remarks

Keynotes
Mike Porath, Founder And Chief Executive Officer, The Mighty
Apr 1208:50
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Keynote Address: Preparing for a new treatment paradigm - gene therapy access and affordability

Keynotes
Jeff Ajer, Executive Vice President And Chief Commercial Officer, BioMarin
Apr 1209:10
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Keynote address: A staged approach to European patient access

Keynotes
Stuart Bell, Vice President, Consulting, Inceptua
Apr 1209:30
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Keynote Panel: Improving access and affordability of new orphan drugs while ensuring continuous investment in rare disease innovation and R&D

Keynotes
Moderator: Jim Lang, Chief Executive Officer, EVERSANA
Daniel Faga, Chief Business Officer, Spark Therapeutics
Sebastien Martel, Senior Vice President and Global Head of Rare Diseases, Sanofi
Paul Levesque, Global President, Rare Disease, Pfizer
Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical
Stuart Peltz, Chief Executive Officer, PTC Therapeutics
Apr 1211:15
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A patients first approach to expanded access

Clinical Development & Regulatory
Edith Eby, Vice President, Pfizer
Apr 1211:15
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Commercial insights during early product development

Commercial
Mike Blum, VP COMMERCIAL STRATEGY, HOMOLOGY MEDICINES
Apr 1211:15
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The role of patient advocacy in ensuring access to effective and transformative therapies

Rare Disease Advocacy World
Amanda Bok, Chief Executive Officer, European Haemophillia Cosortium
Apr 1211:15
Conference pass

US, EUROPE, CANADA: Working with regulatory bodies through the pre-approval stage for gene therapies

Global Market and Patient Access
Janet Rae, Vice President Of Global Head Of Regulatory Affairs, Ultragenyx Pharmaceutical
Apr 1211:15
Conference pass

Orphan drugs in Canada: the evolving landscape

Pricing and Reimbursement
Trevor Richter, Director, Common Drug Review And Optimal Use, CADTH
Apr 1211:15
Conference pass

Translation of Gene Therapeutics in Neurological and Neuromuscular Diseases

Advanced Therapies - Clinical
Brian Kaspar, Chief Scientific Officer, AveXis Inc
Apr 1211:15
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FDA’s efforts to expedite the development of Regenerative Medicine Advanced Therapy (RMAT) Products

Advanced Therapies - Manufacturing
Peter Marks, Director, Center For Biologics Evaluation And Research (Cber), U.S. Food and Drug Administration
Apr 1211:15
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Funding academic rare disease research with huge translational science potential – programs of excellence, grants and collaborations

Pitch and Partner
Ashley Winslow, Senior Director, Portfolio Development, The Orphan Disease Center
Apr 1211:25
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Adapting value frameworks for treatments of ultra-rare diseases and cures: ICER’s experience and recent developments

Pricing and Reimbursement
Rick Chapman, Director Of Health Economics, Institute for Clinical and Economic Review
Apr 1211:35
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The access challenge for rare disease technologies- doing it the NICE way

Pricing and Reimbursement
Sheela Upadhyaya, Assoicate Director HST, National Institute for Health and Care Excellence
Apr 1211:40
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Expanded access programs and real world evidence

Clinical Development & Regulatory
Beverly Harrison, Head, Patient Support, Janssen Office of the Chief Medical Officer, Janssen Research and Development Llc
Apr 1211:40
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How to approach global expansion after initial approval and how strategy may change over time

Commercial
Thomas Lester, Senior Director Of Product Development, BioMarin Pharmaceutical Inc.
Apr 1211:40
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Shining a light on the financial and societal burden of blindness – a patient groups’ role in demonstrating value of treatment to all stakeholders

Rare Disease Advocacy World
Kristin Smedley, President, Curing Retinal Blindness Foundation
Apr 1211:40
Conference pass

CHINA: Patient access, working with companies, expanding regional access to clinical trials and the evolving relationship between patient groups, companies and regulatory authorities

Global Market and Patient Access
Kevin Huang, President, Chinese Organization for Rare Disorders
Apr 1211:40
Conference pass

Combatting resistance in CAR-T clinical trials – developing new approaches to treat patients

Advanced Therapies - Clinical
Marco Ruella, Professor of Medicine, Scientific Director, Lymphoma Program Division of Hematology and Oncology Center, University of Pennsylvania
Apr 1211:40
Conference pass

Strategic advantages of building proprietary gene therapy manufacturing capabilities: scaling from pre-clinical to commercial development

Advanced Therapies - Manufacturing
John Gray, Senior Vice President And Chief Scientific Officer, Audentes Therapeutics
Apr 1211:40
Conference pass

Developing drugs against rare oncogenic targets in pediatric populations - considerations in patient identification, patient recruitment, and working with regulators

Rare Oncology
Michael Cox, Senior Director Of Clinical Development And Medical Affairs, Loxo Oncology Inc
Apr 1211:40
Conference pass

Shaping the future of single ventricle heart disease

Pitch and Partner
Erik Emerson, Executive Vice President, Commercial Operations, Mezzion Pharma
Apr 1211:40
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Shaping the future of Single Ventricle Heart Disease

Pitch and Partner
Erik Emerson, Executive Vice President, Commercial Operations, Mezzion Pharma
Apr 1211:45
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HTA panel discussion with Coordinating HTA and learning from best practices and lessons learned from recent assessments of advanced therapy

Pricing and Reimbursement
Rick Chapman, Director Of Health Economics, Institute for Clinical and Economic Review
Goedele Van Haasteren, Hta Specialist, Swiss Federal Office of Public Health
Sheela Upadhyaya, Assoicate Director HST, National Institute for Health and Care Excellence
Trevor Richter, Director, Common Drug Review And Optimal Use, CADTH
Apr 1212:05
Conference pass

Developing a global expanded access policy to ensure access for rare disease patients

Clinical Development & Regulatory
Paul Aliu, Global Head of Medical Governance, Chief Medical Office, Novartis
Apr 1212:05
Conference pass

Industry and hospital collaboration - Preparing hospitals to administer therapies by having policies and procedures in place along with pre-positioned, highly flexible, multidisciplinary team rare dis

Commercial
Marshall Summar, Division Chief Of Genetics And Metabolism, Children's National Medical Center
Apr 1212:05
Conference pass

One organization, three diseases - bringing together common Cerebral Creatine Deficiency Syndromes to build a platform for research

Rare Disease Advocacy World
Laura Trutoiu, Director Of Research, Association For Creatine Deficiencies
Apr 1212:05
Conference pass

JAPAN: Patient Reported Outcome efforts to help clinical development and company-patient relationships in Japan

Global Market and Patient Access
Yukiko Nishimura, President, ASrid Japan
Apr 1212:05
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The access challenge for rare disease technologies- doing it the NICE way

Pricing and Reimbursement
Helen Knight, Programme Director, Technology Appraisals and Highly Specialized, National Institute for Health & Care Excellence (NICE)
Apr 1212:05
Conference pass

Development of gene therapies for rare CNS disorders

Advanced Therapies - Clinical
Mark Pykett, Chief Scientific Officer, PTC Therapeutics
Apr 1212:05
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CAR-T manufacturing: how biotechs like Mustang Bio have developed early stage scalable manufacturing by optimizing resources while ensuring a long term commercial vision

Advanced Therapies - Manufacturing
Knut Niss, Chief Technology Officer, MustangBio
Apr 1212:05
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Identifying and developing treatment for rare cancers - orphan drug designation, and the challenges of moving into clinical development and beyond

Rare Oncology
Sanjeev Luther, Chief Executive Officer, Rafael Pharmaceuticals
Apr 1212:05
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Pitch 19: INT41, an Intrabody Gene Therapy for Treating Huntington’s Disease

Pitch and Partner
Apr 1212:15
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Pitch 20: ONC201, a novel, small molecule showing clinical efficacy in H3 K27M-mutant glioma, a rare subtype of brain cancers

Pitch and Partner
Lee Schalop, Chief Operating Officer, Oncoceutics Inc
Apr 1212:25
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Pitch 21: AAV2/8-based gene therapy for Mitochondrial NeuroGastroIntestinal Encephalomyopathy (MNGIE)

Pitch and Partner
Apr 1212:30
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How to optimally benefit from tracking a patient’s journey

Clinical Development & Regulatory
Dennis Akkaya, Corporate Development, myTomorrows
Apr 1212:30
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From epidemiology insights to high-risk population screening: innovative genetically based approaches in a commercial framework

Commercial
Oved Amitay, Chief Business Officer, CENTOGENE AG
Apr 1212:30
Conference pass

250,000 Voices: What really matters -- data and insights from patients and caregivers

Rare Disease Advocacy World
Mike Porath, Founder And Chief Executive Officer, The Mighty
Apr 1212:30
Conference pass

AUSTRALIA: Experience with the regulatory framework for orphan drugs

Global Market and Patient Access
Martine Zimmermann, Global Head Of Regulatory Affairs, Alexion Pharmaceuticals
Apr 1212:30
Conference pass

Regulatory considerations for cellular and gene therapy products

Advanced Therapies - Manufacturing
Nirjal Bhattarai, Principal Investigator, Division Of Cellular And Gene Therapies, U.S. Food and Drug Administration (FDA)
Apr 1212:30
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Bringing Kymriah to patients globally - A unique experience

Rare Oncology
Pascal Touchon, Senior Vice President & Global Head Cell & Gene, Novartis Oncology
Apr 1212:35
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Pitch 22: Seclidemstat: A reversible LSD1 inhibitor for treatment of Ewing’s sarcoma

Pitch and Partner
Daniela Santiesteban, Business Development Manager, Salarius Pharmaceuticals
Apr 1212:45
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Pitch 23: HTD1801: a novel multifunctional oral drug candidate for primary sclerosing cholangitis (PSC)

Pitch and Partner
Apr 1213:20
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Lunch Keynote Panel: The patient role continuum - initiating research, starting companies, working with regulators and all the way to reimbursement

Keynotes
Moderator: Melissa Hogan, Founder And President, Project Alive
Jenn Mcnary, Duchenne Parent Advocate, Consultant
Steven Laffoon, President, Wylder Nation
Megan O'Boyle, Principal Investigator, Phelan-McDermid Syndrome Foundation
Lori Sames, Founder And President, Hannah's Hope Fund for GAN
Apr 1214:30
Conference pass

ROUNDTABLE 1: Gene therapy development – challenges, Issues, and solutions from the new frontier of rare disease therapies

Keynotes
Scott Schliebner, Vice President, Scientific Affairs, Rare Diseases, PRA Health Sciences
Apr 1214:30
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ROUNDTABLE 10: State Policy – continued efforts to advance rare disease policy and initiatives

Keynotes
Tim Boyd, Director Of State Policy, National Organization for Rare Disorders (NORD)
Apr 1214:30
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ROUNDTABLE 11: Alternative to viral vectors – reprogramming human T cell function and specificity with non-viral genome targeting

Keynotes
Theodore Roth, Managing Director/Phd Student, University of California, San Francisco
Apr 1214:30
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ROUNDTABLE 12: Gene therapy consortium – elevating patient groups’ collective knowledge of gene therapy and working with companies though the first ever patient group gene therapy consortium

Keynotes
Melissa Hogan, Founder And President, Project Alive
Apr 1214:30
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ROUNDTABLE 13: Disease burden – characterizing the burden of disease to more effectively educate regulators, physicians and payers

Keynotes
Karen Anderson, Senior Director - Medical Affairs And Rare Genetic Diseases, Agios Pharmaceuticals
Apr 1214:30
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ROUNDTABLE 14: Rare in Catalonia – Regional policies for an integrated health care in rare diseases

Keynotes
Roser Francisco Bordas, Head Technician, Rare Disease Program, Catalan Health Service. Spain, Catsalut
Apr 1214:30
Conference pass

ROUNDTABLE 15: FDA Patient Affairs - Learn new ways to share your perspective with the FDA

Keynotes
Samir Shaikh, Deputy Director, Patient Affairs Staff Office Of The Commissioner, Office Of Medical Products And Tobacco, U.S. Food and Drug Administration (FDA)
Andrea Furia-Helms, Director, Patient Affairs Staff, U.S. Food and Drug Administration (FDA)
Apr 1214:30
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ROUNDTABLE 16: Advanced manufacturing – building a cGMP in-house cell & gene therapy facility

Keynotes
Apr 1214:30
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ROUNDTABLE 17: Gene therapy commercial readiness - how to get yourself commercially ready for gene therapy. Practicalities of access agreements, concierge services and technology stewardship

Keynotes
Christina Poschen, Associate Consultant, Partners4Access
Apr 1214:30
Conference pass

ROUNDTABLE 18: Sustainable pricing – will value-based payment models be sustainable for the healthcare system?

Keynotes
Jay Weaver, Divisional Vice President, Enterprise Pharmacy, Blue Cross Blue Shield, Health Care Service Corp.
Apr 1214:30
Conference pass

ROUNDTABLE 19: Real World Evidence – updates from FDA

Keynotes
Khair Elzarrad, Deputy Director, Office of Medical Policy, Center for Drug Evaluation and Research, U.S. Food and Drug Administration (FDA)
Apr 1214:30
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ROUNDTABLE 2: Rare Disease Forecasting - Key drivers of uncertainty and how to deal with imperfect data

Keynotes
Doug Pfaff, Managing Director, Huron Life Sciences
Apr 1214:30
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ROUNDTABLE 20: Compassionate Use--- Ethical considerations for pre-approval access to gene therapy products

Keynotes
Carolyn Chapman, Member, CUPA, Division of Medical Ethics, Postdoctoral Fellow, Nyu Langone Health
Apr 1214:30
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ROUNDTABLE 21: Moving from n=1 to marketing application – clinical and regulatory considerations for accelerating rare disease product development

Keynotes
Zizi Imatorbhebhe, Senior Director, Strategic Development, Head Rare Disease Center of Excellence, PAREXEL International
Apr 1214:30
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ROUNDTABLE 3: Site selection – how can dedicated rare disease trial sites enable studies to be conducted on a timely and effective manner

Keynotes
Han Phan, Founder And Executive Officer, Rare Disease Research
Apr 1214:30
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ROUNDTABLE 4: Ethical considerations – for pharmaceutical and patient group partnerships, one size does not fit all – determining when to accept money and when to share information with patients

Keynotes
Gina Parziale, Executive Director, Alport Syndrome Foundation
Kara Eichelkraut, Senior Manager, Patient Advocacy, Reata Pharmaceuticals
Apr 1214:30
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ROUNDTABLE 6: Patient education - The importance of educating the patient community about rare diseases and newly approved and advanced therapies

Keynotes
Trista Morrison, Vice President Of Communications And Patient Advocacy, North America, Sobi
Apr 1214:30
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ROUNDTABLE 7: Patient voice - Working with external stakeholders, pharmaceutical partners, and regulators to further the impact of patient advocacy groups

Keynotes
Edmund Lim, Co-Founder, We CARE Journey
Julie Raskin, Executive Director, Congenital Hyperinsulinism International
Allyson Lock, President, NEW ZEALAND POMPE NETWORK
Isabelle Lousada, President And Chief Executive Officer, Amyloidosis Research Consortium
Monica Weldon, president, Bridge the Gap -SYNGAP Educatrion and Research Foundation
Vladimir Tomov, Chairman, National Alliance of People with Rare Diseases - Bulgaria
Sarah Procario, Advocacy-Communications Manager, The Hemophilia Foundation of Michigan
Apr 1214:30
Conference pass

ROUNDTABLE 8: Analyzing ICD-11 – challenges and complications around coding for rare diseases in the healthcare setting and understanding if the new ICD-11 will aid rare diseases coding

Keynotes
Christopher Chute, Professor Of Health Informatics, Johns Hopkins University
Apr 1214:30
Conference pass

ROUNDTABLE 9: Updates from Office of Orphan Product Development

Keynotes
Janet Maynard, Director, Office of Orphan Products Development, Food and Drug Administration, U.S. Food and Drug Administration (FDA)
Apr 1214:30
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Pitch 24: Re-thinking clinical trial design in DMD

Pitch and Partner
Apr 1214:40
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Pitch 25: TOL2 for treatment of myasthenia gravis

Pitch and Partner
Apr 1214:50
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Pitch 26: Treating TSC related facial angiofibromas and other skin conditions with topical sirolimus ointment

Pitch and Partner
Qiaolin Ren, Sr. Director, Product Development, Aucta Pharmaceuticals Llc
Apr 1215:00
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Pitch 27: NeOnc Technologies-delivering new drugs to the brain

Pitch and Partner
Thomas C Chen, Chairman And Chief Executive Officer, NeOnc Technologies
Apr 1215:10
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Pitch 28: YELIVA® (opaganib) for Cholangiocarcinoma and RHB-204 for pulmonary NTM infections

Pitch and Partner
Apr 1215:20
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Pitch 29: Fluorescence guided Photodynamic Therapy for the treatment of Glioblastoma and Esophageal cancers

Pitch and Partner
Ravindra Pandey, Founder and Chief Scientific Officer, Photolitec
Apr 1215:30
Conference pass

Pitch 30: Live Cultured Osteoblasts for treatment of Osteonecrosis of the Femoral Head

Pitch and Partner
Satyen Sanghavi, Chief Scientific Officer and Executive Director, Regrow Biosciences LLC , Texas - USA
Apr 1215:35
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Patients role in defining Centers of Excellence & standards of care

Rare Disease Advocacy World
Pamela Gavin, Chief Strategy Officer, National Organization for Rare Disorders (NORD)
Apr 1215:40
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Enabling drug discovery - the need for improved epidemiology in rare diseases

Clinical Development & Regulatory
Mathew Pletcher, Head Of Rare Disease Discovery, Roche
Apr 1215:40
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Executing post-market data collection to meeting regulatory requirements and payer needs

Pricing and Reimbursement
Ashish Dugar, Vice President, Global Medical Affairs, Sarepta Therapeutics
Apr 1215:55
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Rare Diseases International: Gateway to Global Rare Disease Policy and Actions

Rare Disease Advocacy World
Lisa Phelps, Council at Rare Disease International,Director, Strategic Planning & Development, National Organization for Rare Disorders (NORD)
Durhane Wong-Rieger, Council Chair at Rare Disease International, President, Canadian Organization For Rare Disorders
Apr 1216:05
Conference pass

Beyond one disease at a time: Platform approaches to rare disease therapeutics development

Clinical Development & Regulatory
Philip Brooks, Program Director, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)
last published: 20/Mar/19 21:45 GMT