2020 AGENDA

Oxon Hill, MD, 29 April 2020 - 1 May 2020

Schedule

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Apr 2909:00
Conference pass

Opening remarks and overview of payer landscape for orphan drugs

Payers Seminar
Anna Bucsics, Project Advisor, Mechanism of Coordinated Access to orphan medicinal products (MoCA)
Apr 2909:00
Conference pass

Messenger RNA therapy for inherited metabolic diseases

Next Generation Therapies Seminar
Paolo Martini, Chief Scientific Officer, Rare Diseases, Moderna Inc.
Apr 2909:20
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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
Apr 2909:20
Conference pass

Session title to be announced

Next Generation Therapies Seminar
Stephanie Tagliatela, Co-Founder And Chief Scientific Officer, Encoded Therapeutics
Apr 2909:40
Conference pass

Countries collaborating on Pricing and Reimbursement of Medicines in Europe – BeNeLuxA and IHSI

Payers Seminar
Francis Arickx, Head Of The Directorate Reimbursement Of Medicines And Pharmaceutical Policy, National Institute for Health and Disability Insurance RIZIV-INAMI
Apr 2909:40
Conference pass

Patient-customized oligonucleotide therapy for ultra-rare genetic diseases

Next Generation Therapies Seminar
Timothy Yu, Attending Physician, Investigator, Division Of Genetics And Genomics, Boston Children's Hospital
Apr 2910:00
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Patient registries - creation, maintenance, data ownership and data sharing

Patient Data Seminar
Anne Pariser, Director, Office Of Rare Diseases Research, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)
Apr 2910:30
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Pricing, profitability and patient access to rare disease medicines

Payers Seminar
Adam Hutchings, Managing Director, Dolon
Apr 2910:30
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The challenges of bringing gene therapy treatments for rare disease to clinical trial

Next Generation Therapies Seminar
Scott Dorfman, Chief Executive Officer, Odylia Therapeutics
Apr 2910:50
Conference pass

Session title to be announced

Next Generation Therapies Seminar
Sheila Mikhail, President And Co-Founder, Asklepios BioPharmaceutical Inc
Apr 2911:00
Conference pass

Data stewardship: Examining the responsibility financial burden on organizations to store and maintain data long-term

Patient Data Seminar
Speaker to be announced
Apr 2911:10
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Feasibility of targeting multiple diseases in one clinical trial using a platform vector gene therapy approach

Next Generation Therapies Seminar
Philip J Brooks, Program Director, National Center For Advancing Translational Sciences - Ncats, National Institutes of Health (NIH)
Apr 2911:30
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Session title to be announced

Next Generation Therapies Seminar
Apr 2911:40
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How do we ethically manage patient data in an age of integrated technologies and data capture in the clinical setting

Patient Data Seminar
Speaker to be announced
Apr 2912:00
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Communicating the WHY to patients & families around data sharing –easy to understand consent, privacy and long-term strategy

Patient Data Seminar
Speaker to be announced
Apr 2912:00
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Open Source Imaging Consortium against Fibrosing Interstitial Lung Diseases (ILD) - setting up business and legal framework for success

Patient Data Seminar
Zheng Yang, Head Of Technology And Data Innovation, Customer Value, Boehringer Ingelheim
Apr 2913:00
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Orphan drugs in Canada: the evolving landscape

Payers Seminar
Trevor Richter, Director, Common Drug Review And Optimal Use, CADTH
Apr 2913:00
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Creating incentive systems for patient data - exploring potential implications and challenges

Patient Data Seminar
Alex Sherman, Director, Center For Innovation And Bioinformatics, Harvard Medical School
Apr 2913:00
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Modifier gene therapy platform for inherited retinal diseases: one therapeutic for multiple diseases

Next Generation Therapies Seminar
Rasappa Arumugham, Chief Scientific Officer, Ocugen
Apr 2913:20
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Incentivizing data sharing among researchers - demonstrating benefits and fostering collaboration to further rare disease drug development

Patient Data Seminar
Salvatore La Rosa, Chief Scientific Officer, Children's Tumor Foundation
Apr 2913:40
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Rare Diseases: progress in the evaluation of drugs and advanced therapies in Québec, Canada

Payers Seminar
Luc Boileau, Chairman And Chief Executive Officer, INESSS
Apr 2913:40
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The cost savings of internationally sharing genomic data– proof of concept for federated data systems

Patient Data Seminar
Lynsey Chediak, Project Lead, Precision Medicine, World Economic Forum
Apr 2913:40
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Engineered Red Cells to Treat Metabolic Disorders

Next Generation Therapies Seminar
Speaker to be announced
Apr 2914:00
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Session to be announced

Payers Seminar
Steven Peskin, Associate Clinical Professor Of Medicine, Rutgers Rwj Medical School, Executive Medical Director Population Health And Transform, Horizon Blue Cross Blue Shield - New Jersey
Apr 2914:00
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Rare Disease Cures Accelerator (RDCA-DAP) – standardizing data quality and harmonizing with regulators

Patient Data Seminar
Vanessa Boulanger, Director Of Research Programs, NORD
Apr 2914:00
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Using Directed Evolution to Create Improved Gene Therapy Vectors

Next Generation Therapies Seminar
Casey Case, Senior Vice President Of Research And Development, 4D Molecular Therapeutics
Apr 2914:20
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Sponsored session available

Payers Seminar
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 or andre.singer@terrapinn.com
Apr 2914:20
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SPONSORED SESSION AVAILABLE

Patient Data Seminar
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 or andre.singer@terrapinn.com
Apr 2915:30
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PANEL:Having the end when collecting data - considerations for working with your patient community, international data privacy laws & ultimately sharing with external stakeholders to further drug dev

Patient Data Seminar
Moderator: Monica Weldon, President and Chief Executive Officer, Bridge The Gap Syngap Education And Research Foundation
Pamela Mace, President, Fibromuscular Dysplasia Society of America
Romina Foster-Bonds, Programs Director, CureDuchenne
Dimitar Yonchev, Patient Advocate Representative, Pro Retina Deutschland e.V.
Apr 2915:50
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Reimbursement process for orphan products in Argentina and overview of HTA development in South America

Patient Data Seminar
Speaker will be announced soon
Apr 2916:10
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Updates on rare diseases in Russia - HTA and reimbursement

Payers Seminar
Nuriya Musina, Head Of Development, Federal State Budgetary Institution, The Center For Healthcare Quality Assessme, Ministry of Health Russian Federation
Apr 2916:10
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Real World Data collection - With the FDA’s shift towards Real World Evidence to Support Clinical Trials, what types of data should patient groups collect?

Patient Data Seminar
Speaker will be announced
Apr 2916:30
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Orphan Drug specific reimbursement and market authorization in Turkey

Payers Seminar
Kagan Atikeler, Pharmacist, Turkish Medicines and Medical Devices Agency

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Apr 3008:25
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Chairperson’s opening remarks

Keynotes
Ellen Coleman, President And Chief Executive Officer, VOZ Advisors
Apr 3008:30
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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
Apr 3008:55
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Sponsored Keynote Address: Gene therapy: the future is now

Keynotes
Mark Rothera, President And Chief Executive Officer, Orchard Therapeutics
Apr 3009:20
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Keynote Panel: The moment is now for rare diseases, but how do we advance scientific innovation, data sharing, and the commercial landscape to expedite treatments for the remaining 6,000+ diseases

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
Janet Woodcock, Director Of Drug Evaluation And Research Center, Food and Drug Administration (FDA)
Apr 3011:25
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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
Thomas Kassberg, Chief Business Officer And Executive Vice President, Ultragenyx Pharmaceutical
Apr 3011:30
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Next generation human enzymes: Innovative solutions for patients with rare diseases

Clinical Development & Regulatory
Anthony Quinn, President And Chief Executive Officer, Aeglea Biotherapeutics
Apr 3011:30
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The commercial and societal opportunities of platform technologies - transitioning a business from a technology to commercialized products

Commercial
Sandy Macrae, President And Chief Executive Officer, Sangamo Therapeutics
Apr 3011:30
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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
Apr 3011:30
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How to best capture true value beyond the pill – methods for developing value of rare disease treatments

Pricing and Reimbursement
Speaker to be announced
Apr 3011:30
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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
Gavin Corcoran, Chief Research And Development Officer, Axovant Sciences
Apr 3011:30
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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
Apr 3011:30
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First things first – defining AI and its implications for rare diseases

Digital Health and AI
John Reynders, Vice President, Data Sciences, Genomics, And Bioinformatics, Alexion Pharmaceticals
Apr 3011:30
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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
Apr 3011:50
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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)
Apr 3011:50
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Commercializing ultra-ultra rare diseases – Case study of Progeria

Commercial
Jim Shaffer, Cbo, Eiger Biopharmaceuticals
Apr 3011:50
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Patient and caregiver reported outcomes: impacting the full continuum of clinical development, approvals and access

Rare Disease Advocacy World
Melissa Hogan, Founder And President, Project Alive
Apr 3011:50
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GOLD SPONSOR SESSION

Global Market and Patient Access
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 or andre.singer@terrapinn.com
Apr 3011:50
Conference pass

Zolgensma case study: Value and reimbursement challenges of gene therapies

Pricing and Reimbursement
Omar Dabbous, Vice President, Health Economics And Outcomes Research, AveXis
Apr 3011:50
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Regulatory considerations in getting the first RNAi therapy approved - case study of ONPATTRO

Advanced Therapies - Clinical
Sara Nochur, Senior Vice President Regulatory Affairs, Alnylam Pharaceuticals
Apr 3011:50
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GOLD SPONSOR SESSION

Advanced Therapies - Manufacturing
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
Apr 3011:50
Conference pass

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

Advanced Therapies - Commercialization
Parag Meswani, Senior Vice President Of Commercial Strategy And Operations, Axovant Sciences
Apr 3011:50
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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
Apr 3011:50
Conference pass

Access to genetic testing and genetic counselors: breakdown of barriers for rare disease patients

Diagnosis
Jodie Vento, Chair, Access Committee, National Society Of Genetic Counselors, Manager, Center For Rare Disease Therapy, Childrens Hospital of Pittsburgh
Apr 3012:10
Conference pass

Running rare disease registries in the digital age

Clinical Development & Regulatory
Lauren Neighbours, Neighbours, Senior Director, Regulatory Affairs, Optum Digital Research Network, Optum
Apr 3012:10
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Bringing gene therapies to patients through unique funding and unified mission

Commercial
Frederic Revah, Chief Executive Officer, Genethon
Apr 3012:10
Conference pass

Natural History Data & Patient Group/Industry Partnership – Pre-clinical through the Marketplace

Rare Disease Advocacy World
Ron Bartek, President, Director, And Founder, Friedreich's Ataxia Research Alliance
Apr 3012:10
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
Apr 3012:10
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How to further access to diagnostic programs and therapeutics in emerging rare disease markets

Global Market and Patient Access
Kamilla Gaitova, Head Of Center For Health Economics And Health Technology Assessment, Republican Center for Health Development, Kazakhstan
Samuel Agyei Wiafe, Founder Executive Director, Rare Disease Ghana Initiative
Harsha Rajasimha, Founder And Chairman, IndoUSrare
Edmund Lim, abc, We CARE Journey
Apr 3012:10
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
Apr 3012:10
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Making use of accelerated programs and design flexibility to expedite cell and gene therapies through the clinic

Advanced Therapies - Clinical
Speaker to be announced.
Apr 3012:10
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PANEL: Ensuring manufacturing capacity, flexibility, and quality for new drug modalities – risks and costs/benefits of enabling internal capacity versus outsourcing options

Advanced Therapies - Manufacturing
Moderator: Alfred Boyle, Senior Vice President Technical Operations, Alnylam Pharmaceuticals
Kim Warren, Head Of Operations, Avrobio
Scott Nickerson, Senior Vice President, Global Quality, Moderna Therapeutics
Derek Adams, Chief Technology And Manufacturing Officer, bluebird bio
Apr 3012:10
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Getting new advanced therapy products to patients through stable hospital system – considerations for site operations and reimbursement

Advanced Therapies - Commercialization
Chuck Bucklar, Group Vice President, Commercial Operations North America, BioMarin Pharmaceutical Inc.
Apr 3012:10
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Integrating quantitative imaging and machine intelligence into the management of rare pediatric conditions

Digital Health and AI
Marius George Linguraru, Principal Investigator, Children's National Medical Center
Apr 3012:10
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Importance of consulting counselors prior to using consumer genetics

Diagnosis
Speaker to be announced.
Apr 3012:30
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A partnership that lasts - key considerations when working with CROs on rare disease studies

Clinical Development & Regulatory
Jonathan Kornstein, Executive Director, Program Strategy, Premier Research
Apr 3012:30
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Sponsored by Charles River Associates

Commercial
More information will be available soon.
Apr 3012:30
Conference pass

Natural History Data & Patient Group/Industry Partnership – Pre-clinical through the Marketplace

Rare Disease Advocacy World
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 or andre.singer@terrapinn.com
Ron Bartek, President, Director, And Founder, Friedreich's Ataxia Research Alliance
Apr 3012:30
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
Apr 3012:30
Conference pass

GOLD SPONSOR SESSION

Advanced Therapies - Clinical
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 or andre.singer@terrapinn.com
Apr 3012:30
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Communication considerations when commercializing advanced therapies

Advanced Therapies - Commercialization
Sponsored by OPEN Health
Apr 3012:30
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Sponsored by HVH Precision Analytics

Digital Health and AI
More information will be available soon.
Apr 3012:30
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GOLD SPONSOR SESSION

Diagnosis
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 or andre.singer@terrapinn.com
Apr 3013:00
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Lunch Keynote Panel: Examining industry data strategies and use of AI to advance rare disease patient identification, R&D, and commercialization

Keynotes
Pamela Gavin, Chief Strategy Officer, National Organization for Rare Disorders (NORD)
Sean Khozin, Global Head Of Data Strategy, Johnson & Johnson
Thomas Abbott, Head, Real World Data and Evidence, Astellas Pharma
Apr 3014:20
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ROUNDTABLE 1: Reducing study burden – approaches for assessing and reducing the burden of clinical trial participation for patients

Keynotes
Scott Schliebner, Vice President, Scientific Affairs, Rare Diseases, PRA Health Sciences
Apr 3014:20
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ROUNDTABLE 10: Patient and provider experiences - A tailored tech + talent approach to enhance the journey

Keynotes
Jan Nielsen, Division President, Patient Solutions, AssistRx Patient Solutions
Apr 3014:20
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ROUNDTABLE 11: Orphan Drug Act-assessing the impact since 1983 and looking forward

Keynotes
Rachel Sher, Vice President Policy And Regulatory Affairs, NORD
Apr 3014:20
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ROUNDTABLE 12: Real World Evidence – examining recent cases of real world evidence for approvals and reimbursement in Europe

Keynotes
Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
Apr 3014:20
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ROUNDTABLE 14: Rare Cancer – advancing best practices and partnerships for rare cancer diagnosis and drug

Keynotes
Kristin Anthony, President, Pten Hamartoma Tumor Syndrome Foundation
Abby Sandler, Executive Director, Mypart, Center For Cancer Research,, National Institutes of Health
Apr 3014:20
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ROUNDTABLE 15: Title to be announced

Keynotes
Andrea Furia-Helms, Director, Patient Affairs Staff, U.S. Food and Drug Administration (FDA)
Apr 3014:20
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ROUNDTABLE 16: Diversity & inclusion - The importance of D&I in a global company - exploring its impact on employee morale, engagement and retention of workforce, and also the need for D&I in clinical

Keynotes
Sara Nochur, Senior Vice President Regulatory Affairs, Alnylam Pharaceuticals
Apr 3014:20
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ROUNDTABLE 17: Internal Patient Perspective – blocking and tackling actionable implementation of patient voice throughout company functions

Keynotes
Jennifer Helfer, Director, Global Patient Advocacy Lead, bluebird bio
Apr 3014:20
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ROUNDTABLE 18: Splinter Groups - How to advance your organization, unify communities and focus on your mission while navigating challenges with personalities, emotions and leadership differences among

Keynotes
Debbie Drell, Director Of Membership, National Organization for Rare Disorders (NORD)
Apr 3014:20
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ROUNDTABLE 19: Diagnostic access programs – advancing access to rare disease diagnosis in emerging markets

Keynotes
Nataliya Shandarovska, Patient Advocate, National Alliance for Rare Diseases Support - Malta
Vladimir Tomov, Chairman, National Alliance of People with Rare Diseases - Bulgaria
Apr 3014:20
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ROUNDTABLE 2: Synthetic control arms - using RWE to streamline the regulatory process

Keynotes
Chelsea Catsburg, Engagement Manager, BluePrint Orphan
Apr 3014:20
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ROUNDTABLE 3: The patient journey - improving clinical trial performance and delivering better study outcomes through a personalized approach to the patient experience

Keynotes
Scott Gray, Chief Executive Officer, Clincierge
Apr 3014:20
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ROUNDTABLE 4: Early Access – effective measures for successful EAPs

Keynotes
Anne Cropp, Chief Scientific Officer, Early Access Care
Apr 3014:20
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ROUNDTABLE 5: Patient-Focused Drug Development – how to get started

Keynotes
Meagan Spychala, Assistant Vice President Of Patient Engagement And Program Strategy, Rho, Inc
Apr 3014:20
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ROUNDTABLE 6: 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
Apr 3014:20
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ROUNDTABLE 7: Gene therapy CMC – early pDNA considerations for late stage success

Keynotes
Michelle Berg, President, Gmp Nucleic Acids, Aldevron
Apr 3014:20
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ROUNDTABLE 8: Forecasting & epidemiology - rare-disease epidemiology methods and resources to support commercial forecasts

Keynotes
David Lapidus, President, Lapidus Data
Apr 3014:20
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ROUNDTABLE 9: Access Programs - discussion on the value of rare and orphan access programs, next generation registries and post marketing surveillance for products

Keynotes
Becky Thompson, Director Integrated Solutions, PAREXEL
Apr 3015:25
Conference pass

Science-Driven and Patient-Focused: Rare Disease Patient and Caregiver Inclusion in Early Therapeutic Discovery

Clinical Development & Regulatory
Luke Rosen, Head Of Patient Engagement And Digital Health Experience, Ovid Therapeutics
Apr 3015:25
Conference pass

Taking the Leap--Establishing a Global Footprint?

Commercial
Sujay Kango, Chief Commercial Officer, Acceleron Pharma
Apr 3015:25
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
Apr 3015:25
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
Apr 3015:25
Conference pass

Hurdles for long term contracting in the US

Pricing and Reimbursement
Speaker to be announced
Apr 3015:25
Conference pass

Examining challenges in gene therapy clinical development and the future of the field for rare diseases

Advanced Therapies - Clinical
Olivier Danos, Chief Scientific Officer, Regenxbio Inc
Apr 3015:25
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Gene therapy manufacturing and testing at Sarepta

Advanced Therapies - Manufacturing
Palani Palaniappan, Head Of Technical Operations And Andover Site, Sarepta Therapeutics
Apr 3015:25
Conference pass

Setting up international centers of excellence for advanced therapies

Advanced Therapies - Commercialization
Speaker to be announced
Apr 3015:25
Conference pass

Update from FDA on development and integration of digital health and AI tools in clinical review

Digital Health and AI
Speaker to be announced
Apr 3015:25
Conference pass

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

Diagnosis
Linn Parrish, Head Of Csr Innovation And Stakeholder Engagement, Corporate Communications And Public Affairs, Takeda
Apr 3015:45
Conference pass

Case Study: Transforming training protocols to incorporate patients and caregivers’ voice into all aspects of clinical development

Clinical Development & Regulatory
Mary Mcgowan, Executive Director, The Myositis Association
Apr 3015:45
Conference pass

The journey from R&D to commercialization: how to build & lead a growth-ready organization

Commercial
Speaker to be announced
Denise Scots Knight, Chief Executive Officer And Co-Founder, Mereo BioPharma
Apr 3015:45
Conference pass

GOLD SPONSOR SESSION

Rare Disease Advocacy World
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
Apr 3015:45
Conference pass

COLOMBIA: Market access challenges and updated orphan drug regulation

Global Market and Patient Access
Speaker to be announced
Apr 3015:45
Conference pass

Innovative contracting for cell and gene therapy for Europe

Pricing and Reimbursement
Alexander Natz, Secretary General, European Confederation of Pharmaceutical Entrepreneurs (Belgium)
Apr 3015:45
Conference pass

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

Advanced Therapies - Clinical
Wilson Bryan, Director, Office Of Tissues And Advanced Therapies, Cber, FDA
Apr 3015:45
Conference pass

Title to be announced

Advanced Therapies - Manufacturing
Speaker to be announced
Apr 3015:45
Conference pass

How best to work with hospital partners to operationalize delivery of cell and gene therapies to rare disease patients

Advanced Therapies - Commercialization
Speaker to be announced.
Apr 3015:45
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
Apr 3015:45
Conference pass

Accelerating patient diagnosis by combining research insights, education and engagement with key stakeholders

Diagnosis
Brigette Tippin Davis, Senior Vice President, R&D, Ambry Genetics
Apr 3016:05
Conference pass

Patients preference studies and multi-criteria decision analyses - their value and impact for Rare Diseases regulatory submissions

Clinical Development & Regulatory
Leo Russo, Senior Director, Epidemiology Worldwide Safety And Regulatory, Pfizer
Apr 3016:05
Conference pass

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

Commercial
Speaker to be announced
Apr 3016:05
Conference pass

Moving treatments from lab to trial – what are the current barriers and how are patient organizations overcoming these challenges

Rare Disease Advocacy World
Donald Lo, Director, Therapeutic Development Branch, Division Of Pre-Clinical Innovation, National Center for Advancing Translational Sciences - NCATS
Wyatt Yue, Principal Investigator, Associate Professor, Lead Scientist, Rare Disease Research, Oxford University Structural Genomics Consortium
Amy Gray, Chief Executive Officer, Charcot-Marie-Tooth Association
Apr 3016:05
Conference pass

INDIA: Update on advanced therapies regulation and clinical development opportunities for rare diseases

Global Market and Patient Access
Speaker to be announced
Apr 3016:05
Conference pass

Negotiating managed entry agreements vs. money back guarantees

Pricing and Reimbursement
Speaker to be announced
Apr 3016:05
Conference pass

Title to be announced

Advanced Therapies - Clinical
Speaker to be announced.
Apr 3016:05
Conference pass

Changing the definition and outlook of comparability testing for gene therapy products

Advanced Therapies - Manufacturing
Speaker to be announced
Apr 3016:05
Conference pass

Operational consideration for getting new advanced therapy products in stable system – hospital perspective

Advanced Therapies - Commercialization
Cynthia Tifft, Deputy Clinical Director, Office Of The Clinical Director, National Human Genome Research, National Human Genome Research Institute
Apr 3016:05
Conference pass

Use of AI in quantitative imaging for patient identification in rare pediatric conditions - the need for increased collaboration to help standardize data collection and overcoming Heterogeneity of ima

Digital Health and AI
Speaker to be announced.
Apr 3016:05
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
Apr 3016:25
Conference pass

Following patients with rare and orphan diseases from clinical trials to market

Clinical Development & Regulatory
Andrew Ecob, Vice President Of Real World Evidence Strategy, PAREXEL
Apr 3016:25
Conference pass

GOLD SPONSOR SESSION

Commercial
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
Apr 3016:25
Conference pass

GOLD SPONSOR SESSION

Global Market and Patient Access
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
Apr 3016:25
Conference pass

Sponsored by Charles River Associates

Pricing and Reimbursement
More information will be available soon.
Apr 3016:25
Conference pass

Session will be hosted by Catalent Pharma Solutions

Advanced Therapies - Clinical
More information will be available soon.
Apr 3016:25
Conference pass

GOLD SPONSOR SESSION

Advanced Therapies - Manufacturing
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
Apr 3016:25
Conference pass

GOLD SPONSOR SESSION

Advanced Therapies - Commercialization
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
Apr 3016:25
Conference pass

GOLD SPONSOR SESSION

Digital Health and AI
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
Apr 3016:25
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
Apr 3017:25
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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
Apr 3017:35
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Keynote Panel: Enabling delivery of cell and gene therapies to patients: resolving continued manufacturing bottlenecks

Keynotes
Moderator: James Miskin, Chief Technical Officer, Oxford Biomedica
Ran Zheng, Chief Technology Officer, Orchard Therapeutics
Yong Dai, Chief Technology Officer, Prevail Therapeutics
Jan Thirkettle, Chief Development Officer, Freeline Therapeutics
Mayo Pujols, Vice President, Head Of Global Cell And Gene Technical Development And Manufacturing, Novartis

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May 108:25
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Chairperson’s opening remarks

Keynotes
Scott Gray, Chief Executive Officer, Clincierge
May 108:55
Conference pass

Keynote Address: Pricing one-time treatments based on value – impact on patients, the health care system and society

Keynotes
David Lennon, President, AveXis
May 109:20
Conference pass

Keynote address: Industry perspective on value and access strategies – developing a cohesive agenda and unified framework for orphan drugs

Keynotes
Jim Lang, Chief Executive Officer, EVERSANA
May 109:40
Conference pass

Keynote Panel: Creating a new era of access to treatments & cures for rare disease patients

Keynotes
Sophie Schmitz, Managing Partner, Partners4Access
Peter Saltonstall, Chief Executive Officer, National Organization for Rare Disorders (NORD)
Diane Berry, Vice President, Global Health Policy And Government Affair, Sarepta Therapeutics
May 111:30
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
May 111:30
Conference pass

Commercialization strategy and capabilities in the context of M&A and partnership opportunities

Commercial
Vikram Karnani, Executive Vice President, Chief Commercial Officer, Horizon Therapeutics
May 111:30
Conference pass

Determining next steps in developing science: in-licensing from pharma, out-licensing IP or partnering

Rare Disease Advocacy World
Steven Laffoon, President, Wylder Nation
May 111:30
Conference pass

SOUTH KOREA: Overview of the regulatory and reimbursement process or orphan products and the rare disease landscape in South Korea

Global Market and Patient Access
Jeonghoon Ahn, Director, National Evidence-based Healthcare Collaborating Agency
May 111:30
Conference pass

Evolving evidence and bridging toward value-based payments

Pricing and Reimbursement
Shannon Baumann, Senior Director, Access Value And Evidence Strategy, bluebird bio
May 111:30
Conference pass

Effects of innovative technologies in clinical trials: How can AI and digital health solutions lower costs of clinical trials and eventually impact overall drug pricing for rare disease therapies

Digital Health and AI
Dan Tierno, Strategic Implementation Manager, Global Data Sciences And Analytics, Bayer US LLC
May 111:30
Conference pass

Investors Panel: Evaluating venture capital investing in the rare disease & orphan space

Pitch and Partner
Art Pappas, Managing Partner, Pappas Capital
Jeffrey Schwartz, Managing Director, Bain Capital Life Sciences
Daniel O'Mahony, Partner, Seroba Life Sciences
May 111:50
Conference pass

A new era of alternative trial designs informed by patient focused clinical reported outcome measures – case study on CORE program

Clinical Development & Regulatory
Alison Skrinar, Vice President, Clinical Outcomes Research And Evaluation, Ultragenyx Pharmaceutical
May 111:50
Conference pass

From non-profit to for-profit: a path for expediting drug development for Angelman syndrome

Rare Disease Advocacy World
Allyson Berent, Chief Operating Officer, Genetx Biotherapeutics, Chief Science Officer, Foundation for Angelman Syndrome Therapeutics
Paula Evans, Chief Executive Officer, Genetx Biotherapeutics, Chairperson, Foundation for Angelman Syndrome Therapeutics
May 111:50
Conference pass

CHINA: China's policy on rare disease & orphan drugs: opportunities and challenges

Global Market and Patient Access
Kevin Huang, President, Chinese Organization for Rare Disorders
May 111:50
Conference pass

Case Study: SPINRAZA in SMA - broadening access to new populations through real world evidence and data generation

Pricing and Reimbursement
Nicholas Simmons-Stern, Associate Director, Global Value And Access, Biogen
May 111:50
Conference pass

Capacity crunch? The future of manufacturing within cell & gene therapy

Advanced Therapies - Manufacturing
Sponsored by Catalent Paragon Gene Therapy
May 111:50
Conference pass

The power of AI to improve rare disease patient outcomes at scale: a case study in NTM

Digital Health and AI
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
Jonathan Woodring, Executive Vice President And General Manager, IPM.ai
Areejit Phukan, Director, Analytics And Forecasting, Insmed Inc
May 112:10
Conference pass

Panel: Best practices for venture philanthropy and industry partnership

Rare Disease Advocacy World
Debra Miller, Founder And President, CureDuchenne
May 112:10
Conference pass

JAPAN: Update on rare disease opportunities and barriers in Japan

Global Market and Patient Access
Yukiko Nishimura, President, ASrid Japan
May 112:10
Conference pass

PANEL: Novel approaches to analytical methods across modalities: engaging regulators on analytical testing requirements, approaches to control strategies, and matching analytic capacity for clinical a

Advanced Therapies - Manufacturing
Mark Galbraith, Head Of Quality Control And Analytical Sciences, Spark Therapeutics
May 112:10
Conference pass

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

Diagnosis
Khrystal K Davis, President, Texas Rare Alliance
May 112:30
Conference pass

GOLD SPONSOR SESSION

Clinical Development & Regulatory
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 112:30
Conference pass

GOLD SPONSOR SESSION

Commercial
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 112:30
Conference pass

GOLD SPONSOR SESSION

Global Market and Patient Access
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 112:30
Conference pass

GOLD SPONSOR SESSION

Pricing and Reimbursement
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 112:30
Conference pass

GOLD SPONSOR SESSION

Advanced Therapies - Commercialization
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 112:30
Conference pass

GOLD SPONSOR SESSION

Digital Health and AI
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 112:30
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GOLD SPONSOR SESSION

Diagnosis
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 112:30
Conference pass

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

Rare Oncology
Dmitry Petrov, Head of Medical Writing and Pharmacovigilance, ICRC Weyer GmbH
May 114:20
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ROUNDTABLE 1: Guided by patients – how, when, and why to build authentic partnerships with patients and patient organizations

Keynotes
Scott Schliebner, Vice President, Scientific Affairs, Rare Diseases, PRA Health Sciences
May 114:20
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ROUNDTABLE 10: Investing in digital - venture capital perspective on digital health and AI investment landscape to facilitate patient identification, clinical enrollment and trial data capture for rar

Keynotes
Brett Cook, Senior Associate, F Prime Capital
May 114:20
Conference pass

ROUNDTABLE 11: Drug Repurposing – Approaches to help industry repurpose drugs and existing research to further therapies for rare diseases and cancers

Keynotes
Diego Ardigo, R&D Rare Diseases Unit Head, Chiesi Farmaceutici SpA
Barbara Goodman, President And Chief Operating Officer, Cures Within Reach
May 114:20
Conference pass

ROUNDTABLE 12: Value-based contracting – description to be determined

Keynotes
Marianne Hamilton Lopez, Research Director, Duke Margolis Center for Health Policy
May 114:20
Conference pass

ROUNDTABLE 13: Newborn Screening – Achieving the promise of newborn screening for rare diseases through smart advocacy and sound policy

Keynotes
Rebecca Abbott, Office Of Government Affairs, March of Dimes
May 114:20
Conference pass

ROUNDTABLE 14: Patients and Pharma - examining best practices for partnering between patient groups and industry

Keynotes
Vivian Fernandez, Director Of Patient Advocacy, Regenxbio Inc
May 114:20
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ROUNDTABLE 16: Pediatric populations – emerging ethical issues surrounding genetic interventions in infants and children, including research design, patient autonomy, sibling and family involvement, f

Keynotes
Lesha Shah, Co-Chair, Pediatric Gene Therapy And Medical Ethics Working Group, Assistant Professor Of Psychiatry, Icahn School Of Medicine, The Mount Sinai Hospital
May 114:20
Conference pass

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

Keynotes
Akshay Kumar, Partner, Partners4Access
May 114:20
Conference pass

ROUNDTABLE 20: Value Framework - examining the potential impact of Quality Adjusted Life Year (QALY) metric on access to medicines for those living with chronic conditions – what has changed and what

Keynotes
Sara Traigle Van Geertruyden, Executive Director, Partnership to Improve Patient Care (PIPC)
Thayer Surette, Deputy Director, Partnership to Improve Patient Care (PIPC)
May 114:20
Conference pass

ROUNDTABLE 21: Primary endpoints – using patient reported outcomes as primary endpoints instead of secondary

Keynotes
Terry Jo Vetters Bichell, Founder And Director, COMBINEDBrain
May 114:20
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Roundtable 24: Dosing and durability – challenges in gene therapy clinical development

Keynotes
Rafael Escandon, Vice President Of Development Operations, BridgeBio
May 114:20
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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
May 114:20
Conference pass

Roundtable 4: Expanded Access – practical considerations to turn early access strategy into a reality

Keynotes
Ruth Rostron, Vice President Of Project Management, Inceptua Medicines Access
May 114:20
Conference pass

Roundtable 5: The discussion will be sponsored by Covance

Keynotes
More information will be available soon.
May 114:20
Conference pass

ROUNDTABLE 6: Integrating epidemiology, natural history and nonclinical pharmacology/toxicology data to inform clinical trial design and the regulatory strategy

Keynotes
Janice Lansita, Practice Director, Biopharmaceuticals and Pharmaceuticals, EpidStrategies, A Division of ToxStrategies
Jon Fryzek, Practice Director, Epidemiology, EpidStrategies, A Division of ToxStrategies
May 114:20
Conference pass

ROUNDTABLE 7: Patient Support Programs – best practices in designing patient support programs in rare patient populations

Keynotes
Chad Forinash, Senior Director, Pharmacy And Clinical Services, PharmaCord
May 114:20
Conference pass

ROUNDTABLE 8: : Investing in Ultra-Rare: Strategies for raising capital, allocating clinical resources and determining roadmaps for small patient populations

Keynotes
Anjan Aralihalli, Venture Partner, C.T.I. life Sciences Fund
Shawn Ritchie, Chief Executive Officer, Med-Life Discoveries
May 114:20
Conference pass

ROUNDTABLE 9: 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 (FDA)
May 115:25
Conference pass

GOLD SPONSOR SESSION

Clinical Development & Regulatory
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 115:25
Conference pass

GOLD SPONSOR SESSION

Commercial
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 115:25
Conference pass

GOLD SPONSOR SESSION

Rare Disease Advocacy World
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 115:25
Conference pass

GOLD SPONSOR SESSION

Global Market and Patient Access
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 115:25
Conference pass

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

Pricing and Reimbursement
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
Francis Pang, Vice President Global Market Access, Orchard Therapeutics
May 115:25
Conference pass

GOLD SPONSOR SESSION

Advanced Therapies - Manufacturing
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 115:25
Conference pass

GOLD SPONSOR SESSION

Advanced Therapies - Commercialization
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 115:25
Conference pass

GOLD SPONSOR SESSION

Digital Health and AI
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 115:25
Conference pass

GOLD SPONSOR SESSION

Diagnosis
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 115:25
Conference pass

GOLD SPONSOR SESSION

Rare Oncology
If you are interested in sponsoring this session, contact André Singer now at +1 646 619 1797 orandre.singer@terrapinn.com
May 115:45
Conference pass

Epidemiology in the new era of Real World Evidence

Commercial
Kui Huang, Rare Disease Epidemiology Lead Strategist, Pfizer
May 115:45
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
May 115:45
Conference pass

Overview over of current US proposals to restrain drug prices

Pricing and Reimbursement
John Rother, Chief Executive Officer, National Coalition On Health Care
May 115:45
Conference pass

Newborn Screening Efforts- Collaborating with Patient Communities to Develop Empowering Educational Resources for Advocates

Diagnosis
Emily Fields, Senior Manager, Patient Advocacy, bluebird bio
May 115:45
Conference pass

MyPART: My Pediatric and Adult Rare Tumor Network – a platform for patient engagement in rare solid tumors research

Rare Oncology
Karlyne Reilly, Senior Associate Scientist And Section Head, National Cancer Institute
May 116:05
Conference pass

The Burden of Access on Patients and Families

Rare Disease Advocacy World
Mindy Cameron, Advocacy Director, Little Hercules Foundation
May 116:05
Conference pass

Case Study: ScreenPlus pilot program - unique cost structure, multiple-assay result verification framework and long-term follow up and data capture strategy

Diagnosis
Melissa Wasserstein, Chief, Division Of Pediatric Genetic Medicine, Department Of Pediatrics,, Montefiore Medical Center
May 116:25
Conference pass

Addressing future hurdles for highly priced drugs - The potential need for payment in preapproval access programs

Clinical Development & Regulatory
Kelly Folkers, Senior Research Associate, Division Of Medical Ethics,, NYU Langone Medical Center
May 116:25
Conference pass

ITALY: The Italian experience - Tools for market access and reimbursement for orphan products in Italy

Global Market and Patient Access
Arrigo Paciello, Assessor, AIFA
May 116:25
Conference pass

Patient and clinician-facing digital technologies to capture the full patient journey

Digital Health and AI
Michael Brudno, Scientific Director Of Centre For Computational Medicine, Hospital for Sick Children
May 116:25
Conference pass

The need for broader stakeholder engagement and coalition building in preparation for expanded newborn screening, services and access

Diagnosis
Pam Crowley Andrews, Co-Founder And Executive Director, Firefly Fund
Allison May Rosen, Representative, Firefly Fund
last published: 21/Jan/20 15:45 GMT