2019 Agenda

 

Oxon Hill, MD, 10 - 12 April 2019

Schedule

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Apr 1108:30
Conference pass

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 Fireside Chat: Re-thinking and reshaping “rare” – continuing the march towards curative therapies for the rare disease community by fostering scientific and regulatory increased collaboration

Keynotes
Christopher Austin, Director, National Center for Advancing Translational Sciences (NCATS), National Center for Advancing Translational Sciences - NCATS
Apr 1109:20
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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
Sandy Macrae, President and Chief Executive Officer, Sangamo Therapeutics
Mark Rothera, President and Chief Executive Officer, Orchard Therapeutics
Andre Choulika, Chairman & 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, VP Patient Affairs and Community Engagement, 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 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
Apr 1111:25
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AUSTRALIA: Overview of Prescription Medicines Regulation Pathway and three new alternative pathways to register orphan drugs in Australia

Global Market and Patient Access
Kaye Robertson, Assistant Director, Australian Government Department of Health
Apr 1111:25
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Update on LUXTURNA implementation and outcomes-based efforts

Pricing and Reimbursement
Sarah Pitluck, Head, Global Pricing & Reimbursement, Spark Therapeutics
Apr 1111:25
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Regenerative Medicine Advanced Therapy Designation

Advanced Therapies - Clinical
Julie Tierney, Senior Policy Advisor for Strategic Planning and Legislation, F.D.A. U.S. Food and Drug Administration
Apr 1111:25
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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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Investors Panel: Multi-stakeholder Investing in Cell therapy – evaluating modality risk, understanding the science

Pitch and Partner
Moderator: Brian Bronk, Head of External Innovation, Rare Diseases, Sanofi
Heather Behanna, Principal, Sofinnova Ventures
Debra Miller, Founder and President, CureDuchenne
Walter Kowtoniuk, Principle, Third Rock Ventures, LLC
Apr 1111:50
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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, Founder and Chief Executive Officer, Solid GT, LLC
Apr 1111:50
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Taking on existing commercialized products

Commercial
Vikram Karnani, Executive Vice President, Chief Commercial Ofiicer, Horizon Pharma
Apr 1111:50
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MENA: updates on market access regulation, rare disease clinical trial enrollment, and patient populations

Global Market and Patient Access
Amin Alamiri, Asst. Undersecretary of Public Health Policy & Licensing, United Arab Emirates Ministry of Health and Prevention
Apr 1111:50
Conference pass

In-housing vs outsourcing manufacturing and process development – assessing benefits and risk

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, Associate Director, Developmental Therapeutics Program Division of Cancer Treatment and Diagnosis,, National Cancer Institute
Apr 1112:15
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Patient advocacy in clinical development for rare diseases

Clinical Development & Regulatory
Juliet Moritz, Executive Director, Program Strategy, Rare Disease & Pediatrics/Patient Engagement, Premier Research
Apr 1112:15
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Biopharma sponsored genetic testing programs - accelerating R&D and getting on-market therapies to patients and clinicians who need them

Commercial
Daniel Anderson, Head of Commercial Partnerships, Invitae
Apr 1112:15
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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
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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
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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
Deborah Gill, Professor, Oxford University Press
Apr 1112:15
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How Cellectis evolves its gene-editing enabled off-the-shelf CAR-T manufacturing to meet cell therapy commercial standards

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 & CEO, Friends of Cancer Research
Apr 1112:15
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How the first orphan drug accelerator is successfully advancing rare genetic disease therapies

Pitch and Partner
Imran Babar, Chief Business Officer, Cydan II, Inc.
Apr 1112:40
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Patient-focused rare disease clinical trial protocols: patient-centered outcomes and beyond

Clinical Development & Regulatory
Margaret Vernon, Vice President and General Manager, Outcomes Research, Evidera
Kevin Marsh, Senior Research Scientist and European Director of Modelling, Evidera
Apr 1112:40
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Use of real world data to support the path to approval for advanced therapies in rare diseases

Advanced Therapies - Clinical
Mariah Baltezegar, Executive Director Clinical Development, Syneos Health
Apr 1113:00
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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 & Scientific Officer, Worldwide Clinical Trials
Apr 1114:31
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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 1114:33
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ROUNDTABLE 3: Epidemiology & forecasting – value, challenges, and methods for rare diseases

Keynotes
David Lapidus, President, Lapidus Data
Apr 1114:34
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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 1114:35
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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 1114:36
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ROUNDTABLE 6: Patient centered development – developing truly patient centric clinical trials for rare diseases

Keynotes
Jamie Arnott, Senior Project Director, Rho, Inc
Apr 1114:37
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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 1114:38
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ROUNDTABLE 8: Driving diagnosis – steps industry can take to improve the diagnosis of rare disease

Keynotes
Kate Grady, Senior Director, Huron Life Sciences
Apr 1114:40
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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, National Center for Advancing Translational Sciences - NCATS
Apr 1114:41
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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 1114:42
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ROUNDTABLE 12: Chilling investment? – the potential implications of increased public scrutiny of rare disease drug prices in the US

Keynotes
Walter Kowtoniuk, Principle, Third Rock Ventures, LLC
Apr 1114:43
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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 1114:44
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ROUNDTABLE 14: Rare in India – engaging patients with rare diseases in India with global clinical trials and resources

Keynotes
Harsha Rajasimha, Co Founder, Organization for Rare Diseases India
Apr 1114:45
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ROUNDTABLE 15: Commercial analytics – how to use data to overcome challenges in rare diseases

Keynotes
Vikram Karnani, Executive Vice President, Chief Commercial Ofiicer, Horizon Pharma
Apr 1114:46
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ROUNDTABLE 16: 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
Apr 1114:47
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ROUNDTABLE 17: Internal organization and policy - data sharing and ownership, internal structure and funding, scientific advisors and more

Keynotes
Eric Hartman, Executive Director, Choroideremia Research Foundation Inc
Alastair Kent, Ambassador, Genetic Alliance U.K.
Terri Klein, President and CEO, National M.P.S. Society
Apr 1114:48
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ROUNDTABLE 18: RWD from EAPs – Clinical outcomes data capture in global early access programs

Keynotes
Stuart Bell, Vice President, Consulting, Inceptua
Apr 1115:40
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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
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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 contracts models for cell and gene therapies one time treatment

Pricing and Reimbursement
Alexander Natz, Secretary General, EUCOPE
Apr 1115:40
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Title to be announced

Advanced Therapies - Clinical
Ilan Irony, Deputy Director Division of Clinical Evaluation and Pharmacology, Food and Drug Administration
Apr 1115:40
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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 1116:00
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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
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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

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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Innovative Early Phase Clinical Trials Designs

Advanced Therapies - Clinical
Julie Lin, Global Project Head, Early Clinical and Business Development, Sanofi Genzyme Corp
Apr 1116:20
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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, US Food and Drug Administration/HSS
Apr 1116:20
Conference pass

Topic to be announced

Global Market and Patient Access
Anant Murthy, Vice President of Market Access & Pricing, Alnylam
Apr 1116:20
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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
Marianne Hamilton Lopez, Research Director, Duke Margolis Center for Health Policy
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
Apr 1117:40
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Keynote Panel: Patient Data – collection, ownership, regulatory considerations and its role in ending the diagnostic odyssey

Keynotes
Simon Kos, Chief Medical Officer, Microsoft
Yann Le Cam, Chief Executive Officer, EURORDIS
Peter Saltonstall, President and Chief Executive Officer, National Organization for Rare Disorders
Linn Parrish, Head Of Corporate Responsibility, Shire

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Apr 1211:15
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 1211:15
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Transitions in the commercial development

Commercial
Mark Baglin, Vice President of Global Marketing, Alnylam Pharmaceuticals
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
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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
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Orphan drugs in Canada: the evolving landscape

Pricing and Reimbursement
Trevor Richter, Director, Common Drug Review and Optimal Use, CADTH
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, Biologics Evaluation And Research, Food And Drug Administration UK
Apr 1211:15
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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
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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
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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
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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 1211:40
Conference pass

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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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

The access challenge for rare disease technologies- doing it the NICE way

Pricing and Reimbursement
Sheela Upadhyaya, Associate Director Highly Specialised Technologies, National Institute for Health and Care Excellence
Apr 1212:05
Conference pass

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 1213:20
Conference pass

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

Keynotes
Moderator: Melissa Hogan, President, Project Alive
Jenn Mcnary, Patient advisory lead, Early Access Care
Steven Laffoon, Founder and President, Wylder Nation
Megan O'Boyle, Principal Investigator, Phelan-McDermid Syndrome Foundation
Lori Sames, Founder and President, Hannah's Hope Fund
Apr 1214:31
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 1214:32
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ROUNDTABLE 2: Rare disease forecasting – the importance of patient numbers and how to deal with imperfect data

Keynotes
Doug Pfaff, Senior Director, Huron Life Sciences
Apr 1214:33
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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 1214:34
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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, share information with patient, and how to work

Keynotes
Gina Parziale, Executive Director, Alport Syndrome Foundation
Kara Eichelkraut, Senior Manager, Patient Advocacy, Reata Pharmaceuticals
Apr 1214:35
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ROUNDTABLE 5: Excessive pricing - a deep dive into US and European approaches, recent cases and possible implications for industry and pricing strategies

Keynotes
Behrang Kianzad, Faculty, University of Copenhagen
Apr 1214:36
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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 1214:37
Conference pass

ROUNDTABLE 7: Patient voice - Working with external stakeholders, pharmaceutical partners, and regulators to further the impact of patient advocacy groups

Keynotes
Sook Yee Lim, Co-founder and President, We CARE Journey
Brad Crittenden, Executive Director, Canadian Association of Pompe
Allyson Lock, NEW ZEALAND POMPE NETWORK
Isabelle Lousada, President and CEO, Amyloidosis Research Consortium
Linda Cooper, Director of Special Initiatives, Association For Creatine Deficiencies
Anne Bruns, Executive Director, The Atypical HUS Foundation
Monica Weldon, Chief Executive Officer, 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
Chris Moen, President, Choroideremia Research Foundation Inc
Apr 1214:38
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, Bloomberg Distinguished Professor of Health Informatics, Johns Hopkins University
Apr 1214:40
Conference pass

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
Apr 1214:41
Conference pass

ROUNDTABLE 11: Alternative to viral vectors – reprogramming human T cell function and specificity with non-viral genome targeting

Keynotes
Theodore Roth, MD/PhD Student, University of California, San Francisco
Apr 1214:42
Conference pass

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, President, Project Alive
Apr 1214:43
Conference pass

ROUNDTABLE 13: Disease burden – characterizing the burden of disease to more effectively educate regulators, physicians and payers

Keynotes
Karen Anderson, Senior Director - Medical Affairs & Rare Genetic Diseases, Agios Pharmaceuticals
Apr 1214:44
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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 1214:45
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ROUNDTABLE 15: FDA Patient Affairs - Learn new ways to share your perspective with the FDA

Keynotes
Samir Shaikh, Deputy Director, F.D.A. U.S. Food and Drug Administration
Apr 1214:46
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ROUNDTABLE 16: Advanced manufacturing – building a cGMP in-house cell & gene therapy facility

Keynotes
Apr 1215:40
Conference pass

Enabling drug discovery - the need for improved epidemiology in rare diseases

Clinical Development & Regulatory
Mathew T. Pletcher, Head of Rare Disease Discovery, Roche
Apr 1215:40
Conference pass

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 1215:40
Conference pass

Tying quality of BLA submission with full process development control

Advanced Therapies - Manufacturing
Dorit Harati, Vice President Of Quality Assurance, Gamida Cell Ltd
Apr 1216:05
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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
Apr 1216:05
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How regulators can work collaboratively with industry to advance and improve GMP and quality standards for advanced therapies

Advanced Therapies - Manufacturing
Nirjal Bhattarai, Principal Investigator, F.D.A. U.S. Food and Drug Administration
last published: 13/Nov/18 16:05 GMT

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