2024 Conference Agenda
Boston, MA, 23 - 25 April 2024
Schedule
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Apr 2312:00
Conference pass
REGISTRATION OPENS
Networking Break
Pre-conference day registration opens
Hall C
Apr 2312:50
Conference pass
Chair's Remarks
Early Access Program Seminar
Rachel Harrison, Associate Director, Pre-Approval Access Programs, Argenx
Meeting Room 052A
Apr 2313:00
Conference pass
Come help shape the innovations in rare disease clinical practice and see those that WODC attendees helped originate in 2023
Workshops
We will present innovations based on insights from WODC workshops in 2023 where we leveraged knowledge of the crowds to make a change for patients.
We will be presenting some of the impact of real new technologies that you as our workshop attendees informed us about and showing you why Volv workshops help to accelerate new clinical possibilities, new this year:
- Earlier detection tools
- Fast progression and subpopulation identification for priority needs assessment
- Impact on health systems capacity management
Come and be part of the drive for change.
Leon van Wouwe, Clinical Innovation Director, volv global
Meeting Room 050
Apr 2313:00
Conference pass
Trends in the evolving landscape of early access programs
Early Access Program Seminar
Moderator: Rachel Harrison, Associate Director, Pre-Approval Access Programs, Argenx
Harpreet Ram, President, EVR Consulting
Dorian Readnour, Executive Director, Global Patient Access, Sarepta Therapeutics
Meeting Room 052A
Apr 2313:00
Conference pass
Understanding and addressing the unique needs of rare disease patient advocacy leaders and communities
Advocacy Support Seminar
We will begin this session with the screening of a 16 minute film from The Disorder Channel showing the stories of families facing rare diseases and how they went on to become founders of Patient Advocacy Organizations. The film will be followed by a facilitated group discussion highlighting the challenges faced by rare disease patient advocacy organizations with a focus on their leaders.
Challenges include mental health stressors, compassion fatigue, "founder syndrome," adding professional staff who do not have the lived experience with the disease, PAG leader burnout, PTSD, feelings of isolation, and grieving.
Attendees - whether PAG leaders themselves, rare disease community members, or industry partners - can expect a thought-provoking discussion that deepens their understanding and inspires action to increase support for these essential organizations and their leaders.
Albert Freedman, Counseling Psychologist / Rare Dad, Rarecounseling.com
Meeting Room 051
Apr 2313:00
Conference pass
Workshop: 2024 - The year of adoption for innovative payment models
Payers Seminar
Moderator: Mark Trusheim, Strategic Director NEWDIGS, Center for Biomedical System Design, Tufts Medical Center
Meeting Room 052B
Apr 2314:00
Conference pass
Monetizing hope
Payers Seminar
Moderator: Matt Salo, Former Executive Director, National Association of Medicaid Directors (NAMD), Founder and Chief Executive Officer, Salo Health Strategies
Patroski Lawson, Chief Executive Officer, The KPM Group DC
Meeting Room 052B
Apr 2314:20
Conference pass
Running EAP’s in sanctioned countries
Early Access Program Seminar
Annika Kollén, Executive Vice President, Global Supply Chain & Operations, Inceptua Group
Meeting Room 052A
Apr 2314:40
Conference pass
Short Afternoon Break
Networking Break
Enjoy a quick break before seminars and workshops resume
Apr 2314:59
Conference pass
Chair's Remarks
Patient Data Seminar
Meeting Room 052A
Apr 2315:00
Conference pass
Patient advocacy: Collaboration or competition
Advocacy Support Seminar
Moderator: Kim Stephens, Executive Director, Muenzer MPS Research & Treatment Center
Melissa Penn, Director Patient Engagement Research & Development, Bayer
Josie Godfrey, Co-Founder and Chief Executive Officer, Realise Advocacy
Meeting Room 051
Apr 2315:00
Conference pass
Patient Data
Patient Data Seminar
Pamela Gavin, Executive Vice President, National Organization for Rare Disorders (NORD)
Meeting Room 052A
Apr 2315:00
Conference pass
Personalized endpoints in clinical trials: Exploring goals with AI and social listening
Workshops
Max Flurie, Senior Data Scientist, TREND Community
Meeting Room 050
Apr 2315:00
Conference pass
Sickelcell legislation: Fight for affordable curatives
Payers Seminar
Meeting Room 052B
Apr 2315:20
Conference pass
Revolutionizing care: Unlocking the value of patient data
Patient Data Seminar
Meeting Room 052A
Apr 2315:20
Conference pass
Unlocking the value of patient data (draft)
Patient Data Seminar
Apr 2315:40
Conference pass
Clinical and caregiver data collection in Rett Syndrome: A successful registry launch
Patient Data Seminar
Melissa Kennedy, Chief Executive Officer, International Rett Syndrome Foundation
Meeting Room 052A
Apr 2315:40
Conference pass
Identifying patients and communities underserved by rare - How we are changing the face of MPS
Advocacy Support Seminar
Terri Klein, President & Chief Executive Officer, National M.P.S. Society
Meeting Room 051
Apr 2316:00
Conference pass
Complicated: The Film - Supporting rare disease In the margins of medicine
Advocacy Support Seminar
Meeting Room 051
Apr 2316:00
Conference pass
The five principals of self-advocacy and your mental health toolbox
Workshops
Meeting Room 050
Apr 2316:00
Conference pass
The value of peer collaborative data collection in a rare disease with no specific symptoms
Patient Data Seminar
Meeting Room 052A
Apr 2316:20
Conference pass
Coordination of Rare Diseases at Sanford (CoRDS): Centralized international patient registry for all rare diseases
Patient Data Seminar
Meeting Room 052A
Apr 2316:40
Conference pass
Co-Producing data for daily care and regulatory submissions
Patient Data Seminar
Meeting Room 052A
Create your personal agenda –check the favourite icon
Apr 247:45
Conference pass
EXPO HALL & REGISTRATION OPENS
Networking Break
Join us in the expo hall
Hall C
Apr 248:35
Conference pass
Apr 248:55
Conference pass
Expediting drug development for rare disease
Keynotes
Moderator: Pamela Gavin, Executive Vice President, National Organization for Rare Disorders (NORD)
Peter Marks, Director, Center For Biologics Evaluation And Research (CBER), U.S. Food and Drug Administration
Hall C
Apr 249:25
Conference pass
Charting the path forward: Regulatory innovations and orphan drug designations in rare disease development
Keynotes
Hall C
Apr 249:45
Conference pass
The innovator dilemma, a call to action
Keynotes
Alaa Hamed, Global Head, Medical Affairs Rare Disease and Rare Blood Disorders, Sanofi
Hall C
Apr 2410:05
Conference pass
Challenge and opportunity with the current regulatory and legislative landscape in the US
Keynotes
Peter Marks, Director, Center For Biologics Evaluation And Research (CBER), U.S. Food and Drug Administration
Kinnari Patel, President & COO, Rocket Pharma
Mark Trusheim, Strategic Director NEWDIGS, Center for Biomedical System Design, Tufts Medical Center
Jamie Sullivan, Vice President of Public Policy, EveryLife Foundation for Rare Diseases
Hall C
Apr 2410:45
Conference pass
NETWORKING, POSTERS, EXPO HALL
Networking Break
Networking, expo, and posters
Hall C
Apr 2411:24
Conference pass
Chair's Remarks
Clinical Development & Regulatory
Melissa Penn, Director Patient Engagement Research & Development, Bayer
THEATER 5 - Meeting Room 051
Apr 2411:24
Conference pass
Chair's Remarks
Advanced Therapies Clinical Development
THEATER 7 - Meeting Room 052B
Apr 2411:24
Conference pass
Chair's Remarks
Rare Disease Advocacy World
HALL C - THEATER 1
Apr 2411:24
Conference pass
Chair's Remarks
Commercial
Afshan Hussain, Managing Director, Global Rare Disease Lead, Havas Life Rare
THEATER 6 - Meeting Room 052A
Apr 2411:24
Conference pass
Chair's Remarks
Clinical Development & Regulatory
Melissa Penn, Director Patient Engagement Research & Development, Bayer
THEATER 5 - Meeting Room 051
Apr 2411:24
Conference pass
Chair's Remarks
AI & Digital Health
HALL C - THEATER 3
Apr 2411:25
Conference pass
Empower patients and medical teams with innovative tools to successfully fight rare diseases
AI & Digital Health
HALL C - THEATER 3
Apr 2411:25
Conference pass
NIH as your technology development and commercialization partner
Commercial
THEATER 6 - Meeting Room 052A
Apr 2411:25
Conference pass
Strategies to save time and cost in rare disease drug development
Pricing & Reimbursement
Marshall Summar, Chief Executive Officer, Uncommon Cures
HALL C - THEATER 2
Apr 2411:25
Conference pass
The Intersection of sports and science: Creating a better world for people impacted by rare diseases
Rare Disease Advocacy World
Robert Long, Executive Director, Uplifting Athletes
HALL C - THEATER 1
Apr 2411:25
Conference pass
The pivotal role patient advocacy organizations play in driving rare disease therapeutic development
Clinical Development & Regulatory
- Understand how patient advocacy organizations drive therapeutic development in the rare disease space
- How to effectively engage and partner with patient advocacy organizations to develop meaningful treatments
- Gain insight into the “how, when and where” patient advocacy can significantly impact the drug development process
THEATER 5 - Meeting Room 051
Apr 2411:25
Conference pass
Unlocking Insights: How decentralized clinical trials and advanced therapies are revolutionizing pediatric rare disease research
Advanced Therapies Clinical Development
- This session will explore solutions to challenges in pediatric rare disease research, specifically in consideration of decentralized clinical trials (DCT) and cell and gene therapy development. You will learn:
- The complexities of executing cell and gene therapy programs in pediatric populations
- Key considerations for instituting a DCT approach for cell and gene therapy programs in a pediatric population
- Strategies for seamless operationalization of DCTs
THEATER 7 - Meeting Room 052B
Apr 2411:25
Conference pass
Venture capital panel: Charting the path for rare disease therapies innovation: Unveiling opportunities, trends, and challenges
Pitch and Partner
Moderator: Ken Kengatharan, Managing General Partner, Atheneos Ventures
Geeta Vemuri, Managing Partner, Agent Capital
HALL C - THEATER 4
Apr 2411:45
Conference pass
An honest discussion: The challenges of genotype interpretation and phenotype variation in rare disease
Advanced Therapies Clinical Development
THEATER 7 - Meeting Room 052B
Apr 2411:45
Conference pass
Commercialization in rare disease
Commercial
THEATER 6 - Meeting Room 052A
Apr 2411:45
Conference pass
How you can deliver consequential projects using AI in multi-year deployments for patient
AI & Digital Health
- AI projects in rare diseases can start with limited scope and then take different directions and start to evolve into longer programmes of work over time
- We present 4 case studies demonstrating how the expectations and also objectives can change over time to develop more impact for patients
- We will also show top-line results for each of these case studies
- Q&A will be taken at the Volv booth (513)
Leon van Wouwe, Clinical Innovation Director, volv global
HALL C - THEATER 3
Apr 2411:45
Conference pass
Is there a need for convergence in the use of scientific evidence by decision makers in healthcare?
Clinical Development & Regulatory
THEATER 5 - Meeting Room 051
Apr 2411:45
Conference pass
Potential for optimal patient access and reimbursement
Pricing & Reimbursement
HALL C - THEATER 2
Apr 2411:45
Conference pass
Preparing for the valley of death in drug development
Rare Disease Advocacy World
HALL C - THEATER 1
Apr 2412:05
Conference pass
Building commercial teams for a successful pre-launch
Commercial
THEATER 6 - Meeting Room 052A
Apr 2412:05
Conference pass
Pricing, reimbursement and payer challenges for rare diseases in the US
Pricing & Reimbursement
Moderator: Kollet Koulianos, Senior Payor Advisor, National Hemophilia Foundation
Durhane Wong-Rieger, President, Chief Executive Officer & Chair, Canadian Organisation for Rare Disorder & Rare Diseases International
Kristin Viswanathan Wolff, Senior Director, Global Government Affairs And Public Policy, bluebird bio
HALL C - THEATER 2
Apr 2412:05
Conference pass
Rare and orphan disease programs of the critical path institute: Case examples of the CP-RND and RDCA-DAP
AI & Digital Health
Laura Hopkins, Associate Director, Critical Path Institute
HALL C - THEATER 3
Apr 2412:05
Conference pass
Sustainability of N-of-1
Advanced Therapies Clinical Development
Larissa Lapteva, Associate Director, FDA
THEATER 7 - Meeting Room 052B
Apr 2412:05
Conference pass
Towards a World Health Assembly Resolution on Rare Diseases
Rare Disease Advocacy World
HALL C - THEATER 1
Apr 2412:05
Conference pass
Uniting for the uncommon: Successes and lessons in pharma-patient advocacy partnerships
Clinical Development & Regulatory
What the audience will learn:
- This will be a transparent discussion on the types of collaborations and agreements that have worked (or not worked) between rare disease patient advocacy groups and drug development companies.
- Learn what you’re doing right (or wrong!) when trying to build bridges with advocacy organizations.
- Learn key questions to ask about advocacy organizations and drug companies before starting a partnership.
THEATER 5 - Meeting Room 051
Apr 2412:25
Conference pass
A first-in-class revolutionary platform: jCell® Retinal Progenitor Cells (RPC) restoring vision in patients with blinding diseases
Pitch and Partner
John Sholar, Chief Executive Officer, jCyte Inc
HALL C - THEATER 4
Apr 2412:25
Conference pass
Advocacy journey and policy partnerships
Rare Disease Advocacy World
HALL C - THEATER 1
Apr 2412:25
Conference pass
An approach in using AI and landmarking to quantify at-home or in-clinic video assessments
AI & Digital Health
Nicholas Valle, Senior Product Director, iTakeControl, Red Nucleus
HALL C - THEATER 3
Apr 2412:25
Conference pass
Navigating cell and gene therapy studies in rare diseases
Clinical Development & Regulatory
- Site Identification and Competition
- Overcoming Interdepartmental Challenges
- Logistics Management
- The Impact of Box Warnings
THEATER 5 - Meeting Room 051
Apr 2412:35
Conference pass
Innovative biologics delivery for rare disease
Pitch and Partner
HALL C - THEATER 4
Apr 2412:45
Conference pass
Adaptable decoys for the quick and selective removal of anti-drug and anti-vector antibodies from circulation
Pitch and Partner
THEATER 4 - HALL C
Apr 2412:45
Conference pass
From science to solution: Navigating the clinical development journey
Advanced Therapies Clinical Development
THEATER 7 - Meeting Room 052B
Apr 2412:45
Conference pass
Innovative financing solutions for rare disease treatments: Balancing cost, access, and sustainability
Pricing & Reimbursement
Moderator: Mark Trusheim, Strategic Director NEWDIGS, Center for Biomedical System Design, Tufts Medical Center
Kollet Koulianos, Senior Payor Advisor, National Hemophilia Foundation
Steven Peskin, President and Chief Executive Officer; Former Executive Medical Director Population Health, Horizon Blue Cross Blue Shield of NJ, SRP Advisors, LLC
HALL C - THEATER 2
Apr 2412:45
Conference pass
Patient engagement strategy earlier in commercial planning
Commercial
THEATER 6 - Meeting Room 052A
Apr 2412:45
Conference pass
Redefining patient roles- Speeding up time to treatment through AI-based digital health
AI & Digital Health
HALL C - THEATER 3
Apr 2412:45
Conference pass
The critical role of partnerships in rare disease drug advancement: From lab bench to bedside
Clinical Development & Regulatory
THEATER 5 - Meeting Room 051
Apr 2412:45
Conference pass
Valuing rare disease treatments in healthcare: Real experience, real impact
Rare Disease Advocacy World
Richard Chapman, Chief Science Officer, Innovation and Value Initiative
HALL C - THEATER 1
Apr 2412:55
Conference pass
TransCytos' Non-Viral Ex-Vivo Slipstream™ Technology – a Transformation of Cell and Gene Therapy Delivery
Pitch and Partner
HALL C - THEATER 4
Apr 2413:05
Conference pass
From patient organization to biotech
Pitch and Partner
HALL C - THEATER 4
Apr 2413:05
Conference pass
International Rare Disease Research Consortium (IRDiRC): A multi-stakeholder approach to understanding the needs in developing Rare Disease diagnostics and therapies
Rare Disease Advocacy World
HALL C - THEATER 1
Apr 2413:05
Conference pass
Meet the moment for advanced therapies: Past learnings, present state and collaborating for the future
Commercial
Michael Lehmicke, Senior Vice President of Science and Industry Affairs, Alliance for Regenerative Medicine
THEATER 6 - Meeting Room 052A
Apr 2413:05
Conference pass
Mobile visits are critical for successful rare disease research
Clinical Development & Regulatory
THEATER 5 - Meeting Room 051
Apr 2413:05
Conference pass
Optimizing patient recruitment by leveraging existing online communities: Success stories
Advanced Therapies Clinical Development
Ethan Ash, Senior VP of Business Development, BioNews
THEATER 7 - Meeting Room 052B
Apr 2413:05
Conference pass
Revolutionizing support: MyMejo's innovative solutions for caregivers by caregivers
AI & Digital Health
Melissa Kennedy, Chief Executive Officer, International Rett Syndrome Foundation
HALL C - THEATER 3
Apr 2413:15
Conference pass
Metabolic replacement therapy for the treatment of rhizomelic chondrodysplasia punctata
Pitch and Partner
HALL C - THEATER 4
Apr 2413:25
Conference pass
NETWORKING, POSTERS, EXPO HALL
Networking Break
Food for purchase in the expo hall
Hall C
Apr 2413:35
Conference pass
Unlocking patient-centric insights and feedback to elevate engagement
Keynotes
Moderator: Anthony Scatamacchia, Senior Vice President, Innovation, Patient Services, Eversana
Hall C
Apr 2414:15
Conference pass
Roundtable 01: Shifting strands: Operational challenges and ethical dilemmas of genetic testing
Roundtables
Hall C
Apr 2414:15
Conference pass
Roundtable 02: Patient Recruitment for Neurodegenerative and Neuromuscular Clinical Trials
Roundtables
- How to reach more patients: clinical trial agnostic and clinical trial specific approaches
- How Patient Navigators play a crucial role in the patients’ CT journey
- Understand how to involve all stakeholders through the use of platform
Jytte van Huijstee, Director Clinical Trial Operations, myTomorrows
Terri Ellsworth, Patient Advocate Partnerships, myTomorrows
Madeleine Carrier, Patient Navigator, myTomorrows
Apr 2414:15
Conference pass
Roundtable 03: Benefit or a threat? Beware the AI generated patient
Roundtables
Kim MacDonnell, Associate Director, Rare Disease, COE, Parexel
Apr 2414:15
Conference pass
Roundtable 04: Running EAP’s in sanctioned countries
Roundtables
Sponsored by Volv Global
Paul Stanton, Senior Director, Global Strategy, Early Access, Inceptua Group
Apr 2414:15
Conference pass
Roundtable 05: Forecasting & business development: Sizing the market opportunity
Roundtables
Sponsored by Orphan Drug Consulting
Joe Musumeci, President, BluePrint Orphan
Chelsea Catsburg, Engagement Manager, BluePrint Orphan
Apr 2414:15
Conference pass
Roundtable 06: Meeting the challenges of fast-track approval for orphan drugs
Roundtables
Sponsored by HEORStrategies, A Division of ToxStrategies LLC
Naomi Cohen Sacks, Vice President, HEORStrategies, A Division of ToxStrategies LLC
Lauren Bylsma, Supervising Epidemiologist, EpidStrategies, A Division of ToxStrategies
Jon Fryzek, Practice Director, EpidStrategies, A Division of ToxStrategies
Amy Kimzey, Toxicologist, ToxStrategies LLC
Apr 2414:15
Conference pass
Roundtable 07: Impact of rare disease on siblings
Roundtables
Joslyn Crowe, Executive Director, National Niemann Pick Disease Foundation Inc
Apr 2414:15
Conference pass
Roundtable 08: PAG to Pharma - transitioning from the rare disease patient community to an advocacy role within industry
Roundtables
Apr 2414:15
Conference pass
Roundtable 09: Early access programs Q&A
Roundtables
Rachel Harrison, Associate Director, Pre-Approval Access Programs, Argenx
Apr 2414:15
Conference pass
Roundtable 10: Grassroots to Science-Ready: Patient-Created Research
Roundtables
Moderated byCushing's Support & Research Foundation (CSFR)
Apr 2414:55
Conference pass
SHORT BREAK BEFORE AFTERNOON BREAKOUT SESSIONS
Networking Break
Short break
Apr 2414:59
Conference pass
Chair's Remarks
Rare Disease Advocacy World
HALL C - THEATER 1
Apr 2414:59
Conference pass
Chair's Remarks
Advanced Therapies Clinical Development
THEATER 7 - Meeting Room 052B
Apr 2414:59
Conference pass
Chair's Remarks
Commercial
Afshan Hussain, Managing Director, Global Rare Disease Lead, Havas Life Rare
THEATER 6 - Meeting Room 052A
Apr 2414:59
Conference pass
Chair's Remarks
Clinical Development & Regulatory
Jennifer McNary, Founder, One Rare
THEATER 5 - Meeting Room 051
Apr 2415:00
Conference pass
Challenges of diagnosing and treating rare diseases in adult patients
Rare Disease Advocacy World
Durhane Wong-Rieger, President, Chief Executive Officer & Chair, Canadian Organisation for Rare Disorder & Rare Diseases International
Jida El Hajjar, Executive Director,, ALS Action Canada
HALL C - THEATER 1
Apr 2415:00
Conference pass
Collaboration for rare oncology patients
Rare Oncology
HALL C - THEATER 3
Apr 2415:00
Conference pass
Maximizing therapeutic impact: Exploring the potential of basket trials for concurrent treatment strategies
Advanced Therapies Clinical Development
THEATER 7 - Meeting Room 052B
Apr 2415:00
Conference pass
Patient-first innovation: FDA shifting rare diseases endpoints
Clinical Development & Regulatory
Jamie Sullivan, Vice President of Public Policy, EveryLife Foundation for Rare Diseases
THEATER 5 - Meeting Room 051
Apr 2415:00
Conference pass
Pricing challenges for rare diseases in Europe
Pricing & Reimbursement
Alexander Natz, Director General, EUCOPE - European Confederation of Pharmaceutical Entrepreneurs
HALL C - THEATER 2
Apr 2415:00
Conference pass
Revolutionizing vision restoration: Mutation-independent optogenetic therapy for inherited retinal diseases
Pitch and Partner
HALL C - THEATER 4
Apr 2415:00
Conference pass
Use of machine learning to improve time to diagnosis of patients with probable MPS II: A case study
Commercial
THEATER 6 - Meeting Room 052A
Apr 2415:10
Conference pass
How to prepare for the genomic gold rush in pharma
Pitch and Partner
HALL C - THEATER 4
Apr 2415:20
Conference pass
A new legislative era in the EU: How an integrated approach can help smaller pharma and biotech achieve a sustainable model to deliver equity of access in short timeframes
Advanced Therapies Clinical Development
Ruth Rostron, Director of Program Management, Uniphar
THEATER 7 - Meeting Room 052B
Apr 2415:20
Conference pass
Breaking barriers: Gene therapy access for neurodegenerative patients
Rare Disease Advocacy World
HALL C - THEATER 1
Apr 2415:20
Conference pass
Commercializing in the ultra-orphan space: Unique challenges and opportunities
Commercial
Moderator: Kristin Marvin-Keller, Managing Director, Havas Life Rare
THEATER 6 - Meeting Room 052A
Apr 2415:20
Conference pass
Five headwinds facing rare cancer drug developers (and what to do about it)
Rare Oncology
Moderator: Benjamin Brown, Executive Director, American Society of Pharmacovigilance
Mridula Shukla, Head of Regulatory Affairs, Gene Therapy, Poseida Therapeutics, Inc.
HALL C - THEATER 3
Apr 2415:20
Conference pass
Rare but not forgotten: Challenges and opportunities for rare disease drugs
Clinical Development & Regulatory
Samantha Zappia, MS, RAC, Principal Consultant, SK Regulatory Solutions
THEATER 5 - Meeting Room 051
Apr 2415:20
Conference pass
ST-002: A promising therapy for the metabolic effects of hypercortisolemia and Cushing syndrome
Pitch and Partner
HALL C - THEATER 4
Apr 2415:20
Conference pass
Tackling financing and reimbursement challenges for transformative therapies – insights from an investor and ex-payer
Pricing & Reimbursement
HALL C - THEATER 2
Apr 2415:30
Conference pass
Zelenirstat: Safety and efficacy - ready to move to clinical investigation for Acute Myeloid Leukemia (AML)
Pitch and Partner
HALL C - THEATER 4
Apr 2415:40
Conference pass
Long acting glucagon analog for the treatment of CHI (Congenital Hyper Insulinism)
Pitch and Partner
HALL C - THEATER 4
Apr 2415:40
Conference pass
Pediatric to adult healthcare transition - continued care
Rare Disease Advocacy World
HALL C - THEATER 1
Apr 2415:40
Conference pass
The future of rare disease funding: Saudi Arabia's investment wave
Clinical Development & Regulatory
Lavni Varyani, Founder, Pharma Business Partners
THEATER 5 - Meeting Room 051
Apr 2415:40
Conference pass
The growing divergence in orphan approvals: A retrospective cohort study comparing the FDA and the EMA from 2011 to 2020
Pricing & Reimbursement
HALL C - THEATER 2
Apr 2415:40
Conference pass
Treating the nano-rare: Redefining clinical management, one patient at a time
Advanced Therapies Clinical Development
THEATER 7 - Meeting Room 052B
Apr 2415:50
Conference pass
FLAG-003 for the Treatment of DIPG (Diffuse Intrinsic Pontine Glioma) in Children
Pitch and Partner
HALL C - THEATER 4
Apr 2416:00
Conference pass
Cell and gene therapy access and reimbursement challenges in Europe
Pricing & Reimbursement
Moderator: Alexander Natz, Director General, EUCOPE - European Confederation of Pharmaceutical Entrepreneurs
HALL C - THEATER 2
Apr 2416:00
Conference pass
Conducting clinical studies in rare diseases
Advanced Therapies Clinical Development
Moderator: Marshall Summar, Chief Executive Officer, Uncommon Cures
Ron Bartek, President, Co-Founder, Friedreich's Ataxia Research Alliance
THEATER 7 - Meeting Room 052B
Apr 2416:00
Conference pass
First-in-class KCC2 potentiators as novel orphan drugs to treat CNS disinhibition and drug-resistance in rare neurological disorders
Pitch and Partner
HALL C - THEATER 4
Apr 2416:00
Conference pass
Mental health and rare diseases: Mental health is part of total health
Rare Disease Advocacy World
HALL C - THEATER 1
Apr 2416:00
Conference pass
Navigating difficult pathways in rare drug development: How, when and why sponsors should engage with FDA ombuds
Clinical Development & Regulatory
Moderator: Jennifer McNary, Founder, One Rare
THEATER 5 - Meeting Room 051
Apr 2416:00
Conference pass
What it takes to order that one test by physicians
Commercial
THEATER 6 - Meeting Room 052A
Apr 2416:00
Conference pass
Winning approaches for rare disease patient recruitment
Rare Oncology
Discover Effective Tactics and Methods for Rare Disease Patient Recruitment Campaigns, Drawing from Real-World Case Studies, Examples, and Best Practices to Ensure Enrollment Success in Studies.
HALL C - THEATER 3
Apr 2416:10
Conference pass
To be announced
Pitch and Partner
HALL C - THEATER 4
Apr 2416:20
Conference pass
Ensuring reliability in quantifying patient populations for orphan drugs: challenges and solutions
Pitch and Partner
HALL C - THEATER 4
Apr 2416:20
Conference pass
The importance of being believed: Medical gaslighting in the rare disease space
Rare Disease Advocacy World
HALL C - THEATER 1
Apr 2416:30
Conference pass
Rising to the challenges of rare disease treatment
Pitch and Partner
HALL C - THEATER 4
Apr 2416:40
Conference pass
SHORT BREAK BEFORE KEYNOTES
Networking Break
Short break before keynotes
Apr 2416:45
Conference pass
Using biomarkers to enable successful development of disease-modifying therapies for rare disease
Keynotes
Hall C
Apr 2417:05
Conference pass
From patient finding to payer pull-through: leveraging advanced analytics to fuel the rare disease revolution
Keynotes
Hall C
Apr 2417:35
Conference pass
Apr 2417:40
Conference pass
Portrait unveiling by Beyond the Diagnosis
Keynotes
Hall C
Apr 2417:45
Conference pass
COCKTAIL RECEPTION
Keynotes
Join us for cocktail hour
Hall C
Create your personal agenda –check the favourite icon
Apr 258:00
Conference pass
EXPO HALL & REGISTRATION OPENS
Networking Break
Join us in the expo hall
Expo Hall
Apr 258:50
Conference pass
Chair opening remarks
Keynotes
Hall C
Apr 259:00
Conference pass
Fireside chat: Access for rare disease patients in Europe and beyond
Keynotes
Yann Le Cam, Founder and past-CEO, EURORDIS
Hall C
Apr 259:20
Conference pass
Can you overcome complexity and deliver value for rare disease patients in the EU health ecosystem?
Keynotes
Sponsored by Sciensus
Grant Castor, SVP Commercial Strategy & Operations, Sentynl Therapeutics
Hall C
Apr 2510:00
Conference pass
Unlocking access: Transformative policies and strategies for orphan drug accessibility
Keynotes
Moderator: Jayson Slotnik, Partner, Health Policy Strategies, LLC
Christoph Glaetzer, Chief Global Value and Access Officer, Johnson & Johnson
Ron Bartek, President, Co-Founder, Friedreich's Ataxia Research Alliance
Hall C
Apr 2510:40
Conference pass
Breaking borders: Advancing cross-state access to care for rare diseases
Keynotes
Matt Salo, Former Executive Director, National Association of Medicaid Directors (NAMD), Founder and Chief Executive Officer, Salo Health Strategies
Hall C
Apr 2511:20
Conference pass
NETWORKING, POSTERS, EXPO HALL
Networking Break
Sponsored by Bionical Emas
Apr 2511:59
Conference pass
Chair's Remarks
Clinical Development & Regulatory
THEATER 5 - Meeting Room 051
Apr 2511:59
Conference pass
Chair's Remarks
Drug Discovery
Pete Thornton, Sr. Director, Strategic Business Development, Invitae
HALL C - THEATER 2
Apr 2511:59
Conference pass
Chair's Remarks
Commercial
THEATER 6 - Meeting Room 052A
Apr 2512:00
Conference pass
Access to rare disease medicines in LMICs
Global Market Access
Harpreet Ram, President, EVR Consulting
THEATER 7 - Meeting Room 052B
Apr 2512:00
Conference pass
Call to action: Update newborn screening
Diagnosis
HALL C - THEATER 1
Apr 2512:00
Conference pass
CureAP-4 drug discovery for ultra-rare CNS disease
Drug Discovery
Kasey Edwards, Co-Founder, Community Relations Specialist, Cure AP-4
HALL C - THEATER 2
Apr 2512:00
Conference pass
Industry perspectives: Partnering in the rare disease space
Pitch and Partner
Shira Landskroner-Eiger, Director, External Innovation Rare Disease, Ipsen
Kathie Bishop, SVP, Head of Rare Disease and External Innovation, Acadia Pharmaceuticals Inc.
HALL C - THEATER 4
Apr 2512:00
Conference pass
New research examining the patient impact of the FDA accelerated approval program
Policy
Silas Martin, Head, Access & Policy Research, Johnson & Johnson
HALL C - THEATER 3
Apr 2512:00
Conference pass
Preparing early for ex-US commercialisation – a patient access perspective
Commercial
Josie Godfrey, Co-Founder and Chief Executive Officer, Realise Advocacy
THEATER 6 - Meeting Room 052A
Apr 2512:00
Conference pass
Putting science first in rare disease studies
Clinical Development & Regulatory
Anthy Tsatoumas, Manager, Project Management, Veristat
THEATER 5 - Meeting Room 051
Apr 2512:20
Conference pass
AI, insights and technology transformations within rare diseases
Commercial
THEATER 6 - Meeting Room 052A
Apr 2512:20
Conference pass
How to successfully achieve reimbursement in rare diseases: From inception to submission
Global Market Access
THEATER 7 - Meeting Room 052B
Apr 2512:20
Conference pass
Leveraging at-scale genetic testing data to accelerate precision drug discovery for rare diseases
Drug Discovery
Alix Lacoste, AI Computational Biology Director Genetic Insights, Invitae
HALL C - THEATER 2
Apr 2512:20
Conference pass
Newborn screening, sequencing, and the future
Diagnosis
Moderator: Christopher Porter, Vice President, Government Affairs and Policy, Travere Therapeutics
Kate Donigan, Senior Director, Science & Regulatory Policy, Global Policy, Government, & Patient Affairs, Sarepta Therapeutics
Nicole Miller, Vice President, Molecular Diagnostics, Global Medical Affairs, Ultragenyx
HALL C - THEATER 1
Apr 2512:20
Conference pass
Understanding the implications of state policies on patient access
Policy
Jayson Slotnik, Partner, Health Policy Strategies, LLC
Melanie Lendnal, Senior Vice President, Policy and Advocacy, The ALS Association
HALL C - THEATER 3
Apr 2512:40
Conference pass
Development of an enzyme replacement therapy for batten disease CLN1 and other neurodegenerative diseases
Pitch and Partner
HALL C - THEATER 4
Apr 2512:40
Conference pass
Evolving trends in gene therapy
Clinical Development & Regulatory
Mridula Shukla, Head of Regulatory Affairs, Gene Therapy, Poseida Therapeutics, Inc.
Apr 2512:40
Conference pass
Evolving trends in gene Therapy
Clinical Development & Regulatory
Mridula Shukla, Head of Regulatory Affairs, Gene Therapy, Poseida Therapeutics, Inc.
THEATER 5 - Meeting Room 051
Apr 2512:40
Conference pass
In-house drug development for patient advocacy groups
Drug Discovery
Julia Taravella, Exececutive Director, Rare Trait Hope Fund
HALL C - THEATER 2
Apr 2512:40
Conference pass
Transformative launch strategies: Commercializing orphan products for success
Commercial
Grant Castor, SVP Commercial Strategy & Operations, Sentynl Therapeutics
Tsveta Milanova, Chief Commercial Officer, Agios Pharmaceuticals
THEATER 6 - Meeting Room 052A
Apr 2512:50
Conference pass
To be announced
Pitch and Partner
Patrick Lu, President and Chief Executive Officer, Sirnaomics
HALL C - THEATER 4
Apr 2513:00
Conference pass
360 view: How do we improve access while we continue to promote innovation
Policy
Durhane Wong-Rieger, President, Chief Executive Officer & Chair, Canadian Organisation for Rare Disorder & Rare Diseases International
Alexander Natz, Director General, EUCOPE - European Confederation of Pharmaceutical Entrepreneurs
HALL C - THEATER 3
Apr 2513:00
Conference pass
Engaging minority communities: Collaborative practices and long-term success
Clinical Development & Regulatory
Mary McGowan, Chief Executive Officer, Foundation for Sarcoidosis Research
Victoria Arteaga, Co-founder, SHER Hispanic Society for Rare Diseases
THEATER 5 - Meeting Room 051
Apr 2513:00
Conference pass
Interactive Discussion: A new data frontier: Harnessing AI to unlock rare disease insights from social media conversations
Drug Discovery
Colton Flowers, NLP Engineer, TREND Community
Max Flurie, Senior Data Scientist, TREND Community
HALL C - THEATER 2
Apr 2513:00
Conference pass
The promise of local cell therapy processing
Global Market Access
THEATER 7 - Meeting Room 052B
Apr 2513:00
Conference pass
The role of genomic sequencing in advancing precision medicine
Diagnosis
Ben Willis, Director of Business Excellence, Egetis Therapeutics
HALL C - THEATER 1
Apr 2513:00
Conference pass
To be announced
Pitch and Partner
Sponsored by Comend
HALL C - THEATER 4
Apr 2513:10
Conference pass
XDemics: Making tomorrow’s cell and gene therapies
Pitch and Partner
Colin Cook, Technical Founder, XDemics Corporation
HALL C - THEATER 4
Apr 2513:20
Conference pass
Advanced therapies in rare diseases: perspectives of patients and caregivers at a global level and recommendations for a successful introduction in Latin America.
Global Market Access
Diego Gil Cardozo, President, Enfermedades Raras en el Caribe y América Latina (ERCAL)
THEATER 7 - Meeting Room 052B
Apr 2513:20
Conference pass
Rescuing ‘orphaned’ drug programs & creating a viable commercial pathway for ultra-rare disease treatments
Commercial
THEATER 6 - Meeting Room 052A
Apr 2513:20
Conference pass
To be announced
Pitch and Partner
HALL C - THEATER 4
Apr 2513:40
Conference pass
SHORT BREAK BEFORE ROUNDTABLES
Networking Break
Short break
Apr 2513:45
Conference pass
Roundtable 01: Connecting journeys: Building an online community for support and insight
Roundtables
Sponsored by TREND Community
Apr 2513:45
Conference pass
Roundtable 02: Patient-engagement in a new digital world, from diagnostics to holistic care
Roundtables
Raghu Vishwanath, Global Head, Franchise Strategy and Operations, Rare Diseases Specialty Care, Sanofi
Apr 2513:45
Conference pass
Roundtable 03: RDCA-DAP task forces: How to facilitate drug development in rare disease?
Roundtables
Kasey Woleben, Founder, Cure Mito Foundation/Rare Village
Sophia Zilber, Board member, patient registry director, Cure Mito Foundation
Hall C
Apr 2513:45
Conference pass
Roundtable 04: Evolving the value proposition toward equity for adults living with rare conditions
Roundtables
Durhane Wong-Rieger, President, Chief Executive Officer & Chair, Canadian Organisation for Rare Disorder & Rare Diseases International
Louise Vetter, President & CEO, Huntington's Disease Society of America
Hall C
Apr 2513:45
Conference pass
Roundtable 05: Breaking down barriers for individualized medicines for rare disease
Roundtables
Irina Antonijevic, Chief Medical Officer, EveryONE Medicines
Apr 2513:45
Conference pass
Roundtable 06: Navigating the unknown - Undiagnosed, partial diagnosis, incorrect diagnosis
Roundtables
Apr 2513:45
Conference pass
Roundtable 07: Income diversification for patient advocacy organizations
Roundtables
Apr 2513:45
Conference pass
Roundtable 08: Health literacy & life skills training for an equitable world
Roundtables
Apr 2513:45
Conference pass
Roundtable 09: Radical Inclusion
Roundtables
Donna Mackey, Vice President, Clinical Operations, Akouos, Inc.
Apr 2513:45
Conference pass
Roundtable 10: PaVe-GT, AAV gene therapies
Roundtables
Apr 2514:25
Conference pass
NETWORKING, POSTERS, EXPO HALL
Networking Break
Food for purchase on the expo floor
Hall C
Apr 2514:30
Conference pass
AI and digital health for rare diseases: Making data inclusive
Keynotes
Spencer Huggett, Founder, Synapze
Hall C
Apr 2515:14
Conference pass
Chair's Remarks
Clinical Development & Regulatory
THEATER 5 - Meeting Room 051
Apr 2515:14
Conference pass
Chair's Remarks
Drug Discovery
Pete Thornton, Sr. Director, Strategic Business Development, Invitae
HALL C - THEATER 2
Apr 2515:15
Conference pass
Accelerate and advance drug development: From Patients to approvals
Drug Discovery
Sarah Gheuens, Chief Medical Officer, Head of R&D, Agios Pharmaceuticals
HALL C - THEATER 2
Apr 2515:15
Conference pass
Global collaboration to support timely diagnosis and care for persons living with a rare disease
Diagnosis
Mary Wang, Programme Director, Rare Diseases International
HALL C - THEATER 1
Apr 2515:15
Conference pass
Innovative & sustainable ways to increase access to treatments
Global Market Access
THEATER 7 - Meeting Room 052B
Apr 2515:15
Conference pass
Manufacturing improvements for gene therapy for orphan drugs
Advanced Therapies
Sheila Mikhail, Executive Director, Columbus Children's Foundation
THEATER 6 - Meeting Room 052A
Apr 2515:15
Conference pass
RARE Revolution: Authentic storytelling in a challenging regulatory landscape
Clinical Development & Regulatory
Nicola Miller, Editor in Chief, RARE Revolution Magazine
THEATER 5 - Meeting Room 051
Apr 2515:15
Conference pass
Regulatory overview for drug development for rare diseases
Policy
HALL C - THEATER 3
Apr 2515:35
Conference pass
A candid conversation: What rare really needs
Pitch and Partner
HALL C - THEATER 4
Apr 2515:35
Conference pass
Changing landscape for drug repurposing
Policy
HALL C - THEATER 3
Apr 2515:35
Conference pass
Equity in healthcare: Bridging the gap for diverse clinical representation
Global Market Access
THEATER 7 - Meeting Room 052B
Apr 2515:35
Conference pass
How caregiver and patient driven medical discoveries impact rare diseases
Drug Discovery
HALL C - THEATER 2
Apr 2515:35
Conference pass
Individualized medicines for rare diseases – Opportunities and challenges
Advanced Therapies
Irina Antonijevic, Chief Medical Officer, EveryONE Medicines
THEATER 6 - Meeting Room 052A
Apr 2515:35
Conference pass
Measuring diversity of patient in clinical trials: Do we have the right measures to measure the right things?
Clinical Development & Regulatory
THEATER 5 - Meeting Room 051
Apr 2515:35
Conference pass
The Global Commission, a partnership to end the diagnostic odyssey for children with rare diseases
Diagnosis
Sponsored by Sanofi
Yann Le Cam, Founder and past-CEO, EURORDIS
Pamela Gavin, Executive Vice President, National Organization for Rare Disorders (NORD)
HALL C - THEATER 1
Apr 2515:55
Conference pass
Advances in treating the multisystemic manifestations of metabolic diseases – delivering enzyme everywhere it is needed
Advanced Therapies
THEATER 6 - Meeting Room 052A
Apr 2515:55
Conference pass
A-Synaptic’s patent protected transdermal replacement for billion dollar oral childhood epilepsy treatment
Pitch and Partner
HALL C - THEATER 4
Apr 2515:55
Conference pass
Collaboration: the TREAT-NMD model bringing therapies to patients with a rare disease
Clinical Development & Regulatory
David Allison, Chief Executive Officer, TREAT-NMD
THEATER 5 - Meeting Room 051
Apr 2515:55
Conference pass
Drug discovery for treating rare disease patients
Drug Discovery
Sarah Gheuens, Chief Medical Officer, Head of R&D, Agios Pharmaceuticals
HALL C - THEATER 2
Apr 2515:55
Conference pass
Facebook in diagnosis? Gathering insights from everyday discussion of amyotrophic lateral sclerosis (ALS) treatments
Diagnosis
HALL C - THEATER 1
Apr 2515:55
Conference pass
How policy moves forward with patients at the center
Policy
HALL C - THEATER 3
Apr 2515:55
Conference pass
Why Investing in Rare? Innovative Solutions for Health Systems Financing
Global Market Access
Rare diseases pose unique challenges to healthcare systems worldwide due to their low prevalence and complex treatment requirements. Traditional financing mechanisms often fail to adequately address the needs of patients with rare diseases, leading to disparities in access to treatment and care. However, innovative solutions are emerging to bridge this gap and ensure equitable access to healthcare for individuals affected by rare diseases. This session will explore the importance of investing in rare diseases and showcase innovative financing strategies that can support health systems in providing comprehensive care to patients with rare diseases.
Moderator: Anne-Sophie Chalandon, Head of Global Public Affairs, Rare Diseases and CGT Policies, Sanofi
Roberto Lunes, Senior Health Economist, World Bank
THEATER 7 - Meeting Room 052B
Apr 2516:15
Conference pass
Building a learning healthcare network for innovation in diagnosis and precision medicine in rare diseases
Diagnosis
HALL C - THEATER 1
Apr 2516:15
Conference pass
Developing patient-reported outcomes for orphan disease: Challenges and solutions
Clinical Development & Regulatory
THEATER 5 - Meeting Room 051
Apr 2516:35
Conference pass
WORLD ORPHAN DRUG CONGRESS CONCLUDES
Keynotes
Mark your calendars for World Orphan Drug Congress 2025!
- Date: April 23-25, 2025
- Location: Boston Convention and Exhibtion Center (BCEC)
- If interested in speaking reach out to gina.mchugh@terrapinn.com
- If interested in sponsoring reach out to justin.franks@terrapinn.com
last published: 18/Apr/24 21:05 GMT
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