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, Pre-Approval Access Lead, 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, Pre-Approval Access Lead, 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.

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
Paul Stanton, Senior Director, Global Strategy, Early Access, 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
Kinnari Patel, President & COO, Rocket Pharma
Peter Marks, Director, Center For Biologics Evaluation And Research (CBER), U.S. Food and Drug Administration
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

Pricing & Reimbursement
HALL C - THEATER 2
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 Health
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
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

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

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

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
Steven Peskin, President and Chief Executive Officer; Former Executive Medical Director Population Health, Horizon Blue Cross Blue Shield of NJ, SRP Advisors, LLC
Kollet Koulianos, Senior Payor Advisor, National Hemophilia Foundation
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
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
Terri Ellsworth, Patient Advocate Partnerships, myTomorrows
Madeleine Carrier, Patient Navigator, myTomorrows
Jytte van Huijstee, Director Clinical Trial Operations, myTomorrows
Hall C
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
Hall C
Apr 2414:15
Conference pass

Roundtable 04: Running EAP’s in sanctioned countries

Roundtables
Hall C
Apr 2414:15
Conference pass

Roundtable 05: Forecasting & business development: Sizing the market opportunity

Roundtables
Chelsea Catsburg, Engagement Manager, BluePrint Orphan
Joe Musumeci, President, BluePrint Orphan
Hall C
Apr 2414:15
Conference pass

Roundtable 06: Meeting the challenges of fast-track approval for orphan drugs

Roundtables
Lauren Bylsma, Senior Epidemiologist, EpidStrategies, A Division of ToxStrategies
Jon Fryzek, Practice Director, EpidStrategies, A Division of ToxStrategies
Amy Kimzey, Toxicologist, EpidStrategies, A Division of ToxStrategies
Naomi Cohen Sacks, Vice President, HEORStrategies, A Division of ToxStrategies LLC
Hall C
Apr 2414:15
Conference pass

Roundtable 07: Impact of rare disease on siblings

Roundtables
Joslyn Crowe, Executive Director, National Niemann Pick Disease Foundation Inc
Hall C
Apr 2414:15
Conference pass

Roundtable 08: PAG to Pharma - transitioning from the rare disease patient community to an advocacy role within industry

Roundtables
Hall C
Apr 2414:15
Conference pass

Roundtable 09: Early access programs Q&A

Roundtables
Rachel Harrison, Pre-Approval Access Lead, Argenx
Hall C
Apr 2414:15
Conference pass

Roundtable 10: Grassroots to Science-Ready: Patient-Created Research

Roundtables

Moderated byCushing's Support & Research Foundation (CSFR)

Hall C
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 Health
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
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
THEATER 5 - Meeting Room 051
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

NPC, Galactosemia and Barth Syndrome: Developing modifier therapies for neurological and metabolic disorders

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

XDemics: Making tomorrow’s cell and gene therapies

Pitch and Partner
Colin Cook, Technical Founder, XDemics Corporation
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 2417:35
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
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

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
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

Diagnosis
Mary Wang, Programme Director, Rare Diseases International
HALL C - THEATER 1
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, VP, Pharma Services & Strategic Partnerships, 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
Kathie Bishop, SVP, Head of Rare Disease and External Innovation, Acadia Pharmaceuticals Inc.
Shira Landskroner-Eiger, Director, External Innovation Rare Disease, Ipsen
HALL C - THEATER 4
Apr 2512:00
Conference pass

New research examining the patient impact of the FDA accelerated approval program

Policy
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

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
Nicole Miller, Vice President, Molecular Diagnostics, Global Medical Affairs, Ultragenyx
Kate Donigan, Senior Director, Science & Regulatory Policy, Global Policy, Government, & Patient Affairs, Sarepta Therapeutics
HALL C - THEATER 1
Apr 2512:20
Conference pass

Understanding the implications of state policies on patient access

Policy
Melanie Lendnal, Senior Vice President, Policy and Advocacy, The ALS Association
Jayson Slotnik, Partner, Health Policy Strategies, LLC
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
Tsveta Milanova, Chief Commercial Officer, Agios Pharmaceuticals
Grant Castor, SVP Commercial Strategy & Operations, Sentynl Therapeutics
THEATER 6 - Meeting Room 052A
Apr 2513:00
Conference pass

360 view: How do we improve access while we continue to promote innovation

Policy
Alexander Natz, Director General, EUCOPE - European Confederation of Pharmaceutical Entrepreneurs
Durhane Wong-Rieger, President, Chief Executive Officer & Chair, Canadian Organisation for Rare Disorder & Rare Diseases International
HALL C - THEATER 3
Apr 2513:00
Conference pass

Engaging minority communities: Collaborative practices and long-term success

Clinical Development & Regulatory
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
Max Flurie, Senior Data Scientist, TREND Community
Colton Flowers, NLP Engineer, TREND Community
HALL C - THEATER 2
Apr 2513:00
Conference pass

Supercharging rare patient groups with technology

Pitch and Partner

Sponsored by Comend

Albert Wang, Co-founder, CEO, Comend
HALL C - THEATER 4
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
HALL C - THEATER 1
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
Hall C
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
Hall C
Apr 2513:45
Conference pass

Roundtable 03: RDCA-DAP task forces: How to facilitate drug development in rare disease?

Roundtables
Sophia Zilber, Board member, patient registry director, Cure Mito Foundation
Kasey Woleben, Founder, Cure Mito Foundation/Rare Village
Hall C
Apr 2513:45
Conference pass

Roundtable 04: Evolving the value proposition toward equity for adults living with rare conditions

Roundtables
Louise Vetter, President & CEO, Huntington's Disease Society of America
Durhane Wong-Rieger, President, Chief Executive Officer & Chair, Canadian Organisation for Rare Disorder & Rare Diseases International
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
Hall C
Apr 2513:45
Conference pass

Roundtable 06: Navigating the unknown - Undiagnosed, partial diagnosis, incorrect diagnosis

Roundtables
Hall C
Apr 2513:45
Conference pass

Roundtable 07: Income diversification for patient advocacy organizations

Roundtables
Hall C
Apr 2513:45
Conference pass

Roundtable 08: Health literacy & life skills training for an equitable world

Roundtables
Hall C
Apr 2513:45
Conference pass

Roundtable 09: Radical Inclusion

Roundtables
Donna Mackey, Vice President, Clinical Operations, Akouos, Inc.
Hall C
Apr 2513:45
Conference pass

Roundtable 10: PaVe-GT, AAV gene therapies

Roundtables
Hall C
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
Hall C
Apr 2515:14
Conference pass

Chair's Remarks

Diagnosis
Mary Wang, Programme Director, Rare Diseases International
HALL C - THEATER 1
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, VP, Pharma Services & Strategic Partnerships, 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
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

Pamela Gavin, Executive Vice President, National Organization for Rare Disorders (NORD)
Yann Le Cam, Founder and past-CEO, EURORDIS
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

Facebook in diagnosis? Gathering insights from everyday discussion of amyotrophic lateral sclerosis (ALS) treatments

Diagnosis
HALL C - THEATER 1
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 Iunes, 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: 20/Apr/24 15:35 GMT

Get Involved At World Orphan Drug Congress USA

 

To Sponsor Or Exhibit

 
 

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

 

 

 

To Speak

 
 

Gina McHugh
gina.mchugh@terrapinn.com
t/ +1 646 619 1775

 

 

 

Marketing & Press Opportunities

 
 

Kaylie Brogan
kaylie.brogan@terrapinn.com