- 14th - 15th November 2024
- Boston, MA, USA
22nd Orphan Drugs & Rare Diseases Global Congress 2024 Americas
Enhance Information Sharing, Improve Collaboration to Increase Drug Approval for Rare Diseases
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Orphan Drugs & Rare Diseases Global Congress 2024
Dear Colleagues,
Facilitate Live is again proud to present our Orphan Drugs and Rare Diseases
Global Congress 2024 Americas. It’s the 20th in the series of our Flagship Orphan Drugs &
Rare Diseases event. This congress will provide you with a comprehensive overview of the
critical issues shaping the future of Orphan Drugs and Rare Diseases.
In the United States, 1 in 10,000 people, or around 30 million Americans are affected by more
than 10,000 known rare diseases. Rare disease are classified as any disease which affects less
than 200,000 Americans. Most rare diseases are life-threatening and many does not have
treatment. Half of the sufferers are children and 30 percent of them will die before the age of 5,
due to lack of available treatment. The complex biology and lack of understanding of the
natural history of rare diseases makes drug, biologic, and device development in rare diseases
very challenging.
Accurate diagnosis and treatment for these diseases has also been teeming with regulatory and
logistical barriers. There is an urgent need for a regulatory framework that encourages safe
therapeutic innovations and helps ensure their timely access to patients.
According to reports (Fortune Business Insights), the global Orphan Drugs Market size is
projected to reach USD 368.43 billion in 2030, at a CAGR of 11.6% during the forecast period,
2023-2030. Biological Orphan drug which has been use in treating rare diseases and in oncology
therapy area possesses a substantially higher share in the global market as new cancer-related
disease emerges and series of FDA approvals will result in increased commercialization of these
drugs. The growing demand for immunomodulators will also contribute to the orphan drugs
market growth. Increasing R&D initiatives and investments by prominent players and patient
advocacy involvement have resulted in major pharmaceutical breakthroughs, and the
development of blockbuster drugs for the treatment of several rare diseases will create
opportunities and contribute exponentially to the overall market growth.
The two-day Congress aims to offer insights into how to improve the diagnostic process and
access to treatments. Strategies and best practices, innovations, technologies and concepts that can promote rare diseases product development will be discussed in an interactive, thought-
provoking and uplifting manner.
We look forward to welcoming you to the congress!
Jocelyn Raguindin
Jocelyn is Conference Director at Paradigm Global Events (PGE). She has over 15+ years experience in organizing pharma-industry conferences and has been at the helm of every PGE conference since 2013. Jocelyn is based in London, UK.
GAIN LATEST INSIGHTS ON:
- Commercialization of Orphan Drugs: Top issues facing the community today
- What are the major drivers and therapy areas that possesses a higher market share
- Key developmental strategies carried out and Influencing factors that may affect market
share to stand out in this industry - Increasing R&D initiatives and investments by prominent players and patient advocacy
involvement that resulted in major pharmaceutical breakthroughs - Patients perspective: what really matters to rare disease patients and caregivers?
- Regulatory framework that encourages safe therapeutic innovations and helps ensure
their timely access to patients. - Impact of orphan drug legislation on innovation and solidification of the biotech industry
- Trends and evolution of Advanced Therapy Medicinal Products
- Trends and Strategies in the Development of Orphan Drugs for Oncology Indications
- Recent discoveries, development and innovation that impacts rare diseases
- How to improve the diagnostic process and access to treatments
- The importance of patient networks and incorporating diverse patient voice in diagnosis
and clinical trial recruitment - Global collaborations to improve diagnosis and care for persons living with a rare disease
- Sustainability, pricing legislation, and multi-stakeholder management in addition to the
patient - Opportunities and challenges for Rare Disease policy shaping in Emerging Markets
- Rare diseases and Mental health
- Opportunity to network with Peers, potential Partners and Investors
WHO SHOULD ATTEND?
This congress is specially created for valued stakeholders in the Rare Disease community:
Presidents, Heads/Chiefs, VPs, Directors, and Managers in the area of:
- Clinical Research Organizations
- Research and Development
- Personalised Medicine
- Regenerative Medicine
- External R&D Innovation
- Innovative Medicine
- Rare and Ultra-Rare Diseases
- Cell and Gene Therapy
- Translational Science
- Molecular Geneticist
- Program Management
- Patient Advocacy Groups
- Public Affairs
- Medical Affairs
- Regulatory Affairs
- Market Access
- Pricing and Reimbursement
- Health Economics Outcomes Research
- Commercial Development
- Investments and Funding
- Product Specialists
- Global Strategic Services
- Business Planning and Operations
- Pharmacies
- Academia
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Key Industry Expert Speakers
Dan Lewi
Head, Business Development and Patient Advocacy
Pulse infoframe Inc., Co- founder, CATS Foundation
Schedule
Content Rich Program Agenda! Featuring Keynote Presentations, Stream Sessions, Panel, and Round Table Discussions.
Conference Agenda
Day 1: Thursday - 14th November 2024
MACRO OUTLOOK AND CURRENT TRENDS
- What innovative changes are coming in research, clinical development, manufacturing, registrational paths, and commercial models that will impact the rare disease community
- Regulatory Framework for Drugs for Rare Diseases
- Insights and recommendations for those preparing for their go-to-market
- Stakeholders collaboration to continue fulfilling unmet needs?
- What R&D strategies can be implemented to accelerate the market access for orphan drugs?
- Challenges in bringing orphan drugs to patients after approval
Moderator:
Panelist:
- After FDA approval, what are the accessibility challenges that rare disease patients face (esp those on state programs)?
- What are some policy changes that could increase the ability for patients to access treatments in a timely manner?
- How are states grappling with the sustainability challenges in the orphan drug space?
Erica Barnes, Executive Director of the Minnesota Rare Disease Advisory Council
- Understand the impact of advanced therapies on commercial models
- Examine innovative ways in patient finding
- Discuss ideas for getting patients to the right treatment center
Bennett M. Smith, SVP, GM, North America, Orchard Therapeutics
- This presentation will focus on the role of RWD in natural history and patient registries.
- How data can be utilized to help inform study design
- The use of RWD as an external control and to find eligible patients for trials
- Using RWD with prior clinical trial data to better understand patient outcomes.
Dr. Femida Gwadry-Sridhar, Founder and CEO, Pulse infoframe Inc.
- Determining accurate patient population estimates for rare diseases is critical at all stages of drug development from R&D through to securing orphan drug status and commercialisation.
- Standard methods pose significant limitations due to the paucity of data, which presents risks to decision-making processes.
- The availability of genetic databases has enabled a new “bottom-up” approach to establishing accurate data on patient populations, and mitigating such risks.
Mohamed H. Ladha, President & GM, North America, RECORDATI RARE DISEASES
- Overcoming hurdles to getting innovative therapies to more rare diseases patients around the world
- Improving clinical development by new-age clinical trial design and recruitment
- An effective framework for developing the delivery system for the next generation of medicine
- Unconventional payment models to ensure innovative treatment can be accessed by rare disease patients
- Technologies that improve scalability, decrease the cost of goods, enhance overall safety, or increase durability
- Making manufacturing and commercialization viable
Moderator: Alison Schecter, Chief Executive Officer, Molecules to Medicine Advisory Group
Panellist:
- What is FDA’s Project Optimus Oncology Initiative and draft Optimal Dosage Regimen (ODR) guidance for oncology?
- How does one apply FDA’s Project Optimus and ODR guidance to developing drugs for orphan oncology indications?
- For orphan oncology indications, does the requirement of having an acceptable balance between benefit and risk for approval conflict with the requirements of Project Optimus and the ODR guidance?
David Young, President, Research & Development, Processa Pharmaceuticals, Inc
- In vivo phenotypic screens can identify therapeutic targets underlying common pathological problems in neurological and rare disorders
- KCC2 potentiating therapies for neurological and rare disorders involving excitation/inhibition imbalance and neuronal circuit disinhibition in the CNS
- Orphan drug indication prioritization and commercialization: Portfolio of KCC2 therapies to match target-product-profile (TPP) and modality to patient needs
Shane Hegarty, Chief Scientific Officer & Co-Founder, AXONIS Therapeutics, Inc.
- Definition of Orphan and Ultra-orphan Indications in Oncology
- Challenges of Development: The Regulatory and Developmental Environment
- Role of Big Pharma: Recent Trends
- Prospective Strategies to Overcome the Challenges
Sanjeev Luther, President & CEO, Cornerstone Pharmaceuticals
- To Follow Shortly
Amanda Moore, CEO, Angelman Syndrome Foundation
- To Follow Shortly
Christian Rubio, Executive Director, EverythingALS
- Overview of current methods for their prevention, diagnosis, and treatment.
- Strengths and limitation of current development pathways for new drugs, medical devices, and biologics for rare diseases
- Drug repurposing for rare diseases
- Special challenges that rare diseases create for research and product regulation
- Current public policies relevant to product development for rare diseases
— END OF DAY 1 —
Conference Agenda
Day 2: Friday - 15th November 2024
ACCESS & COLLABORATION
- Everything patients and families want you to know but haven’t had the platform to share
- An inside look of the patient/family ecosystem and how clinical research fits in
- Changing the paradigm – the ROI on the investment needed to ensure the most important stakeholders in clinical trials feel valued
Jennifer McNary, Founder, One Rare
- Reducing Isolation and improving best practices for clinicians through structured networking and engagement
- Creating tools for improved patient communication
- Moving beyond story telling to joint strategies to improve care and patient outcomes.
Tricha Shivas, Chief of Staff and Strategy, Foundation for Sarcoidosis Research
- Siblings are an often-overlooked member of the rare disease ecosystem but play a valuable role and have unique insights into rare disease.
- Siblings can, and should, be seen as caregivers in rare disease as they often play a pivotal role in activities of daily life within the home
- The phrase “unaffected siblings” is inaccurate for while a sibling may not have the rare disease, they are still affected in many aspects of their life
- How can we generate more evidence to support the important role that siblings play in caregiving?
Joslyn Crowe, Vice President, International Niemann-Pick Disease Alliance (INPDA)
- We are focused on putting rare disease patients at the center of everything we do and are committed to letting the patient voice shape who we are
- We have the opportunity to remain close to the patients, caregivers and their providers, always learning from them and responding to their needs.
Stuart Siedman, Global Head Patient Advocacy, Rare Diseases, Chiesi
- To follow
Daniel Chung, Chief Medical Officer, SparingVision
- Emphasis of the role of education in uniting industry stakeholders and patients in healthcare.
- Informed patients power to make better decisions and enhance treatment access.
- The potential of educational programs by grassroots patient organizations
Chisa Nosamiefan, Co-founder, The Labalaba Foundation for Lupus
- The number one reason patients don’t participate in clinical trials is because of the burden of travel. We can solve that problem.
- Patient advocates spend too much time away from their family to fundraise. We can solve that problem.
Luke Rosen, CEO, Rescue7, CEO, KIF1A.ORG
- Mental Health is part of total Health
- The incidence of mental health disorders is significantly higher in individuals with a rare disease, compared to the general population.
- Remove the stigma concerning Mental Health and asking for help through constant conversation.
- Coping and Awareness of Mental Health in the Rare Disease Community
- Rare Disease organizations and Pharmaceutical companies should make Mental Health a priority
- Who and where to ask for help for Mental Health?
Frank Rivera, Co-Founder/President, Stronger Than Sarcoidosis
- Rare disease programs have historically leveraged creative methods to deal with difficulties inherent in orphan disorders
- The industry is evolving to address new roles in sustainability, pricing legislation, and multi-stakeholder management in addition to the patient
- Partnerships can make rare disease programs more successful in meeting and surpassing new industry standards
- Breaking down barriers for access
- Strengthening health systems around the world and accelerating pathways to diagnosis including EU-sponsored international initiatives
- Stakeholders to address the rare diseases’ ecosystem gaps to collaboratively build a sustainable roadmap for better health and a brighter future for the patients suffering from rare diseases.
- Challenges and opportunities in creating sustainable healthcare for all.
Moderator:
Panelist:
— END OF CONFERENCE —
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Testimonials
what people say
From a patient advocacy organization perspective, was a very informative meeting. Learned a lot from presentations and networking that will inform how we can add value to the advocacy-patient-drug developer dialogue.
Dr. Nadine Tutton
Scientific Director, Research, Association for Frontotemporal Degeneration
I was very impressed with the format, the content was interesting and well done. I felt I learned a lot and was glad to be able to attend.
Senior Director
Business Development at Pharm-Olam, LLC
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Location
- Boston, MA, USA
- info@paradigmglobalevents.com
- +44 (0) 207 193 3485