22nd Orphan Drugs and Rare Diseases Global Congress 2024 Americas
Enhance Information Sharing, Improve Collaboration to Increase Drug Approval for Rare Diseases
Boston, MA, USA
Thursday 14th - Friday 15th November 2024
Facilitate Live is again proud to present our Orphan Drugs and Rare Diseases Global Congress 2024 Americas. It’s the 22nd in the series of our Flagship tri-annual Orphan Drugs & Rare Diseases event. This congress will provide you with a comprehensive overview of the critical issues shaping the future of Orphan Drugs.
In the United States, 1 in 10 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 will provide an interactive, cutting edge, and comprehensive discussion and networking format led by key experts with intimate knowledge in the industry. Gain practical strategies and best practices on challenges, innovations, technologies, and concepts surrounding orphan drugs and rare diseases.
We look forward to meeting you at the Congress!
Sincerely yours,
Jocelyn Raguindin
Conference Director
Facilitate Live
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 —