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
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:
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