Conference Agenda
18 Orphan Drugs & Rare Diseases 2022 Americas - Day 2
Day 2: Wednesday - 30th November 2022
Kelly Athman, Sr. Director, Medical Affairs, InformedDNA
ACCESS & COLLABORATION
- The foundational value of building collaboration through trust, respect, transparency, and empathy
- Not all advocacy organizations are alike: assessing the landscape
- Real-world examples of innovative digital solutions driving collaboration among multiple stakeholders
- Value of partnering with advocacy organizations to create innovative patient-driven solutions
Moderator:
Jodie Sherman Gillon, SVP Corporate Affairs & Patient Advocacy, Peptilogics
Panellist:
Jennifer McNary, Exec. Director, Head, Patient Advocacy & Engagement, Fulcrum Therapeutics
Frank Rivera, Co-Founder/President, Stronger Than Sarcoidosis
Jeremy Griffin, Executive Director, New York City Hemophilia Chapter
Craig Martin, CEO, Global Genes, President, Rithm Health
- Attendees will come away with a better perspective of when, and how to engage patients, advocacy organizations and caregivers
- Talk will cover the benefits of early and consistent engagement on recruitment, retention, regulatory and access issues.
Jennifer McNary, Exec. Director, Head, Patient Advocacy & Engagement, Fulcrum Therapeutics
- Patients often take on the role of research partner or Citizen Scientist. The benefits of such a partnership include
increased public access and involvement of patients in research, immersion of patient community values relevant
to research, outreach and educational potential with the community, and ultimately, the democratization of
science
Dr Neena Nizar, Founder & Executive Director, The Jansen’s Foundation
- Creating patient-clinician relationships that create a sense of ease and trust
- Addressing issues on practical challenges
- Improving access and supporting patients
- Multi-disciplinary approach and patient centered support to improve accessibility and further advances which will impact the next generation aiding patients of the future
Alan J. Balch, Chief Executive Officer, Patient Advocate Foundation
- Creating opportunities for rare disease innovations through state and federal policy
- The power of rare disease collaborations
- Keeping patient voice prominent in policy and collaborations
Tara J. Britt, Founder & President, Rare Disease Innovations Institute, Inc.
- Education of the process of clinical trials( in easier terms so patients can understand)
- Listen to the Patient needs and help them to understand what the focus of the trial is. (Break the stigma of Us vs.
Them, Pointing of fingers) - Understanding Pharmaceutical costs of trials and research
- Dealing with an illness with multiple organ involvement. (how multiple organ disease hinders a patient’s
involvement in trials) - Rare Disease ( Limited pool for Trials, Patient distrust, Diversity in Patients)
Frank Rivera, Co-Founder/President, Stronger Than Sarcoidosis
- A case study from a pioneering company that blazed a trail with a first-of-its-kind tissue-based regenerative
medicine launch - Challenges and opportunities to consider when launching a commercial product for the rare disease community
- Best practices from R&D to manufacturing to commercialization
Johanna Rossell, Chief Commerical Officer, Enzyvant
- Contextual considerations for value evidence in Rare Diseases
- HTA and patient access for orphan drugs – state of affairs
- How can we build an ecosystem for rare diseases?
Simu Thomas, Vice President & Global Head HEOR, Alexion, AstraZeneca Rare Disease
- Successful example of value-based pricing in Rare Disease
- Potential changes in treatment paradigms to improve effectiveness and reduce cost of treatment
- Legislative incentives to encourage development of orphan rare diseases therapies
- Impact of orphan diseases treatment on healthcare payment system
Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech. Evaluation, Independence Blue Cross
- Breaking down barriers for access
- Strengthening health systems around the world and accelerating pathways to diagnosis
- 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:
Dr Neena Nizar, Founder & Executive Director, The Jansen’s Foundation
Panellist:
Tara J. Britt, Founder & President, Rare Disease Innovations Institute, Inc.
Frank Rivera, Co-Founder/President, Stronger Than Sarcoidosis
Kathleen Coolidge, Patient Advocacy, Pulse Infoframe
Kelly Athman, Sr. Director, Medical Affairs, InformedDNA
