- A plethora of issues and challenges experienced by person with rare disease and their caregivers
- The impact on patient and of caring for someone with a rare disease
- What support options are available for carers of people with a rare disease(s) from health and social
care providers, charities or support groups. - What challenges still remain to increased equity for people living with a rare disease and their
families?
Moderator:
Panelists:
- Allyson Damikolas, Mother & Carer to a Rare disease patient
- Audrey Damikolas, Rare Disease Patient
- What are the growing trends in Rare Disease treatment
- How can governments and orphan drug companies help support each other’s initiative to provide access to patients?
- 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:
Femida Gwadry‐Sridhar, Founder & CEO, Pulse Infoframe
Panelists:
- Review common issues
- Trends in approvals and INDs
- International collaborations
- PFDD and COAs for Rare diseases and the role of the patient
- Recent guidances; Pre-IND, Natural History, Eligibility Criteria
- How do you generate RWD and evidence in support of your initiative to ensure meaningful results?
- What data types and structure is needed in support of your research?
- The important role of patients in evidence generation and how to keep them engaged?
Femida Gwadry‐Sridhar, Founder and CEO, Pulse Infoframe
- Key considerations accelerating Orphan Drug development and commercialization
- Learn about challenges facing the developer and strategies to overcome them
- Best practices for developing orphan drugs, and how to apply them
David Rintell, Vice President, Head of Patient Advocacy, BridgeBio.
- How an EAP aligns with orphan drug development and commercialization plans.
- Impacts of CoVid-19 on the Early and Expanded Access environment.
- The role of Real World Data and how EAP’s can provide an ideal platform to collect the data..
- EAP’s as a long term plan for the launch in non-commercialized countries.
- Practical guide to initiating a successful EAP.
Robert Donnell, Executive Vice President, Medicines Access, Smartway Pharma
- How is this advanced therapy fundamentally changing the way rare diseases patients are treated?
- 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
- Making manufacturing and commercialization viable
- Unconventional payment models to ensure innovative treatment can be accessed by rare disease
patients
Panelist
Monica L Weldon, President/CEO/Founder, SYNGAP1 Foundation
- Addressing challenges for Rare diseases therapy development
- Current strategies to advance rare diseases therapy development
- Efficient and effective models for therapy development that are scalable and sustainable
- ATP hydrolysis by ATP synthase can co-exist with ATP synthesis.
- CV ATP hydrolysis is increased in conditions of impaired respiratory function.
- Epicatechin is a competitive mimetic of ATPIF1, selectively inhibiting ATP hydrolysis without inhibiting
synthesis. - Selective inhibition of CV ATP hydrolysis prevents ATP depletion independent of respiratory
dysfunction./li>
Cristiane Benincá, Imaging Core Director, UCLA Metabolism Theme
- Addressing challenges on timely and adequate recruitment of eligible trial participants.
- How to overcome different regulations and requirements of regulatory authorities?.
- Development of efficient trial designs relevant to small populations to gain the most information from
the available data. - > How, in this highly regulated sector, to get this innovative treatment onto the market in time to help
patients with orphan conditions? - > Promoting international collaboration and advance rare diseases research worldwide
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