Day 1 Congress

Femida Gwadry‐Sridhar, Founder & CEO, Pulse Infoframe

  • 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



  • 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

Femida Gwadry‐Sridhar, Founder & CEO, Pulse Infoframe


  • 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

  • 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

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
  • Selective inhibition of CV ATP hydrolysis prevents ATP depletion independent of respiratory

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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To Speak

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Tel. +44 203 567 1321
Tel. +44 203 567 1321
Tel. +44 203 567 1321