Day 2 Congress

  • Growing up with a fatal hereditary rare disease in my family and knowing I had a 50/50 chance of getting it
  • The disease took the lives of 12 family members before we were given a name Hereditary Sensory Autonomic Neuropathy Type 1E (HSAN1E)
  • It was through genetic testing from my brother and sister we were able to get a diagnosis.
  • Caregiving for my brother and sister
  • Deciding to get tested for HSAN1E, living with a negative result
  • Starting a non-profit to find other families with HSAN1E

Rachelle Dixon, President / Co-Founder, HSAN1E Society

  • Why must we ask why there is no treatment?
  • How can we achieve the goal?
  • The way forward

Richard A. Basile, Co Founder and CEO, BioPontis Alliance for Rare Diseases

  • Review of drivers of pre-approval drug access and the “right to try”
  • Overview of relevant regulation and legislation in the US
  • Consideration of program guidelines in other countries
  • Perspectives of key stakeholders: patients, families, advocates, regulators, media, payers, companies
  • Implications for effective program planning
  • Special considerations for rare and genetic diseases
  • Best practices in program design, implementation and evaluation

Mark Krueger , Chief Strategic Officer, MK&A Inc.

  • Engaging key stakeholders: patients & caregivers, clinicians and advocacy groups
  • Empowering patients (and their families) as the experts
  • Understanding the patient journey - participate, listen and learn
  • Building partnerships that address unmet needs
  • Why support must extend beyond our medicines – the role of industry in the community

Vikram Karnani, Executive Vice President, Chief Commercial Officer, Horizon Pharma

  • What are the unique needs of collaborations for rare disease patients?
  • What are more novel areas for collaboration? unmet needs, diagnosis, access, ancillary support
  • What are novel methods and models for collaboration?
  • Which other stakeholders should be involved and how?
  • What’s the ideal we should strive for?


Jodie Gillon, Global Medical Lead, Patient Engagement Rare Diseases, Pfizer Inc.
Isabelle Lousada, CEO & President, Amyloidosis Research Consortium

  • Patient participation in rare disease management is critically important for success
  • Trusted and purposeful relationships between patients and pharmaceutical companies drives patient resources and support programs
  • CASE STUDY: Rarity’s 360° Patient Experience

Mahesh Kolar, Vice President, Strategy and Business Development, Rarity Health

  • How are activists carrying the collective voice of the patients and represent the patients’
  • How can leading groups advance understanding and awareness of rare diseases by unifying patients
  • Case studies: stories of success and failure
  • Strategic implications for biopharma companies, planning to expand into Europe

Moderator:
Richard A. Basile, Co Founder and CEO, BioPontis Alliance for Rare Diseases
Panelists:
Khrystal K. Davis, CEO and Patient Advocate, Zebra Leaf Publishing
Michael A. Swit, Esq., FDA Legal Counsel, Law Offices of Michael A. Swit
Mahesh Kolar, Vice President, Strategy and Business Development, Rarity Health
David Cory, President and CEO, Eiger BioPharmaceuticals

  • Current situation in Europe from a regulatory and a market access perspective
  • Pricing and reimbursement: key success factors/li>
  • Case studies: stories of success and failure
  • Strategic implications for biopharma companies, planning to expand into Europe

Matthias P. Schönermark, Managing Director, SKC

  • Strategy of providing early access in OD
  • Key logistics in providing early access
  • From early to greater access

Stephan Toutain, SVP, Operations, Anavex Life Sciences

  • We are committed to treating rare and ultra-rare diseases
  • Drug development is a team sport and it is critical to attract and retain top talent
  • Collaboration across the organization is necessary for advancing programs effectively

Bob Ward, Chairman, Chief Executive Officer, Eloxx Pharmaceuticals

  • Successful example of value-based pricing in Rare Disease
  • Potential changes in treatment paradigms to improve effectiveness and reduce cost of treatment
  • Implementing measures designed to limit their exposure to costs associated with orphan drug reimbursement.
  • Legislative incentives to encourage development of orphan rare diseases therapies
  • Impact of orphan diseases treatment on healthcare payment system

Moderator:
Christian Girard, Co-Founder & CEO, Overlord Pharmaceuticals
Panelists;
Matthias P. Schönermark, Managing Director, SKC
Stephan Toutain, SVP, Operations, Anavex Life Sciences
Bob Ward, Chairman, Chief Executive Officer, Eloxx Pharmaceuticals
Ken Kengatharan, CEO, Auxesia Orion and Managing Partner, Atheneos Ventures

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