Day 1 Congress

  • How can governments and orphan drug companies help support each other’s initiative to provide access to patients?
  • Manufacturers collaboration with governments, payers and patients to continue fulfilling unmet needs?
  • How are small biotech start-ups contributing to the orphan drugs market?
  • How can developers demonstrate value for orphan drugs?
  • How can manufacturers meet global patient demand for access to medicines prior to approval?
  • What R&D strategies can be implemented to accelerate the market access for orphan drugs?

Christian Girard, Co-Founder & CEO, Overlord 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


Ken Kengatharan, CEO, Auxesia Orion and Managing Partner, Atheneos Ventures

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

Matthias P. Schönermark, Managing Director, SKC Beratungsgesellschaft mbH

  • Why should drug repurposing considered a sustainable strategy for rare diseases?
  • Assessing the commercial roadblocks for growth of repurposed drugs
  • Is there a major role for repurposing of old drugs for new uses in orphan and rare disease space?
  • Examples of collaborative models to drive generic drug repurposing in rare diseases

Cesare Spadoni, PhD MBA, Chairman, aPODD Fundation (UK)

Rick Thompson, CEO, Findacure

Current technological approaches and challenges
Review of the cutting-edge research programs and clinical trials
Advances in gene therapy
What is on the horizon for gene therapy for orphan drugs?

  • Challenges faced when embracing a completely new model of healthcare
  • Technology needed to develop and deliver targeted therapeutics
  • How to design clinical trials for small patient group

  • How to navigate the current regulations
  • How are these regulations influence time to market for orphan drugs?
  • Regulatory obstacles faced by manufacturers during the registration process
  • Designing best strategies for a successful regulatory approval

Pedro Franco, Director of Europe for Global Regulatory & Scientific Policy, Merck KGaA*

Review of the EMA’s orphan designation program
Range of incentives to encourage the development of medicinal products for the treatment of rare diseases
Current development on EU case law and legislations
Orphan drugs pipeline in the EU

  • What can stakeholder do to make orphan drugs more affordable to patients?
  • Stakeholders strategies to encourage progress on orphan drugs policies
  • How are government collaborating with stakeholders to make orphan drugs readily available to patient?
  • Stakeholder cooperation to overcome challenges in orphan medicine development

Mercedes Martínez Vallejo, Technical Advisor, Ministry of Health, Consumption and Social Welfare, Spain*

T Anthony Howell, Esq., Co-Founder & Digital Community Builder, rareLife solutions, Creators of
The collaborative model with biotech /pharma companies,
Support clinical development through EU funding and
Creation in partnership with the french Venture capital Sofinnova Partners, of a dedicated fund to invest in early stage project in the field of rare/genetic diseases to accelerate development of new therapies.

Lucia Faccio, Director of Research & Development, Fondazione Telethon

  • How can manufacturers work with regulators in understanding regulatory issues and challenges to minimise risks during development?
  • How should the R&D and commercial leaders in pharma anticipate and sort out the public policy risks and benefits of adopting an orphan drug development and commercialization strategy?
  • The future of approving drugs thru orphan drugs designation pathway particularly in near and long-term changes that are coming in the future with respect to orphan drugs approval process