Day 1 Congress – 5th March 2019

  • 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
Zizi Imatorbhebhe, Sr Director, Strategic Dev't, Head of PAREXEL’s Rare Disease Center of Excellence, PAREXEL International
Alain Rinaldi, CEO & Founder, MEDExpansion, CMO & Co-founder, Overlord Pharmaceuticals
Pedro Franco, Director of Europe, Global Regulatory & Scientific Policy, Merck KGaA

  • 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

Markus Peters, IGES Institute North America, former Chief Commercial Officer Agilis Biotherapeutics

  • 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
Pan Pantziarka, Program Director, Drug Repurposing, Anticancer Fund
Farhat Khanim, Research Scientist, University of Birmingham

  • 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 advances in science are revolutionising the treatment of rare diseases
  • Putting patients first in the treatment of rare diseases requires creating equitable access solutions
  • Our shared responsibility to leave no patient with a rare disease behind

Owen Marks, Country Lead Rare Diseases, Pfizer

  • An overview of different legislations & regulatory framework
  • Different policies
  • Different definitions of orphan drugs & rare diseases
  • Limit industry and academia resources

Pedro Franco, Director of Europe, 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

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

  • The foundational value of building collaboration through trust, respect, transparency and empathy
  • Not all advocacy organizations organizations are alike: assessing the landscape
  • Real world examples of innovative digital solutions driving collaboration among multiple stakeholders
  • It isn’t easy so why do it? Innovative partnering yields unique benefits boosting capabilities, medicine development and

T Anthony Howell, Esq., Co-Founder & Digital Community Builder, rareLife solutions, Creators of

Frank Grossmann, Founder & Executive Director, Orphanhealthcare Foundation and CEO, Orphanbiotec AG

  • 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

  • New ideas, new paradigms, new levels for discussion are needed to foster technological and medical progress in the area of rare diseases and orphan drugs development in Central and Eastern European countries due to healthcare systems resistance to rare
  • “PATIENTS’SCIENTIFIC INSTITUTE”creates new quality & opportunities in entire area of rare diseases and orphan drugs development. It’s created by patients and for patients. It’s also created by young scientists, students of medical and life sciences and experienced researchers. PSI is a think-thank, functioning on non-profit basis, focused on R&D in rare diseases and providing such research directly to patients.
  • We believe that for rare disease patients access to possible treatments= access to research, including clinical trials designed according to new paradigms with strong patient involvement from the early stage of studies;
  • No political activities, no-policy-making;
  • Involvement of local biotechnological SMEs as well as big pharmaceutical companies;
  • Disease-focused working groups within PIN working on patient/ disease registries, codes of conduct;
  • PIN is governed by patients and patient- centred, patient-focused initiative.

Rafal Swierzewski, President of Board, Foundation for Patients with Rare Diseases COCARDA

  • 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