- 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
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*
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*
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