Day 1 Congress

• 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
• Updates on the natural history studies
• Issues for evaluating and validating biomarkers as surrogate endpoints
• Flexibility on the non-clinical aspects of the evaluation of novel drug compounds
• Consideration of safety questions and added information on changes to drug substance or drug product manufacturing process
• What can be done by healthcare providers to be proactive in monitoring side effects that can put patient safety at risk and effectively communicate results to the developer?
• Why should patients’ information about their treatment experience be collected and how can this steer the evolution of orphan drugs?
• How social media plays a role for developers and patients to communicate and how companies can utilize social media to innovate and create new drugs for patients?
• How are advance therapy fundamentally changing the way rare diseases patients are treated
• How policy makers and regulators approach Advance Therapy Medicinal Products (ATMP)
• What are the new development in Advance Therapy Medicinal Product
• Incentives Supporting Development of ATMP
• 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
• Making commercialization viable by tackling high cost
• Improving clinical development by new age clinical trial design and recruitment
• Overcoming challenges in manufacturing and supply chain
• Understanding diseases thru national history
• 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
• Current state of RNAi therapeutics development
• Overcoming challenges in developmental process of RNAi treatment
• Overcoming the challenges of delivering RNAi to new cell types
• Leveraging the potential of RNAi technology
• RNAi therapies in the pipeline
• Selection of the study population
• Selecting appropriate study design for research
• Statistical analyses, the use of biomarkers, primary endpoint definition and proving drug efficacy
• Pharmacovigilance considerations for characterisation and risk management
• Timely and adequate recruitment of eligible trial participants
• Development of efficient trial designs relevant to small populations to gain the most information from the available data
• Practical conditions to be considered when conducting a clinical trial for rare diseases
• Promoting international collaboration and advance rare diseases research worldwide
• Patients as Key Partners in Drug Development
• Role of Real World Evidence in Rare Disese
• Value of Registries in Rare Disease – pros and cons
• Understanding the collective voice of the patients and represent the patients’ interest
• Patient involvement is crucial for identifying the questions to ask and the outcomes to assess
• Patient involvement is essential for achieving true translational research
• The identification and proper handling of patient needs in clinical research will yield advantages in terms of clinical and economic benefits.
• Overview of current methods for their prevention, diagnosis, and treatment.
• Strengths and limitation of current development pathways for new drugs, medical devices, and biologics for rare diseases
• Special challenges that rare diseases create for research and product regulation
• Current public policies relevant to product development for rare diseases

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