Agenda at a Glance

Orphan Drugs Conference Day 1

Day 1: Tuesday 17th March 2020

  • What is it like living with a rare disease?
  • What impact does rare disease have to the patient and caregiver?
  • Experiences when communicating with healthcare professionals
  • Social and economic implications of living with rare disease

  • How to find the right patient and design a collaborative clinical trial?
  • Developing strong economic models in demonstrating the value of subsidizing patient healthcare costs
  • The need for accurate forecasting to ensure a successful market access
  • Optimizing a supply chain management system
  • Establishing a well-informed sales and marketing plan
  • Engaging and empowering patient

  • How are this advance therapy fundamentally changing the way rare diseases patients are treated?
  • How are policy makers and regulators approach gene therapy products?
  • Improving clinical development by new age clinical trial design and recruitment
  • Effective framework for developing the delivery system for the next generation of medicine.
  • Making manufacturing and commercialization viable
  • Reimbursement models for gene therapies
  • Gene therapies in the pipeline

  • 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

  • Current state of RNAi therapeutics development
  • Overcoming challenges in developmental process of RNAi treatment
  • Addressing the challenges of delivering RNAi to new cell types
  • RNAi therapies in the pipeline

  • Understanding the global rare disease policy landscape
  • New steps to meet the challenges of rare diseases
  • What are the key elements across diverse rare disease programs and how this can affect patients access to care
  • Bridging gaps between policy and practice to guarantee access for patients

Commercialization, Current Trends & Collaborations

How can governments and orphan drug companies help support each other’s initiative to provide access to patients?

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?

How does collaboration help clinicians and patients with rare diseases wanting to access new treatment?

International Collaborations in Orphan Drug Development – Making It Work

Challenges in bringing orphan drugs to patients after approval

Access, Pricing & Reimbursement

Advancing Orphan Drugs Innovation and clinical development to facilitate patient access

Breaking down barriers to Patient Access: Bringing the stakeholders together

Challenges surrounding funding for orphan drugs specifically for HTA

Strategies for Implementing value- based pricing and reimbursement for orphan drugs

How Are Payers Treating Orphan Drugs?

Means of ensuring access and affordability of Orphan drugs

Innovations, Discovery & Clinical Development

Accelerating Scientific development for rare disease from breakthrough technologies

Regenerative medicine therapies for Rare Diseases

Special challenges that rare diseases create for research and product regulation

The Importance of Patient Networks and Advocacy Groups in Designing of Clinical Trials and Patient Recruitment?

Designing and running adequate clinical trials for orphan drugs

Unique approaches in clinical trial design for rare diseases

Advance Therapy Medicinal Products

Advance therapies: Promises and potentials for rare disease

Clinical Considerations for gene therapy for rare disease

Advance therapy fundamentally changing the way rare diseases patients are treated

Leveraging the potential of RNAi technology

Patients and the Value of Rare Therapies

Gene Therapy Trial with Randomized Enrichment Design with integrated Real-World Evidence

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