Event Schedule

The biggest mistake providers make is following antiquated diagnostic criteria that neglects the real diversity of the phenotype.  For autoinflammatory disorders like Still’s Disease, current research lacks adequate tools for monitoring or quantifying both primary and secondary efficacy treatment endpoints.

  • Review advances in serology, targeted treatments, and key systemic findings
  • Target both pediatric and adult rheumatology and immunologists
  • Create earlier options for detection, research, and collaboration

Rare is different. We know that. We hear time and again how important the voice of the patient is in drug development and commercialization. We buy into it. We believe it. We work to make it happen. But then, when it comes to some important decision makers, sometimes it feels as though there is no consideration for the rare disease patient in the determinations they render. This presentation will review 3 very recent experiences in which the plight of the rare disease patient was not considered.

  • Determine value of rare disease therapies
  • Use Real World Evidence to show orphan drug value and inform HTA evaluations 
  • Center the role of patients in demonstrating the impact of orphan drugs
  • Ensure regular interaction with the chronic illness patient and advocate community
  • Streamline communication with those doing the research and inspire them to do more
  • Expand genetic research efforts while keeping genetic information confidential and safe