The Challenge

Rare Diseases affect 25 to 30 million Americans collectively1. While there is a lot of research being done, there is still a need for all stakeholders (patients, caregivers, health care providers, academics, and industry leaders) to come together to understand how to serve and empower this patient population.

Life science consultants working on high tech laptop

What We Did

  • Identification and creation of a comprehensive list of community and research based HCPs working with rare disease patients
  • Coordinated professionals (Health Care Providers) to participate in advisory boards across the world
  • Created an agenda to gather insights with both synchronized and asynchronized meeting activities
  • Developed detailed discussion prompts, presentations materials, and pre-read content scientific publications expanding on the current etiology of disease, diagnosis, symptoms, prognosis and the delicate attributes pertaining to pediatric diagnosis and care
  • Coordinated thought leader presentations for the synchronized portion of the agenda and discussion prompts for both segments (synchronized and asynchronized) to promote conversation and insight gathering

Results Delivered

  • Independently curated a list of professionals (Health Care Providers) as advisors from publications review and biography assessments
  • Developed presentation materials for Advisory board Meetings
  • Planned and managed a virtual platform, for multiple countries for live Rare Disease Advisory Board Meetings with Health Care Providers
  • Hosted an asynchronized web portal for Health Care Providers to access research on Rare Disease with a follow up live session with advisors
  • Readout with key insights as a result of an in-depth discussion amongst Health Care Providers to express the diagnosis, prognosis, and management of the Rare Disease

Key Outcomes

  • Gained insights into unmet needs of the Rare Disease
  • Uncovered regional differences in the diagnosis and management of the Rare Disease
  • Delivered strategic input regarding:
    • Challenges in diagnosing
    • Patient journey and concerns upon diagnosis (e.g., treatments, inheritance patterns, prognosis, and ways to slow progression)
    • Obstacles that can be overcome with education for patients, families, HCPs, and Payors
    • The need for new Rare Disease treatments

Reference:

  1. https://www.nih.gov/about-nih/what-we-do/nih-turning-discovery-into-health/promise-precision-medicine/rare-diseases#:~:text=Rare%20diseases%20were%20once%20considered,30%20million%20Americans%20are%20affected