Case Study – January 14, 2020

Rare Disease Task Force – Africa

Situation

FYMCA Medical Ltd, a UK-based company that develops improved rare disease education and services for the developing world, wanted to identify problems impacting the care patients with rare diseases receive in resource-limited countries across Africa. Significant advancements and solutions are typically inhibited by a lack of coordination between medical professionals, patient groups, diagnostic laboratories, healthcare funders, and government decision-makers.

Goals

To address these challenges, the company formed a task force of rare disease clinicians and patient groups in Africa to identify communal issues surrounding rare disease diagnosis and treatment, and then attempt to find solutions. Objectives for the task force included:

  1. Allow clinicians and patient advocacy groups to collaborate with one another
  2. Collect information to develop models and support the evolution of services for the African rare disease community
  3. Gather input on diagnostic capabilities

Solution

  • 23

    Countries

  • 22

    Task force members

  • 27

    Questions

FYMCA used Within3 to hold a virtual session for the task force to work toward its goals. The Within3 platform met the company’s needs in the following ways:

  1. Enabled task force members across multiple time zones to contribute at their convenience over several weeks
  2. Did not require travel for task force members
  3. Allowed for the likelihood of intermittent or unreliable internet connectivity
  4. Provided auto-translation for non-English speaking advisors

All delegates previously attended FYMCA training events and engaged with the team on post-event platforms. Prior to the start of the 29-day virtual advisory board session, task force members received an email with a pre-recorded training video and slide deck that provided instruction on how to use the Within3 platform. This eliminated the need for a live webcast, which could be interrupted or compromised by sporadic network access. Other platform features that ensured the success of the virtual session included draft auto-save, which allowed advisors to continue commenting where they left off in the event of a loss of internet connectivity. Participants could also use translation capabilities to eliminate any language barriers and were able to engage in conversations by using the platform’s key engagement features such as replying and tagging.

Results

  • 90

    Transcript pages

  • 428

    Task force comments

  • 19

    Comments per advisor

During the over-time meeting, a high level of engagement from both moderators and task force members yielded a robust online discussion. Overall, participation was much higher than expected – although the company expected a participation rate of about 50%, actual participation was more than 80%. Of the task force members who participated in a post-session survey, 100% agreed that:

  1. They had adequate time to contribute
  2. Platform functionality was sufficient to communicate ideas
  3. They would participate in additional virtual advisory session

Due to the high quality of discussion produced during the session, the task force successfully outlined four specific topics to include in future sessions:

  1. Establishing a definition for rare diseases in Africa
  2. Development and adoption of specific rare disease treatment guidelines
  3. Gather information around genetics and diagnostics
  4. Determining registries and surveillance definitions

In addition to further discussion and development of topics raised during the meeting, the company will translate the task force findings into a peer-reviewed article for publication in a medical journal. Future plans include presenting the results to intergovernmental organizations, with the goal of continuous improvement of the diagnosis and treatment of rare diseases in Africa.