How Has COVID-19 Impacted Patients with Rare Conditions?

How Has COVID-19 Impacted Patients with Rare Conditions? By Judy Chandler Over 600,000 Inspire members are affected by rare conditions. As a vital partner to 40 rare disease non-profit advocacy organizations, Inspire’s rare disease patient and caregiver members represent over 3,000 rare conditions. Serving as a common point across these diverse conditions, Inspire was uniquely positioned to learn how rare disease communities were being affected by COVID-19.   First, we noticed a surge of COVID-19 posts across all of Inspire through the month of March. Inspire’s research team began by reviewing some of these member communications to identify [...]

Patients Are The Best Resource For Defining Study Endpoints

Patients Are The Best Resource For Defining Study Endpoints By Robert Gardner In September, the World Orphan Drug Congress USA Fall Webinar series opened with a program called: “Understanding the Novel Challenges in Rare Disease Development: Perspectives From Experts.” They took an audience poll, asking attendees to identify their “biggest challenge in rare disease studies” from five choices, including competition for patients; lack of understanding of disease progression; development of appropriate endpoints; lack of data; and global regulatory pathways. Almost half the respondents -- 46% -- chose “development of appropriate endpoints” as the biggest challenge in rare [...]

Next Generation Online Health Communities

Next Generation Online Health Communities: Data empowering patients and caregivers improves care and accelerates research By Richard Tsai The World Orphan Drug Congress USA 2020 (WODC 2020) provided four days of powerful panels and keynotes about collaborations, successes and failures in creating what rare disease patients and families need and desire most – effective treatments for their conditions. In one failure turned to success, Kim Stephens shared her story involving inappropriate clinical trial endpoints. Stephen’s son has Hunter Syndrome, an extremely rare inherited condition where children lack an essential enzyme in their cells that eliminates waste . [...]

Accelerating Rare Disease Research

Accelerating Rare Disease Research By Richard Tsai COVID-19 has challenged the non-profit arena’s fundraising arm, which is largely based on face-to-face events like galas, runs and walk-a-thons. This limit on the number and amount of donations has occurred just as rare disease patient organizations have reached a tipping point in their influence on government and industry. According to Malcolm Gladwell, the “tipping point” is “the moment of critical mass, the threshold, the boiling point,” where “ideas...spread like viruses do.” 1In this case, the tipping point refers to the efforts made by rare disease patient organizations to provide [...]