The Fighting for Kaiden Foundation and rareLife solutions have partnered to bring oneSMAvoice, a new online community and disease knowledge platform, to the spinal muscular atrophy (SMA) community.
Following a successful 2021, the Digital Experiences team at rareLife solutions marked two important milestones in February, including the rollout of an all-new onevoice community and the visual redesign of one of our longest running and largest onevoice sites. The newest member of the onevoice family (and the first of several launching over the course…
Our newest onevoice community, oneSMAvoice empowers people impacted by spinal muscular atrophy.
While there is a growing recognition that the world of rare disease is different and comes with unique challenges, the body of knowledge around how to address those challenges is very much a work in progress. At rareLife solutions, we’re helping to build that knowledge base by sharing what we’ve learned from our work with…
The rareLife Solutions Engagement team was honored to have their work selected as one of the award-winning abstracts at the 49th Annual SCDAA National Convention. Reintroducing Hydroxyurea as an Effective Treatment for Kids and Teens is a community-based educational campaign designed to raise awareness of hydroxyurea usage for children and teens through the use of…
The annual National Organization for Rare Disorders Summit is one of the largest multi-stakeholder events in rare disease. It includes communities ranging from from patient advocacy groups and industry, to government and academia, to discuss topics related to rare diseases and orphan products. The rareLife solutions team was selected to include several posters at this…
There is a growing recognition that the world of rare disease is different and comes with unique challenges, but the body of knowledge around how to address those challenges is very much a work in progress. At rareLife solutions, we’re helping to build that knowledge base by sharing what we’ve learned from our work with…
https://www.rarelifesolutions.com/wp-content/uploads/2021/08/onevoice-explainer-v2-pro-narration-1080p-210822-1.mp4
Currently, all trials, including those in rare diseases, that do not demonstrate a statistically significant benefit (i.e., P < 0.05) on the primary endpoint are classified as “negative”. This classification does not take into account a myriad of factors, including whether the trial was sufficiently powered, or had other statistically significant endpoints, or was terminated before completion, or was even initiated. This paper proposes reclassification of these trials into 5 categories: true negative, underpowered, inadequate, terminated or uninitiated, and valid. These categories reflect the trial characteristics more accurately and will be more useful to all stakeholders, especially the patients who participated in the trial and their healthcare providers.
Individual privacy has come under increasing scrutiny since the passage of the General Data Protection Regulation. While patient privacy is crucial in all publications, the challenge is particularly severe in rare diseases where, by definition, there are very few patients, thereby increasing the risk of identification.
We searched PubMed and Google for articles on patient privacy in rare diseases. Search terms included “rare” OR “orphan” AND “disease” AND “privacy” and were restricted to those published in English. Results: Two authors (PP, MN) independently evaluated the results for relevance to the topic, and any disagreements were resolved through discussion with all authors. The selected documents were analyzed further. We identified 5 high-risk situations in which the privacy of patients with rare diseases can be breached.
