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 patients, advocates, caregivers, healthcare professionals, treatment industry leaders, and beyond. We share our thinking with the community here and via email. To be added to our email list and get future resources directly in your inbox, please get in touch.
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…
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…
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.
In recent years, there has been a push towards expanding the role of patients, advocates, and caregivers (also referred to as patient and public involvement in medical literature; “patient” includes advocates and caregivers in this context) in medical research and publications on ethical, normative, and substantive grounds.
At the end of 2019, we published an article about the paucity of publications in rare diseases. But rather than stop there, we decided to do something about it. We combined our expertise in building immersive digital experiences, desire to communicate more effectively in rare, and our publications prowess, to develop rareJourney. How does rareJourney…
Patients, advocates, and caregivers are the market-shapers in the world of rare disease. A powerful rare disease community can improve drug development and commercialization, influence regulatory decisions, shape treatment paradigms, and educate others about the disease. What’s more, survey data show that a significant majority of patients would provide feedback to drug developers, and would choose treatments from companies that are engaged with patients over those that are not.
Because we are immersed in rare diseases, both professionally and personally, our team understands the intricacies of rare. Patient finding and community building are cornerstones of rare disease problem solving as they directly benefit individuals living with a rare disease and the rare community at large, while enabling improvements in patients’ experience and care.
Peer-reviewed medical publications remain a mainstay of trusted communication of disease and treatment information. They influence everything from prescribing options to treatment guidelines and payer decisions. They are even more essential in rare diseases as PACs seek to educate themselves and often their physicians.