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.

Our Latest Posts

NORD Rare Summit: rareLife posters

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…

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“Negative” Clinical Trials in Rare Diseases and Beyond: Reclassification and Potential Solutions

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.

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Privacy Considerations in Medical Publications – A Rare Perspective

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.

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What makes a rare disease community powerful?

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.

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