Turning the tide for rare from the inside: the first 100
Estimated reading time: 6 minutes
Nicola Miller, editor-in-chief at RARE Revolution, reflects on emerging themes as we mark the milestone of our first 100 articles published on RARE Revolution insider®
At the heart of the conception of RARE Revolution insider® was the desire to take our extensive learning and expertise from the development of RARE Revolution Magazine® (RRM) and craft it into a tailored platform to cater to the content needs of our industry audience. Rather than detract from the voices of lived experience on RRM, RARE Revolution insider® (RRi) allows us to bridge these worlds and share insights, thought leadership and breakthrough innovation to our niche professional audience.
This month, as we come to the end of our first full quarter since launch, we are proud to mark a meaningful milestone—100 pieces of quality content published. This rich tapestry of articles brings together voices from across the global rare ecosystem: where scientists, academics, advocates, industry leaders and patients are united by a shared mission. A mission to transform the lives of those affected by rare diseases and perhaps most importantly, to share that mission-driven progress for the betterment of all—turning the tide for rare disease—together.
In our first 100 articles some key themes emerged from this collection:
Rethinking pharmacovigilance: safety beyond the status quo
As we would expect, one of the most pressing areas of focus and challenge in rare disease treatment development and innovation is ensuring drug safety, particularly in populations too small for traditional monitoring systems. Several articles explore how pharmacovigilance—the practice of preventing, detecting and reporting adverse drug effects—is being reimagined.
Innovators are turning to real-world data (RWD), artificial intelligence (Ai) and patient-reported outcomes (PROs) to fill the gaps left by conventional methods. These tools are not only enhancing safety but are empowering patients to play a more active role in their treatment’s development journey.
Innovation in clinical trials: smarter, faster, fairer
Rare disease trials face unique hurdles as addressed in our first 100. Small patient pools, ethical concerns and the urgent need for effective treatments are barriers to be addressed in the pursuit of progress. Insights shared in RRi showcase how adaptive trial designs, biomarker-driven studies and digital endpoints (data collected via digital health technology, e.g. wearables and smart applications) are revolutionising the way new treatments are tested.
One standout theme is the co-design of clinical trials, ensuring research reflects real-world need and values. And importantly there is now a growing ground swell of appreciation in the immense value of bringing caregivers insights into this design process. This shift toward patient and caregiver-centricity is not just ethical—it’s essential for meaningful progress.
Global empowerment: science without borders
From Latin America to indigenous communities, our first 100 highlights efforts to decentralise scientific leadership and build capacity in underrepresented regions. Insights from advocates and industry alike underscore the importance of local expertise, cultural sensitivity and community-driven research in tackling rare disease as a global issue.
Amplification of all voices reminds us that innovation thrives when everyone has a seat at the table.
Women in RARE: the women leading the way
Our first 100 collection includes 10 Women in RARE features, shining a much-needed spotlight on female leaders who are breaking down barriers in genomics, biotech and advocacy. These profiles are more than just inspirational for future generations of women in rare—they are a call to action for greater gender equity in science and healthcare leadership.
A common thread weaves through this collection: resilience, vision and an unwavering commitment to improving the lives of others.
Power to patient voices: from subjects to stakeholders
As a platform designed to bridge the worlds of advocacy, community and industry our first 100 collection, of course, includes the elevation of patient, caregiver and advocate perspectives. Whether it’s shaping clinical trials protocols or redefining success metrics, patients and their advocates are no longer passive participants—they are co-creators.
Our surveys and interviews reveal a strong desire for transparency, trust and ongoing dialogue between researchers and the rare communities they strive to support. This cultural shift of bringing patients and researchers together is paving the way for more ethical, effective and inclusive research.
Ethics and equity in research
Challenging perceptions and ideas are critical in the role of RRi, and our first 100 does not shy away from the tough questions. Thought leaders explore with us the ethical dilemmas of randomised controlled trails in ultra-rare conditions where withholding treatments can have stark consequences. Others examine how biomarkers can personalise care while raising concerns about access and equity.
We also delve into the, at times, dark history of medical research on minority communities and the very real need for greater endeavours by all in building trust within these communities, along with the younger generation of healthcare consumers. Discussions reflect a growing awareness that scientific rigor must be balanced, unbiased and led by compassion and justice.
The human side of leadership: behind the scenes
Our incognito series offers a rare glimpse into the behind the scenes lives of rare disease leaders—CEOs, trustees, communicators and more. Their candid insights reveal the real-life challenges they face when working in this unique space and reveal both the emotional toll and daily triumphs that come hand-in-hand with working in a career where a deep sense of purpose drives the work.
It’s a reminder that behind every policy or breakthrough is a person with a story to tell, their own ‘why’ and that with the safety of anonymity they can share the insights that often as professionals we dare not share!
Looking ahead: to the next 100
As we look ahead it is hard to say what the focus of our next 100 articles will be. In many ways the whole rare ecosystem is shifting and in what direction we don’t know. But what we do know is that success for this community will be incremental and painstaking and it is our collective responsibility to keep informed on not just the successes and challenges of today, but the direction of travel for tomorrow. Our aspiration is that RARE Revolution insider® will become one of your valued and trusted experts along that journey, keeping you informed, connected and dialled in to all things RARE. Here’s to the next 100.
If you would like to share your insights and/or submit and incognito contribution we would love to hear from you. Please contact Nicola Miller viaeditor@rarerevolutionmagazine.com
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