What If Communities Living With Rare Conditions held the Key to a More Humane Medicine? Report from the World Orphan Drug Congress 2025, Amsterdam.

The next scientific revolution will be led by patients’ wisdom and human dignity, and it won’t be televised.

Last week, at the World Orphan Drug Congress in Amsterdam, I had a profound experience. As I moved between conversations—wearing my different hats as a scientist, a regulatory executive, a patient living with Western-science-labeled chronic conditions, and a medicine decolonization writer questioning the very foundations of medical science—I felt a powerful, common thread.

It wasn’t just about developing new therapies. It was about the palpable, collective yearning for a new kind of relationship between medicine and the people it serves. I spoke with patient advocates, researchers, and regulators who were all, in their own ways, trying to think outside the box because the old one isn’t big enough to hold our humanity.

It left me with a clear and hopeful thought: The rare disease community is uniquely positioned to lead medicine’s most necessary evolution.

Why Rare Disease? The Power of the Multidimensional Perspective

In most of medicine, the system is designed for large populations. It seeks averages and common pathways. It can, often unintentionally, see patients as data points first.

But in the rare disease world—meaning the often-unchallenged medical reality created by medicine and statistics—that model breaks down. You cannot find an “average” in a community of dozens or hundreds. We are forced to see the person in all their unique, complex detail. This isn’t a limitation; it’s a superpower.

The rare disease experience naturally cultivates what I call a “multidimensional perspective.” It’s an understanding that:

• A treatment’s success isn’t just a lab value; it’s the ability to share a meal, to hug a loved one, to have a good day.
• The “burden of proof” doesn’t rest solely with the patient proving they are sick, but also with the system proving it is trustworthy.
• Data and dignity are not separate concerns. They are intertwined. An innovative treatment that comes with condescension, gaslighting, or bureaucratic cruelty is not a success.

This interpretation from the invaluable wisdom of people forced to live in this Western-science-made rare disease reality. Patients already know this. They live this truth every day. And this precise expertise is what the entire medical system is struggling to learn.

An Invitation to Claim a New Space: The Authority of Patient Dignity

So, I want to offer an invitation, inspired by the countless conversations in Amsterdam. What if we, the community of people living with Western-science-labeled rare diseases, consciously stepped into a new role, not as supplicants, but as the guiding authorities on Patient Dignity?

This isn’t about taking over labs or writing prescriptions. It’s about formally recognizing and codifying the wisdom patients already possess. It’s about building a framework that the rest of medicine can learn from.

Imagine if we collectively built:

• A “Dignity-First Charter” for clinical research, co-created by patients and families, that becomes the benchmark for any company wanting to collaborate with your community.
• A certification for “Dignity-Compliant” care that lets every patient and family know which clinicians and institutions truly see the whole human, not just the rare diagnosis.
• A new model for collaboration, where patient organizations don’t just advise but co-design research from the very first question, ensuring it serves life as it’s actually lived.

“Aren’t we already doing that?” scientists and regulators might ask.

No, we don’t. I’m not begging for patient wisdom to be assimilated in Western science, as it is now. I’m inviting them to build their own authority. Regulatory authorities are the authorities on science. Patient organizations are the authorities on patient dignity, and they have the right to have equivalent powers. They have the right to reorganize themselves to provide patient dignity, enforceable regulations, guidelines, advice, and certification. I’m talking about power dynamics, beyond today’s symbolic representation in science and regulation that can be rescinded with a mere breeze of political change, new pharmaceutical regulation, or changes in healthcare system standards. It’s about sustainability.

Patient organizations dealing with rare diseases are in a unique position to trailblaze this empowering reality:

• There is an urgent need to amplify statistically marginalized voices.
• Many patient advocates express the wish to share scarce resources to face a shared reality.
• The “rare” ecosystem already demonstrated how scientific and regulatory flexibility is a must to navigate this reality. Patient dignity authorities led by patients are in the most favorable position to reflect this necessary fluidity. Traditional regulatory authorities might even learn from them.

The Ripple Effect

By championing this in the rare disease space, this idea could create a blueprint for all of medicine. The solutions forged in the fires of rare disease—where compassion is not a nice-to-have but a necessity—are precisely what a healthcare system needs to heal itself when patients experience it as weary and distrustful.

The energy in Amsterdam made it clear: the will is there. The expertise is there. The moral authority is there.

The world doesn’t need patients to fit into its existing boxes. It needs creative people to show it what lies outside of them.

We all have a role to play. As a medical science decolonization writer, I help patient advocates and trailblazing scientists to unlearn limiting, harmful scientific narratives. I empower countless curious, dedicated people to think outside the box.

How do you want to contribute to this new reality? Where do you see the first, most powerful opportunity to embed this principle of dignified authority?