Rakiya A.Muhammad
Grounding hopefulness in evidence, grounding hype in data, and being fundamentally honest to the reading public plea is a plea from the rare disease community through science.
This was a key takeaway from the presentation by renowned physician and innovator, Professor James Levine at the 2025 National Press Foundation(NPF) Rare Diseases Reporting Fellowship.
Professor Levine, President of Fondation Ipsen, an international science foundation focused on rare disease innovation, addressed 62 journalist fellows from 29 countries across five continents.
“It’s too easy to say that there are new gene therapies, hope is on the horizon,” he pointed out.
“But can you imagine what that’s like for a family who’s been looking after a child, whether that’s in a wealthy country or a less wealthy country, and they read that there’s sort of hope where in fact hope with respect to an immediate cure does not exist.”
Accurate, well contextualised journalism, he said, is essential to scientific advancement: it is the is the “oxygen of science” that can breathe hope in the futures of people living
Uncommon Disorders: Communicating the Scale
Levine described Rare Diseases as the world’s largest invisible medical community, affecting 350 million lives across 7000 conditions -yet 95% remain untreated.
The word “rare” is almost a paradox in that it suggests these conditions must be uncommon, the healthcare innovator observed.
“But when you combine all these rare diseases together, you’re talking about an agency of more than 300 million people living with rare diseases, and you’re talking about the size of the United States,” he highlighted.
“And because of this misconception, you can understand why this minimizes the urgency in the political attention.”
He also pointed out: New therapies are on the way and they’re coming fast but because sometimes many of the rare diseases by definition affect only a few people, some people argue that there’s not enough of an incentive to develop therapies for them.
Levine counters: “If you look at the data, approximately $50 billion of business was done with mergers and acquisitions in the rare disease space in 2023.”
“There’s a lot of activity in this space and there are reasons for this. Some of it is related to the ability to deliver therapies, but also part of it is related to the spillover economics.”
He added: “So you can develop a therapy, for example, for an ultra-rare obesity condition, but the spillover can be into more commonplace disorders.”
“And so, there are significant opportunities. So because their disease is rare, that doesn’t mean people aren’t interested in finding cures. It’s just not true.”
From Victims to Visionaries: Breaking Pity Narrative
Levine called for a paradigm shift: change the story from one of victimhood to one of resilience and agency:: Break the pity narrative, from victims to visionaries.
“The community is not looking to be saved. They’re looking to be supported, the people are looking for diagnoses and people are looking for cures, but they’re not looking to be saved,” he said.
“There’s a very different communication between agency and tragedy. And the way the language is written in whichever language you are writing in is extremely important with respect to how members of the public who are reading your articles perceive the shapes of possibility.”
He spotlighted resilience through inspiring stories—like that of a Fondation Ipsen author living with a severe neurological disorder yet thriving as a journalist.
“She’s met three popes. She’s written 18 books, which are romance novels and has been in a wheelchair her whole life,” he stated.
“Can you imagine what it takes to do that? People with rare diseases are not looking to be pitied. They’re looking to be elevated. They show agency over adversity. They have so much to teach us.”
After more than three decades in medicine, Levine has often witnessed families eager to share their viewpoints.
“The people living with rare diseases are more than willing to share not only their own data or even the data of their children, but even genetic data because the solutions come from sharing this data,” he observed.
“As journalists, you’re going to find a remarkable willingness to share lifetime stories and personal stories as it ultimately is going to raise awareness of the actual condition and accelerate cure.”
Language of Hope: Focusing on Progress
He encouraged journalists to honour each step forward, fostering an optimistic spirit that shines a light on advancement.
“Focus on progress—not perfection,” he said, stressing the ‘grammar of hope’.
He highlighted a shift in language and sees journalists as guides, helping society embrace this fresh outlook.
“Medicine is becoming creative engineering; the lexicon shifts from cure to construction, patient to partner,” he stated.
“Journalists can teach society this new grammar. Every story you tell expands the public vocabulary of progress.”
To guide storytelling, Levine introduced his DACMAR framework—six vital questions:
Disruption: What’s changing?
Adoption: Who’s using it?
Collaboration: Who’s partnering?
Management: How is it governed?
Adaptability: Can it scale?
Resources: What’s the cost?
He shared invaluable tips for interviews:
For scientists, ask: “What keeps you awake at night?”—uncovering doubts, fears, triumphs, and the emotional core behind discoveries. “Ask about failures, not just successes; explore the personal cost of persistence; find the moment of uncertainty before breakthrough; connect the science to the scientist’s story.”
For patients, ask: “What do you want the world to know?”—empowering them to tell their stories on their terms. Move from extraction to collaboration, let patients co-author their story, he added.
The researcher emphasised the journalist’s role in weaving together three distinct voices: The Scientist, representing rigorous research and clinical expertise; the Parent, offering lived experience and advocacy; and the Algorithm, providing data analysis and pattern recognition.
“Stay inspired, stay honest,” Levine advised. “Exaggeration erodes credibility. Your reporting must be rooted in evidence—realistic, transparent, and hopeful.”
