Connecting Biotech’s Promise with Real Progress in the Rare Disease Realm

Contextual reporting about biotechnological advances can improve the futures of people living with rare diseases As a leading voice in the rare disease community, James Levine of Fondation Ipsen wants international journalists to amplify the impact of scientific innovation and fuel hope for people living with rare diseases and their caregivers. by Rachel Jones, National Press Foundation When an infant named K.J. Muldoon received a successful gene-editing therapy performed by Children’s Hospital of Philadelphia researchers earlier this year, media coverage of that biotechnological landmark flooded the zone of health and science reporting. It also helped raise the profile of rare genetic diseases that affect tens of millions of people around the world. Baby K.J.’s miraculous story, paired with the 2019 CRISPR-based bone marrow treatment that ended Victoria Gray’s lifetime struggle with sickle cell disease, is a powerful example of the promise that scientific innovation holds for people living with a rare disease. Journalists need to fully grasp the implications of these developments while also amplifying the persistent gaps in access and equity that block these innovations from reaching the vast majority of patients. That was the central message shared by Dr. James Levine, president of the Paris-based non-profit Fondation Ipsen on Nov. 17. Levine launched the 2025 NPF Rare Diseases Reporting Fellowship with a potent call to arms about amplifying technology’s role in reshaping the rare disease ecosystem. Levine told the 62 journalist fellows from five continents and 29 countries that they have the power to bridge the gap between the promise of biotech and meaningful progress for people living with rare disease. “It’s too easy to say that there are new gene therapies, hope is on the horizon. 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,” Levine said. “So 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 the science.” In fact, Levine believes that accurate contextual journalism is the “oxygen of science” that can breathe hope in the futures of people living with rare disease. Here are some of the highlights of his conversation: The heart of the rare disease story is not about pity It can be tempting to lean into the most complex, dramatic elements of a particular rare disease patient’s journey, Levine said. Resist that temptation. “The community is not looking to be saved. They’re looking to be supported, that people are looking for diagnoses and people are looking for cures, but they’re not looking to be saved. There’s a very different communication between agency and tragedy,” Levine said. “Please be the protagonist and not allow our patients to be footnotes. 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.” One of the authors published by Fondation Ipsen offers a perfect example. She lives with a severe neurological disease, Levine says, but “she’s a working journalist. She’s met three popes. She’s written 18 books, which are romance novels and has been in a wheelchair her whole life. Can you imagine what it takes to do that? People with rare diseases are not looking to be pet. They’re looking to be elevated. They show agency over adversity. They have so much to teach us.” Communicating the scale of rare disease is important Levine advised that journalists must always remember that the word “rare” is almost a paradox, in that it suggests these conditions must be uncommon. “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. And because of this misconception, you can understand why this minimizes the urgency in the political attention.” We are living in a ‘golden era of hope’ for people living with rare disease New therapies are on the way and they’re coming fast, Levine said. 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 disputes this. Speaker: James Levine, President, Fondation Ipsen Summary, transcript and resources: https://nationalpress.org/topic/connecting-biotechs-promise-with-real-progress-in-the-rare-disease-realm/ This training is sponsored by Fondation Ipsen. This video was produced within the Evelyn Y. Davis studios. NPF is solely responsible for the content.