James Levine Transcript — Nov. 17, 2025

Rachel Jones/NPF (00:00):

Welcome to the 2025 National Press Foundation, rare Diseases Reporting Fellowship program. We’re pleased to welcome 62 journalists from five continents and 29 countries for this journalism training. They’ll spend this week immersed in the research and advocacy challenges faced by the more than 300 million people around the world who live with rare diseases. All of these briefings have been made possible by our sponsor Foundation Ipsen, the Paris based nonprofit foundation, which focuses on increasing knowledge and awareness of rare diseases. Speaking of re-imagining the future, we are extremely privileged to begin our training by hearing from Foundation Ibsen’s President James Levine. He’ll set the tone for our week of briefings by asking us to embrace our opportunity to ignite our understanding about the science that can yield a better future for people living with rare disease. James, thank you so much for joining us today.

James Levine/Fondation Ipsen (01:08):

Absolutely a pleasure. Thank you, Rachel, and thank you the whole National Press Foundation, and most importantly, thank all of you here with us today. This is such a critical issue, unmet need in the medical world, and I really appreciate you spending your time and commitment to join us. May I please, Rachel share a few slides with everybody?

Rachel Jones/NPF (01:30):

Yes, you may.

James Levine/Fondation Ipsen (01:36):

Can everybody see what we can see?

Sydney Clark/NPF (01:41):

Yes, it looks great.

James Levine/Fondation Ipsen (01:44):

Thank you all so much again, and what I’d like to do today is give you a little bit of a data-driven background on what we know about rare disease communication from the rare disease ecosystem, and then share with you some common myths about rare disease and then some significant opportunities with respect to respect to improving awareness and your capacity within the rare disease community. So thank you for your time and off we go. Well, as you’re going to hear again and again, this is one of the most substantial international unmet needs in the world. With 350 people living with rare diseases, the vast majority without a treatment, 7,000 conditions, this is a massive unmet medical need. Our foundation, Ipsen, our focuses are shown here to support education, advocacy, and cure. We’re a non-for-profit based in France, but with a wide global reach shown here. Our publishing house has reached just over a million books in circulation.

(02:57):

Our digital reach without counting this meeting direct is about 11 million people and you can see our global presence across 240 organizations. We’re a science-based organization, which is critical to the way we approach the rare disease space, and we work with several hundred independent experts in the rare disease space, and I should tell you all of whom function without any form of fiscal compensation. And where we start is here, we wanted to understand specifically from the rare disease community what are their communications needs. And we contacted 45. There are over a thousand rare disease organizations. Please recall there are 7,000 rare diseases. And we contacted and worked with 45 rare disease associations and used a scientific approach to understand exactly what are the communication needs and pitfalls that have occurred with respect to communications in rare disease. And I’d like to please share a few of these reflections with you.

(04:01):

Now in order to disseminate this. The first call from the community was that there is no communications approach in rare diseases. And so a book was produced by the foundation and it’s available for free at this website. The first thing that you are going to hear again and again and again is the invisibility of people living with rare diseases. Literally millions of people live without diagnosis. Hundreds of hundreds of millions of people have rare diseases, most have no cure, but the diagnostic delay can be five to seven years. So many children and adults are living their lives without actually knowing what’s wrong with their loved one. And so this is sort of fodder for headlines if you like, in the sense that as these stories become unveiled, these are massive, not only scientific discoveries but human stories. And our sense is that the journalistic capacity that you have is the oxygen we need in the rare disease community. The oxygen of science is that so many of these stories as you can appreciate with the new gene therapies, the new approaches to scientific discovery get suffocated in jargon. And your mission from our perspective with a massive thank you is to bridge two cultures. The culture of the society that needs to hear these words and the scientific community that are often unable to express them.

(05:40):

The technology is moving very, very fast, but the public capacity to understand the technology is lagging. And at a significant part of the course that you’re about to endure and huge thanks to Rachel and her team is to explain to you, to explain to the public quite what these new technologies are and how they impact people. I mean, for example, we can sequence the genome overnight, but it takes months to actually tell the story. There’s a new language of health arising. All of you, or many of you have heard about CRISPR technologies, asos, which are antisense, oligo nucleotides, new types of gene therapy. The lexicon is shifting very, very fast and our ability to share this technology with the public is getting more and more difficult.

(06:36):

So what is I think important to appreciate? Again, what we learned from the rare disease community is that data convinces not only those in society who do not understand the struggle of people living with rare diseases, but policymakers and importantly funders. And so it is the link between the heart and the head that the rare disease community needs. There’s a lot of data out there, but the ability to communicate these human stories is exceptionally difficult. Therefore, please not only write with precision but write with heart. And what you’ll find for those of you who have not reported in this space before, is that so many of these stories are heartrending, both in terms of the struggle faced by families, caregivers, children with rare diseases and adults with rare disease, but also the struggle to not only achieve cures, which often as you know don’t exist, but even the care necessary to carry people through their day.

(07:37):

And it’s this link between scientists, parent and algorithm that is so challenging. We live in a world where even the concept of how algorithms drive scientific discovery, connect parents to scientists is an extremely complex space. And over the next several days, we’ll all gain a greater appreciation of how those connections occur. But I can imagine, I’m not a journalist, I’m very jealous of all of you who are, but the ability to be able to write in these spaces and express the complexity of these issues in a humanistic way I think is exceptionally important at the moment. And remembering the size of the challenge, literally 360 million people who need your help, I beg your indulgence to appreciate some of the complexity, but then transmit it into a human voice.

(08:34):

One of the great problems arises of course, in a space where you can imagine writing a story, and there are many, I’m afraid of parents who are raising children with these sort of quite significant issues regarding physiology, pathology, and psychology is that then of course there’s a great hyper associated with whether it’s a genetic therapy and then false hope occurs. And there’s a sort of an equation we think of in the red disease community, and this came out of the needs assessment is there’s almost an equation between public curiosity, skepticism and credibility. And the general request from the rare disease associations is a sort of a commitment to transparent honesty. 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. 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.

(09:56):

One of the common faults made in communicating rare diseases into the public space is the focus on pity. And all of you will be aware of this issue that it is too easy to relay pity, whereas that is in fact not what is wanted by people living with rare diseases. In fact, quite the opposite. People with rare diseases, 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. Please be 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. And just to give you a sense of how critically important what you’re doing on this course is from the last course led by Rachel and her team, the total global impact was 235.9 million people. That was the number of people who read all of the,

Rachel Jones/NPF (11:21):

Hello? Can we remember to keep our audio muted please? Okay, sorry about that James.

James Levine/Fondation Ipsen (11:33):

No problem at all. But it’s a lovely moment just to reflect. I dunno if all of you heard that the total global impact of this program last year was 235.9 million people, the stories you’re about to write are actually exceptionally important in this space and for the worldwide medical community. So from the rare disease community, from the communications assessment we did scientifically across the community, there were certain aspects of the scientist interview that sort of came up again and again. In other words from the perspective of the community is how do you make science vibrant and exciting? All of this information will be known to you, I suspect, but what again comes is that scientists are not sort of locked in their laboratories, sort of boringly typing in data. Being a scientist, I can tell you I’ve been a bench scientist for 25 years, is very passionate, very exciting, full of failures, full of crying, I can tell you full of joy when you make massive discoveries.

(12:43):

And conveying the beauty and the passion of the obsession of scientists is extremely important and very awakening and is a true bastion of hope for the community in the sense that you have literally, on one hand you have a huge amount of suffering in the world, but on the other hand you have a community, the scientific community battling night and day late into the night and long into the day to find cures. Science is a beautiful, passionate, but very, very hard job. On the other hand, the patient interview is sort of the contrary to this. As I mentioned before, one of the big complaints that comes out of the rare disease community is that people living with rare diseases are not victims should not be pitied. They have the ability, I just had somebody living with autism to write a book. Can you imagine what it takes to overcome all these barriers?

(13:40):

Another author we work with has a severe neurological disease and she’s a working journalist actually 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. And what we learned so much is not only the dignity, but also the capacity of not only people living with their diseases but their care caregivers to collaborate. And so what we start to sense as a grammar of hope, I mean here you see four pictures and I’ve just very microscopically tell you about the four people. You can see the person standing being photographed is clawed. He has a rare disease. He just for his 55th birthday, ran through France 55 kilometers accompanied by communities simply as his birthday present to himself.

(14:43):

But he was joined by an entire community. The gentleman in the middle holding his book is Maxine. He wrote a book, he has autism and he wrote a book about autism with social media support. This reached 1.3 million people. The little boy in the top right is a boy called George, and the insert is his dad called Jonathan. Jonathan started up a biotech company to support George and they’ve just sort of signed a biotech deal. On the bottom right is a fear. She’s reading a book, she’s reading a magazine. We produce actually about understanding rare diseases in Africa. And she, she’s holding her little issue magazine in Cape Town, South Africa. She lives in a township and the magazine she’s holding is the only school book, the only paper document she owns with respect to her education. And this magazine reaches 300,000 children living in townships. There’s a lot of stories of hope.

(15:49):

And what we’re able to do, again using the scientific approach is to provide six sort of constant questions as to how an innovative story can actually be impactful. And these are on the slide deck. It’s a free book, but it gives you a framework in which to address a journalistic question as to whether the issue you’re discussing is disruptive in terms of therapy. Are the resources available? Can it be scaled? In other words, this is just a cure for one people. Can this be adapted into middle and low income countries? How is the innovation being managed? What’s the collaborative environment and who’s actually going to benefit from the innovation? So the only point is that as you’re structuring your stories, there are frameworks in the innovative space in rare diseases around which to base them. So those are the reflections from the rare disease community.

(16:51):

And what I’d like to do next is talk about two things. First of all, 10 common myths about rare disease and then 10 key opportunities in the rare disease space. And I’ll talk through them. The slide deck will be available to you, so I’m going to sketch through these, but they are quite consistent myths and opportunities. So here are the 10 myths. The first one is, I’m sure you’re going to hear this again and again and again, is the idea that the word rare is of course a sort of almost a paradox that of course something’s rare is and therefore 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.

(17:44):

So it’s actually a key issue. Scale shapes strategy. The second myth about rare diseases is that if a rare disease is genetic, and in fact most are nothing can be done. In other words, if somebody is born with a rare disease, can they actually be treated? And we live in a new world where we’re even talking about therapies now where a single person with a genetic disorder can be treated, in other words, a completely bespoke genetic therapy. In addition, not only are some diseases not treatable, 94% of people living with rare diseases have no treatment as of today. But there are multitude of supportive therapies that can be provided for people living with rare diseases. And it’s important because when a patient comes to a doctor, when a patient’s interviewed, it is critical to appreciate that although a disease may not exist today, there may be one coming tomorrow.

(18:38):

But in addition to that, multiple types of therapy are available and important for people living with rare diseases. A person living with rare diseases should never be told that there is no hope. Number three, rare diseases always appear at birth. That is not true. A LS Huntington’s, and there are many metabolic disorders that appear later in life, and sometimes symptoms can be triggered by environment or hormonal shifts such as menopause and misdiagnosis can occur because rare diseases are often misconstrued as only being occurring from early childhood onwards. And this awareness is important as there are lifespan issues. This is another great myth. Often when people think about rare diseases, they think about people, particularly children. We’ve already discussed that myth, but living in wheelchairs all very disfigured and so on and so forth, that’s simply not true. Many rare diseases are in fact invisible. You can think of certain metabolic diseases, you can think about certain psychological issues that are simply unaware.

(19:43):

One very interesting example that we actually discussed last year is the issue of sometimes men, aia, which is a significant bleeding during menstruation, can in fact be associated with rare bleeding disorders. And so a lot of rare diseases are often missed completely because of lack of obvious physical symptoms. Another myth is that there are no treatments for rare diseases. You even heard me twice mention that for 94% of people there are no therapies. And that’s true. However, that does not exclude that there aren’t other therapies in the marketplace. So in fact, first of all, there are large global pipelines being developed across biotech and biopharma. Second of all, there are many therapies coming online. And importantly, a theme you’re going to hear again and again is that there are platform technologies. Those platform technologies for example, include gene therapies and that those platforms can be used for multiple rare diseases.

(20:41):

And so the landscape is changing, fast therapies are coming. Whilst it is true, many therapies do not exist today. We are in the golden era of hope for people living with rare diseases. New therapies are on the way and they’re coming fast. Another myth is that because sometimes many of the rare diseases by definition have few people who are living with these rare diseases, you could argue that there’s no economic equation to make it worthwhile developing therapies that is simply not true. If you look at the deal data, approximately $50 billion of business, if you like, was done with m and a mergers and acquisitions in the rare disease space in 2023. There’s a lot of activity in this space and there are reasons it for this. Some of it is related to the ability to deliver therapies, but also part of it is related to the spillover economics. 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.

(21:56):

People with the same rare diseases are all alike. Again, that’s another myth. So somebody has one type of Duchenne’s muscular dystrophy and is very disabled. Another person may have Duchenne and actually appeared to be completely normal. And what’s important is to appreciate that. Of course, that’s true of many diseases. You think of type two diabetes. Some people have almost no sequela type two diabetes. Other people are battling blindness. And so there’s huge variability equally so in the rare diseases as in common diseases, individuality is the key. This is a critical issue that the eighth myth is that once rare disease care ends, sorry, rare disease care ends once a diagnosis is given, it’s quite the opposite. You’ll find in stories that in patients you meet that the diagnostic odyssey can take sometimes up to eight years. And often it’s almost a sort of a moment of relief, almost a moment of celebration when a diagnosis is made.

(22:58):

But that’s the problem actually in reality, the challenge starts with the diagnosis. So once one has a diagnosis, then an entire package of care is necessary to carry the person across the finish line to give people a full life that every individual deserves. The other. The next myth, and again you’ve heard me fall into this caveat already, is that we’re beholden to technology to automatically solve all rare diseases. Now, that’s important because some of the stories are going to come across is are things like drug repurposing where drug has already been discovered for another purpose, but can be redeployed into a rare disease. So that type of thinking, in other words, we don’t need technology to solve every one of these 7,000 rare diseases. Some of the solutions are already out there, but they need to be repurposed. Last patient organizations that are going to be an extremely important part of this storytelling have limited influence that is in rare diseases is particularly important, that rare disease associations have disproportionate influence than you would anticipate.

(24:11):

For example, knowing about cardiovascular disease and so on and so forth. So for example, at the FDA, at the EMA and at the MHRA, these are the regulatory agencies for medicines in rare diseases in the regulators, the FDA is a big example. It is expected that members of rare disease associations actually come to the floor and express the need for the new therapy. So rare disease associations are paramount in advocacy, but even in therapeutic design and in raising money to build new therapies, rare disease associated are particularly important. As you’re writing stories connecting to the rare disease Association, they will be thrilled to provide you with information and further links and so on and so forth. So those are the 10 links. So what are the levers for hope? What are the 10 levers for hope that we can apply? And you will be inspired for sure by other speakers, but let me at least share with you 10 things that I have seen commonly in this community.

(25:16):

The Diagnostic Odyssey, this issue comes up again and again and again. It can take up to a decade to receive a diagnosis and some people never get one. Rachel is fully aware that my stepmom has a rare disease, undiagnosed, she was a English lecturer at uc, Berkeley and lost her speech, lost her ability to walk, lost her bodily control. And Rachel, unfortunately, she died 10 days ago. She never got a diagnosis because there are fragmented pathways across specialists, across testing. And you think about the countries you are writing from this degree of fragmentation reaching a specialist that the city visit is called in Ghana, where you go to actually meet the specialists for the first time. That in of itself is a huge challenge. So the delays in treatment, whether that’s through genetic testing, through the ability to access specialists, simply through lack of medical training, is a critical, consistent theme of stories that you’re going to come across.

(26:26):

The therapeutic desert. I’ve already discussed most people with rare diseases don’t actually have a therapy, and this is because of a variety of factors. But therefore, you can understand and appreciate that when you come across stories where therapies are emerging, and this is, as I said, happening almost every day. Now, how exciting and thrilling this is. However, the caveat is please don’t over express. There’s hype versus reality. So it’s the emphasis is on the reality because those stories are also going to be read by families of other people living with the same rare disease diseases. Fragmentation in healthcare pathways is an extremely part of the solution pack that you can help deliver into the world and the fragmentation in rare diseases, and think about it for a second, some rare diseases may only have one person, some may have 50 people, but those 50 people are living all across the world.

(27:26):

And so you can imagine that the only expert happens to be in Germany actually had dealt with this. About three or four weeks ago, I was contacted by somebody from the states saying, could I connect the family to the world expert who happens to be in Germany? It was easy to do, no problem. But you can imagine across the world that the entire healthcare system with respect to rare diseases is fragmented. And this complexity results in suffering. And so as you come across your stories, you’re going to find extraordinary success stories of people who’ve overcome this degree of fragmentation to find solace, to find diagnosis and treatment inequity and access is a huge issue across the world, and you can immediately appreciate that whole genome sequencing, whether that’s in France, the United States, the UK is simply not accessible in many middle and low income countries, let alone the cost of therapies that can run to hundreds of thousands of dollars.

(28:31):

However, these do not represent end game stories. There are so many organizations around the world making first of all, whole genome sequencing. Exome sequencing is becoming less and less expensive. The facilities that brode in Boston are shared with in countries in Sub-Saharan Africa, there are lots of methods for improving access to diagnosis. All the pharma companies that I’m aware of have programs to deliver in some shape or form their therapies to people who can less afford them and so on and so on and so forth. But it’s something you should be aware of. The fifth issue is the rare disease data paradox. You’re going to find this again and again. On one hand, we’re all aware of the importance of preserving protection with respect to our own medical data. We’re all aware of this in critical issue, but what you’re going to find in the rare disease community is something quite different.

(29:35):

And I’ve been in medical practice for over 30 years, I’ve never seen it before, but you’ll find again and again, 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 these datas in this data internationally. 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. One of the most disheartening stories, and obviously I’m aware of this from my dad’s perspective, he looked after he looked after my stepmom for six years with this ongoing condition, it’s emotionally, physically, financially devastating for caregivers. And you’re going to hear this story around the world. We produced data on the financial burden. We just conducted studies on the financial burden of caregiving.

(30:40):

There’s gender bias. 80% of caregiving is often by women, is provided by women. People give up their careers, people relocate and so on and so forth. But caregiving is one of the most heartrending stories imaginable. Clinical research in rare diseases, you can immediately imagine that if you’re going to recruit for a trial in type two diabetes, affecting what, six to seven and a half percent of the population, it doesn’t take that long. But if you are now trying to recruit for a trial where there only 50 people, even with rare disease, it can take years to find the right number of patients. And so you’re going to find this sort of scientific bottleneck. So when you hear the success stories, all of these types of stories about how difficult it is to conduct the science in rare diseases will come to the fall.

(31:31):

The silence problem, invisibility creates silence. And we’re all aware of this. And one of the big problems, and we’ve seen this from articles from journalists who went through the program last year and the year before, is this idea of stigma. It’s one thing to sort of celebrate the success of a successful entrepreneur, a successful businesswoman, the successful writer I told you about. And these stories are astonishing. But to then hear the stories of stigma that people living with rare diseases experience at work, imagine just going out for a drink after work and everybody goes, but the person in the wheelchair is sort of left behind that sort of thinking. But that constant stigma that people with rare disease experiences is not only reportable, but again, it just opens your heart. It just opens your heart. And at a broader level, if you think about it at a societal level, if we think this way as individuals, imagine that we think this way as societies. This is why people with rare diseases are underrepresented, not only just in terms of their medical needs, but in terms of funding awareness policy. So often, politicians, individuals, ourselves, look away when we should be looking forwards.

(32:55):

One of the completely misunderstood areas in rare diseases that people living with rare diseases who are surviving go through all the stages of life and go through all the challenges of life, whether that’s employment, that can include sexuality, that includes children, relationships, divorce, and all the way through all the social parts of life, but also the medical parts of life. And aging with rare disease is one of the things that people is very, very rarely discussed at all. And therefore, caregiving with rare disease. There’s just the most heartbreaking story that was reported on where a mom living and she reported this publicly on a webinar, a mom living who was looking after her daughter with rare disease was driving one day in New York City and it crossed her mind to crash the car on purpose. Why did she consider this? She considered crashing the car on purpose because she was getting older.

(34:00):

She had a heart condition, she feared her own death, and she realized that if she were to die, there’d be no one to look after her handicapped daughter sitting in the backseat of the car. And these are the stories that one needs to be aware of. And lastly, the rare disease ecosystem. Much of the rare disease associations I’ve mentioned to you who are absolutely critical in rare disease and rare disease, important and rare disease reporting, excuse me, are often, often underfunded, but also only have a few staff, many of whom have children or are themselves experiencing the diseases they’re representing, but they are absolutely central to the ability to report on and to advocate for in given individuals. And so being aware of these patient associations is critically important. And so first of all, thank you for spending the time listening. And all I would say is you are the oxygen for change. The science is happening at a very fast speed. It’s very sophisticated, it’s very technical. The opportunities we’re at a remarkable dawn of opportunity, and I would thank you all for listening and participating in this course. I’d like to thank Rachel and her team, but please, I beg of you report with your heart, report with your mind and help us mobilize impact. Thank you,

Rachel Jones/NPF (35:27):

James. I have to start by extending my absolute deepest condolences and also behalf of the National Press Foundation on the loss of your stepmother.

James Levine/Fondation Ipsen (35:43):

Thank you.

Rachel Jones/NPF (35:44):

You’ve graced me with sharing some information about your parents’ journey in that regard. And I am so, so very sorry. And so Tripoli grateful to you for taking time out to be with us. We are going to try to see if we can get over some of the technological challenges here. I’ve noted some of the questions in the chat and I’m going to ask Asco Tucci of Japan, if you would unmute yourself, because I think your question is very much at the core of our challenges as journalists in this pursuit. So please go ahead and introduce yourself briefly and ask your question.

Asako Takaguchi | Kyodo News  (36:32):

Okay, thank you so much. Thank you, James, for a wonderful story. So impressive. So my name is Asako and I am from Japan. So I’ve been working as a reporter at the news agency in Japan. And also I am also a rare deceased patient myself. And that’s the reason I joined this fellowship today.

James Levine/Fondation Ipsen (36:56):

Thank you.

Asako Takaguchi | Kyodo News  (36:57):

Yeah, thank you so much. And also, my question was, it was very similar to Lee Wade’s question because let me see, let me check. Because what I’m feeling as a problem as a patient is I don’t know how we should speak up as a patient because like you said, rare disease is very invisible. So my symptom is also invisible. So they don’t know that I am patient, but if we don’t speak up, they don’t know what challenges we face and things like that. But I feel as living in Japan, I feel that all the patients with my same disease, they are so shy and they don’t, they’re just too afraid to speak up about themselves. They don’t want it to be known as a patient. But if we don’t speak up, they never know what challenges we face. And if we speak up, if we are to be able to speak up, they will know what challenges and they need to know, they will know what they can do to support us. So that’s my question. What is a very effective way to speak up as a patient? If you have any ideas, could you share with me

James Levine/Fondation Ipsen (38:26):

Asaka? So thank you so much for your question. I think in a way you’ve just framed the reason for this course in the sense that the big answer to your question is to recruit top journalists from around the world to speak, to report on these things. And as I said last, the impact of this program reaches the last program reached over 250 million people. That’s an extraordinary number as individuals. I think one has to think critically in the sense that depending on one’s culture, sometimes one doesn’t want to speak out about one’s condition, one wants to be private because as the data suggests, we fear of stigmatization, we fear discrimination, and we don’t want to be viewed as having a situation. So I think first of all, I’m very respectful of people who want to be very secret about what’s going on medically and why shouldn’t somebody choose to be private.

(39:40):

On the other hand, if somebody chooses not to be, for example, we work with influencers with 30, 40,000 followers, social media provides a massive method for reaching more people than we can ever imagine. I think there are two extremes. On one hand, at the big level, we have world-class journalists who are going to write about these issues. We have individuals who can use social media very powerfully to express their stories and share their stories and reach out to other people. But on the other hand, I’m very respectful of people who don’t want any mention at all of any aspect of their medical care. And I think therefore there’s a whole variance. And thank you for your question.

Rachel Jones/NPF (40:26):

I am going to now shift to Evans. Jonah, you asked very many very great questions, Evan, but I’m going to ask you to focus on just one, and that’s the one about innovators and their need to learn or unlearn. Can you please introduce yourself and ask that question?

Evans Jona | Post On Sunday  (40:52):

Thank you so much, Rachel. My name is Evans Jona and I’m a reporter from Zimbabwe Post on Sunday. I would like to ask a question basically on innovation and rare diseases. Many people believe that where I come from here in Zimbabwe, that red diseases only benefit innovation when it comes to red diseases only benefit world nations. So how can we shift this narrative and highlight global inclusive progress for everyone?

James Levine/Fondation Ipsen (41:24):

It’s Evans. Thank you. It’s a really, really big question and I spent a lot of my time in the innovation space, so I’ve got sort of two answers for you actually. And again, I’ve got the link if you can’t find it. But if you look at the UNESCO World report on science focused on biotech, what you’ll see in the report is a sort of a blossoming of biotech innovation in middle and low income countries, right? Particularly, I mean, you think about what’s happening in Nairobi, for example, the biotech city in Nairobi. So in other words, I think there are significant economic opportunities. In other words, as biotech is innovating, it’s a great opportunity for countries, not typically not in Silicon Valley, not in London, et cetera, et cetera, to take opportunity of this. And I think there are big economic development opportunities to develop biotech infrastructure.

(42:22):

So of course there needs to be policy driven and so on and so forth. But I think there are big opportunities in that regard. So that’s number one. At the other end of the spectrum, however, is I think a core issue that you’re going to come up with again and again and again, which is the affordability of new treatments as they come out the pipelines. And I think, I mean, it’s a global issue and I don’t have the answer. The only micro answer I have is that I was at a meeting in Delhi where one of the ministers for health was there, and we’re talking about building innovative capacity in India. And the fundamental idea there is, I’m not sure if you’re aware, but again, I dunno the exact number, but a huge number of medicines are actually made in India.

(43:16):

The manufacturing capacity in India for making medicines is enormous. And one of the ideas we discussed is this idea is gene therapy becomes more and more established and the mechanisms and the machines to do it better and better, the ability to mass produce genetic therapies at about three $50 per person is going to make these kind of therapies much more available. So the sort of conclusion is that, I mean, it’s no real answer to your question. I haven’t gotten an answer to your question. And there is inequity and it’s, there’s a moralistic undertone to all of that. But nonetheless, I think therapies are going to become more affordable through the capacity to mass produce. There are going to be much like you saw with the HIV global fund, the ability to deliver medicines to underserved countries actually very successfully. I think those are the big opportunities. But in addition to that, I think biotech development in underserved countries is a massive opportunity. But great question. Thank you for your question.

Rachel Jones/NPF (44:22):

I want to shift now to Sharon Zaki. She asked an interesting question around balancing excitement, the need to protect families. Sharon, introduce yourself and ask your question, please. She may be having some technical difficulties. Let me ask it. You spoke about the risk of,

Sharon Muzaki | Nature Africa (44:44):

Okay,

Rachel Jones/NPF (44:47):

Sharon,

Sharon Muzaki | Nature Africa (44:48):

Thank you so much, James. Thank you for everyone for attending. And thank you Rachel. My name is Sharon Muzaki. I’m an environmental and science journalist with Nature Africa. And thank you for the presentation. You spoke about the risk of false hope in rare disease reporting. How can journalists like me balance the excitement around the emerging therapies? There are many and gene editing, of course, with a need to protect families from misinformation, especially in regions like my country, Uganda and the rest of the Saharan Africa, where scientific literacy is law. Thank you.

James Levine/Fondation Ipsen (45:29):

I mean, I think it’s a fantastic question. And I suppose the answer to your question is in the articles, you’re right in the sense that as Ron writes, and I’m not a journalist, as I’ve said to you, but as that the story is presented to your public, my feeling is that there needs to be a sort of reasonable balance between, this is actually a thrilling advancement of the capacity of science. But the reality is that just as we heard before that not everybody in the world gets access to these therapies, and it almost creates a quite interesting talk in the story in the sense that this is actually very exciting, this is happening. But on the other hand, there are sort of international political geopolitical struggles to bring good therapies right across the world or to scale up these therapies or to bring them in sort of a price effective manner. And so I think there’s nothing, if I was reading a story and I was to read that balance, that wouldn’t eliminate the hope and it wouldn’t sort of turn me off from reading the story, but it would actually intellectually give me a true balance as to what the journalist was expressing.

Rachel Jones/NPF (46:48):

I’m going to now turn to, excuse me, Davis Biondo. He has an interesting question about traditional medicine. Davis, are you there?

Davis Buyondo | New Vision (47:00):

Hi everyone. And condolences to you, James.

James Levine/Fondation Ipsen (47:04):

Thank you. Thanks for everybody. I wasn’t going to talk about this, but it just came out and I think it’s correct, so thank you.

Davis Buyondo | New Vision (47:11):

Sorry, it brought back your memories. Okay, my concern is you talked about therapies and usually in Uganda, this is where I’m based right now. In Uganda, one thing happens, traditional therapies are over underlooked, underlooked, and they’re considered to be outdated in the way since it requires deep research. And which research is usually endorsed or approved by one body, which is what health organization. So I’m looking at how we can connect traditional therapies that are working with the modern therapies, or maybe I can also say something that, sorry, I took your time. Most of the time, journalists, we step out to write about right diseases, but ourselves as journalists, we are suffering and we can’t come out to say something about it. For example, I got a call goon lost a child, a baby, nine months, nine months. And this guy who was working with one of the biggest media organizations in Uganda called The Daily Monitor. And this guy came out only when the kid was at the verge of death. But it’s been writing about other rare diseases, but it couldn’t come out to say I got a child suffering. So as journalists, I think we should also learn from what we’ve seen. We saw people with autism coming out to speak, to write. So we shouldn’t

Rachel Jones/NPF (49:07):

Thank you for that question, Davis. I think the important one is the one about, and certainly we journalists need to have that empathy, but address his first question about traditional medicine.

James Levine/Fondation Ipsen (49:20):

I think Davis is actually a super question. And actually at the foundation, we’ve just completed a book with a professor writing about what’s called in the United States, integrative medicine in rare diseases. And it comes right to your point. So integrative medicine is sort of where the traditional medicines, herbal medicines, yoga, therapy, et cetera, et cetera, are integrated into medical care. The only distinction that’s important is that these are not replacement medicines, like an herbal medicine should not replace a potential therapy, they’re integrated. So that’s the only comment there. But what’s I think very important is with that understood that a traditional medicine shouldn’t replace or displace a trial based therapy, but also remembering that the vast majority of people without rare diseases have no therapy. What’s therefore important that some of these integrated medicines, some of these complimentary approaches are actually very important as providing hope for people living with rare disease.

(50:42):

So in other words, what is particularly tragic is that somebody with an undiagnosed rare disease goes to a doctor and the doctor says, dunno, what’s wrong with you? Sorry. And the person leaves, I mean, with no hope, right? On the other hand, again, following up from your question, the same person goes to the doctor and says, well, actually, we don’t know exactly what this illness is, but there are six things we would recommend for you. And while some of those are certain types of herbal tea that help with the anxiety, we also recommend art therapy or narrative therapy. We recommend yoga therapy to increase your flexibility and so on and so on and so forth. And what the patient actually leaves with, again, without it being hype to come back to Sharon’s point, but without it being hyped, but is actually an evidence-based therapeutic program, but not a curative one. So therefore, Davis, I think quite interestingly, there’s a lot of room for this sort of thinking, and I think it’s sort of quite powerful. And again, you do not want, however, I’m a physician, I do never want to say to a patient I’ve met, I’ve met patients, have patients with terminal cancer and so on and so forth. You never want to say to a patient, so sorry, nothing I can do. So there’s always something we can do.

Rachel Jones/NPF (52:07):

I think we are coming near the end of our session, but I wanted to include one more question from Sina because I think it is a very important one. Sina, Navi, can you ask your question?

Safina Nabi | Independent (52:22):

Yeah. Thank you so much for giving me the chance to speak. So basically, as a journalist, when we try to write stories about rare diseases, we are already struggling because we don’t know how the disease is impacting a person then conducting all of those interviews. And when it comes to writing the story, it is very important for us to have experts included in the piece who can defer all of those questions, technical terms. But I always struggle to figure out who to speak to, how to speak to, and at times they goose you at the last moment when you have to submit the piece, even if they agree to speak to. So my question is that how will we navigate those challenges? Is there any database or do you people have any kind of an information that you can share with us so that we come up with all of those challenges?

James Levine/Fondation Ipsen (53:12):

Yeah, yeah, it’s a great question and it’s very, very important. So the way I actually always address this is the same way, actually, the first thing I do is go to PubMed, national Library of Medicine, PubMed in internationally. It’s available everywhere in the world. And then I type in the rare disease and then see what publications come up. And then what you can do is search for the authors and their contact information from there. And that’s number one. Number two, as I mentioned several times, rare disease patient associations are surprisingly not willing, but enthusiastic to provide you with contacts because they want journalists to advocate for the rare disease. So that’s my second recommendation. And Rachel, I will tell you that on occasion people have contacted me or my team, and if you’re totally stuck, we can help you go through the same process. But those are the two things I always do. Rare disease association, start with them and then PubMed to see the scientific experts. And I am aware of the ghosting, so I always have the theory of find three experts and get one opinion.

Rachel Jones/NPF (54:28):

You have provided me, and I’m sure the journalists as well, with probably one of the most elegant call to arms that I’ve ever received as a journalist. And that is to think of journalism as the oxygen of science. We have the opportunity to breathe life into this story through our reporting and through our context. I want to ask you to close us out by telling us what is your vision for this fellowship program? What has it been when you started, I guess, the initial conversations with Sonny Efron and where we are now? What is your hope as we move forward?

James Levine/Fondation Ipsen (55:09):

Well, I mean, first of all, just gratitude to every single person on this call for taking. I know it’s a proper fellowship. I know it’s for some people and so on and so forth, but that’s not the point. I am a scientist and a physician, and I see the pain and suffering as a scientist. I see the vehicles of opportunity and hope coming down the line every single day actually. But who is going to tell that story in the world? It’s not me, and it’s not my colleagues. It’s not people at the clinic, it’s you. It’s you who are listening today. You are going to spread this word of hope. And I’ve seen this program emerge over the last three or four years now, and every year the quality of the stories get better. The impact of what you do is extraordinary, and there is a massive community of need who are at your doorstep today. So please take on the challenge. Please write beautifully with your head and heart, and please put oxygen into the rare disease community.

Rachel Jones/NPF (56:27):

James, you have often spoke of rare disease patients and families as being heroes, and you today are our hero in this program and in this effort to educate journalists. So once again, our deepest condolences to you and our deepest gratitude to James Levine, president of Foundation Ipsen, for launching the 2025 Rare Diseases Reporting Fellowship. Thank you so much.

James Levine/Fondation Ipsen (56:55):

Thank you all. Thank you.