‘Invisible’ diseases like primary biliary cholangitis (PBC) can be difficult to recognise, diagnose and to manage effectively. Here Prof. Gideon Hirschfield (University of Toronto, Departments of Medicine, Immunology and Medical Sciences) and Robert Mitchell-Thain, (PBC Foundation UK) discuss the disease, how it presents and the challenges facing people living with this rare liver disease.
Watch the video below containing some excerpts of this discussion, focusing on what people with PBC and doctors can do to better understand each other and have a better conversation, which may lead to better outcomes for people with PBC.
You can never truly understand what it’s like to live with an ultra-rare condition unless you’ve experienced it for yourself. And with this in mind, we recently embarked on a project to help healthcare professionals experience what it’s like to live with Fibrodysplasia Ossificans Progressiva (FOP). The ‘Life in a Day’ project is an immersive experience that helps participants to understand the physical and emotional impact that a disease, such as FOP, can have on the day-to-day lives of those it affects.
Debuting at the ASBMR Congress in Austin, our aim was to give delegates a glimpse of what it’s like to live with FOP through an immersive booth experience.
We asked delates to share their learnings. Here’s what one of them had to say:
“FOP is an ultra-rare disease, with an estimated prevalence of 1.4 per million individuals. Those who live with the condition experience heterotopic ossification (bone growth) which occurs outside of the normal skeletal system. Working in rare diseases, I’ve heard this description and said these words many times, but since taking part in Ipsen’s FOP immersive experience they’ve taken on a new meaning.
The FOP experience gave me a glimpse into what it’s like to live with FOP, simulating the morning routine: getting out of bed, getting dressed and having breakfast but with certain restrictions to demonstrate how those living with FOP experience these daily tasks. It sounded simple, but I soon realized that even the simplest of tasks can be challenging when you’re living with locked joints or restrictive movements. And to make it more impactful, as we took on the challenges, we listened to audio clips of patients sharing their experiences.
Let’s start with getting out of bed…or sleeping for that matter. Because of the extra bone formation, FOP can cause locked joints – to simulate this, I put on a leg and arm brace to restrict movement. It completely limited my mobility and made it incredibly difficult to get out of bed. Eventually I had to roll myself out of bed, although I almost asked for assistance before following through with this. I suddenly realized the extra care I would need to take to ensure I could get out of bed safely. This is important in FOP where a bump or fall can lead to a ‘flare up’ and trigger more heterotopic ossification.
One of the patients I heard from talked about the fact that she’d lost the ability to turn in bed so she couldn’t move from lying on her back. She joked that people often say “oh I don’t want to get out of bed” but her reality is that she can’t get out of bed by herself. Another patient talked about the strength required to get herself out of bed, comparing it to a sit up, “I use my whole strength from my middle rather than my legs or my arms to get out of bed… it’s actually physically hard, it’s really really tiring.” It made me appreciate what I take for granted every day.
I then tried to eat breakfast. For this, my arms were strapped to the table and I wore a neck brace. After several attempts I realized just how hopeless it was trying to eat with a normal fork and opted for a telescopic one. One of the patients we heard from said that cutlery is almost pointless to her, and I could see what she meant. The process was slow and required a huge amount of dexterity. On a couple of occasions I managed to get the food close to my mouth, only for it to fall just before I could get it in. It was incredibly frustrating. Adding to this challenge, many people with FOP live with locked jaw, so I had to try and cut food up into tiny pieces for it to fit into my mouth. Again, the patient stories were so thought provoking. One patient told us she could only really have “smoothies and soft foods like rice and some pasta”. She explained “my mouth opening is pretty much the size of a fingernail so it’s very hard for me to eat. I have to shove food in there” which makes her not want to go out in public because it’s very messy. I really sympathized with this and how this would take away the joy and social element of eating out.
Finally, exhausted from the first two activities, it was time to get dressed. Armed once again with braces to restrict my movement, I attempted to put my socks on, and I realized just how difficult it would be to dress yourself when you can’t bend over. I was given an assistive device to help and without it, it would have been impossible. But even with the device I struggled to put on my socks. One of the inspirational patients we heard from said, “it’s very difficult to find clothing that is easy for me to do independently which is a big thing for me. I’m very independent and I don’t like asking for help”. I started to imagine was this must feel like – the loss of independence – and the thing is, it’s something that people with FOP have to deal with every single day.
Overall, what struck me was just how long everyday tasks take when you have limited mobility and how frustrating it can be. The patients who shared their experiences were remarkable, their resilience and courage in the face of this awful disease, hugely motivational. Despite the challenges of FOP, they found solutions. They adapted and continue to adapt as their condition progresses.
I encourage everyone to take the time; to listening and to learn about the lived experiences of patients. Less than 1,000 people in the whole world know what it’s truly like to live with FOP, but this glimpse has helped me to understand some of the realities these people face on a day-to-day basis. For me, this was a 30-minute uncomfortable experience. For those living with FOP, it’s a lifetime.”
In December 2021, Ipsen received ISO 37001 certification for its anti-corruption management systems by Eurocompliance, a certification organization dedicated to the prevention of corruption.
This certification recognizes the robustness and effectiveness of Ipsen’s anti-corruption management system, supported by a strong engagement of all the organization, allowing the identification, control, and mitigation of risks of corruption and bribery.
Last July, after additional on-sites visits by EuroCompliance, the certification has been renewed for 2022. This renewal is the demonstration of our strong commitment to the fight against corruption, and to Business Ethics at large.
“Our growth platforms have delivered strong sales performance, reflecting improving commercial execution and the strength for our portfolio.” CEO David Loew
Ipsen announces that it becomes a founding sponsor of the second Biolabs innovation brain hub in Europe at Hopital Hotel Dieu, AP-HP, in Paris, France. The announcement builds on a 2018 commitment to a partnership with Biolabs for the development of a network of life sciences incubators in Cambridge, Massachusetts, and a second co-working facility in Watertown, Massachusetts.
“We are proud to renew our long-standing partnership with Biolabs by becoming a founding sponsor of their second life science innovation hub in Europe, at the historical Hopital Hotel Dieu, AP-HP. There is great science in France and Europe that can benefit patients around the world”, said Ipsen EVP and Chief Business Officer Philippe Lopes Fernandes, who made the announcement live yesterday at HealthTech Innovation Days (HTID). “With biotechs facing a funding drought, high inflation, and riskier choices, we remain firmly committed to fostering the discovery of innovative medicines through partnerships on both sides of the Atlantic.”
As one of two pharma founding sponsors of Biolabs-Hotel Dieu, Ipsen will be part of the member selection committee. We will harness our expertise in development of transformative medicines to help grow and nurture the whole community of residents, as well as participating into a ’Golden Ticket’ program to provide hands-on support to a biotech of choice each year.
“We are delighted to expand access to state-of-the-art facilities for scientists and entrepreneurs in France and Europe together with our long-established partner Ipsen,” said Johanna Michielin, head of BioLabs France. “France has a proud heritage in the advancement of medicine. The innovation hub Biolabs-Hotel Dieu, will be accessible to all scientists and start-ups looking to innovate in the field of biotechnology and digital health in partnership with the public hospital”, added Johanna Michielin.
Our strong pursuit of external innovation in life sciences is embedded in Ipsen’s growth strategy. In the past couple of years, the partnering team inked deals for one commercialised product and more than 20 investigative programs across our areas of interest – oncology, rare disease and neuroscience. With our sights set on forging new partnerships, the team will seek for further opportunities at Bio Europe in Leipzig, Germany in late October.
In France, we are running a biotech mentoring program together with France BioTech. Over the past year, our R&D team in Les Ulis has been providing guidance to several entrepreneurs on how to devise their research and development plans.
Continuing medical education is vital to maintaining the quality of clinical practice, which in turn can improve patient outcomes. As a pioneer in botulinum neurotoxin type A (BoNT-A) treatment, Ipsen has worked with a scientific steering committee of international experts to devise a high-quality, expert-level educational network to help experienced physicians improve their management of patients with cervical dystonia or focal spasticity who receive BoNT-A injections.
The Ixcellence Network® is an international educational program that targets physicians who already have some experience in the use of BoNT-A injections in the fields of cervical dystonia or focal spasticity. Its specific training seeks to deepen understanding the impact of spasticity on the patient and then to tailor the treatment of the patient based on these assessment outcomes.
The value of this important program, which is considered the only toxin educational program with published findings,1–3 is also highlighted in seven abstracts at high profile congresses such as TOXINS and MDS International Congress of Parkinson’s Disease and Movement Disorders (MDS).
Despite the two-year hiatus in face-to-face training over the pandemic during which we utilized virtual training, over 800 highly experienced physicians have benefitted from the program, regarded as the most comprehensive training of its kind which disseminates international best practices to improve patient care with modules ranging from goal setting and evaluation, muscle identification and guidance techniques, and multi-disciplinary team patient management.
It is perhaps best for a founder member of our Ixcellence® steering committee to convey the impact that the program has had. Listen to Dr. Jorge Jacinto PM&R Senior Consultant, Head of Department of Adult Neuro-rehabilitation, Head of Movement & Gait Analysis and Head of the Adult Botulinum Toxin at the Clinics Centro de Medicina de Reabilitao de Alcoito, Alcabideche, Portugal.
The Ixcellence Network® has improved standard of care for thousands of patients with cervical dystonia and spastic paresis treated with BoNT-A injections. It uniquely brings together specialists in neurology and rehabilitation and to facilitate information exchange and networking. The involvement of the steering committee experts in cervical dystonia and spasticity and the shared learning experience have led to new ideas to develop the courses further and we look forward to another decade of supporting learning with agile and high‑quality education.
We see a bright future for the Ixcellence Network® as it continues to grow in stature, and we are proud of our commitment to the industry to help form specialists of the future delivering optimal patient management. The network is a globally-driven training program, organized via our local medical departments; if you wish to hear more about the network, please contact the Ipsen Medical Science Liaison (MSL) for your area.
By Hamzah Baig, Senior Global Medical Affairs Director for Neuroscience at Ipsen
References
I was honored to be invited to take on the challenge to live ‘A Life in a Day of a patient with FOP’ – but also apprehensive. The challenge simulated a day spent living as someone who has fibrodysplasia ossificans progressiva (FOP). FOP is a debilitating, ultra-rare disorder where bone forms outside of the skeleton in muscles, tendons, and ligaments,1 which can be a response to flare-ups, which are often brought on by trauma, such as a fall or an intramuscular injection.2 This incorrect bone formation leads to locking of joints and debilitating reduced mobility over time.3,4 It is chronic and progressive and has a huge impact on the daily lives of those it affects…3,4 as I was soon to find out…
Patient centricity is a mantra for many companies, but at Ipsen patients truly are at the heart of everything we do; we work with them, and we work hard to empathize with the challenges that they face, to improve what we do. Our partnership with The Method, a company that encourages patient centric thinking and behavior through immersive experiences, has allowed Ipsen employees like me to gain a very small insight into the realities of living with an ultra-rare disease.
Overview of the experience
The immersive experience was brought to life through an app with regular interactive challenges, live role play, and some aids to simulate the physical challenges of people who live with FOP.
The day started when the app pinged the first challenge to me at 7.00am. I was provided with instructions to use a range of innovative wearables that served to restrict my movement for the day. They even managed to simulate a swelling on my leg using a foam ball and strapping, as a sign of a potential flare up…
Over the course of the next 12 hours, I received regular instructions to help me experience what living with FOP might be like – and to make me contemplate how I would cope with the knowledge that my mobility was likely to become more and more restricted as time went on.
It was completely immersive.
Every aspect of my life was impacted; getting dressed, eating, commuting, typing on my keyboard, using the bathroom. All of it presented challenges in ways I could not have anticipated. There was a real difference between reading or hearing about the impact of the condition on patients, and actually attempting to live like a patient.
It went well beyond the physical challenges though… I had to consider the impact on friends and family, my career, and my emotional well-being. I lived with the anxiety of falling and injuring myself, and of losing more mobility. I had to engage with the way other people (including colleagues) perceived me and my condition – and there were interactive role play calls to assess what adaptions would be needed to my house to help me live in it as my mobility deteriorated. A challenging call from an hypothetical employer questioned my capabilities and a conversation with a patient support group made me realise just how much I would need to think about as my condition progressed.
It lasted 12 hours, but it truly was ‘A Life in a Day’. I was transported through a lifetime of issues and challenges that impacted me in ways I could not have imagined. The ongoing instructions from the app were relentless and exhausting, and while it is nothing compared to living with the condition, it gave me a powerful glimpse into the physical, social, and emotional challenges that are an everyday part of living with FOP.
On the day of the experience, I happened to be with my extended family in the small village where I’d grown up and where many had known me since childhood. I was given some dice as part of the challenge, which I had to throw throughout the day to determine the severity of incidents that I experienced, such as the outcome of dental checks, or the severity of a fall. The countdown to news and having to wait until later in the day to find out the impact of an incident really brought home to me that this condition is subjective in how it takes over of the bodies of the people it affects.
In one challenge, I had to feed myself with a telescopic spoon. Sounds possible, but with restricted joint movement in both arms and – by that late point in the day – an inability to bend at the waist and sit up, the spoon was useless. Wearing a sterile bib and eventually having to give up and be fed by my son, it was a disempowering experience. Eating was no longer fun.
In another role play, I talked to a patient group member who is living with FOP and many of the symptoms I was experiencing, including a locked jaw – he was talking to me as if I was a patient myself and I found it very hard to ‘join in’ knowing that all the problems I had were so very temporary. He had a humor and kindness that still make me emotional to think about now.
The challenges had a huge impact, not only on me, but also on my family and for the people I bumped in to during the day. My mum was overcome and deeply affected – she did exactly what you’re not meant to do…felt sorry for me all day, answered for me, took away my choices, wouldn’t let me do things for myself. It was as interesting watching and learning from the reactions of those around me as it was observing my own.
Overall, the experience was visceral and incredibly emotional and I’m grateful for having had the opportunity to experience in some small way what patients with FOP live with every day. It felt like a privilege; indeed, it was a privilege. It was humbling, frightening, and eye-opening, but importantly it was also an opportunity to raise awareness.
I’m sharing my experience because I want to communicate the impact it had on me and the potential effect it could have on others. As far as we know, only 900 people in the world truly understand what it’s like to live with FOP.5 Getting a small glimpse of what they face reminded me of what’s at stake. Putting ourselves in the shoes of those we support is not only inspirational and motivational but also fundamental in ensuring we are equipped to deliver against our Ipsen promise: to focus. Together. For patients and society.
I encourage others to walk in the shoes of patients with FOP. Only then can we really begin to understand and appreciate the challenges they face.
By Abbie Pound, Culture and Engagement Director, Patient Centricity Champion, CSR Champion. UK & Ireland Global Hub, Ipsen
In October 2021, Ipsen pledged ambitious action on climate change. Today, we announce that Ipsen’s Climate Targets were approved by The Science Based Target initiative (SBTi). This approval confirms that our greenhouse gas (GHG) emission reduction target is in line with a 1.5°C-aligned trajectory, the most ambitious goal of the Paris Agreement.
The Science Based Targets initiative is a collaboration between CDP, the United Nations Global Compact, World Resources Institute (WRI) and the World Wide Fund for Nature (WWF), which defines and promotes best practice in science-based target setting. The SBTi is regarded as the global authority in the independent assessment and validation of science-based targets.
Ipsen is a member of the Business Ambition for 1.5oC campaign which commits Ipsen to decarbonize on a science-based trajectory. SBTi approval is an essential step in demonstrating that our targets are truly science based. However, it is our actions that are the best demonstration of our commitment.
“Our commitment to driving societal impact includes our climate pledge. I have spoken many times on the need to step up to meet climate as one of the defining challenges of our time. Our environmental approach is fully integrated into our strategy: Focus. Together. For patients & society,” says David Loew, CEO. “SBTi approval demonstrates our commitment to meaningful action and to have a positive impact for future generations.”
Hear what CEO David Loew has to say about the official close of its acquisition of Epizyme.
The International Liver Congress (ILC)1 provides a key opportunity for us to meet and discuss the management and research of liver diseases. Given the online format of recent meetings, it was great to have the chance to interact, discuss and debate in person this year.
A key focus of my clinical and academic work is primary biliary cholangitis (PBC), a so-called ‘rare’ liver disease that carries a substantial burden for patients, including an ongoing risk of developing cirrhosis. Given PBC is a less frequent condition than many other liver diseases (one in 1,000 women over the age of 40 years old live with PBC),2 dynamic international meetings such as the ILC, enable us to raise awareness amongst those without specialist knowledge of how we can effectively diagnose, manage and monitor disease.
The industry sponsored symposium entitled “Current challenges in Primary Biliary Cholangitis: Debating the care odyssey” was a good example of an opportunity to discuss, learn and disseminate knowledge about PBC. This meeting captured many facets of PBC relevant to clinicians and patients. I was invited by Ipsen, along with two colleagues who are also experts in PBC, Prof. Cynthia Levy (USA) and Prof. David Jones (UK), to debate whether delayed diagnosis is the greatest challenge in PBC. We were all really on the same side, to raise the quality of care for patients with PBC by recognising aspects of disease that impact quality of life.
It was clear from the discussions that timely diagnosis was agreed to be key for the effective management of PBC, particularly as the disease can progress to a point at which a response to therapy becomes less likely. However, the counter argument highlighted that delayed diagnosis is not the only unmet need in PBC. Without effective, disease modifying treatments that also address symptoms, management of PBC can be challenging. Early-stage disease needs to be diagnosed efficiently and successfully, but this should not detract from the need for effective therapy in already identified high-risk patients. In addition, symptom control and quality of life matter greatly, and this should be considered when developing new treatments and making management decisions.
The debate led to many clinical points about PBC care being discussed. The management of PBC is rightly increasingly dynamic, as new pharmacological innovations are in development, and our practice needs to adapt to these changes.3–5 ILC offers an excellent opportunity to open a dialogue with clinicians from all over the world. As a result, we continue to step closer to the goal of providing the best care to every patient, no matter where they live.
PBC, and related cholestatic liver diseases, were hot topics throughout ILC, with presentations on research in the inflammatory and cholestatic components of disease, disease-specific quality of life assessment tools, and population-based data reporting care practices. Other important topics discussed included treatment targets and how we can provide lifelong and personalized treatment to patients with this chronic autoimmune liver disease. The identification of high-risk patients, with insufficient response to standard of care, who require second-line and subsequent treatment also remains a challenge. Finally, the vexed topic of overlap syndrome remains an important theme for many conversations about PBC. However, there is hope that as we are better able to treat patients with PBC with more effective therapies, earlier in their disease, we may indeed see less patients with overlap features as part of their disease course.
As we look to the future, we are entering a new era, where a greater understanding of the disease alongside therapeutic advances, will equip us to offer more impactful interventions for patients living with PBC.
Prof. Gideon Hirschfield
University of Toronto, Canada