Dr. Amauri Soares, VP Head of Early Development & Innovation Oncology at Ipsen uses
music to explain the importance of understanding the genetics of cancer and how the emerging field of
epigenetics could unlock a new paradigm for precision medicine.

By Dr. Amauri Soares, VP Head of Early Development & Innovation Oncology

At 15 years old, my mind was made up. I was going to be a doctor. My interest was driven by curiosity.
There was still much to understand about medicine, and I became passionate early on about discovering
solutions that could potentially save lives.

In medical school, this ambition led me to hematology. From leukemias and lymphomas to anemias and
other rare blood disorders – the field was vastly diverse, and the complexity of something that seemed
so simplistic, our blood, drew me in.

Now, with more than 20 years of experience in medical affairs and research and development, I’m
surrounded by others who are innately curious and share my passion to find innovative solutions.

The emerging field of epigenetics

Our team at Ipsen is focused on bringing new medicines to people living with cancer. Innovation in
oncology is especially exciting because we’re in an era of precision medicine. Advances in the field of
epigenetics, particularly in hematology, mark a completely new paradigm for precision medicine –
building on scientific successes to harnessing the immune system and our knowledge of biomarkers to
meet cancer where it forms.

By understanding different targets, drug mechanisms and the underlying disease drivers, we can often
treat cancer using combinations of therapies with complementary approaches or even medicines that
rewrite affected parts of our DNA.

Epigenetics are an innovative approach to developing investigational cancer medicines by focusing on
identifying and targeting key aspects of the DNA that can alter or dysregulate the basic structural
components of chromosomes (chromatin) to slow or even stop tumor cell proliferation.

Creating Harmony

There’s no doubt that the field of epigenetics is complex and is considered by many as “deep science.” As
a lover of medicine and music, I’ve found the most simplistic way to understand and explain how it
works is with a metaphor.

Think of DNA as the sheet music. While there are only a certain number of musical notes, the variety of
compositions are endless because of the arrangement of the notes, the pitch and a musician’s
interpretation of the tempo, melodies, and the tone. Now, imagine the cells in our body as the
instruments that each have a unique function – the base drum, the guitar, even the piccolo. In smaller
groups the instruments can form a symphony of woodwinds, strings, brass, or percussion, whereas in
our body together they complete a full symphony.

While each of these instruments (cells) has a role in the symphony (our body), epigenetics can be
considered the conductor of the music, the environment, or the musical style of the composition. For
example, envision a Brazilian Bossa Nova song with a steady base, syncopated chords on the piano or
guitar and a melody from a horn or vocalist. Now, if those same notes were influenced by Elton John’s
ballad style tone, tempo, and melody, that influence is an example of an epigenetic alteration or
dysregulation that drastically changes the arrangement and in the case of your body, influences how
your DNA is interpreted by individual cells.

Epigenetics also explains why we have such diversity inside the cells of our own bodies and how DNA can
create brain cells, blood cells, and every other kind of cell in our bodies. In the same way as the infinite
amount of musical compositions with the same core musical notes, epigenetics is the reason each
human is unique even though we share a common genome.

However, sometimes the tone, tempo or melody change just doesn’t work for the composition. Certain
instruments are too loud or soft, sound off-key or the melody no longer fits the lyrics. This is similar to
when certain types of cells become too dominant, proliferate too rapidly, or create dissonance with
others and ultimately can become cancerous cells.

Epigenetics allows us to “tag” or mark different notes (genes) in the cell to turn them “on” or “off.” For
cancer cells, this means epigenetics can “tag” certain genes or proteins to suppress tumor proliferation.
Since there are many applications and impacts of epigenetics, there is also the ability to use epigenetics
to add or remove notes (tag writing and erasing), clarify the length of the note (tag reader), or even
change the pitch of a note (tag remodeling).

While we are only at the beginning of this novel approach to identifying epigenetic-related targets in
different cancer cell lines, our platform has already identified hundreds of “tags” across more than 400
cancer cells. Epigenetics unlocks the potential to develop treatments that alter or dysregulate cancer
cells by targeting the source of the mutations. Or put simply, we have the potential to silence certain
instruments in the symphony when they’re playing out of tune.

A future of possibilities

With Elton John reverberating in my head, it’s easy to get excited about all of the possibilities of
epigenetics in treating hematological malignancies like follicular lymphoma. We can apply what we know
today about biomarkers, tumor targeting and even drug delivery, but also look into the future to imagine
synergies with the tools available today for potential combination therapies.

We also must remember that scientific advances can move at different tempos – sometimes a rapid, fast
step forward, and other times the science builds on itself with many different influences to provide
incremental advances. We have to celebrate the accomplishment of both, as this is what keeps our field
moving forward.

©2023 Ipsen Biopharmaceuticals, Inc. All rights reserved. DS-US-000091. 12/2023

Debbie has been living with a blood cancer, follicular lymphoma (FL), since 2011 when she was initially diagnosed during her studies in England. While living with an indolent cancer has had its challenges and can be filled with uncertainty, Debbie seeks to find joy and live every moment to the fullest.

For Debbie, being diagnosed with FL came completely out of the blue. At the time, she was attending university in England and attributed many of her initial symptoms, like fatigue and weight loss, to studying and being in a new environment. But after two weeks of tests, she was told she had FL and needed to start treatment immediately.

FL is a cancer of the lymphatic system and a type of non-Hodgkin lymphoma (NHL) that develops when the body makes abnormal B lymphocytes. In FL, an indolent, slower growing cancer, the lymphocytes slowly build up in the lymph nodes or other body organs, making it difficult to detect. [1]

“It’s a roller coaster,” Debbie describes her experience with FL. “Some days are better than others, but you have no idea how you’re going to feel at any given moment.”

From the beginning, Debbie was grateful for the support of her friends and family – specifically her husband who has been an important emotional support, holding Debbie’s hand “both metaphorically and physically.”

While there is no cure for FL, it can be treated, allowing people to often live for many years with the disease. However, it is likely that the lymphoma will return at some point. For individuals who relapse or become refractory, second-line therapies are often successful in providing another remission. Oftentimes, the age and overall health of the individual plays a role in treatment decisions as well, and an “active surveillance” or “watch and wait” approach might be used. [2]

Debbie has been in remission for nearly ten years, and she says there’s a part of her that’s always waiting for it to come back. She lives with the day-to-day uncertainty around if or when the cancer might come back.

“The pressure grows the further you go without having relapsed,” she says. “It hangs over you constantly.”

While living with an indolent cancer has its challenges, Debbie seeks to find joy and live every moment to the fullest. In addition to spending time with her family, friends, and pets, she has taken up calligraphy since her diagnosis and says it helps to take her out of her everyday life.

For others who are recently diagnosed with FL, Debbie encourages you to “find someone to talk to and not to compare your journey to anyone else’s” adding, “we’re all very different.”

Learn more about Debbie’s story:

Hub experienced a hemorrhagic stroke in 2021 as a result of bleeding in the brain. Like many stroke survivors, Hub experiences spasticity, a condition in which muscles contract uncontrollably leading to pain, stiffness and limited movement. As a father of three, Hub was determined to recover and put significant emphasis on seeking the care he needed, including spending five weeks in a rehabilitation facility.

Spasticity is a common condition that can be experienced by those who have had a stroke, affecting 25% to 43% of people in the first year following the stroke. It can impact day-to-day life, including a person’s ability to move and can cause pain that impacts sleep or doing simple tasks like dressing, walking, or getting in and out of a chair. In addition to the physical challenges, the emotional impact of living with spasticity can trigger feelings of sadness, frustration, and isolation related to the significant changes to a person‘s previous lifestyle.

Hub experiences spasticity in his hand, causing issues with some tasks that were previously second nature, such as writing or typing on a computer. To practice writing, his young son brings home extra writing homework, and the pair practice together.  Hub is thankful for this time with his son, saying, “It’s very fun and it’s about the experience.”

Although faced with a long road to recovery, Hub credits his support system for helping him stay positive and motivated – specifically the support of his family, friends, and doctors. Speaking to one of his favorite memories when he returned home, he explains that his oldest son loves cooking on the grill and he invited friends over for a grill party, where Hub cooked burgers and ribs for everyone.

Hub notes that you can’t expect a change overnight in a recovery journey. While it is challenging, he recommends being patient and looking at how far you’ve come since the beginning. Some days are more difficult than others, and Hub remembers that progress is what’s important, saying, “Sometimes bad things happen and you got to keep moving forward. You can’t give up. We can’t stop. Keep moving on.”

Learn more about Hub’s story:

Savannah – or Sassy Savvy as she’s known by her family – is a caring sister, a strong-willed daughter and above all else, overfilling with creativity, happiness and laughter. Savannah is also living with a rare disease, fibrodysplasia ossificans progressiva (FOP), and while it has a great impact on her life, she nor her family let it define her.

FOP is an ultra-rare disease that impacts the lives of fewer than an estimated 400 people in the U.S. The disease causes new, abnormal bone to form in muscles and joints, which over time severely restricts mobility and function, such as eating and walking independently or providing selfcare.

For Savannah, the physical challenges of FOP have led to limitations in what she can do when compared to others her age. However, her passion for singing remains unchanged. She practices with her keyboard and often encourages her dad to join in (although he admits he’s “not as good” as Savannah). 

Despite the day-to-day challenges of life with FOP, such as the pain resulting from the abnormal bone formation, Savannah’s mom Christina explains that “she is so strong-willed.”

Currently, Savannah is also home schooled, so her family is her primary social network. “The social aspect of Savannah having FOP has been big,” shares her father, Jamal. “We are the people she sees for the most part. We’re her best friends, so she’s not getting interactions with kids her age.”

Savannah’s big sister Saniya is her biggest advocate and provides incredible support. Yet, Saniya recognizes that the support is two ways – Savannah “helps me with my nerves. She helps me to feel confident in myself. And she helps me just really strive and just be the best that I can be.”

Her family agrees her “positivity is a big thing that she’s taught this family. She has the ability to be so impactful to other people. Without her we wouldn’t be as positive as we are and I never want her to lose that.”

For others living with FOP, Savannah wants you to know “FOP is rare and that means you’re special.”

Learn more about Savannah and hear her family’s story:

Nora and her husband, Hector, met 39 years ago, got married shortly after, and have been inseparable ever since. In 2021, Hector was diagnosed with metastatic pancreatic cancer, which threw their entire family into navigating a very difficult diagnosis. Nora has taken on the role of Hector’s advocate and caregiver. 

Metastatic pancreatic cancer is one of the hardest to detect and most difficult cancer types to treat when compared to all solid tumor types. Emotionally, metastatic pancreatic cancer can affect daily life, negatively impacting a patient’s psychological and emotional well-being just as quickly as their physical health. Since the diagnosis is serious with an average survival of less than two years, people diagnosed and their loved ones experience stress, uncertainty and a mix of other emotions.

For Hector, the first sign he had that something wasn’t quite right was when he started having problems with his stomach. A visit to the GI doctor revealed spots on his pancreas, which he was told were not a big deal. But, after about three more months and numerous visits to the hospital, one day, Nora noticed that Hector’s skin had a yellow hue. The couple rushed to the hospital, where Hector was diagnosed with stage IV metastatic pancreatic cancer. Speaking to the severity of the diagnosis, Nora said, “we both knew what it meant.” It was devastating news for their family, especially after being told nothing was wrong. 

But, the couple decided they would not give up. “We’re going to fight it. We’re going to do everything we can,” explains Nora. She made it her mission to become Hector’s best advocate by joining him at all of his doctor’s appointments, and keeping a notebook of everything to remember – from names of doctors to ideas about how she could support Hector with nutrition. 

Nora reminds people who are caring for a loved one that it’s important to make time for yourself and maintain your own physical and mental health as a caregiver. She notes that by taking care of yourself, you’re enabling yourself to best support your loved one. For her, she finds respite when listening to music and loves going for hikes to clear her head. 

Although Hector and his family have a very difficult diagnosis of metastatic pancreatic cancer, Nora believes in the power of positivity and offers advice to others going through a similar situation: “Don’t give up. There’s always hope.” 

Learn more about Nora and Hector’s story:

Sabrina is a lover of nature, mother and grandmother, whose outlook on life is to make every moment count. Sabrina lives with primary biliary cholangitis (PBC), which means every day is different depending on the severity of her symptoms, but this doesn’t stop her from doing the things she loves the most. 

Sabrina’s favorite ways to pass the time include family time and enjoying the great outdoors. Growing up in rural Washington state and growing her own family with her husband Jason, the place she calls home is a true source of love and adoration. Sabrina finds herself spending weekends with family camping, hiking, barbequing, and enjoying the natural beauty of where she grew up ─ when she has the energy to do so, and on top of her full-time job and responsibilities as a mother and grandmother. 

“You need to make every moment count,” describes Sabrina on her ambition to live life to the fullest. “I want to live life while I can live it. I want to have those experiences with my family. I want to do those things with my kids and my grandkids. Don’t give up hope.” 

PBC is a rare, progressive, autoimmune cholestatic liver disease in which bile ducts in the liver are gradually destroyed. The damage to bile ducts can inhibit the liver’s ability to rid the body of toxins, and can lead to scarring of liver tissue, known as cirrhosis.¹ The symptoms that impact Sabrina on a daily basis are severe itching (pruritus) and debilitating fatigue. Every day is different but on a typical day, Sabrina lives with symptoms that tend to be invisible to others. 

“It can be downright awful,” Sabrina describes her PBC. “I have upper right quadrant pain quite frequently. I have itching. It can happen during the day. There’s no rhyme or reason.” 

Sabrina also experiences challenges in her personal ability to access care while living in a rural area. Her local hospital does not have any PBC specialists and Sabrina, similar to many other people living with PBC, often finds herself educating healthcare providers on the disease and advocating for herself. When Sabrina needs to see her hepatologist in Seattle, she and her husband take a seven-hour drive from her home. Advocating for herself includes monitoring her symptoms closely, including checking her blood pressure daily.  

Life with PBC for Sabrina has not been easy, but she has developed a support system and leaned on her family and loved ones. Finding a community of other people, especially women, living with PBC has been very important for Sabrina’s journey.  

“I’ve met so many people I would have never known that I love dearly,” shares Sabrina on finding her community of others living with PBC. “They’ve become family.” 

Learn more about Sabrina and hear her story:

This year marks the 17^th anniversary since the discovery of the ACVR1 gene was announced, a major scientific milestone in fibrodysplasia ossificans progressiva (FOP) research that helped us understand what causes the condition. On April 23, we marked FOP Awareness Day to honor this important anniversary, raise awareness of this ultra-rare genetic condition, and celebrate the strength of the FOP community, who face challenges every single day that most of us would find unimaginable. At Ipsen, we recognize the importance of advocating for the unique and complex needs of people living with FOP and although FOP Awareness Day is one very important day, we will never lose sight of the fact that the FOP community deserves our ongoing and long-term commitment every day. In order to succeed in addressing the significant unmet needs that exist, working together with the FOP community is of paramount importance.

We believe it is our responsibility to listen to the first-hand experiences of the FOP community in order to drive positive change. One way we can do this is through studies that truly put those living with FOP at the center of our research, such as burden of illness surveys and natural history studies, which are vital tools to expand our knowledge in rare diseases. Research like this allows us to design clinical trials that explore those aspects of a disease that are the most impactful and relevant to patients, such as disease progression, physical, social, emotional, and quality of life outcomes.

It would be impossible for most of us to understand what someone living with an ultra-rare, chronic, painful and progressive condition like FOP is going through.^1,2 What we do know, however, is that FOP causes ongoing, irreversible bone growth in soft tissues outside of the normal skeletal system, which is known as heterotopic ossification (HO).³ This new bone growth leads to joints becoming immobile, causing progressive loss of movement – many people with FOP need to use a wheelchair by 30 years of age.^2,4 Extra bone growth associated with FOP can also cause the jaw to lock, creating difficulty eating and speaking. Additionally, restrictions in the chest can lead to cardiorespiratory failure and ultimately lead to many people living with FOP to have a reduced life expectancy.⁵

The closest we can get to understanding the realities of living with a rare disease like FOP, is by actively listening to patients about the true impact that it has across every aspect of their lives. By ensuring the patient voice is embedded throughout the end-to-end process of clinical development, including dissemination of findings, the resulting research can be used to bring meaningful change.

Refining rare disease research

Research in rare and ultra-rare diseases brings challenges that are different to those in other disease areas – these include very small patient populations that make it difficult to find individuals who are willing and able to participate in research,⁶ lack of wide-spread disease specific knowledge⁶ and expertise to build upon,^4,7 clinical trial complexity with a lack of known and agreed upon endpoints,⁶ high research and development costs⁸ with a notable failure rate.⁹

To try to break down some of these barriers and better understand the day-to-day realities for people living with FOP, their family members and caregivers, as well as the healthcare professionals (HCPs) who support them, we designed two complementary projects: a prospective, natural history study and a burden of illness survey. Both studies were created in partnership with the FOP community – who not only helped to design them, but who continue to be pivotal as we work to understand and disemminate the insights.

Firstly, the Natural History Study — a first-of-its-kind in FOP. Previously, there was limited research in this area, so the study was designed to more comprehensively describe the natural disease progression and was the first time a global, prospective, longitudinal evaluation of FOP has been carried out, with data collected over a period of 36 months. Findings demonstrated the debilitating impact and progressive nature of FOP, and key insights include:

• The greatest accumulation of new HO occurs during childhood and early adulthood.¹⁰
• The use of aids, assistive devices, and adaptations (AADAs) increased substantially, with more than 90% using at least one new AADA during the study, suggesting the use of AADA could be a real-world indicator of decreased mobility in FOP.¹⁰

As a complement to this, we also partnered with FOP community advisors to carry out the first international FOP Burden of Illness (Bol) survey to capture the multifaceted impact of FOP, including the effect on families and caregivers. The Bol survey revealed the extent of the social, economic and quality of life impact of FOP, with findings demonstrating:

• Progressive loss of joint mobility and function is associated with a considerable, negative impact on quality of life for those living with FOP.¹⁰
• As disability increases over time, the way in which FOP is managed changes, with a greater focus on daily care and support, an increased reliance on assistance from family members for performing daily activities, and limitations in mobility restricting the ability to travel.¹¹
• Individuals with the most severe physical limitations reported quality of life scores substantially worse than those generally reported by people living with diabetes mellitus, cancer, and multiple sclerosis. Results also suggest loss of joint function leads to a negative financial impact due to rising costs as the need for living adaptations, likelihood of changes to career plans, and missed days of work for these individuals and their families increase.¹¹

Driven by the voices of FOP

At Ipsen, our teams are working everyday to support those in the rare disease community. But the only way we can truly begin to comprehend what life is like for someone living with a rare disease, is by asking them. The Natural History Study and the Bol survey were important tools that allowed us to ask questions and listen to the community, to try to understand their experiences. We would like to personally thank the FOP community who helped make these studies impactful and who have provided opportunities for better understanding and the sharing of these powerful insights.

Only through true understanding can we continue to make the right decisions to support the FOP and rare disease communities.

References

1. Baujat, G et al., 2017, Prevalence of fibrodysplasia ossificans progressiva (FOP) in France: an estimate based on a record linkage of two national databases, Orphanet Journal of Rare Diseases, vol.12, no.1, pp.1-9.

2. Connor JM, Evans DA. Fibrodysplasia ossificans progressiva. The clinical features and natural history of 34 patients. J Bone Joint Surg Br. 1982;64(1):76-83.

3. Kaplan FS, et al. 1993, The Histopathology of Fibrodysplasia Ossificans Progressiva. J Bone Joint Surg Am, Vol 75, no. 2, pp. 220-30.

4. Pignolo, R. et al., 2020. Self-reported baseline phenotypes from the International Fibrodysplasia Ossificans Progressiva (FOP) Association Global Registry. Bone, 134, p.115274.

5. Kaplan FS, Zasloff MA, Kitterman JA, et al. Early mortality and cardiorespiratory failure in patients with fibrodysplasia ossificans progressiva. J Bone Joint Surg Am. 2010;92(3):686–691.

6. Neez E, Gentilini A, Hutchings A.  2021. Addressing unmet needs in extremely rare and paediatric-onset diseases: how the biopharmaceutical innovation model can help identify current issues and find potential solutions.  EFPIA, viewed 28 September 2022, https://dolon.com/rare-knowledge/publications/addressing-unmet-needs-in-extremely-rare-and-paediatric-onset-diseases.

7. Qi, Z., Luan, J., Zhou, X., Cui, Y., & Han, J. 2017. Fibrodysplasia ossificans progressiva: Basic understanding and experimental models. Intractable & rare diseases research, 6(4), 242–248. https://doi.org/10.5582/irdr.2017.01055.

8. Jayasundara K, et al 2019, ‘Estimating the clinical cost of drug development for orphan versus non-orphan drugs.’, Orphanet Journal of Rare Diseases, Vol.14, no. 12.

9. Wong CM, et al 2019, ‘Estimation of clinical trial success rates and related parameters’ Biostatistics, Vol. 20, no. 2, pp. 273–286.

10. Pignolo et al. The Natural History of Fibrodysplasia Ossificans Progressiva: A Prospective, Global, 36-Month Study. Genetics in Medicine. 2022. https://doi.org/10.1016/j.gim.2022.08.013.

11. Mukaddam MA. et al. 2022. The impact of fibrodysplasia ossificans progressive (FOP) on patients and their family members: results from an international burden of illness survey, Expert Review of Pharmacoeconomics & Outcomes Research. DOI: 10.1080/14737167.2022.2115360.

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

1. Kaplan FS, et al. The medical management of fibrodysplasia ossificans progressiva: current treatment considerations. Proc Intl Clin Council FOP 1:1-111, 2019.
2. Pignolo RJ et al. The Natural History of Flare-Ups in Fibrodysplasia Ossificans Progressiva (FOP): A Comprehensive Global Assessment. J Bone Miner Res. 2016;31(3):650-656.
3. Kaplan FS et al. Early Mortality and Cardiorespiratory Failure in Patients with Fibrodysplasia Ossificans Progressiva. J Bone Joint Surg Am. 2010;92:686–91.
4. Pignolo RJ et al. Fibrodysplasia Ossificans Progressiva: Diagnosis, Management, and Therapeutic Horizons. Pediatr Endocrinol Rev. 2013;10(2)437-448.
5. IFOPA, What is FOP?, IFOPA. [Last accessed 30 August 2022].  https://www.ifopa.org/what_is_fop

Hear what CEO David Loew has to say about the official close of its acquisition of Epizyme.

Today, we are broadening our commitment to providing everyone with the opportunity to read and understand our research. From July 2022, we will publish, as a minimum, a 250-word plain language summary alongside all company-sponsored journal publications from human studies.

By Will Gattrell

This was made public at the European Meeting of the International Society of Medical Publication Professionals (ISMPP) alongside our findings that show that plain language summaries remain rare, with a prevalence of 61 per million articles listed on PubMed (1).

We believe that developing plain language summaries will make our research more accessible and inclusive (2), thus expanding on our original commitment to publishing our journal articles with open access.

Readers regularly express their desire to read primary scientific literature to educate themselves from a reliable source, and their frustration at how inaccessible it can be. For instance, putting the findings of research in simple words was rated as a priority in a patient survey that we conducted last year and that asked 103 patient organizations worldwide about ways of delivering patient value.

Plain language summaries put complex information into everyday language so that it can be understood by all audiences, including patients, patient advocates, caregivers, healthcare professionals, policy makers, and those whose first language is different to that in which the article is written.

At Ipsen, the Patient Centricity and Global Medical Publications and Communications teams have co-created a process for plain language summary development, which includes review by a panel of non-specialists. The process is in sync with the first published recommendations on how to develop plain language summaries for medical journal publications that we worked on together with Open Pharma last year (2).

Ultimately, plain language summaries can help enhance the conversations between patients and their physicians, which in return can result in improved patient outcomes. Through this move, we hope to encourage the industry to adopt them more broadly.

If you have any questions or would like any further information on our commitment, please contact: GMPC@ipsen.com

References

1. Gattrell W. et al. Prevalence and characteristics of plain language summaries indexed in PubMed. Presented at the European Meeting of the International Society of Medical Publication Professionals Virtual Meeting, January 25-26, 2022.
2. Curr Med Res Opin. 2021 Nov;37(11):2015-2016. doi: 1080/03007995.2021.1971185. Epub 2021 Sep 12.