Patient Empowerment
Featuring Managing Pain, Skin Health and Children’s Health
“Despite being chronic, it is possible to lead a fulfilled life with psoriasis.”
Tassneem Miah, Communications Lead, The Psoriasis Association Page 04
“Trials are designed without reflecting the realities of children’s lives or involving families early enough.”
Maria Cavaller Bellaubi, Patient Engagement & Therapeutic Development Director, EURORDIS Page 06
VTS affects up to 15-35% of twin pregnancies.
~Olivia Bowen, TV Personality and Content Creator Read more on Children’s Health (Pages 06–08)
Photo credit: James Rudland
A community-first approach to bridging the diagnostic gap
Chronic musculoskeletal conditions, such as osteoarthritis and persistent, undiagnosed joint pain, are a leading cause of disability and healthcare utilisation in the UK.1
Delays in diagnosis, particularly for infectious arthritis, can result in prolonged pain, missed windows for early treatment and an overburdened secondary care system.
Faster arthritis diagnosis breakthrough OrthoDx are seeking to address this challenge with a transformative diagnostic platform: Synchovior. This synovial fluid test uses biomarker analysis to rapidly distinguish inflammatory arthritis from other types of joint pain, offering a clear diagnostic direction at the point of care.
In a European-first, the Norfolk and Norwich University Hospital (NNUH) has become the first in Europe to trial a Synovial Fluid Triage test in a clinical setting. The evaluation is being led by leading consultant rheumatologists and supported by the National Institute for Health and Care Research (NIHR).
Early results are promising, showing that the test has the potential to reduce unnecessary admissions to hospitals, provide faster access to appropriate care and significantly reduce demand for beds and the costs associated with care.2
Diagnostics support NHS and community care
The real-world deployment of Synchovior at NNUH (Norfolk Norwich University Hospital) illustrates how innovations in diagnostics can be safely integrated into NHS services, starting in an A&E setting, but with clear scalability into primary and community care.
With most joint pain patients initially presenting to A&E with undiagnosed underlying causes, empowering frontline clinicians with tools like Synchovior can cut waiting times, improve triage accuracy and enable earlier intervention, especially in underserved areas.
By shifting diagnostics closer to the patient, healthcare professionals and policymakers can support a patientcentred, community-first model of care. Synchovior provides not only clinical insight but also system-wide efficiencies
— minimising hospital visits, optimising referrals and aligning with the NHS Long Term Plan’s focus on integrated and preventative care.
Data-driven healthcare transformation
As the burden of chronic conditions continues to rise, early and accurate diagnosis is the gateway to better outcomes. The Government’s recent announcement of a £600 million investment in a new Health Data Research Service — aimed at centralising NHS data to accelerate medical research and improve access to new treatments and technologies — underscores the critical role of integrated efforts in advancing patient care.
By aligning with such initiatives, we aim to ensure that innovations like Synchovior are effectively integrated into the healthcare system, ultimately improving outcomes for patients with chronic musculoskeletal conditions.
References:
Understanding the person with chronic pain
Pain affects 28 million people in the UK1 and can affect every part of life. Here, three voices show how research, healthcare and lived experience drive change together.
The British Pain Society’s (BPS) new strategy is championing care that’s multidisciplinary, research-informed and person-centred.
Pain science meets lived experience
Pain research must be shaped by those it aims to help. Dr Kirsty Bannister, Associate Professor of Pain Neuroscience, says: “Basic science is vital to understanding the mechanisms of pain, such as how the nervous system encodes and maintains chronic pain. In Parkinson’s disease, for example, linking sensory dysfunction to neurodegeneration allows us to target therapies more effectively, but scientists can’t operate in isolation. Involving people affected by pain helps us shape research questions that reflect real-life concerns. Only by connecting preclinical science with patient experience can we build the future of personalised pain care.”
Women’s pain gap
Katy Vincent, Professor of Gynaecological Pain, adds: “Pain is staggeringly common in women. Yet, historically, women’s pain has been dismissed or seen as psychogenic. More than a third of teenage girls report moderate or severe period pain, and up to half of post-menopausal women develop painful osteoarthritis. Biological differences between sexes mean we need to understand what treatments work for women and ensure early access. Recognising and addressing this gap is crucial — not only for the individual, but for families, the workforce and wider society.”
Equity-driven pain care reform
Amirah Ashouri, member of BPS’ Expert Patient and Carer Committee, puts this all into context: “The BPS validates lived experience by giving us a voice in shaping pain management. Having chronic pain since childhood, I’ve experienced the limitations of care focused just on symptoms. A diagnosis of fibromyalgia in my 20s led to holistic, person-centred NHS care that empowered me to reclaim quality of life. Yet, access to such support remains inconsistent. By working alongside a wide range of healthcare professionals, we’re shifting thinking, promoting equity and driving meaningful change.”
Our strategy recognises pain as a serious public health issue and commits to equity, evidence and empathy, plus a vision for care that no one living with pain should feel unseen or unsupported.
Chronic pain: medicine’s silent struggle
Chronic pain affects an estimated 28 million people in the UK,1 yet alongside patients’ suffering lies another hidden burden: clinicians struggling to manage an increasingly complex condition.
Pain UK’s survey of clinicians at the 2024 Royal College of GPs Annual Conference revealed frustration and helplessness, tempered by empathy, as dominant experiences in managing patients with chronic pain. Many report emotional exhaustion and isolation, knowing they often lack the tools and resources to ease their patients’ suffering adequately. This relentless pressure contributes significantly to clinician burnout, creating an unsustainable cycle that ultimately diminishes patient care.
Worrying gap between clinical guidelines and everyday practice
A worrying gap exists between clinical guidelines and everyday practice. Recent surveys highlight a crisis of confidence among healthcare professionals tasked with managing chronic pain. Despite updated guidelines promoting holistic, multidisciplinary approaches, clinicians often feel ill-equipped and under-trained. Pain management remains severely underrepresented in medical training, leaving many professionals unsure how best to approach patient care beyond medication. Consequently, providers are left juggling conflicting pressures: adhering to best practices versus managing patient expectations and practical constraints.
Relationships in crisis
WRITTEN BY Jo Brown Executive Director, British Pain Society
Chronic pain’s invisibility frequently strains patient-provider relationships. Patients can feel misunderstood, disbelieved or dismissed, eroding trust and making meaningful dialogue increasingly difficult. Clinicians, on their part, frequently express exasperation and guilt, sensing
they fail their patients despite best intentions. Consultations can devolve into emotionally charged encounters, worsening outcomes for both patient and provider. Yet, evidence consistently demonstrates that strong, empathic relationships significantly enhance chronic pain outcomes, highlighting the critical need for continuity, compassion and open communication.
Systemic changes to address chronic pain
Clinicians urgently need better training, equipping them with confidence and competence in managing complex, chronic conditions. Crucially, the healthcare system must prioritise genuine multidisciplinary approaches, enhancing timely access to specialists, psychological support and physiotherapy services. Redefining chronic pain management requires investing in relationships, safeguarding continuity of care and fostering open dialogue between clinicians and patients. By confronting these hidden burdens and investing in tangible support for healthcare providers, we can transform chronic pain care into a more sustainable, compassionate and effective process for all.
Reference 1. BMJ Open. 2016 May 25;6(6):e010364. doi: 10.1136/ bmjopen-2015-010364.
WRITTEN BY Ashley I. Simpson Consultant Peripheral Nerve Surgeon, Pain UK
The misunderstood mental and physical impact of psoriasis
Psoriasis is a chronic condition resulting from an overactive immune system. Affecting the skin and joints, this long-term condition can affect all aspects of one’s day-to-day life.
Psoriasis is an immunemediated inflammatory disease (IMID), meaning the immune system is not functioning correctly. In psoriasis, the immune system is overactive, causing symptoms on the skin and sometimes joints. Despite being chronic, it is possible to lead a fulfilled life with psoriasis, overcoming the challenges the condition brings.
Symptoms and types of psoriasis
When thinking of psoriasis, dry, itchy skin and plaques may come to mind. Whilst plaque psoriasis is most common, it is not the only type. Guttate psoriasis affects younger individuals as well as rarer, severe forms like generalised pustular psoriasis (GPP) and erythrodermic psoriasis, which requires urgent medical attention. Psoriatic arthritis (PsA) is a chronic condition associated with psoriasis, affecting the joints and causing pain, swelling and stiffness.
Day-to-day impact of psoriasis
From uncomfortable clothing, impacting one’s self-esteem, to restless sleep or painful movement, living with psoriasis or PsA can negatively impact quality of life. The encouraging news is that many over-the-counter treatments are available to manage these symptoms. This may include topical
creams, pain relief or psychological support available via the NHS. Louis, who lives with psoriasis, shares: “Anyone with psoriasis knows it’s not just about managing the physical pain and discomfort, but also the mental toll. I went from being confident and outgoing to someone who shied away from social situations out of fear of being stared at or mocked for my skin. But now, I’ve learned that you can’t control what others think, and you certainly shouldn’t hide yourself away because of it.”
What support is available?
Your GP should be the first point of contact if you notice changes to your skin or pain in your joints. They can provide first-line treatments or a dermatologist or rheumatologist referral. If the condition affects your mental health, your GP can explore options such as medication or talking therapies. While there is currently no cure for psoriasis, with the right treatment and advice, you can live well with the condition.
The unseen story behind living with psoriasis
How do you manage a condition that is visible but also invisible; living with symptoms daily, but people only sympathise when they can see it? That’s what it’s like to live with psoriasis.
Psoriasis is a condition that is so used to being characterised by the skin element that when the skin is clear, people struggle to understand that the symptoms, effects and mental toll never leave.
Let’s talk about psoriasis
Psoriasis can be so easily dismissed as just a ‘skin problem’. While, yes, the patches appear on the skin; it isn’t the whole condition. In fact, psoriasis is classified as a chronic, autoimmune condition due to how the immune system mistakenly attacks healthy skin cells, creating the patches. It is important that we break down these misconceptions in order to not only understand the chronic illness better but also the impact that it has on those living with the condition.
Psoriasis is classed as an chronic, autoimmune condition due to the immune system mistakenly attacks healthy skin cells creating the patches.
Invisible yet itchy condition
While so many parts of psoriasis can be visible, the invisible urge of the itch is, in my opinion, one of the worst parts of the condition. It is something that people who don’t live with the condition don’t seem to realise: my skin is constantly irritated, whether I am covered in psoriasis or clear. In fact, having lived with the condition for 10 years now, I have no recollection of what it feels like to go a whole day without having the urge to scratch and tear at my skin.
The stares are worse after Covid-19
One thing that I have noticed since Covid-19 is that people stare more at the patches on my face. Perhaps wondering if my skin is contagious or not. Perhaps wondering how I could leave the house looking the way I do. It makes the prospect of a flare even worse. So, try not to stare. We understand the curiosity, but we aren’t contagious — we’re just incredibly itchy.
The more we raise awareness of our chronic conditions, the more we can learn to live with them in society and not have to silently suffer.
WRITTEN BY Tassneem Miah Communications Lead, Psoriasis Association
WRITTEN BY Jude Duncan
Psoriasis and Psoriatic Arthritis (PsA) Advocate
Mohs micrographic surgery: gold standard care for skin cancer
Experts consider Mohs surgery the most effective treatment for skin cancer, with up to a 99% cure rate and minimal scarring.
Mohs micrographic surgery is a specialised procedure that accurately removes skin cancers on the face in a single appointment. At the forefront of this advanced treatment is our team at St John’s Institute of Dermatology at Guy’s Hospital, where we have been treating skin conditions for over 150 years.
Gold standard treatment for skin cancer
Mohs surgery is seen as the gold standard treatment for basal cell carcinomas (BCC) and squamous cell carcinomas (SCC), the two most common types of non-melanoma skin cancer.
BCC grows slowly, while SCC grows faster and may spread, but both can be successfully treated with Mohs surgery. In almost all cases, BCC and SCC are both caused by UV radiation from exposure to the sun.
What is Mohs surgery?
“Mohs surgery offers the highest cure rate and least scarring for treating facial cancers,” explains Dr Rakesh Patalay, consultant dermatologist and dermatology surgeon. “Our team is highly trained in this technique, with Guy’s Hospital performing the most Mohs procedures of any centre in the country.”
How long does Mohs surgery take?
Mohs surgery offers the highest cure rate and least scarring for treating facial cancers.
Mohs surgery was first developed in the 1930s and involves removing the carcinoma layer by layer. Each sample is analysed by our dedicated on-site laboratory to determine whether any cancer is present. If any cancerous cells remain, the next layer of tissue is removed and examined the same way, with the process repeated until the surgeon reaches a layer with no cancerous cells.
The processing of each layer of tissue can take 60-90 minutes, so patients should be prepared to spend several hours in the clinic. With our dermatology specialists dedicated to the patient for the entire day, they can return home just hours later, confident that the cancer has been completely removed.
World-renowned dermatology experts
Our Mohs surgery specialists work at Guy’s Hospital, which is home to St John’s Institute of Dermatology, a world-renowned centre for treating skin conditions. At Guy’s, we have the largest Mohs centre in the UK, and we’re proud to now bring that expertise to our state-of-the-art, private facilities at 77-79 Wimpole Street in the Harley Street Health District. To find out if Mohs surgery is right for you, speak to our team on 020 3808 0083 or email gstt.PrivatePatientEnquiries@nhs.net
Newborn screening for rare conditions needed now
Just weeks ago, the World Health Assembly (WHA) passed a resolution for rare diseases.
The text noted that diagnosis of a rare condition can take over five years, and some never get a diagnosis; around 70% of rare conditions are genetic in origin; and nearly half of these start in childhood.1
United for rare disease equity
The WHA recognised ‘that insufficient screening programmes, including newborn screening and unequal access to diagnostic services, infrastructure and expertise contribute to delayed diagnosis and management.’
Rare Diseases International developed a global coalition of stakeholders, 280 strong, to support this resolution, bringing together people living with rare conditions and their families, clinicians, researchers, industry, government ministries, NGOs and others.
Recognising the benefits of early diagnosis
The overwhelming support for ‘recognising that early identification can prevent the onset of disease symptoms or delay the progression of both common and rare diseases, thereby reducing child mortality and morbidity, improving the quality of life of persons living with a rare disease and conferring
families, their caregivers and society as a whole’ was astounding, with this resolution passing unanimously.
Global push for newborn screening
This resolution shows global solidarity for change. There is a groundswell of support internationally for newborn screening, early interventions and pre-symptomatic treatment to save lives and give the best quality of life to those with life-long conditions. In the UK, a treatment must be available before a condition can be added to the newborn blood spot test. However, for many conditions, even if there are no treatments, narrowly understood, there are early interventions which would increase quality of life.
Early screening supports informed families
Complex conditions can present in a range of ways, with families being ping-ponged around the health system, costing money, time off work, impacting relationships and mental health. Newborn screening can also help people make informed family planning decisions around future children.
Newborn screening is a human right
I sit on the Lancet Commission for Rare Diseases, and we are framing our upcoming report through the human rights lens, building on the 2021 U.N. Resolution on Rare Diseases. Newborn screening is a human rights issue. Babies have a right to be diagnosed and receive early interventions and treatment to achieve the best life possible.
Reference:
1. World Health Organization. 2025. Seventy-eighth World Health Assembly – Daily update: 24 May 2025.
Project improves access to medicines for children
Across Europe, too many children with rare diseases are still waiting for effective treatments. The conect4children project has laid the foundations for a faster, more inclusive research system.
Across Europe, children, particularly those living with rare diseases, still face major barriers in accessing the medicines they need.
Urgency of rare disease research
A disease is considered rare when it affects fewer than one in 2,000 people, but with over 6,000 rare diseases identified, they collectively affect at least one in 20 people. Around 75% of rare diseases affect children, and 70% have their onset during childhood — yet most still lack approved, effective treatments.1
Clinical trials for children are often delayed due to fragmented infrastructure, limited expertise and regulatory hurdles. Too often, trials are designed without reflecting the realities of children’s lives or involving families early enough. The result is slower research, lower enrolment and missed opportunities for innovation.
Clinical trials for children are often delayed due to fragmented infrastructure, limited expertise and regulatory hurdles.
What c4c changed
The conect4children (c4c) project — a public–private partnership funded under the Innovative Health Initiative — set out to change that. Over seven years, this large-scale collaboration brought together over 200 sites in 21 countries, creating a coordinated, patientcentred model for paediatric research. EURORDIS – Rare Diseases Europe was a key partner in ensuring the voice of children and families helped shape trial design from the outset.
C4c established national hubs, expert advisory groups and trial support services across Europe. It launched a centralised feasibility and site identification service, as well as standardised data and training protocols tailored to paediatric needs. It also embedded young people and caregivers as co-designers — not just participants — in the research process.
Among its achievements are the creation of the foundation c4c Stichting, a Supervisory Board and a fully operational IT platform. Strategic partnerships with five major companies, cooperation agreements with 15 countries and the processing of 24 trial service requests show its scale and readiness.
A legacy to build on
As the project concludes, its challenge now is sustainability. With the right support, c4c’s legacy can serve as a lasting blueprint for faster, smarter and more inclusive medicines development for Europe’s children.
Reference 1. European Commission. Rare Diseases and European Reference Networks. Rare diseases.
WRITTEN BY Kirsten Johnson Chair of the Council, Rare Diseases International
WRITTEN BY Maria Cavaller Bellaubi Patient Engagement & Therapeutic Development Director, EURORDIS
Rare diseases affect millions of children in Europe:
how do we deliver the treatments?
We must address the challenges of expanding the reach of medicines for rare childhood diseases in Europe. Too many children are unable to access new medicines. Here’s how we can fix it.
Rare diseases, including childhood cancers, are a major cause of death for children. Rare diseases affect up to 36 million people in the EU;1 three-quarters are known to affect children,2 and about 3 in 10 children affected by a rare disease will die before their fifth birthday.3 Only 5% of rare diseases have effective treatments, leaving most without options.4 A fifth of children with cancer will not survive5 and up to 90% of survivors live with the burden of late effects.6
Turning new science into actual medicines
Scientific innovation is constantly advancing the standard of medicines for rare childhood diseases, but securing equitable access is challenging and complex. Many of the best new medicines are developed by small biotechnology companies that focus on cutting-edge science but lack specific expertise and resources to
get their medicines widely approved and distributed.7 Many of these companies are US-based, and there is a significant delay in paediatric medicines reaching patients in the rest of the world.
At Norgine, we know that local expertise is vital, especially in Europe. As a mid-sized pharma company, we are able to partner with small biotechs to bring much-needed medicines to Europe, a process that needs an innovative mindset. It’s important to recognise that innovation does not end in the laboratory. It involves navigating the complex and divergent healthcare systems in Europe to find ways to bring medicines to patients.
Innovation across the whole healthcare process
It is tempting to think that a lifesaving children’s medicine will naturally make its way to patients, but this is not the reality. Many obstacles occur long after scientific innovation. Medicines for children present
unique challenges. There are, rightly, special ethical considerations for children in clinical trials; achieving the best risk-benefit balance is extremely important. Trials in rare diseases are especially difficult due to small patient numbers, drawn from diverse groups over wide geographies.8
To solve all of these challenges means working with multiple patient organisations and scientific centres of excellence in line with various nuanced medical guidelines.
Paediatric approvals face complexity Navigating Europe’s paediatric drug approval processes can be particularly complicated. The new EU Health Technology Assessment (HTA) process and national HTA processes are complex and require different clinical data and study designs, depending on the healthcare system and country. National health insurance structures vary; clinical data is interpreted based on specific country healthcare system requirements, and there are differing regulations and reimbursement criteria.
To add to the complexity, paediatric medicines must go through additional regulatory steps, requiring preapproval of detailed plans studying the effects in children of different ages9 — which is time-consuming but vital for safety. Each step must be supported by state-of-the-art manufacturing and local distribution to guarantee consistent quality and supply.
Children’s health can’t afford delays None of this can be taken for granted. Consequently, many promising treatments for children face significant delays or may never reach patients at all in parts of Europe and beyond. Understanding the path, however complex, is the first step towards finding solutions. Norgine’s agility and our deep regional expertise make us uniquely placed to navigate these processes. We’re proud to do so because the children we serve don’t have time to wait.
References:
1. European Commission. 2025. Rare diseases.
2. Murdoch children’s research institute. Rare genetic disorders.
3. National Stem Cell Foundation. Rare Childhood Diseases.
4. US Government Accountability Office. Rare Disease Drugs: FDA Has Steps Underway to Strengthen Coordination of Activities Supporting Drug Development.
5. Lam CG, et al. Science and health for all children with cancer. Science. 2019;363(6432):1182–86.
6. National Cancer Institute. Late Effects of Treatment for Childhood Cancer(PDQ®)–Health Professional Version: General Information About Late Effects of Treatment for Childhood Cancer.
7. Vital Transformation. 2020. The US Ecosystem for Medicines. How new drug innovations get to patients.
8. Nony P, et al. A methodological framework for drug development in rare diseases. Orphanet J Rare Dis. 2014;9:164.
9. Zisowsky J, et al. Drug Development for Pediatric Populations: Regulatory Aspects. Pharmaceutics. 2010;2(4):364–88.
WRITTEN BY
Janneke van der Kamp CEO, Norgine
Vanished, but
not forgotten:
the impact of vanishing twin syndrome
During a routine scan, Olivia Bowen, and her husband Alex, were told one of their twins was no longer developing—a condition Olivia later discovered was vanishing twin syndrome (VTS).
“Ididn’t even know there was a name for it,”
Olivia recalls. “It happens when you’re carrying multiples and one baby stops developing early on. The tissue is then reabsorbed into the body. There’s often no physical sign—no bleeding, no trauma— which makes it more difficult. It is like they were never there, but they were.”
A hidden loss in early pregnancy
VTS affects up to 15-35% of twin pregnancies,1 though many cases go undetected. With earlier and more frequent scans, the condition is becoming more recognised. “I had a scan at five weeks and saw both babies,” she explains. “Even at 12 weeks, the second baby was visible— smaller, but there. Some people never know, and that’s part of what makes it so hard to process.”
experiencing early pregnancy loss. “Charities like Tommy’s and Footprints are incredible, and the NHS offers a certificate of recognition, no matter how early the loss. That kind of validation means a lot.”
She also emphasises the importance of compassionate care. “At one appointment, someone congratulated me on having twins after I had lost one—as I left, I had a panic attack. Another nurse attempted to change my notes, so I would not keep reliving my loss. The way that was dealt with, how she was thinking of me the whole time, that changed my experience.”
I felt so upset that I had lost one, but so happy I still had a healthy baby. It’s overwhelming.
Grieving what was, and what could have been The emotional toll can be profound. “You begin picturing life with two babies; then that vision is gone. It’s a complicated grief—I felt so upset that I had lost one, but so happy I still had a healthy baby. It’s overwhelming.”
Olivia acknowledges the support available for those
Breaking the silence
By sharing her story, Olivia hopes to incite conversation. “I never could have imagined how many people this has affected. I’ve had countless messages from women—some in their sixties— who had never told anyone before. The response was unbelievable, and I am so glad people could speak to me.”
Reference: 1. https://www.sciencedirect.com/science/article/abs/pii/ S1521693422000487
Newborn screening can also help people make informed family planning decisions around future children.
~Kirsten Johnson, Chair of the Council, Rare Diseases International