The FH Europe Foundation (FHEF), the international network of over 30 patient organisations, dedicated to supporting individuals and families affected by familial lipid disorders, has issued an urgent statement to the Federal Joint Committee (G-BA) in response to the decision on ANGPTL3 inhibitor (Evinacumab) for patients with homozygous familial hypercholesterolaemia (HoFH) aged 12 years and older in Germany.
The G-BA is the highest decision-making body of the joint self-government of physicians, dentists, hospitals and health insurance funds in Germany. It issues directives for the benefit catalogue of the statutory health insurance funds (GKV) for more than 74 million insured persons and thus specifies which services in medical care are reimbursed by the GKV. The G-BA conducts also benefit assessment of medicinal products, where it puts every new active pharmaceutical ingredient through an early benefit assessment within six months after it is launched on the German market. During the early benefit assessment, the G-BA examines whether the drug is really something new: if it offers patients greater benefit than comparable treatments that are already available. Click here for more information.
The FHEF statement has been prepared in collaboration with international HoFH patient ambassadors and health care and policy experts and endorsed by the leading scientific society – the European Atherosclerosis Society (EAS). It emphasizes the importance of considering the underlying genetic causes of the disorder and the latest available scientific evidence, while recognising the burden of the disease and the patient lived experiences in health decisions in Germany. The statement underscores the need for personalized care and the recognition of patient safety and health economic arguments in the treatment of HoFH.
Read the statement here.
HoFH is an exceptionally severe and rare form of FH. This genetic disorder characterized by extremely high levels of low-density lipoprotein (LDL) cholesterol due to mutations in specific genes from birth. This condition significantly increases the risk of premature cardiovascular diseases, including atherosclerosis, heart attacks, and early death. Understanding the genetic underpinnings of HoFH is crucial. HoFH results from mutations in genes critical for LDL cholesterol metabolism. Targeted treatments that directly address the genetic cause of the disorder, providing more effective management than traditional therapies. In other words, traditional therapies often fall short in managing this condition, leaving patients with limited and less effective options.
HoFH is not just a medical condition; it deeply affects the daily lives and well-being of patients and their families. The disorder leads to physical disability, social exclusion, and significant psychological stress. Along with strict dietary restrictions, patients with HoFH endure frequent and invasive medical treatments like apheresis (if available) and face economic challenges due to the demanding nature of their care. The need for effective, targeted treatment options is vital to alleviate these burdens and to offer quality of life.
Read the story of Elsie Evans, a person living with HoFH here
Patient stories, like the one of Elsie’s, underscores the importance of quality of life beyond mere treatment. Elsie is not just a patient; she is professional advocate, a teacher, a coffee lover, and a traveller. Through her involvement with organisation like FH Europe Foundation, she aims to create a supportive community where patients uplift each other and improve their well-being. Elsie’s story reminds us that resilience, hope, and scientific progress can transform lives—even in the face of daunting challenges.
The treatment of HoFH necessitates precision medicine, which tailors medical treatment to the individual characteristics of each patient, taking into considerations their genetic mutations as well as sex, age, wellbeing and quality of life, among many other specific factors. Given the genetic complexity of HoFH, treatments must be specifically designed to address these unique genetic profiles.
"I eagerly anticipate the evolution of personalised treatments into practical approaches that prioritise quality over quantity of life. I envision a future where there won't be a need to advocate or share our stories, as the preventative measures we are fighting for now will change the environment into one where we are all equal when it comes to health."
Elsie’s words highlight the importance of individualised treatment, precision medicine, and access to the right therapies for each person. It is a powerful reminder that for patients with chronic, rare and severe diseases living a good quality of life is as important as staying alive and goes beyond mere treatment. It is about holistic well-being and support.
We believe that every patient deserves access to the best possible, personalised treatment options. Given the gravity of HoFH and the limited effective and appropriate innovative treatment options available in Germany, we urge the G-BA to reassess this decision and to prioritize the health and well-being of patients with HoFH in the country.
We stand ready to engage in constructive dialogue to ensure that the needs of HoFH patients are adequately addressed. Thank you for your attention to this critical matter.
Read the statement here.
After several months of fruitful collaboration, we are delighted to announce that FHCARE Singapore will be joining our Network. FHCARE Singapore was founded in 2016, by a small group of clinicians, headed by Professor Tavintharan Subramaniam at Khoo Teck Puat Hospital, Singapore.
The focus for FHCARE Singapore has been providing support for patients living with FH and progressively for patients with elevated Lp(a) and raining awareness of those conditions among the wider public.
Over recent months Prof Tavin and his team have identified patients with both Heterozygous FH and Homozygous FH. However, with an estimated 1 in 140 Singaporeans with HeFH and 1 in 200,000 HoFH, there is much work to do!
FH Europe Foundation team have been supporting FHCARE Singapore with strengthening the patient engagement whilst being mindful of cultural sensitivities. The biggest step to date has been a group video call at the end of June 2024 with FH patients from both organisations coming together to share experiences, culminating in a creation of a dedicated WhatsApp group specifically for FH patients in Singapore.
With a grant secured from the Singapore Ministry of Health, Prof Tavin and his team have the objective of:
In the meantime, the groups have collaborated on a significant research publication in the area of behavioural science and exploring genetic testing acceptance in FH. A joint paper under the lead of Assoc Prof Konstadina Griva (Lee Kong Chian School of Medicine, Singapore) is in the submission phase for publication.
Who Is FHCARE Singapore?
FHCARE Singapore is a dedicated organisation committed to supporting individuals and families affected by familial hypercholesterolaemia (FH). Their mission aligns perfectly with ours—to raise awareness, provide education, and advocate for better care and management of FH. An interesting fact, Singapore is an island country and city-state in maritime Southeast Asia, with a population of almost 6 million people and 3 main ethnic groups (74.0% Chinese, 13.5% Malay, 9.0% Indian, 3.4% other).
Finally meeting in person at the EAS Annual Congress in Lyon, France. From the left: Prof. Anna Abreu, Magdalena Daccord, Emma Print, Elsie Evans, Prof Tavintharan Subramaniam, Dr Wann Jia Loh.
What Does This Mean for Our Community?
With FHCARE Singapore on board, our network expands now to the Asia-Pacific region. Together, we can amplify our joint efforts to improve the lives of people with familial hyperlipidaemias (HeFH, HoFH, elevated Lp(a) and FCS), while learning about specific challenges and opportunities and developing new projects in the new region.
“It is a great honour and privilege for FHCARE Singapore to be part of the FHEF Community. Being part of this joyous and dynamic family where every voice is valued and respected, inspires our team to persevere and optimistically move forward and take meaningful positive steps to improve lives of individuals, families and communities living with FH”. (Prof Tavintharan Subramaniam)
On July 4th, the Hungarian EU Presidency kicked off with a landmark event focused on cardiovascular health (CVH), held at the Ministry of Interior in Budapest. This high-level meeting, organised in collaboration with the European Society of Cardiology, emphasized the need for a dedicated European Action Plan on Cardiovascular Health, as advocated by the European Alliance for Cardiovascular Health (EACH). International key stakeholders, including health ministers, health experts, doctors and researchers, leading health organisations like WHO Europe as well as representatives of EACH partner organisations and patient representatives were present. Representing FH Europe Foundation were CEO Magdalena Daccord, Dr Marius Geanta, Chair of FHEF Policy and Innovation Committee, Nicola Bedlington, Senior Policy Advisor, and Dr Ákos Gábor Gesztes /FH Patient Ambassador from the Hungarian Patient Organization and member of the Network.
Dr. Péter Takács, Hungary's Minister of State for Health, opened the event, followed by insights from Prof. Dr. Franz Weidinger, President of the European Society for Cardiology, and Prof. Dr. Béla Merkely, Honorary President of the Hungarian Society of Cardiology. They highlighted the staggering impact of cardiovascular disease, which costs Europe 282 billion euros annually and is responsible for 40% of deaths in women. The event underscored the critical role of air pollution, a topic addressed at COP 28, and the need for more research—currently, only 4% of clinical trials focus on cardiovascular disease. Despite the commonality and cost of rehospitalization, 80% of premature cardiovascular episodes and strokes are preventable.
Watch this 15min Masterclass in compelling data presentation on cardiovascular diseases burden by Prof. Franz Weidinger. The President of ESC underlines the human cost of CVD, the economic burden, disparities (especially gender) and the need for innovation in research and investment in primary and secondary prevention strategies across Europe.
The meeting featured four expert panels discussing and a Ministerial roundtable. These panels framed cardiovascular health as a "path-finder" and emphasized the importance of trust and data-driven health systems.
A significant segment of the event was a ministerial roundtable with health ministers from Hungary, Ireland, Poland, Romania, and Spain, alongside Dr Hans Kluge, WHO Regional Director for Europe. The panellist had an opportunity to showcase some of the practises already implemented in their respective countries in the effort to tackle CVD. Yet, it was very clear that more should be happening to resolve the issue. The roundtable affirmed the value of a comprehensive European Action Plan on Cardiovascular Health, advocating for an integrated approach that transcends individual policies and emphasizes collaboration across non-communicable diseases and across different branches of the economy, learning from exciting examples like the cancer plan and showcasing synergies which will drive better, long lasting health outcomes.
The event offered a unique opportunity to discuss with high-level stakeholders the needs and the progress and achievements made in the familial lipids’ advocacy space, which echoed in the sessions.
Although inherited CVD risk factors and genetics were not the focus of session 1, it was great to hear a personal account told in the final remarks by Prof. Dr Paul Dandale. Known for his dedication to prevention and interest in familial hypercholesterolaemia (FH), he shared his experience of some close relations affected with FH and his stance on the need for early screening and detection of genetic high cholesterol.
Specific reference to FH Paediatric Screening was made by Prof. Fausto Pinto, Past President of WHF, in session 2, when talking about secondary prevention and tangible examples of successful implementation in some Member States. In the discussion part of the session, Magdalena Daccord addressed the panellist and the audience highlighting the urgency to act and the importance to consider familial lipid disorders as CVD risk factors which affected us all. Considering the prevalence of elevated Lp(a) and FH, there were potentially 30 people in the plenary room affected with either of the conditions. She then asked about the opportunity to leverage already existing experiences and learnings from best practices implementation in countries like Slovenia, Croatia, Cyprus, as the MoH representatives of those nations were present in the room.
Several informal yet important exchanges dedicated to FH and elevated Lp(a) took place over the networking dinner reception on the evening prior to the conference and continued in the breaks on the day. The Hungarian conference, following the Slovenian technical meeting in 2021, and the Czech Senate meeting in 2022, marks the third EU Presidency high-level event and an important milestone in the mission to prevent the preventable.
The FH Europe Foundation delegation, equipped in Prague Declaration and with PERFECTO FH and the Lp(a) international Taskforce projects as examples, advocated tirelessly for childhood FH screening and was raising awareness of elevated Lp(a) as another independent CVD risk factor requiring urgent attention, actions and investment.
The discussions on the day, both on stage and in the corridors, reinforced the necessity for a standalone cardiovascular health plan, given its profound human and financial impacts. The event concluded with a commitment to make Europe a global leader in cardiovascular research, management, and care, with the emphasis on cardiovascular health. This ambitious vision requires political leadership, belief, and courage to implement a coherent and effective plan.
The Budapest historic event marks a significant stride towards a healthier future for Europe, underscoring the collective commitment to tackling cardiovascular disease through comprehensive and innovative strategies.
The European Cardiovascular Health Plan builds on the ambitions and vision of EACH and provides a strategic framework for Europe and an incentive for developing all-important national plans to tackle early CVD detection, CVD prevention, timely and appropriate treatment to prevent dire complications, increased investment in CVD research and rehabilitation.
The event in photos:
FH Europe Foundation is thrilled to welcome Stitching VrouwenHart from The Netherlands to be part of the FHEF Network of patient organisations.
VrouwenHart is a leading non-profit organisation dedicated to women’s heart health in the country, run by volunteers.
Why This Matters
Cardiovascular disease remains the leading cause of death globally, affecting millions of lives each year. Shockingly, women are often underrepresented in research, diagnosis, and treatment. VrouwenHart recognises this disparity and is committed to bridging the gap.
FHEF and VrouwenHart have worked together closely for a few years. On the occasion of the FH Awareness Day 2023, VrouwenHart Founder Caroline Verhage formed part of the expert panel of international female speakers for our webinar “Misdiagnosis and Gender Bias in Women with FH”.
Caroline established VrouwenHart in 2016 after her own experience and misdiagnosis for SCAD (spontaneous coronary artery dissection). She wanted to help other women self-advocate and get the treatment they need sooner. With Caroline’s relentless work and the support of a growing team, VrouwenHart now has over 17,000 followers and has helped numerous women across Europe. In fact, it is through VrouwenHart that we have one of our board members – Annelies Dol!
VrouwenHart’s Mission
VrouwenHart, which translates to “Women’s Heart,” has been at the forefront of raising awareness about heart disease in women. Our mission is threefold:
Together, Engaged for Change!
This collaboration demonstrates our joint commitment to making women’s heart health and geneder differences more prominent on the international cardiovascular health agenda, with a special focus on familial lipids like FH, elevated Lp(a) and FCS.
Join Us on This Journey
We invite everyone—patients, healthcare providers, researchers, and policymakers—to join us on this critical journey. Together, we can create a future where women’s hearts are protected, and CVD is no longer the silent killer it once was.