Congenital Heart Disease Priority Setting Partnership
Congenital heart disease is the most common type of birth defect, affecting 13 children born every day in UK, with many requiring one or more open heart operations or interventions.
In recent years, outcomes have continued to improve and nowadays around 97% of children born with congenital heart disease are expected to survive into adulthood. However many develop late complications related to their condition or previous surgery/interventions and almost all require life-long follow-up.
In 2021-22, the James Lind Alliance Congenital Heart Disease PSP brought together patients, parents, charities, and clinicians to establish national clinical priorities for research in children and adults with congenital heart disease. The two Top 10 lists of priorities were launched at the BCCA annual conference in November 2022 and published in Open Heart.
A national strategy to address these priorities has been developed, with endorsement from professional bodies and national charities, and supported by NHS England Congenital Heart Disease Clinical Reference Group and the Women and Children’s Programme of Care. It includes establishing the Congenital Heart Research Network, a UK & Ireland collaborative network for multi-centre studies including clinical trials, and a national Congenital Heart Disease Patient and Public Involvement group.
The Congenital Heart Disease Priority Setting Partnership brought together parents, adult patients, charities, and clinicians from across both the spectrum of congenital heart disease and the UK to identify priorities for research. It was a collaboration between the Children’s Heart Federation, The Somerville Heart Foundation, the British Congenital Cardiac Association, and the Society for Cardiothoracic Surgery, was hosted by the Institute for Cardiovascular Sciences at the University of Birmingham. It was funded by George Davies, the high street fashion entrepreneur behind brands such as Next, Per Una, and George at Asda, through a generous donation from the George Davies Charitable Trust to the Birmingham Children’s Hospital Charity.
The PSP focused on the management of congenital heart disease throughout life, including prior to birth, in three areas of the patient and families experience:
- Diagnosis, during pregnancy or after birth.
- Treatment: medical therapy, catheter intervention, surgery including mechanical support & transplantation, lifestyle, or psychosocial intervention.
- Outcomes of the conditions and/or treatments and the impact on patients and their families, including the physical, psychological, and social effects of living with congenital heart disease.
We did not look at areas which fall outside of these, such as the causes of congenital heart defects, acquired heart disease in those without a congenital heart defect, or aspects of congenital disorders that do not affect the heart.
Stage 1: Establishing the partnership
Using the methodology established by the James Lind Alliance, the Congenital Heart Disease PSP sought to identify and prioritise evidence uncertainties or ‘unanswered questions’ relating to the management of congenital heart disease in the UK. We therefore established a steering committee of parents, adult patients, national charities, and clinicians from across the UK and engaged with a wide range of partner organisations to raise awareness of the PSP through their members and supporters.
Stage 2: Initial survey
The initial survey sought to identify evidence uncertainties through a consultation process with all stakeholders, and asked one simple question: What questions would you like to see answered by research, relating to the diagnosis, treatment, or outcomes of congenital heart disease?
The survey was available online and in PDF format from June-October 2021 and was widely promoted via social media, partner mailing lists, and newsletters.
The steering committee specifically identified South Asian communities as being disproportionately affected by congenital heart disease and experiencing worse outcomes from interventions, whilst being underrepresented in consultations. With input from the Centre for Ethnic Health Research at the University of Leicester, the PDF survey was available in Bengali, Urdu, Gujarati, and Hindi, in addition to English, Welsh and Polish.
The initial survey received 524 responses proposing 1,373 individual questions.
Stage 3: Evidence checking
The survey responses were analysed, removing any out-of-scope questions and those already answered in the literature, to generate a long-list of unanswered but answerable summary questions.
These were split into two parallel tracks, with 56 uncertainties related to children/antenatal care and 47 uncertainties related to adults with CHD.
Stage 4: Prioritisation surveys
Two follow-up surveys to prioritise the identified uncertainties were conducted between March-May 2022, receiving 250 responses to the child/antenatal survey and 252 responses to the adult CHD survey.
The 26 most high-ranked unanswered summary questions in each track were taken forward to the workshops.
Stage 5: Priority setting workshops
Two workshops were held in June 2022, one child/antenatal and one adult, bringing together patients, parents, charities, and healthcare professionals, with a range of conditions/expertise from across the UK and Ireland, and facilitated by the James Lind Alliance. Consensus was reached relatively quickly at both workshops and two final Top 10 lists of national research priorities were agreed.
Post-PSP: Developing a national strategy to address the priorities
After completion of the PSP, we engaged with partners and funders to disseminate our findings. The two Top 10 lists of priorities were launched at the BCCA annual conference in November 2022 and published in Open Heart.
We developed a national strategy to address the priorities including the establishment of the Congenital Heart Research Network, a UK & Ireland collaborative network for multi-centre studies including clinical trials, and a national Congenital Heart Disease Patient and Public Involvement group. We are now working to implement this strategy through multi-centre clinical trials and other studies to improve the clinical care and day-to-day lives of children and adults born with congenital heart disease.
The Congenital Heart Disease PSP was conducted through the Institute of Cardiovascular Sciences at the University of Birmingham, in conjunction with Birmingham Children’s Hospital Charity, and overseen by the James Lind Alliance.
Key partner organisations:
- Children’s Heart Federation
- Somerville Heart Foundation
- British Congenital Cardiac Association (BCCA)
- Society for Cardiothoracic Surgery in Great Britain and Ireland (SCTS)
Other partner organisations:
- Little Hearts Matter
- Tiny Tickers
- Max Appeal
- Birmingham Children’s Hospital Charity
- Young at Heart
- Paediatric Critical Care Society (PCCS)
- Congenital Cardiac Anaesthesia Network (CCAN)
- Paediatricians with Expertise in Cardiology Specialist Interest Group (PECSIG)
- Society of Clinical Perfusion Scientists (SCPS)
- Congenital Cardiac Nurses Association (CCNA)
- British Adult Congenital Cardiac Nurse Association (BACCNA)
- Primary Care Cardiovascular Society (PCCS)
- BHF Clinical Research Collaborative (BHF-CRC)
- Centre for Ethnic Health Research at the University of Leicester
The Congenital Heart Disease PSP was overseen by a steering group comprised of parents, adult patients, national charities, paediatric and adult cardiologists, congenital cardiac surgeons, and a paediatric cardiac intensivist, along with two information specialists, supported by an administrator and chaired by a Senior Advisor from the James Lind Alliance.
Patient and carer representatives:
Julie Wootton, Parent and Chair of the Children’s Heart Federation
Julie is chair of trustees for Max Appeal, for people with 22q11DS, which she established following the death of her son in 1999. She is chair of trustees of the Children’s Heart Federation, a trustee of CCP-UK, the patient arm of the British Cardiovascular Society, and Genetic Alliance UK.
Michael Cumper, former Patient and President, Somerville Heart Foundation
Michael Cumper was President and a Trustee of the Somerville Heart Foundation. Prior to becoming President, he was Vice President and Chairman of the charity for many years. He was a patient with congenital heart disease and represented the Somerville Heart Foundation on a number of committees including being a member of the NHS Standards Group that wrote the Standards for the care of adults with congenital heart disease for England. He was an outspoken advocate for patients and sadly passed away in July 2022.
Jara Weinkauf, patient with congenital heart disease
Jara was born with a severe congenital heart defect called Tricuspid Atresia. After multiple openheart surgeries, she knows first-hand, how difficult it can be to navigate all things surgery, doctor visits, keeping a positive spirit, and most of all staying independent and active. She is eager to contribute the experience gained growing up with a heart condition and hopes to make life easier for fellow cardiac patients.
Fraser Pender, patient with congenital heart disease
Fraser is an adult patient with congenital heart disease, born with transposition of the great arteries. He underwent a ’switch’ procedure as a newborn and a balloon septostomy at 1 years of age. Although he leads a healthy and sporty lifestyle, he has to undergo yearly medical examinations and will require a heart valve replacement in the future.
Sarah Murray, Parent and Chair of the BHF Clinical Research Collaborative Cardiac Surgery PPI group
Sarah is the chair of the National Cardiac Surgery Clinical Trials Initiative PPI Group, and is also the mother of a son with a congenital heart defect. She is passionate about finding answers to some of the fundamental questions patients ask.
Alex Miskin, Parent of child with congenital heart disease
Alex is passionate about improving the treatment and research into Congenital Heart Disease as the Mother of Evie Miskin who was born with the congenital heart condition, Truncus Arteriosus. Evie has undergone heart surgery to separate her aorta and pulmonary artery. This surgery happens every few years as she has grown or the conduit requires replacement. Along side these interventions she has had many catheter procedures and has had stents fitted due to the narrowing of her arteries, all under the care of the fantastic team at Birmingham Children’s Hospital, which has allowed her to thrive.
Sasha Rooprai, Parent of child with congenital heart disease
Sasha is parent of a child with a heart condition called complete heart block (at stage 3) She is passionate about creating awareness of heart disease through different projects. She is also a friend of the ‘ Young at Heart Charity.’
Mr Nigel Drury, Clinician Scientist and Consultant in Paediatric Cardiac Surgery, Birmingham Children's Hospital and PSP National Lead
Mr Nigel Drury is Consultant in Paediatric Cardiac Surgery at Birmingham Children’s Hospital, and Clinician Scientist and BHF Intermediate Clinical Research Fellow at the University of Birmingham. He is Chief Investigator of the only two multi-centre randomised clinical trials in children’s heart surgery in the UK (BRICC, DESTINY).
Prof John Simpson, Consultant Paediatric and Fetal Cardiologist, Evelina London Children’s Hospital, and President, British Congenital Cardiac Association (BCCA).
Professor John Simpson studied medicine at the University of Edinburgh. He trained in children's and fetal cardiology at Guy’s Hospital, London and at the University of California, San Francisco, USA. He has been a consultant at Evelina London Children’s Hospital since 1999 and leads the echocardiography service. He is interested in the use of advanced imaging of congenital heart disease during fetal life, including 3D imaging of the heart.
Dr Katherine Brown, Consultant in Cardiac Paediatric Intensive Care, Great Ormond Street Hospital, London
Katherine Brown is consultant in Paediatric Cardiac Intensive Care at Great Ormond Street Hospital and Associate Professor and Lead for Children’s Heart Diseases Research at UCL Institute of Cardiovascular Science at Great Ormond Street Hospital. Katherine has an interest in health services research related to: registry or population based analyses for paediatric cardiac surgery and congenital heart disease, and longer term disease impacts such as morbidities, psychosocial or neurodevelopmental outcomes and quality of life.
Dr John Thomson, Consultant Interventional Cardiologist, Leeds Teaching Hospitals
John is an experienced interventional cardiologist working in the field of congenital heart disease. His unit is large and he has performed many thousands of catheter procedures on children and adults in a variety of clinical settings.
Mr Rafael Guerrero, Chief of Congenital Cardiac Surgery, Alder Hey Children’s Hospital, Liverpool, and Chair, Congenital Cardiac Surgery sub-committee, SCTS
Rafael is an international leader in Paediatric and Adult Congenital Cardiac Surgery, as well as a trainer, innovator and advocate and promoter of the use of advanced technology in healthcare. He is Chief of Congenital Cardiac Surgery and Director of Cardiac Services at Alder Hey Children’s Hospital, and an Honorary Professor at the University of Liverpool. He is also co-founder of the award-winning Alder Hey Innovation Centre and as the Director of Innovation has been instrumental in establishing the vision and long-term strategy. He is the current Chair of the Congenital Committee of the Society for Cardiothoracic Surgery.
Dr Louise Coats, Consultant Adult Congenital Cardiologist, Freeman Hospital, Newcastle
Dr Louise Coats is an Adult Congenital Cardiologist at the Freeman Hospital, Newcastle upon Tyne and Adult Lead for the North East and North Cumbria Congenital Heart Disease Network. She leads the Congenital Heart Disease Research Group, with sits as part of the Reproduction, Development and Child Health Theme at Newcastle University. She is interested in outcomes for adults with congenital heart disease, in particular understanding what matter to patients, how this should inform care provision and how we better detect problems early.
Dr Clare Herd, Information Specialist, Institute of Applied Health Research, University of Birmingham
Clare has worked for the last ten years as an information specialist and systematic reviewer. Most of her work has used Cochrane methods to produce comprehensive reviews of interventions for a variety of conditions.
Dr Giovanni Biglino, Senior Lecturer in Cardiovascular Bioinformatics, University of Bristol
Giovanni is a bioengineer with an interest for interdisciplinary research and patient and public engagement and involvement in research. He is currently Senior Lecturer in Biostatistics at the Bristol Medical School, Honorary Clinical Senior Lecturer at Imperial College London and a Future Leader in Innovation Enterprise and Research (FLIER) at the Academy of Medical Sciences.
Sharmaine Afferion, Administrator, Institute of Cardiovascular Sciences, University of Birmingham
Sharmaine has worked at the Institute of Cardiovascular Sciences for 4 years. She is involved in all the social media aspects of the PSP.
James Lind Alliance:
Katherine Cowan, Senior JLA Advisor and Chair of the Steering Group
Katherine is Senior Adviser to the James Lind Alliance (JLA) and is chair of the Congenital Heart Disease Priority Setting Partnership (PSP) Steering Group. She has been a key contributor to the development of the JLA method.