Haematology specialty profile
Why you should delivery haematology research in the UK
Haematology studies often bring a unique set of recruitment challenges, yet the life-sciences industry need research studies to be set-up and delivered quickly, effectively, to target and within agreed timelines. The UK is uniquely placed to meet this need.
Our uniqueness stems from the way that our research leadership and workforce is organised to deliver and evaluate care under one common National Health Service (NHS). This is underpinned by us, the Clinical Research Network. We provide researchers with the practical support they need to make research happen - making the UK the optimum base for high quality clinical trials and research programmes in haematology.
We also bring together communities of clinical practice, called specialties, to provide national networks of research expertise. The Haematology Specialty is one of 30 such specialties and is made up of leading research-interested clinicians and practitioners at both national and local levels. Our job is to ensure that the haematology studies that are included in our national portfolio of research receive the right support to ensure they are delivered successfully.
We offer a number of services to life-sciences companies which include advising about aspects of study design, particularly regarding patient numbers and the likely location of those patients. We can provide:
Our UK-wide network of leading academic clinicians is experienced in the efficient delivery of high quality studies in haematology. Our Specialty covers the following sub-specialties:
- Bleeding Disorders
- Immune thrombocytopenic purpura
- Immuno haematology
- Obstetric Haematology
- Paediatric haematology
- Paroxysmal Nocturnal Haemoglobinuria
- Platelet Disorders
- Red cell disorders
- Sickle-cell disease
- Thrombotic microangiopathy
Access to patients
The UK is the only country in the world that has a comprehensive haemophilia database which maintains a record of patients with bleeding disorders. Our focus on haematology has enabled us to collaborate with the National Haemophilia Database. This means that we can advise on the incidence of bleeding disorders, as well as the location of patients around the country. Such information is anonymised, but provides the intelligence we need to aid in the feasibility assessment of new commercial studies investigating novel therapies in this patient group. So, for example, we can compare the National Haemophilia Database against study inclusion/ exclusion criteria and within 24-48 hours we can determine the approximate number of potentially eligible patients in the UK. We can also identify where those patients are treated, and check if those centres are already undertaking similar haemophilia studies that might compete for patients locally and affect the success of both studies.
The UK has an outstanding unified system for the clinical care of haemophilia patients. All patients attend a small number of treatment centres coordinated by the UK Haemophilia Centre Doctors’ Organisation (UKHCDO) – another organisation that we collaborate with. This means that all potentially eligible patients are already known to investigators. This is a major advantage to the commercial companies that choose to use our research support services.
We work closely with our colleagues that make up the Children’s Specialty within our Network structure to support the recruitment of children into haemophilia research studies.
Our local teams of research staff are specialists in the recruitment and retention of patients in haematology studies, and in engaging families and carers.
We have dedicated research support staff to assist with study delivery at site level. We also offer a range of practical services that are designed to help you set-up and deliver commercial research studies to time and target, these include:
- Feasibility assessment services
- Site identification
- Performance monitoring
- Costing templates
- Model Clinical Trial or Investigation Agreements
Over 6,300 patients were recruited into Haematology studies in 2018/19.
The Haematology Specialty delivered 79 per cent of commercial studies to time and target in 2018/19 with 95 acute trusts recruiting a patient into a Haematology study.
Since 2015, the Haematology Specialty has achieved six global first recruitment of participants to studies, demonstrating our ability to quickly and effectively set up studies.
TRAIT was a research study to describe the real world use of Thrombopietin-Receptor Agonists in the management of Immune Thrombocytopaenia in the UK. The study ran from 2016 to 2018 and utilised the early contact and engagement, site intelligence and performance monitoring services provided by the NIHR CRN.
The study recruited 244 participants across 16 UK sites, to time and target, with the involvement of the HaemSTAR network of Clinical Haematology registrars, who are supported by the NIHR.
More information is available in the Blood Journal.
BMN 270-301: Gene therapy study in severe haemophilia A patients
This study is a phase 3, open-label study to evaluate the efficacy and safety of BMN 270. BMN 270 is an adeno-associated virus vector-mediated gene transfer of human factor VIII in haemophilia A patients. The study is open and has exceeded its recruitment target of 10 participants, ahead of time in six NHS sites with support from the NIHR CRN.