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Author: Louise Stanley

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Advancing UK Regulatory Science Strategy in the Context of Global Regulation: a Stakeholder Survey

Written by Louise Stanley on . Posted in Birmingham Health Partners.

New study published in Therapeutic Innovation & Regulatory Science today (17th February 2021) from the Birmingham Health Partners Centre for Regulatory Science and Innovation

Background

The UK’s transition from the European Union creates both an urgent need and key opportunity for the UK and its global collaborators to consider new approaches to the regulation of emerging technologies, underpinned by regulatory science. This survey aimed to identify the most accurate definition of regulatory science, to define strategic areas of the regulation of healthcare innovation which can be informed through regulatory science and to explore the training and infrastructure needed to advance UK and international regulatory science.

Methods

A survey was distributed to UK healthcare professionals, academics, patients, health technology assessment agencies, ethicists and trade associations, as well as international regulators, pharmaceutical companies and small or medium enterprises which have expertise in regulatory science and in developing or applying regulation in healthcare. Subsequently, a descriptive quantitative analyses of survey results and directed thematic analysis of free-text comments were applied.

Results

Priority areas for UK regulatory science identified by 145 participants included the following: flexibility: the capability of regulations to adapt to novel products and target patient outcomes; co-development: collaboration across sectors, e.g. patients, manufacturers, regulators, and educators working together to develop appropriate training for novel product deployment; responsiveness: the preparation of frameworks which enable timely innovation required by emerging events; speed: the rate at which new products can reach the market; reimbursement: developing effective tools to track and evaluate outcomes for “pay for performance” products; and education and professional development.

Conclusions

The UK has a time-critical opportunity to establish its national and international strategy for regulatory science leadership by harnessing broader academic input, developing strategic cross-sector collaborations, incorporating patients’ experiences and perspectives, and investing in a skilled workforce.

Read the full study on the publication website (opens as PDF).

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Funding boost to research multimorbidity in hospital patients

Written by Louise Stanley on . Posted in Health inequalities, Inflammation and chronic disease.

Scientists have been given almost £4m to improve understanding of multimorbidity in hospital patients.

Research led by Newcastle University will focus on multiple long-term conditions in hospitalised patients and is funded by the Medical Research Council (MRC) and National Institute for Health Research (NIHR).

The four-year ADMISSION study aims to transform understanding of how different long-term conditions cluster or group together, why people are affected more often by some groups of conditions than others, and how hospital systems look after them.

NHS pressures

The number of people who have more than one long-term health condition, known as multimorbidity, is growing. This is increasing pressure on healthcare providers, such as the NHS, as these patients have complex needs – often staying in hospital for longer and taking more time to recover.

However, hospital systems are designed to treat single health conditions. Patients with multimorbidity often find their care is inefficient and unsatisfactory. For providers, this inefficiency translates into more costly care and potentially worse outcomes.

To find new ways to deliver hospital care, experts want to understand how long-term health conditions develop, and why particular conditions occur in groups.

Despite the recognised importance of multimorbidity in hospital patients, there has been little research in this area to date.

Professor Miles Witham, Deputy Lead for NIHR Newcastle BRC’s Ageing Syndromes theme, is co-investigator of the ADMISSION study.

He said: “We are delighted to have received this substantial award from the MRC and NIHR to fund our research.

“The results of ADMISSION will provide a springboard for developing, testing and delivering novel approaches to transform care for people with multiple long-term conditions before, during and after admission to hospital.”

ADMISSION will use cutting-edge data science, computing and statistical approaches to analyse ‘big data’ from routinely-collected healthcare records, along with information from the UK Biobank and the Scottish Health Research Register (SHARE).

Its focus will be on clusters of conditions: to describe how they occur across the population, to understand the mechanisms that explain them, and to examine their impact on patient pathways through healthcare.

Future care

This knowledge will inform the design of future care and treatments, with potential both to reduce costs, and to improve health outcomes for the millions of patients with multimorbidity admitted to hospital each year.

Newcastle University is leading the study, collaborating with Newcastle Hospitals NHS Foundation Trust, University of Birmingham, Manchester Metropolitan University, University College London and the University of Dundee.

Professor Liz Sapey, Professor of Acute and Respiratory Medicine at BHP founder-member the University of Birmingham, said: “As a doctor working in acute medicine, I see many people admitted with multiple long-term conditions.

“Currently, we do not understand how and why some illnesses cluster together, and so do not know the best ways to care for these patients.

“Our expertise in using ‘big data’ from patient records through our PIONEER Health Data Hub here in Birmingham forms an important part of the ADMISSION programme.

“Describing how these illnesses cluster together, as part of the ADMISSION collaborative, is a crucial first step to improving care for patients with multiple long-term conditions.”

For more information, visit: www.admissioncollab.org

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New med-tech partnership to assess quality of life in clinical trials and care

Written by Louise Stanley on . Posted in Clinical trials, Health inequalities.

BHP founder-member the University of Birmingham has today announced a partnership with med-tech company Aparito to co-develop digital platforms to assess patient quality of life and symptoms in clinical trials and routine NHS care.

The partnership will see the configuration of Aparito’s flagship software platform Atom5™, and brings together international experts in patient-reported outcomes (PROs) methodology and input from patients and clinicians, with cutting-edge and innovative technology.

The aim of the partnership is to co-develop multiple digital PROs for use in a wide range of disease groups to assess treatment safety and effectiveness from the patient perspective and enhance the patient experience of clinical trials and routine care. These data will support patient care and provide evidence to inform regulators and policy makers such as the Medicines and Healthcare products Regulatory Agency (MHRA), and the National Institute for Health and Care Excellence (NICE).

Professor Melanie Calvert, National Institute for Health Research (NIHR) Senior Investigator and Professor of Outcomes Methodology at the University of Birmingham’s Centre for Patient-Reported Outcomes Research (CPROR), said: “It is essential that we capture information on the impact of disease and treatment on patient symptoms and quality of life.

“This information can help regulators decide if a treatment is safe and effective and answer important questions from patients such as ‘how will it make me feel?’

“We are delighted to be partnering with Aparito to use cutting edge methodology and technology to advance this area and benefit patients.”

Dr Elin Haf Davies, CEO of Aparito, which is based in Wrexham in the UK and Leiden in the Netherlands, said: “We are highly honoured to enter into this partnership with the CPROR at the University of Birmingham.

“Professor Melanie Calvert and her team are highly regarded and international leaders in PRO methodology. We very much look forward to expanding on this work to provide a digitalised and personalised solution, in 2021 and beyond.”

One of the projects is the new National Institute for Health Research Surgical Reconstruction and Microbiology Research Centre (NIHR SRMRC) ‘PRiORiTy’ (Patient Reported Outcomes Research in Trauma) study.

In this study, the team of experts at BHP founder-members the University of Birmingham and University Hospitals Birmingham NHS Foundation Trust will assess patient symptoms following traumatic brain injury to help tailor care to patient needs.

This is an important issue for patients, as explained b ypatient advocates involved in the design of the study, Luke and Jackie Flavell: “We feel it is really important for patients to report symptoms of traumatic brain injury as early as possible and doing this electronically would save valuable time and improve patient care. We are very much looking forward to working with CPROR on the PRiORiTy study.”

Daniel Lewi, Head of Business Development at Aparito, added: “Working with the team at University of Birmingham to provide a technology solution for PRiORiTy has highlighted how deeply clinicians care about the patient experience and how they can improve treatment within the patient cohort.

“Having such an approachable and knowledgeable team has allowed the University of Birmingham to detail very specifically how we can adapt our Aparito Atom5™ technology to really change a patient’s life and we cannot wait to work with the team again on future projects.”

Clinical trial confirms digoxin is effective for treatment of atrial fibrillation

Written by Louise Stanley on . Posted in Clinical trials, Inflammation and chronic disease.

A clinical trial has shown that digoxin has the same effect on physical wellbeing as beta-blockers when used to treat patients with permanent atrial fibrillation and symptoms of heart failure.

Beta-blockers have long been the drug of choice for controlling rapid heart rates in patients with atrial fibrillation (AF), but a clinical trial led by Professor Dipak Kotecha of Birmingham Health Partners has shown that digoxin is just as effective, but with less adverse effects.

The ‘Rate Control Therapy Evaluation in Permanent Atrial Fibrillation’ (RATE-AF) trial was the first of its kind to compare the effectiveness of digoxin and beta-blockers to treat AF. Beta-blockers, such as bisoprolol, are one of the most common groups of drugs used in clinical practice to reduce heart rate and improve pump function. Digoxin primarily works to slowly improve the contraction of the heart but also has other broad range effects which at low-dose can potentially be helpful to counter the body’s response to AF and heart strain, and is usually only used when other treatments are unsuccessful.

The RATE-AF trial showed there was no difference in physical wellbeing between digoxin and beta-blockers and there was no difference in the effect on long-term heart rate between the two drugs. Importantly, digoxin at low dose was found to cause substantially and significantly less adverse effects than beta-blockers, lessened the impact of AF on the daily lives of patients by improving symptoms, and reduced a marker of heart strain, natriuretic peptide.

AF is caused by disorganised electrical impulses firing from different places in the top chambers of the heart and patients usually require medication to control an irregular heartbeat. Patients can also have a reduced quality of life, be admitted to hospital more frequently and have a higher chance of strokes and heart failure.  This trial, embedded in the NHS, involved 160 patients aged 60 or older. It has addressed a major evidence gap in the management of patients with permanent atrial fibrillation. The research team will plan a larger trial to see if digoxin can reduce hospital admissions in this patient group.

Chief Investigator Professor Kotecha said: “I hope that the results of this trial show the importance of randomised clinical trials to see how treatments actually work. On behalf of the research team and all the patients who designed and took part in the RATE-AF trial, we are delighted to show that digoxin is a drug that can be used to improve the lives of patients with AF.”

The trial was publicly funded by the National Institute for Health Research (NIHR). RATE-AF was coordinated by the Institute of Cardiovascular Sciences at BHP founder member the University of Birmingham, a Patient & Public Involvement Team and the Birmingham Clinical Trials Unit. Patients and staff involved in the trial were from BHP founder member University Hospitals Birmingham NHS Foundation Trust as well as Sandwell and West Birmingham Hospitals NHS Trust and local General Practitioners.

‘Weak’ and ‘strong’ cells bonding boosts body’s diabetes fight

Written by Louise Stanley on . Posted in Inflammation and chronic disease.

Scientists have broadened our understanding of how ‘weak’ cells bond with their more mature cellular counterparts to boost the body’s production of insulin – improving our knowledge of the processes leading to type 2 diabetes.

Type 2 diabetes mellitus occurs when β-cells cannot release enough insulin – a tightly controlled process requiring hundreds of such cells clustered together to co-ordinate their response to signals from food, such as sugar, fat and gut hormones.

An international research team – led by scientists at BHP founder-member the University of Birmingham – have discovered that immature β-cells (PDX1LOW/MAFALOW) are able to overcome their relative deficiencies by partnering with ‘stronger’ counterparts to drive insulin release.

Publishing their findings in Nature Communications, the researchers reveal that subtle differences in the levels of PDX1 and MAFA proteins (found only in β-cells) , and more broadly, differences in β-cell maturity, contribute to how clusters of insulin-producing cells, known as islets, function.

The corresponding author David Hodson, Professor of Cellular Metabolism, at the University of Birmingham, commented: “Our research shows that differences in β-cell maturity, defined using PDX1 and MAFA levels, are needed across the islet for proper insulin release. Unexpectedly, increases in the proportion of mature β-cells, is associated with islet failure. It seems that, rather like society, the islet needs cells with all ages to be properly functional.

“Redressing the balance between immature and mature β-cells restores islet function under conditions of metabolic stress – an excess of sugar and fat in the diet – providing evidence that both ’weak’ and ‘strong’ β-cells could contribute to proper islet function and insulin release.”

“This is the first glimpse that immature cells might contribute to the regulation of insulin release across the islet. Our study indicates a promising line of investigation that could be leveraged to make islets more resilient during type 2 diabetes or when generating new islets in a ‘dish’ for the purpose of transplantation.”

Normally, mature and immature β-cells co-exist within the adult islet and can be grouped into subpopulations according to differences in their levels of specific genes and proteins. Immature β-cells are generally considered to be poorly functional when viewed alone, as single cells.

Researchers found that islets containing proportionally more PDX1HIGH and MAFAHIGH β-cells showed defects in cell function (metabolism, ionic fluxes and insulin secretion). The team believes maintaining a mix of ‘strong’ and ‘weak’ β-cells is important for effective insulin production.

Birmingham secures National Training Centre for the Advanced Therapies Skills Training Network

Written by Louise Stanley on . Posted in Experimental medicine.

The National Horizons Centre, RoslinCT and the University of Birmingham have been selected to deliver high impact physical and digitally-delivered training courses as part of the growing ATSTN programme. The three centres bring with them complementing capabilities and a vast wealth of experience across GMP/GxP, manufacturing and bioprocessing, and their expertise within virtual reality training will prove instrumental for driving the successful development of cell and gene therapy as well as vaccine manufacturing staff across the UK, through the delivery of these industry-leading training courses.

The core aim of the Advanced Therapies Skills Training Network (ATSTN) is to develop National Training Centres to deliver specialist on-site courses, including innovative digital training utilising virtual reality, providing learners with the hands-on expertise and experience to succeed in the advanced therapies and vaccine manufacturing sector. The ATSTN programme also includes an Online Training Platform focused on upskilling existing staff within the industry and a Career Converter which measures an individual’s transferable skills from outside the sector and recommends applicable roles within advanced therapies and vaccine manufacturing.

Professor Phil Newsome at BHP founder-member the University of Birmingham and Professor Ivan Wall at Aston University said:
“We are excited that Birmingham will play a central role in delivering these much-needed skills for the UK’s advanced therapies sector. This will ensure the UK retains a world-leading position in the manufacture and delivery of advanced therapies. Moreover, it will further strengthen and leverage the rapidly-growing health and life science sector in Birmingham and the Midlands.”

Mayor of the West Midlands, Andy Street, commented: “Hosting an ATSTN National Training Centre in Birmingham is testament to the expertise we have developed in advanced therapies for the benefit of patients with complex conditions.  Developing  the skills to enable the therapies of the future, including new vaccines, to be delivered to patients is critical to our region and nation. And as well as leading on training, the University of Birmingham is proposing the development of new clean room facilities at Birmingham Health Innovation Campus, providing businesses with access to the critical infrastructure and expertise they need to develop, commercialise and manufacture at scale.

“To capitalise on this opportunity, extend Birmingham’s position as a leading player in advanced therapies, and deliver on the Government’s goal to be an international leader in the field, we will be seeking further support to help the delivery of innovation infrastructure in this Campus.”

Matthew Durdy, Chief Executive Officer at Cell and Gene Therapy Catapult commented: “The involvement of the National Horizons Centre, RoslinCT and the University of Birmingham is a major step in the effective development and rollout of impactful training courses which will enable the ATSTN to upskill and attract talent into the advanced therapies and vaccine manufacturing industry. It is also indicative on how the ATSTN is a truly collaborative initiative which continues to be developed in partnership with industry and academia, and the valued expertise from these three centres will provide users access to in-person training centres which complement each other and the wealth of online resources already provided on the ATSTN platform. I look forward to witnessing the great strides which will be made over the course of next year.”