Making ENT research count
Ear, Nose and Throat (ENT) research in the UK has gone from strength to strength over the last decade. It has transformed from what was once considered a specialty with a deficit in high quality research, to one that has a thriving research community and a national research strategy. Insight Monthly talks to two key researchers who are driving recent developments in this area.
“The aim is to define the right question and best approach to answer that question to make the research really count”
The Network’s ENT specialty covers a wide range of conditions ranging from life-threatening infections and cancers of the head and neck, to hearing loss and conditions such as Meniere’s disease (see casestudy), the impact of which on patients’ life are perhaps less well known. One person who fully understands the magnitude of ENT conditions and the importance of high value research is Professor Anne Schilder, Director of evidENT at the Ear Institute, University College London (UCL), and ENT National Specialty Lead for the NIHR Clinical Research Network. A practicing clinician for more than 30 years, Professor Schilder (pictured above) recalls a time when clinical research was lower on the ENT agenda:
“As an ENT trainee I was fortunate to work in several European countries with different health systems; the UK, Netherlands and Belgium. This sparked my curiosity – why are ENT infections in children treated differently in these countries? I realised that one of the reasons was that there was little evidence to guide doctors and parents in making decisions about the management of these conditions.
“At that time, the 1990’s, there was a shortfall in high quality clinical research in ENT, compared to other medical areas. Many ENT treatments involve surgery and this reflected what was happening in surgery in general. Surgeons have busy clinical practices and often little time for research. Many felt that running clinical trials in surgery was too complicated. This means there were few surgeons who could set an example and teach others how to develop and deliver surgical trials. In ENT I became one of few surgeons with expertise in area.”
Anne began a programme of clinical trials looking at the benefits of common ENT operations in children while she was working as a clinician at the University Medical Centre Utrecht. In 2011 she was invited to join UCL to set up a programme of clinical trials in ENT here in the UK. In that same year, she was awarded an NIHR Research Professorship. She describes how research activity in ENT, Hearing and Balance has grown during that time:
“It has been wonderful to experience how the NIHR has invested to promote both ENT and surgical research. The Clinical Research Network has played a key role in getting research on the surgical agenda…”
“It has been wonderful to experience how the NIHR has invested to promote clinical research in both ENT and surgery in general. The Clinical Research Network has played a key role in getting research on the surgical agenda.
“Awareness raising was the first step. Initially people were just not aware of the support available for research within their local Clinical Research Network and trust. The benefits of having a study adopted onto the Clinical Research Network Portfolio and what this can offer patients – having access to new and potentially better treatments – were not widely known.
“When I took the role of ENT National Specialty Lead we worked hard as a group to share our knowledge and expertise of conducting research within the NHS. As a result, ENT surgeons and audiologists began to design more and better studies and submit them to receive Network support. We also got better at delivering these studies, so our study performance statistics began to improve.
“In parallel we engaged proactively with the life-sciences industry. Traditionally companies would go to a local ENT surgeon to trial a new product. Now they see the benefits of being able to broaden a study to more sites via the Network. Being on the NIHR Portfolio also is perceived as a quality stamp for commercial studies. It demonstrates that the study is of high quality and of value to the NHS, which helps these companies get more surgeons and sites on board. This has attracted more companies to the UK. Our group has worked hard on this, the Network is now the default route for commercial ENT research.”
So with both non-commercial and commercial research firmly on the agenda how do the ENT Specialty’s stats look? Over the last six years (since 2009/10) the number of ENT studies on the Clinical Research Network Portfolio has steadily increased by 75 per cent. And since Clinical Research Network records began in 2008/09, almost 100,000 patients have been recruited into ENT studies supported by the Network.
Now, in September 2016, the ENT research community is taking their collegial approach to the next level. Under the guidance of Professor Schilder, Natalie Bohm, ENT Clinical Academic Lecturer, and member of the UCL evidENT team, has led the creation of a strategic research agenda for the field. She explains:
“Professor Schilder entered discussions about developing a national research agenda for ENT, Hearing and Balance with tour professional organisations: ENT UK (which is the professional body for ENT surgeons), the British Academy of Audiology, and the British Society of Audiology. As a result the jointly-funded GENERATE project came into being with the aim of patients and professionals working together to promote a strategic approach to high value research in our field.
“I was asked to lead a research priority setting exercise to inform the agenda and took advice from the NIHR-funded James Lind Alliance . It was a huge undertaking which took almost two years to complete. We made sure all key stakeholders were on board: patients, charities, health professionals, researchers, service commissioners, managers and research funders.
“We collected the views of more than 700 patients and professionals via a survey asking what research questions they want answered, and why. We also held five focus groups to discuss ideas in more depth. We analysed the more than 1000 research suggestions by categorising them to topics and themes. A group of independent experts helped us narrow this long list down to 99 research ideas; the final process of setting the 21 research priorities took place during the GENERATE Consensus Conference in September last year.”
‘The Research Agenda for ENT, Hearing and Balance Care ’ was launched at a public event in December 2015. Natalie feels privileged to have been involved in a project that brings patients, clinicians and researchers to the same table:
“It was unique to see patients and professionals equally represented. What struck me was how keen people have been to share their ideas and opinions, working in this collaborative space with everyone committed to the same end goal was a real honour. All groups agreed to work together to ensure that future research is patient-centred and addresses the issues that matter to patients and those who care for them.”
Professor Schilder echoes this point:
“The agenda unearthed real questions and concerns that people have; these go beyond ‘What is the best treatment for this condition?’. Examples of priorities are: ‘What is the impact of ENT, hearing and balance conditions on people and the society?; What is the best patient pathway; how can patients self-manage their condition and what can be managed in the community rather than in a hospital?’. Many of the priorities relate to ageing and the impact of sensory loss – hearing, balance taste and smell.”
But creating a research agenda is just the beginning. Professor Schilder concludes by looking to the future:
“Now the ENT, Hearing and Balance communities need to deliver the agenda. People are organising workshops, bringing together the groups involved in setting the priorities to take a topic from the agenda and develop initial research proposals. The first workshop is in September looking at implantable hearing aids. We will work on defining the right question and best approach to make the research really count.”
“Of course the next important step is to get the research funded and delivered. The NIHR and other research funders have been very involved throughout the initiative and have been very supportive. We, as a specialty within the Clinical Research Network, will have an important role to play in delivering the studies once they hit the NIHR Portfolio.”
Casestudy: Clinical trial progress in Meniere’s disease
There is currently no medical cure for Ménière’s disease – a rare disorder affecting the inner ear which can cause crippling vertigo, tinnitus and hearing loss. When Colin, from Norfolk, was diagnosed with the condition in 2014, he experienced weekly bouts of dizziness where he was pinned to the floor, unable to move or stop himself from being sick. This could often go on for hours, leaving Colin exhausted and bedridden for days.
Having been told that the condition is currently incurable, Colin didn’t know how he would be able to cope:
“I thought, ‘how am I going to carry on working? I could lose my job, my livelihood and all as a result of the condition’.”
But joining a pioneering clinical research trial delivered by the Clinical Research Network, across the UK has helped him overcome his condition, he recently celebrated over a year free of symptoms.
Recognising that the NHS currently has limited treatment options for Meniere’s disease, a doctor at his local GP surgery suggested he go to see the ENT team at the Norfolk and Norwich University Hospital to see if he would be able to take part the trial funded by life-science company Otonomy. Colin was found to be eligible and was offered the opportunity to take part.
After receiving just two of the intended four injections of a new drug called OTO-104 in the affected ear, Colin no longer suffered from any dizziness or related sickness at all. This good health has now continued for well over a year since the trial concluded. Colin says:
“I’m so grateful for what’s happened. This research trial has changed my life. I’ve had nothing but the best of health since participating in the clinical trial…”
“I’m so grateful for what’s happened. This research trial has changed my life. I’ve had nothing but the best of health since participating in the clinical trial.”
Mr John Phillips, Consultant ENT Surgeon leading the study at the Norfolk and Norwich University Hospital, acknowledges the impact of research and the importance of patients getting involved:
“The success of the trial may profoundly increase our understanding of Ménière’s disease but this success hinges on the willingness and courage of participants such as Colin, who we are extremely grateful to for getting involved”.
Following the completion of the trial which Colin took part in, further research is now underway at NHS Trusts across the UK and in Europe and the United States to gather further data on the effects of OTO-104 and establish if the drug could be of value to patients within the NHS in the future as a potential treatment for Meniere’s disease.
Carl LeBel, Chief Scientific Officer at Otonmony, believes that the Network has played a positive role in this research programme to date and is keen to explore further opportunities to partner with the Network’s ENT specialty:
“The partnership with the NIHR CRN ENT was essential in being able to announce recently the successful completion of a one-year, multiple-dose clinical safety trial of OTO-104 in Ménière’s disease that was conducted in the UK. Based on this success, we have expanded the CRN ENT collaboration as part of an ongoing Phase 3 clinical trial (AVERTS-2) designed to assess the effectiveness of OTO-104 in Ménière’s disease patients that is being conducted in multiple European countries.”