Discover how direct engagement with patients, facilitated by the NIHR, has had a positive impact on Servier’s phase II trial design.
Published: 09 April 2021
The NIHR Patient Engagement in Clinical Development Service connects life science companies with patients who want to help shape and improve the design and delivery of commercial clinical research.
Sjögren’s Syndrome is a rare autoimmune condition that affects moisture-secreting glands in the body, characterised by symptoms such as a dry mouth and dry eyes. Although it can affect anyone at any age, it is most common in women aged 40-60 and may be associated with hormones or other immune conditions such as lupus and rheumatoid arthritis.
Symptoms can range in severity and can seriously diminish patients’ quality of life. As Sjögren’s Syndrome is a long-term condition with no cure, treatment involves alleviating symptoms with the aim of limiting the complications associated with the condition, such as eye and lung problems. It can be difficult to diagnose as there are a number of other conditions that share common symptoms with Sjögren’s Syndrome. A lip biopsy is sometimes required to confirm the diagnosis.
A potential new drug, being developed by Servier, to improve the treatment of symptoms of Sjögren’s Syndrome is currently in the early stages of trials. This is the first ever phase II trial for a possible new treatment for Sjögren’s Syndrome that Servier has conducted. Researchers at the company wanted to understand the condition from a patient perspective and gain patient input on the trial design to ensure the research meets the needs of the intended participants.
Patient Engagement Activities
Back in January 2020, Servier co-hosted the first of two patient engagement sessions in partnership with the NIHR. The first session was a face-to-face discussion, similar to a focus group, attended by a diverse group of seven Sjögren’s Syndrome patients, a carer, researchers from Servier and facilitators from the NIHR.
The sessions encouraged open discussion about personal experiences of living with the condition and their perceptions of the disease. The patients also discussed what they would need or like from a potential future new drug. Patients and carers were asked to input on specific aspects of the trial design such as the tests involved and whether the length and number of clinical visits would be feasible for them if taking part in the trial. They also provided insight into how they would like the results to be measured during the trial, compared to how their symptoms are currently monitored by their clinicians.
In addition to the face-to-face session, patients and carers also fed back on the wording and content of the accompanying Informed Consent Form (ICF) document for the trial. This session was conducted via telephone as it took place when national lockdown restrictions were in place due to the coronavirus pandemic.
Ken and Carol attended the patient engagement session and are actively involved in a support group for people with Sjögren’s Syndrome. Carol suffers from the condition and is supported by her husband Ken. They were both keen to be involved to help others who may have the condition now and in the future.
“In terms of Sjögren’s, it’s probably too late for us now to be involved in any major discovery, that’s for the next generations, and as we’ve seen, people in the generations beneath us are suffering in just the same way. It would be great if there was some sort of a breakthrough that would help them avoid the position we’re in.”
Accessible transcript for the video 'Patient perspective: NIHR facilitates patient input into Servier’s first Sjögren’s Syndrome trial'.
The patients and carers were forthcoming with their ideas to improve and adapt the trial design to the needs of Sjögren’s Syndrome patients. As a result of their feedback, a symptom diary is now being considered as a way to monitor symptoms throughout the trial. Patient and carer input on the ICF document has also been taken into consideration and visual elements have now been added to help to convey the protocol and aid participant understanding.
Life science company perspective
In light of patient and carer feedback, researchers are looking at ways to make the trial more flexible. For example, researchers are now considering splitting site visits into two shorter days to accommodate those with fatigue, which is a symptom of Sjögren’s Syndrome. However, the most significant impact on the trial design is that lip biopsies will be made optional rather than mandatory. Although lip biopsy is seen as a major burden to participation due to the uncomfortable procedure, it is an important clinical measure of the impact of the treatment. Having the flexibility of an optional lip biopsy is something that patients and carers felt strongly about and making these changes will certainly help to improve recruitment and adherence to the trial.
Changes to the protocol design made in light of patient and carer feedback will aim to improve the accessibility of the trial, increasing the diversity and number of participants recruited. These changes will also improve the feasibility of the trial and help to limit participant dropouts from the study, ultimately improving the probability of answering the research question successfully.
Marta, R&D Chief Patient Officer at Servier, attended the patient engagement sessions and really valued the direct patient contact:
“I think the best thing was to have first-hand real experience of a relevant number of people, very diverse, living with the condition and happy to collaborate with us to try to design new drugs to help them.”
But finding patients who are willing to engage in this way can be challenging, which is where the NIHR’s Patient Engagement in Clinical Development Service comes in. The NIHR Clinical Research Network covers the whole of England and uses its national reach to help identify and engage patients country-wide. Consequently, the NIHR is the perfect partner for connecting companies with a diverse range of patients in terms of diseases and conditions, trial experience, socio-demographics, ethnicity and geography.
As an independent intermediary, the NIHR is also able to offer neutral ground for engagement activities. By taking care of the logistics and associated costs, for example by organising and hosting engagement sessions in venues around the UK and online, the service removes many of the common quandaries companies encounter. For instance, providing too much or too little in terms of catering, travel, accommodation and reimbursement. An NIHR representative will also facilitate the direct patient contact at each session. These seemingly little things are essential foundations for productive, unbiased and honest conversations which are invaluable to those designing the protocol.
The Patient Engagement in Clinical Development service also addresses legal and compliance requirements through a template Letter of Agreement, alleviating further
burden from life science industry companies while enabling flexibility where required. The NIHR also acts as the data controller, handling all patient data and GDPR processes. In summary, the NIHR has navigated much of the regulatory pathway for companies.
Marta commented on the ease of using the NIHR Patient Engagement in Clinical Development service and the quality of the sessions:
“The approach that they have with the patients is very transparent, [...] the whole set up of the meeting and the process is very professional.”
Accessible transcript for the video 'Company perspective: NIHR facilitates patient input into Servier’s first Sjögren’s Syndrome trial'.
Servier have now undertaken similar engagement activities in Japan and Spain and have been working in collaboration with Sjögren’s Europe to crosscheck some aspects of the trial design. Partly due to the success of the patient engagement sessions, the UK has been selected as one of the locations to conduct the trial. Servier is looking to recruit patients in 2021 with ongoing support from the NIHR throughout the trial.
- Find out more about this service by visiting our Patient Engagement in Clinical Development Service web page.
- Read a related case study: Pfizer and the NIHR leading patient engagement in lockdown.