4 Committee discussion

Clinical effectiveness

4.1 Christensen et al. (2006) and the observational studies reported significant improvements in patient-reported outcome measures. The committee noted some uncertainty in the quality of this evidence including because Peristeen is self-administered, so there are limitations with patient-reported outcome measures.

4.2 The clinical and patient experts explained that for people with bowel dysfunction, even small improvements in these patient-reported outcome measures can translate into significant quality-of-life benefits and could mean the difference between adequate bowel control and incontinence. The committee concluded that the evidence with which it had been presented may underestimate the quality-of-life benefits of Peristeen.

4.3 The patient experts emphasised that using Peristeen has vastly improved their lives, allowing them a degree of functional independence (such as going on holiday and maintaining a permanent job) that was not possible with the standard bowel care they had previously received. The committee also heard from the clinical experts that using Peristeen may lead to improved attendance and participation in school and social life for some children with bowel dysfunction.

Drop-out rates in the trials

4.4 The committee discussed the high initial drop-out rates in the clinical trials, and was advised by the experts that this accurately reflected their own clinical practice experience. People who try Peristeen are likely to know within the first 1 or 2 months if it is going to be suitable for them. The patient experts explained that it takes up to 2 months to become confident with using Peristeen and that people wishing to use Peristeen must be motivated and determined to succeed with the technology.

Peristeen's use in children

4.5 The committee noted that the evidence for Peristeen in children is less robust than in adults. However, it recognised that clinical studies are difficult in children with a wide range of underlying conditions, particularly because of challenges with communicating patient-reported outcome measures. One clinical expert had experience of using Peristeen in teenagers with megarectum. This group used the device on average once a week and were able to maintain bowel control that allowed them to attend school. Several comments were received during consultation about the successful use of Peristeen in children at centres across the NHS, including a report from the UK Paediatric Colorectal Group of the British Association of Paediatric Surgeons.

4.6 The committee acknowledged that Peristeen is successfully used in children in the NHS and that there is anecdotal evidence describing its long-term use. The committee noted observations that the drop-out rate for children using Peristeen was not as high as for adults. The expert advisers considered that this is likely to be because of ongoing encouragement and support from parents and carers for children using Peristeen. Despite limitations in the published evidence, the committee concluded that Peristeen may offer significant benefits for children with bowel dysfunction. The committee was aware that the economic modelling only considered adults, but it judged that using Peristeen in children is unlikely to cost any more than standard bowel care.

Comparators

4.7 The patient experts explained that, before trying Peristeen, their symptoms were severe enough for them to have considered more invasive treatments such as colostomy.

4.8 The clinical experts stated that stoma surgery may represent an improvement in quality of life for some people with bowel dysfunction who are severely disabled by their symptoms and find a colostomy easier to manage. ACE procedures may also be offered to patients and are often an option in children. The experts advised, however, that stoma surgery is associated with a risk of subsequent hernias and the need for revision surgery.

NHS and system impact considerations

4.9 The committee was aware that there are other transanal irrigation devices available in the NHS. It considered that clinicians should discuss the different options with the patient to help identify the device which is most appropriate.

4.10 The clinical and patient experts explained that the high initial drop-out rates associated with using Peristeen may be reduced by ensuring good quality training and support for both patients and staff. The company has a team of nurses in the UK that provide training for patients and for bowel care specialists, with additional non-clinical resources including a patient support phone programme.

4.11 The committee was advised that Peristeen is usually first prescribed by specialist bowel care teams with ongoing prescription in primary care. It considered that there is a need for improved awareness of transanal irrigation in the NHS as a treatment option for bowel dysfunction.

4.12 The clinical and patient experts explained that Peristeen should be offered as part of a supportive bowel care programme. People using Peristeen should have training from a specialist healthcare professional. The committee heard that most people will have 1 face-to-face appointment to learn how to use Peristeen, and then have further follow-up support in the community (usually over the phone). The experts noted that it takes most people a few months to get used to Peristeen. Even once someone is confident with using the device, they still need access to a professional support system (such as easily accessible contact details of a specialist nurse) to provide advice as needed.

4.13 The patient experts commented that the support of dedicated specialists was essential to their being able to use Peristeen effectively. They added that they would have found a patient support group helpful.

Cost modelling

4.14 The committee accepted the external assessment centre's (EAC) suggested changes to the company's model, and concluded that its results were more plausible than the company's base case. The EAC's updated model showed that using Peristeen could result in cost savings of £2,867 per patient over 37 years. These savings mainly come from fewer healthcare professional visits and less carer time, reduced incidence of faecal incontinence needing incontinence pads, reduced incidence of urinary tract infections and fewer hospitalisations.

4.15 The committee considered that there was considerable uncertainty in the cost modelling for Peristeen. The audit data used in the model was not available for scrutiny and a number of assumptions used in the model were not sourced. Although the EAC was unable to source more robust assumptions, it identified that the hospitalisation rates for pressure ulcers and urinary tract infections were higher than expected so included these in its changes to the model.

4.16 The committee discussed the frequency of administration because the cost of each catheter is an important factor influencing the overall cost of treatment. The instructions for use recommend that it should be used every other day after an initial few weeks of using it every day. The clinical experts confirmed that this was the average frequency of use for most people with neurogenic bowel dysfunction using Peristeen.

4.17 The committee noted the EAC's sensitivity analysis which showed that Peristeen would become cost incurring if it were used more than 4 times per week. The patient experts stated that although they normally use the device every other day, there are times when they need to irrigate more frequently (such as when travelling or after a change in diet).

4.18 The committee considered that Peristeen can provide important clinical benefits in most people with bowel dysfunction, including improving quality of life and promoting independence. It acknowledged that it may take several weeks before a person is comfortable with using Peristeen, so the device is most effective when offered with specialist training and structured patient support. The committee concluded that although the cost modelling is uncertain, it is likely that using Peristeen in people with bowel dysfunction does not cost any more than standard care.

  • National Institute for Health and Care Excellence (NICE)