Advice

Clinical and technical evidence

Clinical and technical evidence

A literature search was carried out for this briefing in accordance with the interim process and methods statement. This briefing includes the most relevant or best available published evidence relating to the clinical effectiveness of the technology. Further information about how the evidence for this briefing was selected is available on request by contacting mibs@nice.org.uk.

Published evidence

There are 3 studies summarised in this briefing, including a total of 46 patients.

These studies include 1 feasibility study for a randomised controlled trial (RCT) and 2 economic studies (1 published as an abstract only).

The clinical evidence and its strengths and limitations is summarised in the overall assessment of the evidence.

Overall assessment of the evidence

CFHealthHub was first launched in 2015 and is still at an early stage of evidence generation. This means there is limited published evidence showing its effectiveness. The only comparative effectiveness evidence is from a feasibility RCT, which was only powered to show the feasibility of a larger RCT and not to report clinical outcomes.

The full-scale RCT of 607 people with cystic fibrosis (CF) having usual care or CFHealthHub completed in 2019 and is expected to publish later in 2020. The setting and primary outcomes for this trial will be highly relevant to the NHS. They will include clinical outcomes around medicines adherence and the rates of exacerbations. The results of this RCT will also inform the economic modelling that has been done to examine the cost effectiveness of CFHealthHub, which has relied on assumptions about its effectiveness. Another study in preparation for publication in 2020 is an analysis of the cost savings associated with medicines optimisation using CFHealthHub. This study analyses the costs of 330 people with CF in 13 CF centres. This will help to support the model-based cost analyses by providing evidence of real-world costs saved.

Hind et al. 2019

Study size, design and location

A randomised feasibility study of 64 adults with CF in 2 CF centres in England. The aim was to see whether the full-scale RCT (due to publish later in 2020) was feasible.

Intervention and comparator(s)

There were 33 people randomised to have care using CFHealthHub and 31 randomised to have usual care, which was typically face-to-face reviews every 3 months. Each person in the control group used a nebuliser that recorded its use, but these data were not available to people in this group.

Key outcomes

Primary recruitment and retention were satisfactory. Over the 5‑month study, no statistically significant differences in the rate of exacerbations was seen between groups. However, the study was not powered to detect these.

Strengths and limitations

There is limited reporting of user outcomes because this is a feasibility study for a larger RCT.

Tappenden et al. (2019)

Study size, design and location

A model-based cost utility analysis of the potential cost-effectiveness of CFHealthHub using a state‑transition model.

Intervention and comparator(s)

CFHealthHub compared with costs of standard care.

Key outcomes

In the model, CFHealthHub was assumed to reduce the number of days that people with CF needed intravenous antibiotics to treat infections with Pseudomonas aeruginosa. It was assumed that CFHealthHub would reduce exacerbations by 1 per person every year, when each exacerbation needs 14 days of home or hospital intravenous antibiotic treatment.

The study concluded that if CFHealthHub is effective then it will produce an additional 0.19 quality‑adjusted life years and cost savings of £64,000 per patient every year. Over 5 years this could generate costs savings of £49.5 million.

Strengths and limitations

This study uses assumptions about the effectiveness of CFHealthHub and so should be viewed with caution. The authors intend to update the model with the real effectiveness data from the ongoing RCT.

Sadler et al. (2016)

Intervention and comparator(s)

CFHealthHub compared with standard care. The study used a Markov model from the NICE technology appraisal guidance on colistimethate sodium and tobramycin dry powders for inhalation for treating pseudomonas lung infection in cystic fibrosis. The model was modified to estimate the incremental cost effectiveness of CFHealthHub. Sensitivity analyses were done to explore uncertainties in the assumptions about CFHealthHub, including its clinical effectiveness.

Key outcomes

All analyses showed that CFHealthHub would produce health gains and costs savings compared with standard care. Over 5 years, CFHealthHub was expected to generate cost savings of £17,852 per patient. This is equivalent to £106.5 million for the 5,864 adults with CF in the UK.

Strengths and limitations

Because this is a conference abstract, there is very limited reporting of the methodology used in the analysis. The sources for the clinical-effectiveness data in the modelling are not given.

Recent and ongoing studies