3 Approach to evidence generation

3.1 Evidence gaps and ongoing studies

The committee was made aware of one ongoing study, the Clinical translation and commercialisation of ORBIT study (NIHR205467).

The study is investigating the potential use of ORBIT within the NHS and aims to:

define how the technology should be used
assess the usability and acceptability of the platform
conduct a budget impact assessment
develop a commercialisation and NHS adoption strategy.

Table 1 summarises the evidence gaps. Information about evidence status is derived from the EAG's report; evidence that does not meet the scope and inclusion criteria is not included. The table shows the evidence available to the committee when the guidance was published.

**Table 1 Evidence gaps and ongoing studies**
Evidence gap	ORBIT
Impact of ORBIT on people's symptoms and health-related quality of life	Limited evidence Ongoing study
Resource use	Limited evidence Ongoing study
Clinical and cost effectiveness in different subgroups	Limited evidence Ongoing study

3.2 Data sources

Data should be sourced from the ORBIT‑UK randomised controlled trial, as well as the ongoing ORBIT study.

3.3 Evidence collection plan

Quantitative and qualitative data on the clinical impact of ORBIT was collected as part of the ORBIT‑UK study at 3, 6, 12 and 18 months, and could be made available for future cost-effectiveness assessments. Additional evidence around the clinical impact of ORBIT, resource use, useability and acceptability is currently being collected through the ongoing NIHR205467.

Statistically robust data about the effectiveness of the technology in different subgroups requires very large study populations that are not feasible in this context. Analysis of any data that are available at the end of the evidence generation period will nevertheless support future NICE decision making.

The study should enrol a representative population; that is, people who would be offered standard care, including behavioural therapy, without digital technologies. This may include face-to-face appointments and monitoring. Eligibility for inclusion, and the point of starting follow up should be clearly defined and consistent during the study, to minimise selection bias.

Data should be collected from the point at which a person would become eligible for standard care. The study should also capture information on people who were eligible but chose not to use the technology or could not access it. Ideally, the studies should be run across multiple centres, aiming to recruit centres that represent the variety of care pathways in the NHS.

Incomplete records and potentially demographically imbalanced selection can lead to bias if unaccounted for. Data collection should follow a predefined protocol and quality assurance processes should be put in place to ensure the integrity and consistency of data collection. See NICE's real-world evidence framework, which provides guidance on the planning, conduct, and reporting of real-world evidence studies.

3.4 Data to be collected

Study criteria

At recruitment, eligibility criteria for suitability of using the digital technology and inclusion in the real-world study should be reported, and include:
- a clinical diagnosis of tic disorder or Tourette's syndrome
- position of the technology in the clinical pathway
- the point that follow up starts.

Baseline information and patient outcomes

Information about individual characteristics at baseline, for example, sex, age, ethnicity, socioeconomic status, clinical diagnosis (and date of diagnosis), details of any comorbidities and treatments. Other important covariates should be chosen with input from clinical specialists.
Changes in tic severity using the Yale Global Tic Severity Rating Scale total score, the Clinical Global Impression Score – Improvement, and ideally the Goal Based Outcomes scale, at baseline and over follow up.
Qualitative information about the impact of ORBIT on daily life, ideally including self-esteem, social interactions and school or work attendance and performance.
Information on healthcare resource use and exacerbation-related hospitalisation costs related to tic disorders and Tourette syndrome. This should include emergency department visits, hospital admissions, length of stay and GP visits.
Changes in a person's medication and any referrals to other services.

Implementation

Costs of digital technologies for supporting treatment of tic disorders and Tourette syndrome, including licence fees, healthcare professional staff time and training costs to support the service and costs of integration with NHS systems.
Access and uptake, including the number and proportion of eligible people who were able to, or accepted an offer to, access the technology, and reasons for refusal.
Engagement and drop-out information, including reasons for stopping treatment.

Safety monitoring outcomes

Any adverse events arising from using digital technologies to support treatment of tic disorders and Tourette syndrome.

Data collection should follow a predefined protocol and quality assurance processes should be put in place to ensure the integrity and consistency of data collection. See NICE's real-world evidence framework, which provides guidance on the planning, conduct, and reporting of real-world evidence studies.

3.5 Evidence generation period

The evidence generation period should be 3 years. This aligns with the planned duration of NIHR205467.

Digital therapy for chronic tic disorders and Tourette syndrome: early value assessment

Evidence generation plan for digital therapy for chronic tic disorders and Tourette syndrome: ORBIT