The aim of the Health Technology Assessment (HTA) Programme is to ensure that high quality research information on the clinical effectiveness, cost-effectiveness and broader impact of healthcare treatments and tests are produced in the most efficient way for those who plan, provide or receive care from NHS and social care services. The commissioned workstream invites applications in response to calls for research on specific questions which have been identified and prioritised for their importance to the NHS, patients and social care.
Research question
Does pectus excavatum corrective surgery improve functional cardiopulmonary outcomes and is it cost effective?
- Patient group: Patients with material pectus excavatum deformity (e.g., a Haller Index of >3.25) and concomitant physiological impairment, excluding emergency cases (Applicants to define and justify).
- Intervention: Corrective pectus surgery by the Nuss or Modified Ravitch procedure. Applicants to define and justify choice of surgery.
- Comparator: Applicants to define and justify an appropriate comparator group which can account for normal growth and development in a similarly aged population group.
- Important outcomes: Change in a single or composite primary outcome measure that best captures potential material functional improvements to, or resolution of, cardiopulmonary impairments typically associated with PE, ideally using an established measure of minimally clinically important difference (MCID), for example but not limited to syncope or other measures.
Other outcomes: Proxy measures of cardiopulmonary function (such as VO2 max or O2 pulse); quality of life including measures of impact on mental well-being; acceptability; need for revision surgery; cost-effectiveness. Existing Core Outcomes should be included amongst the list of outcomes unless a good rationale is provided to do otherwise. Applicants are encouraged to report recruitment and findings disaggregated by sex (and other demographic factors where relevant). - Setting: Secondary care.
- Study design: Applicants to propose and justify a study design that will provide robust information about effectiveness of the intervention.
- Minimum duration of follow-up: Applicants to define and justify. Longer-term follow up: If appropriate, researchers should consider obtaining consent to allow potential future follow-up through efficient means (such as routine data) as part of a separately funded study.
Rationale
Pectus excavatum (PE) is the most common congenital chest wall deformity and occurs in approximately 1 in 400 to 1000 births, with males affected 3 to 5 times more frequently than females. It usually becomes most apparent during the adolescent growth spurt resulting in potentially significant depression of the breastbone (sternum) in what is often called “funnel chest”.
While significant impact on body image and associated quality of life and psychological outcomes are universally recognised there continues to be conflicting evidence and much debate on if and how much PE impacts cardiopulmonary outcomes.
Corrective surgery via the open Modified Ravitch Procedure (developed in the 1940s), or more commonly now with the non-invasive Nuss Procedure (developed in the 1990s), is now routine across much of the world. This also used to be the case in the NHS before NHS England changed their commissioning policy in 2019 when the decision was taken not to routinely fund such surgeries due to a lack of sufficient evidence that this corrective surgery makes material difference to objective cardiopulmonary outcomes, over and above any improvements that might be seen in psychological and associated quality of life outcomes.
NHS England continues to receive individual funding requests for this surgery as well as policy change requests from the clinical community, but there are still material gaps in the supporting evidence. There are a very small number of patients who present, with clinically urgent (potentially life threatening), cardiopulmonary symptoms in association with PE and where there is a potentially stronger clinical case for surgical treatment. A revised clinical policy proposal is currently being considered by NHS England in this cohort, but this leaves a wider group of patients who also have significant PE deformity and some physiological impairment, but for whom surgery is not available through the NHS.
To provide additional high-quality evidence to inform future commissioning decisions, the NIHR, through the HTA programme seeks to fund a study which can demonstrate the potential for material objective functional improvement in cardiopulmonary outcomes from corrective PE surgery.
Additional commissioning brief background information
A background document is available that provides further information to support applicants for this call. It is intended to summarise what prompted the call and the existing evidence base, including relevant work from the HTA and wider NIHR research portfolio. It was researched and written on the basis of information from a search of relevant sources and databases, and in consultation with a number of experts in the field. If you would like a copy please email htaresearchers@nihr.ac.uk.
Making an application
If you wish to submit a Stage 1 application for this call, the online application form can be found on the Funding opportunities page. To select this call, use the filters on the right of the screen or search using the call name and/or number.
Your application must be submitted online no later than 1pm, 29 March 2023. Applications will be considered by the HTA Funding Committee at its meeting in May 2023.
Guidance notes and supporting information for HTA Programme applications are available.
Important: Shortlisted Stage 1 applicants will be given 8 weeks to submit a Stage 2 application. The Stage 2 application will be considered at the Funding Committee in September 2023. Applications received electronically after 1pm on the due date will not be considered.
For commissioned topics, the Programme strongly discourages the practice of the same Co-Applicant joining more than 1 competing team. There may be unusual circumstances where the same person could be included on more than 1 application, e.g. a Lead from a named charity or a unique national expert in a condition.
For such exceptions, (i) each application needs to state the case as to why the same person is included, (ii) the shared Co-Applicant should not divulge application details between teams and (iii) both teams should acknowledge in their application that they are aware that one of their Co-Applicants is part of a competing application and that study details have not been shared.
Should you have any queries please contact us: htacommissioning@nihr.ac.uk.