Case Example 7Designing a Registry for a Health Technology Assessment

DescriptionThe Nuss procedure registry was a short-term registry designed specifically for the health technology assessment of the Nuss procedure, a novel, minimally invasive procedure for the repair of pectus excavatum, a congenital malformation of the chest. The registry collected procedure outcomes, patient-reported outcomes, and safety outcomes.
SponsorsNational Institute for Health and Clinical Excellence (NICE), United Kingdom
Year Started2004
Year Ended2007
No. of Sites13 hospitals
No. of Patients260 patients

Challenge

The Nuss procedure is a minimally invasive intervention for the repair of pectus excavatum. During a review of the evidence supporting this procedure conducted in 2003, the National Institute for Health and Clinical Excellence (NICE) determined that the existing data included relatively few patients, few quality of life outcomes, and did not sufficiently address safety concerns. NICE concluded in the 2003 review that the evidence was not adequate for routine use and that more evidence was needed to make a complete assessment of the procedure.

Proposed Solution

Gathering additional evidence through a randomized controlled trial was not feasible for several reasons. First, a blinded trial would be difficult because the other procedures for the repair of pectus excavatum produce much larger scars than the Nuss procedure. Surgeons also tend to either perform only the Nuss procedure or only another procedure, a factor which would complicate randomization efforts. In addition, only a small number of procedures are done in the United Kingdom. The sample for a randomized trial would likely be very small, making it difficult to detect rare adverse events.

Due to these limitations, NICE decided to develop a short-term registry to gather evidence on the Nuss procedure. The advantages of a registry were its ability to gather data on all patients undergoing the procedure in the UK to provide a more complete safety assessment, and its ability to collect patient-reported outcomes.

The registry was developed by an academic partner, with input from clinicians. Hospitals performing the procedure were identified and asked to enter data into the registry on all patients undergoing the intervention. Once the registry was underway, the cases in the registry were compared against cases included in the Hospital Episodes Statistics (HES) database, a nationwide source of routine data on hospital activity, and nonparticipating hospitals were identified and prompted to enter their data.

Results

NICE conducted a reassessment of the Nuss procedure in 2009, comparing data from the registry to other published evidence on safety and efficacy. The quantity of published literature had increased substantially between 2003 and 2009. The new publications primarily focused on technical and safety outcomes, while the registry included patient-reported outcomes. The literature and the registry reported similar rates of major adverse events such as bar displacement (from 2 to 10 percent). Based on the registry data and the new literature, the review committee found that the evidence was now sufficient to support routine use of the Nuss procedure, and no further review of the guidance is planned. Committee members considered that the registry made a useful contribution to guidance development.

Key Point

The Nuss registry demonstrated that a small, short-term, focused registry with recommended (but not automatic or mandatory) submission can produce useful data, both about safety and about patient-reported outcomes.

From: Chapter 3, Registry Design

Cover of Registries for Evaluating Patient Outcomes: A User's Guide
Registries for Evaluating Patient Outcomes: A User's Guide. 2nd edition.
Gliklich RE, Dreyer NA, editors.

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