Clinical Context

Aortic valve stenosis (or narrowing) most often occurs due to excessive calcium buildup on the valve that prevents it from fully opening. This restricts the amount of blood that can flow out of the heart to supply the rest of the body. To overcome this, the heart must work harder, potentially leading to heart damage. The options available to treat the dysfunctional valve are open heart surgery and transcatheter aortic valve replacement.

Device Description

The SAPIEN 3 Transcatheter Heart Valve (THV) System (Premarket Approval P140031/S085) replaces a damaged aortic valve with a biological tissue valve using a catheter-based delivery system, thereby avoiding open heart surgery. It is a Class III percutaneously delivered aortic valve prosthesis.

Indications for Use

The Edwards SAPIEN 3 Transcatheter Heart Valve System is indicated for relief of aortic stenosis in patients with symptomatic heart disease due to severe native calcific aortic stenosis who are judged by a heart team, including a cardiac surgeon, to be appropriate for the transcatheter heart valve replacement therapy.

Initially indicated for patients with symptomatic heart disease due to calcific aortic stenosis who are at high or greater risk for open heart surgery, the label for the SAPIEN 3 THV System has been expanded three times to include a wider patient population; see Summary of Safety and Effectiveness Data for 2015, 2016, 2017, and 2019. The 2019 approval (P140031/S085) expanded the indications for use to include patients with symptomatic heart disease due to calcific aortic stenosis deemed to be at low risk for surgical aortic valve replacement.

Context of Clinical Outcome Assessment (COA) Use

Various COAs were included in the three submissions expanding the patient population indications for this device. The New York Heart Association functional class (clinician-reported outcome, or ClinRO) was used as part of the study eligibility criteria and secondary endpoints in those submissions. Three patient-reported outcome (PRO) instruments were used in the submissions to measure quality of life (EuroQoL-5D visual analog scale, Kansas City Cardiomyopathy Questionnaire (KCCQ), and the physical and mental component of the Short Form (SF)-36. The 6-minute walk test or 5-meter walk test (performance outcomes or PerfOs) were used to assess physical performance in the submissions. While COAs were used as descriptive effectiveness endpoints in the majority of the SAPIEN 3 THV System submissions, the 2019 label expansion relied upon the KCCQ as one of six hypothesis-tested secondary effectiveness endpoints.

Lessons Learned

The SAPIEN 3 THV System has historically leveraged COAs as part of the evidence to support label expansions for the device. Over time, a PRO has played an increasingly important role in the reporting of study outcomes for transcatheter heart valve technologies. This increased emphasis on COAs has resulted in their increased utilization and prioritization; see Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims.