Evicenter
P&T meetings

RP-A501 (AAV9.LAMP2B)

Danon disease

Regulatory submission
BLA planned for late 2028
Last updated
300 sources

Product information and disease descriptionProduct descriptionProduct information

Effect of RP-A501 on quality measures

12 evidence topics · 9 sources

Product-specific effect on quality measures

No evidence found.

Quality measures specific to Danon disease or hypertrophic cardiomyopathy

No evidence found.

HEDIS Cardiac Rehabilitation (CRE): heart transplantation as a qualifying event

“The percentage of persons 18 years and older who attended cardiac rehabilitation following a qualifying cardiac event, including myocardial infarction (MI), percutaneous coronary intervention (PCI), coronary artery bypass grafting (CABG), heart and heart/lung transplantation or heart valve repair/replacement.” (opens the source at this quote in a new tab)

“The percentage of persons who attended 2 or more sessions of cardiac rehabilitation within 30 days after a qualifying event.” (opens the source at this quote in a new tab)

“The percentage of persons who attended 36 or more sessions of cardiac rehabilitation within 180 days after a qualifying event.” (opens the source at this quote in a new tab)

HEDIS Plan All-Cause Readmissions (PCR): definition

“For persons 18 years of age and older, the risk-adjusted ratio of observed-to-expected unplanned acute readmissions (inpatient and observation stays) for any diagnosis within 30 days of an acute hospitalization (inpatient and observation stays).” (opens the source at this quote in a new tab)

Medicare Part C Star Ratings: Plan All-Cause Readmissions (measure C18)

“Percent of plan members aged 18 and older discharged from a hospital stay who were readmitted to a hospital within 30 days, either for the same condition as their recent hospital stay or for a different reason.” (opens the source at this quote in a new tab)

“The percentage of acute inpatient stays during the measurement year that were followed by an unplanned acute readmission for any diagnosis within 30 days, for members 18 years of age and older using the following formula to control for differences in the case mix of patients across different contracts.” (opens the source at this quote in a new tab)

Medicare Part D Star Ratings: Medication Adherence for Hypertension (RAS antagonists) (measure D09) and its sacubitril/valsartan exclusion

“This measure is defined as the percentage of Medicare Part D beneficiaries, 18 years and older, who adhere to their prescribed drug therapy for renin angiotensin system (RAS) antagonists: ACEI, ARB, or direct renin inhibitor medications.” (opens the source at this quote in a new tab)

“One or more prescriptions for sacubitril/valsartan during the treatment period” (opens the source at this quote in a new tab)

CMS Hospital Readmissions Reduction Program: heart failure among the measured conditions

“CMS includes the following condition or procedure-specific 30-day risk-standardized unplanned readmission measures in the program:” (opens the source at this quote in a new tab)

“Heart failure (HF)” (opens the source at this quote in a new tab)

“The payment reduction is capped at 3 percent (that is, a payment adjustment factor of 0.97).” (opens the source at this quote in a new tab)

CMS Merit-based Incentive Payment System, Quality ID 005: renin-angiotensin system inhibitor therapy for heart failure with ejection fraction of 40% or less

“Percentage of patients aged 18 years and older with a diagnosis of heart failure (HF) with a current or prior left ventricular ejection fraction (LVEF) ≤ 40% who were prescribed ACE inhibitor or ARB or ARNI therapy either within a 12-month period when seen in the outpatient setting OR at each hospital discharge.” (opens the source at this quote in a new tab)

CMS Merit-based Incentive Payment System, Quality ID 008: beta-blocker therapy for heart failure with ejection fraction of 40% or less

“Percentage of patients aged 18 years and older with a diagnosis of heart failure (HF) with a current or prior left ventricular ejection fraction (LVEF) ≤ 40% who were prescribed beta-blocker therapy either within a 12-month period when seen in the outpatient setting OR at each hospital discharge.” (opens the source at this quote in a new tab)

ACC/AHA heart failure performance measures: ejection fraction assessment and its heart transplant exclusion

“Percentage of patients age ≥18 y with a diagnosis of heart failure for whom the quantitative result of prior (any time in the past) LVEF assessment, using any imaging modality, is available in the medical record” (opens the source at this quote in a new tab)

“Patients post–heart transplant or with an LVAD are excluded, because these patients were excluded from clinical treatment trials for low LVEF” (opens the source at this quote in a new tab)

ACC/AHA heart failure performance measures: counseling on implantable cardioverter-defibrillator implantation

“Percentage of patients age ≥18 y with a diagnosis of heart failure with current LVEF ≤35% despite ACE inhibitor, ARB, or ARNI and beta-blocker therapy for at least 3 mo who were counseled regarding ICD implantation as a treatment option for the prophylaxis of sudden death” (opens the source at this quote in a new tab)

ACC/AHA heart failure quality measures: patient-reported outcome questionnaires

“To reflect the increasing importance of patient-reported outcome measures, 2 patient-reported outcomes quality measures were added that use heart failure patient-reported outcomes questionnaires currently accepted by the US Food and Drug Administration.” (opens the source at this quote in a new tab)

“Two surveys are well validated: The Kansas City Cardiomyopathy Questionnaire” (opens the source at this quote in a new tab)

ACC/AHA heart failure measures, 2024 update: new measures including counseling on contraception and pregnancy risks in cardiomyopathy

“New performance measures include optimal blood pressure control in patients with heart failure with preserved ejection fraction, the use of sodium-glucose cotransporter-2 inhibitors for patients with heart failure with reduced ejection fraction, and the use of guideline-directed medical therapy in hospitalized patients.” (opens the source at this quote in a new tab)

“patient-centered counseling regarding contraception and pregnancy risks for individuals with cardiomyopathy” (opens the source at this quote in a new tab)

Heart transplant program performance metrics monitored by the Organ Procurement and Transplantation Network

“The new transplant program performance monitoring system involves four risk-adjusted measures related to the patient journey:” (opens the source at this quote in a new tab)

“90-day graft survival hazard ratio: A program’s rate of graft failure from date of transplant to 90 days post-transplant, relative to the expected rate based on transplants with similar recipient and donor organ characteristics across the nation.” (opens the source at this quote in a new tab)

“The pre-transplant mortality model reports a program’s pre-transplant mortality rate relative to the mortality rate experienced by similar patients across the nation over a 2-year evaluation” (opens the source at this quote in a new tab)