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Current opinion in endocrinology, diabetes, and obesity
Apr 01, 2025;32 (2): 89-95.

Perspectives on early health economic evaluations of RNA therapies targeted at lipoprotein(a).

Angela Burvill, Gerald F Watts, Zanfina Ademi
Author Information
  1. Angela Burvill: Internal Medicine, Sir Charles Gairdner Hospital, Hospital Avenue.
  2. Gerald F Watts: School of Medicine, University of Western Australia, Nedlands.
  3. Zanfina Ademi: Health Economics and Policy Evaluation Research (HEPER) Group, Centre for Medicine Use and Safety, Faculty of Pharmacy and Pharmaceutical Sciences.
PMID: 39936471 DOI: 10.1097/MED.0000000000000904

Abstract

PURPOSE OF REVIEW: Early health economic evaluations of new medications are useful, as they consider the implications for health services.We reviewed recent literature on expected clinical outcomes of lowering of elevated plasma lipoprotein(a) [Lp(a)] in secondary prevention, which is essential information on effectiveness for economic evaluations.We reviewed a recent early economic evaluation of RNA therapies targeted at Lp(a).
RECENT FINDINGS: RNA-based therapies, if approved, would likely be used initially in adults with established atherosclerotic cardiovascular disease (ASCVD) and very high Lp(a). Adults with ASCVD have high absolute risk of recurrent events and elevated Lp(a) serves as a risk-enhancing factor.Potent lowering of Lp(a) in secondary prevention may be associated with significant relative risk reductions of coronary heart disease or ASCVD events; this needs confirmation in currently ongoing and future clinical trials.One economic evaluation has estimated the value of olpasiran and pelacarsen, at various willingness-to-pay thresholds, compared with standard-of-care secondary prevention.
SUMMARY: Early economic evaluations estimate longer-term clinical benefits and cost consequences associated with new medications.Existing casual evidence of Lp(a) and cardiovascular disease can be used in early economic evaluations as best available evidence, while awaiting results from major cardiovascular outcomes trials.

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MeSH Term

Humans
Lipoprotein(a)
Cost-Benefit Analysis
Secondary Prevention
Atherosclerosis
Oligonucleotides
Cardiovascular Diseases
Dicarboxylic Acids
Fatty Acids
RNA, Small Interfering

Chemicals

Lipoprotein(a)
ALN-PCS
Oligonucleotides
8-hydroxy-2,2,14,14-tetramethylpentadecanedioic acid
Dicarboxylic Acids
Fatty Acids
RNA, Small Interfering
Journal Article Review

Word Cloud

Created with Highcharts 10.0.0economicevaluationsLphealthclinicalsecondarypreventionearlytherapiescardiovasculardiseaseASCVDEarlynewmedicationsWereviewedrecentoutcomesloweringelevatedlipoproteinevaluationRNAtargetedusedhighriskeventsassociatedtrialsevidencePURPOSEOFREVIEW:usefulconsiderimplicationsservicesliteratureexpectedplasma[Lp]essentialinformationeffectivenessRECENTFINDINGS:RNA-basedapprovedlikelyinitiallyadultsestablishedatheroscleroticAdultsabsoluterecurrentservesrisk-enhancingfactorPotentmaysignificantrelativereductionscoronaryheartneedsconfirmationcurrentlyongoingfutureOneestimatedvalueolpasiranpelacarsenvariouswillingness-to-paythresholdscomparedstandard-of-careSUMMARY:estimatelonger-termbenefitscostconsequencesExistingcasualcanbestavailableawaitingresultsmajorPerspectives

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