PDB24 An EARLY Cost-Utility MODEL of Mrna-Based Therapies Compared with Standard of Care in EARLY-Onset Propionic Acidaemia in the United Kingdom

This chapter builds upon Chapters 2 and 6 by introducing the reader to the history and concepts of health-related quality of life, cost–utility analysis, quality-adjusted life years (QALYs), and payer thresholds. The aim of this chapter is to outline in more depth the role of applied cost–utility analyses in the economic evaluation of public health interventions. The chapter goes on to reproduce a paper by Owen and colleagues at the National Institute for Health and Care Excellence (NICE) in the United Kingdom. This paper shows that many public health interventions often have a cost per QALY considerably lower than the £20,000 payer threshold conventionally used by NICE in the United Kingdom.

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Study protocol: optimising newborn nutrition during and after neonatal therapeutic hypothermia in the United Kingdom: observational study of routinely collected data using propensity matching

BMJ Open ◽

10.1136/bmjopen-2018-026739 ◽

2018 ◽

Vol 8 (10) ◽

pp. e026739 ◽

Cited By ~ 2

Author(s):

Cheryl Battersby ◽

Nick Longford ◽

Mehali Patel ◽

Ella Selby ◽

Shalini Ojha ◽

...

Keyword(s):

United Kingdom ◽

Therapeutic Hypothermia ◽

Late Onset ◽

Standard Of Care ◽

Blood Stream ◽

Research Database ◽

Population Database ◽

Scientific Publications ◽

Link Type ◽

The United Kingdom

IntroductionTherapeutic hypothermia is standard of care for infants born ≥36 weeks gestation with hypoxic ischaemic encephalopathy (HIE); consensus on optimum nutrition during therapeutic hypothermia is lacking. This results in variation in enteral feeding and parenteral nutrition (PN) for these infants. In this study, we aim to determine the optimum enteral nutrition and PN strategy for newborns with HIE during therapeutic hypothermia.Methods and analysisWe will undertake a retrospective cohort study using routinely recorded electronic patient data held on the United Kingdom (UK) National Neonatal Research Database (NNRD). We will extract data from infants born ≥36 weeks gestational age between 1 January 2008 and 31 December 2016, who received therapeutic hypothermia for at least 72 hours or died during therapeutic hypothermia, in neonatal units in England, Wales and Scotland. We will form matched groups in order to perform two comparisons examining: (1) the risk of NEC between infants enterally fed and infants not enterally fed, during therapeutic hypothermia; (2) the risk of late-onset blood stream infections between infants who received intravenous dextrose without any PN and infants who received PN, during therapeutic hypothermia. The following secondary outcomes will also be examined: survival, length of stay, breast feeding at discharge, hypoglycaemia, time to full enteral feeds and growth. Comparison groups will be matched on demographic, maternal, infant and organisational factors using propensity score matching.Ethics and disseminationIn this study, we will use deidentifed data held in the NNRD, an established national population database; parents can opt out of their baby’s data being held in the NNRD. This study holds study-specific Research Ethics Committee approval (East Midlands Leicester Central, 17/EM/0307). These results will help inform optimum nutritional management in infants with HIE receiving therapeutic hypothermia; results will be disseminated through conferences, scientific publications and parent-centred information produced in partnership with parents.Trial registration numberNCT03278847; pre-results,ISRCTN47404296; pre-results.

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