Year: 2010
Type: dissertation
Abstract: Cystic fibrosis (CF) is one of the most common genetic diseases in the UK.CF is a chronic, progressive, and life-shortening disease.As a multi-system disease, CF requires complex and constant care that can place a significant burden on constrained NHS resources.This thesis focuses on the economic impact on the NHS of paediatric CF in the East of England as well as investigating the cost-effectiveness of newborn screening for CF.The literature indicates that there is a relatively large variation (US$4069 to $29849 per case per year) in the direct cost of care from a health system perspective.The only newborn screening study from a UK perspective is now relatively old (1998) and the emergence of new therapies and service delivery methods could alter the findings of that study.First, a retrospective (1998,(2004)(2005)(2006)(2007) longitudinal cost of illness study was carried out on paediatric CF patients in the east of England.Patients" outcome and resource use information was gathered from patient notes and computerised systems.The study takes into account direct costs to the NHS only, and uses NHS tariffs to attach a unit cost to resource use.Regression analysis was used to analyse the data for indicators of cost drivers, controlling for age, and diagnosis category.Second, a cost-utility model was built to look at the long-term impact of newborn screening versus no screening (clinical diagnosis).Newborn screening for CF in the UK uses a bloodspot test which is tested using the immunoreactive trypsin test, a second confirmatory immunoreactive trypsin test and DNA analysis.The model was a probabilistic cohort simulation model that uses Markov processes to allow patient history over time.Once a patient had been diagnosed, they could enter one of four health states: Successful treatment, intermittent infection, chronic infection, and death.The results of the study show that the total cost of care for paediatric CF patients in the East of England has increased over time (1998)(1999)(2000)(2001)(2002)(2003)(2004)(2005)(2006)(2007)) from approximately £1,040,087 to £1,062,008 and that the median cost per patient has increased from £3852 to £4249.The regression analysis shows that disease severity has a significant negative impact on costs and being in the youngest and oldest age group indicates having a higher cost.The results of the cost-effectiveness analysis indicate that no screening is associated with a cost of £110,238 and a QALY gain of 11.23 and screening is associated with a cost of £110,417 and a QALY gain of 11.51.Therefore, newborn screening has a cost per QALY of £641 when compared with clinical diagnosis.This analysis has shown that newborn screening for CF is cost-effective, and that the government has made the correct decision by rolling out a national programme.Once identified by screening, treatment can begin immediately which may have a positive impact on future health for the patient.As shown by the regression analysis, keeping people healthier reduces the overall disease and cost burden on the NHS.The results of this thesis are most applicable to the UK context, however if treatment regimes and screening processes are similar in other countries, the results could be applicable in other contexts.The methods used in this thesis are applications of well known and utilised economic evaluation and statistical methods, and are appropriate for any diseases where prognosis and disease severity is variable.Chapter 6: Discussion .....
Author: Jerome Jarrett
Language: English
Subfield: Pulmonary and Respiratory Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: green
OpenAlex ID: W823463507