Verteporfin photodynamic therapy for neovascular age-related ...

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2 Introductionon-going or new clinical studies that are designed to generate robust and relevant outcome data,including data on optimum treatment regimens, long-term outcomes, quality of life and costs’(Box 1). 6Uncertainties about the effectiveness of VPDT were highlighted by the NICE technologyappraisal. 6 Around the time of publication of the NICE guidance, the NHS R&D HealthTechnology Assessment programme, on behalf of the Department of Health, approachedpotential investigators about carrying out further research on VPDT to address theseuncertainties. The overall aim was to characterise the cohort of patients referred for and treatedwith VPDT and to collect data about their visual acuity outcomes, quality of life and use of healthand social care resources.Initially, it was envisaged that this research should focus on the group of patients referred to inparagraph 1.2 of the NICE guidance (see Box 1), that is patients with ‘predominantly classic’CNV lesions. However, discussions with the regional NHS commissioners and the Royal Collegeof Ophthalmologists led to the scope of the research being expanded to include all patientstreated with VPDT, irrespective of the subtype of the CNV. The VPDT cohort study was set upto meet these objectives. It built on surveillance programmes and research proposals active at thetime and allowed patients with nAMD to access VPDT through the NHS.The VPDT cohort study investigators aimed to address a number of questions which wererelevant to NICE and to the NHS which were unanswered at the time of its guidance in 2003.These are set out in Chapter 2. However, the study was also of interest to other stakeholders, forexample commissioners of NHS care. There is a need to develop robust methods of managingthe introduction of new technologies into the NHS, especially when these are expensive, andthe VPDT cohort study is one model by which this might be achieved. At the outset, therewas the ambition that establishing a treatment register would allow a new technology (in thiscase, VPDT) to be introduced to a pre-specified service standard ensuring best possible carefor patients, its use to be monitored effectively by commissioners and uncommon/rare adverseevents (AEs) not identified in pre-licensing trials to be detected. A further benefit might betraining clinical sites in research methods and processes to facilitate future clinical trials andresearch relevant to the NHS.BOX 1 Extract from NICE guidance to the NHS on VPDT 61.1 Photodynamic therapy (PDT) is recommended for the treatment of wet age-related macular degenerationfor individuals who have a confirmed diagnosis of classic with no occult subfoveal choroidalneovascularisation (CNV) (that is, whose lesions are composed of classic CNV with no evidence of anoccult component) and best-corrected visual acuity 6/60 or better. VPDT should be carried out only byretinal specialists with expertise in the use of this technology.1.2 PDT is not recommended for the treatment of people with predominantly classic subfoveal CNV (that is,50% or more of the entire area of the lesion is classic CNV but some occult CNV is present) associatedwith wet age-related macular degeneration, except as part of on-going or new clinical studies that aredesigned to generate robust and relevant outcome data, including data on optimum treatment regimens,long-term outcomes, quality of life and costs.1.3 The use of VPDT in occult CNV associated with wet age-related macular degeneration was not consideredbecause the photosensitising agent (verteporfin) was not licensed for this indication when this appraisalbegan. No recommendation is made with regard to the use of this technology in people with this form ofthe condition.
DOI: 10.3310/hta16060Health Technology Assessment 2012; Vol. 16: No. 63Visual acuity and health-related quality of lifeA limitation of many randomised controlled trials (RCTs) of ophthalmological interventionsis that the researchers conventionally choose best-corrected monocular distance visual acuity(BCVA) as the primary outcome. Reporting the effect of a new treatment as the average BCVAbenefit relative to a control group allows ophthalmologists to consider the probable value of thenew treatment compared with the best existing treatment (or alternative treatments) for the samecondition. However, the limitations of clinical measures of outcome are now widely appreciatedand many governmental and non-governmental organisations emphasise the importance ofpatient-reported outcomes or health-related quality of life (HRQoL) for measuring treatmenteffectiveness and health-care performance. 7,8 Moreover, the benefits of ophthalmic interventionsare difficult to compare with other health-care interventions without being able to describe themin a common currency, for example quality-adjusted life-years (QALYs, see below and The healthand social service costs of nAMD and associated treatments). 9Health-related quality of life is a complex concept. A spectrum of instruments have beendesigned to measure HRQoL, from ones focused closely on functional performance to thoseassessing broader domains and rating the importance to an individual patient of a perceivedloss of physical, social or emotional function. 10 This spectrum can be investigated specificallywith respect to the condition affecting a respondent (condition-specific instruments) or to hisor her wider life experience (generic instruments). The National Eye Institute Visual FunctionQuestionnaire (NEIVFQ), which lies towards the functional performance end of the HRQoLspectrum, is perhaps the most widely used vision-specific HRQoL instrument. 11A subset of generic HRQoL instruments explicitly recognises underlying preferences fordifferent health states. These preference-based measures, such as the European Quality ofLife-5 Dimensions (EQ-5D) and the Short Form questionnaire-6 Dimensions (SF-6D), 12,13report HRQoL on a scale with ‘anchors’ at 0 for death and 1 for perfect health. Preferencebasedmeasures of HRQoL are important because they can be combined with the relativeeffects of interventions on life expectancy to report QALYs. 9 QALYs allow comparison ofinterventions that may improve HRQoL but not life expectancy (such as many ophthalmicinterventions) with interventions in other disease areas that can improve life expectancy buthave little effect on HRQoL (e.g. statins to prevent coronary heart disease). Such comparisons,using HRQoL measures that take preference weights (i.e. societal values) or utilities from thegeneral population, 14,15 underpin health policy in many publicly funded health systems. Theyenable policy-makers to decide the relative worth of a new treatment in a wider context, thatis compared with the value of health-care treatments for all other conditions that compete forfunding from a finite budget.Many studies have examined cross-sectional associations between visual acuity and HRQoLusing a variety of HRQoL instruments including preference-based measures. 16,17 However,few have examined these associations longitudinally. Also, several studies that have reportedpreference-based measures of HRQoL have reported utilities elicited directly from patients 18rather than by the recommended process of taking these preferences from the generalpopulation. 14,15 Previous studies that have attempted to use preference-based measures of HRQoLto assess the gains from ophthalmic interventions for policy-making purposes have highlightedthe deficiencies in existing studies. 19The VPDT cohort study collected clinical measures of vision, measures of HRQoL and measuresof resource use to achieve its principal aims of estimating the effectiveness and cost-effectiveness© Queen’s Printer and Controller of HMSO 2012. This work was produced by Reeves et al. under the terms of a commissioning contract issued by theSecretary of State for Health.
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90 Appendix 1THE VERTEPORFIN PHOTOD
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FeedbackThe HTA programme and the a
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