Verteporfin photodynamic therapy for neovascular age-related ...

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.