EXECUTIVE SUMMARY

  This submission is from a systematic reviewer for the NICE guidance programme. The main issues it addresses are that the work to assess treatments is complicated and time consuming, explaining results to a lay audience is difficult and may not be happening adequately at the moment and that groups without a strong lobby are missing from the decision making process. NICE is vital to protect patients from expensive new treatments that are no better than currently used alternatives.

  I work within a team at the University of Birmingham Department of Public Health and Epidemiology to produce Technology Assessment Reports for the NICE guidance programme. The work that we do for NICE includes systematic reviews of the published and unpublished evidence on a particular topic and an economic model. In the past I have worked on the reports on Coronary Artery Stents and Photodynamic Therapy for Age-Related Macular Degeneration and am currently leading on Structural Neuroimaging in First Episode Psychosis and Use of Tumour Necrosis Factor Alpha Inhibitors for Crohn's Disease Multiple Technology Assessment Reports. I also teach academics how to perform systematic reviews of the evidence at masters' degree level. Some of these students are now either working at NICE or in Evidence Review Groups producing technology appraisal reports for NICE. I have no financial investment in any drug or device company.

FACTUAL INFORMATION AND RECOMMENDATIONS FOR ACTION

  1.  This submission is largely based around my experience of the NICE process that I witnessed while I was doing the technology appraisal on Photodynamic Therapy for Age Related Macular Degeneration (PDT for AMD).

  2.  The UK drug licence for photodynamic therapy with verteporfin was based around a subgroup analysis on a particular subgroup of patients in a single randomised controlled trial (RCT). When the second relevant RCT was published, the subgroup analysis no longer appeared to be a true estimate of the effectiveness of the treatment. This suggested that the drug licensing process was based on a subgroup analysis that was developed for the reporting of the first trial (predominately classic AMD) rather than a clinically accepted subgroup known before the trial was conducted (classic wet AMD).

  3.  Whilst doing the systematic review we encountered some very difficult academic issues around how to measure vision and how to measure quality of life in patients with vision loss. This was needed in order to establish the cost effectiveness of treatment.

  4.  The issues that arose were very complicated so getting them across to a lay audience was very difficult. I listened to press coverage of this topic when it was covered, particularly on BBC Radio 4, and they struggled to present the necessary detail required to really understand the issues.

  5.  At the same time it came to my attention that patient groups were receiving information about photodynamic therapy that was overemphasising the clinical effectiveness of the drug and not giving due weight to the known potential side effects (blindness in a small percentage of patients) and the uncertainties around the effectiveness of treatment. Patient groups had also not been told that the potential budget impact to the NHS was enormous and that there were insufficient ophthalmologists to administer the treatment.

  6.  The general impression I was given from press articles was that photodynamic therapy would "save vision" and "improve vision". However, it was only ever shown from the RCT results that photodynamic therapy could slow the rate of vision loss in some patients who had the wet form of AMD.

  7.  It appears to me that public confidence in NICE may be waning. This could be due to two main factors. Firstly the information needed to really understand the issues properly is complicated so explaining it to a lay audience is tricky. I am unsure that NICE has really been able to tackle this area adequately because I do not know if they have the remit or staff to do it. Secondly, some drug companies are providing some funding for patient groups, and at the same time letting them have promotional material for their products. The patient groups are not having access to independent appraisal of this evidence. So when NICE pronounce on a particular topic, it does not tally with what patient groups have been told so they tend to complain, particularly if they are told they cannot have a treatment they think they should have.

  8.  The types of people who are currently disadvantaged by the NICE process are those who do not have a strong lobbying voice. For example, in PDT for AMD, it may have been more cost effective and may have benefited far more people if the money that could have been used for PDT instead had been used for much more extensive rehabilitation services for people going blind. However, the rehabilitation services do not have a strong lobbying voice so this perspective was lost to the decision-making process.

  9.  With regard to the speed of publishing guidance—I am more concerned that the guidance is correctly based on the best evidence available rather than rushed through. The issues involved in many of the Technology Appraisals are very complex and can't be evaluated without sufficient time for thought. I think that it is entirely appropriate that there is an appeals process.

  10.  If we didn't have NICE then what would have happened for photodynamic therapy would have been considerable pressure from the drug company to ophthalmologists to provide this treatment for all patients with wet AMD irrespective of whether they would really benefit from the treatment. They would also not have considered what services would not have been offered because the money was being spent on PDT. Some ophthalmologists would have provided the treatment; some would not, resulting in postcode prescribing and other more basic essential services without groups to lobby for them being squeezed out. It is essential that NICE provides a way to impartially assess these very expensive new treatments because they aren't necessarily any better than our currently available treatments.

Dr Catherine Meads

University of Birmingham

7 March 2007