You have heard of NICE in the news or through your GP or hospital – here is a short insight into their work, as well as why and how it affects your access to certain drugs.
Patients and the wider public can play a role in influencing decisions taken by NICE. For further detailed information, please visit the NICE website
IMPORTANT UPDATE: NICE has issued their FINAL decision on the 4th March 2010.
They do NOT RECOMMEND the use of azacitidine (Vidaza) for MDS on the NHS. This will mean that PCT’s will most certainly reject requests for funding for this particular drug – other than in exceptional circumstances.
The MDS UK Patient Support Group – as well as 2 other patient groups (Leukaemia Society and Rarer Cancers Forum) are appealing against the NICE decision – please see our Campaigns page on azacitidine for further details. We need your urgent assistance.
The National Institute for Health and Clinical Excellence (NICE) is responsible for recommending the use of new medicines and treatments within the NHS. Appraisals involve a consultation and evaluation process featuring interested parties and an independent academic centre. The process ends with the Appraisal Committee – details of how it operates are given below.
Submissions and Evidence Review Reports
The Appraisal Committee receive Submissions and Evidence Review Reports. The main elements of appraisal are clinical effectiveness (including efficacy and side effects of the proposed treatment) and cost effectiveness.
Decisions are evidence driven, with RCT (randomized controlled trial) evidence being preferred. Expert and patient opinion may often also be required. Rigour, quality and transparency are very important in submissions.
Clinical evidence from trials is rarely sufficient and modelling will be required in most cases to provide evidence. Methods of modelling are closely specified in guidance documentation to produce a “reference case”.
The reference case requires the use of Quality Adjusted Life Years (QALY’s) – allowing gains in both quality of life and length of life changes to be encompassed in a single measure. The incremental cost per quality adjusted life year gained (cost/QALY) is used in estimating cost effectiveness.
Quality Adjusted Life Years (QALY’s)
The QALY consists of two elements – an estimate of quality of life anchored at 1 (perfect health) and 0 (death), and length of life. The quality of life weighting is derived from the general population using a process of comparisons between possible choices to generate weights.
As a general rule NICE treats a QALY to be of equal worth and irrespective of other circumstances of the disease (such as severity) or the patient (such as age, sex, parental responsibility etc). The only exception is the “end of life” consideration.
Special factors are required if the incremental cost per quality adjusted life year gained exceeds £20,000.With a fixed budget in place, this represents the estimated cost of marginal activities that would be displaced by introducing a new technology.
Criteria for Appraising End of Life Treatments
In appraising end of life treatments, the following conditions have to be met:
- life expectancy of less than 24 months,
- treatment offers an extension of at least 3 months,
- no alternative treatments available,
- small patient population.
Application of Criteria
If these conditions are met for “end of life” the Appraisal Committee will consider the impact of giving greater weight to QALYs in the later stages of terminal diseases (the extended survival period is assumed to give a Quality of Life equal to that of a healthy individual of the same age); and the size of additional weight that would be necessary to make the incremental cost per QALY gained fall below the usual £20,000 threshold.
Appraisal Committee Members
The Appraisal Committee has 24 members. Their roles are both technical (to interpret evidence and arguments) and evaluative (to reach a decision as a reasonable member of the public) using agreed criteria.
The rough composition of the Appraisal Committee is 15 practising doctors, 2 economists, a statistician, health managers, patient group representatives, a drug industry health economist and a devices industry health economist.
Process at Appraisal Committee
The Appraisal Committee process consists of documentary evidence, presentations, expert and patient commentary with questioning by the committee, deliberation and then a vote.
Nature of NICE Decisions
NICE may endorse a product wholly or in part. It may deem it not cost effective and therefore not recommended and it may argue that the evidence is currently inadequate and endorse “in research only” use. Decisions to support drugs but restrict access to them are commonplace.
The article above summarises information originally prepared by Ron Akehurst, (Professor of Health Economics and Dean of School, the University of Sheffield), with permission.