This annex
     
a discusses the general principles for selection of particular new interventions/products for appraisal, and
b suggests how these could be turned into specific selection criteria.
     
Context
     
2 Para 10 of the main paper summarises the government's proposals for a national process which will
     
  i identify significant new interventions well before they are made available for routine use in the NHS and
  ii invite the sponsoring company (if any) to submit evidence as the basis of
  iii an appraisal by a multi-professional group working under the oversight of NICE, leading to advice to NHS commissioners and clinicians on clinical cost-effectiveness.
     
  This process will in due course be extended to the most significant of existing interventions. This annex focusses on step 3 of the overview, the application of objective criteria to decide which interventions should be submitted for appraisal.
     
General principles
     
Objectives
     
3 The main objective of the selection process is to focus the resources (both in terms of expertise and money) available for appraisal onto those interventions where guidance will be of most benefit to the NHS and patients.
     
4 At the same time, the selection process must be
     
  i demonstrably even-handed - in particular between products directly competing for the same "market", but also between broad disease or patient areas. For instance, we would not want the process to be applied more rigorously to life-saving treatments than to those for chronic conditions (unless this could in turn be justified in terms of some overarching criterion);
  ii as far as possible transparent and objective, taking into account factors which are relevant to the judgement to be made and setting aside irrelevant factors.
     
Suggested principles
     
5 Bearing these objectives and constraints in mind, the government proposes that the selection should be based on the following principles:
     
  i overall significance to the national health. The appraisal process is intended just as much to promote the faster dissemination of products likely to achieve worthwhile health gain as to discourage the dissemination of ones which are not clinically cost-effective. Before the appraisal, by definition, we do not know whether benefits are likely to outweigh costs or not. However, it may be possible to make informed estimates of either or both of
 

a) the total potential NHS cost of using the intervention to treat the condition for which it is intended; and

 

b) the maximum likely benefit (including non-NHS benefits) of treating the condition.

  ii

significant management challenge in effective introduction of the intervention. Some innovations, however desirable in principle, may nevertheless require careful attention by NHS management if they are to be successfully introduced. This is not just a question of securing financial resources (though that may be an issue at least in the short term) but also the potential impact on other, non-financial NHS resources including

  • specialist diagnostic services
  • hospital inpatient services
  • GP services
  • emergency services.
  iii likelihood of significant misallocation of resources in the absence of guidance. Other things being equal, guidance will be more valuable if it "adds value", ie encourages clinicians and patients to take better informed and more consistent decisions than they would have done unaided. The following are likely to be the most common circumstances in which guidance could have significant impact:
 

treatments for life-threatening or disabling conditions for which no good treatment yet exists, and for which any new treatment is likely to arouse enormous expectations (whether or not justified by actual evidence of effectiveness);

 

treatments where the most readily measurable clinical endpoints do not readily translate into measures of patient well-being, ie where there is uncertainty or controversy over the fundamental clinical significance of the measured effects;

 

treatments over which there might be serious ethical concerns;

   

treatments where there is serious doubt whether they are appropriate for NHS funding for the generality of patients;

   

existing treatments where there is controversy over best practice or where there is evidence of significant departures from best practice.

     
Operational criteria
     
6 For practical use, each of the suggested principles in para 5 will have to be turned into a usable criterion. The following paragraphs suggest a possible approach.
     
Step 1: total NHS cost of treating the condition
     
7

This should be relatively straightforward. Total NHS cost for 100% treatment of the condition can be written as

Cost = Population with condition x proportion able to benefit x unit cost

where the unit cost will either be cost per treatment or cost per annum depending on whether the treatment is for acute episodes or treatment of a chronic condition. Of the factors in the equation

  i the relevant population can (in most cases) be estimated;
  ii the proportion able to benefit should normally be estimated at 100% unless there is good evidence that only a subgroup are likely to be treated, and that clinicians in practice would be able to limit treatment to this sub-group;
  iii for an existing treatment, the market-weighted average of treatments already on the market could be used. For an innovative treatment, the sponsoring company would be asked to give a broad figure or range for the unit cost.
   
Step 2: maximum benefit
     
9 The following questions will be considered
  i are any non-financial resources (eg specialist manpower, infrastructure) likely to be a constraint on introduction on the scale potentially indicated?
  ii is this a breakthrough treatment for a group of patients for whom there is no current effective treatment available, and who are otherwise at risk of death or significant disability?
  iii

are there likely to be significant uncertainties over the interpretation or significance of the clinical evidence, which in the absence of guidance could lead to variations in clinical practice - for instance over

a) the magnitude of the expected clinical benefits

  • the relation between the clinical outcomes measured by the trial and the clinical outcomes of real significance to patients or carers
  • the value which patients/carers/society place on the clinical benefits?
  iv are there likely to be ethical concerns over the use of the treatment?
  v is there any doubt over whether this is a suitable treatment for NHS funding, for the generality of patients?
   
Step 4: making decisions
     
  The intervention will be provisionally selected for assessment if
  i estimated total NHS costs exceed a given threshold; or
  ii costs are below that threshold but still significant and other factors indicate substantial value to the NHS and patients from appraisal; or
  iii the scale of the potential health gain, together with other factors if relevant, indicates substantial value to the NHS and patients from appraisal.
     
Updating
     
11 As explained in the main paper, the provisional selection will be reviewed from time to time as the intervention comes closer to launch, in the light of comments from industry, the NHS and patient groups and as more information becomes available. It is expected that some of the initial uncertainties would be resolved, ie that a more definite answer could be given to some of the questions at steps 1-3, and that as a result some of the overall assessments may change. The final selection will be made using information available about 1 years before the expected launch of the intervention.