Select Committee on Health Minutes of Evidence

Annex 3



Understanding of the Therapeutic Area and/or Use of Medicine

    —  Is the therapeutic area one where medical practice and/or the use of medicines are rapidly changing? [Rapid change increases the technical complexity of appraisal, but may also increase the potential value to the NHS of early advice. Alternatively, rapid change may render early advice redundant within a short time period, and may militate against early or any appraisal.]

    —  Is understanding of the use of medicine to treat the disease well or poorly developed, or is the medicine the first to be used for this condition? Are there any effective non-pharmaceutical interventions for the condition? [These points are related. In areas where there are no previous pharmaceutical treatments comparisons will necessarily be against non-pharmaceutical interventions (or against best supportive care) and may be more difficult. Moreover, appropriate outcome measures relevant to assessment of clinical effectiveness may not yet have been developed. However, the potential value to the NHS of early advice may be greater for genuine therapeutic breakthrough products.]

    —  Is the medicine the first/second/third/subsequent to be introduced in its class? [In general, any difficulties over appropriate choice of outcome measures etc are most likely to arise with the first in class. Depending upon the timing of introduction of subsequent entrants, these difficulties are likely to be resolved by followers in the class.]

Potential Indication and Patient Population

    —  Is the indication for the most refractory patients, e.g. third line treatment, or in other respects for a more restricted patient population than is likely to be the case later? Are subsequent indications or changes in formulation likely to have greater impact on numbers of patients, on quality of NHS care, and on NHS resource use? [It is very common, in particular with cancer and other life-saving treatments, for companies to seek an initial indication for "rescue" therapy and to go on to seek further indications for earlier disease. In general, the evidence base for earlier disease stages is likely to be more comprehensive (e.g. Randomised Clinical Trials on overall survival rather than non-randomised studies of tumour response rates).]

Data Considerations

    —  Is there a shared understanding of appropriate endpoints for this condition - if not are improvements in prospect? [This is most likely to be an issue with new treatments in therapeutic areas where there has up till now been no effective intervention. As already noted, these may also be circumstances in which early advice to the NHS may be at a premium.]

    —  How relevant are the end-points in the data which are expected to be available at launch to an assessment of clinical and cost effectiveness?

    —  Do these endpoints relate to the expected length of treatment? If not, are the available endpoints likely to be good predictors of long-term clinical and cost effectiveness?

    —  What plans are there to collect further post-registration data relevant to clinical and cost effectiveness? How far will the available data be relevant to clinical and cost effectiveness in the UK setting? How quickly are these data likely to be available? [These questions are closely related. The underlying issue is whether the nature of the condition or the specific intervention make it inherently difficult to collect information appropriate to the assessment of clinical and cost effectiveness at time of launch; and if so whether there are active plans to collect such information post launch.]

    —  Would early appraisal affect the conduct of further research, and if so, how? [In some circumstances early appraisal might reduce the incentive to additional research or - conceivably - make it more difficult ethically to carry out. In other circumstances, early appraisal could lead to advice which would positively promote further research in the NHS.]

Service Provision

    —  Is service provision well developed for the therapy area?

    —  Is the introduction of the new pharmaceutical likely to have a significant impact on the way in which services in this therapy area are delivered, and if so how accurately can this impact be predicted? [These questions are closely related. It may be desirable to appraise interventions likely to cause major impact on the delivery of services. However, if predictions of service impact are founded on assumptions with a high degree of uncertainty, this may make it difficult to produce robust guidance at time of launch.]

January 2002

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Prepared 8 July 2002