Select Committee on Health Appendices to the Minutes of Evidence


Memorandum by Sheila M Bird (NC 11)

  Individual submission from first statistician-member of NICE's Appraisal Committee. For potential conflicts of interest, please see publicly available Declarations of Interest re NICE.

  I preface my remarks by paying tribute to the tremendous enthusiasm, dedicated hard work and intellectual openness of the professional and administrative staff at NICE; and to Professor Barnett's splendid, indefatigable chairmanship of the Appraisal Committee. Members commit time, energy and thought because rigorous appraisal of the cost-effectiveness of technologies is an emerging, exciting, applicable science. Methodologies are being researched and importantly refined, including by the pharmaceutical industry, in response to the challenges that NICE presents.

  Appraising the cost-effectiveness of future technologies demands that attention is paid now, both by sponsors and by the National Health Service, to the design and data requirements of cost-effectiveness information systems [C-EIS]. Typically, C-EIS goes beyond randomised controlled trials of short-term efficacy: it shines a new cost-effectiveness spotlight on the statistical and economic modelling of disease progression. Critically, ethical consideration needs to be given to patients' informed consent to data acquisition for C-EIS, which may entail the salient use of disease registry of NHS costs data or access to anonymized individual patient data from industry or other pivotal randomised trials that predate the C-EIS conception. My recommendations are:

Recommendation 1: Invocation of Medicines Act

  When research is commissioned by NICE to improve the cost-effectiveness evidence base on which the Appraisal Committee will make its decision, NICE should be able to invoke the powers of the Medicines Act so that the research team has access to anonymized individual patient data from pivotal randomised controlled trials. NICE-commissioned research into cost-effectiveness of disease-modifying treatment of multiple sclerosis was not accorded full access by all pharmaceutical companies. Consideration should be given to more general circumstances than NICE-commissioned research when NICE may invoke disclosure of data under powers similar to the Medicines Act.

Recommendation 2: NICE forum for sponsors to pre-discuss Cost-Effectiveness Information System [C-EIS]

  Appraisal of cost-effectiveness uses information sources beyond the pivotal randomised controlled trials on which licensing was approved on the basis of quality, safety and efficacy in the short-term. In particular, costs, utilities and longer-term effectiveness may be assessed outwith the original trials, and the latter may be sensitive to the chosen statistical model for disease progression as well as to the choice of data sources by which to estimate the model's parameters.

  Model and data uncertainty, as outlined, mean that the challenge of appraising cost-effectiveness is much greater than for efficacy. The methodologies, including Bayesian model criticism, for doing so are only just being developed for practical application. Pharmaceutical companies, many of which have responded impressively to the challenge, should therefore have a forum at NICE for pre-discussion of the cost-effectiveness model that they intend to use and of the data sources by which they will derive parameter estimates for their proposed model: in short, a forum for pre-discussion of their proposed cost-effectiveness information system [C-EIS], just as USA's Food and Drugs Administration engages in pre-discussion of the design of a sponsor's pivotal randomised controlled trials.

Recommendation 3: Operational criteria for selection of technologies for appraisal at NICE

  The least transparent, and therefore potentially the least scientifically defendable, aspect of NICE Appraisal is how the selected technologies are chosen. Scientifically-sound publicly-disseminated operational criteria should be developed for the sifting of technologies for NICE Appraisal. Each proposed topic should be documented [for example on NICE website] as to: date of proposal, proposer, date of classification, classifier, actual classification on each of the operational criteria for sifting, date of selection decision, triage decision [NICE Appraisal recommended, pended, rejected].

  I suggest that, once defined, the new objective procedure should be applied in retrospect to the last 500 proposed topics that were filtered informally by health departments and Ministers. Moreover, post-appraisal classification by NICE on each of the operational criteria should be compared with the departments' publicised pre-appraisal classification for technologies which were subject to NICE Appraisal so that feedback improves the operational criteria or their implementation, as necessary.

Recommendation 4: Alternative to NICE Appraisal

  Since members of Appraisal Committees are unpaid, their and their employers' generosity of time and effort should not be squandered on inappropriately selected topics, but used to the maximum benefit of patients and staff in the National Health Service.

  Alternative to NICE Appraisal should therefore be available to Ministers when, for example, an appraisal topic is identified with high NHS budget impact or pre-appraisal guesstimated high cost but low effectiveness on which there are not even any properly conducted, relevant randomised trials of efficacy. In addition, the attention of UK Royal Colleges and of the Medicines Control Agency, or its European counterpart, should be drawn to such topics if licensing approval in the last decade has been given on non-randomised evidence.

Recommendation 5: Enhanced data quality by the date of NICE re-appraisal

  If Appraisal Committee has made recommendations for an enhanced cost-effectiveness evidence-base, then consideration needs to be given by Ministers to whether re-appraisal of cost-effectivess should proceed (or alternative mechanism be invoked) if there has been no enhancement by the sponsor or others in the evidence-base by the re-appraisal date.

January 2002

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