EXECUTIVE SUMMARY

  Sanofi-aventis endorses the aims of NICE, which is to provide national guidance for more equitable access to modern treatments in England and Wales, on the basis of cost effectiveness rather than affordability. NICE in a relatively "youthful" organisation which has undergone a constant process of review since its inception, including the Health Select Committee (HSC) Inquiry of 2002.  The recent introduction of the new Single Technology Appraisal (STA) process is to be particularly welcomed. However, as with any new process, there are some issues and inconsistencies that need to be addressed.

  Implementation of NICE guidance remains subject to regional variations, and a mechanism should be found to ensure that NICE guidance is fully implemented across England and Wales.

  Full engagement in all elements of NICE's work, and that of other UK HTA agencies, is extremely resource intensive for all stakeholders. Serious consideration should be given for a review on the suitability of the current arrangements for HTA in the UK. This includes whether three independent review bodies add value or whether a single UK body might be more appropriate. An expanded NICE, with a national remit to assess all products at or prior to launch, must only be considered in close collaboration with other stakeholders.

INTRODUCTION TO SANOFI-AVENTIS

  1.  Sanofi-aventis is Europe's largest pharmaceutical company, and ranked number three in the UK. Backed by a world-class R&D organization, Sanofi-aventis is developing leading positions in seven major therapeutic areas: cardiovascular medicine, thrombosis, oncology, internal medicine, metabolic disorders, diseases of the central nervous system, and vaccines.

  2.  Our portfolio of medicines mirrors the priorities of the National Health Service which are to: reduce mortality from heart disease; reduce mortality from cancer; reduce mortality from suicide; reduce adult smoking rates and tackle obesity and support people with long term conditions. We also have an equally exciting pipeline of new medicines and are well placed to help meet the present and future needs of patients and healthcare professionals alike.

  3.  Sanofi-aventis is committed to the UK, as shown by our investment in six sites in the UK, which reflect the full range of product life cycle activities—from pre-clinical development of new molecules through to manufacture and distribution, marketing, regulatory compliance, product education and professional support.

  4.  Sanofi-aventis (as both legacy companies and as Sanofi-aventis) has extensive experience of NICE. We have undergone twenty-two NICE appraisals, and Sanofi-aventis contributes as a stakeholder to the development of several other appraisals and guidelines.

NICE'S EVALUATION PROCESS

  5.  The last HSC inquiry into NICE (2002) made several recommendations which resulted in various refinements to NICE's evaluation process. These improvements are welcome but we still have concerns about some key phases of the appraisal processes:

Topic Selection Process

  6.  Sanofi-aventis welcomes the recently revised topic selection process. However, we have concerns that this new process is not sufficiently transparent and needs further refinement. Having introduced new topic consideration panels, the Institute now publishes the notes of their meetings on its website. However, in their present form the notes are insufficiently detailed and too vague, in respect to pharmaceutical technologies, to understand why the decisions were made in the first place. Furthermore, the role and quality assurance of the National Horizon Scanning Centre in Birmingham in the new process is not clear.

  7.  There is extensive consultation between NICE and the manufacturer about the draft remit and scope of a particular technology appraisal, before the topic selection process is completed, and the Minister decides, which technologies are selected for inclusion in the next wave of appraisals. The consultation on "minded" referrals is a very useful exercise but, overall, the current process can take many months before final referrals are made. This can therefore result in increased delay to the assessment of new technologies and is inefficient for the Institute.

  8.  Following the scoping exercise, NICE and the Department of Health, collate the feedback and prepare a recommendation to Ministers on which technologies should be included in the next wave. The recommendations are not released to stakeholders meaning there can be no transparency as to the rationale and criteria behind the recommendations. Ultimately, when a wave is announced, it is often unclear why certain technologies have been selected for appraisal, and why certain assessment processes—multiple, or single technology appraisal process or guidelines—are considered most appropriate for the technologies in question.

  9.  We believe that attention should be given to further reducing the time taken to make final referrals to the Institute. Furthermore, greater transparency regarding the methodology, rationale and criteria for the topic selection process, including publication of the conclusions and recommendations put to Ministers, should be introduced.

  10.  There should be more transparency regarding the methodology, rationale and criteria for the topic selection process, including publication of the conclusions and recommendations that are put to Ministers.

  11.  Time taken in the topic selection process should be reduced by having the Department of Health and Ministers take final decisions on topic selection within one month of publication or each wave.

Single Technology Assessments (STAs)

  12.  We welcome the new STA process. It is hoped that the newly introduced STA process will speed up evaluations by the Institute, and result in the more speedy and efficient publication of guidance to the NHS and healthcare professionals when it is both appropriate and evidence permits. However, producing this guidance should not be at the cost of full, thorough and open consultation. The Expert Review Group (ERG) report, which is an assessment of the manufacturer's submission and forms the basis of the Consultation panel's deliberations, is not made available to manufacturers before it goes for consideration by the assessment panel. Industry should be given the opportunity to make representations about the ERG's report to the Consultation Panel. Currently the only way to raise any issues is at the ACD or appeal stage. A more collaborative approach, earlier in the process, would be beneficial to all parties and would lead to more efficient decision making.

  13.  A more collaborative approach should be sought for STAs, to enable industry to make representations about the ERG report, prior to a decision being made.

  14.  As a relatively new process, we appreciate that many elements of the STA methodology are yet to be fully tested in practice. A key example is the actual process to define the "decision problem". This element of the process requires further clarity to set out how it works in practice. Particularly when the decision problem submitted by a manufacturer is not accepted by the Institute in itself, which has an impact on further negotiations on the timelines for appraisal

  15.  Greater clarity is required around the process of establishing the "decision problem" in the STA process.

SPEED OF PUBLISHING GUIDANCE

  16.  Sanofi-aventis recognises that as NICE's work programme increases, it will need to work hard to provide the NHS with holistic guidance that updates, cross references and reflects the interdependent nature of NICE's different work programmes. Therefore, the speed of publication of guidance may vary significantly, and may be subject to delay when the Institute is developing related technology appraisals and clinical guidelines in parallel.

  17.  Given the continual expansion of the Institute's work programmes, further transparency is required to clarify how related streams of work interact and influence one another. For instance, how a guideline development group and an appraisal committee should work together in developing their recommendations. Clearly, the two groups should work in close collaboration where remits overlap, but there is no explicit process as to how this should take place. For example, at which stages in the production of the guidance/guidelines do the two groups collaborate? How should any disagreements or conflicts be resolved? What contingencies are in place if the two groups are unable to reach agreement?

  18.  Greater clarification is required to define how the apraisal development groups and the guideline developing group should work together.

APPEAL SYSTEM

  19.  Despite changes implemented as a result of the recommendations in the last HSC inquiry, Sanofi-aventis continues to question the transparency of the appeal process. The appeal panel continues to be chaired by the Chairman of NICE and is predominantly staffed by non executive directors of the Institute. Limited public places are now available for the actual appeal hearing (which is a step in the right direction), but the panel's deliberations are still held in private. This end-stage of the appraisal process is therefore neither compatible nor consistent with the Institute's stated aims of transparency.

  20.  Additionally, it is at the discretion of the Appeal Panel Chair as to how an appeal is heard. Sanofi-aventis has noted that this occasionally results in inconsistency between appeals which is a disadvantage to NICE stakeholders.

  21.  The Appeal panel's evidence and deliberations should be fully transparent and made public within a reasonable and consistent time frame.

NICE COMPARISON WITH SCOTTISH MEDICINES CONSORTIUM (SMC) AND ALL WALES MEDICINES STRATEGY GROUP (AWMSG)

  22.  NICE's remit covers England, Wales and Northern Ireland. In parallel, the AWMSG plans to prepare its own guidance for all cardiovascular and oncology technologies at launch, as well as for other existing high cost, low volume technologies. The SMC is responsible for producing guidance for Scotland, although NHSScotland will recognise MTA guidance, but currently not STA recommendations. This situation is confusing and potentially perpetuates the so called "post code lottery". HTA stakeholders in the UK are required to deal with three separate bodies, each with different purpose, requirements and methodologies. This is time consuming, resource intensive and results in duplication of effort. Sanofi-aventis recommends that a review be undertaken to consider the suitability of the current arrangement and assess whether three independent review bodies add value or whether moving towards a single UK body might be more appropriate.

  23.  A review should be undertaken to consider the suitability of the current arrangement and assess whether three independent review bodies adds value.

IMPLEMENTATION OF NICE GUIDANCE

  24.  Whilst we welcome the establishment of NICE's Implementation Directorate and the increased prioritisation of support for implementation, we remain concerned that implementation of NICE guidance can vary widely across the country. We hope that the Health Select Committee will propose solutions for NICE guidance to be fully implemented, in a fair and equitable fashion, across the country and the NHS.

  25.  NICE has put significant resource into establishing its own in-house implementation team to provide support to PCTs on the practical and financial considerations of implementation. This is admirable given that NICE has no formal jurisdiction to ensure implementation of its guidance. However, it is difficult to assess how effective the in-house NICE Guidance Implementation Team has been to date. This is because variation in the implementation of NICE guidance across the country remains striking.

  26.  A thorough review should be undertaken to assess the impact of the NICE implementation team, and the value to the NHS of its outputs.

  27.  NICE has recently initiated a new work programme to review ineffective practice in the NHS. Whilst supporting the programme's overall objectives, Sanofi-aventis questions whether it is appropriate to label products as "ineffective" before they have been fully assessed. Furthermore, it is unclear why a separate work programme is required to assess these products and why guidance could not be achieved using the multiple technology appraisal process which is already in place and well established.

  28.  Finally, Sanofi-aventis request that the Institute clarifies its process for selecting ineffective practice review topics. The current topics in progress were selected in-house by the Institute which is inconsistent with its topic selection process for all other work programmes.

  29.  There should be a review of the ineffective practices programme to consider whether its aims could be achieved under the existing technology appraisal processes.

SUMMARY OF RECOMMENDATIONS FOR ACTION

—  There should be more transparency regarding the methodology, rationale and criteria for the topic selection process, including publication of the conclusions and recommendations that are put to Ministers.

—  The topic selection process should be reduced and Ministers required to make final decisions regarding topic selection within one month of publication of each wave.

—  A more collaborative approach should be sought for STAs, to enable industry to make representations about the ERG report, prior to a decision being made.

—  Greater clarity is required around the process of establishing the "decision problem" in the STA process.

—  Greater clarification is required to define how the appraisal development groups and the guideline developing groups should work together.

—  The Appeal Panel's deliberations should be transparent and made public within a reasonable and consistent time frame.

—  A review should be undertaken to consider the suitability of the current arrangement and assess whether three independent review bodies (SMC, AMWMSG and NICE) adds value.

—  A thorough review should be undertaken to assess the impact of the NICE implementation team, and the relative effective change it is bringing to the NHS.

—  There should be a review of the ineffective practices programme to consider whether its aims could be achieved under the existing technology appraisal processes.

—  There should be greater transparency and clarity around the review of the ineffective practices programme.

Sanofi-aventis

March 2007