ABOUT BMS

  Bristol-Myers Squibb Pharmaceuticals Ltd (BMS) is a global research and development-based pharmaceutical company and one of the leading suppliers of both hospital and GP-prescribed medicines to the NHS. We develop and supply medicines for some of the NHS' highest priority treatment areas, including cancer, cardiovascular disease, severe mental illness and HIV and we are proud to have launched five new medicines into the UK in the last three years. Our business strategy is based around the development of innovative medicines in areas of greatest unmet medical need. Our medicines have been the subject of several NICE appraisals affording us significant experience of working with NICE, of its process and the impact of its guidance on patient care.

INTRODUCTION

  We welcome the decision of the Health Select Committee to conduct an inquiry into NICE, given increasing public concern about the availability of medicines in the NHS. It is becoming clear that NICE is, in effect, operating as a "fourth hurdle" and that medicines must demonstrate not only the quality, safety and efficacy required by the licensing authorities, but also "cost-effectiveness", as defined by NICE, before they can become widely available to patients. The perception in much of the NHS—if not the legal actuality—is that the funding of medicines is contingent on them being "approved" by NICE. To some extent this may represent opportunisitic behaviour by budget-holding primary care trusts constantly searching for financial economies. However, it is also indicative of the ambiguity of NICE's status which is central to the issues we have with it.

  NICE was established with the objective of accelerating the uptake of effective new technologies and of reducing or eliminating geographical disparities in their use (so called "postcode prescribing"). It has made some, but only limited, progress towards meeting these objectives for those technologies it recommends. Even here, however, the uptake of NICE guidance is patchy and incentives for implementing "positive" guidance are weak.

  Of even greater concern is that NICE would appear to have moved away from these objectives in more recent time. It now has an implicit, if not explicit, rationing function, where its recommendations on specific technologies stem directly from the question of what can be afforded, not what is best for patients. This, we believe, is not what Parliament intended, and we recommend that the Health Select Committee focuses its inquiry on the question of whether NICE has gone beyond its original remit in this way.

  We believe it is time Parliament called NICE to account and the Health Select Committee inquiry is an excellent way of doing this. The inquiry is even more timely in light of the recent report by the Office of Fair Trading into the Pharmaceutical Price Regulation Scheme. The OFT proposes a central role for NICE in defining a medicine's value, as a critical input into determining the price the NHS will pay for that medicine. It is important that the Committee fully considers the significance of this proposal as it conducts the inquiry.

  The OFT report is complex and far-reaching in its implications and it is too early to offer a full assessment of it at this stage. However, an initial observation is that while the principle of value-based pricing merits examination, the practicalities of how such a system will work are highly complex. In particular, we do not believe that NICE is ready and able to take on the enhanced role that the OFT envisages. In effect it would be asking NICE to run before it can walk, and consequently we believe the government should exercise great caution before moving in the direction the OFT proposes.

  We recognise the Committee's observation, reflected in the inquiry remit, that NICE's decisions are increasingly being challenged and that public confidence in NICE is waning. We believe there are a number of reasons for this which we set out and discuss below.

  The points highlighted in this submission are those that are of particular importance to Bristol-Myers Squibb. We are members of the ABPI and fully endorse the points made by the ABPI in its own submission to the enquiry, even if we do not specifically echo them below. In particular, we share the ABPI's concern about the consistency and quality of assessments carried out by the economic review groups (ERGs) as part of the STA process and about the lack of opportunity for companies to have dialogue with either the ERGs or the appraisal committee in the course of the review. Given the flaws in the NICE appeals process—also documented in the ABPI's submission and known to BMS through direct experience—anything that makes the process more inclusive, thereby helping to reduce the incidence of appeals, should be warmly embraced by NICE.

BMS COMMENTS

1.   The Role of NICE

—  Health technology assessment—the process of making judgements about the cost and clinical effectiveness of a medicine or medical technology—has an important part to play in informing choices made by doctors and patients. The stated purpose of NICE is to provide "national guidance" and BMS supports this role.

—  Increasingly, however, the way in which NICE is being interpreted is to undermine choice since, if NICE recommends against a technology, then it is extremely difficult, if not impossible, for that product to receive funding by the NHS. In effect, the "choice" is being made by NICE, not by the doctor or patient.

—  Moreover, a "yes" or "no" decision from NICE now centres around whether the technology is judged "cost-effective" against a "cost per QALY" threshold for which the maximum acceptable level is £30,000. There is also evidence of this threshold being driven closer to £20,000. This is not a cost-effectiveness threshold, since there is no law of health economics that says a technology suddenly becomes non cost-effective above a particular cost per QALY point (or cost-effective below it). It has become a de-facto affordability threshold, based on a judgement about what level of cost-effectiveness the NHS is prepared to pay for.

—  In summary, therefore, NICE is making judgements about what products should or should not be paid for by the NHS (and therefore reach patients), based on considerations of affordability. This is neither NICE's stated role (NICE, for example, refuse even to acknowledge that a threshold exists, though evidently it does), nor what Parliament envisaged. While NICE has a role in informing choice, to delegate choice to NICE in this way in anathema to broader government policy.

2.   The Accountability and Transparency of NICE

—  It is reasonable that an expert body such as NICE should carry out the technical function of assessing the clinical and cost-effectiveness of different medical interventions and making recommendations about their use in the NHS. It is not right however that NICE should be asked to make highly political decisions related to what the NHS can afford to provide for the population.

—  As noted above, NICE currently uses a cost per QALY threshold approach to determine when it does or does not approve a treatment for use. This threshold has simply emerged: it has never been mandated through any political or democratic process. We believe this is wrong given that decisions about patients' access to medicines revolve around this threshold-based judgement made by NICE. Such public policy decisions should be exposed to the full oxygen of public and political debate.

—  Although NICE is supposed to assess both the cost and clinical effectiveness of medicines and medical technologies, in practice it is a product's cost per QALY against threshold that determines the outcome. The balance of factors taken into account by NICE in appraising a technology is not made clear. Nor does the process acknowledge that assessing cost-effectiveness is an imprecise science, based on a variety of inputs and assumptions, and that for any product there is a range of plausible cost-effectiveness ratios. The manner by which NICE absorbs such imprecision yet is capable of making definitive and binding decisions about technologies is frequently unclear. This exposes the process to the suspicion that it is driven by subjective judgements (such as around affordability) rather than objective ones.

—  It is critical for public confidence in NICE that there is proper transparency in all its processes. In particular, it should be much clearer how the threshold is determined and, crucially, that the Government, rather than NICE, takes full accountability for it.

—  Many of these problems and concerns stem from the fact that NICE is not a truly independent body. It is part of the NHS, and therefore part of the organisation that has to fund its decisions. We believe this to be a conflict of interest that needs to be resolved if NICE is to command the confidence of the wider public.

3.   Cost-effectiveness and value

—  We have concerns that the appraisal methodology NICE employs does not properly consider a medicine's full value to the patient, to the NHS and to society more broadly. It is in part because of this that NICE's decisions are increasingly being challenged.

—  NICE's methodology is over-reliant on the cost per QALY as a measure of cost-effectiveness and, indeed, value. The QALY is in fact just one piece of evidence that should be considered as part of a full assessment of the value of a medicine.

—  Judgements about the value of a medicine to patients and the NHS should be taken on a broader basis, encompassing both the clinical benefits of the medicine as well as its cost-effectiveness. Factors that should be taken into account include, for example, whether the product fulfils a genuine unmet medical need, the availability of other treatments, the benefits that are of most importance to the patient and his or her carers and wider societal benefits and savings such as in social security costs.

—  We believe that the health technology assessment carried out by NICE should focus more on the additional clinical benefit to patients that the new medicine provides, and this should be given at least equal weight in forming the overall recommendation. A measurable assessment of the clinical benefit of the medicine separate to the cost per QALY assessment would add both clarity and balance to the process.

—  A potential model would be the French Amelioration du Service Medical Rendu, or ASMR, where the clinical benefit is expressed as a classification between 1 & 5, as follows:

1  =  major improvement, delivered to innovative product of significant therapeutic benefit, 2  =  important improvement, delivered to product of therapeutic benefit in terms of efficacy and/or reduction in side effect profile,

3  =  moderate improvement in terms of efficacy and/or reduction in side effect profile, delivered when already existing product, where equivalent pharmaceuticals exist,

4  =  minor improvement, and

5  =  no improvement

—  The problems associated with making definitive cost-effectiveness judgements on new medicines are exacerbated by the fact that the default mode for NICE is becoming an assessment of a product made at or around the time of launch. By definition, the full data required to form a rounded judgement of a product's full value are not available at this time: such data can only be gathered through extensive use of the product in a real-life setting. Long-term outcomes or survival data, for example, will not generally be available at this stage.

—  While we recognise the desire of the NHS to have guidance available close to when a new product is launched—a desire that, understandably, NICE wants to fulfil—it has to be recognised that all that can be produced at this early stage is precisely that—guidance. The problem with assessment at launch is that NICE is now making, in effect, once and for all value judgements about a new medicine at this stage. This is completely inappropriate, and has the effect of constructing a barrier to entry at launch that is damaging potentially both to patients and to incentives to innovate for the pharmaceutical industry.

—  The cost-effectiveness approach adopted by NICE takes almost no account of the fact that medicines designed to treat highly-specialised diseases will always be more expensive unit per unit because there is a much smaller patient base from which the company can recoup the costs of research and development. NICE has recognised the need for a different approach to cost per QALY analysis in the case of so-called "ultra-orphan" drugs (those affecting up to 1000 people in the UK). We believe that this needs to go further: if cost per QALYs are to play such a pivotal role (and, as we argue above, this is debatable) then at least there should be scope for adopting different thresholds for different products, depending on their type and patient base. The overall budgetary impact should be kept in mind in making these judgements.

4.   Assessment at launch

—  As set out above, we recognise the desire for NICE to issue guidance as close to launch as possible. We would point out however that there are real practical issues with proceeding too early, not least that the final licensed indication(s) is often not known until approximately two months prior to marketing authorisation. It is our experience that appraisals begun before this point cause significant problems for the manufacturer and can result in an unnecessary additional resource burden. We urge the Committee to call on NICE to be as flexible as possible in the timing of appraisals close to launch.

5.   Implementation of NICE guidance and patient access to medicines

—  When NICE has deemed that a treatment is cost-effective, the NHS is required to fund its availability to all appropriate patients. Unfortunately this is still very often not the case and many patients do not receive the best available treatments. There is significant variation in the availability of NICE approved medicines across the country.

—  Anti-platelets are one example of this phenomenon. Anti-platelets, of which aspirin is the most widely used, help to prevent patients with heart disease suffering a further heart attack or stroke. As such they are an important tool in preventing ill-health and death from cardiovascular disease, a key priority for the NHS. Despite this, there is a six-fold variation is usage of anti-platelets between PCTs nationally. This is a stark differential in prescribing of a proven and cost-effective class of treatments which have the potential to contribute to improved public health at a very reasonable cost.

—  We therefore believe the government and the NHS must focus more on what needs to be done to ensure that NICE approved treatments are made widely available to patients. In particular, the Healthcare Commission should be more rigorous in its annual assessment of NHS organisations in reviewing performance in relation to NICE guidance implementation.

6.   Availability of medicines that have not been reviewed by NICE

—  The NHS is increasingly using absence of NICE guidance as a reason not to make medicines available to patients. In many instances, PCTs will not pay for medicines that have not been reviewed by NICE, regardless of the evidence supporting the value of the product. More often, particularly where a medicine is new to the market, bureaucratic hurdles are put in place to make it difficult for clinicians to prescribe it. The result is that patients are denied treatments that could be beneficial to them—often for considerable periods of time if an appraisal overruns, as is sometimes the case, or if NICE decides not to review a particular treatment.

—  Medicines for conditions that are not government priorities are particularly likely to be refused funding. We are aware, for example, of patients with Hepatitis B, a serious and life-threatening condition, who have sought treatment with a new drug that has yet to be appraised by NICE and been told that their PCT will not pay for it.

—  The Department of Health recently re-issued guidance making it clear that lack of NICE guidance was not an acceptable reason to refuse to fund a medicine. It remains to be seen whether the guidance will have any impact. The Department must monitor this situation closely and ensure that patients requiring treatment with non-NICE reviewed medicines are not discriminated against in this way.

Bristol-Myers Squibb Pharmaceuticals Ltd

March 2007