Shortfalls in the way the National Institute for Health and Clinical Excellence operates have compromised public confidence, and could permanently disadvantage patients in England and Wales from benefiting from the major, incremental medical innovations available in other countries, Parliament has been warned.

As the House of Commons Health Select Committee begins hearings in its second inquiry into NICE, the Association of the British Pharmaceutical Industry has told it that the bar to a positive recommendation from NICE is becoming unrealistically high, and there is also pressure on the agency to take decisions on the basis of very limited clinical- or cost-effectiveness data.

In its written evidence to the panel, the ABPI stresses its particular concern about the following aspects of NICE procedures: over-reliance on cost/QALY (qualify of life-adjusted years) in making (rather than informing) decisions; expectations of certainty in decision-making that cannot be achieved, particularly early in a product’s lifecycle; issues of quality and consistency in the work commissioned from Assessment Groups/Evidence Review Groups (AGs/ERGs), including transparency; and an “unnecessarily adversarial” approach to industry within some AGs/ERGs.

These issues result in an increasing trend to resort to an appeal process that is itself seriously flawed, and which has resulted in the past two years in no significant changes to NICE guidance, the industry group adds.

“No doubt there are frustrations for all in the relationship between NICE, AGs/ERGs and industry,” the ABPI acknowledges. It calls for a more constructive and less adversarial approach, which should include, from the outset of each appraisal, dialogue between manufacturers, NICE and AGs/ERGs on the methods, data quality and sources used.

Among the ABPI’s other recommendations are: for the industry to be invited to participate in Appraisal Committee meetings to answer questions and provide clarification on areas of uncertainty; an independent review of the quality of Assessment/ERG Reports; and a root-and-branch review of the appeal process.

There also needs to be: a review of NICE guidance implementation, with recommendations that result in tangible action; “joined-up” commissioning and financial incentives; the automatic inclusion of NICE-approved medicines in local formularies; and mandatory funding for medicines, where reviews of technology appraisals have been moved into clinical guidelines and positive recommendation remains, the group adds.

In total, the Commons inquiry has received written evidence from 92 individuals and groups including government, industry groups and individual manufacturers, the Royal Colleges and other academic bodies, and patient associations.

Among the latter is the Alzheimer’s Society, which is participating next month in the first-ever High Court challenge to a NICE ruling. This seeks to overturn the current NICE decision that patients in the earlier stages of Alzheimer’s disease (AD) should not have access to the four drugs which are licensed to treat the condition - Pfizer/Eisai’s Aricept (donepezil), Novartis’ Exelon (rivastigmine), Shire’s Reminyl (galantamine) and Lundbeck’s Ebixa (memantine).

NICE is being challenged when it is felt that its decisions are poor because these have a huge impact on people’s lives, and a major reason for challenges is the fact that they do not give sufficient weight to evidence from patients and professionals, the Society has told the inquiry.

Health economic analysis must be placed in the context of people’s lives and real-life clinical practice; however, in the Society’s experience, little weight is given to evidence submitted by people with experience of the drugs. “NICE failed to consider that the evidence from patients and carers might be considered on its own merits rather than as pointers for the committee to consider further published research. In addition, the knowledge and experience of clinicians with expertise in the disease area has also been ignored, resulting in guidance that does not translate easily to clinical practice,” it says.

Appeals process slammed

The lack of transparency also leads to challenges, according to the Society, which notes that, during the appraisal of AD drugs, it had been impossible to understand how a number of decisions were reached. Also, little explanation was given as to how the appeal panel reached its decisions and why points raised by appellants were ignored. An independent appeals process needs to be developed, and more detail on how the appeal panel reaches its decisions should be provided to appellants, says the Society.

Pfizer, the lead claimant in the upcoming judicial review, has also provided written evidence to the Select Committee inquiry. In it, the company echoes the ABPI’s call for drugmakers to be permitted to attend appraisal committee meetings, with the same rights as all other stakeholders, and also calls for committee hearings to be held in public.

NICE should revise its appeal process so that it considers the merit of the decision as well as the process, and the appeal process should be conducted and chaired by an independent panel, adds Pfizer, whose evidence also calls for NICE to:

– broaden the perspective of appraisals so they include costs and savings to patients, caregivers and wider society;

– commit to establishing a process (which must include the pharmaceutical company) for defining the evidence expected to be necessary at time of approval to demonstrate cost-effectiveness and value;

– recognise explicitly the inherent limitations of using QALYs to measure the value of medicines; and

– recognise the inherent uncertainty in all economic evaluations and not place undue emphasis on these results when developing guidance.

Fears for innovation

Pfizer also urges the Select Committee to examine the impact of health technology assessment (HTA), as currently practiced by NICE, on the sustainability of pharmaceutical innovation in the UK.

HTA discriminates against innovation in disease areas where current practice is a relatively cheap, off-patent medicine, such as in many areas of cardiovascular disease, mental health and respiratory illness, it says. The argument that this situation creates incentives for pharmaceutical companies to concentrate research in areas of unmet clinical need, rather than where generics are already available, is flawed for two reasons, it adds.

First, the notion that, if treatments exist within a given disease area, there is no longer clinical need, is simply false. Cardiovascular disease is still the biggest killer in the western world - and increasingly a major problem in the developing world – yet treatments exist. More and better treatments are needed in cardiovascular medicine, not fewer. Secondly, associating value only with breakthrough pharmaceutical innovation is fundamentally flawed. Normally, medicines evolve incrementally over time and, within a new area of medical innovation, the first medicine is rarely the best.

Unless this is addressed, there is a real prospect that no new medicines will be introduced in the UK into disease areas where common practice is to use off-patent medicines and, by 2020, this could realistically extend to all disease areas, warns Pfizer. By Lynne Taylor