Transparency in health technology assessments

The running battle over which National Health Service patients with dementia should have access to the dementia drug donepezil (Aricept) is to progress to the courts. Last year, guidance from the National Institute for Health and Clinical Excellence (NICE) restricted use of the drug to patients with moderate and severe Alzheimer's disease, thereby denying its use for 60% of patients with Alzheimer's disease who have mild dementia.1

NICE's decision was based on modelling Aricept's clinical and cost effectiveness through a contract with Southampton University's Health Technology Centre. NICE makes its own internal work accessible to the drug industry so that its processes are open to critical appraisal. Furthermore, it requires industry to supply all its evidence in an “executable” form, so that differing assumptions can be modelled. However, NICE's appraisal guide clearly states that the contracted work of external academic assessment groups, such as the Southampton University's Health Technology Centre, will be available just in a “read only” form in which different modelling assumptions cannot be re-run.2 NICE argues that this is essential to protect the intellectual property rights of assessment groups. This lack of transparency has never been challenged before, and although NICE's rules might be noble, protection of just this part of the assessment process may be unwise. However, with the resources at their disposal industry might be able to replicate the model from the read only version.

Shire, in conjunction with the Japanese biotechnology company Eisai, which owns the licence for donepezil, have challenged the failure of NICE to provide access to the Southampton model in a judicial review.3 NICE has expressed regret at the high opportunity cost of this challenge, which will divert its resources into legal processes rather than technology assessment. It is determined to defend its position robustly.4

This conflict, and its narrow focus on the one area where NICE lacks transparency, may be an attempt by the drug industry to enhance its profits from a marginally cost effective drug. It might also be part of a more subtle drive to undermine processes of assessing health technology, which are designed to ensure the delivery of effective treatments to patients and value for money to tax payers.

Australia has a similar system of assessing the cost effectiveness of drugs and devices and attempts have also been made to undermine its processes. The Australian Pharmaceutical Benefits Scheme was the first systematic attempt to develop health technology assessment to ensure that health systems adopt cost effective interventions. Its creation in 1993 was contentious for the drug industry, as the industry is a strong advocate of free trade, even though it is protected by patents and other regulatory devices that enhance monopoly power and profits. Some academics and former committee members of the Pharmaceutical Benefits Scheme fear that it will be undermined by pressure from the United States. For instance, negotiations over the 2005 free trade agreement between the US and Australia focused on removing regulatory barriers to trade such as technology appraisal epitomised by the Pharmaceutical Benefits Scheme.5 The US government is arguing that technology appraisal, such as that in the Australian Pharmaceutical Benefits Scheme, creates obstacles to the free trading of drugs as it bars some products from reimbursement and affects their “free market” price to the detriment of producers. The recent visit to the United Kingdom of the US deputy secretary for health has raised similar concerns in the UK. Mr Azar was quoted as saying that “mechanisms such as those of NICE for rationing drugs to keep costs down stifle innovation,” and concern was expressed that this was part of the efforts of drug companies “to have unrestricted access to the NHS as part of a free market.”6

The work of NICE is essential if resources are to be targeted towards patients who will benefit the most. Generally its processes are transparent and sensible. However, the constraints under which it works can be improved.

Firstly, NICE currently has to accept the product prices set by industry and is unable to bargain them down to more reasonable levels that might facilitate acceptance and use by the NHS. Price setting is influenced by the Pharmaceutical Price Regulation Scheme, which offers industry the freedom to set prices and protect target profit levels of 17-21% return on capital. The Office of Fair Trading has criticised the Pharmaceutical Price Regulation Scheme and advocated pricing in relation to “economic value.”7 Calculation of economic value may deflate future drug prices, and if NICE were given this role the need for transparency in its decision making would be important.

While NICE may estimate the financial consequences on the NHS of its guidance, it is assumed that primary care trusts can fund all its guidance. This is clearly incorrect as implementation will be uneven and produce new forms of postcode rationing.8 Also, NICE guidance is increasing as the growth of NHS funding is declining. In setting NICE's work agenda, ministers need to prioritise removing inefficient technologies rather than adding new technologies to the NHS.9

NICE is an essential institution for improving the efficiency of the NHS. It will never be perfect because evidence from clinical trials and economic modelling may be corrupted by poor science in the practice of clinical and economic evaluation.10 11 12 This makes transparency essential; surely it would have been better to have compensated Southampton for its loss of property rights in its model of treatment for Alzheimer's disease rather than become entangled in litigation?

With the NHS seeking to control expenditure and target the use of drugs to improve the health of the population in a cost effective manner, and industry wanting to maximise its profits, conflict is inevitable. It is essential that the trade off between health and wealth is managed with transparent and good science by all participants—both public and private.