Public funding of new cancer drugs: Is NICE getting nastier?

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Abstract

Background

Decision-making processes that determine cancer drug availability vary internationally. The National Institute for Health and Clinical Excellence (NICE) assesses clinical and cost-effectiveness, but recent restrictions on the availability of cancer drugs suggest that NICE may be getting tougher.

Objectives

To determine whether NICE is rejecting a higher proportion of cancer drugs and whether the reasons for restricting technologies have changed.

Methods

NICE decisions on cancer drugs from May 2000 to October 2008 were classified as ‘positive’, ‘restricted’ or ‘negative’, and decisions taken before and after a change in NICE’s appraisal methods in August 2006 were compared. NICE’s stated reasons for its restrictions were analysed.

Results

Fifty-six cancer drugs in 38 appraisals were analysed. The proportion of ‘negative’ appraisals increased from 4% in period 1 to 27% in period 2. Findings were similar when analysed by drug assessment (11% versus 26%).

Conclusions

The higher rejection rate for cancer drugs is partly explained by the new appraisal process, but the principal reason for the observed change is the shift from an absence of evidence on cost-effectiveness to evidence of an absence of value-for-money.

Introduction

Throughout the developed world, the access to, and affordability of, expensive, potentially life-saving new cancer drugs is of keen interest and concern to patients, the public and policy makers.1, 2, 3, 4 In response, some jurisdictions have adopted special funding mechanisms for cancer drugs. For example, Denmark earmarks grants for high priority areas such as cancer treatments;5, 6 in Belgium, high-cost inpatient cancer drugs are funded by increased drug budgets or reimbursed separately;7 in Canada, the Province of Ontario has established the ‘New Drug Funding Programme’ to reimburse hospitals and cancer centres for certain new and expensive intravenous cancer drugs;8 Ireland9 and New Zealand10 also operate special schemes for cancer treatments. These features reflect countries’ willingness to pay for cancer treatments as well as the need to manage financial risk and avoid destabilising local health economies.

The methods used to decide whether cancer drugs will be reimbursed or paid for from the public purse are also subject to wide international differences.11, 12 In most jurisdictions there is no separate evaluation process for cancer drugs, but some countries, such as Norway and Sweden, explicitly consider disease severity in the decision-making process,13, 14 which may give life-saving treatments a higher profile. Canada has pioneered a Joint Oncology Drug Review,a although recommendations are not binding upon individual provinces, which make their own funding decisions.11

Within the United Kingdom (UK), several bodies make decisions on National Health Service (NHS) funding for new drugs, but none uses a separate process for cancer treatments. The Scottish Medicines Consortium (SMC) advises local health bodies in Scotland on the use of all newly licensed medicines, all new formulations of existing medicines and major new indications for established products. Pharmaceutical manufacturers are required to make a submission before a product is launched. The SMC considers whether the evidence indicates that the product will offer good value-for-money to the NHS in Scotland, then issues advice as soon as practical after the product’s launch. The SMC may choose to accept, restrict, or reject use of a product.15 If manufacturers fail to submit evidence, the SMC advises against use in NHS Scotland.16

The National Institute for Health and Clinical Excellence (NICE) issues guidance on the use of drugs and other health technologies for the NHS in England and Wales. The process used by the institute is lengthier and more complex than that followed by the SMC. For multiple technology appraisals (MTAs), NICE seeks to issue guidance within 12–15 months from the start of the process. In contrast, the single technology appraisal (STA) process, which only considers individual technologies, should take around 6 months and guidance should be issued within 13 weeks of marketing authorisation.17 The evidence informing MTAs and STAs also varies: both involve the assessment of evidence from manufacturers, but MTAs also incorporate an analysis from an independent academic group.18 Whilst NICE rejects the use of an absolute cost-effectiveness threshold, higher cost per QALY (quality-adjusted life-year) is associated with a lower likelihood of positive guidance,19 and a threshold in the order of £20,000–30,000 per QALY gained is deemed acceptable.20 Directions issued by the Secretary of State for Health in December 2001 require local health authorities to make funding available for interventions recommended by NICE within three months of guidance being issued.21 The directions also place a duty on NHS hospital trusts to co-operate with this process, but there is no statutory obligation on physicians to prescribe treatments recommended by NICE. The directions should encourage uptake, but geographical variations in access suggest that compliance is non-uniform.22

A previous analysis examined all NICE’s published appraisals of cancer drugs up to June 2006.23 Over that 6-year period, there had been 23 appraisals of cancer drugs and just one appraisal had been completely ‘negative’: NICE guidance was that the drug was not to be available on the NHS. It therefore, appeared that cancer drugs had ‘fared quite well’ under NICE.23 Over the following year, NICE rejected a higher proportion of cancer drugs,24, 25 but the small number of appraisals precluded robust conclusions. This paper reports an updated analysis, which aims to explore whether there has been a change in NICE guidance on cancer drugs, and, if so, why.

Section snippets

Materials and methods

We reviewed all published appraisals of cancer drugs up to October 2008 and extracted data on NICE’s decisions using two units of analysis: technology appraisals and individual drug assessments. Appraisals can cover more than one drug and each drug considered within an appraisal is counted as a separate drug assessment. For example, NICE appraisal number 33 assesses three drugs for advanced colorectal cancer.26

We analysed the recommendations and classed them as positive, negative or restricted.

Results

From May 2000 to October 2008, NICE published 38 appraisals covering 56 cancer drug assessments. This included 9 STAs of cancer drugs, all of which were published during period 2. Table 1 gives the details of how NICE recommendations on cancer appraisals and cancer drugs varied between the two time periods. Fig. 1 shows the percentages of appraisals and of drug assessments that were positive, negative or restricted. Relative to period 1, the proportion of negative recommendations increased in

Discussion

It seems that NICE is making negative decisions on cancer drugs more often, although this trend cannot currently be confirmed at the conventional level of statistical significance. The principal reason for this appears to be a shift in the nature of the evidence base for cost-effectiveness on cancer drugs. Consequently, NICE is more often citing value-for-money as a reason for limiting availability of these drugs.

There are a number of limitations to this research. First, statistical testing

Conclusions

Mechanisms for determining the availability of cancer drugs vary internationally; NICE exemplifies a system of decision making based on assessments of clinical and cost-effectiveness. NICE appears to be making negative decisions on cancer drugs more frequently. Although this could be a chance finding, it may represent a trend. The new appraisal process of STAs explains only part of the observed changes, but it is clear that the nature of the evidence base for cost-effectiveness has shifted from

Funding

The authors received no funding for the research reported in this paper. There was no requirement for ethical approval.

Authorship

M.D. conceived the original idea for the study. A.M. developed the idea, extracted and analysed the data and wrote the original draft. M.D. critically revised the draft and refined the interpretation of the data. Both authors have approved the final version of the paper.

Conflict of interest statement

The authors have worked on technology assessment reviews, for which the Centre for Health Economics at The University of York receives funding from NICE. A.M. and M.D. have received funding for research from, and undertaken consultancy projects for, several pharmaceutical companies. M.D. is also Chair of one of NICE’s Guideline Review Panels.

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