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Keywords:

  • enforcement;
  • multiple technology appraisal;
  • NICE;
  • single technology appraisal

Introduction

  1. Top of page
  2. Introduction
  3. Technology Assessment within NICE
  4. Timeliness and the Introduction of Single Technology Appraisal (STA)
  5. Impact
  6. Closing Remarks
  7. Question Period

Health technology assessment to determine relative cost-effectiveness has been in operation in the UK since 1999. Although Scotland has its own separate and independent processes for assessing drugs and health technologies, the main agency undertaking these activities is the National Institute for Health and Clinical Excellence (NICE), an independent organization responsible for providing national guidance on promoting good health and preventing and treating ill health. Premised on a fixed budget set by the Treasury, NICE was not founded to save money, but rather to make the best use of the money available, to eliminate inequalities in access to care, and to promote best practices through the development of clinical guidelines. As a result, the past decade has realized a buildup of infrastructure and the development of systematic processes that have facilitated the engagement of governments with the findings of the research community such that active knowledge transfer is affecting the decisions of all types of government agencies in the health field.

NICE Activities

Importantly, NICE does not report through the Department of Health, but advises the National Health Service directly. That is, it advises directly those statutory bodies responsible for purchasing and providing care. Because of the implications for spending, the decisions of NICE may be used to justify future changes to health-care budgets through the working of regular spending reviews by the Treasury.

NICE is organized around four programs:

  • 1
    Technology Assessment Program—assesses the effectiveness and cost-effectiveness of drugs and devices (diagnostics about to be added).
  • 2
    Public Health Program—assesses the effectiveness and cost-effectiveness of public health interventions (includes educational initiatives targeted at improving health outcomes).
  • 3
    Interventional Procedures Program—assesses the effectiveness (but not cost-effectiveness) of surgical procedures, interventional diagnostics, etc.
  • 4
    Guidelines Program—development of best practice guidelines (based on technology assessment when necessary and taking intermittent cognizance of costs).

In addition to these four programs, there is also a research arm and an implementation arm. Only aspects of the Technology Assessment Program are discussed here.

Technology Assessment within NICE

  1. Top of page
  2. Introduction
  3. Technology Assessment within NICE
  4. Timeliness and the Introduction of Single Technology Appraisal (STA)
  5. Impact
  6. Closing Remarks
  7. Question Period

Processes

The Technology Assessment Program was the first program to be initiated and covers both drugs and devices, although the majority of appraisals to date have been of drugs. Some older and some new (but not all) drugs have been evaluated by this program. Historically, the review, synthesis of evidence, and economic modeling of the technology under appraisal has always been undertaken by NICE, which outsourced the work to one of seven university Assessment Teams in a process now known as a “Multiple Technology Appraisal.” Drug companies were invited to submit a drug value dossier that was one component of the analysis and review of evidence relating to the effectiveness and cost-effectiveness of the drug that was conducted by the commissioned team. The university teams produced high quality publications on technologies and methods as a result of these activities and the work was attractive to the groups concerned.

Working in conjunction with the Assessment Team, NICE developed the pertinent questions about the drug that were to be addressed within the context of existing technologies, a process known as scoping. Scoping defined the nature of the decision problem to be considered and the comparisons to be included through extensive dialog between NICE and the drug companies. A set of clinical or patient guidelines or a review of patient flow under the current standard of care was often a typical starting point for the discussion, with the key questions being how the new technology would fit in to the existing patient flow, and what the key comparators were within the existing system. Although drug companies were able to input into the process of defining the decision questions, NICE was ultimately responsible for ensuring the assessment was comprehensive and unbiased.

Once the scope was defined, the Assessment Teams were then responsible for producing the final drug assessment documentation. This documentation then formed the basis for The Appraisal Committee to reach a decision on whether the drug was to be supported (and therefore reimbursed) within the National Health Service via a three-stage process of preliminary decision, consultation, and final decision (plus the possibility of appeal by interested parties). Of note, this approach meant that the burden of proof that a drug was or was not cost-effective lay with NICE rather than with the drug companies themselves.

Despite some concerns that the assessment process was not transparent enough and inevitable press criticisms, by international standards the process is very transparent. All reports, comments, responses, and the basis for decision are published. Indeed, it appears the reports are very widely read, with about 4 million hits on the reports on the website per year with many hundreds of thousands of downloads, approximately half of which are in North America. In fact, NICE has earned a good reputation with stakeholders because of the perceived robustness and transparency of its processes.

Decisions

On examining the history of decisions, the majority of new devices and new drugs that have been appraised have been supported by NICE to some degree. However, most usually, the recommendation is more restrictive than the label. In general, NICE will further define the circumstances in which the use of the technology is appropriately cost effective; for example, by specifying its use in particular patient groups, or in specified situations.

Politics

Health care is overwhelmingly tax-financed in the UK and the constraints of a fixed budget means that in some cases, the decisions made by NICE are highly contentious. Not surprisingly, this makes NICE vulnerable to political, media, and lobbyist pressures. For example, recently, in response to parliamentary pressure, NICE adopted policy changes to accommodate access to some very expensive end-of-life drugs targeted at prolonging the life of cancer patients by a few months. Subsequently, however, the House of Commons Health Select Committee proffered harsh criticism of the changes. Given the divergent interests of the stakeholders, which include government, local Commissioners of Health Services, drug companies, and the public, it would seem inevitable that NICE will be subject to constant criticism. However, on the positive side, as a conduit, NICE has brought to the fore a very public discussion about the appropriate threshold of cost-effectiveness for medical interventions.

Timeliness and the Introduction of Single Technology Appraisal (STA)

  1. Top of page
  2. Introduction
  3. Technology Assessment within NICE
  4. Timeliness and the Introduction of Single Technology Appraisal (STA)
  5. Impact
  6. Closing Remarks
  7. Question Period

Although NICE had achieved much credibility during its first 5 years of operations, by 2005, both parts of the drug industry and some politicians identified slow NICE procedures to be significantly contributing to slowing access to new drugs (despite by far the biggest contribution to tardiness in NICE pronouncing on new drugs being delay by Health Ministers in deciding to refer the drug for consideration). The Institute was required to develop faster systems. In response, NICE introduced a shortened appraisal process known as STA, whereby a company with a new drug or a new device is invited to make a submission to NICE, which is then evaluated close to the time of licensing. The highly structured and methodologically guided submissions are now sent to university-based review teams for commentary and critique. Unlike the earlier system, which continues in parallel, the burden of proof that a new drug or device is cost-effective lies with the manufacturer, who must present the case supporting the effectiveness and cost-effectiveness of the technology? This allows NICE to reject technologies, rather than look for evidence to support their use, if the case is not satisfactorily made, or, alternatively, to make limited recommendations such as for research use only, or a recommendation with evidence development.

The adoption of STAs has speeded the assessment process so that NICE is performing more evaluations and expects to be reviewing all new drugs close to the time of launch in the near future. However, although the process itself is faster, it appears that the use of STAs has in fact reduced the proportion of approvals. Furthermore, and of some concern, universities that provided analytical services in the past are much less interested in providing review services because reviews do not easily lead to quality publications. This resulted in some universities reducing their commitment or withdrawing their expertise altogether.

Impact

  1. Top of page
  2. Introduction
  3. Technology Assessment within NICE
  4. Timeliness and the Introduction of Single Technology Appraisal (STA)
  5. Impact
  6. Closing Remarks
  7. Question Period

It is clear that the results of NICE appraisals have a significant impact on product sales figures; if a drug or intervention is not recommended by NICE, for the most part, it will not be marketable in the UK. On the other hand, in part because local budgets have to accommodate new expenditures, a recommendation from NICE does not guarantee that a technology will be immediately adopted. Furthermore, it has become apparent that in some cases, technologies were never adopted despite a recommendation from NICE. As a consequence, in recent years, NICE has concentrated more on implementation, and it is now mandatory that local commissioners must show evidence of at least plans for implementation of interventions that have been recommended by NICE within 3 months of the guidance being issued.

Although difficult to prove cause and effect, it is widely believed that NICE appraisal has increased the uptake of a number of effective drugs in several disease areas including cancer, asthma, and coronary heart disease.

Closing Remarks

  1. Top of page
  2. Introduction
  3. Technology Assessment within NICE
  4. Timeliness and the Introduction of Single Technology Appraisal (STA)
  5. Impact
  6. Closing Remarks
  7. Question Period

It is noteworthy that NICE appears to have produced a quite remarkable and rather sophisticated awareness among health-care staff and the general public, of the issues in allocating health resources. This has led to a surprisingly large number of people engaging in the debate around the cost-effectiveness threshold that should be adopted. It may well be that this legacy of involvement is the essential element in our achieving successful decision-making about which health technologies we can afford.

Question Period

  1. Top of page
  2. Introduction
  3. Technology Assessment within NICE
  4. Timeliness and the Introduction of Single Technology Appraisal (STA)
  5. Impact
  6. Closing Remarks
  7. Question Period

Audience Question: Here in the US, we have significant numbers of pharmacoeconomists and university-based programs. When you talk about the buildup of infrastructure to facilitate the appraisal system, specifically, what kind of infrastructure are you talking about?

Author: My understanding is that currently the US does not have a decision-making strategy based around cost-effectiveness. So, it is not just a matter of having capacity in numbers of pharmacoeconomists and analysts, but it may be that some different kinds of skills will be necessary if you are going to try to address questions of cost-effectiveness.

Audience Question: If ensuring the best treatments were being applied across the UK rather than cost-effectiveness was initially the motivator for NICE, is cost containment now taking priority?

Author: Although, of course, cost containment is always important, NICE was not expected to produce cost savings. However with fixed budgets, implementation of NICE recommendations means that something else is displaced. Ultimately, it is the value of what is displaced that determines the threshold of cost-effectiveness. NICE therefore must ensure that their recommendations are better value than that which gets displaced.

Audience Question: My impression is that in the UK cost-effectiveness is more accepted as a policy tool or technique than it might be in Canada or the US. Could you comment on why that is?

Author: Well, I think it's certainly true that it's more accepted; we have very centralized budgetary systems that make it quite clear to people that there are fixed budgets and this draws attention to the fact that cost-effectiveness matters.

Source of financial support: None.