How NICE decides which drugs to make available on the NHS

Dr Melinda Goodall gave NB Live delegates an insight into the complexities of deciding whether eye health treatments should be made available on the NHS. Mary McDonald reports.

With a limited pot of money for NHS health care, we have to make hard choices about how best to spend it. The National Institute for Health and Care Excellence (NICE) is the independent body which makes these tough decisions.

If NICE recommends a new more expensive treatment for one group of patients, something else has to give. Turning it down, however, risks denying a treatment to patients who might have benefited from it.

Dr Melinda Goodall of NICE explained the body aims to make decisions that will improve the health of the whole population in England.

So how do you prioritise between a cancer treatment that prolongs life for a few, an asthma intervention that could help millions, and a new drug to treat an eye condition that could improve the quality of life of thousands of older people?

What is a NICE technology appraisal?

Goodall explained, “NICE evaluates existing and new treatments by a process called a technology appraisal. A “technology” is generally a drug treatment, but could be any intervention with the potential to improve health.”

How for example, Goodall asked NB Live delegates, would you decide between funding one very expensive treatment or funding an ECLO service to help people newly diagnosed with sight loss?

In recent years NICE has approved many treatments for eye conditions such as macular degeneration and macular oedema. Some are recommended for use only with patients who meet particular criteria.

Goodall explained how NICE go about it: “Our aim is to review the clinical and economic evidence, and then make recommendations on the appropriate use of both new and existing medicines. A single treatment can be reviewed in isolation but sometimes NICE looks at all the treatments for a particular condition, including any new ones, and then recommends the most cost effective approach.”

Considering the health impact

The first step is to gather evidence to get a full picture about a proposed treatment. NICE talks to anyone who will be affected by the decisions: patients, manufacturers of the treatment, and the manufacturers of any treatment that might be displaced by the proposed new one, and to clinicians and other professional groups affected.

An independent academic group reviews the evidence, and the public are consulted. An appraisal committee of clinicians, statisticians and health economists consider the issues. All this is orchestrated by NICE staff, who provide the tools to help make these complex decisions.

Goodall stressed how powerful patient statements can be, “as it is vital that the appraisers understand the impact of having a condition, and of having or not having the treatment.”

Patient statements for eye conditions can spell out the practical effect of losing your sight: on everyday independence, looking after yourself, being able to read or go out.

How cost effective is it?

NICE number crunchers need a formula that can compare the cost effectiveness of very different treatments, whether it’s a hip replacement or an injection to stop the deterioration in vision. Their aim is to fairly prioritise the treatments that will most benefit the health of the nation.

“The appraisal takes into account the cost not only of the drug but also of the nurses and doctors’ time in administering and managing the treatment and any special equipment needed. The appraisal also looks at the likelihood and cost of managing any side effects. And to balance this, it looks at the benefits to length of life and quality of life and the impact of any adverse events (side effects).”

So to compare two treatments, NICE calculates the difference in cost (the extra spend), and divides it by the difference in effect (the extra benefit). This is known as an incremental cost effectiveness ratio or ICER.

Quality of life or longer life?

For eye treatments the health benefit is more likely to be improved quality of life, whereas a cancer treatment might extend someone’s life. To compare two such different benefits, NICE calculate a figure called a Quality of Adjusted Life Year (QALY).

In crude terms the more it costs to achieve a QALY, the better the argument has to be for NICE to approve a treatment. While there is no fixed threshold, in practice treatments that cost less than £20,000 per QALY are likely to be considered cost effective.

Treatments costing between £20,000 and £30,000 would raise more questions. NICE looks carefully at the certainty of the outcomes, whether the quality of life issues have been measured adequately, and if it offers innovations that bring benefits. An innovation for an eye treatment might be that less frequent injections of a new drug achieve a good outcome, so more patients could be treated within current clinic capacity. Treatments costing over £30,000 per QALY need increasingly strong cases for them to become recommended treatments.

Social value judgements

Goodall made it clear that it isn’t all about money. Social value judgements are important considerations too: “Appraisers are mindful of reasons why society might be willing to pay for a treatment.”As examples, Goodall asserted that appraisers would appreciate that sight is the sense that people most fear losing, and that there is strong public support for treatments that might save a child’s life.

Pricing deals

In addition pharmaceutical companies can propose patient access schemes to the Department of Health to improve the cost effectiveness of a treatment. These confidential agreements guarantee a discount when the drug is sold to the NHS. Patient access schemes often help a medicine to meet NICE’s cost effectiveness criteria. Lucentis was approved with a patient access scheme to treat wet age-related macular degeneration and diabetic macular oedema.

From clinical trial to reality

A particular challenge when appraising new treatments, is that NICE only has the limited information arising from clinical trials. Uncertainty about prevalence of a condition or the frequency of treatment, can have enormous cost implications when the treatment is made available to the population at large. NICE looks at the clinical trial data and produces mathematical models to predict the likely costs.

In the case of Lucentis there was uncertainty over how many injections patients were likely to need in the first few years of treatment. Mathematical modelling proved that even if patients needed four more injections than included in the model, the treatment would still remain under the £30,000 per QALY.

Impact of eye treatments

When Lucentis was appraised there was clear evidence that patients’ visual acuity improved when treated with it. Some improvement was also seen with laser treatment. But there was insufficient evidence that combining Lucentis with laser treatment would result in an improved outcome.

Clinicians told NICE that they believed that the laser treatment is likely to have longer term benefits. If evidence proves this in the future, it would be important to reappraise the recommended treatment for diabetic macular oedema.

An added complexity for eye treatments is that the quality of life benefits a patient experiences when their best seeing eye is treated, can be very different from the benefit gained by treating their worst seeing eye. Again mathematical models help NICE to predict which treatments will have the greatest impact.

Where a treatment is recommended, NICE publishes guidelines outlining who it is for and how treatment should be delivered and managed. “The easiest way to find NICE guidelines for eye conditions,” said Goodall, “is to go to the NICE pathways for eye conditions.”

She summed up by encouraging delegates to respond to consultations, and to encourage patients to contact NICE with their impact stories.

Dr Melinda Goodall is a Technical Adviser in the Technology Appraisals team at NICE.

Further information

NICE guidelines for eye conditions.