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Yesterday (Thursday 29 November), the National Institute for Health and Clinical Excellence (NICE) opens a public consultation on its preliminary recommendations on the NHS use of the drug, pirfenidone (known commercially as Esbriet). The draft guidance assesses whether it should be recommended for use in people who have the lung condition, idiopathic pulmonary fibrosis.

Idiopathic pulmonary fibrosis is a progressive disease associated with scarring of the lungs, making breathing difficult. Each year around 4000 adults in the UK are diagnosed with idiopathic pulmonary fibrosis.

Pirfenidone is an oral medication that has a UK marketing authorisation for the treatment of mild-to-moderate idiopathic pulmonary fibrosis in adults. It is believed to slow down the decline in lung function. After evaluating the evidence provided by its manufacturer, InterMune, NICE's draft guidance does not recommend pirfenidone for treating mild-to-moderate idiopathic pulmonary fibrosis. This is because there are uncertainties over its clinical benefits in the long term, and based on the available evidence, it would not be a cost-effective use of NHS resources.

The draft guidance recommends that any adults who are taking the drug should have the option to continue on the treatment until they and their doctors consider it appropriate to stop.

Professor Carole Longson, Director of the Centre for Health Technology Evaluation at NICE said: “Our committee found that pirfenidone seemed to have a modest but measurable effect on slowing the decline in lung function, but that it was uncertain whether this benefit persisted over time because the clinical trials were short in duration. Also, it was unclear whether the drug could improve overall survival for people with the disease because of uncertainty about the correct classification of deaths in the clinical trials as being related to idiopathic pulmonary fibrosis or not, a low number of deaths in both treatment and placebo arms and short follow-up.

“Given these uncertainties and when compared with best supportive care, our committee concluded that treatment with pirfenidone would not represent a cost-effective treatment option for the NHS. Therefore, our draft guidance does not recommend its use for mild-to-moderate idiopathic pulmonary fibrosis.

“We encourage healthcare professionals, people with idiopathic pulmonary fibrosis and other relevant third parties to comment on our committee's provisional recommendations during this consultation period so that they can contribute to the development of this guidance.”

Those wishing to comment on NICE's draft recommendations have until 5pm on Wednesday 19 December to do so. NICE's independent committee will meet again in January to review the comments received. NICE then expects to publish its final guidance for the NHS in April 2013.

If NICE's recommendation does not change once the final guidance publishes, this will mean that NHS settings in England and Wales will not be legally required to allocate funding for its use as it is not cost effective.

Until NICE issues its final guidance, decisions regarding the use of pirfenidone for idiopathic pulmonary fibrosis should continue to be made at local NHS levels.

Notes to Editors

About the draft guidance (appraisal consultation document)

1. To download the appraisal consultation document of pirfenidone for treating idiopathic pulmonary fibrosis, please visit the NICE website.

2. For further information about idiopathic pulmonary fibrosis, please visit NHS Choices

3. Pirfenidone is an oral medication that is manufactured by InterMune and marketed as Esbriet. It has a UK marketing authorisation for treating mild-to-moderate idiopathic pulmonary fibrosis. Pirfenidone is believed to slow down the progression of idiopathic pulmonary fibrosis by decreasing fibroblast growth, reducing the production of proteins and cytokines that are associated with fibrosis, and by reducing the response to growth factors.

4. The recommended dosage of pirfenidone is 3x267 mg capsules taken 3 times a day. Assuming no wastage, the annual cost of ongoing treatment with pirfenidone is £26,171.72. This excludes procurement discounts that local NHS settings may have negotiated, as well as the patient access scheme that InterMune agreed directly with the Department of Health. InterMune has requested for the size of this discount to be confidential.

5. NICE's independent committee concluded that the incremental cost effectiveness ratio (ICER) for the comparison of pirfenidone and best supportive care in patients with mild-to-moderate pulmonary fibrosis was in excess of £36,300 per QALY gained and was considered to sit at the lower end of a very wide range (the manufacturer has requested that this ICER remains confidential). This is greater than the £20,000-30,000 range that NICE would typically deem to be a cost-effective use of NHS resources. For further information about how NICE measures cost effectiveness, please visit the NICE website.

6. NICE's committee heard from clinical specialists that life expectancy for mild-to-moderate idiopathic pulmonary fibrosis would typically exceed 2 years. The committee concluded that treatment with pirfenidone did not fulfil NICE's ‘end of life' criteria. The ‘end of life´ criteria are supplementary pieces of advice that committees consider when appraising treatments that may extend the life of patients with a short life expectancy and that are licensed for indications that affect small numbers of people with incurable illnesses.

7. NICE technology appraisals are intended for NHS settings in England and Wales. The Scottish Medicines Consortium expects to publish guidance for NHSScotland in February 2013.

About NICE

1. The National Institute for Health and Clinical Excellence (NICE) is the independent organisation responsible for providing national guidance and standards on the promotion of good health and the prevention and treatment of ill health

2. NICE produces guidance in three areas of health:

• public health - guidance on the promotion of good health and the prevention of ill health for those working in the NHS, local authorities and the wider public and voluntary sector
• health technologies - guidance on the use of new and existing medicines, treatments, medical technologies (including devices and diagnostics) and procedures within the NHS
• clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.

3. NICE produces standards for patient care:

• quality standards - these reflect the very best in high quality patient care, to help healthcare practitioners and commissioners of care deliver excellent services
• Quality and Outcomes Framework - NICE develops the clinical and health improvement indicators in the QOF, the Department of Health scheme which rewards GPs for how well they care for patients

4. NICE provides advice and support on putting NICE guidance and standards into practice through its implementation programme, and it collates and accredits high quality health guidance, research and information to help health professionals deliver the best patient care through NHS Evidence.