In draft guidance out today (Thursday 21 March), the National Institute for Health and Clinical Excellence (NICE) states its intention to recommend pirfenidone (also known as Esbriet and manufactured by InterMune) as a treatment option for certain people receiving NHS-funded care who have the chronic lung condition, idiopathic pulmonary fibrosis. This differs to an earlier draft that NICE sought feedback on last November, which provisionally did not recommend the drug.

Each year around 4,000 adults in the UK are diagnosed with idiopathic pulmonary fibrosis. It is a progressive disease in which breathing becomes increasingly difficult because of scarring to the lung tissues.

Pirfenidone is an oral capsule taken three times a day. It aims to slow down the irreversible damage in the lungs caused by idiopathic pulmonary fibrosis.

In its latest draft guidance (called a final appraisal determination or FAD), NICE recommends pirfenidone as a treatment option across the NHS for people with idiopathic pulmonary fibrosis who have a forced vital capacity (FVC) between 50% and 80% predicted - FVC is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible.

NICE's positive draft recommendation comes after InterMune submitted additional evidence to demonstrate the potential benefits of the treatment in the group for whom it is recommended and also revised the terms under which it would make pirfenidone available to the NHS (i.e. its patient access scheme, which is agreed directly with the Department of Health).

NICE currently estimates that around 6,800 people in England and Wales with idiopathic pulmonary fibrosis could be eligible for treatment in line with its draft recommendations.

Professor Carole Longson, Director of the Centre for Health Technology Evaluation at NICE said: “We are pleased that InterMune provided further evidence during our public consultation and revised its terms to make pirfenidone available to the NHS. As a result, our independent committee concluded that the drug is a clinically and cost effective treatment option for certain people who have idiopathic pulmonary fibrosis in England and Wales.

“There is no cure for the disease and few effective treatments, and so our positive draft recommendation is good news for patients.

“We have published this draft as the last opportunity for our stakeholders to decide whether to appeal against our proposals before we issue final guidance for the NHS next month.”

NICE's draft guidance states that patients should stop taking the pirfenidone if their disease continues to worsen (i.e. if their FVC declines by 10% or more predicted over a twelve month period). Also, people who are currently taking the drug for their idiopathic pulmonary fibrosis but who do not fulfil the treatment criteria, should have the option to continue until they and their doctors consider it appropriate to stop.

NICE expects to publish its final guidance for England and Wales at the end of April. Once this happens, the guidance will replace decision-making at local levels across the NHS. NHS settings will have three months to allocate funding and resources for the drug.

Ends

Notes to Editors

About the draft guidance (final appraisal determination)

1. The final appraisal determination of pirfenidone for treating idiopathic pulmonary fibrosis will be available from 00.01 Thursday 21 March 2013. Embargoed copies are available on request from the NICE press office.

2. Further information about idiopathic pulmonary fibrosis is available from 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 revised 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 considered that InterMune's probabilistic incremental cost-effectiveness ratio (ICER) of £24,000 per QALY gained for the subgroup of people with FVC between 50% and 80% predicted was an acceptable cost-effectiveness estimate. Typically NICE deems treatments to be cost effective if their ICERs fall below £20,000 per QALY gained. When ICERs are higher than this, decisions will take account of the factors such as uncertainty about the estimate of cost effectiveness, whether the benefits have been captured in the QALY measurement and the innovative nature of the technology. 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. See the NICE website for further information.

7. NICE technology appraisals are intended for NHS settings in England and Wales. The Scottish Medicines Consortium is currently developing guidance for NHS Scotland.

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
• social care - the Health and Social Care Act (2012) sets out a new responsibility for NICE to develop guidance and quality standards for social care. To reflect this new role, from 1 April 2013 NICE will be called the National Institute for Health and Care Excellence (NICE) and it will become a Non-Departmental Public Body.

3. NICE produces standards for patient care:

• quality standards - these describe high-priority areas for quality improvement in a defined care or service area
• 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
• CCG Outcomes Indicator Set (formerly known as COF) - NICE develops the potential clinical health improvement indicators to ensure quality of care for patients and communities served by the clinical commissioning groups (CCGs)

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.