Celgene, the company that makes pomalidomide, did not provide sufficient evidence of the effectiveness of the drug compared with current care. Even by using the company’s analyses, the drug, also known as Imnovid, did not offer enough benefit to justify its high price.

Multiple myeloma is a type of cancer that affects plasma cells, a type of white blood cell found in the bone marrow. Although it is incurable, there are a number of treatment options to help slow the progress of the disease and improve quality of life. The choice of treatment would differ for each individual, but NICE recommends thalidomide for most patients as a first line treatment. NICE also recommends bortezomib for people who are unable to take thalidomide and also as an option after the first treatment has stopped working. In people who have had at least two prior therapies NICE recommends lenalidomide. This appraisal considers the use of pomalidomide for treating multiple myeloma after third or subsequent relapse.

Commenting on the draft guidance, Sir Andrew Dillon, NICE chief executive, said: “Multiple myeloma is an incurable disease, but NICE-recommended treatment options thalidomide, bortezomib and lenalidomide – which can slow the progress of the condition and help relieve symptoms – have greatly improved the length of time someone can live with the disease and their quality of life.

“We are disappointed not to be able to recommend pomalidomide in this preliminary guidance, but the analyses submitted by Celgene, the company that makes the drug, did not show how well the drug works compared to the other treatments available. Also, Celgene’s own cost effectiveness analysis clearly demonstrated that the drug does not offer enough benefit to justify its price.”

Consultees, including the manufacturer, healthcare professionals and members of the public are now able to comment on the preliminary recommendations which are available for public consultation. Comments received during this consultation will be fully considered by the Committee and following this meeting the next draft guidance will be issued. 

Until final guidance is issued, NHS bodies should make decisions locally on the funding of specific treatments. Once NICE issues its final guidance on a technology, it replaces local recommendations across the country. 

Ends

For more information call the NICE press office on 0300 323 0142 or out of hours on 07775 583 813.

Notes to Editors

About the draft guidance

1. Pomalidomide, in combination with dexamethasone, is not recommended within its marketing authorisation for treating relapsed and refractory multiple myeloma in adults who have had at least 2 prior treatments, including lenalidomide and bortezomib, and whose disease has progressed on the last therapy.
2. The draft guidance will be available at /guidance/indevelopment/GID-TAG448 from 15 October 2014. Embargoed copies of the draft guidance are available from the NICE press office on request.
3. The Committee concluded that because of the design of the MM 003 study, the extent of the benefits associated with pomalidomide was uncertain, and that the MM 003 results were of limited value in comparing pomalidomide with ‘established practice without pomalidomide’ as specified in the scope of this appraisal.

1. Pomalidomide is administered orally. The recommended dosage is 4 mg once daily, taken on days 1 to 21 of repeated 28 day cycles. Treatment should continue until disease progression. Adverse reactions may be managed by interrupting or reducing the dose, as specified in section 4.2 of pomalidomide’s summary of product characteristics. The price of a pack (21 tablets) of 1 mg, 2 mg, 3 mg or 4 mg tablets is £8884 (excluding VAT). Costs may vary in different settings because of negotiated procurement discounts.
2. The cost per QALY gained (Quality Adjusted Life Year) presented by the company was over £50,000 compared with bortezomib. The Committee heard from the clinical expert that although there is no standard of care for people with relapsed and refractory multiple myeloma, bendamustine was likely to be the most commonly used therapy in this setting in England. When comparing pomalidomide with bendamustine plus thalidomide and dexamethasone, all cost per QALYs presented were over £70,000.
3. The Committee was not persuaded that the estimates of the extension to life were robust, objective or plausible based on the company’s economic modelling. It concluded that pomalidomide did not fulfil the criteria for being a life-extending, end-of-life treatment. The Committee further concluded that even had pomalidomide fulfilled these criteria, the weight that would have to be placed on the QALYs would be too high for pomalidomide to be considered a cost-effective use of NHS resources.

7. Pomalidomide is not recommended for use within NHS Scotland (http://www.scottishmedicines.org.uk/SMC_Advice/Advice/972_14_pomalidomide_Imnovid/pomalidomide_Imnovid).  
8. The company estimates a patient population of 669 in England.