NICE, the health­care guidance body, has issued new draft guidance on the use of lena­lido­mide (Revlimid, Celgene) for treating the blood cancer multiple myeloma after one prior treat­ment with bor­tez­o­mib. With the current in­for­ma­tion avail­able, the committee's pre­lim­i­nary guidance (now being issued for consultation) does not recommend lena­lido­mide for this indi­ca­tion [1].

Multiple myeloma is a type of cancer that develops from cells in the bone marrow and there is cur­rently no cure for the disease, only treat­ments to stop the progress of the con­di­tion and help relieve symp­toms. Although the choice of treat­ment would differ for each individual, a thalido­mide regi­men would be con­sidered suitable for most patients as a first line treat­ment. Bortezomib is also recommended by NICE as an option for people who are unable to take thalido­mide. This appraisal con­siders the use of lena­lido­mide as an option for people whose disease has re­lapsed once after receiving one treat­ment with bor­tez­o­mib, and are unable to receive thalido­mide and for whom bone marrow trans­plan­ta­tion is not appro­pri­ate.

Commenting on the draft guidance, Sir Andrew Dillon, NICE Chief Executive said: “In the original guidance published in 2009, NICE recommended lena­lido­mide as treat­ment option for multiple myeloma in people who have received two or more prior ther­a­pies. This recom­men­da­tion has not changed and patients at that stage in the treat­ment path­way will still be able to receive lena­lido­mide through the NHS.

“Since then, NICE has also recommended bor­tez­o­mib as a first line treat­ment. We are now looking specifically at how well lena­lido­mide works after someone has received bor­tez­o­mib, and whether it provides value for money. However, from the in­for­ma­tion provided by the manu­fac­turer it was unclear if lena­lido­mide was as effective as re-treatment with bor­tez­o­mib and the manu­fac­turer's own economic model showed that the drug would not be cost effective at this stage - because of this we are unable to recommend the drug in pre­lim­i­nary recom­men­da­tions. We hope that the manu­fac­turer, Celgene, will look again at their sub­mission.”

“Since we recommended lena­lido­mide in 2009, no clin­i­cal studies have been under­taken specifically looking at how well lena­lido­mide works compared to other treat­ments for these patients. Celgene only presented robust trial data com­par­ing lena­lido­mide to a placebo, not to other chemo­ther­a­pies and there is very little evi­dence in the pop­u­la­tion we are cur­rently appraising this drug for.”

In the guidance published in 2009 the manu­fac­turer of lena­lido­mide submitted a patient access scheme, where they bear the costs of the drug beyond 26 cycles (normally for a period of 2 years) which enabled the Committee to be able to recommend the drug. No patient access scheme has been submitted for this new patient group in this current appraisal.

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.

Explanation of terms

1. Lenalidomide in combination with dexamethasone is not recommended for treating multiple myeloma in people: who have relapsed once, and who have received 1 prior treatment with bortezomib, and for whom thalidomide is contraindicated or cannot be tolerated and for whom bone marrow transplantation is not appropriate.

This appraisal is a part review of TA172: Lenalidomide for the treat­ment of multiple myeloma in people who have received at least one prior ther­apy. http://publications.nice.org.uk/lenalidomide-for-the-treatment-of-multiple-myeloma-in-people-who-have-received-at-least-one-prior-ta171

About the guidance

1. The draft guidance will be available at http://guidance.nice.org.uk/TAG/452 from 14 March 2014.
2. The Committee considered all the cost effectiveness models submitted by the manufacturer to be fundamentally flawed because of several issues. The Committee concluded that the most plausible costs per QALY for lenalidomide compared with bortezomib or standard chemotherapies were more than £30,000, whether bortezomib retreatment was or was not appropriate.
3. The manufacturer did not provided any evidence to indicate the drug would meet the standards to be considered under the end-of-life criteria.
4. Lenalidomide is available as a 21-capsule pack. The cost per pack varies according to capsule size: £3570 (5 mg), £3780 (10 mg), £3969 (15 mg) and £4368 (25 mg). The recommended starting dose is 25 mg orally once daily on days 1-21 of repeated 28 day cycles..
5. The Scottish Medicines Consortium has not published advice on the use of lenalidomide for this group of patients as yet: http://www.scottishmedicines.org.uk/SMC_Advice/Forthcoming_Submissions/lenalidomide_Revlimid_
6. The decision to partially review TA171 was made in response to comments from Celgene which suggested that an update of TA171 was required as the marketing authorisation for lenalidomide also includes treatment after one prior therapy. This was not originally considered during the development of TA171 but since the publication of TA171, NICE has recommended first line treatment with bortezomib in TA228 for people who are unable to tolerate or have contraindications to thalidomide.

About NICE

The National Institute for Health and Care Excellence (NICE) is the independent body responsible for driving im­prove­ment and excellence in the health and social care system. We develop guidance, standards and in­for­ma­tion on high-quality health and social care. We also advise on ways to promote healthy living and prevent ill health.

Formerly the National Institute for Health and Clinical Excellence, our name changed on 1 April 2013 to reflect our new and addi­tional responsibility to develop guidance and set quality standards for social care, as outlined in the Health and Social Care Act (2012).

Our aim is to help practitioners deliver the best possible care and give people the most effective treat­ments, which are based on the most up-to-date evi­dence and provide value for money, in order to reduce inequalities and variation.

Our prod­ucts and resources are produced for the NHS, local author­i­ties, care providers, charities, and anyone who has a responsibility for commissioning or providing health­care, public health or social care services.

To find out more about what we do, visit our website: www.nice.org.uk and follow us on Twitter: @NICEComms.

Source: NICE.