2002/043 Final appraisal determination on imatinib (Glivec) for chronic myeloid leukaemia
Issued: 12th August 2002
Final appraisal determination on imatinib (Glivec) for chronic myeloid leukaemia
The National Institute for Clinical Excellence has published a final appraisal determination (FAD) on the use of imatinib (Glivec) for chronic myeloid leukaemia (CML) on its website (www.nice.org.uk). NOTE: This document does not constitute formal guidance to the NHS.
The process NICE follows in ensuring that the guidance it issues to the NHS is robust. NICE produces two consultatitive documents, the first is an Appraisal Consultation Document that outlines the Committees initial thoughts on the evidence and the second is the FAD which takes into account feedback from the consultation on the ACD.
As part of its commitment to openness and transparency NICE publishes these documents on its website.
The FAD has been sent to the stakeholders involved in the appraisal so they can consider if they wish to appeal against the guidance. For this appraisal the appeal period closes at 5pm on Tuesday 27th August.
Andrew Dillon, Chief Executive, said, "NICE follows an open and consultative process to allow for the stakeholders involved in the appraisal to comment on the provisional recommendations of the committee. The committee consider these comments, along with any additional evidence, at the second committee meeting. We have always been clear that this means the recommendations in the final appraisal determination may differ from the provisional recommendations.
"The FAD on imatinib makes it clear that during the recent consultation period, the committee received further evidence and the assessment group carried out additional work. As a result of the consultation and subsequent evidence the recommendations of the Committee were changed."
He added "One of the reasons we were established was to look at technologies where there is genuine uncertainty regarding their value and to end this uncertainty by providing clear authoritative guidance for the NHS in England and Wales, once we have completed this process guidance will exist for imatinib explaining where the evidence suggests it should be used."
Notes for editors
- The process NICE follows to develop its guidance involves patients, health professionals, the manufacturers of the technology and an independent advisory committee. It provides organisations representing patients, professionals and manufacturers with the opportunity for consultation and appeal. In line with the Institute's commitment to transparency, consultation documents are published on the NICE web site.
- In developing its guidance NICE commissions an independent review of the published evidence and the manufacturers own data, this 'Assessment Report', together with evidence submitted by patient/carer organisations, health professionals and manufacturers, is considered by an independent appraisal committee. Patient/carer and professional representatives, nominated by their own organisations, attend and inform the committee meeting.
- Following their first consideration of the evidence the independent appraisal committee consult on their initial thoughts with all of the stakeholders including the patient/carer and professional groups. This consultation document is called an ACD (Appraisal Consultation Document). The ACD is also published on the NICE web site and it is important to note that this document does not constitute the Institute's formal guidance on this technology. The recommendations made in it are preliminary and may change after consultation.
- The feedback from the consultation process is considered by the appraisal committee at a further meeting and they then draft a further document (the FAD) which is sent to NICE. NICE considers the committee's recommendations, and sends their proposed guidance to the same stakeholder groups who have the opportunity to appeal. If there are no appeals this guidance is issued to the NHS.
- Section 4 of the FAD describes in detail the considerations of the committee.
- In summary, the FAD for imatinib shows that whilst NICE were consulting on the ACD more results from a trial became available. These results showed that in 85% of patients, the CML had not progressed (that is got any worse) 24 months after treatment. It also showed that overall 91% of patients had survived for 24 months after treatment (with or without progression of the CML).
- In addition to this the Assessment Group carried out extra work to compare the effectiveness of imatinib with other treatments for CML. This work aimed to put the clinical effectiveness of the drug into context by comparing it with other treatments.
- HR stands for haematological response. This means that blood cell counts return to normal as a result of treatment.
- CR stands for cytogenetic response. This means that the abnormal stem cells produced by the bone marrow that develop into white blood cells have been reduced (partial CR) or eliminated (complete CR). Major CR in clinical studies is defined as either complete CR or a partial CR.
- The extra work carried out by the Assessment Group on data from a study of patients in the chronic phase of CML suggested that patients who have a major CR 3 months after treatment are likely to have a better outcome 18 months after treatment, compared to those who did not have major CR.
- The additional analysis also showed that although survival rates 1 year after treatment with imatinib were the same as those of other treatments, imatinib produced much higher HR and/or CR.
- The Committee reviewed additional evidence from two sources in support of the importance of HR and CR as ways to judge the likelihood of longer-term survival. Having reviewed this evidence the Committee considered that it showed a sufficiently strong link between HR and CR and longer-term survival of people with CML. More information can be found in Section 4 of the FAD.
- Patients, professionals and manufacturers were sent the FAD document on the 5th August 2002 and have until 5pm on 27th August to determine if they wish to appeal.
- Appeals must fall within one or more of the following grounds:
a. The Institute has failed to act fairly and in accordance with the appraisal procedure set out in the Institute's Guide to the Technology Appraisal Process.
b. The Institute has prepared guidance which is perverse in the light of the evidence submitted.
c. The Institute has exceeded its powers.
- If there are no appeals the guidance will be confirmed and issued in October 2002.
This page was last updated: 30 March 2010