HAIRBEAR_UK and @jm954 represented patients at a recent NICE appraisal of Acalabrutinib and NICE has now determined that Acalabrutinib will be available to CLL patients in the following groups:
* previously treated CLL patients.
*untreated patients with 17p deletion or TP53 mutations.
*untreated patients who are unsuitable for chemotherapy treatments, such as FCR or BR. This would be because perhaps their kidney function is poor or they are too frail to undergo chemotherapy.
Unfortunately NICE was unable to make a determination on the suitability and cost effectiveness of Acalabrutinib in the untreated patients who are suitable for chemotherapy because of a lack of evidence submitted by Astra Zeneca (AZ). As a charity we were very disappointed with this and advocated that Acalabrutinib should be available for all untreated CLL patients and we will continue to advocate that that should be the case.
The main clinical trial evidence that AZ presented to NICE came from the ELEVATE clinical trial, which compared Acalabrutinib (+/- obinutuzumab) with a combination of chlorambucil and obintizumab. This trial showed a significant improvement in progression-free survival (i.e. an increase in the length of time people survive without their CLL getting worse) in patients who took acalabrutinib, compared with those who had the chlorambucil. Patients for whom chlorambucil would've been an option at that time would have been the less fit patients who could not have tolerated FCR or BR.
Those patients who are fit for FCR or BR chemotherapy may choose to take the chemotherapy (perhaps if they have favourable genetic markers) or there is the option of a one year course of Venetoclax + Obintuzumab which was recently approved by NICE to be available via the Cancer Drugs Fund.
Unfortunately, there is now a potentially serious health inequality gap between the patient groups who have access to several targeted treatments and the untreated and fitter (generally younger) patients who only have one option (V+O) via the CDF.
We are grateful that AZ were able to reach an agreement with NICE regarding a favourable pricing structure which helped the appraisal reach a positive conclusion.
The use of Acalabrutinib in Scotland is currently being assessed by the Scottish Medicines Consortium (SMC) . We will report on the outcome of this assessment when it has made a determination on its use.