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Following a recent assessment, the Scottish Medicines Consortium (SMC) has this week approved the routine use of blinatumomab for the treatment of adult patients with Philadelphia chromosome negative relapsed or refractory B-precursor acute lymphoblastic leukaemia (ALL) in Scotland.
Acute lymphoblastic leukaemia (ALL) is a rare and rapidly progressing form of leukaemia. It is a cancer that affects the white blood cells, in particular the B lymphocytes. Stem cells divide to produce either more stem cells or immature cells that become mature blood cells over time. ALL is caused by a disruption to this process where the cells continue to divide, but do not mature. A result of this means that the body finds it difficult to fight infection. The most common signs and symptoms including anaemia, tiredness, fever, unusual bleeding and bruising are caused by the bone marrow being unable to produce enough normal blood cells.
During the assessment process, the benefits of blinatumomab (marketed by the manufacturing pharmaceutical company Amgen as Blincyto®) were examined by the appraisal Committee with both clinical and cost effectiveness taken into consideration. Clinical trial data found that blinatumomab was a novel therapy that had potential to improve remission rates and survival for patients. Importantly, it also enabled 40% of the patients to reach a point where they could receive a stem cell transplant. In a hard to treat area, this is a welcome result.
The recommendation of blinatumomab for ALL patients was accepted after consideration under the SMC’s Patient and Clinician Engagement (PACE) process for medicines that treat end of life and very rare conditions (in addition to the clinical evidence). As a part of the appraisal, the PACE process gives both patient and clinician’s an opportunity to provide their views. Patient organisations and clinicians highlighted that many patients are diagnosed when the disease is at a later stage and there are limited effective treatment options available (and therefore a poor prognosis).
Due to the limited number of treatments available for patients in this setting and blinatumomab’s potential to bridge relapsed patients to transplant (considered a “curable” treatment) the drug satisfied the SMC criteria for recommendation. As such, relapsed ALL patients in this setting will soon have access to an additional treatment option that could prepare them for a transplant.
The final advice concluded that “as blinatumomab is an orphan medicine, SMC can accept greater uncertainty in the economic case”, identifying that although the cost of the drug is high, there is a very real unmet therapeutic need for these patients. The positive decision to introduce blinatumomab into routine practice for patients in Scotland will offer an effective treatment option for patients in this setting, enabling some to be able to receive a transplant.
Blinatumomab is also currently being assessed for NHS use in Wales by the All Wales Medicines Strategy Group (AWMSG) and by the National Institute for Health and Care Excellence (NICE) for use in England, with the decisions expected to be published within the next eighteen months. These bodies, like the SMC, decide if a drug is clinically and cost effective enough to be recommended for routine use within the NHS.
Recent policy changes that have affected the way that drugs are appraised and funded for clinical practice in England could see many NHS patients in England with a rarer cancer (including many blood cancers) miss out on life-extending treatment (like blinatumomab for ALL). The changes incite further inequalities in that they could actually be available for patients in Scotland or Wales. Due to the method of drug appraisal in England, drugs struggle to meet the criteria for a positive recommendation and whereas previously there has been separate funding processes to fund such drugs (the Cancer Drugs Fund), the policy changes means that this is no longer an option.
Rare cancer drugs are currently appraised through NICE’s standard appraisal method. A result of this is that many drugs intended for the treatment of rarer cancers failed to demonstrate as clinically and cost effective enough to be recommended for routine use – this is largely because the existing methodology used by NICE doesn’t fairly assess disease indications where there is limited clinical data available (because of small patient populations). The chief executive of NICE, Andrew Dillon, has even stated that applying their “standard approach to treatments for very small groups of patients would result in us always recommending against their use. This would be unfair.”
The Cancer Drugs Fund (CDF) was established in 2011. Its purpose was to help remedy this “unfair” process, funding drugs that had not been successfully recommended during the NICE appraisal process. The CDF has benefitted over 84,000 patients since it was introduced but by 2015, it had severely overspent. Following a recent review and consultation period, the newly proposed process was revealed. The changes will see the CDF move to the beginning of the appraisal process to temporarily fund drugs whilst more data is being collected to confirm their clinical and cost effectiveness but there will be clear exit criteria (to prevent drugs from being funded this way indefinitely).
Urgent reform is needed to address the broken methodology of appraising drugs for rare cancers in order to ensure retained and fair access to innovative and effective treatment options for all existing and future NHS patients. The way that drugs are currently appraised just does not appropriately assess cancers that have small patient populations. Following the recent changes to the CDF, many drugs manufactured for the treatment of rarer cancers will still struggle to be recommended for routine use and there will now be no further funding sources. The way drugs are appraised by the SMC for patients in Scotland considers orphan drugs (drugs for rarer cancers) in a separate process, amending its criteria to help make more innovative treatments accessible to NHS Scotland patients. This process should be mirrored in NICE’s appraisal process for drugs in England to ensure increased access to life-extending treatments.
Patients who are already receiving treatments affected by these policy changes will continue to do so but the changes could affect future treatment options for existing patients (should their current one stop working and they need a new line of treatment) as well as new patients that have yet to be diagnosed. Leukaemia CARE believes that every blood cancer patient has a right to fair and equal access to the most effective treatments available.
To help ensure blood cancer patients in the UK can access the life-saving drugs they need and end this “Cancer Lottery”, we are encouraging people to get involved by signing our petition and sharing the campaign on social media.