Patients in Scotland with advanced prostate cancer will be able to get routine access to Sanofi Genzyme's Jevtana on the NHS after all, after cost regulators changed track and endorsed the drug as a cost effective use of resources.
The Scottish Medicines Consortium has now published advice accepting Jevtana (cabazitaxel) to treat advanced forms of the disease, following consideration through its Patient and Clinician Engagement (PACE) process, which is used for medicines to treat end of life and very rare conditions.
During this process, patient groups and clinicians highlighted the limited treatment options for patients at this stage of the disease and that the problems they may experience, such as spinal cord compression and a significant decrease in quality of life, can impact considerably on both them and their carers, it said.
The drug is considered "a therapeutic advance", offering patients the potential for increased survival time with manageable side effects, the SMC noted, after having previously rejected it on grounds that the overall health benefits were not sufficient to justify its cost to the NHS.
Jevtana has also been cleared by sister body the National Institute for Health and Care Excellence for use on the NHS in England and Wales.
"This is positive news for men in Scotland who now have the same access as other men in the UK to treatment that increases their chance to live longer," said Roger Wotton, Chairman at prostate cancer patients' charity TACKLE.
"Any extension of time a man with prostate cancer is able to get back to be with family and friends is highly important. The availability of Jevtana means men in Scotland now have an alternative effective treatment option. Likewise, physicians now have another medicine in their arsenal to treat patients."
"There are currently very few options for patients at this stage of treatment and as such cabazitaxel represents an advance," said Professor Jonathan Fox, chair of SMC. "Having heard the evidence from patient groups and clinicians through our PACE process, we know this decision will be welcomed".
On the downside, the Committee was unable to accept Roche's Perjeta (pertuzumab) for the routine treatment of certain types of breast cancer.
Despite the added flexibility that PACE gives to the decision making process, committee members were concerned about the company's evidence around the long term survival benefits of the medicine.
Richard Erwin, general manager of Roche UK, said the decision is "a devastating blow to women in Scotland with this very aggressive form of breast cancer".
Last month the National Institute for Health and Care Excellence (NICE) approved Perjeta in combination with for the same clinical indication.
"The discount accepted by NICE was offered in Scotland yet the medicine was rejected by the SMC who found the link between overall survival and the clinical endpoint (pathological complete response) evaluated in neoadjuvant HER2-positive early breast cancer trials to be too uncertain".
"The endpoint was accepted by regulatory authorities in the EU and US to grant Perjeta its marketing authorisation for neoadjuvant therapy. With novel endpoints becoming increasingly common, a conditional funding mechanism - allowing access to new medicines while additional data are gathered - would be a positive step forward for patients in Scotland," said Erwin.
Further rejections following evaluation via the PACE process were handed down to: Vertex' Kalydeco (ivacaftor) for the treatment of cystic fibrosis in adults with the R117H genetic mutation, because of too much uncertainty around the overall clinical benefits the drug may bring in relation to its high cost; MSD's Keytruda (pembrolizumab) for advanced melanoma (skin cancer) in patients who have previously been treated with ipilimumab, because the Committee was not satisfied that evidence on the long term survival benefits was strong enough to justify the drug's cost to the NHS; and Shire's Plenadren (modified release hydrocortisone) for the treatment of adrenal insufficiency, because of a lack of robust evidence in the submission on the clinical benefits and value for money of the medicine when compared to other available treatments.
The Committee was also unable to accept Shield Therapeutics' Ferracru (ferric maltol) for the treatment of iron deficiency anaemia (IDA) in patients with Inflammatory Bowel Disease (IBD). This was because of uncertainty around the evidence about the clinical and cost benefits of the medicine in relation to other currently available treatments for this condition, the regulator said.
"It is disappointing that the Committee was unable to accept the other medicines considered in November," said Professor Fox. "Four of these medicines were considered through our PACE process and, while that gives us additional flexibility in our decision-making for medicines for end of life and very rare conditions, we have to consider value for money and take account of the needs of all patients being treated in NHS Scotland, not only those affected by the condition under consideration".