Category Archives: SMC

SMC approves licence for liver cancer treatment

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Stivarga® (regorafenib) has been accepted by the Scottish Medicines Consortium (SMC) as a monotherapy for the treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with Nexavar® (sorafenib).1

Regorafenib is the first medicine to be specifically licensed for second-line use in patients with HCC who had formerly been treated with sorafenib, the German multinational pharmaceutical company Bayer has announced.

The medicine is taken orally and works by slowing down the growth and spread of cancer cells by cutting off the blood supply that keeps cancer cells growing.2

Judi Rhys, chief executive of the British Liver Trustsaid: “A diagnosis of hepatocellular carcinoma (HCC) is truly devastating – it is a horrendous type of liver cancer that is often diagnosed very late with few treatment options.

“We are delighted that the Scottish Medicines Consortium (SMC) has accepted the Trust’s evidence on behalf of patients and agreed to the use of this drug for patients in Scotland.

Evidence shows that outcomes for people with advanced liver cancer are particularly poor, so this is an important step.”

She added the decision “highlights a two tier system where patients in other parts of the UK are denied access to this new treatment that can improve outcomes”.

The positive SMC announcement follows the recent decision from the National Institute for Health and Care Excellence (NICE) to not recommend the use of regorafenib on the NHS in England.3

Amanda Cunnington, head of patient access, Bayer UK said regorafenib was “the first advancement in licensed treatment for liver cancer patients in nearly a decade”and that it offers “the first and only approved second-line systemic treatment option which could significantly improve patients’ overall survival”.

Regorafenib is licensed based on data from the international, multicentre, placebo controlled Phase III RESORCE [Regorafenib after Sorafenib in patients with hepatocellular carcinoma; NCT 01774344] trial. The trial investigated patients with HCC whose disease had progressed during treatment with sorafenib.4

In the trial, regorafenib plus best supportive care (BSC) was shown to provide a statistically significant and clinically meaningful improvement in overall survival (OS) versus placebo plus BSC (10.6 vs. 7.8 months, respectively, (HR 0.62; 95% CI 0.50-0.79; p=0.000017)) which translates to a 37% reduction in the risk of death over the trial period.4

Adverse events observed in the RESORCE trial were generally consistent with the known safety profile of regorafenib.4 The most common (>=30%) treatment-emergent adverse events were hand–foot skin reaction, diarrhoea, fatigue and hypertension.4

HCC is the most common type of primary liver cancer.5 Liver cancer is a difficult-to-treat cancer with an annual mortality rate of 48,000 in the EU.6 Globally, it is the second leading cause of cancer-related deaths.6In the UK, there are over 5500 new cases of primary liver cancer diagnosed each year, which is around 15 patients each day.7


  1. SMC. regorafenib 40mg film-coated tablets (Stivarga®). SMC No 1316/18. Bayer plc. April 2018. Available at: (Last accessed May 2018).
  2. European Medicines Consortium (EMC) Stivarga® Patient Leaflet. Available at: (Last accessed April 2018).
  3. National Institute for Health and Care Excellence (NICE) Regorafenib for previously treated advanced hepatocellular carcinoma. Technology appraisal guidance [TA514] Published date: 21 March 2018.  Available at:  (Last accessed April 2018).
  4. Stivarga® (regorafenib) Summary of product characteristics. Bayer HealthCare. September 2017.… (Last accessed April 2018).
  5. Cancer Research UK. Liver Cancer Types. Available at: (Last accessed April 2018).
  6. GLOBOCAN 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide in 2012. (Last accessed April 2018).
  7. Cancer Research UK. Liver Cancer Incidence Statistics. Available at  (Last accessed April 2018).


Darzalex, Epclusa will be funded on NHS Scotland

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Patients in Scotland with multiple myeloma and those with some types of hepatitis C have won NHS access to new treatment options after the Scottish Medicines Consortium backed routine use of Janssen’s Darzalex and Gilead’s Epclusa.

The cost watchdog has now endorsed use of Darzalex (daratumumab) for the treatment of multiple myeloma, a rare cancer of the white blood cells that may result in complications including severe bone pain, kidney damage and a depleted immune system, reversing an earlier negative decision.

Following consideration through the SMC’s Patient and Clinician Engagement (PACE) process for medicines used to treat end of life and very rare conditions, the drug has now been accepted for the treatment of patients who have received at least three prior treatment regimens.

Patient groups and clinicians highlighted that there are limited treatment options available at this stage of the disease and that, while there is currently no cure, Darzalex may provide an opportunity to improve control of the condition.

Elsewhere, Epclusa (sofosbuvir/velpatasvir) was accepted for the treatment of specific sub-types of hepatitis C, offering a new route to possibly eradicating the virus.

Through a submission to SMC, patient groups highlighted that this medicine could enable patients to be treated with minimum disruption to their working and family lives.

Funding for the drug is, however, being restricted patients with genotype 2, 5 or 6 chronic HCV infection, or for those with decompensated cirrhosis, irrespective of chronic HCV genotype.

The regulator has issued separate advice accepting the use of Epclusa for the treatment of patients with genotype 3 chronic HCV infection.


NICE recommends use of Janssen’s Imbruvica for Waldenström’s macroglobulinaemia

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The National Institute for Health and Care Excellence (NICE) in the UK has recommended the use of Janssen Pharmaceutical’s Imbruvica (ibrutinib) on the Cancer Drugs Fund (CDF) to treat adults with a type of non-Hodgkin lymphoma called Waldenström’s macroglobulinaemia (WM).

Ibrutinib is a Bruton’s tyrosine kinase (BTK) inhibitor and blocks cell survival signals transmission within the malignant B cells by forming a covalent bond with the BTK protein.

The recommendation covers NICE’s marketing authorisation and patients who have had a minimum of one prior therapy.

NICE based its positive opinion on clinical data obtained from a single-arm, open-label Phase II trial performed in the US.

Janssen UK Health Economics, Market Access and Reimbursement (HEMAR) and Advocacy director Jennifer Lee said: “This rare form of blood cancer has for too long been without a standard of care treatment, and ibrutinib is a real step-change in the management of WM.

“The decision to make ibrutinib available on the CDF makes it possible for additional clinical data to be collected to add to the evidence base for WM.”

NICE also issued an appraisal consultation document (ACD), recommending the firm to not use Imbruvica for adults with relapsed or refractory mantle cell lymphoma (MCL).

The institute said that the proposal for inclusion in the CDF will be considered and the drug will continue to be available on the previous CDF.

Responding to ACD, Jennifer Lee added: “This is particularly discouraging news for patients with MCL in the UK, as the medicine is already routinely available to patients with the same condition in Scotland, following the Scottish Medicines Consortium (SMC) recommendation last year.

“Should this guidance become final, UK patients with MCL could be left without access to this standard of care for the condition and with few treatment options, and none beyond chemotherapy.”