Access for new melanoma treatment remains uncertain for patients despite UK launch

Jan 29 2016

Novartis announced today that Tafinlar^® (dabrafenib) + Mekinist^® (trametinib), the first oral combination therapy to be licensed for advanced melanoma, is now available in the UK for the treatment of adult patients with unresectable or metastatic melanoma with a BRAF V600 mutation. However for patients with the most aggressive form of skin cancer, access to the new combination is uncertain because the Cancer Drugs Fund is not listing new medicines until at least April 2016, and NICE guidance for the combination is not scheduled until after that time.

Metastatic melanoma is the most serious and life-threatening type of skin cancer, with only a 20% survival rate five years after diagnosis. There are more than 13,000 cases of malignant melanoma a year in the UK, around 37 a day. Melanoma rates and deaths have continued to rise over the past three decades, with the number of sufferers rising faster than the ten most common cancers. It has been predicted that within a decade, melanoma will be the fourth most common cancer for men and women in the UK.

Like many cancers, abnormal gene changes (mutations) can lead to the development of melanoma. Approximately half of all melanoma cases have what is known as a BRAF mutation, which can be identified by genetic testing of the tumour so the most appropriate targeted treatment can be chosen.

The BRAF mutation results in an abnormal activity of certain proteins that are usually responsible for regulating skin cell growth and development. This leads to uncontrolled growth of the skin cells and the development of tumours. The combination of dabrafenib + trametinib targets two different proteins that are involved in the development of tumours to block their activity and slow growth of the tumours. Using both medicines as a combination has been shown to be more effective than a BRAF inhibitor on its own.

“Understanding what drives melanoma at a genetic level has transformed the way we treat this devastating cancer and this combination is an important new option for patients. It has been shown to be more effective than monotherapy in extending the time before the cancer progresses and for the first time we are seeing median overall survival of more than two years,” said Dr Paul Nathan, Consultant Medical Oncologist, Mount Vernon Cancer Centre, Middlesex. “But equally important for patients is their quality of life. The studies of this combination also demonstrate an improvement in quality of life and an acceptable safety profile, which is crucial for patients with advanced melanoma.”

Access uncertainty from the start

Despite the UK launch, the prospect of patients gaining access to this combination therapy immediately looks bleak following the recent announcement that no new drugs will be included on the Cancer Drugs Fund list for the foreseeable future.

“While the CDF was not sustainable, it was the only mechanism by which new cancer medicines could get funding between launch and an appraisal by NICE”, said Margaret Dean, General Manager, Novartis Oncology UK and Ireland. “It is unfortunate that the CDF is closed to all new cancer medicines – with no transition mechanism in place. What this means for the first oral targeted combination therapy for advanced melanoma is that no patients can benefit from July 2015 until April 2016 at the earliest and patients are falling through the gap.”