Watchdog approves skin cancer drugs ipilimumab and vemurafenib

Patients with an advanced form of skin cancer were given new hope today after the health watchdog recommended that two life-extending treatments should be available on the NHS.

In final draft guidance, the National Institute for Health and Clinical Excellence (Nice) said that drugs ipilimumab and vemurafenib should be used for the treatment of advanced malignant melanoma - the most dangerous type of skin cancer.

The prognosis for advanced melanoma is very poor, and those who are diagnosed often have just months to live.

But under new proposals, patients in England and Wales will be able to access the life-extending treatments.

"Advanced melanoma can significantly affect patients' quality of life and without effective new therapies, the prognosis for advanced disease is very poor," said Professor Carole Longson, director at the Health Technology Evaluation Centre at Nice.

"For many years the treatments available for this condition have been very limited and in some cases restricted to palliative care. However, there are now a number of new treatments being developed and vemurafenib and ipilimumab are the first two that Nice has been asked to review.

"These new draft recommendations represent really good news for skin cancer patients.

"Vemurafenib and ipilimumab are breakthrough treatments that can potentially significantly affect prognosis for these patients and we are very pleased that the manufacturers have worked with us so that we are now able to recommend both ipilimumab and vemurafenib."

In initial guidance, Nice did not recommend the use of the treatments. But the watchdog has since received data about the cost effectiveness of the drugs and in its final draft guidance it has recommended the treatments on the basis that the manufacturers provide them with a discount.

Vemurafenib, which is also known as zelboraf and is manufactured by Roche, has been recommended for the treatment of metastatic melanoma.

And ipilimumab, made by Bristol-Myers Squibb, is recommended for the treatment of advanced malignant melanoma in people who have received prior chemotherapy.

Patient Richard Jackson, 44, who was diagnosed with metastatic malignant melanoma in 2006, said that ipilimumab, also known as yervoy, gave him a chance to see his two children grow up.

Mr Jackson, from Little Sutton, Wirral, Cheshire, said: "Being diagnosed with metastatic malignant melanoma in 2006 gave me very little chance of long-term survival.

"No standard treatment existed, and the only course of action was to agree to undergo trials.

"The compassionate use programme allowed me to be treated with yervoy in 2007. The effects were instant and dramatic, skin lesions, about the size of golf balls, flushed with colour and visibly reduced.

"Although the side effects were debilitating, by November 2008 I was back at work and living cancer free.

"Yervoy gave me hope and inspired me to keep going. Without Yervoy there is little chance I would still be here to see my two children grow up."

Until Nice issues final guidance on the drugs, NHS bodies should make decisions locally on the funding of specific treatments, a spokeswoman said.

Professor Alan Ashworth, chief executive of the London-based Institute of Cancer Research, said: "It represents a great stride forward in the treatment of advanced malignant melanoma, and is a brilliant example of what new-generation targeted cancer therapies can achieve.

"Zelboraf validates the importance of research into the fundamental causes of cancer, since it works by blocking a very specific mutant molecule that fuels the growth and spread of melanomas.

"It can take time to move research from the laboratory into the clinic, but zelboraf today becomes the latest in a string of targeted cancer therapies that will deliver real benefits for NHS patients."

Gill Nuttall, spokeswoman for the patient support group Factor 50, added: "I am absolutely delighted that Nice have overturned their original decision and that the patients I speak to every day can start to get the treatments that they so desperately need.

"Some of the patients who were here at the time of the original decision are sadly no longer with us and today will be tinged with sadness for some people.

"I am really pleased that Nice paid attention to all the comments made by the patients and clinicians and that those patients can now start to benefit from this innovative new treatment."

Nina Goad, spokeswoman for the British Association of Dermatologists, added: "The medicines may extend the lives of some of those with widespread melanoma who otherwise would have had little treatment available to help their advanced disease."

PA