National Institiue for Health and Clinical Excellence

Annual Review - 2012/2013
tech appraisals

Ensuring equal access to clinically and cost effective treatments

NICE technology appraisals aim to ensure equal, and consistent access to new and existing medicines and treatments deemed clinically and cost effective for use in the NHS.

This year, we published 30 technology appraisals on a range of diseases and conditions covering cystic fibrosis, chronic heart failure, prostate cancer, melanoma and macular oedema. The majority of these appraisals contained positive recommendations for use in the NHS.

In April 2012, NICE recommended fingolimod (Glenya), the first oral treatment for patients with relapsing multiple sclerosis.

Multiple sclerosis is a condition where white blood cells attack the coating of nerve cells which help messages from the brain travel to the rest of the body.

The most common form of this condition is relapsing-remitting multiple sclerosis (RRMS). This is where symptoms come and go, and periods of good health are followed by a sudden onset of symptoms. Around 27,500 people in England and Wales are thought to have RRMS, and the treatments available so far had to be injected.

Fingolimod (Gilenya), a medicine for the treatment of RRMS is taken by mouth, works by preventing white blood cells from attacking nerve cells in the brain and spinal cord. NICE recommended the drug for use among adults with RRMS who have an unchanged or increased relapse rate, or ongoing severe relapses in comparison with the previous year, despite taking beta interferons.

In addition, the recommendation for the use of fingolimod only applies if the drug manufacturer provides the drug with a discount agreed as part of a patient access scheme.

Professor Carole Longson, Director of the Health Technology Evaluation Centre at NICE, said: “We are pleased to recommend fingolimod as a treatment option for the specific patient population for whom it has been demonstrated to be cost effective.

“Multiple sclerosis can be a disabling condition and so we hope that this novel treatment will help to reduce relapses for these people."

In December 2012, NICE published guidance on two treatments for skin cancer, both of which were positive. Ipilimumab (Yervoy) was recommended for the treatment of advanced malignant melanoma in people who have received prior chemotherapy. And vemurafenib (Zelboraf) was recommended for the treatment of unresectable locally advanced or metastatic melanoma that is associated with mutations in a cell protein called BRAF V600.

Both treatments were recommended on the basis that manufacturers provide them with a discount, agreed in separate patient access schemes for each appraisal.

Professor Carole Longson said: “Advanced melanoma can significantly affect patients' survival and quality of life and without effective new therapies, the prognosis for advanced disease is very poor. For many years the treatments available for this condition have been very limited and in some cases restricted to palliative care.”

She added: “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 February 2013, NICE conducted a rapid review for ranibizumab (Lucentis, Novartis) for diabetic macular oedema (DMO).

NICE originally published guidance on ranibizumab in November 2011, not recommending the drug for treatment of DMO. However, the guidance was updated following a revised Patient Access Scheme submitted by the manufacturer, together with updated analyses showing the drug's superior relative effect among a sub-group of people with DMO.

Professor Longson said: “In November 2011, NICE published guidance which did not recommend the drug as an effective use of NHS resources. However, following the submission of a revised patient access scheme, we have conducted a rapid review of the original guidance."

She added: “The manufacturer also included updated analyses showing that ranibizumab could be expected to have a superior relative effect among people with central retinal thickness greater than 400 micrometres.”

Speedy production of new guidance

NICE frequently produces guidance within months of a drug becoming available.

One example of this was the publication of final guidance by NICE on apixaban, for the prevention of stroke in patients with atrial fibrillation. The final guidance came a mere four months after the drug was licensed.

Part of the reason for this is that the drug was referred before it received a licence for use in the UK by regulatory authorities. There were additionally no issues with the evidence submitted by the manufacturer, and there were no appeals on draft recommendations produced by NICE on use of the drug.

Jenniffer Alty, Associate Director of Planning and Operations for Appraisals at NICE's Centre for Health Technology Evaluation, said “For apixaban, the Appraisal Committee felt the evidence was clear-cut, and no additional consultation was needed.

"This, together with no appeals received, helped speed up the process for the production of final guidance from NICE."

Visit our website for more information on the improving production times for new pharmaceuticals.

The majority of our appraisals contained positive recommendations on medicines and treatments deemed clinically and cost effective for use in the NHS.