This draft guidance follows a previous draft published by the National Institute for Health and Clinical Excellence (NICE) on June 2, which recommended both thalidomide (Thalomid) and Velcade (bortezomib) as first line treatments for multiple myeloma patients ineligible for stem cell transplant or high-dose chemotherapy.

NICE’s most recent draft states that the clinical effectiveness of Velcade and thalidomide are comparable, yet designated Velcade as a backup treatment to thalidomide after analyzing the costs of the two treatments.

These recommendations followed consultations with a number of government, patient, and professional multiple myeloma organizations. Determinations of cost effectiveness were made based on reports submitted by Janssen-Cilag, the distributor of Velcade in the U.K., and Celgene, the manufacturer of thalidomide.

The statements presented in the draft have raised concerns at Janssen-Cilag about the accessibility of Velcade as first-line treatment for multiple myeloma patients in the U.K. The company said in a statement that it will appeal against NICE’s recommendations.

The appeal period for this draft will close on September 10, and a final guidance is expected to be published by NICE later that month.

Both drugs are currently recommended as second-line treatments for the treatment of multiple myeloma.

More information about the draft can be found at the NICE website.

15th Congress Of The European Hematology Association – As a reminder to Beacon readers, the European Hematology Association (EHA) will hold its 15th meeting from June 10 to 13 in Barcelona, Spain.  European researchers and physicians will meet to present the latest research findings about multiple myeloma and other blood disorders.  The Myeloma Beacon will also be covering the event on its website.  For more information, please visit the EHA Congress website.

MMRF Race For Research – On June 13, the Multiple Myeloma Research Foundation (MMRF) will hold a Race For Research 5K Walk/Run in New Canaan, CT, to raise awareness and funds for multiple myeloma research. Registration is scheduled for 7:30 a.m., and the race starts at 9 a.m. For more information, please see the MMRF website.

For a more detailed listing of myeloma-related events, please check the Myeloma Beacon Events Calendar.

The Price Of Life – Directed by Adam Wishart, this BBC documentary is the first to film the decision-making process of the National Institute for Health and Clinical Excellence (NICE). NICE is an independent organization that provides medical opinions to the National Health Service (NHS), the government-funded health care system in the U.K. The documentary gives insight into NICE’s process of deciding whether or not Revlimid (lenalidomide) should be approved for use and funded by the NHS. It also gives the perspective of patients and an American drug company. For more information, please visit BBC Web site.

For a more detailed listing of myeloma related events, please check the Myeloma Beacon Events Calendar.

NICE is an independent organization that provides medical opinions to the National Health Service (NHS), the government-funded health care system in the U.K.

Based on NICE’s recommendation, the NHS will only pay for Revlimid for patients who have received at least two prior therapies. The NICE guidelines recommend that patients currently taking Revlimid who have not received two or more prior therapies continue their current treatment until the patient and doctor consider it appropriate to stop.

In November 2008, NICE opposed funding Revlimid due to its cost.

In response, Celgene, the manufacturer of Revlimid, proposed a cost-sharing plan in which Celgene would cover the cost of treatment beyond 26 cycles (normally two years).

NICE agreed to the cost-sharing plan and released a preliminary announcement in January that recommended Revlimid in combination with dexamethasone be used in relapsed/refractory myeloma patients with two or more prior therapies. The final decision was announced in April.

For more information, see the press release on the NICE Web site.

Revlimid is a therapeutic agent that has often proven to be effective in the treatment of relapsed patients. Clinical trials report extended survival times and recession in signs and symptoms in the majority of patients.

In the U.K., NICE decides which treatments will be made available under the state-funded National Health Service (NHS). Revlimid has been unavailable to U.K. patients because of a decision by NICE to stop funding the drug due to its high cost. However earlier this year, NICE released a preliminary decision to once more fund Revlimid after Celgene Limited, the manufacturer of Revlimid, suggested a cost-sharing plan. Under this plan, the NHS will pay for the first 26 cycles of treatment. Celgene will cover any further cycles of treatment, which last approximately one month each.

With its preliminary and now final recommendations, NICE has determined that Revlimid is a cost effective use of the resources of the NHS.

Although multiple myeloma is still an incurable disease, Gareth Morgan, Professor of Haematology, Royal Marsden NHS Foundation Trust, is encouraged by the possible impact of this step. “New therapies like Revlimid are offering significant extensions of survival for patients and moving the disease toward becoming a chronic, manageable condition,” he told Pharmiweb. “[This] is a major step toward providing a critical new option for patients in the U.K.”

Eric Low, Chief Executive of Myeloma U.K., added, “This is a great win for myeloma patients. I applaud the efforts of the myeloma community, the manufacturer, NICE and the Department of Health in working together to allow access to Revlimid to patients who need it.”

For more information, please see the press release on Pharmiweb.

The news comes after Celgene proposed a new cost-sharing plan where the NHS will pay for the first two years of treatment, and Celgene will pick up the bill for any further treatment. It is estimated that approximately 17 percent of myeloma patients treated with Revlimid will require treatment after two years.

The revised guidance has yet to be finalized and is being put out for consultation until February 20 by the National Institute for Health and Clinical Excellence (NICE). In England and Wales, NICE decides which treatments will be available to patients under the state-funded NHS.

NICE, under the revised guidance, will recommend that Revlimid be used in combination with dexamethasone in relapsed or refractory multiple myeloma patients who have received two or more prior therapies.

The new deal is the latest advancement in controversial drug coverage decisions made by NICE in the past year. In November, NICE announced that it would not fund the use of Revlimid because of its £36,000 ($52,000) per-year cost. On January 2, however, NICE issued a new guidance that approved the use of drugs that cost as much as £80,000 ($116,000) per year in cases where a patient was expected to live less than two years, the treatment could extend life by at least three months, and no less expensive treatment could offer similar results.

In Scotland, the Scottish Medicine Consortium (SMC), similar to NICE in England and Wales, decided not to recommend use of Revlimid under the National Health System of Scotland. However, a Scottish Government official added that “even when a drug is not recommended by the SMC, in exceptional circumstances where a clinical case may be made, special arrangements under ‘exceptional prescribing’ will be considered by the relevant NHS Board.”

The proposed deal in England and Wales, however, has garnered much praise and support, as there are nearly 3,800 new cases of multiple myeloma in the U.K. each year.

Rachel Rowson, from Macmillan Cancer Support, said “We are delighted that the manufacturer of Revlimid has decided to put patients before costs and will be making this treatment more affordable to the NHS. Revlimid can give some myeloma patients extra time which can be extremely valuable to them and their families.”

Additionally, Andrew Robertson, the general manager of Celgene in the U.K. and Ireland, stated “We understand that time is a precious commodity for patients with multiple myeloma and we are committed to ensuring that the Revlimid treatment continuation scheme is successfully implemented so that all patients who can benefit will have rapid and sustained access to treatment.”

More information about the proposed agreement can be found at the BBC News Web site.

In England and Wales, the National Institute for Health and Clinical Excellence (NICE) decides which treatments will be made available under the state-funded National Health Service.  Until recently, NICE refused to approve the use of any drug costing more than £30,000 ($44,000) for each extra year of good health attributable to the drug.

On January 2, NICE issued new guidelines to approve, under certain circumstances, life-extending drugs that cost as much as £80,000 ($118,000) per extra year of good health.  The previous £30,000 threshold may now be exceeded when a patient is expected to live less than 24 months, strong evidence exists that the costly treatment could extend life by three months, and no less expensive treatment can offer similar benefits.

The decision is a surprising reversal.  Just two months ago, NICE announced that it would not fund the myeloma treatment Revlimid (lenalidomide) because of its £54,000 ($79,000) per-year cost. (Read about the November NICE announcement here.)  The Revlimid rejection rekindled public debate about NICE’s funding criteria, as it was just the latest in a series of controversial NICE decisions related to important new cancer drugs.

Macmillan Cancer Support, a U.K. based organization that provides support for cancer patients, estimated that 10,000 cancer patients will benefit from this new policy each year.

Further details about the new NICE guidelines can be found at The Argus, and the full guidelines can be found on the NICE Web site.

The agency responsible for controlling health care costs in those countries has issued a preliminary ruling that treating myeloma with Revlimid is not cost effective.

The agency – the National Institute for Health and Clinical Excellence (NICE) – admits that the drug is clinically effective. The once-a-day capsule has been shown to extend the lives of myeloma patients by nearly three years. As a result, Revlimid is now a standard treatment for myeloma in much of Europe and the United States and is used in more than 60 countries across the world.

NICE, however, argues that Revlimid’s cost is too great for the benefit it typically provides. In England and Wales, Revlimid costs about £4,500 ($7,300) per month of treatment.

If NICE’s preliminary ruling is reflected in the agency’s final decision – due next January – it would prevent most multiple myeloma patients in England and Wales from being treated with Revlimid. The government funded National Health Service, which must adhere to NICE’s guidelines, provides almost all health care in England and Wales.

Myeloma patient organizations in the United Kingdom are dismayed by the NICE decision. The chief executive of Myeloma UK said “This is yet another instance of patients losing out because NICE and the pharmaceutical company have failed to effectively interpret an impressive set of [clinical trial] data in what is no more than a game of professional one-upmanship.”

“NICE and a pharmaceutical company have once again failed to achieve excellence in what they do.”

Some analysts believe, though, that NICE and Revlimid’s manufacturer, Celgene, will reach a compromise that will allow Revlimid to be used regularly in England and Wales.

NICE and the manufacturer of another myeloma medication, Velcade, were able to reach such a compromise last year.

In the Velcade case – as with Revlimid – NICE initially ruled that the drug was not cost effective. NICE changed its guidance in its final ruling, however, when Velcade’s manufacturer agreed to cover the cost of the drug for patients who do not respond to it.

Revlimid’s manufacturer, Celgene, believes NICE’s recent ruling ignores important issues. Andrew Robertson, the company’s general manager for the U.K. and Ireland, said: ‘Whilst we recognize that NICE has an important role in recommending medicines that offer greatest value for money to the [National Health Service], we believe that the current appraisal process disadvantages patients with rarer diseases, where it is proportionately more expensive to develop new medicines.”

Robertson added, though, that “Celgene is committed to working with NICE, the Department of Health and the myeloma community to ensure that the final NICE guidance will be positive and patients with myeloma can have the opportunity to live longer with their cancer controlled. “

Further details on the NICE decision can be found in an article from the Daily Mail and in another article from PharmaTimes.Com. The full text of NICE’s ruling is available as a PDF document here.