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Sarah Houlton, PhD, is Pharmaceutical Executive’s international correspondent.
Genentech and Biogen's MabThera has been awaiting NICE appraisal for three years. Merck KGaA's Erbitux has waited two-and-a-half years. A ruling on AstraZeneca's Arimidex isn't expected for more than a year.
The availability of new medicines—or lack thereof—has been thrown into the limelight once more, with UK's National Institute for Health and Clinical Excellence (NICE) being called upon to make dramatic improvements to the speed at which it carries out evaluations.
Just because a medicine is approved by the European Medicines Agency (EMEA) doesn't mean that doctors can immediately start prescribing it. There is another layer of bureaucracy that has to be overcome first, at member-state level, regarding decisions on such issues as reimbursement. So patients in one state may get a new drug quickly after approval, while in another, they could be waiting for years.
In the UK, the situation is further complicated by the existence of NICE, which carries out investigations into new treatments, and decides how effective they are. Although the drugs may technically be available on the National Health Service (NHS), local health authorities are invariably reluctant to spend their limited budgets on expensive new medicines before NICE requires them to—and the evaluation can take years. This creates "postcode prescribing"—in which the drugs you can take depend on where you live.
There is hope that this situation may be helped in the future. NICE is having discussions with the Department of Health over proposals for a revised process. This would enable important new drugs to be appraised more quickly, and in some cases, allow NICE to issue guidance shortly after a drug becomes available on the NHS.
Patient groups have been extremely vocal in their calls for reforms to the NICE system. Cancer information charity CancerBACUP claims that 23 licensed cancer treatments are currently beset by "unacceptable delays" before being made widely available on the NHS. The most extreme case is Genentech and Biogen Idec's MabThera (rituxumab), a treatment for non-Hodgkin's lymphoma that has been awaiting appraisal for three years. Merck KGaA's Erbitux (cetuximab) for advanced colorectal cancer has been waiting for two-and-a-half years.
Patient power has, however, had some headline-hitting success. A nurse with early-stage breast cancer that is susceptible to Herceptin (trastuzumab) won a court case that forced her local health authority to pay for the drug. This, despite the fact that it's only licensed for use in the advanced stages of the disease—manufacturer Roche is still assessing the drug's safety, and whether the benefits outweight the risks in widespread prophylactic use. The company won't be in a position to submit an ANDA until February, meaning a license is unlikely until next summer. NICE has already begun its appraisal, which is unusual.
CancerBACUP has published a detailed policy paper that calls for a root-and-branch reform of the entire appraisal system. It says cancer treatments should be examined within three months of a marketing license being granted, and some should even be fast-tracked. It also believes that NICE should set up specific expert-appraisal committees for different disease areas, such as cancer, and these should be able to give interim guidance on drugs. Finally, the charity believes the appraisal process should be made shorter, and NICE should be given the resources it needs to operate efficiently.
The situation regarding drug availability is different even within the UK. In Scotland, decisions are made by the Scottish Medicines Consortium (SMC), rather than NICE. In September, the SMC recommended that AstraZeneca's Arimidex (anastrozole) be used in post-menopausal, early-stage breast cancer patients. The drug has been shown to be 25 percent more effective in these patients than the existing gold-standard treatment, tamoxifen. But in the rest of the UK, where NICE rules, Arimidex is still stuck in the bureaucratic quagmire, and a decision isn't expected for well over a year.
Ironically, when NICE was set up back in 1999, it was supposed to eliminate this kind of postcode prescribing. Initially intended to look at the clinical effectiveness of new treatments, by the time NICE came into being it had been transmogrified into a body that was also mandated to determine cost-effectiveness. And, rather than being a body that rules on how well something works, it is being used as an excuse by local health authorities not to prescribe new treatments that—while expensive—provide hope to extremely sick cancer patients.
"It's heartbreaking for the nurses on our helpline to have to tell callers that new treatments will not be available on the NHS for several years," says CancerBACUP's chief executive Joanne Rule. "Only reform of NICE will ensure these vital treatments are widely available to the patients who desperately need them."
Sarah Houlton, PhD, is Pharmaceutical Executive's global correspondent. She can be reached at firstname.lastname@example.org