The Power of Informed Patients in the UK

e-Patients are first and foremost informed patients. Here is an incredible story from the UK showing how an alliance of Informed Patients, Cancer Advocacy Organizations and Pharmaceutical Companies have forced the UK National Health System to accept a drug treatment it originally refused for cost control reasons. The flexible cost plan offered by the drug maker, reimbursing the NHS if the treatment fails, is a first and may become a model for future new treatments.

In Europe, National healthcare systems who provide almost universal coverage are acting strongly to reduce costs. In the UK, cost analysis of new treatments is the responsibility of NICE (The National Institute of Health and Clinical Excellence). Based on its analysis NICE decides which medicines are paid for by the National Health Service. Final recommendations by NICE are invariably accepted by the English government. Last July, NICE issued a draft guidance with negative opinion about Velcade (bortezomib), the only drug known to have efficacy for relapsed Multiple Myeloma, saying the value of the drug was unclear and therefore should only be used in clinical trials. The drug is currently available in Scotland, Wales and Northern Ireland and throughout the rest of Europe where it is accepted as treatment for relapsed Myeloma.

Soon after NICE issued its negative guidance a joint appeal was lodged by 3 cancer charities (Myeloma UK, Cancerbackup and Leukaemia CARE), who argued that NICE’s initial rejection of Velcade was based mainly on the grounds of cost, rather than efficacy, and that this decision was therefore “perverse and unfair”, particularly as the cost of the drug was just over the £30,000 threshold for NHS drugs.

In early 2007, NICE agreed to review its own ruling and asked for opinions, from expert patients, charities and clinical specialists.

Yesterday NICE issued a new guidance, now recommending that “all suitable patients” have access to the treatment, with the NHS continuing to fund the drug for those who show a full or partial response.

Patients who do not benefit from a partial response after a maximum of four cycles of treatment should be taken off the treatment, with the cost refunded by the manufacturer, NICE said.

This new Draft Guidance is itself subjected to 3 weeks of reviews and comments at Multiple myeloma - bortezomib: appraisal consultation document.

The main reason NICE changed its guidance were the “Velcade3″. Three informed and very vocal patients who lobbied hard to get the drug accepted by NHS.

Jack Stilgoe, a senior researcher at the thinktank Demos, said the decision reflected the increasing pressure on NICE to fund new drug treatments. He said the high-profile campaigns for certain drugs by patients - with the backing of drug companies - were shaping public debates about which treatments were funded by the NHS.

Myeloma patient Jacky Pickles, one of the ‘Velcade 3’, who have been campaigning for NICE approval of the drug, said: “The entire myeloma community has been campaigning for access to Velcade for three years. The announcement today has given us all hope that soon no patient will die of myeloma without first being given access to Velcade. It could keep us alive long enough for a cure to be found. … If they agree to let clinicians use Velcade on the NHS, it will be like the myeloma community winning the lottery. Without it we are sentenced to death.”

The “Velcade 3″ started in August 2006, when they confronted Health Secretary Patricia Hewitt, and accused her of condemning them to death by denying them a new drug. They now have, of course, their own website where you can read the following:
“We, ‘the Velcade Three’, welcome the news of NICE’s (National Institute for Health and Clinical Excellence) revised preliminary positive guidance which recommends that Velcade is made available on the NHS to some patients with multiple myeloma.

Last August NICE issued an negative decision that would deny patients access to a treatment that would give hope . However after 10 long months of campaigning against that decision which has resulted in a successful appeal, we now feel that the road ahead is more positive. We feel we have been given a second bite of the cherry to ensure us patients get the treatment we need.

The outcome we now need to see is that Velcade is made available to all myeloma sufferers at the point of need after consultation with their consultants and that there is no discrimination of patients in any way.”

Clearly, informed patients can help to radically transform the healthcare environment, globally.