The MDS Beacon™

Survey results show that 18 percent of myelodysplastic syndromes (MDS) sufferers in the United Kingdom could have lived longer if they had access to treatments not yet approved for funding in the U.K.

The National Institute for Clinical Excellence (NICE) is a government agency in the U.K. which reviews the clinical effectiveness and cost-effectiveness of medical treatments. Based on the review of clinical and cost information, NICE makes a recommendation about whether the treatment should be funded by the National Health Service (NHS), which provides almost all health care in England and Wales.

The survey also revealed that 56 percent of blood cancer experts surveyed believe that less priority is given to rarer cancers compared to common cancers.

Other results from the study include that 89 percent of blood cancer experts surveyed have been in situations in which they have been unable to provide potentially life-extending treatments because the treatments were not yet available on the NHS or approved by NICE.

Additionally, 12 percent of the experts surveyed indicated concerns about informing patients of new treatments not yet approved for funding. In particular, it can be distressing to patients to be informed about a treatment that they cannot have.

NICE can take up to three years from the time a drug is first licensed in the U.K. to appraise and approve the drug for funding. This delay in approving treatments is a concern for medical professionals who treat patients with life-threatening conditions such as cancer.

“Despite significant advances in the treatment of MDS, the majority of patients cannot get access to and in some cases are not even informed about new life-extending drugs until they have been appraised and approved by NICE,” said Ghulam Mufti, a professor of hemato-oncology at King’s College Hospital and Chairman of the U.K. MDS Forum.

The results of the survey were announced one month after NICE released its preliminary decision to not recommend Vidaza (azacitidine) for state funding in the U.K. They found the treatment to be more expensive than the manufacturer, Celgene, presented. The committee reviewing Vidaza is currently in a period of re-review before announcing a final recommendation to the NHS (related Beacon news).

The U.K. MDS Patient Support Group initiated the study, titled The Blood Cancer Treatment Access Survey. Surveyors collected data through telephone interviews with 100 U.K. hematologists earlier this summer.

For more information, visit the U.K. MDS Patient Support Group press release.