UK Estimates of Burden of Disease Associated with Management of Intermediate-2 or High-Risk Myelodysplastic Syndromes

Previous UK studies into the burden of disease (BoD) of intermediate-2 (INT-2) or high-risk myelodysplastic syndromes (MDS) have focused on the potential budget impact of azacitidine. No estimates have been published for the UK that incorporate the costs associated with basic disease management in the UK for this group of patients.

To estimate the annual burden of MDS in the UK.

The research comprised a systematic review of studies in haematological cancer, and a subsequent economic analysis. The systematic review extracted all relevant BoD information from identified papers, including not only patient or healthcare elements, but also the burden on carers and family. The economic analysis combined all available, relevant evidence into annual, UK-specific cost estimates.

The systematic literature review identified and extracted information from 23 papers. Study designs were comprised of economic evaluations (n=6), observational studies (n=2), RCTs (n=2), retrospective analyses involving registries or hospital data (n=6), review articles (n=3) and surveys (n=4). The National Institute for Health and Clinical Excellence appraisal of azacitidine was also included as was information from the pivotal trial of azacitidine, AZA-001. Across the literature reviewed, three main types of standard care were identified: supportive care (SC) alone, low-dose chemotherapy (LDC) and, more rarely, standard dose chemotherapy (SDC).

Many papers provided estimates of transfusion burden, which had the largest BoD impact in patients who were considered transfusion dependent. Use of erythropoiesis-stimulating agents was identified as an additional important resource, and the societal burden of haematological cancer was also quantified in one study, which examined time required of carers and family for transfusion visits. The most recent UK epidemiological estimates were used to convert average per-patient BoD figures to national estimates. Each type of standard care had a different associated burden of blood transfusions; therefore, the subsequent economic analysis weighted the estimated BoD according to the proportion of patients expected to be eligible for BSC, LDC and SDC.

It is expected that 761 INT-2 and high-risk MDS patients will be treated each year. Among these, it was estimated that a total 5,121 blood transfusions would be required per year. These transfusions are expected to be associated with a total UK annual cost of approximately £1.4 million to the NHS, as well as £17,955 to carers and family members based on expected time off work to attend transfusion sessions with the patient. Values ranging between £10 million and £14 million per year were estimated for the likely national BoD on hospitals for inpatient stays, and the national cost of NHS community nurse visits was estimated to be £115,877 per year. The societal cost of inpatient stays was also experimentally calculated based on hospitalisation statistics from the AZA-001 trial; this produced an estimated annual burden of £312,010 for the UK.

The annual burden of disease associated with MDS in the UK is estimated at between £12 million and £16 million. Major components of this burden include hospitalisation and transfusion costs. Indirect costs associated with MDS such as productivity costs for patients and carers appear relatively modest but are nonetheless an important consideration and should not be overlooked.

Brereton:Celgene Ltd: Consultancy. Nicklasson:Celgene Ltd: Employment. Mufti:Celgene: Consultancy, Research Funding.