Health technology assessment and haemophilia

Abstract

Although the funding of rare diseases such as haemophilia in developing countries remains a low priority, pressures on the funding of haemophilia treatment are also emerging in developed economies affected by the global economic downturn and the other demands on health care budgets. This is leading advisory bodies and payers alike to explore the tools of Health Technology Assessment (HTAs) in deriving recommendations for reimbursement policies. In particular, the use of cost utility analysis (CUA) in deriving costs per quality adjusted life year (QALY) for different interventions is being used to rank interventions in order of priorities relative to a threshold cost per QALY. In these exercises, rare chronic disorders such as haemophilia emerge as particularly unattractive propositions for reimbursement, as the accepted methodology of deriving a CUA. For e.g. the use of prophylaxis in haemophilia leads to a range of costs/QALY which exceed the willingness to pay thresholds of most payers. In this commentary, we review the principles utilized in a recent systematic review of the use of haemophilia products carried out in Sweden as part of an HTA. We suggest that ranking haemophilia related interventions with the standard interventions of therapeutics and public health in CUA comparisons is inappropriate. Given that haemophilia treatment is a form of blood replacement therapy, we propose that such comparisons should be made with the interventions of mainstream blood transfusion. We suggest that unequivocally effective treatments such as haemophilia therapies should be assessed differently from mainstream interventions, that new methodologies are required for these kinds of diseases and that evidence of a societal willingness to support people with rare disorders needs to be recognized when reimbursement policies are developed.

Estimates of utility weights in hemophilia: implications for cost-utility analysis of clotting factor prophylaxis

“…Despite the lack of clear evidence of utility of CSPBMs over generic measures, development of new measures continues to occur, motivated in large part by concerns over the insensitivity of generic measures [81,82]. An alternative is to abandon CUA and QALYs in favor of other metrics [11]. …”

“…This paper does not ask whether prophylaxis is cost-effective, in part because the criteria for cost– effectiveness are variable and often arbitrary [10]. Some have suggested that different economic criteria be used, such as cost per bleed prevented or monetary benefit based on willingness to pay [11]. Instead, the focus of this paper is on the sources and implications of uncertainty in the utility scores used to assess cost–effectiveness in CUAs.…”

Using Value-of-Information Methods when the Disease Is Rare and the Treatment Is Expensive—The Example of Hemophilia A

“…The results from past cost-utility (modeling) studies conducted in the UK, Germany, and Canada varied significantly, with incremental cost-effectiveness ratios ranging from low £37,000 per quality-adjusted life years (QALYs) to above €1million per QALY when comparing FVIII prophylaxis with on-demand therapy. 37 Our study results must be interpreted in the light of potential limitations. We made assumptions about several parameters, including patient accrual fraction, time horizon of the decision, and trial costs, that may have been incorrect.…”

Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)

“…These frameworks have the advantages of providing sufficient flexibility to incorporate all value elements of importance and, as shown in a pilot study of a rare disease MCDA framework, are capable of incorporating different stakeholder perspectives [21]. In contrast, where cost-effectiveness frameworks have been used to assess OMPs, they have often failed to be sufficiently flexible to capture all relevant elements and perspectives [49–51]. …”

“…Cost effectiveness analysis (particularly when restricted to a health service perspective) is not optimal for the assessment of OMPs, as the QALY fails to account for all of the core elements of value relevant to rare diseases [49–51]. If it is to be used to inform assessments of OMPs, the ICER thresholds should be modulated to reflect all the specificities of rare diseases as described in Fig.…”