Health Technology Assessment for Rare Diseases: Evaluation of Preferences toward treatments for Patients with Haemophilia
Creators: name: Scalone, Luciana
Publisher: name: Università degli Studi di Napoli Federico II
Year: 2008

description: Rationale 
Despite the success of recent investments in health technology for the treatment in hemophilic patients, today, inhibitor development is a major complication of hemophilia treatment, as inhibitors increase the risk of uncontrollable bleeding, make surgery more problematic, increase the risk of severe physical disabilities, reduce wellbeing, and represent a potential cause of premature mortality. 
The introduction of new options, such as immune tolerance induction and the use of bypassing agents, has significantly improved treatment success in inhibitor patients in even the most challenging situations (e.g. emergencies, home treatment, and surgery). However, these treatment advances are paralleled by significant increases in the cost of care for these patients, and economic constraints can cause limited access to optimal therapy.  In order to optimize the benefits derived from use of the available resources, it is necessary to know and compare both present and future effects and costs of alternative options. Identification of the most efficient option allows implementation of appropriate investments, which must be considered as opportunities to improve patients’ health and wellbeing. Implementing rational investments may also facilitate economic benefits in the future, with potential advantages not only for patients, but also for their families and for society as a whole. 
However, there is still a lack of consensus on how to optimally treat haemophilia patients with inhibitors. Considerable uncertainty remains about the optimum treatment choice in different clinical circumstances. Furthermore, in several healthcare systems, resource constraints are main potential obstacles for access to the most efficient treatment options. As a result, clinicians have for several years made decisions on how to manage their patients according to their own experience and opinions. These decisions can differ  depending on the type of patient, on the bleeding situation, and on possible considerations related to safety, immunogenicity, practicalities, product availability, and costs. 
The management of these patients actually involves a complex interaction between different parties, i.e. physicians, patients or their caregivers, and budget holders. Each of these parties has their own set of preferences, which is influenced by the role they play in the healthcare system, their experiences, and their expectations. In order to optimise the appropriateness of the decisions, it is necessary to be both informed and aware of the opinions and preferences of the interested parties. This knowledge can be useful in order to better understand the potential benefits of treatments, thereby improving the success, and possibly also increasing the efficiency, of the intervention.
	
Objective 
This work aimed to evaluate preferences towards the characteristics of different coagulation factor concentrates for haemophilia inhibitors patients, from the perspective of patients or their caregivers, haematologists, pharmacists. 

Methods
A discrete choice study was conducted. Potential products were described with eight selected characteristics: perceived viral safety, risk of anamnestic response, possibility of undergoing major surgery, frequency of infusions in prophylaxis, number of infusions to stop bleeding, time to stop bleeding, time to pain recovery, cost. Participants received 16 pairs of potential products and chose from each pair the option they considered better. Data were analyzed with a random-effects conditional logistic model. 

 
Results
1614 observations were obtained from 37 patients/caregivers, 39 physicians, and 25 pharmacists from Italy. Cost, in terms of additional healthcare taxes, was the most important attribute to every group: the relative importance assigned to this characteristic was 21% by the patients/caregivers and 26-27% by the physicians and pharmacists, suggesting that unlike to what it may be thought, cost is an important issue also to people not directly involved in the payment for these products. 
For patients/caregivers the next most important factors were: risk of anamnestic response (RI = 20%), possibility of undergoing major surgery (RI = 18%), perceived viral safety (RI = 18%). For physicians the next most important characteristics were: risk of anamnestic response (RI = 18%), number of infusions to stop bleeding (RI = 14%), possibility of undergoing major surgery (RI = 13%). For pharmacists the next most important factors were: time to stop bleeding (RI almost 20%), time to pain recovery (R = 14%), possibility of undergoing major surgery (RI = 13%).

Discussion
To our best knowledge this is the first study estimating and comparing the value given by patients (or their caregivers), physicians, and pharmacists to the different characteristics of coagulation factor concentrates used for the treatment of haemophilia patients with inhibitors. Not only outcome attributes, such as those related to safety, like viral and risk of anamnestic response, those related to effectiveness, like the time to stop a bleeding, the time to alleviate the pain and possibility of undergoing major surgery, but also process attributes (frequency of infusions to stop a bleeding or to follow a prophylaxis regimen) and cost are considered important for a product used in patients with inhibitors. 
Decisions on treatments must take into account patients’ characteristics and their clinical needs; however, preferences can also play an important role in the choice and success of treatments. The results of this study could, therefore, help decision-makers to optimise the overall benefits of treatments.