Perception of Actual Factor Concentrates and Expectations Towards Future Treatment with New Long-Lasting Products - Results of a Survey Among Haemophilia Patients in Germany
Authors: von Mackensen Sylvia , Heinrich Struck , Kalnins Werner , Oldenburg Johannes 
Published: October 2019

Abstract
Background: Prophylactic treatment allows patients with haemophilia (PWH) to live a normal life, however the need of frequent weekly injections may impact negatively treatment adherence which might result in increased annual bleeding rate and impaired quality of life. Therefore there is a need for half-life extended products which will be launched in the next years. However, little is known about patients' perspectives on the use of new products with extended half-life and their willingness to switch to these products.
Aim: The German patient organisation DHG is interested to better understand PWH's expectations and concerns towards these new products in order to adapt their support for the haemophilia community.
Methods: The study consisted of two phases: 1) conduct of separate focus groups with adult haemophilia patients and parents of haemophilic children with respect to the new long-lasting products across Germany; 2) based on the focus group results construction of a questionnaire for a systematic postal survey among DHG members with haemophilia A or B (n=1,499).
Results: The postal survey included questions concerning haemophilia, knowledge about half-life of factor concentrates and attitudes towards factor concentrates (FC). In total 697 questionnaires were sent back (46.5%); 518 patients and 177 parents of haemophilic children. The majority of patients had haemophilia A (83.7%), were severely affected (77.8%), received prophylaxis (61.4%), mostly three times a week (47.8%) and used recombinant products (57.8%). 14.1% had a history of an inhibitor. Most of the patients and parents did not know the correct half-life of the actual FC [FVIII: 30.1%, FIX: 74.5%]. In general they were satisfied (M=4.4±0.8) with their actual FC; only 4.1% were unsatisfied, mainly with short half-life, difficult manageability and need to store it in the fridge. They expected from the new products to have prolonged half-life, better stability, better transparency concerning production of the FC and improved manageability. 59% would be willing to switch to new products if they have a prolonged half-life, provide more security when travelling and have the same safety of the actual FC. Reasons for not willing to switch were fear of inhibitors and uncertain safety of new product as well as satisfaction and no side effects of actual FC. They have almost no information about the new up-coming products, wish more information about half-life, possible side-effects and efficacy and would consider changing product if the prolongation of the half-life is at least double as high as the actual FC. The majority wants to receive information about new products from their haemophilia treater (79%), leaflets/newsletter of DHG (77%) and information letter from HTC/DHG (32%).
Analyses between clinical subgroups showed significant differences for knowledge of half-life of FVIII for type of haemophilia (p<.0001), severity (p<.0001), treatment (p<.0001) and age (p<.031). Significantly more patients on prophylaxis would be willing to switch to new products because of their prolonged half-life (92%; p<.0001) and benefits for doing sports (53%, p<.0001), while on-demand patients would do so because of their longer stability outside the fridge (69%; p<.0001), advantages for surgeries (68%; p<.0001), easier application (44%; p<.001) and 24% would be motivated to switch to prophylaxis. No differences were found for satisfaction with actual FC and the willingness to switch to new products. Presence of inhibitors did not have an impact.
In the five focus groups 23 adult haemophilia patients (78.3% HA; 78.3% severely affected) and 9 parents of haemophilic children (88.9% HA; 77.8% severely affected) were interviewed (Hamburg, Cologne, Munich, 2x Dresden).
Conclusions: In this representative survey among German patients and parents of children with haemophilia they reported to be satisfied with their actual FC. Nevertheless, unsatisfactory aspects of their actual FC are half-life, manageability and storage conditions. The majority did not have a lot of information of the new long-lasting products, but would be willing to switch from the actual FC to these products assuming the half-life is prolonged and has the same safety of the actual FC. These findings will help patient associations and pharmaceutical companies to better inform patients about the new up-coming products taking patients needs into consideration.

Disclosures
von Mackensen: SOBI: Consultancy. Heinrich:SOBI: Consultancy. Kalnins:SOBI: Consultancy. Oldenburg:SOBI: Consultancy.