Expectations and Concerns Towards New Extended Half-Life (EHL) Products - Results of Surveys Among Haemophilia Patients from the Dach Region
Authors: von Mackensen Sylvia , Kalnins Werner , Lino Hostettler , Joerg Krucker , Albisetti Manuela , Weiss Josef , Pabinger Ingrid , Oldenburg Johannes 
Published: October 2019

Abstract
Background: In Western Europe most patients with haemophilia (PWH) have the opportunity to get regular prophylaxis and can life normal lives due to the availability and funding of factor concentrates. The majority of haemophilia patients have to inject themselves every other day or three times a week.The need of frequent injections may impact negatively patients' treatment adherence which might result in increased annual bleeding rate and impaired quality of life. Therefore there is a need for half-life extended (EHL) products which will be launched in the next years in Europe for treatment of haemophilia A and B and are expected to improve patients' quality of life. However, little is known about patients' perspectives on the use of these new EHL products and their willingness to switch to these products.
Aim: National Member Organisations (NMO) of haemophilia patients from the DACH Region (D=Germany, A=Austria, CH=Switzerland) were interested to better understand PWH's expectations and concerns towards these new EHL products in order to adapt haemophilia treatment to real patients' needs.
Methods: A systematic postal survey was sent out to 2,644 patients and parents of children with haemophilia A or B in Germany (2015), Switzerland (2016) and Austria (2016). The postal survey included questions concerning demographic and clinical data of the patient, knowledge about half-life of actual factor concentrates (FC) and attitudes towards FC in terms of satisfaction with actual factor concentrate, expectation towards new EHL products, willingness to switch to new EHL products, required information for decision making, preferred information sources and facilitation of therapy in general.
Results: In total 1,013 questionnaires were sent back (38.3%); 2 patients had to be excluded from further analysis since these patients had VWD. 747 adult haemophilia patients and 262 parents of haemophilic children participated, 2 respondents could not be classified. The majority of patients had haemophilia A (84.1%), were severely affected (73%), received prophylaxis (56.6%), mostly three times a week (40.9%) and used recombinant products (59.4%). 14% had a history of an inhibitor (past, actual). Most of the patients and parents did not know the correct half-life of the actual FC [FVIII: 30%, FIX: 75%]. In general they were satisfied (M=4.4±0.8) with their actual FC; only 4.1% were unsatisfied, mainly with difficult manageability and short half-life of FC. They expected from the new products to provide less frequent injections, better efficacy and safety/no side effects. 59.6% would be willing to switch to new products if they have a prolonged half-life and have the same safety of the actual FC. Reasons for not willing to switch were fear of inhibitor development and no side effects of actual FC. They had 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. 20.6% would switch already with a half-life prolongation of 1.2-1.5 times longer; 3.3% would not switch at all. The majority wanted to receive information about new products from their haemophilia treater (77.1%), newsletter of their NMO (73.4%) and information letter from their HTC/NMO (35.7%). They would wish smaller packages, different mode of administration and better manageability of the product in order to facilitate their therapy.
There was a significant difference across countries regarding treatment regimen (p<.0001) and used product category (p<.0001); in Germany more patients were on prophylaxis (71.3%) and in Switzerland more patients used recombinant products (75.8%). No differences were found for the satisfaction with their actual product, their willingness to switch to new EHL products in general and the time of required half-life prolongation. In Switzerland more patients required information on costs of the new EHL products for an adequate decision making process for switching (p<.0001).
Conclusions: In this representative survey among patients and parents of children with haemophilia of the DACH Region it could be shown that even though they were generally satisfied with their actual FC the majority would be willing to switch from their actual FC to the new up-coming EHL products assuming the half-life is prolonged and has the same safety of the actual FC.

Disclosures
von Mackensen: Shire: Research Funding; SOBI: Research Funding. Kalnins:SOBI: Research Funding. Lino:SOBI: Research Funding. Joerg:SOBI: Research Funding. Albisetti:Boehringer Ingelheim: Other: Pediatric Expert Working Group. Weiss:Shire: Research Funding.