Perception of Actual Factor Concentrates and Expectations Towards Future Treatment with New Long-Lasting Products - Results of a Survey Among Haemophilia Patients in Germany - Analysis of Open Questions

Background: Prophylactic treatment allows patients with haemophilia (PWH) to live an approximately normal life. The majority of German PWH receive prophylaxis. The need of frequent weekly injections may impact negatively treatment adherence. This is the expected reason which might result in increased annual bleeding rate. Therefore there is a need for half-life extended products which will be launched end of this year in Germany. 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 actual factor concentrates (FC) and attitudes towards actual FC and open ended questions concerning the expectations towards new products and how therapy could be facilitated. The open ended questions should give the participants the chance to write down other aspects, problems, laments and other wishes. Several aspects could be mentioned by the same subject.

Out of the sent 1,499 questionnaires 697 questionnaires were sent back; mainly from adult patients (n=518), 177 from parents of haemophilic children. Patients had haemophilia A (83.7%), in a severe form (77.8%), were on prophylaxis (61.4%), mostly three times a week (47.8%) and used recombinant products (57.8%). 14.1% have had an inhibitor.

Open questions were answered by 462 respondents, at least 1 to maximum 5 aspects were mentioned.

Open questions on the current situation ["facilitation of therapy"] (n=302): less packaging waste (n=129), no cooling required (n=61), sc injections/oral medication/nasal medication (n=54), better supplies like perfect injection needle etc. (n=27), prefilled syringe (n=25), diversity of package size (125 iU to 4000 iU) (n=24), support in home care therapy (n=17), simpler treatment documentation (n=9), improved logistic/delivery for home treatment (n=8).

Open questions for future situation (n=160) ["expectations towards new long-lasting products"]: less injections (n=492), same safety/efficacy/compatibility (n=44), better quality of life (n=22), no fridge (n=16), treatment costs/reduction-no increase (n=15), higher trough level (n=9), smaller packaging (n=8), easier handling (n=8), and many more mentioned 1- 3 times.

Conclusions: The answers to the open questions in this representative survey among German haemophilia patients and parents of children with haemophilia provide important additional information and insights in the unmet needs of haemophilia patients, which will help the German patient association and pharmaceutical companies to better inform patients about the new up-coming products taking patients needs into consideration.