Clinical trial NCT04775888: Accessibility of Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies
Official title: Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies: a Comparison of Perceived and Observed Accessibility in the Auvergne-Rhône-Alpes Region (France)
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Health Services Accessibility
Blood Coagulation Factor Deficiencies
Keywords: haemophilia
accessibility
on-demand treatment
prophylactic treatment
Lead sponsor: RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône
Sponsor class: OTHER
Collaborators: name: Hospices Civils de Lyon
First posted: 2021-03-01
Enrollment: 500
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The included subjects comprise people with haemophilia or other coagulation deficiencies, with no limitation of age, treated by prophylaxis or on-demand treatment, in one of the authorized hospital pharmacies in the Auvergne-Rhone-Alpes region (France).

Brief summary: The current treatment of people with haemophilia and other bleeding deficiencies is largely based on clotting factor replacement therapy. The injections can be repeated several times a week according to a personalized schedule. To date, medications are exclusively dispensed in hospital pharmacies to ensure traceability and safety. This retrocession imposes accessibility constraints on patients and on their caregivers, increasing the burden of the disease, particularly in the organization of personal and professional daily life. The PHAREO study aims to investigate patients' perception of accessibility to anti-haemophilia drugs in relation to an evaluation of spatial accessibility in the Auvergne-Rhône-Alpes region (France) in order to consider, if necessary, ways of improving the pathway for patients and their caregivers.

Detailed description: Haemophilia is a rare constitutional hemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy. For many years, the reference treatment, particularly in children, has been based on the use of anti-haemophilic drugs for prophylaxis requiring repeated injections several times a week according to a personalized schedule. In contrast, on-demand treatment is less and less used, particularly in patients with severe forms of haemophilia. Clinical and biological diagnosis, as well as the implementation and monitoring of treatments, are carried out within specialized hospital care structures affiliated with the French national reference center. Medications are dispensed as part of hospital retrocessions. This organization imposes constraints on patients and their caregivers due to their limited accessibility.

The burden related to this disease is probably due to the systematic use of specialized hospital teams such as doctors, nurses for self-injection training for example, and pharmacists. It now seems important to reflect on the evolution of patient pathways that were previously exclusively hospital-centred towards ambulatory care. To do this, various reflections need to be undertaken, including that relating to the accessibility of medication. Indeed, the monthly renewals of these chronic treatments force patients and their caregivers, as well as the parents of children, to go to the hospital, which frequently makes the organization of daily and professional life more cumbersome.

The PHAREO study aims to investigate patients' perception of accessibility to anti-haemophilia drugs in relation to an evaluation of spatial accessibility in order to consider, if necessary, ways of improving the pathway for patients and their caregivers.

The expected benefits of the study are to have: 1) an exhaustive description of the spatial accessibility of the cohort of people living with haemophilia to anti-haemophilia medication within the Auvergne Rhône Alpes region (France) and 2) a better understanding of their needs and their perceptions regarding access to on-demand and prophylactic treatments.

Eligibility criteria: Included:

* Living person on the study start date and presenting with haemophilia or other coagulation deficiencies;
* Treated with prophylaxis or on-demand anti-haemorrhagic treatments;
* Location of medication dispensing within the hospital pharmacies of the HEsora network (Hémophilie Soins Rhône Alpes): 27 eligible hospital pharmacies in Rhône Alpes + \<10 eligible hospital pharmacies in Auvergne

Excluded:

- Home delivery (not included in the study via hospital pharmacy)

Primary outcomes:
measure: To assess the perception of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment
timeFrame: Through study completion, on average 6 months.
measure: To measure the accessibility of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment
timeFrame: Through study completion, on average 6 months.
measure: To compare the perception and the spatial accessibility of persons with haemophilia and other coagulation deficiencies regarding prophylactic and on-demand antihemorrhagic treatment
timeFrame: Through study completion, on average 6 months.
Secondary outcomes:
measure: To determine if the severity of the disease would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
timeFrame: Through study completion, on average 6 months.
measure: To determine if the age of the patient would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
timeFrame: Through study completion, on average 6 months.
measure: To determine if the condition of access to hospital would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
timeFrame: Through study completion, on average 6 months.
measure: To determine if the level of autonomy of the participant would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
timeFrame: Through study completion, on average 6 months.
measure: To determine if the availability of the hospital pharmacy would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
timeFrame: Through study completion, on average 6 months.
Other outcomes:
Locations:
city: Albertville
country: France
facility: Centre Hospitalier Albertville-Moûtiers
city: Annecy
country: France
facility: Centre Hospitalier Annecy Genevois
city: Annonay
country: France
facility: Centre Hospitalier d'Ardèche Nord
city: Aubenas
country: France
facility: Centre Hospitalier d'Ardèche Méridionale
city: Aurillac
country: France
facility: Centre Hospitalier Henri Mondor d'Aurillac
city: Belley
country: France
facility: Centre Hospitalier de Belley
city: Bourg St Maurice
country: France
facility: Centre hospitalier de Bourg St Maurice
city: Bourg-en-Bresse
country: France
facility: Centre Hospitalier Fleyriat
city: Bourgoin-Jallieu
country: France
facility: Centre Hospitalier Pierre Oudot
city: Bron
country: France
facility: Groupement Hospitalier Est, Hospices Civils de Lyon
city: Chambéry
country: France
facility: Centre Hospitalier de Chambéry - Hôpital Savoie
city: Clermont-Ferrand
country: France
facility: Centre Hospitalier Universitaire de Clermont-Ferrand
city: Contamine-sur-Arve
country: France
facility: Centre Hospitalier Alpes Léman
city: Gleizé
country: France
facility: Hôpital Nord Ouest de Villefranche-sur-Saône
city: La Tronche
country: France
facility: Centre Hospitalier Universitaire Grenoble - Alpes
city: Lyon
country: France
facility: Hôpital Edouard herriot, Hospices Civils de Lyon
city: Lyon
country: France
facility: Hôpital de la Croix-Rousse, Hospices Civils de Lyon
city: Montbrison
country: France
facility: Centre Hospitalier du Forez
city: Montluçon
country: France
facility: Hôpital de Montluçon
city: Montélimar
country: France
facility: Centre Hospitalier de Montélimar
city: Moulins
country: France
facility: Centre Hospitalier de Moulins-Yzeure
city: Oyonnax
country: France
facility: Centre Hospitalier Du Haut Bugey
city: Privas
country: France
facility: Centre Hospitalier des Vals d'Ardeche
city: Roanne
country: France
facility: Centre Hospitalier de Roanne
city: Romans-sur-Isère
country: France
facility: Hôpitaux Drôme Nord
city: Saint-Genis-Laval
country: France
facility: Pharmacie Centrale, Hospices Civils de Lyon
city: Saint-Julien-en-Genevois
country: France
facility: Centre Hospitalier Intercommunal Sud-Léman Valserine
city: Saint-Priest-en-Jarez
country: France
facility: Centre Hospitalier Uuniversitaire Nord, Saint-Étienne
city: Sallanches
country: France
facility: Hôpitaux du Pays du Mont-Blanc
city: Tarare
country: France
facility: Hôpital Nord-Ouest Tarare-Grandris
city: Thonon-les-Bains
country: France
facility: Hopitaux du Léman
city: Valence
country: France
facility: Centre Hospitalier de Valence
city: Vichy
country: France
facility: Centre Hospitalier de Vichy
city: Vienne
country: France
facility: Centre Hospitalier Lucien Hussel
Study officials:
affiliation: RESCUe-RESUVal
name: Laurie Fraticelli, PhD
role: STUDY_DIRECTOR
affiliation: RESCUe-RESUVal
name: Julie Freyssenge, PhD
role: STUDY_DIRECTOR
References:
citation: Leroy V, Freyssenge J, Renard F, Tazarourte K, Negrier C, Chamouard V. Access to treatment among persons with hemophilia: A spatial analysis assessment in the Rhone-Alpes region, France. J Am Pharm Assoc (2003). 2019 Nov-Dec;59(6):797-803. doi: 10.1016/j.japh.2019.07.006. Epub 2019 Aug 9.
citation: Susen S, Gruel Y, Godier A, Harroche A, Chambost H, Lasne D, Rauch A, Roullet S, Fontana P, Goudemand J, de Maistre E, Chamouard V, Wibaut B, Albaladejo P, Negrier C. Management of bleeding and invasive procedures in haemophilia A patients with inhibitor treated with emicizumab (Hemlibra(R) ): Proposals from the French network on inherited bleeding disorders (MHEMO), the French Reference Centre on Haemophilia, in collaboration with the French Working Group on Perioperative Haemostasis (GIHP). Haemophilia. 2019 Sep;25(5):731-737. doi: 10.1111/hae.13817. Epub 2019 Jul 11.