Patient preferences in the treatment of hemophilia A: impact of storage conditions on product choice

Abstract

ObjectivesTo gain insights into the usage of factor VIII (FVIII) products by patients diagnosed with moderate/severe hemophilia A, and to assess the impact and perceived importance of product storage.MethodsIn this study, 200 patients diagnosed with moderate or severe hemophilia A across seven countries participated. Data were collected via a 30-minute, face-to-face interview in six countries and via a web-based survey in the seventh country. The questionnaire evaluated the effect of six features associated with FVIII products on the choice of the product; the structure and flow of data collection was designed to eliminate potential bias.ResultsTwo-thirds of the respondents were using recombinant FVIII products. Only 17% were generally dissatisfied with current FVIII products, whereas >40% of the respondents were dissatisfied with frequency of administration and storage issues when traveling. The majority noted restrictions in their daily activities, particularly travel and sports. Most of them (85%), stored their product in the refrigerator and of these, 88% believed that it should always be stored there. These patients were also less satisfied with the product overall, more concerned about storage temperature, more restricted in daily activities, and spent more time on preparation and injection compared with patients who stored their product at room temperature. Conjoint analysis revealed that origin of FVIII (plasma-derived vs recombinant) was the strongest driver of product choice among all respondents, followed by storage flexibility (temperature), reconstitution device, and administration frequency. In this study, we did not investigate the efficacy and safety of the product.ConclusionNot refrigerating FVIII products was associated with greater patient satisfaction and less restriction on daily activities. If efficacy and safety are unaffected, then storing FVIII at room temperature might have a positive impact on product choice. Few patients were aware that FVIII can be stored without refrigeration, suggesting that health care professionals who treat hemophilia should communicate this aspect to the patient (depending on the labeled option); this approach might offer patients greater flexibility when traveling and require less time for reconstitution.

Treatment preferences in people with haemophilia A or caregivers of people with haemophilia A: A discrete choice experiment

“…27 In concordance with segment 2, others have also reported greater satisfaction with non-refrigerated Factor VIII products. 28 Treatment preferences identified by patients may differ from those identified by physicians. For example, while both patients and physicians value efficacy of treatment highly, patients may be willing to trade-off convenience for improved efficacy.…”

Patient Perspectives on Novel Treatments in Haemophilia: A Qualitative Study

“…Despite these advancements [6], a cure for haemophilia is not widely available yet and current treatment is still far from optimal. According to patients, products could be improved for frequency of administration [7], efficacy of coagulation products (preventing bleeds) [8], mode of administration [7], easier storage [7,8], fewer side effects (potential transmission of pathogens, antibodies against infused factor VIII or IX) and packaging (size, components of medication, logistics) [7,8]. Intravenous infusion of coagulation factor may pose a problem, especially for young children with delicate veins or for older people, for example, owing to an increased difficulty in self-administration with increasing age [9].…”

The Effect of Fluctuating Temperature on the Stability of Turoctocog Alfa for Hemophilia A

“…Increased understanding of patient/caregiver perspectives and preferences in terms of hemophilia treatment is leading to a growing awareness that factors such as product storage, portability, and usage are directly related to successful disease management [3]. Indeed, a wide range of temperature storage conditions has been cited as one of the biggest drivers of product choice among adult patients and a significant factor in patient satisfaction [4]. Furthermore, flexible factor storage conditions may reduce wastage caused by improper storage [3].…”

“…The stability of FVIII products under varying temperature conditions has been reported as an important element of patient satisfaction, as it allows patients more freedom in their daily lives, particularly with regard to sports and traveling to countries with high daytime temperatures [3, 4]. As such, an important aspect of any FVIII replacement product is its storage flexibility [3].…”