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. 2024 Aug 14:18:1713-1727.
doi: 10.2147/PPA.S470091. eCollection 2024.

The Concizumab Pen-Injector is Easy to Use and Preferred by Hemophilia Patients and Caregivers: A Usability Study Assessing Pen-Injector Handling and Preference

Niklas Kahr Rasmussen  1 , Birgitte Berg  2 , Anne Sia Louise Christiansen  3 , Jesper Skov Neergaard  1 , Gitte Ter-Borch  2 , Emily A Hildebrand  4 , Maya Gonczi  4 , Thomas Sparre  1
Affiliations

The Concizumab Pen-Injector is Easy to Use and Preferred by Hemophilia Patients and Caregivers: A Usability Study Assessing Pen-Injector Handling and Preference

Niklas Kahr Rasmussen et al. Patient Prefer Adherence. .

Erratum in

Abstract

Introduction: Concizumab is a once-daily prophylactic treatment developed for patients with hemophilia A or B (HA/HB) with or without inhibitors. It is the first treatment for hemophilia patients to be delivered subcutaneously using a pre-filled, multi-dose pen-injector with a 4 mm, 32 G needle.

Aim: To investigate patient and caregiver handling and preference for the concizumab pen-injector compared with current injection systems used to treat hemophilia.

Methods: This preference and handling study was conducted in accordance with authority guidelines for approval of new devices and included adults and adolescents with HA/HB with or without inhibitors and caregivers currently administering factor replacement therapy or factor VIII mimetic (emicizumab) therapy. All participants underwent a training session, followed by a test session during which participants independently administered a single pen-injection into an injection pad or manikin. Time to train, time to prepare and inject, and number of complete independent injections handling the pen were assessed. Participants evaluated handling and preference via the Hemophilia Device Handling and Preference Assessment Questionnaire.

Results: 80 participants (44 adults, 21 adolescents, 15 caregivers) currently using factor replacement therapy (n=41, 51%) or emicizumab (n=39, 49%) participated. Average training time and time to complete an injection were 7 min 49s and 1 min 21s. In total, 98% of independent complete injections were achieved at first attempt. 98% (n=78; 95% confidence interval [CI] 91-100%) of participants assessed the pen-injector as either "easy" or "very easy" to use. 88% of participants preferred the pen-injector (n=70; 95% CI 78-94%) over their current injection system, and 9% (n=7) reported "no preference".

Conclusion: Participants found the concizumab pen-injector easy to learn and easy to use and preferred it over their current injection systems.

Keywords: concizumab; pen-injector; preference and handling study; questionnaire; subcutaneous.

Plain language summary

People with hemophilia lack clotting factors that help stop bleeding after an injury. If left untreated, it can lead to life-threatening bleeding episodes. Treatment such as replacement therapy with clotting factors needs to be administered at regular intervals to prevent bleeding. The administration of intravenous (inside the vein) medication can be challenging due to the required skills, injection pain and availability of a good vein to inject into. Factor VIII mimetic therapy now allows people to inject medication under the skin (subcutaneously). However, using vials and syringes still poses challenges with the time to prepare and inject medication. Pen-injectors have been developed to address these challenges and provide additional benefits, including increased dose accuracy, portability, reduced discomfort, and less administration time. Concizumab is a medication developed for once-daily subcutaneous injection for people with hemophilia A or hemophilia B, with or without inhibitors, using a pen-injector. The objective of this preference and handling study was to understand patient and caregiver pen-injector handling experiences, and device preferences compared to their existing devices. In this study, most participants found it easy to learn to use the pen-injector. 98% of participants reported that the pen-injector was easy or very easy to use compared with their current device. 88% of participants reported a preference for the concizumab pen-injector over their current device. This shows that the pen-injector can easily and effectively be used to inject concizumab, providing more control and independence in patients’ treatment while reducing the burden caused by intravenous modes of administration.

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Conflict of interest statement

NKR, BB, ASLC, JSN, GTB, and TS are employees of Novo Nordisk. EH and MG are employees of Research Collective, LLC, Tempe, Arizona, USA. Novo Nordisk is the sponsor of the trial. The study was conducted by an independent CRO. The authors report no other conflicts of interest in this work.

Figures

Figure 1
Figure 1
Concizumab pen-injector components. The concizumab pen-injector is shown with labelled individual components. Each pen-injector is supplied as a pre-filled pen containing 150 mg concizumab to be used with NovoFine® Plus 32G, 4 mm long injection needles, which come with inner and outer needle cap. The pen can deliver a maximum of 80 mg in one injection. The interval on the dose counter is 1 mg.
Figure 2
Figure 2
Overview of study design and implementation.
Figure 3
Figure 3
The ease of learning and ease of use of the pen‑injector as assessed using the HDHPA questionnaire. (A) Responses from participants to HDHPA question 1 on factors associated with ease of handling and use of concizumab pen-injector. Participants were asked to select the response that most closely represented how they felt using the pen-injector. (B) Responses from participants to HDHPA question 2 on overall difficulty or ease of use of the concizumab pen-injector. Responses were assessed on a 5-point scale ranging from very difficult to very easy. Responses for very difficult and difficult were combined to represent negative results and the responses easy and very easy were combined to represent positive results. Data are presented as number of responses with percentages % (n).
Figure 4
Figure 4
Confidence in using and quick preparation of the pen-injector. (A) Responses from participants to HDHPA question 3 on confidence in using the pen-injector correctly. (B) Responses from participants to HDHPA question 3 on confidence that the pen-injector delivered the correct full dose. Responses were assessed on a scale of 5 (not at all confident, a little confident, somewhat confident, very confident, extremely confident). Responses to not at all confident and a little confident were combined to represent negative results. Responses to very confident and extremely confident were combined to represent positive results. (C) Responses from participants to HDHPA question 4 on how slow or quick it was to inject with the concizumab pen-injector. Responses were assessed on a scale of 5 (very slow, slow, neither slow or quick, quick, very quick). Responses to slow and very slow were combined to represent negative results, and responses for quick and very quick were combined to represent positive results. Data are presented as number of responses with percentages % (n).
Figure 5
Figure 5
Comparison of the pen-injector to the patient’s current device as assessed using the HDHPA questionnaire. (A) Responses from participants to HDHPA question 5 on factors associated with ease of handling and use of the concizumab pen-injector compared to their current device. (B) Responses from participants to HDHPA question 6 on overall ease or difficulty of use of the concizumab pen-injector compared to their current device. Responses were assessed on a scale of 5 (much more difficult, more difficult, neither more difficult nor easy, easier, much easier). Responses for much more difficult and more difficult were combined to represent negative results, and responses for easier and much easier were combined to represent positive results. Data are presented as number of responses with percentages % (n).
Figure 6
Figure 6
Preference for the pen-injector over the patient’s current device. (A) Responses from participants to HDHPA question 9 on device preference. Device preference was recorded on a scale of 3 (pen-injector used in the study, no preference, my existing device). (B) Responses from participants either on factor replacement therapy or FVIII mimetic therapy (emicizumab) on device preference. (C) Responses from participants to HDHPA question 10 on strength of the device preference for those who preferred concizumab pen-injector (n=70) recorded on a scale of 3 (not very strong, fairly strong, very strong).
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