As combination products incorporating drug-delivery devices—particularly autoinjectors and prefilled pens—become increasingly common in biologics and specialty therapeutics, regulatory and clinical development teams face growing pressure to demonstrate that patients can use these devices safely and effectively in real-world conditions. Regulators in the EU and beyond expect sponsors to integrate human-factors evidence, including actual-use studies and patient usability assessments, directly into clinical programmes rather than treating device evaluation as a post-approval afterthought. Understanding how other sponsors have structured these components within authorised trial protocols is therefore a practical priority for teams designing studies under the EU Clinical Trials Regulation (536/2014).
This analysis draws exclusively on trial records published through the EU Clinical Trials Information System (CTIS) to identify protocols in which a genuine actual-use study, patient or caregiver usability assessment, or device human-factors component was built into the trial design for an autoinjector, prefilled pen, or comparable self-injection device, with patients or caregivers performing the injections. EPAR assessment reports were excluded from the dataset. The review covers the search methodology used to distinguish substantive usability elements from routine self-administration language, the qualifying trials identified, and the specific design features and outcome instruments those protocols employed.
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Actual-use and patient usability studies of autoinjectors and prefilled pens in EU CTIS trial protocols
Key findings
Three distinct designs recur in the protocols:
- Dedicated device usability / human-factors actual-use sub-studies at selected sites, in which participants perform observed injections and investigators record injection success, use errors, close calls, and handling difficulties. These are the closest fit to a classic actual-use study embedded in a therapeutic trial.
- Patient-reported self-injection usability captured through a validated PRO, most commonly the Self-Injection Assessment Questionnaire (SIAQ) administered PRE (before first self-injection) and POST (immediately after self-injecting), plus device preference/usability questionnaires.
- Self-administration training, certification, and user-error/device-malfunction surveillance, where patients or caregivers are trained and certified to inject at home and the protocol monitors device malfunctions and use errors, without a standalone usability sub-study.
Tier 1 — Dedicated autoinjector/prefilled-pen actual-use and usability sub-studies
- GlaxoSmithKline, Benlysta (belimumab) 200 mg prefilled pen (Phase III, 2023-509370-39-00). The protocol includes a human-factors/autoinjector device usability sub-study at selected US sites. It captures observed actual injections against an injection-step checklist, visual inspection, injection success, use errors, close calls and use difficulties, plus a self-reported usability questionnaire on ease of use, handling and acceptance. The usability component is for participants who self-administer; some participants may need caregiver administration due to hand disability. 219
- Janssen-Cilag, Simponi (golimumab) prefilled syringe and 45 mg prefilled pen (Phase III, 2023-507142-83-00). A Usability Assessment Substudy at sponsor-identified sites collects participant/caregiver experience with the prefilled syringe and the pen (ultra-safe device) after self-dosing at Weeks 62 and 66. 251
- Pfizer, marstacimab prefilled pen (Phase III, 2022-500470-33-00). A Participant Assessment Tool (PAT) sub-study: the actual prefilled-pen user injects, records their experience, inspects the pen window for residual drug, and immediately records injection success and any difficulties after 6 or 12 injections. 252
Tier 2 — Patient-reported self-injection usability (SIAQ and preference/usability PROs)
- Immunovant, IMVT-1402 subcutaneous self-injection — four Phase III protocols (2025-521920-31-00, 2024-515979-35-00, 2024-516020-33-00, 2024-515973-82-00). Each protocol administers the SIAQ as PRE-self-injection and POST-self-injection modules; the POST form is completed only by participants who self-administer, immediately after giving themselves the injection. 199210201203
- Camurus AB, CAM2029 (octreotide subcutaneous depot) (Phase III, 2024-510667-33-00). The SIAQ is completed by patients who choose to self-administer: PRE before first self-administration and POST 20 to 40 minutes after, and after subsequent self-administrations; results tables report self-injection scores. 218
- Sanofi, frexalimab self-injection device (Phase III, 2024-519304-28-00). A patient-facing preference and usability questionnaire assesses preference for home versus clinic injection, ease of device preparation, confidence to self-inject, and whether the participant would recommend the injection method. 163
Tier 3 — Self-administration training, certification and use-error/device-malfunction monitoring
These protocols require patients or caregivers to inject and build in structured training, proficiency certification, and safety surveillance of the device, but do not describe a standalone usability sub-study. Device type is noted because several use a prefilled syringe or vial kit rather than a pen/autoinjector.
- Novo Nordisk, ziltivekimab pen-injector and manual syringe (Phase III, 2022-501939-16-00). Participants or caregivers inject monthly; first administrations are observed/trained at study visits, a patient-administration video is provided in the study app, and injection date/time/site are recorded in the app. 250
- GSK, Benlysta prefilled pen — user-error monitoring (Phase IV, 2023-509146-35-00). Participants and caregivers are trained, first doses supervised, and device malfunction and user errors are collected throughout the study. 224
- GSK, Benlysta prefilled syringe — formative human-factors risk mitigation (integrated Phase II/III, 2023-503219-14-01). Self-administration risk mitigation is based on formative human-factors studies; participants/caregivers are educated and the first two scheduled doses are supervised. 222
- Novartis, MAS825 vial kit for subcutaneous injection (Phase II, 2024-520208-25-00 and 2023-504419-34-00). A two-step training/certification process for self-injection at site and at home, with HCP certification of successful training and patient/caregiver confirmation of confidence to self-inject at home. 168169
- Roche, crovalimab subcutaneous administration (Phase III, 2023-506526-37-00, 2023-506498-36-00, 2023-505089-27-00). Training for self-administration over the first five SC doses in a monitored setting, with HCP evaluation of self-injection capability before unsupervised use. 183184185
- Janssen-Cilag, guselkumab prefilled syringe (2023-504737-41-00, 2023-504716-15-00). Observed first use with supervised self-injection and training (including injection-pad simulation and an eCRF proficiency record); home self-administration is allowed once site staff judge the participant/caregiver can inject safely and effectively. 187180
- Eli Lilly, mirikizumab (Phase III, 2022-502841-91-00). A self-administration sub-study with training, observed first injection by site personnel, then participant/caregiver administration with oversight and optional remote retraining, documented via patient administration training and administration logs. 177
- Sanofi, frexalimab home injections (Phase III, 2024-513527-17-00). Home injections with participant self-evaluation of pain (verbal descriptor scale before, after, and 2 hours post-administration) and local tolerability monitoring. 238
- argenx, efgartigimod PH20 SC ready-to-use prefilled syringe (2023-507885-21-00). Self-administration or caregiver-supported administration at home; participants are educated on and self-monitor injection/infusion-related reactions. 240
- Charité Universitätsmedizin Berlin, Ebglyss (lebrikizumab) prefilled syringe (Phase III, 2025-524435-39-00). Transition to home self-administration after supervised dosing and tolerability confirmation. 243
Summary table
| Trial ID | Sponsor | Device (as stated) | Usability design | Instrument / endpoint | Injector | Cite |
|---|---|---|---|---|---|---|
| 2023-509370-39-00 | GSK | Benlysta prefilled pen | Human-factors actual-use sub-study | Injection-step checklist, injection success, use errors, usability questionnaire | Patient (caregiver if disabled) | 219 |
| 2023-507142-83-00 | Janssen-Cilag | Simponi prefilled pen + syringe | Usability Assessment Substudy | Post-self-dose experience (Wk 62/66) | Patient/caregiver | 251 |
| 2022-500470-33-00 | Pfizer | marstacimab prefilled pen | Participant Assessment Tool sub-study | Injection success, difficulties, pen-window inspection | Patient/caregiver | 252 |
| 2025-521920-31-00 | Immunovant | IMVT-1402 SC self-injection | PRO usability | SIAQ PRE/POST | Patient | 199 |
| 2024-515979-35-00 | Immunovant | IMVT-1402 SC self-injection | PRO usability | SIAQ PRE/POST | Patient | 210 |
| 2024-516020-33-00 | Immunovant | IMVT-1402 SC self-injection | PRO usability | SIAQ PRE/POST | Patient | 201 |
| 2024-515973-82-00 | Immunovant | IMVT-1402 SC self-injection | PRO usability | SIAQ PRE/POST | Patient | 203 |
| 2024-510667-33-00 | Camurus | CAM2029 octreotide SC depot | PRO usability | SIAQ PRE/POST, self-injection scores | Patient | 218 |
| 2024-519304-28-00 | Sanofi | frexalimab self-injection device | Preference/usability PRO | Ease-of-prep, confidence-to-self-inject | Patient | 163 |
| 2022-501939-16-00 | Novo Nordisk | ziltivekimab pen-injector | Training + observed administration | Observed correct administration, app log | Patient/caregiver | 250 |
| 2023-509146-35-00 | GSK | Benlysta prefilled pen | Use-error surveillance | Device malfunction / user error capture | Patient/caregiver | 224 |
| 2023-503219-14-01 | GSK | Benlysta prefilled syringe | Formative human-factors mitigation | Supervised first 2 doses | Patient/caregiver | 222 |
| 2024-520208-25-00 | Novartis | MAS825 vial kit SC | Training/certification | Training checklist, HCP certification | Patient/caregiver | 168 |
| 2023-504419-34-00 | Novartis | MAS825 vial kit SC | Training/certification | Training checklist, HCP certification | Patient/caregiver | 169 |
| 2023-506526-37-00 | Roche | crovalimab SC | Training + capability assessment | HCP proficiency assessment, diary | Patient/caregiver | 183 |
| 2023-506498-36-00 | Roche | crovalimab SC | Training + capability assessment | HCP proficiency assessment, diary | Patient/caregiver | 184 |
| 2023-505089-27-00 | Roche | crovalimab SC | Training + capability assessment | HCP proficiency assessment, diary | Patient/caregiver | 185 |
| 2023-504737-41-00 | Janssen-Cilag | guselkumab prefilled syringe | Training + capability assessment | Injection-pad sim, eCRF proficiency | Patient/caregiver | 187 |
| 2023-504716-15-00 | Janssen-Cilag | guselkumab prefilled syringe | Training + home option | Patient diary | Patient/caregiver | 180 |
| 2022-502841-91-00 | Eli Lilly | mirikizumab | Self-administration sub-study | Administration training/logs | Patient/caregiver | 177 |
| 2024-513527-17-00 | Sanofi | frexalimab home injection | Self-evaluation | Pain verbal descriptor scale | Patient | 238 |
| 2023-507885-21-00 | argenx | efgartigimod PH20 SC prefilled syringe | Home self/caregiver admin | IRR self-monitoring | Patient/caregiver | 240 |
| 2025-524435-39-00 | Charité | Ebglyss prefilled syringe | Home transition | Supervised dosing then home | Patient | 243 |
Reviewer notes and limitations
- The clearest matches to the question (an actual-use or usability study of an autoinjector or prefilled pen with patient injection) are the GSK Benlysta pen human-factors sub-study 219, the Janssen Simponi Usability Assessment Substudy 251, and the Pfizer marstacimab prefilled-pen Participant Assessment Tool 252. The Novo Nordisk ziltivekimab protocol is the other true pen-injector record 250.
- The SIAQ is the dominant validated instrument for patient-reported self-injection usability, appearing across the Immunovant IMVT-1402 programme 199210201203 and the Camurus CAM2029 protocol 218. It is a PRO capturing the patient experience of self-injection rather than an instrumented human-factors actual-use test.
- Several trials in Tier 3 use a prefilled syringe or vial kit rather than a pen/autoinjector; they are included because patients or caregivers perform the injection under a structured training/certification and use-error-monitoring design, but a device reader should treat them as self-administration programmes rather than pen/autoinjector usability studies.
- Coverage caveat: CTIS documents are frequently redacted, and sub-study details may sit in appendices not surfaced by keyword search. This set should be read as a representative map of current sponsor practice, not a complete inventory. A targeted follow-up on any single sponsor or molecule (for example, all Novo Nordisk pen-injector protocols, or every trial using the SIAQ) can be run directly in Rhizome.
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