Children and young people comprise one quarter of the UK’s population. Neurological trauma (e.g., traumatic brain injury and spinal cord injury) is one of the most common causes of death and disability in this population group, yet health technology innovations for these patients lag behind those for adults.

Using the horizon scanning methodologies developed by the Innovation Observatory, systematic searches were performed to identify registered clinical trials, published funding awards, and news articles that focused on innovative devices and digital and diagnostic health technologies developed for use in children and young people (from 28 days after birth up to and including 18 years of age) with actual or suspected neurological trauma. The search results were screened for relevance, and key information on the included technologies was extracted and summarized.

Twenty-nine technologies were identified, of which 10 were commercially available. The majority were developed in the UK or the USA. Overall, the development pipeline was evenly split amongst technologies considered to be a device (37%), digital (34%), or diagnostic (29%). Most technologies were intended for use across settings by healthcare professionals, either for initial onsite assessment, for in-hospital management, or for rehabilitation in hospital or in the community.

Results from this horizon scan show that development of technologies for pediatric neurological trauma is currently limited, with only a small number of the technologies being developed covering an area of unmet need. To complement the horizon scan, we also sought stakeholder insights on medical technologies for this population group. The combined results and final conclusions will be shared in a future publication.

Advanced Therapy Medicinal Products (ATMPs) are innovative biologics (gene, cells and tissue-based products) with the potential to treat diseases with significant unmet clinical need. ATMPs pose distinct regulatory, health technology assessment (HTA) and patient access challenges, hence early identification and prioritization of ATMPs is now recognized as a key concern in England. The National Institute for Health Research Innovation Observatory (NIHRIO) uses a robust methodology to identify and monitor health technologies, including ATMPs that meet the remit of key HTA stakeholders in England. This analysis provides a global overview of the current ATMPs pipeline to administer useful insights for policymakers, funders and innovators.

NIHRIO's database tracks pharmaceuticals from phase I/II onwards, but this analysis focuses on late-stage development. The database (N > 12,000 records) was filtered to identify potential ATMPs using a predefined criteria based on the European Medicine's Agency's classification. Each record is categorized by stage: ‘Active’, (with an estimated three years to European licence); ‘Monitoring’ (in development with no licence date); and ‘Finished’, (output produced/discontinued and no longer tracked). Subsequently, records in ‘Active’ and ‘Monitoring’ were examined further.

Analysis identified 636 ATMPs: five percent ‘Active’, 40 percent ‘Monitoring’ and 55 percent ‘Finished’. ATMPs in the Active/Monitoring stages included: gene therapies (52%), somatic cells (43%) and tissue-engineered products (5%). Of these, 40 percent were oncological with the majority targeting hematological cancers (lymphomas). Prevalent non-oncology areas included musculoskeletal (10%) and ophthalmology (8%). Over one-third of trials were phase IIs, with almost half of all trials were based in the US.

The overarching findings here indicate increasing development of the ATMP pipeline towards indications with significant unmet clinical need. In oncology, the high prevalence of hematological ATMPs is largely due to recent chimeric antigen receptor T cells (CAR-T) innovation. In non-oncology areas, ATMP development is increasing due to advances in regenerative medicine. With a significant number of ATMPs projected to be licenced within three years, and many more in active late-stage trials, HTA bodies and health systems are challenged to prepare for the entry of these innovative therapies.