Horizon Scanning and Early Assessment of Health Technologies for the Treatment of Orphan Diseases

Year : 2024 | Volume : | : | Page : –

Victoria Nazarkina

  1. Professor, Department of Organization and Economics of Pharmacy, National University of Pharmacy Kharkiv Ukraine


The theoretical foundations and regulatory framework of the processes of the formation of an effective health technology assessment (HTA) system at the early stages of the life cycle of medicines are analyzed in the article. Particular attention is paid to horizon scanning (HS) and early assessment of expensive innovative drugs utilized for treating rare diseases. In order to inform policymakers, purchasers, and providers (to prioritize MT research, financial, and operational planning) or facilitate early access (by facilitating controlled dissemination of MTs), Horizon Scanning Systems, also known as “early warning and information systems,” seek to identify, filter, and prioritize new and innovative medical technologies (MTs) with significant foreseeable impacts on health, costs, society, and the healthcare system. Public and private organizations (governments, payers, healthcare systems, venture capitalists, and developers of MTs) around the world have long used formal and informal HS programs. The HS method enables proactive planning and decision-making on the use and payment of novel medications based on first evaluations of their clinical efficacy and financial impact. It is of utmost importance for rare diseases that have significant unfulfilled medical needs. Based on the review of scientific publications, analytical reports, and data from the official websites of regulatory authorities and HTA agencies, it was found that an effective system of HS, early assessments, dialogue, and managed access allows for early identification of innovative MTs that will potentially have a significant impact on healthcare. It allows us to prepare in advance for the implementation of such MTs and to rationally use limited resources and distribute risks.

Keywords: health technology assessment, horizon scanning, early dialogue, innovative medical technologies, medicines, rare (orphan) diseases

How to cite this article: Victoria Nazarkina. Horizon Scanning and Early Assessment of Health Technologies for the Treatment of Orphan Diseases. International Journal of Brain Sciences. 2024; ():-.
How to cite this URL: Victoria Nazarkina. Horizon Scanning and Early Assessment of Health Technologies for the Treatment of Orphan Diseases. International Journal of Brain Sciences. 2024; ():-. Available from: https://journals.stmjournals.com/ijbs/article=2024/view=144626


1. de la Torre BG, Albericio F. The Pharmaceutical Industry in 2023: An Analysis of FDA Drug Approvals from the Perspective of Molecules. Molecules. 2024 Jan 25;29(3):585.
2. Resolution adopted by the General Assembly on 16 December 2021 [on the report of the Third Committee (A/76/454, para. 51)] Addressing the challenges of persons living with a rare disease and their families. Available at: https://digitallibrary.un.org/record/3953765?ln+=+en&ln=ru&v=pdf. Accessed March 15, 2024.
3. The 17 Goals. UN Department of Economic and Social Affairs. Sustainable Development. Available at: https://sdgs.un.org/#goal_section. Accessed March 15, 2024.
4. European Commission. An official website of the European Union Public Health. Rare diseases https://health.ec.europa.eu/european-reference-networks/rare-diseases_en#orphan-medicinal-products. Accessed March 15, 2024.
5. Horgan D, Moss B, Boccia S, et al. Time for Change? The Why, What and How of Promoting Innovation to Tackle Rare Diseases – Is It Time to Update the EU’s Orphan Regulation? And if so, What Should be Changed? Biomed Hub. 2020 Jul 17;5(2):1-11.
6. Health in the European Union – facts and figures. Eurostat Statistics Explained. Available at: https://ec.europa.eu/eurostat/statistics-explained/index.php?title=Health_in_the_European_Union_%E2%80%93_facts_and_figures. Accessed March 15, 2024.
7. EFPIA-EURORDIS joint statement on patient access to medicines for rare diseases. EFPIA, EURORDIS, 2022. Available at: https://www.efpia.eu/news-events/the-efpia-view/statements-press-releases/efpia-eurordis-joint-statement-on-patient-access-to-medicines-for-rare-diseases. Accessed March 15, 2024.
8. Senior M., Hadjivasiliou A. Orphan Drug Report 2022. April 2022. EvaluatePharma, 2022. 17 р. Available at: https://info.evaluate.com/rs/607-YGS-364/images/Evaluate%20Orphan%20Drug%20Report.pdf. Accessed March 15, 2024.
9. Senior M., Hadjivasiliou A. Orphan Drugs 2023-2028. A flattening curve? March 2023. EvaluatePharma, 2023. 15 р. Available at: https://info.evaluate.com/rs/607-YGS-364/images/Orphan%20Drug%20Report.pdf. Accessed March 15, 2024.
10. Vogler S. “Ready for the future?” – Status of national and cross-country horizon scanning systems for medicines in European countries. Ger Med Sci. 2022 Mar 31;20:Doc05.
11. Vogler S. Payer Policies to Support Innovation and Access to Medicines in the WHO European Region. Oslo Medicines Initiative Technical Report. Copenhagen: WHO. 2022. Available at: https://www.ncbi.nlm.nih.gov/books/NBK587873. Accessed March 15, 2024.
12. Grössmann N, Wolf S, Rosian K, Wild C. Pre-reimbursement: early assessment for coverage decisions. Wien Med Wochenschr. 2019 Sep;169(11-12):254-262.
13. Ivanovic J, Capone G, Raffaelli L, et al. Horizon Scanning for pharmaceuticals and effective health care programming: 2 years’ experience at the Italian Medicines Agency. Drug Discov Today. 2021 Feb;26(2):569-576.
14. Marangi M, Ivanovic J, Pistritto G. The Horizon Scanning System at The Italian Medicines Agency. Drug Discov Today. 2019 Jun;24(6):1268-1280.
15. Vignali V, Hines PA, Cruz AG, et al. Health horizons: Future trends and technologies from the European Medicines Agency’s horizon scanning collaborations. Front Med (Lausanne). 2022 Dec 8;9:1064003.
16. Lauvrak V, Bidonde J, Peacocke EF. Topic identification, selection and prioritisation for health technology assessment (HTA) – A report to support capacity building for HTA in low- and middle-income countries. Norwegian Institute of Public Health; 2021. 63 р.
17. Simpson S, Cook A, Miles K. Patient and public involvement in early awareness and alert activities: an example from the United Kingdom. Int J Technol Assess Health Care. 2018 Jan;34(1):10-17.
18. Parsons J, Milverton J, Ellery B, et al. Canberra Horizon Scanning and Early Assessment. Health Technology Assessment Policy and Methods Review. ACT: Australian Department of Health and Aged Care; 2023. 111 р.
19. Horizon scanning of innovative medicines, devices, diagnostics and digital technologies for stakeholders in England. NIHR Innovation Observatory, UK, 2021. Available at: https://www.io.nihr.ac.uk/wp-content/uploads/2022/01/NIHR-HS-Workshop-HTAi2021-Horizon-scanning-of-innovative-medicines-devices-diagnostics-and-digital-technologies-for-stakeholders-in-England_Fina.pdf. Accessed March 15, 2024.
20. Designating an Orphan Product: Drugs and Biological Products. Available at: https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products. Accessed March 15, 2024.
21. Community Register of orphan medicinal products. European Commission. Available at: https://ec.europa.eu/health/documents/community-register/html/reg_od_act.htm?sort=a. Accessed March 15, 2024.

Ahead of Print Subscription Review Article
Received April 1, 2024
Accepted April 25, 2024
Published April 29, 2024