Problems of creating an effective system of health technology assessment at the early stages of the drug life cycle
DOI:
https://doi.org/10.24959/sphhcj.24.317Keywords:
health technology assessment; horizon scanning; early dialogue; innovative health technologies; medicines; rare (orphan) diseasesAbstract
Aim. To study modern approaches to the formation and functioning of the Horizon Scanning (HS) system and early health technology assessment (HTА), in particular, for the treatment of rare diseases with significant unmet medical needs.
Materials and methods. The analysis, systematization and synthesis of data from scientific publications, analytical reports, guidelines, information from official websites of regulatory authorities, HTА and HS agencies were used.
Results. The analysis of the theoretical foundations and regulatory support for the processes of forming an effective HS system at the early stages of the life cycle has been performed. HS systems, the so-called “early awareness and alert systems”, are aimed at identifying, filtering and prioritizing new and innovative technologies with a significant predicted impact on health, costs, society and the healthcare system in order to inform policy makers, purchasers and healthcare providers or improve early access. The HS system allows for proactive planning and decision-making on the use and reimbursement of new medicines based on preliminary assessments of budgetary impact and clinical efficacy.
Conclusions. For the national HTA system, which currently is at the stage of development, it is important to adapt positive international experience in this area and provide functionality for HS, early assessment, early dialogue, managed access with the development of the evidence base. It is advisable to apply the “light” model and join the international cooperation in this area.
References
EFPIA (2023). The Pharmaceutical Industry in Figures. Key Data. 25 р. URL: https://www.efpia.eu/media/rm4kzdlx/the-pharmaceutical-industry-in-figures-2023.pdf
Dmytryk, K. (2021) V osoblyvomu stanovyshchi: za pidsumkamy 2024 r. 18% svitovoho rynku Rx-preparativ nalezhatyme orfannym? [In a special situation: according to the results of 2024, will 18% of the world market of Rx drugs belong to orphans?]. Apteka online, 26(1297). URL: https://www.apteka.ua/article/580249 [In Ukrainian].
Vogler, S. (2022). “Ready for the future?” – Status of national and cross-country horizon scanning systems for medicines in European countries. German Medical Science, 20. doi: 10.3205/000307.
Grössmann, N., Wolf, S., Rosian, K., Wild, C. (2019). Pre-reimbursement: early assessment for coverage decisions. Wiener Medizinische Wochenschrift, 169, 254–262. doi: 10.1007/s10354-019-0683-1.
Ivanovic, J., Capone, G., Raffaelli L., Pantò, V., Marangi, M. (2021). Horizon Scanning for pharmaceuticals and effective health care programming: 2 years’ experience at the Italian Medicines Agency. Drug Discovery Today, 26, 2, 569-576. doi: 10.1016/j.drudis.2020.11.015.
Marangi, M., Ivanovic, J., Pistritto, G. (2019). The Horizon Scanning System at the Italian Medicines Agency. Drug Discovery Today, 24(6), 1268-1280. doi: 10.1016/j.drudis.2019.04.010.
Vignali, V., Hines, P. A., Cruz, A. G., Ziętek, B., Herold R. (2022). Health horizons: Future trends and technologies from the European Medicines Agency’s horizon scanning collaborations. Frontiers in Medicine, 9, 1-20. doi: 10.3389/fmed.2022.1064003.
Lauvrak, V., Bidonde, J., Peacocke E. F. (2021). 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.
Simpson, S., Cook, A., Miles, K. (2018). Patient and public involvement in early awareness and alert activities: an example from the United Kingdom. International Journal of Technology Assessment in Health Car, 34(1), 10-17. doi: 10.1017/S0266462317004421.
Parsons, J. (2023). Horizon Scanning and Early Assessment. Health Technology Assessment Policy and Methods Review. ACT: Australian Department of Health and Aged Care. 111 р.
Vogler, S. (2022). Payer Policies to Support Innovation and Access to Medicines in the WHO European Region. Oslo Medicines Initiative Technical Report. Copenhagen: WHO. Available at: https://www.ncbi.nlm.nih.gov/books/NBK587873/
UK NIHR Innovation Observatory (2021). Horizon scanning of innovative medicines, devices, diagnostics and digital technologies for stakeholders in England. 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
UK PharmaScan. An essential first step to market access for medicines. Available at: https://www.ukpharmascan.org.uk/
International HealthTechScan. Available at: https://ihts.org/
Nazarkina V. M., Nemchenko A. S., Kosiachenko K. L., Babenko M. M. Metodolohiia tsinoutvorennia na likarski zasoby v systemi okhorony zdorovia [Methodology of pricing of medicinal products in the healthcare system]: monohrafiia; za nauk. red. A. S. Nemchenko. Kyiv, 2022. 288 p. [In Ukrainian]
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
Senior M., Hadjivasiliou A. (2022). Orphan Drug Report 2022. April 2022. EvaluatePharma. 17 р. Available at: https://info.evaluate.com/rs/607-YGS-364/images/Evaluate%20Orphan%20Drug%20Report.pdf
Senior M., Hadjivasiliou A. (2023). Orphan Drugs 2023-2028. A flattening curve? March 2023. EvaluatePharma. 15 р. Available at: https://info.evaluate.com/rs/607-YGS-364/images/Orphan%20Drug%20Report.pdf
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
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
Drug Approvals and Databases. Available at: https://www.fda.gov/drugs/development-approval-process-drugs/drug-approvals-and-databases
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