TY  - GEN
A1  - Volodina, Anna
TI  - Multi-country comparative analysis of paediatric regulatory
frameworks and their impact on access to medicines in times of
pandemic and beyond.
CY  - Heidelberg
UR  - https://archiv.ub.uni-heidelberg.de/volltextserver/36663/
KW  - regulatory affairs
AV  - public
N2  - In the field of public health, the importance of access to essential medicines has been a focus
of attention. For children, improving access is even more urgent as the lack of medicines with
paediatric labelling and age-appropriate formulations is a global health problem. The European
Union and the United States have adopted paediatric regulatory framework more than 15 years
ago, that requires and rewards paediatric pharmaceutical research (hereafter referred to as
paediatric legislation). In this way, both regions have been able to stimulate the research and
marketing authorisation of medicines for children and thus to improve access to age-appropriate
treatments.
Paediatric legislation becomes an indispensable commodity in times of health crisis. It ensures
that the medical needs of this vulnerable population are addressed without undue delay. For
example, vaccines against Coronavirus disease COVID-19 have been rapidly developed for
paediatric use. From 2023, the European pharmaceutical legislation is under review with the
aim of further strengthening paediatric research. However, the vast majority of children live
outside of Europe and the United States where the role of regulatory frameworks in access to
paediatric medicines is not well understood.
This research aimed to examine regulatory frameworks in six countries with different health
and economic status and to develop regulatory recommendations for better access globally.
Countries selected for analysis were Australia, Brazil, Canada, Kenya, Russia, and South
Africa. The research employed a mixed-method design with qualitative and quantitative
methods and included three studies each pertaining to a research objective. The analytical
framework from the National Collaborating Centre for Healthy Public Policy in Canada was
used to synthesise and interpret the results obtained.
Results: Authorisation availability of paediatric medicines and formulations in the studied
countries was lower compared to Europe and the United States. Generic medicines generally
did not contain age-appropriate formulations even when they were available from the
originator. Regulatory barriers to access were identified in all countries. These included a lack
of harmonisation of paediatric research requirements, poor availability of medicines with
paediatric use information and formulations. Brazil, Kenya, Russia, and South Africa
additionally described overarching health system barriers, such as poor financing and supply.
Children in these countries continue to suffer from the diseases well-saturated with novel
treatments, which indicates barriers to access at the health system level. The COVID-19 pandemic temporarily reduced regulatory barriers. In middle-income countries, however, the
impact on access to medicines has been limited due to weak health systems. Medicines
developed under paediatric legislation ranged from common childhood vaccines to highly
specialised treatments, some of which were later included in the World Health Organisation´s
lists of Essential Medicines. However, the spectrum of addressed diseases was not fully in line
with the needs, as evidenced by the burden of disease. Among others, medicines for neonatal
and poverty-related diseases were substantially underrepresented. Acceptance of paediatric
legislation was hampered by concerns about its equity and feasibility.
Conclusions: Regulatory frameworks in the studied countries remain unable to support access
to paediatric medicines and formulations in a systematic manner. National marketing
authorisation is essential for equitable access but in case of paediatric medicines it cannot be
achieved without binding regulatory measures. Overall, the research highlights the need for a
globally harmonised paediatric regulatory framework that would contain needs-based rewards
and obligations for pharmaceutical companies. While regional legislative efforts are underway,
they should become global. The impact of regulatory measures may be limited if not combined
with a robust system to deliver medicines to patients and industry activity in a country.
Therefore, efforts to strengthen the health system and support for the domestic manufacturing
sector remain necessary elements of access to medicines. Finally, continued public health
efforts to reach all Sustainable Development Goals are required to effectively reduce the disease
burden in children, particularly in the low-and middle-income countries.
Y1  - 2025///
ID  - heidok36663
ER  -