Conceptual Basis for the Development of Guidance for the Use of Biomarkers of Effect in Regulatory Risk Assessment of Chemicals

Overview

Journal EFSA J

Date 2024 Dec 18

PMID 39691503

Authors

Antonio Hernandez-Jerez

Susanne Hougaard Bennekou

Laurentius Ron Hoogenboom

Henry Mcardle

Christina Pieper

Tanja Schwerdtle

Hendrik Van Loveren

Zainab Al Harraq

Cristina Croera

Anna Christodoulidou

Agnes De Sesmaisons

Chantra Eskes

Sara Levorato

Silvia Valtuena Martinez

Georgia Bompola

Lucian Farcal

Affiliations

Soon will be listed here.

Abstract

This Scientific Report was carried out in the context of the self-task mandate (M-2023-00097) of the EFSA's Scientific Committee on 'Guidance on the use of biomarkers of effect in regulatory risk assessment of chemicals'. In the first phase, the project on biomarkers of effect started with a feasibility study (EFSA-Q-2024-00128), with the intention to look closer at definitions and descriptions of biomarkers of effect, as well as to explore several concepts related to the context of application and other scientific principles to be further considered for its development. In addition, relevant activities, initiatives and knowledge in this area were collected and analysed within a complementary mapping study. The outcome of this phase aimed to create a structured basis for future guidance, to identify challenges and to recommend a way forward for its development. The recommendations refer especially to terminologies, the scope of the guidance and several scientific and technical aspects of the selection and interpretation of biomarkers of effect that need to be addressed in future guidance. Moreover, further recommendation refers to the collaborative process to be established with other regulatory organisations that should support the harmonisation and reduce divergencies in the application of methodologies across organisations or sectors.

References

Lesko L, Atkinson Jr A . Use of biomarkers and surrogate endpoints in drug development and regulatory decision making: criteria, validation, strategies. Annu Rev Pharmacol Toxicol. 2001; 41:347-66. DOI: 10.1146/annurev.pharmtox.41.1.347. View

Turck D, Bohn T, Castenmiller J, de Henauw S, Hirsch-Ernst K, Knutsen H . Guidance for establishing and applying tolerable upper intake levels for vitamins and essential minerals: Draft for internal testing. EFSA J. 2022; 20(1):e200102. PMC: 8784980. DOI: 10.2903/j.efsa.2022.e200102. View

Bodaghi A, Fattahi N, Ramazani A . Biomarkers: Promising and valuable tools towards diagnosis, prognosis and treatment of Covid-19 and other diseases. Heliyon. 2023; 9(2):e13323. PMC: 9884646. DOI: 10.1016/j.heliyon.2023.e13323. View

Burke H . Predicting Clinical Outcomes Using Molecular Biomarkers. Biomark Cancer. 2016; 8:89-99. PMC: 4896533. DOI: 10.4137/BIC.S33380. View

Dent M, Vaillancourt E, Thomas R, Carmichael P, Ouedraogo G, Kojima H . Paving the way for application of next generation risk assessment to safety decision-making for cosmetic ingredients. Regul Toxicol Pharmacol. 2021; 125:105026. PMC: 8547713. DOI: 10.1016/j.yrtph.2021.105026. View

Neveu V, Moussy A, Rouaix H, Wedekind R, Pon A, Knox C . Exposome-Explorer: a manually-curated database on biomarkers of exposure to dietary and environmental factors. Nucleic Acids Res. 2016; 45(D1):D979-D984. PMC: 5210656. DOI: 10.1093/nar/gkw980. View

Carnesecchi E, Langezaal I, Browne P, Batista-Leite S, Campia I, Coecke S . OECD harmonised template 201: Structuring and reporting mechanistic information to foster the integration of new approach methodologies for hazard and risk assessment of chemicals. Regul Toxicol Pharmacol. 2023; 142:105426. DOI: 10.1016/j.yrtph.2023.105426. View

More S, Bampidis V, Benford D, Bennekou S, Bragard C, Halldorsson T . Guidance on harmonised methodologies for human health, animal health and ecological risk assessment of combined exposure to multiple chemicals. EFSA J. 2020; 17(3):e05634. PMC: 7009070. DOI: 10.2903/j.efsa.2019.5634. View

Aronson J . Biomarkers and surrogate endpoints. Br J Clin Pharmacol. 2005; 59(5):491-4. PMC: 1884846. DOI: 10.1111/j.1365-2125.2005.02435.x. View

10.

Baken K, Lambrechts N, Remy S, Mustieles V, Rodriguez-Carrillo A, Neophytou C . A strategy to validate a selection of human effect biomarkers using adverse outcome pathways: Proof of concept for phthalates and reproductive effects. Environ Res. 2019; 175:235-256. DOI: 10.1016/j.envres.2019.05.013. View

11.

. Biomarkers and surrogate endpoints: preferred definitions and conceptual framework. Clin Pharmacol Ther. 2001; 69(3):89-95. DOI: 10.1067/mcp.2001.113989. View

12.

More S, Bampidis V, Benford D, Bragard C, Halldorsson T, Bennekou S . Statement on the derivation of Health-Based Guidance Values (HBGVs) for regulated products that are also nutrients. EFSA J. 2021; 19(3):e06479. PMC: 7970819. DOI: 10.2903/j.efsa.2021.6479. View

13.

Krewski D, Westphal M, Al-Zoughool M, Croteau M, Andersen M . New directions in toxicity testing. Annu Rev Public Health. 2011; 32:161-78. DOI: 10.1146/annurev-publhealth-031210-101153. View

14.

Kraus V, Burnett B, Coindreau J, Cottrell S, Eyre D, Gendreau M . Application of biomarkers in the development of drugs intended for the treatment of osteoarthritis. Osteoarthritis Cartilage. 2011; 19(5):515-42. PMC: 3568396. DOI: 10.1016/j.joca.2010.08.019. View

15.

Zare Jeddi M, Hopf N, Viegas S, Price A, Paini A, van Thriel C . Towards a systematic use of effect biomarkers in population and occupational biomonitoring. Environ Int. 2021; 146:106257. DOI: 10.1016/j.envint.2020.106257. View

16.

Knapen D, Angrish M, Fortin M, Katsiadaki I, Leonard M, Margiotta-Casaluci L . Adverse outcome pathway networks I: Development and applications. Environ Toxicol Chem. 2018; 37(6):1723-1733. PMC: 6004608. DOI: 10.1002/etc.4125. View

17.

. Biological markers in environmental health research. Committee on Biological Markers of the National Research Council. Environ Health Perspect. 1987; 74:3-9. PMC: 1474499. DOI: 10.1289/ehp.74-1474499. View

18.

Lee J, Won E, Raisuddin S, Lee J . Significance of adverse outcome pathways in biomarker-based environmental risk assessment in aquatic organisms. J Environ Sci (China). 2015; 35:115-127. DOI: 10.1016/j.jes.2015.05.002. View

19.

Aronson J, Ferner R . Biomarkers-A General Review. Curr Protoc Pharmacol. 2017; 76:9.23.1-9.23.17. DOI: 10.1002/cpph.19. View

20.

Verma M, Patel P, Verma M . Biomarkers in prostate cancer epidemiology. Cancers (Basel). 2013; 3(4):3773-98. PMC: 3763396. DOI: 10.3390/cancers3043773. View