1932

Abstract

With the passage of the Biologics Price Competition and Innovation Act of 2009, the US Food and Drug Administration established an abbreviated pathway for developing and licensing biosimilar and interchangeable biological products. The regulatory framework and the technical requirements of the US biosimilars program involve a stepwise approach that relies heavily on analytical methods to demonstrate through a “totality of the evidence” that a proposed product is biosimilar to its reference product. By integrating analytical, pharmacological, and clinical data, each of which has limitations, a high level of confidence can be reached regarding clinical performance. Although questions and concerns about the biosimilars pathway remain and may slow uptake, a robust scientific program has been put in place. With three biosimilars already licensed and numerous development programs under way, clinicians can expect to see many new biosimilars come onto the US market in the coming decade. [Note added in proof: Since the writing of this article, a fourth biosimilar has been approved.]

Loading

Article metrics loading...

/content/journals/10.1146/annurev-med-051215-031022
2017-01-14
2024-06-17
Loading full text...

Full text loading...

/deliver/fulltext/med/68/1/annurev-med-051215-031022.html?itemId=/content/journals/10.1146/annurev-med-051215-031022&mimeType=html&fmt=ahah

Literature Cited

  1. 1. US Congress. 1944. Public Health Service Act, Public Law. 788–410
  2. 2. US Congress. 1938. Federal Food, Drug, and Cosmetic Act, Public Law. 75–717
  3. 3. US Congress. 1984. Drug Price Competition and Patent Term Restoration Act, Public Law. 98–417 https://www.gpo.gov/fdsys/pkg/STATUTE-98/pdf/STATUTE-98-Pg1585.pdf
  4. 4. European Union. 2004. The biosimilar pathway: Article. 104 of Directive 2001/83/EC with Amendment 2004/27/EC. http://ec.europa.eu/health/files/eudralex/vol-1/dir_2001_83_cons/dir2001_83_cons_20081230_en.pdf
  5. 5. European Medicines Agency. European public assessment reports http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/epar_search.jsp&mid=WC0b01ac058001d124 [Google Scholar]
  6. 6. US Congress. 2010. Biologics Price Competition and Innovation Act in the Patient Protection and Affordable Care Act, Public Law. 111–148 http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/UCM216146.pdf
  7. 7. USFDA. 2015. Guidance for industry: scientific considerations in demonstrating biosimilarity to a reference product http://www.fda.gov/downloads/DrugsGuidanceComplianceRegulatoryInformation/Guidances/UCM291128.pdf [Google Scholar]
  8. 8. USFDA. 2015. Guidance for industry: quality considerations in demonstrating biosimilarity of a therapeutic protein product to a reference product http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm291134.pdf [Google Scholar]
  9. Allen D, Baffi R, Bausch J. 9.  et al. 1996. Validation of peptide mapping for protein identity and genetic stability. Biologicals 24:255–74 [Google Scholar]
  10. Garnick RL. 10.  1992. Peptide mapping for detecting variants in protein products. Dev. Biol. Stand. 76:117–30 [Google Scholar]
  11. Cho A, Normile D. 11.  2002. Nobel Prize in Chemistry. Mastering macromolecules. Science 298:527–28 [Google Scholar]
  12. Ghasriani H, Hodgson DJ, Brinson RG. 12.  et al. 2016. Precision and robustness of 2D-NMR for structure assessment of filgrastim biosimilars. Nat. Biotechnol. 34:139–41 [Google Scholar]
  13. Wei Z, Shacter E, Schenerman M. 13.  et al. 2011. The role of higher-order structure in defining biopharmaceutical quality. BioProcess Int 9:58–66 [Google Scholar]
  14. Ma M, Tudan C, Koltchev D. 14.  2015. The 10th Annual Bioassays and Bioanalytical Method Development Conference. Bioanalysis 7:569–72 [Google Scholar]
  15. Rieder N, Gazzano-Santoro H, Schenerman MA. 15.  et al. 2010. The roles of bioactivity assays in lot release and stability testing. BioProcess Int 8:33–41 [Google Scholar]
  16. Schenerman MA, Axley MJ, Oliver CN. 16.  et al. 2009. Using a risk assessment process to determine criticality of product quality attributes. Quality by Design for Biopharmaceuticals: Principles and Case Studies AS Rathore, R Mhatre 53–84 Hoboken, NJ: Wiley [Google Scholar]
  17. Rathore AS, Winkle H. 17.  2009. Quality by design for biopharmaceuticals. Nat. Biotechnol. 27:26–34 [Google Scholar]
  18. Kozlowski S, Swann P. 18.  2009. Considerations for biotechnology product quality by design. Quality by Design for Biopharmaceuticals: Principles and Case Studies AS Rathore, R Mhatre 9–30 Hoboken, NJ: Wiley [Google Scholar]
  19. Cavagnaro JA. 19.  2002. Preclinical safety evaluation of biotechnology-derived pharmaceuticals. Nat. Rev. Drug Discov. 1:469–75 [Google Scholar]
  20. 20. USFDA. 2014. Guidance for industry: immunogenicity assessment for therapeutic protein products http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm338856.pdf [Google Scholar]
  21. Shankar G, Arkin S, Cocea L. 21.  et al. 2014. Assessment and reporting of the clinical immunogenicity of therapeutic proteins and peptides—harmonized terminology and tactical recommendations. AAPS J 16:658–73 [Google Scholar]
  22. Kirshner SL. 22.  2014. Mechanisms underlying the immunogenicity of therapeutic proteins: risk assessment and management strategies. J. Interferon Cytokine Res. 34:923–30 [Google Scholar]
  23. Rosenberg AS. 23.  2006. Effects of protein aggregates: an immunologic perspective. AAPS J 8:E501–7 [Google Scholar]
  24. Jiang XR, Song A, Bergelson S. 24.  et al. 2011. Advances in the assessment and control of the effector functions of therapeutic antibodies. Nat. Rev. Drug Discov. 10:101–11 [Google Scholar]
  25. Jefferis R. 25.  2012. Isotype and glycoform selection for antibody therapeutics. Arch. Biochem. Biophys. 526:159–66 [Google Scholar]
  26. Jefferis R. 26.  2009. Recombinant antibody therapeutics: the impact of glycosylation on mechanisms of action. Trends Pharmacol. Sci. 30:356–62 [Google Scholar]
  27. Shields RL, Lai J, Keck R. 27.  et al. 2002. Lack of fucose on human IgG1 N-linked oligosaccharide improves binding to human Fcγ RIII and antibody-dependent cellular toxicity. J. Biol. Chem. 277:26733–40 [Google Scholar]
  28. 28. International Conference for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH). 2005. Guidance for industry: Q5E comparability of biotechnological/biological products subject to changes in their manufacturing process http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm073476.pdf [Google Scholar]
  29. 29. USFDA. 1996. FDA guidance concerning demonstration of comparability of human biological products, including therapeutic biotechnology-derived products http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm122879.htm [Google Scholar]
  30. Schneider CK. 30.  2013. Biosimilars in rheumatology: the wind of change. Ann. Rheum. Dis. 72:315–18 [Google Scholar]
  31. Chirino AJ, Mire-Sluis A. 31.  2004. Characterizing biological products and assessing comparability following manufacturing changes. Nat. Biotechnol. 22:1383–91 [Google Scholar]
  32. Lee JF, Litten JB, Grampp G. 32.  2012. Comparability and biosimilarity: considerations for the healthcare provider. Curr. Med. Res. Opin 28:1053–58 [Google Scholar]
  33. McCamish M, Woollett G. 33.  2013. The continuum of comparability extends to biosimilarity: How much is enough and what clinical data are necessary?. Clin. Pharmacol. Ther. 93:315–17 [Google Scholar]
  34. Woodcock J, Griffin J, Behrman R. 34.  et al. 2007. The FDA's assessment of follow-on protein products: a historical perspective. Nat. Rev. Drug Discov. 6:437–42 [Google Scholar]
  35. 35. USFDA. 2014. Draft guidance for industry: clinical pharmacology data to support a demonstration of biosimilarity to a reference product http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm397017.pdf [Google Scholar]
  36. 36. USFDA. Purple book: lists of licensed biological products with reference product exclusivity and biosimilarity or interchangeability evaluations http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/TherapeuticBiologicApplications/Biosimilars/ucm411418.htm [Google Scholar]
  37. 37. USFDA. 2015. Draft guidance for industry: nonproprietary naming of biological products http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm459987.pdf [Google Scholar]
  38. 38. USFDA. 2016. Guidance for industry: labeling for biosimilar products http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm493439.pdf [Google Scholar]
  39. 39. USFDA. 2016. Draft guidance for industry: labeling for biosimilar products; notice of availability draft guidance for industry; availability https://www.federalregister.gov/articles/2016/04/04/2016-07611/labeling-for-biosimilar-products-draft-guidance-for-industry-availability [Google Scholar]
  40. Danese S, Fiorino G, Michetti P. 40.  2016. Has IBD specialists' awareness of biosimilar monoclonal antibodies changed? Results from a survey amongst ECCO members European Crohn's and Colitis Organisation poster presentation P312: clinical: therapy and observation. https://www.ecco-ibd.eu/index.php/publications/congress-abstract-s/abstracts-2016/item/p312-has-ibd-specialistsx2019-awareness-of-biosimilar-monoclonal-antibodies-changed-results-from-a-survey-amongst-ecco-members.html [Google Scholar]
  41. Anour R. 41.  2014. Biosimilars versus “biobetters”—a regulator's perspective. Eur. Generics Biosimilars Initiat. J. 3:4166–67 [Google Scholar]
/content/journals/10.1146/annurev-med-051215-031022
Loading
/content/journals/10.1146/annurev-med-051215-031022
Loading

Data & Media loading...

  • Article Type: Review Article
This is a required field
Please enter a valid email address
Approval was a Success
Invalid data
An Error Occurred
Approval was partially successful, following selected items could not be processed due to error