The biosimilars journey: current status and ongoing challenges

Kos, Igor; Azevedo, Valderílio Feijó; Neto, Daniel Egg; Kowalski, Sérgio Cândido

doi:10.7573/dic.212543

Cited by 11 publications

(7 citation statements)

References 54 publications

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“…Therefore, it is imperative that these similarity studies are carried out in an appropriate population group. The most sensitive population group must be chosen in order to allow an accurate detection of clinically meaningful differences [ 26 ]. In the case of adalimumab, the best population were patients with either psoriasis or rheumatoid arthritis.…”

Section: Resultsmentioning

confidence: 99%

The Biosimilar Landscape: An Overview of Regulatory Approvals by the EMA and FDA

Gherghescu

Delgado‐Charro

2020

Pharmaceutics

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Biosimilar medicines expand the biotherapeutic market and improve patient access. This work looked into the landscape of the European and US biosimilar products, their regulatory authorization, market availability, and clinical evaluation undergone prior to the regulatory approval. European Medicines Agency (EMEA, currently EMA) and Food and Drug Administration (FDA) repositories were searched to identify all biosimilar medicines approved before December 2019. Adalimumab biosimilars, and particularly their clinical evaluations, were used as a case study. In the past 13 years, the EMA has received 65 marketing authorization applications for biosimilar medicines with 55 approved biosimilars available in the EU market. Since the first biosimilar approval in 2015, the FDA has granted 26 approvals for biosimilars with only 11 being currently on the US market. Five adalimumab biosimilars have been approved in the EU and commercialized as eight different medicines through duplicate marketing authorizations. Whilst three of these are FDA-approved, the first adalimumab biosimilar will not be marketed in the US until 2023 due to Humira’s exclusivity period. The EU biosimilar market has developed faster than its US counterpart, as the latter is probably challenged by a series of patents and exclusivity periods protecting the bio-originator medicines, an issue addressed by the US’s latest ‘Biosimilar Action Plan’.

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Section: Resultsmentioning

confidence: 99%

The Biosimilar Landscape: An Overview of Regulatory Approvals by the EMA and FDA

Gherghescu

Delgado‐Charro

2020

Pharmaceutics

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“…Moreover, despite this increasing trend of biotechnological drug use in rare diseases, information on the pharmacokinetics, pharmacodynamics, and pharmaceutical application of these drugs is still limited in children, especially in those with rare diseases, compared to small-molecule drugs [9]. Besides, the use of biotechnological drugs other than the indications for which they are licensed takes place through extrapolation [24]. Considering their use in rare diseases, it may be suggested that the existing risk management plans of these drugs may be more likely to accompany various uncertainties in rare disease-oriented practice and that additional challenges may arise, indicating the need for additional measures.…”

Section: Discussionmentioning

confidence: 99%

Utilization of biotechnological drugs in rare diseases requiring the use of off-label drugs in children in Turkey

Kırmızı¹,

Aydın²,

Akıcı³

et al. 2021

Turk J Med Sci

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Background/aim Pediatric patients, especially those with rare diseases, represent a population that has a high tendency towards off-label drug use (OLDU) and needs a more careful practice of pharmacotherapy than in adults. We aimed to investigate biotechnological drug use in children with rare diseases requiring OLDU. Materials and methods This retrospective study examined all single-diagnosed OLDU applications (n = 5792) for 4992 children (<18-year) in Turkey. Applications of rare diseases were selected, and their descriptive characteristics were examined, including demographic features of patients, biotechnological drug utilization status, and disease categories. The off-label statuses of the drugs at the end of 2020 were also examined. Results In total, 77.7% (n = 4501) of OLDU applications were made for rare diseases. Biotechnological drug use was higher in rare disease applications than in nonrare diseases (37.9% vs. 19.2%, respectively; p < 0.0001). Canakinumab was the top applied biotechnological drug (73.2%). Compared to that in small-molecule drugs, the mean age of patients was higher in biotechnological drug-containing applications (8.1 ± 5.3 vs. 9.7 ± 4.9, respectively; p < 0.0001). Biotechnological drug use was higher in nonneoplastic rare diseases (40.3%) than in neoplastic rare diseases (26.4%), (p < 0.0001). At the end of 2020, the approval status of the off-label indications covered in 2016 was significantly higher for rare (24.4%) vs. nonrare (5.2%, p < 0.0001) diseases and for biotechnological (32.3%) vs. small-molecule (13.9%, p < 0.0001) drugs. In total, 87.7% of the drugs would have to be still used in the off-label setting at the end of 2020. Conclusion It was seen that more than three-quarters of the pediatric OLDU applications are for rare diseases, and the need for biotechnological OLDU in this group is almost 2-fold of small-molecule drug use. While further projected findings imply a higher approval tendency for rare diseases and biotechnological drugs, there seems to be more room for improvement for pediatric drug use.

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“…Extrapolation of Indications: This topic had been an important regulatory advantage for biosimilars and had a direct impact on costs to the health systems. It involves considering the potential to extrapolate the efficacy and safety data from one already studied condition to the other indications of the reference product, for which the biosimilar was not directly tested [14].…”

Section: Other Regulatory and Clinical Questions On Biosimilars: Extr...mentioning

confidence: 99%

“…It is well known that even different batches of the same reference product can exhibit minimal differences through time. These minimal changes could have a direct impact on pharmacokinetics (PK) and pharmacodynamics (PD), as well on efficacy and safety, so the similarity exercise must include a batch-to-batch evaluation of biosimilars in comparison with the reference product [14].…”

Section: Introductionmentioning

confidence: 99%