Biosimilars Data Package
Although a reduction in the data package for a biosimilar product may be possible, it is still quite substantial when compared to small molecule chemical generic products. The “Quality” dossier required is extensive; first, a full-quality dossier that meets all regulatory standards similar to that of a novel biological product has to be provided; in addition, a comprehensive comparability exercise to the reference product is required for both the drug substance and drug product. The amount of non-clinical and clinical data required depends on how well the molecule is characterized, potential differences between the biosimilar and the reference product and clinical experience with the substance class.
The type and extent of clinical data requirements for biosimilars vary based on three factors;
- complexity of the active substance and how well it can be characterized
- availability of an accepted surrogate end point to compare efficacy
- type and seriousness of safety concerns that have been seen with the reference product or the substance class.
The clinical data required may include:
- Robust and extensive comparative Phase I pharmacokinetic/pharmacodynamic (PK/PD)
- Phase III efficacy and safety studies with immunogenicity assessed throughout the clinical program. Immunogenicity, in particular, is critical to the development of biosimilar medicines
- Post-Authorization Safety Study (PASS)
The originator manufacturer will have undertaken a Phase III study for each applied therapeutic indication; however for a biosimilar, there is the possibility to extrapolate therapeutic similarity shown in one indication to other approved indications of the reference product.
In summary, the development of biosimilar medicines is guided by stringent regulatory requirements ensuring the highest standard of quality, non-clinical and clinical comparability data. The type and extent of this data depends on the complexity of the molecule and, therefore, is decided on a case-by-case basis.