Addressing the health technology assessment of biosimilar pharmaceuticals

Abstract

The growing number of biosimilars presents challenges to regulatory and health technology assessment (HTA) systems. This paper illustrates these challenges by focusing on biosimilars used in the oncological setting. In particular, discordances between data required by regulatory and HTA authorities potentially deprive patients of effective treatments and hinder optimal resource allocation. Regulatory and HTA authorities need to harmonize requirements to foster the development and widespread use of biosimilars, which potentially release considerable resources. The authors believe that often-inappropriate methodology creates a very real chance that HTA authorities will reject some biosimilars. This would effectively extend patent protection and, in the absence of competitor pressure from biosimilars, result in prices remaining unnecessarily high. The authors propose that HTA organizations should accept pharmacokinetic and pharmacodynamic equivalence between the brand and the biosimilar as a proxy of biological comparability. HTA organizations should then adopt, in the absence of compelling reasons otherwise, cost-minimization analysis (CMA) as the basis of the cost-effectiveness deliberations. In the absence of adequate studies demonstrating equivalent efficacy, a prerequisite of CMA, HTA organizations should require threshold analysis. Once approved, biosimilar manufacturers and regulators should maintain rigorous pharmacovigilance to exclude immunoreactivity or other rare adverse events. Furthermore, cancer centres and trusts should regularly audit and publish the impact of biosimilars on clinical outcomes and resource use. When appropriate, regulatory and HTA authorities should demand revised cost-effectiveness analyses from biosimilar manufacturers. This approach would hone the accuracy of the cost-effectiveness analyses, protect patients and allow health services rapid access to low cost treatments.

Key words::

• Biosimilars
• Cancer treatment
• Cost and cost analysis
• Drugs generic
• Technology assessment biomedical
• Therapeutic equivalency

Transparency

Declaration of funding

This review was funded by an unconditional grant from ratiopharm direct. The funder was invited to comment on drafts of the paper but all editorial decisions rested with the authors, who take full responsibility for the content.

Declaration of financial/other relationships

J.B. has disclosed that he has carried out paid consultancy for a wide range of pharmaceutical companies. He received payment from ratiopharm direct relating to this paper and also for contributions to UK health technology assessment submissions for their products. A.S. has disclosed that she has received payment from ratiopharm direct for contributing additional materials to this paper and commenting on drafts and also for participation in an advisory board meeting. P.A. has disclosed that he has received payment from ratiopharm direct for contributing additional materials to this paper and commenting on drafts.

Some peer reviewers receive honoraria from CMRO for their review work. The peer reviewers of this paper have disclosed that they have no relevant financial relationships.

Acknowledgement

Medical writer, Mark Greener, helped with revisions to the initial draft of the manuscript, for which he received payment from ratiopharm direct.

The paper was conceived and written entirely by the authors. J.B. conceived the project, carried out literature searches and prepared the initial draft of the paper. A.S. and P.A. commented on the initial draft and contributed additional materials.