NCCN Biosimilars White Paper: Regulatory, Scientific, and Patient Safety Perspectives

Abstract

Biologics are essential to oncology care. As patents for older biologics begin to expire, the United States is developing an abbreviated regulatory process for the approval of similar biologics (biosimilars), which raises important considerations for the safe and appropriate incorporation of biosimilars into clinical practice for patients with cancer. The potential for biosimilars to reduce the cost of biologics, which are often high-cost components of oncology care, was the impetus behind the Biologics Price Competition and Innovation Act of 2009, a part of the 2010 Affordable Care Act. In March 2011, NCCN assembled a work group consisting of thought leaders from NCCN Member Institutions and other organizations, to provide guidance regarding the challenges health care providers and other key stakeholders face in incorporating biosimilars in health care practice. The work group identified challenges surrounding biosimilars, including health care provider knowledge, substitution practices, pharmacovigilance, naming and product tracking, coverage and reimbursement, use in off-label settings, and data requirements for approval.

Getting Familiar With Biosimilars

“…A white paper from 2011 introduced biosimilars from a regulatory, scientific, and patient safety perspective (Zelenetz et al, 2011). Based on a survey of 277 providers, the paper also pointed to a low level of understanding of biosimilars.…”

Regulatory considerations in oncologic biosimilar drug development

“…Awareness of these upcoming changes may be of substantial value to clinicians, pharmacists, and patients and their caregivers to ensure that lower-cost alternatives enable savings within healthcare systems. [1][2][3][4][5][6] In this review, we provide an overview of the regulatory standards for the development and approval of biosimilars in the European Union (EU) and the United States (US), outlining some of the current differences and their potential implications, and we consider some of the challenges that arise for emerging countries in adopting the biosimilar regulatory paradigm pioneered in the EU/US. Further, we review a few of the key issues for biosimilars that are currently being discussed on a global basis by regulators, biosimilars developers, and other stakeholders.…”

“…Biosimilars are currently being used in clinical practice for the supportive care of patients with cancer, such as hematopoietic growth factors (e.g., erythropoietin, filgrastim). 1 In the next few years, a number of biologics used for the treatment of patients with cancer will lose exclusivity, including the monoclonal antibodies (mAbs) trastuzumab, rituximab, cetuximab, and bevacizumab. This loss of exclusivity enables biosimilar mAbs to be approved by regulatory authorities, and thus enter clinical use.…”

“…1,2,33 Extrapolation is allowed by both the EMA and FDA for biosimilars, although this must be based on a strong scientific justification, which leverages the complete comparability data set (quality, non-clinical and clinical evidence, as well as what is known on the mechanism of action [MOA]). 34 Of note, the EMA has recently allowed extrapolation of indication for rheumatologic and gastrointestinal inflammatory diseases at approval of the 2 infliximab biosimilar mAbs, Inflectra TM (Hospira UK Ltd, Royal Lemington Spa, UK) and Remsima TM (Celltrion Healthcare Hungary Kft, Budapest, Hungary).…”

Comparison of originator and biosimilar therapeutic monoclonal antibodies using comprehensive two-dimensional liquid chromatography coupled with time-of-flight mass spectrometry

“…1 Biosimilar proteins such as haematopoietic growth factors (e.g., erythropoietin, filgrastim) and monoclonal antibodies (mAbs) (e.g., infliximab) 2 are currently being used in clinical practice for the treatment of patients with cancer and immune-mediated disorders. In the next few years, a number of biologics will lose exclusivity, including trastuzumab, rituximab, cetuximab, bevacizumab and adalimumab.…”