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Corrected 21 July 2022 to make a clarification about the products subject to the industry comments.
Stakeholders want the US Food and Drug Administration (FDA) to include biosimilars, as well as certain biologics regulated by the Center for Biologics Evaluation and Research (CBER), in the agency’s guidance on benefit-risk principles when evaluating product quality assessments.
In May, FDA published the Benefit-Risk Considerations for Product Quality Assessments draft guidance which applies to new drug applications (NDA), biologics license applications (BLAs) and supplements. It outlines possible mitigation strategies for product quality issues and the circumstances under which unresolved quality issues may be addressed during a public health emergency or a widespread drug shortage. (Related: FDA offers insight into product quality assessment principles, Regulatory Focus 11 May 2020)
Specifically, FDA says it will take a risk-based approach to product quality assessments to prevent potential shortages and consider mitigation strategies where there may be quality concerns. The risk of unresolved quality issues may be outweighed by the benefit of the product or the ability to bring it market quickly according to regulators, especially if the product addresses an unmet medical need.
In a comment on the guidance from Samsung Bioepis, the company asks FDA to consider including biosimilars as part of the benefit-risk considerations for product quality assessment.
“As outlined in the Biologics Price Competition and Innovation Act of 2009 (BPCIA) enacted as Title VII of the Patient Protection and Affordable Care Act, the new regulatory pathway for biosimilar was created with the goal of reducing the cost of health care and improving how patient receive care which includes greater access,” said Samsung. “Biosimilars can improve broader patient access to biologics through improved affordability (which may be cost savings to various stakeholders) and this in turn enables earlier treatment in some instances of chronic and debilitating diseases in a manner that has the potential to significantly improve overall treatment outcomes.”
Similarly, in its comments, the drug lobby group PhRMA asks FDA to apply the guidance to certain CBER-regulated biologics, such as human gene therapies.
“While PhRMA generally supports the considerations set forth in the draft guidance, we offer a number of recommendations to help enhance clarity and support manufacturers in developing and submitting CMC information to the Agency. Broadly, we recommend that FDA expand the scope of the draft guidance to include products regulated by CBER,” the group said in letter dated 11 July. “While PhRMA recognizes there may be unique considerations to product quality assessments for CBER-regulated products (e.g., gene therapies), the higher-level considerations set forth in this draft guidance would apply to these products.”
In addition, PhRMA asked that FDA explicitly state that in certain circumstances, “…mitigation strategies can obviate the need for a quality postmarketing agreement (QPA). For example, mitigation strategies such as the use of in-filters (for visible particulates) and short in-use periods may adequately mitigate any residual risk that may otherwise be addressed through a QPA.”
The group also asked FDA to provide additional details on QPAs, post-marketing requirements (PMRs), and post-marketing commitments (PMCs). It also wants the agency to align its guidance with risk management principles outlined in International Council for Harmonisation (ICH) Q9 guideline.
Finally, the group recommends that FDA include examples in the final guidance that address new modalities.
“In particular, we encourage FDA to include an example of its approach to benefit-risk considerations on stability performance for new modality products,” said PhRMA.