Biosimilars, Regulatory Science, Cell and Gene Therapy Guidance and China’s Regulatory Landscape

Feature ArticlesFeature Articles | 01 May 2019 | Citation

April features took an in-depth look at biosimilars from a global perspective, the need to update regulatory science to include the use of Minimal Residual Disease (MRD), recent changes to China’s healthcare regulatory authorities and healthcare policy administration and the importance for manufacturers and regulators to ensure manufacturing controls are in place to sustainably produce regenerative medicines.
Gowri Sukumar focuses on global regulations emerging for biosimilar approval, the many challenges impacting the development of biosimilars and how various agencies define biologics, the evolution of guidances and data requirements for market approval, interchangeability and international nonproprietary names. “Biosimilars: A Global Perspective” provides an overview of the biosimilars that, over time, have been approved by regulators in various countries and regions.
The legal framework for the development of generic versions of small, low molecular weight medicines has evolved over several decades. “Similarity Approach for Complex Drugs: Biologics and Non-Biological Complex Drugs” examines regulatory issues related to approvals of biosimilars and Non-Biological Complex Drugs (NBCDs) by both the European Medicines Agency and the US Food and Drug Administration with a focus on issues regarding similarity and bioequivalence. Jon de Vlieger, Kevin Klein, Vinod Shah and Daan Crommelin encourage regulatory professionals to be aware of the new challenges for regulatory systems regarding these products as well as the clinical practice implications for substitution and interchangeability.
How do biosimilars differ from generic drugs? Cecil Nick answers this question and more in “Optimizing Clinical Trial Data to Support Biosimilars for Market Approval.” In covering efforts to gain market approval for biosimilars, the author reviews uncertainties associated with biosimilars, the value of pharmacokinetic data as well as therapeutic equivalence trials and their design. Nick suggests the time is right for regulators and industry to engage in a critical evaluation of the clinical development of biosimilars with a focus on issues where residual uncertainty exists.
FDA and EMA Guidances
Telly Chi and Aditi Sinha discuss the need to update regulatory science to include the use of Minimal Residual Disease (MRD) as a specific measure of tumor burden for multiple myeloma at levels undetectable through conventional laboratory techniques. MRD can potentially be used as a clinical and regulatory endpoint to evaluate a drug’s effect both on patients’ risk of future relapse risk and subsequent treatment survival outcomes, thus serving as a surrogate endpoint for early drug approvals for patients with multiple myeloma. In “Minimal Residual Disease as a Surrogate Endpoint for Product Development for Multiple Myeloma: Comparing FDA and EMA Guidances,” the authors review and compare FDA and EMA guidances and advocate for flexible regulatory approaches to data requirements for the use of MRD.
The term “regenerative medicine” is used to describe treatments that assist, stimulate or replace the body’s own reparative properties using cell therapy, gene therapy or tissue engineering approaches. To successfully and sustainably produce a regenerative medicine product, Mo Heidaran emphasizes how important it is for manufacturers and regulators to ensure manufacturing controls are in place. By having in place sound drug development practices, as well as manufacturing facility plans that cover the product’s entire lifecycle, manufacturers of regenerative medicine products are more likely to see commercial success. “Establishing Manufacturing Controls: A Hurdle for the Cell and Gene Therapy Industry” also identifies specific guidance and support materials that can be used to help direct product development and support sustainable manufacturing processes.
China’s Regulatory Landscape
With the Chinese regulatory landscape constantly changing, it has become necessary to reevaluate the roles and restructure how the healthcare regulatory authorities and healthcare policy administrations are organized. In “Chinese Health Policy and Regulatory Authorities Overview,” Yingying Liu describes the recent changes to China’s healthcare regulatory authorities and healthcare policy administration. Liu outlines how the reorganization and restructuring of the former Chinese Food and Drug Administration (CFDA) and several other organizations in March 2018 affects the responsibilities and functions of the many government departments, agencies and regulatory bodies responsible for overseeing drugs, food, medical devices, testing and evaluation.
What’s Coming in May

May feature articles will examine global regulatory compliance and enforcement, including
FDA’s review framework for medical devices, global cybersecurity compliance and a process for communicating with the Center of Drug Evaluation (CDE) during drug development. Other articles will focus on RMAT designations and accelerated pathways in Japan. Looks for these topics and more on biosimilars in May.

Q2 Regulatory Focus Article Series

Don’t miss the upcoming release of the second electronic Regulatory Focus article series in 2019  featuring global regulatory strategies and best practices on 13 May with an “Ask Me Anything” discussion on 12 June.

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