Biologics: Innovation in Advanced Therapy Medicinal Products
Navigating the Nuances of Biologics Regulations
The term “biologics” corresponds to a broad range of medicinal products, covering recombinant proteins, Gene Therapy Medicinal Products (GTMPs), Somatic Cell Therapy Medicinal Products (sCTMPs), and Tissue-Engineered Products (TEPs).
Regulatory requirements for development, manufacturing, and marketing authorization, as well as subsequent monitoring of biologics, must be integrated within the biological and medical context. Linking scientific and regulatory knowledge at a very early stage in biologics development is critical.
For over ten years, we've been supporting companies in development and regulatory for new biological entities, biosimilars, and Advanced Therapy Medicinal Products (ATMPs). We help clients plan and execute tailored approaches, taking into account specific developmental challenges as well as financial and time constraints.
Coupled with these constraints, certain decisions about which molecules to progress during development, complex and costly R&D activities, and resourcing challenges can all impact a biologics company’s success. Strong, independent, and technical advice from the onset can significantly improve the chances of success.
Therapeutic Proteins (New Biological Entities)
Strategy
- Identify critical developmental steps and align with regulatory risk factors
- Define Target Product Profiles (TPPs)
- Locate medical experts and set up of medical advisory boards
Development
- Review and assess non-clinical, clinical, and quality data packages and perform gap analysis
- Provide study synopses and development roadmaps based on scientific and regulatory gap analysis
- Prepare development plans
- Perform vendor qualification audits for non-clinical, clinical, and manufacturing (GLP, GCP, and GMP) processes
Regulatory
- Analyze risks and harmonize global regulatory requirements with development milestones
- Plan, execute, and debrief scientific advice procedures with global regulatory agencies
- Synchronize biologic development plans with medical device or in vitro diagnostic development
Biosimilars
Strategy
- Residual risk identification and management through targeted biosimilar-specific scientific and regulatory knowledge
- Alignment of global biosimilar development milestones via our regulanet® network of enterprises
- Referencing of medicinal product sourcing strategies
Development
- Preparation of development plans
- Definition of TPP
- Design of comparability exercises
- Non-clinical and early clinical study design
- Support in analytical development to proof biosimilarity (e.g. in vitro potency)
- Vendor qualification audits for non-clinical, clinical, and manufacturing (GLP, GCP, and GMP) processes
- Review and interpretation of analytical data for reference products and biosimilar characterizations
- Comparative immunogenicity assessments
Regulatory
- Risk analysis and synthesis of global regulatory requirements with development milestones
- Planning, execution, and debriefing of scientific advice procedures with global regulatory agencies
Advanced Therapy Medicinal Products (ATMPs)
- Gene Therapy Medicinal Products (GTMPs)
- Somatic Cell Therapy Medicinal Products (sCTMPs)
- Tissue-Engineered Products (TEPs)
Strategy
- Critical developmental steps and alignment with regulatory risk factors
- Definition of TPP
- Location of medical experts and set up of medical advisory boards
Development
- Development plans
- Gap analysis at quality, pre-clinical, and clinical stages
- Manufacturing and controls milestones, characterization studies, and analytical development
- Quality control and comparability concepts
- Non-clinical and clinical study designs
Regulatory
- Support and author communication with health authorities for product categorization and scientific advice, including responses to agency questions
- Regulatory and scientific gap analysis for global development projects (the US and Europe)
- Clinical trial applications
Have a question concerning Biologics? Contact Dr. Katrin Wiemann
