Frequently Asked Questions
Non-Clinical Drug Development & Regulatory Consulting
- What is the regulatory framework for developing an autologous gene therapy product?
- When is the right time to ask for regulatory/scientific advice?
- Which authority to select for a first scientific advice? FDA, EMA or European National Authorities first?
- A non-clinical program without animal studies – is this possible?
- Where to place pivotal GLP studies? What if there is no ideal CRO?
- What to do with unexpected toxicological findings?
- Can immunocompromised mice be used in GLP toxicology studies?
- How to address the genotoxic risk of gene-edited therapeutics?
Risk Assessments and ERAs
- How to qualify impurities? Or is it a metabolite?
- How to perform and interpret computational evaluation of (potential) genotoxic impurities according to ICH M7?
- What is the minimal need for a leachable & extractable assessment?
- How to evaluate PDE values with limited non-clinical information?
- Can the excipient be used in mice, rabbits and humans?
- How to prepare Environmental Risk Assessments for GMOs and other products?
- How to integrate a development plan into a proper project plan and deliver the agreed outcomes according to the scope, timescale and quality of the program?
- How to manage risks, dependencies and critical path activities?
- How to provide communications, reports and progress updates of the project?
Scientific / Regulatory Writing
- Does the research report comply with regulatory requirements?
- Drafting Briefing Dossiers for Scientific Advice – how to make regulators comfortable?
- How to structure a documentation system to facilitate CTD submission later on?
Non-Clinical Development of Medical Devices
- Can the product be defined as a medical device, biologic drug, or cosmetic? How does a non-clinical program differ among the aforementioned products?
- How could a reduced program for a medical device look like?
- Are there differences in biocompatibility evaluation between the EU and the US?
Safety Assessment of Cosmetics
- How to include new compounds into cosmetic formulations?
- What to do with in vivo toxicology studies previously conducted with a cosmetic product?