Regulatory Science as an Important Principle of Good Consultancy Practices (“GCP”)

by Michael Pfleiderer

Regulatory science is the foundation of regulatory decision-making and is used to assess the quality, safety, and efficacy of human medicinal products throughout their life-span. The domains, covered by regulatory science, are considered to include both basic and applied biomedical sciences (such as microbiology, genetics, pharmacology, and biostatistics), clinical trial methodology and epidemiology, and social sciences (such as decision sciences, risk assessment, and communication). Regulatory science aims to contribute to the development of new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of regulated products.

However, for many innovative areas such as Advanced Therapy Medicinal Products, novel concepts to respond to emergency or outbreak scenarios or adaptive pathways to licensure, significantly contributing to the abbreviation of development and review time lines, regulators worldwide have not yet developed adequate principles to further establish a global regulatory science agenda. The aim of such agenda is to transform individual efforts into a coordinated action plan to support global licensure goals. Ultimately, advances in regulatory science will contribute to improved and faster access to innovative medicinal products.

Development, of product or product class specific regulatory agendas, provides a catalyst and unique opportunity for regulators worldwide to develop and propose a global regulatory science agenda for innovative medicinal products. Regulatory oversight is critical to allow access to these products that are safe, effective, and of assured quality. Methods, used by regulators, need to constantly evolve so that scientific and technological advances are applied to address novel challenges, and to provide an increased understanding of benefits and risks of existing products. Regulatory science builds on high-quality basic research and encompasses at least two broad categories. First, there is laboratory-based regulatory science. Illustrative examples include development of correlates of efficacy; or correlates of safety; or of improved product characterization and potency assays. Included in such science would be tools to standardize assays used for regulatory purposes. Second, there is a science to develop regulatory processes. Illustrative examples include adaptive clinical trial designs; or tools to analyze the benefit-risk decision-making process of regulators; or novel pharmacovigilance methodologies. Included in such science would be initiatives to standardize regulatory processes (e.g., definitions of terms for adverse events [AEs] following use of individual products). The aim of a global regulatory science agenda is to transform current national or regional efforts, mainly by experienced regulatory agencies specialized on the regulation of novel products or product classes, into a coordinated action plan to support global development programs.

Biopharma Excellence has regular and detailed insight into the development of innovative products and development concepts and is regularly summarizing and publishing scientific and regulatory conclusions arising from own experience and observations. These conclusions are also shared with regulators facilitating mutual understanding between developers and regulators, ensuring that regulatory guidance will not hamper product development. As such, we are deeply involved in the further development of the regulatory science agenda facilitating improved and accelerated access to novel medicinal products. Our efforts are also helpful to identify challenges to implement a regulatory science agenda and develop strategies and actions to fill gaps identified. Ultimately, a global regulatory science agenda will enable regulators, academics, and other stakeholders to converge around transformative actions for innovation in the regulatory process to support global product development goals.

Attached you will find some recent publications with contributions made by Biopharma Excellence aimed at integrating the rapidly growing field of new disciplines into the regulatory science agenda ensuring coordinated actions taken by developers and regulators.

We are happy to assist you in developing your own product specific scientific and regulatory agenda avoiding misled development strategies or prolonged development time lines.

Publications Michael Pfleiderer:

• Official Batch Control of Influenza Vaccines: Is it still Useful?

• The Value of Multi-Country Joint Regulatory Reviews: The Experience of a WHO Joint Technical Consultation on the CYD-TDV (Dengvaxia®) dossier.

• WHO Preferred Products Characteristics for Next Generation Influenza Vaccines

• Quality Standards for Challenge Strains. Requirements for Challenge Strains for Clinical Trial Applications and Marketing Authorization Applications

Publication Diane Seimetz:

• Advanced Therapies in Wound Management: Cell and Tissue Based Therapies, Physical and Bio-Physical Therapies Smart and IT based Technologies

• ATMPs: How to Successfully Master Challenges and Foster the Regulatory Success Rate?

• The Key to Successful Drug Approval: An Effective Regulatory Strategy

• Expediting Drug Development

• Adaptive Pathways and PRIME: A True Acceleration of Drug Development and Approval?

Publication Gabriele Dallmann:

• Scientific Advice in the EU and US: Practical Experience and Rules of the Game on Both Sides of the Atlantic