Regulatory Affairs

Platform technologies for vaccines – why is there still no regulatory guidance available?

by Michael Pfleiderer

The recent widespread, long-lasting and recurrent outbreaks of Ebolavirus, Chikungunyavirus, Denguevirus, Zikavirus once more prove the need for rapid availability of vaccines and appropriate platform technologies. Read Michael Pfleiderer’s expert opinion on a matter of the highest relevance.


Drug repurposing: how to protect your asset by an integrated regulatory strategy

by Michael Firgens

Drug repurposing is an alternative cost-effective approach to drug development which builds on already approved drugs and compounds. However, the actual commercial success of a repurposed drug depends on achieving effective protection against competitors such as generics and other products with the same active pharmaceutical ingredient. In this article, Michael Firgens explains the key strategies for the protection of a repurposed drug.

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, clinical trial methodology and epidemiology, and social sciences. In the report by Michael Pfleiderer, you can read about the importance of regulatory sciences to contribute to the development of new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of regulated products.

Regulating Bacteriophages: The First Steps into the Post-Antibiotic Era

by Luka Kotrikadze

It was in the year of 1915 when Frederic William Twort published the paper in The Lancet describing the discovery of an infectious agent that causes the lysis of the Staphylococcus hyicus bacteria. This served as the first ever published description of bacteriophages, therapeutic potential of which was only later assumed by Fe´lix d’Herelle. With the advancements into the research soon foregone with the discovery of the antibiotics, nowadays, with the increasing attention towards the antimicrobial resistances, the bacteriophage therapy is slowly stepping into the light of its come back. You can read about the current regulatory and product associated issues of bacteriophages, and the industry standpoint in the article by Luka Kotrikadze.

Ensuring regulatory and scientific compliance with the requirements of the Common Technical Document (CTD) of data generated by manufacturers or manufacturing consortia

by Michael Pfleiderer

Manufacturers of medicinal products operating within and outside the European Union (EU) or the European Economic Area (EEA) who aim at placing their products on the EU/EEA market often find it difficult to correctly interpret data requirements for a valid CTD. Uncertainties on the amount and quality of scientific data to be generated and on the way how to optimally present these data within the individual sections of the CTD very often create a major hurdle that cannot be overcome easily. Most notably, uncertainties and discrepant opinions on these issues can significantly complicate partnering processes between two or more enterprises that are dependent on each other for successful drug development and licensure.

How should we regulate genome editing for pharmaceutical applications?

by Dr. Diane Seimetz

Genome editing technologies are not only having a game-changing effect on research. They also have the potential to transform health care by curing diseases for which there are currently no or no satisfactory therapies available.

In particular with the emergence of novel gene editing technologies such as CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats) in the pharmaceutical arena the question arises how to best regulate these technologies.

On 18 October 2017 the European Medicines Agency (EMA) organized an expert meeting on genome editing technologies used in medicinal product development. Diane Seimetz contributed to the meeting as invited expert and has summarized the discussion and critical aspects in this article.

Hospital Exemption for ATMPs: Perspectives & Regulatory Trends Under Discussion

Advanced Therapy Medicinal Products (ATMPs) bear game changing potential for the treatment of severe conditions for which we have no appropriate therapies today. The product class of ATMPs comprises gene therapy medicinal products (GTMPs), somatic cell therapy medicinal products (sCTMPs), tissue-engineered products (TEPs) as well as combined ATMPs. ATMPs are complex products, and ATMP developing companies face diverse challenges during development as well as for commercialization.

Regulatory Pathways for Orphan Drug Designation

To obtain orphan drug designation, drug developers need to understand how to manoeuvre through the regulatory frameworks and to prepare a sound justification for their drug candidate. The regulatory requirements and evaluation process may vary from country to country and also depend on the policy status and experience of regulators. The individual elements for an orphan drug designation are illustrated by the example of the EU regulatory framework.

Effective Regulatory Strategy: The Key to Successful Drug Approval

The development of new drugs is essential to patients and healthcare systems but requires significant investment and risk taking. At the same time, both costs and time of drug development have increased over the past decades resulting in higher pressure on development programs. To avoid pitfalls, it is critical to define an effective regulatory strategy early on and to integrate it in the development program.