Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

"Lessons learned" for Regulatory and Scientific Advice of Vaccines and Biologicals in Times of COVID-19

Dr. Manja Burggraf (Abschlußjahr: 2021)

Summary
Language: English
As the SARS-CoV-2 pandemic went global it became clear that very fast effective preventive treatment will be necessary. Ideally a variety of vaccines to tackle this crisis had to be available very soon, as the whole population might not be efficiently treated with only one vaccine and production will need much more time, compared to a situation when several developers producing in parallel. Moreover, there is the need for development of medicinal product which can be applied in the therapy of COVID-19 patients. Therefore, the obvious question was asked how to enhance medical product development in general.
In the case of SARS-CoV-2 no vaccine and therapeutic treatment was readily available, making it necessary to develop new medicinal products, or invest on drug repurposing. The development process of a new medicinal product is very complex, time-consuming and requires lots of resources. Before the start of development, a sound scientific understanding about the virus is necessary, in order to find potential targets for treatment. After identification of a potential target, a corresponding medicinal product must be developed considering reproducibly sufficient quality, several parameters during non- clinical tests as well as efficacy and safety as elaborated in clinical studies. Corresponding information will also be relevant during the authorisation process of the medicinal product. In the end the developer must show robust evidence for a positive benefit/risk ratio of the medicinal product which enables the health authorities to grant a marketing authorisation. To give the developer guidance on the overall requirements, comprehensive literature of various degrees of legally binding nature exists.
On this very burdensome way the developer can request Scientific Advice (SA) by the agency, which is a powerful tool to avoid or address obstacles early in the development thereby streamlining and accelerating the process itself. Like all regulatory processes, requesting and receiving SA is a very formal procedure, bound to timelines and a potential high fee. However, during the pandemic an unprecedented flexibility on European and national level was applied to SA procedures, e.g., the concept of rolling review, or ad-hoc SA.
This thesis addresses the questions how the national competent authorities (NCA) addressed the pandemic with a focus on efficient and effective SA. In a survey the NCA (respondent) were asked about kind and occurrence of applied changes of SA procedures.
Moreover, feedback on hurdles during medicinal product development and general pandemic measures implement by the agency to ensure business continuation were requested. Evaluation of the answers to this survey shall provide a conclusion on usefulness and sustainability of the made procedure changes. Areas of product development shall be scrutinized for hurdles which need updated guidance or enhanced support. And with the knowledge about the general pandemic measures taken by the agency an assumption shall be drawn which measure are efficient and effective and therefore part of the strategy for enhanced preparedness of health authorities for potential future health crises.
The results of the survey showed that nearly every agency which provided COVID-19 related SA did adjust its own procedures. As one conclusion most of the respondents emphasized the application of shorter timelines for the provision of SA acknowledging the high need for timely access to effective treatments. Enhanced flexibility regarding the formats of SA, a balanced, reduction of formal requirements and decreased or no fee, were other major applications identified in the survey.
The survey also showed that most of the health agencies were prepared and adapted to the demands of the pandemic with increased flexibility, communication (intern and extern (for professionals and lay language)) and in parts with the employment of specialist, new staff. Adjustment to the COVID-19 related SAs had to be as dynamic as the pandemic itself, especially concerning clinical study design and the upcoming threat of the SARS-CoV-2 variants.
The responses to the survey underlined the importance of early contact with the NCA and demonstrate, that SA is and has been in an integral part of an enhanced development of medicinal products regarding COVID-19 and can be for enhanced authorisation processes in the future. Moreover, continuous critical review of established processes, as well as the documentation of lessons learned will improve the preparedness for future health crises.
Pages: 68
Annexes: 2, Pages: 7