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Mar 6, 2019

The European Medicines Agency (EMA) has published the first of a series of guidance documents to help applicants prepare for obligations stemming from the new EU regulations on medical devices.

The new regulations introduce new roles and responsibilities for EMA and national competent authorities (NCAs) in relation to certain types of medical devices and in-vitro diagnostics. The Agency is working closely with the EU regulatory network and with stakeholders from the pharmaceutical and medical device industries, including notified bodies, to ensure a smooth transition to the new regulatory framework.

Overseas inspections are targeted on foreign pharmaceuticals and medical devices that have been marketed or are going to be marketed in China. The inspections focus not only on manufacturing sites but also on research and development sites. The inspections will apply Risk Management Principles and go through the processes from approvals, oversight responsibilities, analyses, complaints to AE monitoring and safety management.

The U.S. Food and Drug Administration (FDA) is reminding stakeholders that February 21st, 2019 is the effective date for compliance with the final rule on Human Subject Protection; Acceptance of Data from Clinical Investigations for Medical Devices. The rule updates the standards for accepting clinical data from clinical investigations conducted inside and outside the United States to protect human participants, and to help ensure the quality and integrity of data obtained through such investigations.

A survey launched by EMA in September 2016 shows that validation issues occur in 90% of initial marketing authorisation applications (MAAs). They create additional workload for companies and potential delays at a critical moment for the timely start of the procedure. To make the validation process more efficient, predictable and easier to navigate, EMA is encouraging applicants to submit a new validation checklist as part of the MAA dossier.

The initiative is launched as a five-month pilot and is expected to increase the number of ‘first time right’ submissions significantly. Applicants who use the checklist are invited to submit their comments to checklist_ima@ema.europa.eu. EMA will use this feedback and the information collected on the quality of submissions using the new checklist to improve the validation process itself.

The checklist includes a number of questions that will help companies to assess the level of completion and consistency of the various sections in their applications. More information can be found in the updated Pre-authorisation guidance questions and answers (Q&A) on the website.

In addition, EMA has also updated the Q&A section on inspections and compliance with an updated form on good clinical practice (GCP) as well as clarification on statements required in the cover letter for good manufacturing practice (GMP). These will help companies to structure the relevant information required for the organisation of future inspections.

After completion, EMA will analyse the result of the pilot and inform stakeholders about the conclusion in the second half of 2019.

The guideline provides guidance on the structure and data requirements for a clinical trial application for advanced therapy investigational medicinal products (ATIMPs). The guideline is multidisciplinary and addresses development, manufacturing and quality control as well as non-clinical and clinical development of ATIMPs. Considerations on genome editing tools are included.

The REMS Assessment draft guidance aims to increase stakeholder understanding of how to develop a REMS Assessment Plan by specifically discussing how the REMS program goals, objectives, and REMS design may impact the selection of metrics and data sources, which will be used to assess whether the REMS is meeting its risk mitigation goals. It recommends assessing the REMS using both process measures and outcome measures and provides examples of metrics by assessment categories, and data sources that may be utilized to evaluate the performance of the REMS. It also includes considerations for assessing the impact of REMS on patient access to the drug or its burden to the healthcare delivery system. Finally, this guidance provides recommendations on a standard format that may be used to report assessment findings according to the timetable for submission of REMS assessments.

The REMS Survey Methodologies draft guidance provides recommendations to industry on conducting REMS assessment surveys used to evaluate respondent knowledge of REMS-related information. The majority of applicants use surveys to evaluate patients’ and healthcare providers’ understanding of the serious risks associated with, and safe use of, their drugs to assess REMS knowledge goals. This draft guidance discusses general principles and recommendations related to conducting REMS assessment knowledge surveys, including study design, survey data collection and processing, and data analysis. It also provides recommendations for developing patient and healthcare provider survey instruments (i.e., questionnaires).