#10 Practical RWE – Connecting the Dots: Regulatory Assumptions

#10 Practical RWE – Connecting the Dots: Regulatory Assumptions


Let’s start by assuming that you have done your homework, which means:


  1. You’ve classified your study in each region e.g., non-interventional study
  2. You’ve identified the relevant national regulations and guidelines


Now what? 


Now you need to consider whether there are any other conditions that are applicable to your study.  For example:

[1] Has the study been imposed by a regulatory authority as a condition of the marketing authorisation? Is it a  postmarket requirement (PMR = USA) or a post-authorisation safety study (PASS = EU)?

[2] Are there any study-specific research procedures? If so, will this impact the study classification and/or the ability conduct the study in the country of interest? Examples include the inability to take study-specific biosamples in Germany in the context of a (drug) non-interventional study.

[3] Are there any considerations specific to the patient population e.g., minors, minors who may progress to adulthood during the study term, legally incapacitated adults?

The answers to these questions allow you to start building the regulatory assumptions that form the basis of the regulatory roadmap for your study. In this instance, our study is a prospective post-authorisation safety study of a new treatment (Drug X) for prostate cancer in adults that has been imposed as a condition of the marketing authorisation by the EMA.  Based on this information we would build our regulatory assumptions like this…

Regulatory Assumptions

– Non-interventional post-authorisation safety study (PASS)

– Imposed PASS (Category 1)

– PRAC endorsement of protocol required

– EMA PASS protocol and report templates must be used

– Registration on the EU PAS register is mandatory

– Prospective data collection

– Retrospective data collection = Medical history

– Expedited safety reporting (as per GVP Module VI)

– Study conduct and scientific considerations (as per GVP Module VIII)

– Compliance with national regulatory requirements for non-interventional studies required

– Drug X is approved and prescribed in the study country

– Patients are adults (18 years and over)

– Study-specific biosamples required for genetic screening via a central lab (excludes Germany unless the study is conducted as a low-intervention clinical trial there)

These regulatory assumptions provide the foundation for creating a study-specific regulatory playbook and roadmap covering the national approvals, notifications, and study conduct considerations. 

Regulatory assumptions are important.  They detail, in simple language, the key elements of the study that drive the regulatory requirements and operational considerations.  Small changes to these regulatory assumptions can have a big impact on the study in terms of time, cost and feasibility.

Remember…small changes can have a big impact!

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