The article provides a brief overview of the concept of patient engagement from the very first days and also outlines the key points to be considered by the parties responsible for clinical studies. 



The Food and Drug Administration (FDA or the Agency), the US regulating authority in the sphere of healthcare products, has published a guidance document dedicated to patient engagement in the design and conduct of medical device clinical studies. The document constitutes a final version of the guidance based on the draft initially published in September 2019. The authority also mentions that the document is non-binding, moreover, an alternative approach could be applied, provided such an approach complies with the respective legislation and has been agreed with the authority in advance. 

Regulatory Background 

First of all, the authority emphasizes the importance of patients’ involvement in medical device development and assessment processes. In particular, it is stated that the patients’ experience should be taken into consideration by medical device manufacturers and other parties involved in operations with medical devices, especially about the way the device operates when used for its intended purpose. In certain cases, useful information could be provided by patients even if they are not involved in a clinical study, but are merely using the device for their own needs. 

As explained by the Agency, the present document is intended to: 

  1. Help sponsors understand how they can voluntarily use patient engagement to elicit experience, perspectives, and other relevant information from patient advisors (see definition in Section IV) to improve the design and conduct of medical device clinical studies;
  2. Highlight the benefits of engaging with patient advisors early in the medical device development process; 
  3. Illustrate which patient engagement activities are generally not considered by FDA to constitute research or activity subject to FDA’s regulations, including regulations regarding institutional review boards (IRBs); and
  4. Address common questions and misconceptions about collecting and submitting to FDA patient engagement information regarding the design and conduct of a medical device clinical study.

As it was mentioned before, the provisions contained therein are non-binding in their legal nature, and should not be construed as the ones introducing new rules and requirements the parties involved should follow. The document is intended to provide additional clarifications regarding the applicable regulatory requirements, as well as recommendations to be considered to ensure compliance thereto. 

Patient Engagement: Key Points 

According to the guidance, the development of a special framework for patient involvement commenced back then in 2017 and was then reflected in the Clinical Trials Transformation Initiative (CTTI) in 2019. Those two points created a basis for further actions taken by the Agency to ensure the expansion of patients’ involvement in the process of developing and testing medical devices and accessories thereto. As the very first step, the Agency has issued a respective discussion document to collect feedback and suggestions on the matter. 

The authority additionally emphasizes that successful adoption of legally marketed medical devices increasingly depends on patient acceptance of that technology and patients being more engaged in the healthcare process, along with demonstrated public health benefits. As further explained by the Agency, active involvement of patients could be a valuable source of information to be considered by medical device manufacturers concerning the products in question and the way they perform, especially in situations when due to the nature of the treatment the device is used for a long time while interactions between patient and healthcare professional are quite rare. It is also mentioned that the overall improvement of the regulatory framework for clinical trials would be beneficial for the industry in general, as it could reduce the unneeded regulatory burden and reduce the time and efforts needed to place a new medical device on the market. 

Thus, when developing a clinical study, the party responsible for it should consider the way the patients would be engaged in terms of receiving their feedback. The authority expects that active patient involvement could result in:

  • Faster study/research participant recruitment, enrollment, and study completion;
  • Greater study/research participant commitment and retention, resulting in decreased loss to follow-up;
  • Greater study/research participant adherence resulting in fewer protocol deviations/violations;
  • Greater study/research participation by diverse patient populations;
  • Fewer protocol revisions;
  • Streamlined data collection resulting in better quality data; and
  • More relevant data on outcomes that matter to patients.

The authority states that according to the feedback it has received from the industry concerning the matter, the idea of a wider patient involvement was broadly accepted. At the same time, certain issues were identified as well. In particular, the Agency found out that there is a perception that the extent to which patients could be engaged in clinical trials is significantly limited, and the input provided by the patients is not so valuable. Apart from this, the parties responsible for clinical trials usually find it difficult to allocate sufficient resources and, which is more important, to find patients who are familiar enough with the way clinical studies should be carried out. In certain cases, logistical challenges are making it difficult to interact with patient advisors, or even with determining the advisors to be engaged. The industry representatives also pointed out a few more issues associated with patient engagement, some of them are addressed in the present guidance issued by the FDA. 


Applicability of the Guidance 

The authority explicitly states that the input to be received from patients could be valuable at all the stages of the medical device’s lifecycle. However, the present document provides clarifications and recommendations that are mostly related to clinical trials and describes the way patients could be engaged at this particular stage. The FDA also mentions that the aspects related to study/research participant or patient advisor reimbursement or compensation, promotion of investigational devices, or dissemination of clinical study results are falling outside the scope of this guidance. 

In summary, the present FDA guidance is intended to describe the current thinking of the authority about patient engagement in clinical studies. The document provides additional clarifications and recommendations regarding the way the parties responsible for clinical studies could improve the effectiveness of interactions with patients to obtain additional information. 



How Can RegDesk Help?

RegDesk is a next-generation web-based software for medical device and IVD companies. Our cutting-edge platform uses machine learning to provide regulatory intelligence, application preparation, submission, and approvals management globally. Our clients also have access to our network of over 4000 compliance experts worldwide to obtain verification on critical questions. Applications that normally take 6 months to prepare can now be prepared within 6 days using RegDesk Dash(TM). Global expansion has never been this simple.