The second article provides additional details regarding the way patients could be involved in 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 is intended to provide additional clarifications regarding the way the patients could be involved, and how their feedback should be collected and considered. It is important to mention that due to the legal nature of the guidance, the provisions thereof are non-binding, and are not intended to introduce new rules and requirements to be fulfilled by medical device manufacturers or other parties involved. Moreover, an alternative approach could be applied, provided such an approach complies with existing legislation and has been agreed with the authority in advance. 


Patient Engagement: the Concept Explained 

First of all, the Agency states that for guidance, patient engagement stands for intentional, meaningful interactions with patients that provide opportunities for mutual learning and effective collaborations. To do this, the parties responsible for clinical studies may engage patient advisors – the ones who have experience living with a disease or condition, and can serve in an advisory or consultative capacity to improve clinical study design and conduct, but who are not study/research participants themselves or caregivers of study/research participants. At the same time, the authority explicitly states that the above category does not include healthcare professionals. As further explained by the Agency, this category could include patients that participated in similar studies before, as well as the ones that were not selected to participate in the respective trial, or could use medical devices for therapeutic purposes, even though they do not have a corresponding disease or condition. The authority acknowledges that in certain cases it could be difficult to choose patient advisors to be engaged if the disease in question is quite rare. Should a party responsible for the clinical study face any issues with it, it may contact the authority and ask for additional advice. 

Other concepts used by the FDA in the context of patient engagement are:

  • Patients – individuals with or at risk of a specific disease or health condition, whether or not they currently receive any therapy to prevent or treat that disease/condition. This is a general category that covers both patient advisors and study/research participants (as defined below). 
  • Study/research participants – individuals who are or become a research participant, as a recipient of the test article, on whom or on whose specimen the test article is used, or as a control, and may include healthy individuals. The authority also mentions that existing legislation uses the terms “subject” and “human subject” instead, but for this guidance these terms are interchangeable. 

As explained by the FDA, the main difference between study participants and patient advisors is that the latter are not directly participating in a study they are advising for. Hence, study participants could not be engaged as patient advisors for the respective clinical study, and vice versa. 

Questions and Answers 

To assist medical device manufacturers and other parties that may be responsible for clinical studies (sponsors), the Agency provides answers to the most common questions related to patient engagement in medical device clinical studies. In particular, the scope of the guidance covers the following aspects:

  1. The authority recommends sponsors decide on the roles beforehand, and clearly outline them in the respective plan. It will be more efficient to engage as patient advisors those persons who are already familiar with the way clinical studies should be performed, operations of medical devices, and other related matters. Apart from this, sponsors may also provide additional educational materials and training, since this could potentially improve the overall quality of feedback received. According to the guidance, the activities in which patient advisors could be involved include, inter alia, such activities as: 
    1. Preparing informed consent documents to be signed by study participants – patient advisors could assist in making it more understandable and clear for the participants. 
    2. Deciding on methods of data collection to apply the least burdensome approach ensures, however, the appropriate quality of data. 
    3. Discussing challenges that were identified during the study. 
    4. Deciding on the patient-reported outcome (PRO) measures. 
    5. Working with patient advisors to inform the design of patient preference studies that may be used to inform the development of clinical studies or to help understand the benefit-risk tradeoffs among patients for the proposed treatment or multiple treatment options used for the disease/condition.
  2. The way the input from patient advisors could be collected. According to the guidance, study sponsors are encouraged to decide on a way patient advisors would be involved from the very beginning – when the study is being designed and developed. Moreover, it is recommended to engage patient advisors when developing a study plan when it is related to novel technologies, of a new target patient population. At the same time, patient advisors could be also actively involved at the stage of post-study review to analyze the results and decide on the ways for improvement for future studies. Apart from this, patient advisors could be involved in ensuring the study plan focuses on the needs of patients. The authority also mentions that in case an investigational device exemption (IDE) is required due to the nature of the study, the appropriate information should be duly reflected in the documentation related to the study, including the informed consent document. Should a sponsor face any issues with the study, patient advisors could be also involved in searching for the solution. 
  3. Receiving feedback from FDA. As it is stated in the guidance, study sponsors are welcome to provide their input for patient engagement – the matters associated thereto could be discussed with the authority within the scope of the Q-Submission framework. 

In summary, the Agency encourages the further extension of patients’ involvement in clinical studies since this could be useful for obtaining additional information to be considered. The present guidance describes the way patient engagement could impact the process and also highlights the main benefits and challenges associated thereto. Additionally, by the device of this guidance, the authority provides additional recommendations concerning the way patients may be involved in clinical trials to make sure their feedback is duly collected and considered. As it is mentioned by the FDA, the sponsors interested in patient engagement in the clinical studies they are responsible for may contact the authority to discuss the issues associated thereto and obtain additional advice.



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.