January 13, 2025 FDA-2024-N-4561
Thank you for the opportunity to comment on Device Trial Participation Snapshots. The National Center for Health Research is a nonprofit public health think tank founded in 1999 that focuses on the safety and effectiveness of medical products. Our research on medical devices has pointed out that patients and physicians are often unaware of the data used as the basis for the approval of medical devices and so we are very pleased that the Center for Devices and Radiological Health (CDRH) has created “Device Trial Participation Snapshots” to present important device trial information to patients. We reviewed the five example snapshots provided by CDRH at https://www.fda.gov/medical-devices/recently-approved-devices/device-trial-participation-snapshots and have the following recommendations.
Our main recommendation is that the snapshots should use simpler wording and sentence structure and also explain the study designs and outcomes in terms of benefits and risks in ways that will be more meaningful to patients as well as health professionals.
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- Simple and clear explanations of the study design and implications for the quality of evidence. The snapshots should state whether the study evaluated patients who used the device compared to patients with a different treatment (or sham control) and whether comparison groups were randomized, blinded, or matched; the duration of follow-up (including missing data); and whether benefits were measured by clinically meaningful outcomes, composite endpoints, or surrogate endpoints. The implications of these study design and implementation issues should be briefly and clearly explained so that the strengths and weaknesses of the evidence are easy to understand for patients and health professionals.
- Quantifying benefits and risks. The percentage of patients who experience specific complications should be provided and compared to sham or other treatments as absolute risks rather than relative risks, because that is more meaningful to patients. Examples of mild, moderate, or severe adverse events and complications should be in plain English, not in medical terminology. Generalizations such as “the device showed success” or “the treatment was generally well tolerated and adverse events were usually mild or moderate” do not provide meaningful information needed to help make treatment decisions. Benefits should also be quantified in terms of absolute rather than relative benefits, and differences in risks and benefits should be listed by demographic groups, as well as in the summary. Although the summary for clinical audiences can be more technical than for patients, the types of information regarding risks and benefits should be the same.
- Diversity. We applaud the decision to provide information about the inclusion of patients from major demographic groups, but this information should clearly include the numbers of patients in each group, not just the percentages. In fact, the numbers of patients in any demographic group are often more important for providing information than the percentage. For example, a study where 5% of 1000 patients are Black will usually provide more useful data about Black patients’ experiences than a study where 20% of 40 patients are Black. The generalizability of the data should also be clearly explained, whether based on the number of patients or how they were selected for the research.
- Standardizing the Summary for Clinical Audiences. There is considerable variability in the level of detail used to describe endpoints and trial design varies in the five snapshots. This section should include the types of information described in our first bullet (Study design and implications for the quality of evidence). If there are mandated post-market studies they should also be briefly described, including when they will be completed.
- Providing citations for referenced studies. This should include links when available and should specify where the studies were published and if they were published in peer-reviewed journals.
In our example below, we have provided specific examples of the kinds of information and wording that would improve the usefulness of the snapshots without making them too long to be read. Please contact dz@center4research.org for any additional information. We’ve cut and pasted the first example provided by CDRH, with our edits in red font and strike-through deletions, and our comments in green font:
“Summary:
What is this device used for?
The Lumicell DVS is a medical tool used by a surgeon to help look at breast tissue after a piece of tumor has been removed during surgery. The device helps the doctor look at the remaining tissue to determine if all of the cancerous tissue was removed.
How is the device used?
Doctors use this device during a surgery to remove only the breast tumor and a small amount of healthy tissue around the tumor. The device is used with an imaging a dye that helps the doctor see highlight cancerous tissue still in the patient once most of the breast tumor has been removed.
What are the benefits of this device?
In a study of __ patients, this device helps to find residual cancerous tissue within the body after the removal of tumor was removed from the breast. Cancer that had not been removed was found in x% of the patients. [Provide specific results!] This means a doctor would be able to see if there was undetected cancerous tissue left in the breast before ending the surgery in some patients. However, it does not tell us if all undetected cancer was accurately found in all the patients studied.
What are the risks and potential harmful side effects of this device?
There were no device related harmful side effects reported due to the device itself. However, there were harmful side effects reported with the use of the imaging dye that has to be used with the device. The most common side effect reported was abnormal color of urine due to the dye in the drug, and that is not harmful. However, a rare, but serious side effect was anaphylaxis (a dangerous allergic reaction or hypersensitivity to the dye).
Demographics Snapshot:
Figure 1, Participant Demographics by Sex All participants were female.
Figure 1. Participant Demographics by Race: [ List numbers instead of or in addition to percentages on a pie chart]
Figure 2. Participant Demographics by Ethnicity [ List numbers instead of or in addition to percentages on a pie chart]
Who participated in the clinical trial studies?
The clinical study included adult, female patients with breast cancer. This study helped determine if the device was safe and effective. For more information about the demographics of all participants, please see Table 1.
What did the trials evaluate?
These trials were designed to determine how well the Lumicell DVS found remaining cancerous tissue. All patients received the imaging dye. [State if true: There was no comparison group of patients to see how frequently patients benefitted from the Lumicell DVS device compared to those whose surgeon didn’t use the device.] The trial was designed to determine if doctors removed more cancer had better success with complete cancer removal after the imaging system was used compared to before the imaging system was used in the same patient.
Were there any differences in how well the device worked or risks among different sexes, races, ethnicities, and age groups?
All patients were female. Because of the small number of patients in the study, While we know the sex (all participants identified as female), age range, race, and ethnicity of the study patients, we do not have information on whether there were differences in benefits or risks for women of particular ages, races, or ethnicities.
Summary for Clinical Audiences
The trials were created to evaluate the proportion of patients who have residual cancer found in at least one device-guided shave among all patients in the treatment arm. In addition, acceptable sensitivity and specificity in detecting cancerous tissue were evaluated. [These results should be described briefly here.] This was accomplished through a randomized, controlled study format.”