The National Evaluation System for Health Technology Coordinating Center (NESTcc), an organization created and supported by the US Food and Drug Administration (FDA) that focuses on the development of new approaches to data used to generate evidence related to safety and effectiveness of medical devices, issued a framework dedicated to the methods used to process data and information. The document is intended to assist all the NEST stakeholders, including the regulating authorities, healthcare institutions and professionals, groups of patients and industry representatives in establishing the efficient processes related to the safety and performance data.
The document has been developed by the NESTcc Methods Subcommittee that was created to develop the methods used for data analysis. In particular, the framework issued by the Subcommittee is intended to provide all parties involved with the analytical tools to be used during the development, execution, and evaluation of the studies. The principles described in the document could be applied to any and all studies related to medical devices.
As it is indicated in the document, it is based on two core principles:
- pre-specification based on the template created at the first step of the development process, and
- justification of control.
According to the framework, the process of planning any study should be commenced with the development of the document highlighting the most important aspects of the study. This document, the study protocol, should include the description of the following elements:
All these points are generally referred to the acronym PICOTS. To assist parties involved in the study development process, the NESTcc Methods Subcommittee developed a template of the study protocol, which is included in the present framework.
It is required to develop study protocols before the actual enrollment takes place. According to the framework, a study protocol should include the following elements:
- Description of the existing study background, including the details on the disease, applicable therapies and also the risk associated with the use of the device.
- A detailed description of the device itself.
- Objectives of the study.
- Methods used to select particular patients.
- Expected outcomes.
- Exposure of the device and schedules of the outcomes.
- Description of the design of the study, including the comparison of the devices and controls.
- Description of the procedures.
- Size of the sample necessary for the study.
- Information on the registration of the study.
- Plans regarding the monitoring.
- Statistical analysis plan.
The framework also provides that the particular requirements applicable to the evidence should depend on the stage of device development and the risk associated with the use of the device.
Study Background and Medical Device Description
First of all, the study protocol should provide the information regarding the particular disease to be treated by the medical device subject to evaluation, and also the description of all applicable standards related to therapy to be performed in such cases. Such a description should be detailed enough to include the aspects related to the condition of the particular patient, all treatment options available, and the way the new device is expected to impact the situation with the unmet medical needs.
The description of the device itself should cover its composition and design, the way it operates and the main principles it is based on, as well as the information regarding other similar devices already available on the market (also referred to the predicate devices). This part should also include the analysis of all identified potential risks and benefits associated with the use of the device for the intended purpose (which should be indicated on the label of the device).
The document provides a list of principles to be taken into consideration when providing general information on the background. In particular, it should include:
- Information about the targeted disease,
- Overview and summary regarding options of the therapy available at the moment (as well as benefit/risk profiles, analysis of the evidence and results for each particular option),
- A detailed description of the unmet medical needs, including the relevant experience with similar devices, description of the principles the device`s performance is based on, and also the details of potential benefits and risk of the particular device,
- Information about the evidence provided to confirm the compliance with the safety and effectiveness requirements,
- Overview of the previous experience and relevant scientific literature,
- Details about the connection between the performance of the device and the benefits resulting from using it.
Another important part of the description should be dedicated to the device itself, covering all the significant aspects, namely:
- Description of the device, each of its components and accessories intended to be used with the device, and also the unique device identifier (UDI),
- The way the device operates (its mode of action) and its intended use as defined by the manufacturer,
- Benefits associated with the device, and also the description of the measures implemented to mitigate identified risks,
- Information on the outcomes of all pre-clinical studies,
- Technical requirements and the information about sizing,
- Expected performance of the device in the future,
- Indication of the current status for each particular component of the device.
Target Patients, Methods, and Objectives of the Study
Another important point to be highlighted in the study protocol is the description of the study-specific objectives. According to the framework, a study protocol should also include the indication of the objectives of the study, aligned with its overall purpose. The objectives of a study should meet certain criteria – in particular, they should be:
- Based on measurable quantities,
- Attainable within a reasonable timeframe.
According to the general approach, all objectives should be sorted depending on their importance. This part should also include references to the key points of attention and the information future decisions and conclusions would be based on. Thus, it is required to explicitly identify the primary objectives of a study.
The framework also provides that the study objectives should comply with the following principles:
- General SMART principle (Specific, Measurable, Attainable, Relevant, Time-Framed),
- Indication of the particular element subject to evaluation (whether it is a device in general or any of its components separately),
- Associated risk and potential hazards,
- Statistical calculations regarding the safety and effectiveness of the device.
A study protocol should also describe in detail the selection method used to engage patients. According to the general rule, all of them should belong to the population of intended use – the particular part of the population the device is intended to by the manufacturer. A study protocol should contain not only the description of the approach used to engage the patients as the participants of the study but also the details regarding the experience of the healthcare professionals and operators using the device.
Summarizing the information provided above, the new Methods Framework developed by the NESTcc Methods Subcommittee describes the most important aspects related to the way the study to be designed in order for its results to be accurate, relevant and admissible. It is also intended to assist in developing a proper study protocol serving as a basis for future investigation.
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