The Center for Devices and Radiological Health (CDRH), a division of the Food and Drug Administration (FDA), has published detailed information about the Patient Science and Engagement Program – the new approach intended to improve the engagement of patients in the development of medical devices.
The appropriate activities are being carried out by the Patient Science and Engagement Program team consisting of healthcare professionals, scientists, and other experts. The spheres of their activities include, inter alia, clinical outcome assessments, methods of processing patient-generated data, and patient preference information in general. According to the official notice published by the CDRH, the mission of the Patient Science and Engagement Program is to engage with patients, understand their perspectives, and proactively integrate the patients` perspectives into the total product life cycle of medical devices to help protect and promote public health.
General Approach
The CDRH states that the active engagement of patients in all the processes related to the development of medical devices would improve the effectiveness of such processes and help to ensure the safety and effectiveness of medical devices in general. According to the position of the authority, further engagement of the patients would help to understand better the patients` needs and ways medical devices could be more useful.
Patient Preference Information (PPI) in Medical Device Decision-Making
The first concept described by the CDRH is the Patient Preference Information (PPI), which provides that it is important to consider the position and opinion of patients using medical devices on a daily basis to meet their current needs. As it is stated in the appropriate notice, PPI stands for a type of patient experience data that incorporates the patient perspective in CDRH`s regulatory decision-making.
PPI describes the aspects of medical devices that are the most important for the patients. In particular, the patient-preference information describes various perspectives with regard to risks and benefits associated with a medical device. The CDRH additionally emphasizes the importance of distinguishing the patient-preference information and patient-reported outcomes (the structured information deriving from a clinical trial). The patient-preference studies should be conducted in order to identify which particular aspects of a medical device are the ones most important for the patients, and how do they prioritize them.
According to the notice published by the CDRH, the patient preference studies could be conducted by:
- Patient groups,
- Members of the medical device industry,
- Researchers,
- Healthcare providers,
- Public-private partnerships.
Hence, the more patient preference studies are conducted, the better the industry representatives would understand the actual needs of patients using the medical device they manufacture. The information collected by the virtue of such studies could be also used to design clinical studies and evaluate their effectiveness from the patents` perspective.
The CDRH states that the information collected during the patient preference studies could be used for the following purposes:
- To identify the needs of the patients that are not currently met by medical devices already placed on the market,
- To determine the risks and benefits associated with a medical device, that are the most important for the patients,
- To assess the importance of clinical study results and identify the meaningful changes,
- To understand the way the patients usually evaluate the risks and benefits associated with medical devices,
- To understand the way the patients percept the information about medical devices, and also the way they accept uncertainty associated thereto.
The authority states that nowadays patient preference studies are often used in the context of regulatory submissions. The CDRH actively cooperates with various groups of patients to ensure the reliability and variety of the information collected through such studies. It is also important to mention that the interested party intended to carry out a patient preference study may contact the CDRH and arrange a pre-submission meeting in order to clarify the most important aspects in advance. Later the medical device manufacturer shall consider the patient-preference information on all the stages of a product development cycle. However, this information and conclusions based thereon should not prevail over the standards and general requirements to be applied for medical devices.
Clinical Outcome Assessments in Medical Device Decision Making
Another important concept addressed by the CDRH is the Clinical Outcome Assessments (COAs). According to the CDRH guidelines, a clinical outcome assessment describes or reflects how a person feels, functions, or survives and can be reported by a healthcare provider or a non-clinical observer (such as a parent), through the performance of an activity or task, or by the patient. The CDRH distinguishes the following four types of COAs:
- Patient-reported outcomes (PROs);
- Clinician-reported outcomes (ClinROs);
- Observer-reported outcomes (ObsROs); and
- Performance outcomes (PerfOs).
Each of the aforementioned types provides a specific perspective, namely:
- PROs constitute the information provided by the patient himself/herself. Such information could be collected by the virtue of various questionnaires or diaries.
- ClinROs – the reports to be formed by the healthcare professionals describing the observations they make.
- ObsROs – the reports to be formed by someone who observes the patient in daily life.
- PerfOs – the information collected when the patient conducts special pre-determined actions on a daily basis in order to ensure the accuracy and reliability of the measurements.
According to the official information published by the CDRH, the information collected through the COAs should be used in order to improve the benefit-risk assessment. The medical device manufacturers may also use it to improve the labeling of their products by adding information describing the way the medical device impacts the symptoms. Moreover, such information could be used for the purpose of selection of the patients eligible for clinical trials, and also for evaluation and assessment of the actual performance of medical devices during such trials.
The authority actively cooperates with the healthcare professionals, scientists, and patient groups for the purpose of continuous improvement of the way the COAs are used in the context of various regulatory procedures and processes.
As in the case with PPIs, the CDRH encourages the parties interested in the inclusion of COAs in regulatory submissions to contact the authority in advance to discuss the most important aspects. The information provided by the CDRH also describes the ways the information collected from the patients could be used.
Summarizing the information provided here above, the CDRH acknowledges the importance of the information collected directly from the patients and reflecting their perspective with regard to the medical devices and various use aspects associated thereto. In particular, the authority distinguishes various types of such information depending on the way it was collected and processed. In general, the information describing the preferences of the patients and the aspects they find to be the most important should be used by the medical device manufacturer for the purpose of further improvement of medical devices. Moreover, this information could be also used to improve the way the important information about medical devices could be communicated to the patients in a clear and understandable way.
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