Medical Device Regulators Lead the Field
The medical device field has been leading the way in demonstrating the implementation and value of patient input. Over the past several years, the U.S. Food and Drug Administration (FDA), specifically the Center for Devices and Radiological Health (CDRH), has shown an increased focus on integrating patients and patient input into the regulatory approval process. They recognize that patients offer a unique perspective on the benefits and risks of new devices and procedures, which may not always be reflected in clinical trial results assessed by FDA staff and advisory committee members. If the ultimate goal is to improve public health by enabling patients to have access to innovative new therapies, then the views of the patients themselves need to be part of the decision-making process.
A major milestone was met in May 2015 when CDRH released a guidance on the use of patient preference information in regulatory submissions. Although sponsors are not required to include this type of data in their submissions, if they choose to, the guidance provides recommendations on performing patient preference studies and outlines how the FDA will use patient preference information in its review and decision. Since releasing this guidance, CDRH has noted an 80% increase in the inclusion of patient preference studies in regulatory submissions. Over the period of 2009 to 2015, submissions with patient reported outcomes increased by 500%, suggesting the value of patient input has been recognized by industry for quite some time.
New Therapies Primed to Benefit from Patient Engagement
Patient preference information could be particularly valuable for new technologies like focused ultrasound that are non-invasive and offer benefits such as minimal time in the hospital, reduced recovery time and fewer side effects. To obtain these benefits patients may be willing to accept the “risk” of a new therapy where the safety profile and long-term efficacy are less established than standard treatments. The benefit-to-risk ratio is always used in FDA decisions on regulatory submissions but by adding patient feedback into the process, patients may get access to promising new therapies more quickly.
Committee Seeks to Improve Clinical Trial Design
To build on their previous work and continue to develop methodologies for implementation of patient input into the medical device field, earlier this year the FDA established an advisory committee devoted to issues of patient engagement throughout the medical device innovation life cycle. The Patient Engagement Advisory Committee (PEAC) will advise the FDA on patient-related issues with an intent to promote more effective incorporation of patient perspectives in the medical device regulatory process. The committee includes patients, patient advocates, health care providers and an industry representative. Topics that the committee may address include “[FDA] guidance and policies, clinical trial or registry design, patient preference study design, benefit-risk determinations, device labeling, unmet clinical needs, available alternatives, patient reported outcomes and device-related quality of life or health status issues.”
On October 11-12, the PEAC met for the first time in an open public hearing to promote discussions and provide recommendations on the engagement of patients in the clinical trials process. Key areas of discussion included patient input into the design of new trials; recruitment, enrollment and retention into trials; and how best to communicate study results to patients. By partnering with patients and patient organizations early in the design of trials, elements of the trial (inclusion and exclusion criteria, study protocol, and follow-up requirements) may be developed that better reflect the appropriate patient population and won’t hinder their involvement in the study. This could potentially lead to ease of patient recruitment and shorter timelines for completion of studies.
In the design of new focused ultrasound trials, considerations of factors that may be prohibitive to patient recruitment, or the timing and number of visits for the treatment and follow-up, and flexible options for follow-up such as telehealth visits or the use of wearables, are just a few considerations that could be informed by direct patient engagement early on.
Can Patient Input Streamline Studies?
There is a growing community of stakeholders using the science of patient input throughout the life cycle of drugs and devices, including research and development, clinical trials, and post-market assessment. The Medical Device Innovation Consortium (MDIC), a public-private partnership with the FDA focused on issues of regulatory science, has been a leader in this field with significant efforts encouraging and developing processes for the use of the science of patient input in the medical device ecosystem. MDIC’s membership includes the medical device industry and other affiliated stakeholders including the Focused Ultrasound Foundation.
MDIC is also leading efforts that could demonstrate to industry the value or return on investment of using patient input. They have begun an innovative new project to integrate patient preferences into the statistical design of clinical trials, focusing on treatment of Parkinson’s disease as a pilot scenario. Perhaps patient preferences can help reduce the number of patients needed in a particular trial, thus reducing the timeline and costs for regulatory submission and approval and getting a new therapy to patients more quickly. The Foundation sees the relevance and importance of this work to focused ultrasound and to our mission to serve patients through acceleration of the field. We are engaged in some of MDIC’s efforts, including my participation on the MDIC Science of Patient Input Steering Committee and the Maximizing Patient Participation in Clinical Trials Working Group.
So can partnering with patients help advance focused ultrasound? Evidence is mounting to suggest that it can, and we’ll continue to share what we learn from others who are building the science of patient input.