A medical educator once said that "a good
physician treats the disease, the great
physician treats the patient who has the
disease." Inherent in this quote is the importance of
listening to the patient and understanding that
they are people with families, jobs, and
hobbies.
They just happen to be living with a medical
condition.
All of us committed to protecting and promoting
public health also have the opportunity to embody
this profound instruction of putting the patient
first.
I am Michelle Tarver, the director of the Patient
Science & Engagement Program at the Center for
Devices and Radiological Health.
Today, I have the pleasure of sharing with you the
work we are doing to incorporate patient
perspectives across the total product life cycle
of medical devices.
Our Center is galvanized in its mission to protect
and promote the health of patients.
Patients are truly the inspiration for all the
work we do.
This is reflected in our vision statement that
patients in the US have access to high quality,
safe, and effective medical devices of public
health importance, first in the world.
Patients is the first word because they are the most
important customer and the group most impacted by our
decisions.
But in order to understand what is important to the
public and patients, we must listen to their
needs, wants, and concerns.
The benefits of hearing the patient voice can be
realized across the total product lifecycle of
medical devices.
Hearing the unmet needs and wants of people living
with a medical condition may inform the discovery
and ideation phase for a medical device.
Once the prototype is designed, patients can
give input that could increase usability of the
device.
Patient perspectives can inform the design of the
clinical study, yielding more patient-friendly and
patient centric studies that provide high quality
evidence.
The tradeoffs that patients are willing to
make between benefit-risk can be considered by FDA
to help inform regulatory decision-making.
Patients can assist with crafting the language used
to communicate those benefits and risks.
Once the device is on the US market, patients can be
part of the boots on the ground intelligence system
for real-world effectiveness and
postmarket safety information through
patient-driven registries and other types of
patient-generated data.
Our work to incorporate patient input across the
total product life cycle of medical devices has
been a 10-year journey.
It began with an internal conversation with FDA
staff identifying the need to make patient-centrism
more integral to our work.
It was further informed by dialogue held with the
public around the US through town halls held in
2010 and 2011.
The patient voice was so important that we worked
with industry to include several commitments in the
MDUFA IV agreement to help advance the science of
patient input in the regulatory process.
We also made partnering with patients one of the
Center's strategic priorities of 2016-2017.
With the momentum fueled by the success of the
strategic priority, we created the Patient
Science & Engagement program.
Our program is inspired by patients and driven by
science.
We have the fundamental goal to understand the
patients' perspectives and proactively incorporate
them into all our decisions and regulatory
activities where appropriate.
Central to this overarching goal is
fostering a culture of patient engagement, making
engaging with patients part of our daily business
at CDRH.
Flanking this central tenet are two pillars--
consistent regulatory review that aligns with
our current policies and optimization of the
research roadmap.
Collectively, these efforts streamline the
integration of meaningful patient input in medical
device development and evaluation.
Our program has not only led efforts to meet all
the MDUFA IV commitments but has actively explored
ways to expand beyond the commitments.
This slide provides a snapshot of those
commitments, and the FDA's accomplishments in meeting
and exceeding those goals.
As you can see, our program is making
substantial progress to explore and develop
innovative applications of patient science and
engagement.
Let's first start with some definitions that will
better inform the policies supporting one of our
MDUFA IV commitments of conducting consistent
regulatory review of patient input.
In draft guidance, we have defined patient engagement
as intentional, meaningful interactions with patients
that provide opportunities for mutual learning and
effective collaborations.
Fundamental to this definition is partnership.
Patient engagement is the bedrock that undergirds
the science of patient input.
The science of patient input represents
structured, well-defined collection of how patients
feel and function, as in the case of
patient-reported outcomes; or assessments of the
tradeoffs patients are willing to make among
different benefits and risks associated with
their condition and its diagnosis or management
which is patient preference information.
We have issued guidance documents with
recommendations for how to collect quality patient
preference information as well as how to develop
well-defined patient-reported outcome
instruments.
Consistent with our MDUFA IV commitment, we issued a
draft guidance outlining principles for selecting,
developing, modifying, and adapting patient-reported
outcome instruments in August 2020.
This draft guidance further clarifies least
burdensome approaches to "bridging" also known as
modifying existing instruments and for
developing new instruments in an efficient manner.
The use of patient perspective is clearly
mentioned in many of our benefit-risk guidance
documents as well.
The Medical Device Development Tool program
is another mechanism used to increase the regulatory
predictability and efficiency for the use and
review of patient-reported outcomes.
This program qualifies patient-reported outcome
instruments for a given context of use.
This qualification can streamline medical device
development and evaluation, leading to
greater innovation and reduced resource
expenditures.
Going beyond the commitments made as part
of MDUFA 4, the Medical Device Development Tool
program is another mechanism used to help
minimize uncertainty in the regulatory review
process for the medical device industry.
We have qualified four patient-reported outcome
instruments with others in process.
We continue to see the impact of clear regulatory
policy on medical device submissions.
Currently, there are 23 industry-sponsored patient
preference studies completed or in the
pipeline.
Our staff provide to industry early and
frequent consultations during the design phase of
the patient preference studies when requested
through the pre-submission process.
We also have informal discussions with industry
members who are not sure how patient preference
might fit into their development pipeline.
Some of these completed studies have been used to
inform performance goals for a clinical trial and
support expanding the labeled indications.
In addition, we have consistently seen
patient-reported outcomes included in more than 50%
of premarket approval applications, humanitarian
device exemption applications, and de Novo
submissions.
This data supports the value that industry also
places on collecting the patient perspective.
To facilitate consistency in the review of patient
science information, we have hired experts in
qualitative methods, psychometrics and health
economics.
We have also worked to train our staff in key
principles when reviewing patient science
information.
Our efforts to build capacity are not limited
to our center but extend beyond our walls.
We held a bootcamp to train small businesses and
digital health companies about the ways digital
health technology can be leveraged to measure
clinical outcome assessments like
patient-reported outcomes.
By building capacity internally and externally,
we improve the quality and reliability of patient
science.
Next, I would like to turn to the work we are doing
to foster a culture of patient engagement.
The Patient Engagement Advisory Committee is the
only committee like it at the Agency.
The committee is comprised of diverse patients,
caregivers and patient advocates.
They provide recommendations to FDA
about general matters related to medical
devices.
Patient Engagement Advisory Committee, also
called PEAC, has discussed complex scientific topics
ranging from clinical trials to medical device
safety surveillance and cybersecurity.
The recommendations from these Advisory Committee
meetings lead to actionable outputs.
During the inaugural PEAC meeting, the advisory
committee members recommended that FDA work
to debunk perceived barriers which they
believed prevented industry from engaging
with patients early in the clinical trial process.
This topic was specifically mentioned in
the MDUFA IV commitment letter.
On their recommendation, we issued a draft guidance
that clarified ways of engaging with patients as
advisors in the design and conduct of clinical
studies.
With this draft guidance, we have gone beyond the
discussions to take steps towards encouraging that
engagement with patients.
Our staff have the Patient and Caregiver Connection
as a resource to learn about patients'
experiences living with their specific condition
and interfacing with medical devices as they
are reviewing medical device submissions or
performing other work.
This mechanism allows for timely access to
aggregated patients' voices.
We currently have 15 partners as shown on this
slide and have received more than 3300 responses
from patients since the start of this Connection.
Patients that are part of this connection are also
invited to share their perspectives during public
scientific meetings.
This past February, three patients and one parent
shared their perspectives on innovative technologies
in imaging as well as patient-focused
considerations for use in the home environment.
Attendees remarked how this portion of the
meeting revealed challenges that they had
not previously considered.
Lastly, I would like to share ways in which our
staff have been working to optimize the research
roadmap for patient science.
We are working to create examples as well as
solutions to foster greater use and adoption
of patient science methods and tools.
We are collaborating on many ongoing patient
preference studies spanning a wide range of
priority preference-sensitive
topics.
We announced a list of those patient
preference-sensitive topics in the federal
register in 2019 in accordance with the MDUFA
IV commitment.
However, we have gone beyond listing the
priority areas to begin addressing regulatory
relevant questions for these patient
preference-sensitive topics.
Through these studies, we are seeking to measure how
patients weigh the benefits and risks of
various treatment alternatives for their
conditions.
We are also working to refine existing
patient-reported outcome instruments to bridge the
gap to new populations or use with new technologies.
These efforts help us work towards developing a model
for adapting or modifying existing patient-reported
outcome measures, allowing for an efficient and
streamlined approach to measuring endpoints in
clinical studies.
We are working collaboratively in the
precompetitive space to develop new tools to
measure important concepts for innovative
technologies.
Our efforts also include exploring novel approaches
to leveraging patient-generated data to
inform medical device evaluation.
These studies show our efforts are moving beyond
the MDUFA IV commitment letter.
We are nimbly stretching towards where the
technology is headed.
In our commitment to expand the footprint of
patient science across the entire healthcare
ecosystem, we are holding a number of meetings where
stakeholders including industry, regulators,
academicians, patients and payors can share their
experiences, share case examples, and move the
needle of patient science forward.
Here is a listing of FDA websites where you can get
more information on the topics I have discussed.
If you have additional questions, please contact
us.
In closing, I will summarize the work we have
led in the Patient Science & Engagement Program and
leave you with some key takeaways.
Despite the global challenges with the
COVID-19 public health emergency, the Patient
Science & Engagement program and our CDRH staff
have successfully met and exceeded all of our MDUFA
IV commitments.
Our staff at CDRH have worked to consistently and
transparently apply current policies in
patient science, building capacity both internal and
external to CDRH, optimizing the research
road map to help advance the use of patient science
methods and fill gaps in measuring patient-centric
concepts, and fostering a culture of meaningful
patient engagement.
Our efforts are helping to expand the impact patients
have on the evaluation of medical devices designed
to diagnose and treat them.
As we journey from medical device conception to
delivery of care, we should work together to
continue shifting the paradigm.
CDRH has made significant progress in advancing the
science of patient input, integrating that science
into medical device evaluation, and making
interactions with patients part of our daily business
culture.
We encourage all members of the healthcare
ecosystem to strive to understand the patients'
perspectives and proactively incorporate
them into medical device development and
evaluation.
There are additional opportunities for us to
explore novel sources and uses of patient input such
as patient-generated data.
Let's journey together, working collaboratively
across the healthcare ecosystem to seamlessly
integrate the patients and their voice in all aspects
of health and wellness.
Thank you for watching this presentation.
As we all work to protect and promote public health,
I encourage you to remember the inspirational
words of Thomas Carlyle -- "he who has health, has
hope; and he who has hope, has everything."