Enhancing FDA’s approach toPatient Engagement
Current State Analysis and Recommendations

May 2017

DOC ID

Executive summary Patient input is playing an important and increasing role in the development and regulationof medical products.
 A large number of patient engagement activities are underway in FDA’s medical productcenters.
 With an eye to the future, FDA launched a current-state analysis of its patientengagement activities, which revealed that taking steps like clarifying responsibilities andfostering systems and tools that promote transparency, accessibility, and collaboration
within the Agency and with the public would help strengthen patient engagement efforts.
 A resulting proposed future state for FDA patient engagement activities contains thefollowing high-level objectives: (1) develop a nuanced understanding of the patientperspective of disease and (2) support patients and their advocates in understanding
regulatory processes and in navigating FDA.
 To achieve these objectives, we propose a comprehensive portfolio of patient engagement
activities, supported by the following structure:- A new central staff, the Patient Affairs Staff (PAS) (located in OMPT), would be
responsible for (1) managing inquiry triage and navigation, (2) supporting developmentof tools and services, and (3) coordinating outbound communications to patient
stakeholders. The supporting services currently provided by OHCA must be expanded
and enhanced, and new talents and skillsets are needed as are clear mechanisms foraccountability to FDA centers.

www.fda.gov

1DOC ID

Sources of insights for this effort

Interviews
with internal
stakeholders

Conference
calls withexternalpatient
stakeholders

Patient
engagement
materials
from peer
institutions

www.fda.gov

Peerreviewed
literature

Patient
engagement
activities

Current anddraftedlegislation

2

DOC ID

Contents• Context for this effort• Current state assessment• Future state recommendations

www.fda.gov

3DOC ID

Context for this effort

Patient engagement at FDA traces its roots to FDA’s response to the HIV/AIDS
advocacy movement of the 1980s and 1990s.

Over the past three decades, patient engagement has expanded and evolved toinclude a range of institutional mechanisms for patients to engage with FDA (seenext slide).

Today, FDA is advancing the science of incorporating patient perspectives into
regulatory decision-making and policy development.

It is critical to build a forward-looking, collaborative, and patient-centric
engagement program that will carry us into the next generation.

www.fda.gov Source: FDA website, client materials

4

DOC ID

Evolution of patient engagement at FDA

AS OF DEC 1, 2016

 MDIC releases PPI framework
 CDRH and CBER release PPI guidance
 CDRH announces PEAC FDA Patient Network is launched(first patient web pages)
 PFDD initiative is establishedunder PDUFA V

2016
2015
2013

 FDA establishes
Health Professional
Liaison Program
2008

 FDA patient
representatives
receive voting rights

2006
2001

 HIV/AIDS
patient groupis founded

1996
1991

1988

2012

 MedWatch is
moved to OHCA toencourage
voluntary reporting Patient Representative
Program expands, patients now
serve as consultants toreviewers during review cycle

 HIV/AIDS group expands to include
cancer and other special health issues
 First patient representative sits on FDA
Advisory Committee
www.fda.gov Source: FDA website, client materials

 FDA working
group isestablishedto discuss
FDASIA
section 1137

 FDA-EMA Patient
Engagement
Cluster is founded FDA forms Patient
Engagement
Collaborative with
CTTI

KEY
OHCA = FDA’s Office of Health and Constituent Affairs
PFDD = Patient focused drug development
MDIC = Medical Device Innovation Consortium
PPI = Patient preference information
PEAC = Patient Engagement Advisory Committee
EMA = European Medicines Agency
CTTI = Clinical Trials Transformation Initiative
5

DOC ID

Current congressional requirements for patientengagement at FDAPDUFA V and FDASIA introduced
new requirements for FDA’s patientengagement efforts

AS OF DEC 1, 2016PDUFA VI shifts the focus of patient engagement efforts
while MDUFA IV includes its first patient engagement
provisions• PDUFA V Section X.C: Holds CDER

• Draft of PDUFA VI Section I.J.1: Holds CDER and CBER

and CBER responsible for hosting
20 patient-focused drug
development meetings and outlining
approach for incorporating patient
input into regulatory decision-making

responsible for advancing science of patient input by:– Strengthening staff with expertise in advancing scienceof patient input– Drafting guidances on collecting and interpreting patient
input, including patient information collected throughexternally held PFDD meetings– Maintaining a public catalogue of tools for external
stakeholders– Holding a public workshop on this topic• FDASIA Section of 1137: Requires
earlier and greater patientparticipation in medical productdiscussions“Develop a systematic process
to include patients earlier in drug
development than Advisory
Committee Stage throughconsultation with scientificreview divisions”• Draft of MDUFA IV Section IV.F: Holds CDRH responsible

www.fda.gov Source: Published legislation, draft legislation

for advancing science of patient input by:– Strengthening staff with expertise required for
responding to applications with PRO or PPI materials– Holding public meetings on this topic
PRO = Patient reported outcomes6

DOC ID

Patient engagement expectations held by patients and industry
Direct quotes from patients / their advocatesPress clippings regarding industry players“The Centers are all doing good, exciting
work relating to patient engagement…
now we’d like to see a movement towards
coordination”“[It will be important] to get agreement with the FDA
on how to validate these patient informed endpoints
and how to move forward with labeling, and I'm notconvinced the FDA actually knows the answer”- Vas Narasimhan, Global Head of Development forNovartis Pharmaceuticals“Soon the Agency will have to provide
guidance to industry. Companies worry
that premarket interactions will be a COI
issue, but including patients early indevelopment is key”

“Sanofi Appoints Dr. Anne C. Beal to the Newly
Created Position of Chief Patient Officer”- Globe Newswire, March 2014

COI = Conflict of interest“PDUFA VI changes the game. We’reeager to know how FDA plans ongathering actionable patient voice
information now that PFDD meetingsaren’t internal”“The new Advisory Committee could
signal a new, increased role for thepatient. That would be exciting”“[Pharmaceutical] companies say the agency needs
to more clearly define the standards they require forpatient-focused outcomes to become a true measureof a drug’s success”-IB Times, September 2015

“We need a sweet spot [on patient input], and it
hasn't been found. And the FDA has not done agood job of finding that sweet spot”

www.fda.gov Source: Conference calls with patient stakeholders, published reports- Diana Zuckerman, President of the National
Center for Health Research
7

DOC ID

Contents• Context for this effort• Current state assessment• Future state recommendations

www.fda.gov

8

DOC ID

Patient engagement at FDA today “by the numbers”

24
Patient Focused
Drug Development
meetings sponsored
Agency-wide

350,000
Recipients on
MedWatch listserv

96%
Adv Committees
with patient reps

63%
CDRH staff
engageddirectly withpatients in
2016

www.fda.gov Source: Client interviews, Center-provided data

NOT EXHAUSTIVE

Relationships
with more than500

patient advocacy
groups201
patient reps for300
conditions
and diseases

9DOC ID

Current patient engagement activities: OHCA
Engagement coordination

Inventory of activity• Liaise with patient and HCP groups to keep communities
Host & attend
meetings•

Strategy:• Serve as central coordinating

patients, HCPs and advocacy groups
Coordinate Commissioner listening sessions• Coordinate and support expanded access programs• Serve as one of the initial points of contact for individual• Provide point of contact forpatients and their advocates andtools and services for patientengagement across centers

informed, jointly solve problems and shape policy• Facilitate 400-500 ad hoc meetings per year with individual

Respondto requests

office for patient and HCP
engagement across FDA (e.g.,
Patient Council)
Outboundcommunication•

patients, patient advocacy groups and HCPs
Triage and respond to ad hoc patient inquiries on wide
variety of issues (e.g., answer questions about regulatory
process, navigate clinicaltrials.gov and serve as empathetic
sounding board)• Manage MedWatch security alerts• Manage FDA Patient Network (i.e., website, bi-weekly
newsletters, webinars, and Twitter feed)• Identify issues and concerns for patient and provider groups
Solicit targeted
input

to drive Agency communication strategy• Solicit comments on FDA documents (e.g., guidance
documents, regulations, requests for input)

Informregulatory
decisions• Manage Patient Representative Program (recruited andtrained ~200 patient reps since 2010)• Lead Agency participation in EMA-FDA Patient Engagement
Cluster

OHCA also responsible for delivering patient-facing FDA messaging as part of broader OEA mandatewww.fda.gov Source: Client interviews, Center-provided data

OHCA = Office of Health and Constituent Affairs

10DOC ID

Current patient engagement activities: CDER
Engagement coordination

Inventory of activity• Coordinated 24 patient focused drug development meetings
Strategy:

Host & attend
meetings•• Promote awareness of regulatory
processes and resources within
CDER amongst patients, theiradvocates and HCPs throughoutreach and educational
programming

Respondto requests• Increase accessibility of CDER
through creation of a dedicatedoffice with low threshold forfacilitating a meeting

Outboundcommunication(OSP = Office of Strategic
Programs)• Respond directly or set up meetings with relevant CDER
stakeholders in response to specific patient requests (PASE)• Process expanded access requests• Maintain Drug Trial Snapshots website, which presents••(PASE = Professional Affairs andStakeholder Engagement )Solicit targeted
input
Informregulatory
decisionswww.fda.gov Source: Client interviews, Center-provided data

aimed at systematically gathering patient perspectives on
their condition (OSP)
Participate in 30-40 ad hoc meetings per year with individual
patient groups to discuss disease and treatment related
issues (PASE)

demographic data on Office of New Drug clinical trials(PASE)
Host Roadmap for Engagement workshop to orient patientadvocacy groups on how regulatory decisions are made andhow they can best engage CDER (PASE)
Lead Safe Use Initiative to combat medication misuse anderrors (PASE)• Convene patient advocacy group / HCP meetings on
specific topics on behalf of offices / divisions (PASE)• Publish Voice of the Patient reports as resource forreviewers (OSP)• Include patient reps as voting members in most Adv Comms• Participate in EMA-FDA Patient Engagement Cluster
CDER = Center for Drug Evaluation and Research

11DOC ID

Current patient engagement activities: CBEREngagement coordination

Inventory of activity
sorr patietien
nt focu
cuse
sed
d drug develop
lopme
men
nt (PFD(PFDD)D) meetintinggs• Sponso(prim(primary sp
spo
onso
sorr of 3 m
me
eetin
ting
gs; parti
rticipa
cipatiotion
n in oth
the
ers)

tien
nts and patietien
nt grouroup
ps on Rare Di
Disesea
ase Day (i(inn• Host patie
Strategy:• Participate in cross-Center

Host & a
Hos
att
tte
end
mee
me
etingsprograms led by the OC andother centers•• Allow individual offices anddivisions to drive engagement on
specific topics of interest(collaborating across offices
through virtual working group)••

Respond to
Respond
re
requ
ques
ests

OutboundOutboundcommunic
ommunica
ation

Solicit targeted
input

Informregulatory
decisionswww.fda.gov Source: Client interviews, Center-provided data

colla
coll
abora
ratio
tion
n with CD
CDE
ER)
Parti
rticipa
cipatete in Com
Comm
missi
issio
oner liliste
stenning sesessionssionssFacil
Facilitaitate
te ~10-20 ad hoc meetinting
gs per year with
with in
ind
div
ividu
idua
al
patie
tien
nt grouroup
ps about disea
iseasese and tre
trea
atm
tme
ent relarelateted
d issueissuess
Rare disea
isease
se co
coo
ordi
rdin
natin
ting
g co
com
mmitte
ittee
e att
tte
ends NORD to
inte
interact with p
pa
atie
tien
nts and advoca
cacy
cy grouroup
ps each year

Respo
ond to ind
indiv
ividu
idua
al meetingting req
requuests at lev
leve
el of
of offices• Resp

and
an
d div
divis
ision
ions
s

rocessss expanded acce
ccess
ss reqrequ
uests• Proce
Distristrib
bute ta
targ
rge
ete
ted
d co
com
mmunica
icatiotion
ns on sp
spe
ecif
cific
ic to
top
pics of
• Di

•
•
•

interest or con
inte
conce
cern
rn (e.g(e.g.,., ttooppa
aren
rentt grou
roup
ps about vaccine
ccines)s)
Publilish
sh demograp
raph
hic infinfo
orma
rmatiotionn for cliclin
nical tritria
als onon CBE
CBER
R
websiteParticipate in Agency-wide calls for input
Engage individual special govt. employees for feedback on
specific patient communications• Publish Voice of the Patient reports on PFDD meetings• Issue guidance (e.g., on Factors to Consider When Making••

Benefit-Risk Determinations) for industry and FDA staff(August 2016, in collaboration with CDRH)
Include patient reps as voting members in most Adv Comms
Participate in EMA-FDA Patient Engagement Cluster

CBER = Center for Biologics Evaluation and Research

12DOC ID

Current patient engagement activities: CDRH
Engagement coordination

Inventory of activity
Host & attend
meetings

Strategy:• Partnering with patients is one ofthree 2016-2017 strategic
priorities with two defined goals:– Promote culture ofmeaningful patientengagement– Increase use andtransparency of patient input
in decision-making

Respondto requests
Outboundcommunication• Hold Town Hall Sessions with invited patient speakers• Host public workshops about patient-focused topics• Process compassionate use requests• Publish patient-facing materials on website, Twitter feed,
and mailing lists• Establish mechanisms for CDRH staff to engage directly
Solicit targeted
input

•

with patients to obtain patient input on pre- and postmarket
issues (partnered with 30+ advocacy organizations; >60%
staff participated in 2016)
Chartered Patient Engagement Advisory Committee (PEAC)
to obtain patient input on variety of patient-related issues(first meeting anticipated in first half 2017)• Developed Patient-Centered Benefit-Risk Framework and•Informregulatory
decisions•catalog of assessment methods in partnership with MDIC
Issue guidance (e.g., on Factors to Consider When Making
Benefit-Risk Determinations) for industry and FDA staff(August 2016, in collaboration with CBER)
Included patient perspective data in 50% of PMA, de novo
and HDE decisions in 2016 (target of 100% in 2017)• Include patient reps on most Adv Comms• Participate in EMA-FDA Patient Engagement Cluster

www.fda.gov Source: Client interviews, Center-provided data

CDRH = Center for Devices and Radiological Health13

DOC ID

Assessment of current state: Strengths
Internal stakeholder

Area
Conviction andcommitment

Problem solving
with patients

Description

What we heard• Centers perform meaningful patient engagement“People at the FDA are very committed,
they are willing to bend over backwards
when it comes to patient communities”

work, and FDA staff are committed to working withpatients• FDA has a variety of mechanisms for interacting•with patients about their specific needs
FDA staff work flexibly to solve patient issues• FDA leaders agree that patient engagement is an
Leadershipalignment

important priority both for soliciting patient input into
regulatory policy decisions and as part of the
Agency’s public service mandate• Patient engagement staff within centers haveConnectivity
within centers

Understanding
patient
perspectives

relationships and visibility across divisions to
support productive exchanges with patients• Patient engagement efforts have given FDA real
insight into clinically meaningful patient outcomes
and how the disease process affects patients andtheir caregivers• Innovative, forward-looking patient engagement
Innovative
approaches

External stakeholder

experiments are occurring throughout the Agency(e.g., Roadmap for Engagement workshop, benefitrisk frameworks and assessment methods)

www.fda.gov Source: Interviews with FDA staff, conference calls with patient stakeholders“FDA is very helpful in guiding us to thepeople who can help solve our problems”“The question is when and how to engage
patients, not whether or not to do it”“We have a duty as public servants to
respond to inquiries from our constituents”“When a patient group approaches us in
the center, we know who best to include in
the discussion”“These forums have given us key, andoften surprising, insight into what isclinically meaningful for patients”“Advancing both the art and the science of
patient engagement is critical to our
mission of better incorporating patientperspectives into our decisions”
14

DOC ID

Assessment of current state: Opportunities (1/2)
Internal stakeholder

Area
Strategic intent ofpatient
engagement

Description

What we heard• FDA lacks widely understood and clearly defined“FDA is lacking a strategy or operational
plan for engaging with patients, HCPs, andthe general public"

objectives to guide patient engagement activity
across centers; and the degree of strategic clarity
varies from center to center• Limited cross-Agency coordination to share bestpractices, jointly address shared policy questions,and present a single FDA voice to patientcommunities portrays a disjointed image of the
Agency to the public

Cross-Agency
coordination• FDA currently depends on personal relationships /lacks a central repository for patient engagement
information, impeding information-sharing acrossthe Agency and challenging continued progress

Institutionalization• Lack of a single point of contact for patients or
triage system for routing inquiries impedes bothpatients and Agency staff from rapidly accessing
the right resources

Improved accessto FDA• Processes for communicating the outcomes of FDA
Feedback loop topatients /advocates

External stakeholder•

touchpoints are inconsistent and often lacking
Lack of transparency to patients and theiradvocates about how patient voice has been
incorporated into Agency decisions and policies
causes frustrationwww.fda.gov Source: Interviews with FDA staff, conference calls with patient stakeholders“The centers are doing good work, but notcoordinating at all, to our knowledge”"Centers do not always work collaboratively
on cross-cutting issues"“A lot depends on who you know, so when
there’s turnover, you have to ask, ‘is that
information gone now?’”“A central entry point to the FDA would betremendously helpful, so long as preexisting relationships could continue”“When clear expectations are notcommunicated to patients, there is thedanger of setting false expectations”“The lack of transparency is frustrating"15

DOC ID

Assessment of current state: Opportunities (2/2)
Internal stakeholder

Area
Approach toincorporating
patient input

Description

What we heard• Mechanisms for incorporating patient input into

“Once patient information is gathered, whatis the mechanism for incorporating it into
regulatory work across the Agency?”

decision-making are variable (sometimes
appropriately) and often lacking• Approach to engagement is largely reactive, driven
largely by inbound requests or external mandates,
rather than deliberate choice

Proactive posture• Existing tools and processes within patient engagePerformance

Clarification of
mission•ment functions are often inefficient or outdated
Lack of connectivity between centers and OHCA
results in services that do not meet center needs• Lack of delineation between media communication
activities and patient engagement efforts limits thecredibility of the Agency as a patient partner• Absence of patient representatives prior to Adv
Additional forums
for patient voice•Comms (e.g., in EOP1, EOP2) limits patient impact
on drug development
Lack of clarity on acceptable interactions between
sponsors and patients discourages engagement• Absence of clearly defined metrics and mechanisms
Appropriate
metrics

External stakeholder

to review performance prevents Agency-wide
evaluation of patient engagement effectiveness andidentification of improvement opportunities

www.fda.gov Source: Interviews with FDA staff, conference calls with patient stakeholders“Our approach to engagement is highly
reactive, and underserved groups haveless voice than perhaps they should”“There is opportunity to professionalize
some of our tools for patient engagement
to make the experience better for bothpatients and FDA”
“There’s a danger of patients feeling likethe FDA is just paying lip service”“Industry worries premarket interactions will
be a conflict of interest issue, but including
patients early in development is key”

“I’m not sure we measure any of our patientengagement activities today”

16

DOC ID

Feedback from patient advocacy group conference calls

Strengths

Area
Conviction
andcommitment
Problemsolving withpatients

Description

What we heard• Centers perform meaningful patient engagement“People at the FDA are really committed,
they are willing to bend over backwards
when it comes to patient communities”

work and FDA staff is enthusiastically committed toworking with patients• Centers have a variety of mechanisms for•interacting with patients and their specific needs
FDA staff works flexibly to solve patient issues• Cross-Agency coordination would build on valuable

Opportunities

Cross-Center
coordination
Communication
mechanisms
Institutionalization

Transparent
follow-up

ongoing work by encouraging centers to identify bestpractices, jointly address pressing policy issues andpresent a single FDA voice to patient communities• A central FDA entry point / triage system for inquiries
would be helpful (particularly for individual patients),
so long as pre-existing relationships can continue• Institutionalization of patient engagement efforts would
ensure continued progress in a way not guaranteed by
FDA’s current dependence on personal relationships• Transparency in communicating the outcomes ofpatient interactions so that impact is known would
reduce frustration and guide future patient efforts• Inclusion of patient reps prior to Ad Board (e.g., EOP1,
Forums forpatient voice

EOP2) would allow earlier input into development• Clarity on acceptable interaction between sponsor andpatients would encourage use of patient perspective

www.fda.gov“FDA is very helpful in guiding us to thepeople who can help solve our problems”“The centers are doing good work, but
not coordinating at all, to our knowledge.
It would be better to work in tandem”“There are far too many places to go,but, at the same time, sometimes even I
could benefit from an operator”“A lot depends on who you know, sowhen there’s turnover, you have to ask,
‘Is that information gone now?’”“The lack of transparency is frustrating.
Ten years later, if a guidance emerges,
does that mean they were listening?”“Industry worries premarket interactions
will be a conflict of interest issue, but
including patients early in development iskey”
17DOC ID

Contents• Context for this effort• Current state assessment• Future state recommendations

www.fda.gov

18

DOC ID

Core components of a robust patient engagement program

Center-led strategic guidance and evaluation
frameworks that direct patient engagement
efforts

Direction

Support &coordination

Designwww.fda.gov

Systems and processes that support
the coordinated delivery of patientengagement efforts across FDA

Design and delivery of center patientengagement activities andincorporation of patient perspectives
into FDA work

19

DOC ID

Guiding principles for the Patient Affairs Staff (PAS)

CulturePAS is guided by a strong sense of responsibility for supporting centers’
patient engagement work and serving patients / their advocates

Talent & skills

PAS is staffed by talent whose background and skillset are in line withthe office’s work activities / mission and can drive the office forward

Workforce size

PAS is supported by a lean staff model that drives value tothe centers without placing undue burden on resources

Informal networks

www.fda.gov

PAS is an active member of FDA’s medical product community
such that informal networks can develop alongside formal ones

20

DOC ID

Proposed PAS organizational profile
Office of Medical ProductsHigh levelstructureCore
responsibilities

Requiredskillset

Director, PAS

Navigation

Shared database

Communication• Triage and navigation for
inbound patient inquiries• Maintenance of a shared
database and calendar ofpatient engagement
information / events• Management of patientcommunication platform• Resolution of non-center
specific inquiries• Knowledge of FDA policy, • Technical capabilities toprocesses and resources
deploy and maintain CRM
tools• Connectivity with centers• Health and/or advocacy
background• Understanding of“customer” needs (boththose of PAS and centers)• Experience with web
platforms, digital
broadcast and social
media• Communications and/or
editorial skills• Director of PAS must have familiarity with the Agency and possess theleadership qualities required to effectively work across centers and offices
www.fda.gov

21

DOC ID

Potential impact of PAS
Development and communication of a shared mission and vision for patientengagement to clarify expectations for patients and provide high-level framework
for center and office programs



Structured mechanisms for internal communication and collaboration to
reduce duplication of effort, improve coordination, and facilitate cross-pollinationof ideas and innovation

A single point of entry for patients / their advocates and resources for theeffective triage and navigation of inquiries to level the playing field for lessexperienced groups and improve access to FDA overallAn improved set of scalable and sharable tools to institutionalize and efficiently
coordinate patient engagement efforts at FDA

Advancement of the science for integrating patient voice into the regulatory
process to better enable patient perspectives to shape product development andapproval

Identification and monitoring of key indicators of success for FDA’s patientengagement program to enable continuous improvement and greater impactTools and services for patient engagement that can support HCP needs where
there are clear operational synergies

www.fda.gov

22