“Research into the safety of artificial joints would be BeTTER SAID if patients are involved.”

How Can Patient Partners ImproveJoint Replacement Research?

TomConcannonRAND CorporationTufts School of Medicine

BenNowellGlobal Healthy Living Foundation CreakyJoints

ShilpaVenkatachalamGlobal Healthy Living FoundationCreakyJoints

Welcome!

This project is funded through a Patient-Centered Outcomes Research Institute (PCORI) Eugene Washington PCORI Engagement Award (2228-GHLF).

ChrisStakeArthritisPower Patient GovernorBeTTER SAID

Agenda

What is BeTTER SAID? Welcome – 5 mins

What did we learn? Findings – 15 mins

How can patient partners improve research? Models – 60 mins National Evaluation System for Health Technology (NEST)—Implications for

Arthroplasty Research and Surveillance: Rachael Fleurence, PhD Patient Panel Input into ACR/AAHKS Treatment Guidelines: Susan Goodman, MD

How can you get involved? Patients + Researchers – 5 mins

Bringing STakeholders Together for Engagement in Research for the Selection of Arthroplasty Implant Devices (BeTTER SAID)

What is BeTTER SAID?Bringing STakeholders Together for

Engagement in Research for the Selection of Arthroplasty Implant Devices

We engaged stakeholders in 3 steps

Research Information Decision

Step 1. Interactive webinars to explore patient decisions and information needs prior to surgery

Step 2. In-person roundtables to observe the response of clinicians, health systems leaders, industry to patient decisions and information needs

Step 3. Disseminating webinar to share observations with patient ambassadors and other stakeholders

What did we learn?FINDINGS

Among people who were considering having hip and/or knee replacement surgery…

Percentage who want to be informed about the specific implant device

88%

Percentage who want to hold or see a model of their device

71%

Percentage interested in learning about the risks and benefits associated with surgery

88%

Percentage who want to learn about other existing devices on the market

88%

Among people who have had hip and/or knee replacement surgery…

Percentage who were informed about specific implant device

63%

Percentage who saw or held the device 34%

Percentage who learned about the risks and benefits associated with surgery

55%

Percentage who learned about other devices 18%

Important Decisions – Patient PerspectiveSurgery — Whether to have hip/knee joint replacement surgery (arthroplasty)

Timing — When to have surgery

Surgeon — Which surgeon will perform surgery

Facility — Where surgery will be performed (e.g., geographic location, specific hospital or medical center)

Device — Which implant device will be installed during surgery

Approach — Specific approach to surgery (e.g., anterior vs. posterior, bilateral joint replacement)

Other HCPs — Who/which other health care professionals (HCPs) besides the surgeon will be involved in care during and after surgery (e.g., anesthesiologist, physical therapist)

Other Services — What other services will be necessary before, during or after surgery (e.g., mental health)

Which decisions need more information?

Important Decisions – Patient PerspectiveSurgery — Whether to have hip/knee joint replacement surgery (arthroplasty)

Timing — When to have surgery

Surgeon — Which surgeon will perform surgery

Facility — Where surgery will be performed (e.g., geographic location, specific hospital or medical center)

Device — Which implant device will be installed during surgery

Approach — Specific approach to surgery (e.g., anterior vs. posterior, bilateral joint replacement)

Other HCPs — Who/which other health care professionals (HCPs) besides the surgeon will be involved in care during and after surgery (e.g., anesthesiologist, physical therapist)

Other Services — What other services will be necessary before, during or after surgery (e.g., mental health)

Which decisions need more information?

Providers Patients Payers Purchasers Policy makers Product makers Principal investigators

Who makes the decision about device?

Patients Payers Product makers

Who is affected by the decision?

“7 Ps” framework from Concannon et al JGIM 2012

How can patient partners improve research?

MODELS

National Evaluation System for health Technology (NEST)

Improving evidence generation around medical devices

www.MDIC.org

Rachael L. Fleurence, PhDExecutive director NESTcc

May 17, 2017

Created to support move to better, faster, cheaper evidence generation for medical devices throughout the total product life cycle for patients, clinicians, industry, regulatory agencies and other stakeholders

Mission to: Ensure safety for patients Expedite access to safe and effective medical devices Enable innovation

The National Evaluation System for health Technology (NESTcc)

In September 2016 FDA awarded a $3M grant for the NESTcc to the Medical Device Innovation Consortium (MDIC) with an expected $6M per year for five years from MUDFA

The NESTcc organizationalstructure fits within MDIC’s governance model:

Moving from passive surveillance to active real time surveillance

Leveraging better, faster, cheaper RWE to support regulatory decisions

Making better use of data generated in the course of clinical care or by patients themselves

Moving away from lengthy, one-off, cost-prohibitive studies to an ecosystem that supports more routine evidence generation

NESTcc’s vision is to support a paradigm shift to resolve challenges in our current system

Many sources of Real-World Data can support the paradigm shift but strong methods are essential

Real-World Data (RWD) includes data collected from sources outside of traditional clinical trials and Real-World Evidence (RWE) is the evidence derived from aggregation and analysis of RWD elements

Sources and Types of RWD

Medical Device-Produced Data

Patient Data Tracking

Industry Sponsored Studies and Registries

Adverse Event Reporting (e.g. MDR

Reporting)

Hospital Administrative Data

Clinical Data from Electronic Health

Records

Hospital Billing Data

Logistics Supply Chain Data

Social Security, DEATH Index, Census, Socio Economic Data

Claims Data (CMS and Private Payers)

Public Registries (e.g. NCDR)

Patient Reported Outcomes

Wearable Devices

Disease Risk Assessments

Patient Requested Data

Industry Institutions Public Sources Patients

Safety and Effectiveness

Monitoring

Real-World Evidence Heat MapTo gain a better understanding of the activity of RWE application across the TPLC, a heat map was developed using case studies shared by stakeholders and or collected during research

Disease Area

TPLC Phase

Ideation &Discovery

Invention &Prototpying Pre-Clinical Trial Design Feasibility

Pre-MarketComparativeEffectiveness

De NovoClassification

Cost-Effectiveness

Post-MarketSafety and/orEffectiveness

Post-MarketComparativeEffectiveness

Effectivenessin Subpop.

Applicationsin Patient

Care MethodsAnesthesiology,General Hospital,Respiratory, InfectionControl, and Dental

Cardiovascular

Cross-Cutting

In Vitro Diagnostics

Neurological AndPhysical Medicine

Ophthalmic and Ear,Nose, and Throat

Orthopedics

Reproductive,Gastro-Renal, andUrological

Surgical 3

5

2

2

2

5

3

1

24

1

9

1

5

1

4

1

3

2

2

2

2

26

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2

4

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10

11

Sh t 1

-0.53 26.00

Number of Cases

0 26

RWE Applications Across Total Product Lifecycle

Dis

ease

Are

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The NESTcc will be exploring how to improve evidence generation for medical devices over next year

The developments will be of interest for patients and clinicians, medical device industry, payers, regulators, hospitals

Involvement and support from the patient community in all aspects of the work will be important for success !

Short term goals are (1) putting governance in place, (2) identifying demonstration projects and (3) developing a sustainability plan.

NESTcc: Stay tuned for future developments as we launch !

Learn More about MDIC and NESTVisit our website at: http://mdic.org

The Experience of the ACR/AAHKS PerioperativeMedication Management Guideline Project

Susan M Goodman, MD

Incorporating Input from a Patient Panel into Treatment

Guidelines

ACR-AAHKS Guideline for the Perioperative Management of Anti-rheumatic Medications in Patients with Rheumatic Diseases Undergoing Elective Total Hip or Knee Arthroplasty

Infection is a significant risk for rheumatic disease patients undergoing THA or TKA

Medication management may mitigate riskGuidance is needed for common clinical situations even

where data are sparse

Why involve patients? Patients are major stakeholders

Patients and doctors may have different priorities for outcomes and risks of therapies

Physicians were able to identify their patients’ choices for “most important” physical health outcomes 62%

Taking patients’ preferences into account may lead to increased patient compliance, adherence, and satisfaction

Kwoh AC&R 2001

Perspectives vary

Bartlett ARD 2012

Literature Review team

Expert voting panel

Define:Scope of the ProjectResearch Questions

Core team and content experts(Including 2 patients)

GUIDELINEImplementation

Guideline Development When are patients involved?

Patients reach the same conclusions as physicians

Recommendations can be strong Interventions that improve health Little risk of adverse events Convenient and low cost For patients, almost all would select MDs almost all would recommend

High Quality Evidence

Patients’ values and preferences are particularly important when…..Quality of the evidence is not highTrade-offs between benefits and harms are closely

balanced Conditional recommendations: the majority of

informed patients would follow, others would not Recommendations in these situations may be value and

preference sensitiveThe patients’ perspective may have strong influence

on the direction and strength of the recommendations

How do you incorporate patients into projects?

It is not established how to incorporate patients’ perspectives into projects:Include patients on panels?Will they be drowned out? Feedback: they don’t feel empowered to speak upWill they be representative?

Conduct focus groups?

Values and preferencesElicited to give weight to the balance between benefits and

harms Specifically queried on the relative importance of infections,

compared to the importance of flares Encouraged to consider their personal experiences relevant

to the questions and judge the importance of the outcomes accordingly , using their experience as their evidence base

They strongly and unanimously felt flares, which were common, were much less important than infection

Patients rated the importance of infection as far greater than 10-20:1, despite the relative frequency of flares vs. infection

The patients felt that flares represented a “known risk” that they could control there is no “average” infection – only very bad ones, with a risk of much

worse outcomes than flares the perioperative period is a “job” “dealing with a flare was simply part of the hard work.” “I always assume I will be in a flare when I enter surgery and for a while

when I come out, but I’m afraid of infection”. “I always expect to flare.”

Flare vs. Infection

ConclusionsPatients’ preferences were elicited prior to the

decisions of voting panel, and presented to themGuideline demonstrated clear benefit of patient input

in the presence of relatively weak evidence base in shaping the strength and direction of the decisions made by a physician voting panel

Formally determining values and preferences of patients led to patient-centric recommendations that were congruent for patient and physician panels

Findings of this project support formal incorporation of information elicited from separately convened patient panels into clinical practice guidelines

How can you get involved?

PATIENTS + RESEARCHERS

We do this for two reasons: It’s the right thing to do (i.e., helps us achieve intrinsic goods like

justice, equity, autonomy, self determination, and accountability for public dollars)

It can change research (i.e., make research more relevant, understandable and useful)

To do it the right way: Requires commitment on the part of other stakeholders Requires training of not just patients, but other stakeholders No single model is the “right” model for patient involvement

Patients can be partners in research

Program of Involvement:Guiding Questions1. How do you establish a distinction between studying patients and

involving patients?2. Why do you involve patients? (e.g., what's the ethical and scientific

rationale?)3. Do you train patients before you get started? If so, why/how?4. Does your team need training to work with patients as partners? 5. What are specific research activities in which you seek the

involvement of patients? (e.g., before, during or after, or across all phases?)

6. At what level of intensity do you involve patients? 7. What are the specifics of how you will interact with stakeholder

groups?

How would you involve stakeholders?

Group work TelephoneWebinars Focus Groups

Staff meetings Surveys Technical advisory rolesDocument review and comment Data analysis

Telephone Email InterviewsConsensus building exercises Voting exercises

Individual work Multi-stakeholder meetingsProject leadership Patient only

Before Research During Research After Research

?

Researchers

If you’re a researcher, how can you involve patients as partners to study joint replacement?

Engage with the BeTTER SAID cohort 10+ BeTTER SAID participants are Patient Partners in Research Contact Ben at bnowell@ghlf.org

Partner with patient-powered research networks like www.ArthritisPower.org

Patients

If you’re a patient, how can you get involved?

Become a Patient Partner in Research with CreakyJoints for training and activation

Research 101 session Contact Shilpa at svenky@ghlf.org or (646) 427-7891

Get involved with patient-powered research at www.ArthritisPower.org

Thank you for participatingin this discussion!