patient and citizen engagement in hta decision making...model of current patient input in hta...

Patient and Citizen Engagement in HTA Decision Making

E4 Panel session,

CADTH Symposium,

May7th, 2013

1

Agenda• Moderation

o Paul Oh

• Patient perspective

o Durhane Wong-Reiger

• Citizen perspective

o Janet Parsons and The Ontario Citizen‟s Council

• Decision maker/ HTA Body perspectives

o CDR - Elaine MacPhail

o pCODR - Mona Sabharwal

• Discussion (All)

“Nothing About Me, Without Me”

Questions for Reflection and Response

• What is currently being done around patient

and citizen engagement in HTA?

• What has been the impact of patient and

citizen engagement in HTA?

• What does “good” or “better” look like?

• How can Canada become a leader in this

area?

Influence and Decision Making

fadedvideolabels.blogspot.com

Twelve Angry Men

Part 1:

What is currently being done around patient and citizen engagement in HTA?

ADVOCARE

Durhane Wong-Rieger, PhD

Chair, Consumer Advocare Network

Thanks to Janssen for some of the slides and analysis

What are “Best” Outcomes in Healthcare?

• Defined as attaining best individual health

possible and optimal health for population

• Individual makes healthcare choices that lead

to best personal outcomes, based on personal

suitability and shared social values

• System provides healthcare options that allow

all patients to get right care, at right time with

optimal use of healthcare and social

resources

8

How can system Allocate Resources to Achieve “Best” Outcomes?

• Allocation based on shared social values

(necessary, equitable, accessible,

affordable, compassionate)

• Allocation defined based on achieving

optimal individual needs (personal good)

• Allocation to optimize population impact

(accountable, appropriate, sustainable)

9

How does Patient Engagement Improve Health Outcomes?

• For self, patients choose optimal healthcare: make evidence-based cost-effective choice;, adhere to care plan; engage in self-management; give feedback

• For healthcare system, patients help decide care options: identify patient outcomes, define patient values, use assessment and decision-making framework

• For funders, patients take part in allocation of resources: define societal values, develop assessment and decision-making framework, evaluate outcomes

10

Model of Current Patient Input in HTAConsultee, Informant

Input thru Council, Task Force, Collect Info: Survey, Poll, Focus Group

Form of Info: Answers, Opinions, Deliberation

Impact: Advise, DiscretionaryExamples: NICE Citizens Council, IQWiG, Ontario Citizens Council

Patient/Public Member

Input thru Committee, Board, CouncilCollect Info: Experts, Deliberation

Form of Info: Analytical, GuidelinesImpact: Varied, Based on GuidelinesExamples: NICE, AU MASC, CEDAC,

pERC Ontario CED

Individual Patients

Input thru Clinical Trials, TestimonyCollect Info: QoL, PROs, Impact

StatementForm of Info: Ratings, Qualitative Impact: Varied, Emotional Suasion

Examples: SMC, IQWiGQuebec conseil, BC Pharmacare,

Patient Groups

Input: SubmissionCollect Info: Written, Oral, MeetingsForm of Info: Qualitative Statement

Degree of Impact: ResponseExamples: NICE, SMC AU MASC,

CADTH, pCODR, Ontario CED

Patient Representativeness 11

Citizen engagement in HTA:A Brokered Dialogue with the

Ontario Citizens’ Council

Janet Parsons, PhDJim Lavery, PhD

Advisory body to the Executive Officer of the Ontario Public Drug Programs

Provides advice on the values that reflect the needs, culture and attitudes of Ontario’s citizens about government drug policy


http://www.health.gov.on.ca/en/public/programs/drugs/councils/

13

http://www.health.gov.on.ca/en/public/programs/drugs/councils/

CDR Patient and Public Involvement

Elaine MacPhail,

CADTH

15

CADTH patient and public involvement Launched in May 2010

Seeks input via Canadian patient groups

Uses template, guide, online submission form

Respects existing CDR timelines

Incorporates patient input systematically

throughout drug review and

recommendation-making process

Must meet the needs of patients, reviewers

and CDEC

Patient Input

Process

Public members on: Canadian Drug Expert

Committee & Health Technology Expert Review

Panel with full voting rights

16

How patient input is used

Patient input collated, summarized, used to inform protocol & included

review report

Patient input presented, used in deliberations & reflected in

recommendations

CADTH ReviewTeam

Expert Committee

Drug PlansPatient input

shared

Patient Input SubmissionCanadian Patient Groups

Reasons & recommendations

published

Original input & summary sent to

CDEC

Patient input

shared

Patient input

shared

Patient input

shared

17

Patient Engagement in pCODR

Mona Sabharwal

18

Applied Lessons in Patient Engagement

• Must be imbedded in organizational culture• one of pCODR‟s Guiding Principles (Collaborative and

Representative) and part of pERC‟s Deliberative Framework

• Begin at the beginning• engagement of stakeholders front and centre since Day 1

• Genuine consultation and interaction, based

on best practices of HTA agencies, nationally

and internationally• inclusive, responsive, transparent follow-up actions (even if it

means not being able to do something)

• Take “ownership” of what you can • on-going investment in providing information and education,

provide guidance but respect who owns specific steps/issues

© 2013 pan-Canadian Oncology Drug Review19

pCODR Review Process

Updated March 31, 2011

1. Conduct Pre-

Submission Planning activities including

getting input from PAG and notifying Patient

Advocacy Groups

2. Prepare & submit

Request for Drug

Review

4.2Conduct

Economic Review

5. Summarize & Review

with pERC

6. Prepare & Publicly

Post Initial Recomm,

Post Reviews

8. Summarize & Review

with pERC

3.1 Screen Submission and Initiate

Review Process

End‡

Ind

us

try/

Tu

mo

ur

Gro

up

pC

OD

R*

Variable 5 business days 70-90 business days 12 business days 10 business days 20 business days

7.1 Get Feedback

from Submitter

(and impacted manufacturer)

7.3 Get Feedback from

Patient Advocacy

Group

7.2 Get Feedback from PAG

Pati

en

tA

dv

ocac

y

Gro

up

s

9. Prepare & Publicly Post

Final Recomm & Post Input

12 business days

*Includes pCODR Secretariat, Clinical

Guidance Panel, Economic Guidance Panel,

pCODR Expert Review Committee (pERC)

and Provincial Advisory Group (PAG)

4.1.1/4.2.2Clarify info

with Submitter

during review

4.1Conduct Clinical Review

3.2 Collect Patient

Advocacy Group Input

Estimated

99 – 149

business days

7.4

Eligible for

Early

Conversion?

No Yes

‡Next steps could include

Recommendation implementation,

Procedural Review or Resubmission

Direct impact of patient

perspective

Indirect impact of patient

perspective

© 2013 pan-Canadian Oncology Drug Review

pERC Patient Members: 2 voting, 1 alternate

Selection criteria/process:

• Demonstration of personal knowledge of, experience with and

understanding of issues related to cancer and its management

(among other qualifications)

• Broad advertising, independent 3rd party to screen applicants, diverse

panel for decision making

Training:

• On-line training modules, orientation, on-going educational supports

• All members forward suggestions anonymously for specific training

topics, demonstrating lots of overlap in needs

Committee participation:

• Present materials, deliberate and vote at all meetings

• Participate in working groups, regardless of topic

• Present at conferences upon invitation and as available

21© 2013 pan-Canadian Oncology Drug Review

Detailed Description of Each Element of the pERCDeliberative Framework (1)

22

Criteria Sub-Criteria Sub-Criteria Definitions

Overall Clinical Benefit Effectiveness

(systematic review in the Clinical Guidance

Report)

The potential health impact of the

drug compared to the other drug and

non-drug alternatives, measured in

terms of relevant patient outcomes

such as mortality, morbidity, quality

of life. Magnitude, direction and

uncertainty of effect should be

considered.

Safety

(systematic review in the Clinical Guidance

Report)

Frequency and severity of adverse

effects associate with the new drug

compared to other drug and non-drug

alternatives.

Burden of Illness

(Clinical Guidance Report, patient advocacy group

input)

Incidence, prevalence or other

measure of disease burden on the

population.

Need

(Clinical Guidance Report, patient advocacy group

input)

Availability of an effective alternative

to the drug technology.

© 2013 pan-Canadian Oncology Drug Review

Detailed Description of Each Element of the pERCDeliberative Framework (2)

Criteria Sub-Criteria Sub-Criteria Definitions

Alignment with Patient

Values

Patient Values

(patient advocacy group input)

Patient based values which bear on the

appropriate use and impact of the drug.

Cost effectiveness Economic Evaluations

(Economic Guidance Report and pharmaco-

economic model review)

A measure of the net cost or efficiency

of the drug and companion technology

compared to other drug and non-drug

alternatives. The uncertainty of results

should be considered.

Feasibility of Adoption into

Health Systems

Economic Feasibility (evaluation of budget

impact assessment in Economic Guidance

Report)

The net budget impact of the new drug

on other drug and health system

spending, including companion testing

technology.

Organizational Feasibility

(Provincial Advisory Group input)

The ease with which the new drug can

be adopted, with an assessment of

health system enablers and barriers to

implementation, inclusive of all

elements: operational, capital, human

resources, legislative and regulatory

requirements

Part 2:

What has been the impact of patient and citizen engagement in HTA?

ADVOCARE



CDR Recommendations (2011-12 w/Patient Submissions)

N=58 Total With Patient Submission

No Patient Submission

Overall100% (58) 72% (42/58) 28% (16/58)

List12% (7/58) 71% (5/7) 29% (2/7)

List Conditional45% (26/58) 85% (22/26) 15% (4/26)

Not List43% (25/58 ) 60% (15/25) 40% (10/25)

26

pCODR Recommendations (2012 w/Patient Submissions)

N=8 Total With Patient Submission

No Patient Submission

Overall 100% (8) 100% (8/8) 0% (0/8)

List 0% (0/8) 0% (0/8) 0% (0/8)

Not List 13% (1/8 ) 100% (1/1) 0% (0/1)

List Conditional 88% (7/8) 100% (7/7) 0% (0/7)

27

CDR/pCODR (2011-2012)Reasons for Recommendations

List(n = 4)

List Conditional

(n = 22)

Not List(n = 16)

Clinical Trial Outcomes(effectiveness, sufficient,similar, certain)

100% 91% 88%

Safety (adverse effects, similar risks, certain)

25% 28% 31%

Cost-effectiveness (more, less, certain)

100% 87% 56%

Quality of Life (studied, better, specific)

25% 59% 44%

Patient submissions (reference, substantiated

0% 82% 63%28

Impact of Patient Submissions: CDR/pCODRRecommendations

• Patient submissions in 72% of CDR and 100% of pCODR

cases

• Average # submissions: 2.2 CDR and 1.3 pCODR per case

• CT outcomes (effectiveness, comparison to standard,

certainty) most important and frequently cited reason for

recommendation

• Cost-effectiveness (more costly, uncertain CE analysis,

$/QALY) cited in all “list”; most “list conditional”; half of

“do not list

• Safety (adverse effects, uncertain risks) cited in 30% of

cases

29

Impact of Patient Submissions: CDR/pCODRRecommendations

• Patient submissions always discussed; factor in

almost all recommendations to “list

conditional”; three-fifths of “not to list” and not

cited in “to list” recommendations.

• Patient submissions discounted when factors

not included in CTs or not substantiated by

submitted QOL measures; request for “balance

of positive and negative” opinions.

30

Patient/Public Member Impact on HTA

Feedback from Patient/Public Members

• Patient and Public Members believe their participation is valuable but neither equal nor impactful

• Patient submissions gives added credibility to patient/public role; have something to contribute

• Other HTA members not always receptive to patient submissions; qualitative information difficult to integrate; some would prefer quantitative but not sure what would be included

• Patient submissions have little impact on discussion or decision; however, do increase understanding of rare or unusual conditions

• Patient relations with pharmaceutical companies, even if declared, raises question of conflict of interest and diminution of input

• Patient representatives in meetings would increase understanding of the patient submission

Purported and Real Impact of Patient Engagement in HTA

Looks Like Really Happens

Patient perceived outcomes builtinto clinical trials (QOL, PROs)

Scales unable to capture patient experience; no long-term impact

Patient submissions to HTA give qualitative experience; own words; collective group input

No process to integrate patient submissions into quantitative approach; ignore, discount

Patient or public members on HTA bodies assure perspective of patient or society at table

Patient/public member roles poorly defined; lack technical expertise; public ≠ patient

Multi-stakeholder forums, Citizens Councils, consultations are opportunities for input

Most public/patients unaware of processes; technical oracademic

Plain language reports increase patient understanding and acceptance of HTA outcomes

Patients want full access;don’t trust unless present:want right to appeal

What is the impact of citizen engagement?

Overall, OCC is a success in the making: reasons

Nevertheless, important concerns identified:

• The mandate of the OCC is vague

• As a consequence, challenging to assess overall

impact

• Participants are activated to participate in what they see as

important opportunity for political participationo An opportunity to speak directly to government

o Profound experience of engagement

• If their contributions are not recognized/acknowledged, will

lead to disappointment/disillusionment

What is the impact of citizen engagement?

The Evaluation Challenge in Citizen Engagement:

• What impacts should we expect from such initiatives?

• Narrow views of impact are insufficient

• Which outcomes and processes matter and why?

• Making room for unanticipated outcomes

• Recognition that a multiplicity of potential impacts should be assessed:

o Not only on council members (e.g. degree to which their expectations are met)

o Decision-makers (e.g. how are CC recommendations incorporated into decisions by expert committees)

o Broader public (e.g. level of awareness of CC activities, increased understanding of how decisions are made)

What is the impact of this kind of citizen engagement?

What impact(s) of citizen engagement should be expected?


Elaine MacPhail,

CADTH

38

Impact of patient input on decision-making

Informs formulary listing recommendations

Identifies outcomes and issues about condition and

its treatment of importance to patients

Identifies unmet needs

Describes expectations for the new product

Reflected in Final Recommendation document

Results in more relevant recommendation

Identifies where there is no evidence to support the

outcomes or issues of importance to patients

Need better tools to measure impact

Patient groups willing to contribute

74% submissions receive

patient inputAv. 2 patient groups

provide input per drug

Data from May 2010 to April 30 2013

75 different groups

across Canada have

contributed


Mona Sabharwal

41

Patient input being used in pCODR reviews

42

Patient Advocacy Group Engagement - on pCODR Reviews with pERCFinal Recommendations (as of Dec. 31, 2012)

Generic Name / Brand

Name

Cancer Type Type Patient

Input

Patient

Feedback

Pazopanib (Votrient) Metastatic Renal Cell Carcinoma 1 1

Ipilimumab (Yervoy) Advanced Melanoma 2 1

Sunitinib (Sutent) Pancreatic Neuroendocrine Tumours 1 0

Vemurafenib (Zelboraf) Advanced Melanoma 1 1

Eribulin (Halaven) Metastatic Breast Cancer 2 0

Everolimus (Afinitor) Pancreatic Neuroendocrine Tumours 1 0

Crizotinib (Xalkori) Advanced Non-Small Cell Lung Cancer 1 1

Bendamustine (Treanda) Non-Hodgkins Lymphoma (NHL) 2 1

Bendamustine (Treanda) Chronic Lymphomatic Leukemia

(relapsed/refractory)

2 1

Pazopanib (Votrient) Soft Tissue Sarcoma 1 0

Total (%) 14 (100%) 6 /14 (43%)

•43© 2013 pan-Canadian Oncology Drug Review

Providing Feedback on an Initial Recommendation

• Contributing parties can share their interpretations and

perspective on a pERC recommendation

• pERC reviews feedback to redeliberate and reconsider, if

appropriate, the initial recommendation

• Brings greater transparency to process, improving legitimacy

and confidence that pERC really has chance to consider

“everything”

Do you agree with the Initial Recommendation – why or why not?

Did we interpret your input accurately?

Is there contextual information about the patient experience which needs to be

re-considered?

Is there something we could have worded more clearly?

Part 3:

What does “good” or “better”

look like?

ADVOCARE



Are Patients/carers/public happier with NICE?

• Lack of research evidence on patient/carer

views, experiences and preferences

• Quality of life measures often don‟t reflect

issues of most importance to patients

• Weighting on evidence from patients

• Process doesn‟t take account wider societal costs

• Technical language and economics are difficult to

engage with/challenge

• NICE „blight‟/ variability in access to NICE

recommended technologies

*from Victoria Thomas (NICE)

47

http://www.biblepicturegallery.com/Pictures/Lifestyle/Man with glasses saying NO ca.htm

Recommendations for Patient Engagement with HTA

o Dialogue with patient groups to define submission: what information, how to collect, how to present

o Engage patients in CT design to ensure patient values

included in measures

o Train potential HTA patient-public members on technical processes of HTA and decision making

o Provide means for patient-public members of HTA

committees to dialogue with patient representatives

o Train all HTA committee members on methods for integrating qualitative information

o Promote transparent decision-making (records of

deliberation as well as outcomes); open meetings.

o COMING: Patient Experts in Health Technology!

Contact:

Durhane Wong-Rieger

Consumer Advocare Network

www.consumeradvocare.org

416-969-7435

[email protected]

49

http://www.consumeradvocare.org

What does better look like?• Overall, evaluation reveals that OCC is a success in the making

and an important step forward for the province

• Opportunities for improvement

Recommendations:

• Current mandate is vague, needs clarification

• Inviting citizens to the HTA table – be prepared

• Relationship management is critical

• More explicit accounts of how OCC reports are used

• KEY will be: Recognition that these outcomes and processes are

important, and social and political will to encourage governments to

adopt this framework for public engagement

What does better look like?

• Multiple points where communication and process are

important – points where members‟ confidence in the

process is put at risk:

• Selection of members

• Frequency of meetings

• Responses to reports

• Turnover of members


• Value of the evaluation

• It helps identify what kinds of outcomes

matter, what kinds of processes might help to

improve these outcomes, and WHY

Acknowledgments

St. Michael’s Hospital

• Jim Lavery

• Wendy Rowland

• Muhammad Mamdani

• Andreas Laupacis

• Natalie Baker

• Applied Health Research Centre

OCC

• Study participants

Ontario MOHLTC/OPDP

• Diane McArthur

• Blair Parsons

• Ontario Citizens‟ Council Advisory Committee

Funders

• Ontario Drug Policy Research Network and Ontario MOHLTC

55


Elaine MacPhail,

CADTH

56

What does “better” look like: 2011-2012 CDR Patient Input Evaluation

Ensure all stakeholders are aligned on purpose and values of patient input Describe use of patient input in CDR deliberative process (done)

Consider input from individuals and caregivers

Enhance processes Update template

Provide greater advance notice (done)

Dedicate employee to patient engagement (done)

Obtain patient group feedback on summary of patient input

Provide opportunity to rank importance of outcomes

Provide more training opportunities

Goals

Design

Process

SECOR Evaluation now available at www.cadth.ca

What does “better” look like?

Patient engagement a way of life*

“Good” is evolving

Studies that include more outcomes and issues of importance to

patients

Processes that include greater consultation, participation and

communication

Evidence equally valued: patient, clinical and economic

Greater support and training

Increased transparency

* Canadian Foundation for Healthcare Improvement

What does “better” look like

CADTH has a phased approach for implementing

changes

Beginning in 2013

Beginning in 2014 and beyond

CADTH is piloting patient input in therapeutic

reviews

Beginning with: Comparative Clinical and Cost-

Effectiveness of Drug Therapies for Relapsing-Remitting

Multiple Sclerosis


Mona Sabharwal

60

What does “Better” Patient Engagement look like?

For Patients:

• Seeing/feeling that input has impact when it

matters

• Knowing what information to provide and what to

leave out

• Broadening the patient perspectives gathered

• particularly poor prognosis, rare or uncommon

cancers

• geographically remote cancer patients and

families

• cultural and/or socioeconomic barriers to

patient participation61

© 2013 pan-Canadian Oncology Drug Review61

What does “Better” Patient Engagement look like?

For pCODR:• Patients are aware of pCODR, what we do and what

we don’t do

• Patients are interested to participate in review

process

• Patient input and feedback has clear and credible

key messages

• Clinical trials capture outcomes and issues of

importance to patients

62© 2013 pan-Canadian Oncology Drug Review

62

Questions for Reflection and Response

• What is currently being done around patient

and citizen engagement in HTA?

• What has been the impact of patient and

citizen engagement in HTA?

• What does “good” or “better” look like?

• How can Canada become a leader in this

area?

patient and citizen engagement in hta decision making...model of current patient input in hta...

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