Patient and Citizen Engagement in HTA Decision Making. E4 Panel session, CADTH Symposium, May7th, 2013

Transcription

1 Patient and Citizen Engagement in HTA Decision Making E4 Panel session, CADTH Symposium, May7th,

2 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)

3 Nothing About Me, Without Me

4 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?

5 Influence and Decision Making Twelve Angry Men fadedvideolabels.blogspot.com

6 Part 1: What is currently being done around patient and citizen engagement in HTA?

7 ADVOCARE Durhane Wong-Rieger, PhD Chair, Consumer Advocare Network Thanks to Janssen for some of the slides and analysis

8 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

9 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

10 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

11 Model of Current Patient Input in HTA Consultee, Informant Patient/Public Member Input thru Council, Task Force, Collect Info: Survey, Poll, Focus Group Form of Info: Answers, Opinions, Deliberation Impact: Advise, Discretionary Examples: NICE Citizens Council, IQWiG, Ontario Citizens Council Input thru Committee, Board, Council Collect Info: Experts, Deliberation Form of Info: Analytical, Guidelines Impact: Varied, Based on Guidelines Examples: NICE, AU MASC, CEDAC, perc Ontario CED Individual Patients Input thru Clinical Trials, Testimony Collect Info: QoL, PROs, Impact Statement Form of Info: Ratings, Qualitative Impact: Varied, Emotional Suasion Examples: SMC, IQWiG Quebec conseil, BC Pharmacare, Patient Groups Input: Submission Collect Info: Written, Oral, Meetings Form of Info: Qualitative Statement Degree of Impact: Response Examples: NICE, SMC AU MASC, CADTH, pcodr, Ontario CED Patient Representativeness 11

12 Citizen engagement in HTA: A Brokered Dialogue with the Ontario Citizens Council Janet Parsons, PhD Jim Lavery, PhD

13 Ontario Citizens Council 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 13

14 Ontario Citizens Council

15 CDR Patient and Public Involvement Elaine MacPhail, CADTH 15

16 CADTH patient and public involvement Patient Input Process 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 Public members on: Canadian Drug Expert Committee & Health Technology Expert Review Panel with full voting rights 16

17 How patient input is used Canadian Patient Groups Patient Input Submission CADTH Review Team Patient input collated, summarized, used to inform protocol & included review report Original input & summary sent to CDEC Expert Committee Patient input presented, used in deliberations & reflected in recommendations Reasons & recommendations published Drug Plans Patient input shared Patient input shared Patient input shared Patient input shared 17

18 Patient Engagement in pcodr Mona Sabharwal 18

19 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 Review 19

20 Patient Advocacy Groups pcodr * Industry/ Tumour Group Updated March 31, 2011 pcodr Review Process Variable 5 business days business days 12 business days 10 business days 20 business days 12 business days Estimated business days 1. Conduct Pre- Submission Planning activities including getting input from PAG and notifying Patient Advocacy Groups 2. Prepare & submit Request for Drug Review 3.1 Screen Submission and Initiate Review Process 3.2 Collect Patient Advocacy Group Input 4.1 Conduct Clinical Review 4.1.1/4.2.2 Clarify info with Submitter during review 4.2 Conduct Economic Review 5. Summarize & Review with perc 6. Prepare & Publicly Post Initial Recomm, Post Reviews 7.1 Get Feedback from Submitter (and impacted manufacturer) 7.2 Get Feedback from PAG 7.3 Get Feedback from Patient Advocacy Group 7.4 Eligible for Early Conversion? No 8. Summarize & Review with perc Direct impact of patient perspective Indirect impact of patient perspective Yes 9. Prepare & Publicly Post Final Recomm & Post Input End *Includes pcodr Secretariat, Clinical Guidance Panel, Economic Guidance Panel, pcodr Expert Review Committee (perc) and Provincial Advisory Group (PAG) Next steps could include Recommendation implementation, Procedural Review or Resubmission 2013 pan-canadian Oncology Drug Review

21 perc Patient Members: 2 voting, 1 alternate Selection criteria/process: Training: Demonstration of personal knowledge of, experience with and understanding of issues related to cancer and its management (among other qualifications) Broad advertising, independent 3 rd party to screen applicants, diverse panel for decision making 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 2013 pan-canadian Oncology Drug Review 21

22 Detailed Description of Each Element of the perc Deliberative Framework (1) Criteria Sub-Criteria Sub-Criteria Definitions Overall Clinical Benefit Effectiveness (systematic review in the Clinical Guidance Report) Safety (systematic review in the Clinical Guidance Report) Burden of Illness (Clinical Guidance Report, patient advocacy group input) Need (Clinical Guidance Report, patient advocacy group input) 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. Frequency and severity of adverse effects associate with the new drug compared to other drug and non-drug alternatives. Incidence, prevalence or other measure of disease burden on the population. Availability of an effective alternative to the drug technology pan-canadian Oncology Drug Review 22

23 Detailed Description of Each Element of the perc Deliberative 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 pharmacoeconomic 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) Organizational Feasibility (Provincial Advisory Group input) The net budget impact of the new drug on other drug and health system spending, including companion testing technology. 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

24 Part 2: What has been the impact of patient and citizen engagement in HTA?

25 ADVOCARE Durhane Wong-Rieger, PhD Chair, Consumer Advocare Network

26 CDR Recommendations ( w/patient Submissions) N=58 Total With Patient Submission No Patient Submission Overall List List Conditional Not List 100% (58) 72% (42/58) 28% (16/58) 12% (7/58) 71% (5/7) 29% (2/7) 45% (26/58) 85% (22/26) 15% (4/26) 43% (25/58 ) 60% (15/25) 40% (10/25) 26

27 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

28 CDR/pCODR ( ) Reasons for Recommendations List (n = 4) List Conditional (n = 22) Not List (n = 16) Clinical Trial Outcomes (effectiveness, sufficient, similar, certain) Safety (adverse effects, similar risks, certain) Cost-effectiveness (more, less, certain) Quality of Life (studied, better, specific) Patient submissions (reference, substantiated 100% 91% 88% 25% 28% 31% 100% 87% 56% 25% 59% 44% 0% 82% 63% 28

29 Impact of Patient Submissions: CDR/pCODR Recommendations 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

30 Impact of Patient Submissions: CDR/pCODR Recommendations 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

31 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

32 Purported and Real Impact of Patient Engagement in HTA Looks Like Patient perceived outcomes built into clinical trials (QOL, PROs) Patient submissions to HTA give qualitative experience; own words; collective group input Patient or public members on HTA bodies assure perspective of patient or society at table Multi-stakeholder forums, Citizens Councils, consultations are opportunities for input Plain language reports increase patient understanding and acceptance of HTA outcomes Really Happens Scales unable to capture patient experience; no long-term impact No process to integrate patient submissions into quantitative approach; ignore, discount Patient/public member roles poorly defined; lack technical expertise; public patient Most public/patients unaware of processes; technical or academic Patients want full access; don t trust unless present: want right to appeal

33 Citizen engagement in HTA: A Brokered Dialogue with the Ontario Citizens Council Janet Parsons, PhD Jim Lavery, PhD

34 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 participation o o An opportunity to speak directly to government Profound experience of engagement If their contributions are not recognized/acknowledged, will lead to disappointment/disillusionment

35 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)

36 What is the impact of this kind of citizen engagement?

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

38 CDR Patient and Public Involvement Elaine MacPhail, CADTH 38

39 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

40 Patient groups willing to contribute 74% submissions receive patient input Av. 2 patient groups provide input per drug 75 different groups across Canada have contributed Data from May 2010 to April

41 Patient Engagement in pcodr Mona Sabharwal 41

42 Patient input being used in pcodr reviews 42

43 Patient Advocacy Group Engagement - on pcodr Reviews with perc Final 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%) 2013 pan-canadian Oncology Drug Review 43

44 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?

45 Part 3: What does good or better look like?

46 ADVOCARE Durhane Wong-Rieger, PhD Chair, Consumer Advocare Network

47 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

48 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!

49 Contact: Durhane Wong-Rieger Consumer Advocare Network

50 Citizen engagement in HTA: A Brokered Dialogue with the Ontario Citizens Council Janet Parsons, PhD Jim Lavery, PhD

51 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

52 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

53 What does better look like?

54 What does better look like? Value of the evaluation It helps identify what kinds of outcomes matter, what kinds of processes might help to improve these outcomes, and WHY

55 St. Michael s Hospital Jim Lavery Wendy Rowland Muhammad Mamdani Andreas Laupacis Natalie Baker Applied Health Research Centre OCC Study participants Acknowledgments Ontario MOHLTC/OPDP Diane McArthur Blair Parsons Ontario Citizens Council Advisory Committee Funders Ontario Drug Policy Research Network and Ontario MOHLTC 55

56 CDR Patient and Public Involvement Elaine MacPhail, CADTH 56

57 What does better look like: CDR Patient Input Evaluation Goals Design Process 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 SECOR Evaluation now available at

58 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

59 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

60 Patient Engagement in pcodr Mona Sabharwal 60

61 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 participation 2013 pan-canadian Oncology Drug Review 61 61

62 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 2013 pan-canadian Oncology Drug Review 62 62

63 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?