Ethical issues and HTA


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Improving the Value of Patient Submissions on Drug Review Processes.

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Ethical issues and HTA

  1. 1. Improving the Value ofPatient Submissions onDrug Review Processes Durhane Wong-Rieger, PhD Devidas Menon, PhD Tania Stafinski, PhD Gabriel Wong-Rieger, BCom
  2. 2. Purpose: How to Promote theValue of Patient Submissions Challenge that HTA (value of therapy) did not sufficiently account for patient impact  Concern: HTA (as applied to the public drug review processes in Canada) do not adequately reflect the value of the therapy to patients  HTA theoretically includes societal values but evident Need for direct patient input  Beyond Quality of Life scales (does not sufficiently capture all of the value to patients; quantitative summary does not convey patient perspective)  Patient is best positioned to provide experience of disease, previous therapy, new therapy
  3. 3. Introduction of PatientSubmissions Introduced by CADTH, Ontario, and BC (process in Quebec) Opportunity for patients and patient representatives to provide direct input, in their own worlds In most cases, will be presented by the patient/pubic member on the HTA committee (accurately reflect, interpret and advocate for the patient perspective) Challenge: How does committee integrate patient input in HTA process?
  4. 4. Purpose of Workshop Sessions Purpose: Provide Insight on Patient Submissions and Influence on HTA process Questions Addressed  Given all of the data available to HTA, what is perceived value of direct information from patients on impact of disease and therapy  What are ways in which patient data could be meaningfully considered within HTA processes?  Does the process (group deliberation) for considering patient data affect the outcome (decision on value of therapy)
  5. 5. Objectives of Sessions 2 workshops (Ontario and BC) with patients and other stakeholders Objectives  Learn about patient submission process: what, how. why  Learn from patient/public members of HTA committees on perceived value and process for considering patient input  Gain insights on HTA/drug review process through simulation  Compare methods for considering patient input as part of HTA/drug review process  Learn factors that potentially influence drug review
  6. 6. Participants Two Sessions  Ontario: 90 (50% or 45 patients)  BC: 75 (65% or 49 patients) Patient representatives  Patients, patient advocates, board, and staff  Previous participation in HTA training Some experienced in making submissions to CADTH or CED
  7. 7. Session Agenda (Intro) Pre-assessment: knowledge and trust of agencies Introduction to HTA and Committees  Overview of HTA  Overview of CADTH (Common Drug Review) and Patient Submission Process Overview of provincial drug review process Perspectives of patient/public members  CADTH (CEDAC)  Ontario Committee to Evaluate Drugs  Patient organizations
  8. 8. Small Group Simulation of DrugReview Committee Each group reviews 3 of 4 drugs for different conditions  Disease (health condition) and drug information, including safety and efficacy data, size of patient population  Cost-effectiveness data, including alternative treatment May only recommend 2 of 3 drugs Without patient input, make Initial group decision on whether to recommend drug for funding Question: what additional patient information do you want? Provided ―patient submission‖ on impact of condition, current therapy, proposed drug impact Provided with 3 of 4 formats for considering the patient submission along with rest of data
  9. 9. Drugs and Condition Information A: Progressive debilitating; affects 2 in 1000  Current: injection every 4 weeks; reduces relapses, slows progression, mild to severe side effects  New: Injection every 4 weeks, reduces relapses by half; 1% severe disability or death; $20K/year; $68K/QALY B: Genetic, auto-inflammatory; affects 1 in 100,000; debilitating, mental retardation, vision loss, deformities  No approved therapy but RA therapy used off-label, daily injection.  New drug injected every 6 weeks; nonlife-threatening side effects, $96K/year; $110K/QALY
  10. 10. Drugs and Condition Information C: Blood cancer, affects 1 in 20,000  Current: IV chemotherapy, 2-year survival at 25%  New: oral; 3 single-arm CTs = 2-year survival at 93%; 1% risk liver toxicity; life-long therapy $30K/year; $20K/QALY D: Obesity, affects 25% Canadians, linked to diabetes, CVD, cancers  New drug: oral, twice daily. RCT loss of 8kg more than placebo; reduced metabolic CDV risks; 25% psychiatric events with 0.02% suicides; on treatment until healthy weight; $3K/year, $60K/QALY
  11. 11. Group Decision-Making Formats Each group assigned Open Discussion + 2 other formats Open Discussion: Individuals freely share opinions, beliefs, and preferences to arrive at a group decision.  Pro’s: Equal voice, relevant attributes specific to current case, unrestricted conversation  Con’s: Leader/champion influence, lack of prioritization or systematic consideration of all attributes Nominal Group Technique: Individuals formulate and express own opinions prior to influence from others  Pro’s: Initial personal opinion shared; al options considered; identify and discuss personal biases  Con’s: Support own ―uninformed‖ opinion; polarization of views
  12. 12. Group Decision-Making Formats Each group assigned Open Discussion + 2 other formats Dialectical Inquiry/Deliberative Dialogue: All aspects of ―funding or not funding‖ explored prior to making decision.  Pro’s: Attributes relevant to key stakeholders strongly positioned  Con’s: Arriving at consensus; need for representative views Simple Multi-attribute Rating Technique: Systematically analyze the attributes of the drug and arrive at a decision based on a ―weighted‖ assessment of the criteria  Pro’s: Consistent and systematic approach across drugs  Con’s: Restricted discussion of perspectives; too reductionist
  13. 13. Patient Submission Summaries Drug A for Progressive neuromuscular  Impact: Devastating, severe attacks, relapses  Treatment outcomes desired: reduce severity, frequency of attacks; less severe side effects  Drug characteristics: small fatal risk acceptable Drug B for Rare Genetic Disorder  Impact: Fever, pain, joint and tissue deterioration  Treatment outcomes desired: short-term reduction of flare- ups, fever, pain; long-term damage reduction;  Drug characteristics: fewer injections
  14. 14. Patient Submission Summaries Drug C for Blood Cancer  Impact: devastating, low survival, helpless, hopeless  Treatment outcomes desired: survival, reduced blood counts  Drug characteristics: oral, nausea manageable Drug D for Obesity  Impact: Physical and psychological, low self-esteem  Treatment outcomes desired: sustainable weight loss which is very difficult to attain  Drug characteristics: most effective, least risky options; immediate and steady weight loss
  15. 15. Results: Group Decisions Total 19 groups and 57 decisions  Each group: may approve maximum 2 of 3 drugs  Groups segregated as patients and non-patients Small difference in recommendations from patients and non-patients  Patients: approved 67% drugs (2 out of 3)  Non-patients approved 59% (fewer than allowed) Small difference between Ontario and BC groups  BC: 71% approval (1 group approved all 3 drugs)  Ontario: 58% (2 groups approved only 1 drug)
  16. 16. Results by Condition and DrugApprovals (all groups combined) Drug C (Blood Cancer) approved by 100% of groups Drug B (Rare Genetic) approved by 86% of groups Drug A (Progressive) approved by 67% of groups Drug D (Obesity) approved by 0% of groups
  17. 17. Rationale for Drug DecisionsImportant Rationale Drug C: Increased chances for survival; everyone relates to cancer; acceptable $/QALY Drug B: No other treatments; no proof of long-term benefit; small numbers; approve despite very high $/QALY Drug A: Reduced relapses but no impact on progression; severe disease; high $/QALY Drug D: Not significant benefit; no direct disease impact; should not be public funding
  18. 18. Approvals by Discussion FormatNo difference in Approvals by DiscussionFormat Multi-Attribute Rating: 67% Approvals Open Discussion: 63% Approvals Nominal Group: 64% Approvals Dialectic Dialogue: 58% Approvals
  19. 19. Approvals by Drug and DiscussionFormat Progressive Rare Blood Obesity Overall Debilitating Genetic CancerOpenDiscussion 60% 80% 100% 0% 63%NominalGroup 50% 100% 100% 0% 64%Mutli-Attribute 75% 75% 100% 0% 67%RatingDialecticDialogue 75% 100% 100% 0% 58%OVERALL 67% 86% 100% 0% 63%
  20. 20. Frequency of Decision Reasons For positive decisions, frequently cited:  Safety (lack of or manageable side effects)  Cost, cost-effectiveness, or cost mitigated by other benefits  Meeting patient needs and survival benefits  Quality of life; lack of alternatives For negative decisions, frequently cited:  Lack of comparator (placebo only)  Lack of adequate information  Risk of side effects  Lack of sustainability and long-term outcomes  Lack of cost-effectives (cited by only 1/3)
  21. 21. Average Number Reasons Cited byVariable Variable Avg. # ReasonsDiscussion Format Open 2.2 Nominal Group 2.5 Multi-Attribute Rating 1.6 Deliberate Dialogue 1.4Drug Progressive Disease 2.1 Rare Genetic 1.4 Cancer 2.1 Obesity 2.3Decision Yes 1.9 No 2.1All 2.0
  22. 22. Summary—Number of Reasons By decision, more reasons cited for ―no‖ (2.1) than for ―yes‖ (1.9) By deliberation format, fewer reasons with more structured group format (multi-attribute (1.6) and deliberate dialogue (1.4)) than with more open discussions (open format (2.3) and nominal group technique 2.5)) By type of drug, fewest reasons given for rare genetic drug (1.4) than for all others (2.1 – 2.3)
  23. 23. Preferences for Discussion Format Preferences for Discussion Formats  MA Rating: best liked; easy to do; sorted options for easy decision making  Open: Usual process; best when high consensus; leader may set direction  Nominal: Assured all opinions heard; allowed differences to surface  Dialectic: Difficult to ―role play‖; helpful in hearing patient perspective
  24. 24. Advocare Simulation: Patient InputShifts Discussion Groups felt patient information was very important for conditions that were unfamiliar to them, specifically the progressive debilitating disease and the rare genetic condition. Information about the patient experience of the challenges of the conditions were cited as important, as was information about the current therapies, or lack thereof. Prior to receiving patient information, groups said they were more focused on cost, cost per QALY, and affordability. Introduction of patient submission focused discussion, regardless of the format, on the patient’s perception of the burden of the condition, the challenges of current therapy, and the potential benefit on hope, manageability, and quality of life.
  25. 25. Advocare Simulation: How DoesPatient Input Impact Discussion? Groups reported that the patient submission helped them to visualize the patient, see him/her as a person rather than statistic The ―lack of concern‖ about potentially fatal (but very rare) side effect was important Risk of imminent death (blood cancer) was a significant factor and had greater weight than debilitating condition. Drug C for blood cancer always chosen regardless of alternatives. Slowing progression was not as important as reducing risk of developing organ, joint, tissue damage. Drug A for progressive condition NOT chosen if alternatives were B (genetic disorder) AND cancer.
  26. 26. Advocare Simulation: Findings ofPatient Submission Workshops Direct patient input (submissions) enhance discussion of patient impact and consideration of patient values and preferences Cost factors (individual cost/patient and $/QALY) are important factors but can be superseded by patient impact (especially if rare disease or not other treatments) Survival (even for limited time) given more weight than reducing risk of future serious organ/joint damage and reducing progression of debilitating disease Groups liked rating tool; easy way to integrate variety of factors and rank options. Mutli-attribute Rating technique also resulted in the highest percentage of approvals overall Drug fo common condition (obesity) not given priority over serious diseases.
  27. 27. Patient Trust in Drug ReviewAgencies Pre-and Post-WorkshopTrust knowledgeable and Ontario Ontario BC BCfair process by… (agree Pre- Post Pre Postsomewhat and stronglYHealth Canada approving 90% 100% 95% 92%drugCADTH (Common Drug 65% 84% 43% 65%Review) recommendingProvincial Drug Program 55% 68% 45% 425funding drugPhysician recommending 90% 74% 95% 92%drugPatient Group informing re: 90% 100% 100% 92%drug and options
  28. 28. Recommendations for PatientEngagement with HTA  Dialogue with patient groups to define submission: what information, how to collect, how to present  Engage patients in CT design to ensure patient values included in measures  Train potential HTA patient-public members on technical processes of HTA and decision making  Provide means for patient-public members of HTA committees to dialogue with patient representatives  Train all HTA committee members on methods for integrating qualitative information  Promote transparent decision-making (records of deliberation as well as outcomes); open meetings.  COMING: Patient Experts in Health Technology! 28
  29. 29. Contact: Durhane Wong-RiegerConsumer Advocare 416-969-7435