Hta basic introduction
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This document discusses the importance of patient involvement in health technology assessment (HTA). It notes that HTA evaluates new drugs and medical technologies but typically does not incorporate patient perspectives on factors like quality of life. The document advocates that patients should play a role in HTA processes by providing input on treatment outcomes, tolerability, and other real-world considerations to ensure funding decisions align with patient values and needs. It provides examples of how patients could be engaged as representatives, through surveys or public input, and highlights the need for HTA to consider individual patient impacts rather than just average population effects.
Hta basic introduction
- 1. HTA: What every Patient Needs to Know Durhane Wong-Rieger, PhD Consumer Advocare Network
- 2. Scenario 1: Regulator has approved a new drug for your condition. There are already four other effective therapies available. § As a pa&ent, what ques&ons would you ask before switching to this new drug? § Benefits vs. Risks § Is it safe? Is it effec&ve? § What are the side effects? § Comparison to current § How does it compare to old therapies? § Is it easier or more difficult to use? § How happy am I with my current therapy?
- 3. Scenario 1: Regulator has approved a new drug for your condition. There are already four other effective therapies available. § As a drug plan manager (funder), what ques&ons would you ask before making the drug available? § What are added benefits of new therapy? § What are new risks of therapy? § How does “cost” affect the balance? Benefits Risks
- 4. Scenario 2: Regulator has approved three new drugs for three different conditions. § As the drug plan manager, you recognize each is likely to have a significant impact on your drug budget, so you could not afford all three. Total budget impact of each is about the same. What do you choose to do? Which One? Hi$, Few Patients Low$, Many Patients Very Hi $, Life Saving
- 5. Scenario 2: Role of patient preference As a pa&ent, what factors would you suggest the drug plan consider? What if you had one of the condi&ons? Quality of Life Disease Impact No OtherTherapy Life Saving StopProgression Fewer Side Effects Easier to Manage Return to Work
- 6. HTA has Role in Access 6 Researcher: Does drug work in lab tests? Is it safe? Company: Does drug work in patients? What are adverse effects? Regulator: Do benefits outweigh harms? What follow-up monitoring required? HTA: How does drug compare to existing therapies? Is it worth the cost? Payer: What is budget impact? Affordable, or spend on something else? Patient Impact
- 7. ü Patients ü Public ü Industry ü Regulator IS THERE A PROBLEM? Healthcare Demand vs. Fiscal Constraint USER vs. PAYER ² HTA ² Government ² Other payers ² Other services We need better, faster, more tolerable therapies We need to manage rising costs, demands, expectations Healthcare Provider?
- 8. Roles of Regulator and HTA Regulator HTA Conducted by government authority, has legal implications and grants right to sell drug Conducted by a variour entities, recommends what “should” be funded and not legally binding Evidence: From clinical trials; controlled conditions; compared to placebo or standard of care Evidence: Compared to best alternative; real-world usage, costs and savings of intervention Evaluate based on benefits outweigh risks (potential harms) Assess benefits-risks compared to alternatives and relative costs Process: Researcher develops, industry collects CTs and submits, regulator reviews Process: Company submits CTs and cost-effectiveness data; HTA comparison to alternatives Approve based on benefits-risks; post-market monitoring to deal with uncertainty Recommend based on cost to benefits, added cost for added benefit, or value of alternatives 8
- 9. CEA: Cost Comparison 9 Patient Population Submitted Drug Comparator Drug • Current standard of care • Drugs in a similar group • Nothing/no treatment • Previous standard of care
- 10. CEA: Cost Minimization 10 Patient Population Submitted Drug Comparator Drug Similar clinical effects Consider costs - Drug costs - Total cost (drug & health care)
- 11. CEA: Cost Effectiveness 11 Patient Population Submitted Drug (1) Comparator Drug (2) Clinical effects (1) Costs (1) COMPARE: Clinical effects (2) Costs (2) Effect Drug 1 Cost Drug 1 Effect Drug 2 Cost Drug 2 > ?
- 12. Decision Matrix Drug A Costs more and is less effective than Drug B (Choose B) Drug A Costs more and is more effective than Drug B (Do Cost-Effectiveness) Drug A Costs less and is less effective than Drug B (Do Cost-Effectiveness) Drug A Costs less and is more effective than Drug B (Choose A) 12 Effectiveness (life years, QALY’s, etc.) COST$
- 13. Cost-utility Analysis Same principle as cost-effectiveness Compare increase in cost with increased “Quality of life” benefits Cost A - Cost B = Cost-Utility Ratio QALYA - QALYB § Use units of Quality of Life in denominator as “utility” § Represents Measure of “satisfaction” with state of health 13
- 14. HTA Cost-Effectiveness Measures § QALY = Years of additional life, adjusted for quality § “Quality of life” from questionnaire asking physical and cognitive abilities, well being, work and social activities § QOL for specific disease health status is “score” based on sum of capabilities and sense of well being § Assumes all life years are equivalent, regardless of age, health status, disease prognosis § QALY -= Quality-Adjusted life year based on QOL score, where “1” = perfect health and “0” = death § ICER = Incremental cost effectiveness ratio § Cost for additional QALY (how much is a year of life worth?) § Opportunity cost = Other benefits for the same $ § Funds are limited and can’t spend the same $ twice § Could you get more health benefits if spent on another therapy or disease?
- 15. May 2015 Harmonized Orphan Drugs/Rare Diseases Policy -- Latin America 15 Patient Engagement => Optimal Use Patient defines treatment outcomes, quality of life, tolerability Patient has legal, ethical, moral right to informed choice Patient helps define start/ stop criteria; uses drug appropriately Patient collaborates to monitor outcomes; provide feedback; adjust therapy Patient partners to improve access criteria and support optimal use
- 16. Lifecycle Approach with Patient Input & Real-World Data 16 Researcher/ Clinician: Disease Knowledge; Drug Discovery; Treatment Guidelines Company: Clinical Trials & Outcome Measures; biomedical, clinical, PROs, Real-World Impact Regulator: Approval on Benefits-Risks- Uncertainties; Use & Real- World Monitoring HTA: Comparison Benefits, Risks, Cost w/ Alternatives; Place in Therapy Payer: Budget Impact; Access Criteria; R-W Data Collection Patient Input
- 17. How Could Patients Engage in HTA Consultee, Informant 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 Patient Representative 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 INESSS, 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 ParticipationinSystem
- 18. Need Patient Values As Alternative to System Values System Values Patient Values “Faint hope” “Chance for life” Convenience Quality of Life Standards of care for average patient Personalized treatment protocol Delayed access based on long-term outcomes (real- world data) Timely access based on sufficient data (coverage with evidence development) Restrictive practice guidelines to avoid inappropriate use Facilitative guidelines to allow optimal prescribing Prefer: Small benefits to many Allow: Large benefit to “1”
- 19. Contact: Durhane Wong-Rieger Consumer Advocare Network www.consumeradvocare.org 416-969-7435 durhane@sympatico.ca