Value Of Biotech To Patients General

TREATING PATIENTS AS VALUED PARTICIPANTS Durhane Wong-Rieger, PhD Chair, Consumer Advocare Network President, Canadian Organization for Rare Disorders President & CEO, Institute for Optimizing Health Outcomes

VALUE OF BIOTECHNOLOGY TO PATIENT 1990: height of awareness that 1/3 of hemophilia patients had been infected with HIV through tainted blood products. 7-year old Alex had never received clotting factor because of parental fears. Great hope was recombinant clotting factor. Canada denied access because it was considered “too expensive” June 8, 2009

VALUE OF BIOTECHNOLOGY TO PATIENT 2002: 32-year-old Mary has been diagnosed with rheumatoid arthritis for 10 years and has already had more than a dozen joint-replacement surgeries. Was in hospital at least half dozen times a year; soon to be wheelchair dependent. Received Enbrel on extended clinical trial and was able to resume normal activities. Public drug plan denied access because of lack of long-term data and cost. June 8, 2009

VALUE OF BIOTECHNOLOGY TO PATIENT 2004: 44-year old Ken, retired engineer because of Fabry disease; kidney transplant and enlarged heart. Received enzyme-replacement therapy through clinical trial and responded with immediate reduction in symptoms. Assumed would be funded similar to ERT for Gaucher’s. Public drug plan initially denied because of cost and lack of long-term data. Subsequent 3-year funding agreement, but only for patients with severe symptomology. June 8, 2009

VALUE OF BIOTECHNOLOGY TO PATIENT 2008: 12-year-old Justin with MPS II was excluded from clinical trail because lack of symptoms at time. Now, progressively deteriorating with permanent loss of hearing and mobility reduced in walking, raising arms, and uncurling fists. 10-year-old Nick with MPS II was excluded from clinical trial because of cognitive involvement. Public drug plan has just approved ERT but only to children with symptoms and has denied coverage to patients because they were not represented in clinical trials. New evidence suggests ERT may improve cognitive functioning. June 8, 2009

VALUE TO WHOM? Value to individual patient: treatment with most health benefits Value to healthcare system: treatment with best health benefits relative to costs Value to payer: treatment with lowest cost Value to society: treatments with health and social benefits relative to other uses for public funds June 8, 2009

HEALTHCARE VALUE MODEL June 8, 2009 Value to Recipients Value to Payers Value to Funder Desired Outcome Cost-effective Driving force HTA to support cost containment Principle Least cost Individual Tactics : Testimonials, Lawsuits Influence: Sympathy, Guilt, Right Success Factors: Personality, Integrity Best Outcomes: Win Case, Raise Awareness Social Value Desired Outcome Most access to all Driving Force Informed public, expert support Principle People’s Choice Individual Patient Outcome Treatment to those in need Driving Force Need for treatment, rescue Principle Rule of rescue, individual good Patient and Health System Desired Outcome Most treatment Driving Force: HTA to support most patient access Principle Level Playing Field

VALUE OF HEALTHCARE Value of Healthcare is Degree of Benefit Experienced Like Value of Education, Arts, Sport, or Entertainment Based solely on the benefits to the recipient No further justification of contribution in other ways necessary Value should not be confused with cost Cost is what it takes to bring the good to recipient Something may be valuable and not very costly; something may be costly and not much value Cost refers to what payer is able or willing to pay to make “good” available June 8, 2009

SOCIAL VALUES IN HEALTHCARE Social values are principles, morals, ethics sometimes used by society to decide which “goods” to make available Anomaly of Canadian Healthcare: Based on Strong Social Values Universal: Achieve sufficiency for all Comprehensive: Essential core needs Portable: Similar service everywhere Medically Necessary: Needed to sustain life Accessible: Available When Needed Sustainable: Funded, Affordable Drugs are Excluded from Canada Health Act and not subject to social values June 8, 2009

DRUGS NOT IN CANADA HEALTH ACT Drug Access is: Not Universal but based on ability to pay or social welfare Essential medicines provided only in hospital or to seniors, disabled Drug coverage varies considerably across provinces Even “life-saving” medically necessary drugs denied Access may be delayed for long time Sustainability (cost control) driving factor Drug Access Controlled by Formulary Listing June 8, 2009

MOST COUNTRIES: RATIONING OF DRUGS Phenomenon is evident to varying degrees in almost every country, with rules for drug access and rationing that are not applied to most other medical services “ Who gets what, when, and how much of it” not determined as personal decision by your doctor but is based on a “group decision” made by a “disinterested” panel of experts and/or bureaucrats June 8, 2009

WHOSE VALUES? DEPENDS ON WHO’S PAYING Value of Regulators: Balance harms and benefits to maximize public good (not individual patients) Value of Payers: Allocate scarce healthcare$ to maximize benefit to patient roster (not individual patients) Value of Industry: ROI high enough to justify marketing and low enough to get reimbursement Value of HC Associations/Academics: Guidelines or “standard of care” (evidence-based for most patients) Value of Clinicians: Easy access (formulary for most patients) versus “Individual Patient Request” (requires justification) Value of Patients: Individualized protocol (Right product for right patient at right time) June 8, 2009

UNCERTAINTY OF BIOTECHNOLOGICAL THERAPIES Risks very high To industry from discovery to market To regulators where safety issues may take a long time to be known To patients because of individual variable in response To funders because cost of product is high Benefits may not be well established Costs often very high June 8, 2009

CHALLENGE: PUBLIC FUNDING TO VALUE “INCREMENTAL” BENEFIT OF BIOTECHNOLOGY Biologics are enhanced therapies to replace traditional Biologics are therapies often targeted to smaller patient populations Recombinant Factor product for hemophiliacs replacing plasma-based Biologics for arthritis: more advanced degree; where traditional therapies no longer effective Cancer therapies as 2 nd and 3 rd line, for those with more advanced disease For rare diseases June 8, 2009

WELL BALANCED SOCIETY  BARACK OBAMA PLAYING BASKETBALL Ibbitson (Globe &Mail, Canada): “Don’t underestimate power of Obama’s hoop skills” Well-balanced society is like a pyramid with a detachable top being spun around an internal gyroscope The three sides = faith, tradition, and reason Top never spins too close to one side where it could fall down and the whole thing would be in danger of toppling Obama, held in suspicion by the American public for his intellectual elitism (reason) and affiliation with “religious fringe” (faith) is saved by his ability to make a “3-pointer” (tradition) June 8, 2009

WELL-BALANCED DRUG VALUATION Drug Valuation: A Pyramid Balanced by 3 sides: ration, values, and real-life impact Ration = evidence-based rules, including cost-effectiveness calculations Social values are principles of serving public good Real-life impact = personal benefits, patient reported outcomes, consumer impact One-sided Drug Plan => No wonder patients experience the system as “not working” Values are set aside because no one can agree on which values to apply Real-life impact, or personal appropriateness, has precedence only when other considerations are negligible, that is, when drugs are abundantly available (very cheap) Ration claims dominance because it is supposedly objective June 8, 2009

HEALTH TECHNOLOGY ASSESSMENT: RATIONAL APPROACH Drug Plans Prefer Rational Market Approach Cost-Benefits Analysis (Buying a Car) Cost-Effectiveness Analysis (Shopping at Walmart) Cost-Utility Analysis (Lifeboat Decision) “ Quasi-scientific” approaches that can be defended Analyses based on “benefits” and “costs” that are often nonconsensual, controversial and even capricious Process can arrive at “objective” answers, even though different committees using same process and inputs could arrive at very different conclusions Provide rational basis for committees to “work through” differences in opinion Provides rationale why exceptional cases can be rejected June 8, 2009

HTA INHERENTLY BIASED AGAINST BIOLOGICAL PRODUCTS First recombinant (non-blood derived) clotting factor for hemophiliacs: more expensive, same efficacy, but no evidence of greater safety Newer therapies (biologics for rheumatoid arthritis, HIV, cancer) replacing pharmaceuticals but without long-term clinical or survival outcomes or long-term safety data (skews risks-benefits analysis) First therapies for life-threatening disorders (ERT) may use surrogate measures without evidence of impact on clinical outcomes or long-term survival (makes cost-effectiveness comparisons challenging); lack of natural history of disease for comparison Early positive results of RCTs for “life-extending” kidney cancer drug compelled company to allow patients to “cross-over”; regulators agreed and approved. Reimbursement committee rejected based on “lack of significant difference” with remaining patient data. First approved drug for age-related macular degeneration: reimbursement committee initially rejected based partly on comparison with “much cheaper” similar drug being used “off-label” June 8, 2009

USE OF SOCIAL VALUES INSTEAD OF (IN ADDITION TO) HTA Leveling Playing Field: Help everyone achieve “basic” well-being Rule of Rescue: Saving an individual in imminent danger (at all costs) Most in Need: Saving worst off (regardless of cost and potential benefit) Fair Innings: Help the young (lives not yet lived) Longevity Reward: Help those who will gain most (regardless of age) People’s Choice: Numerous small benefits versus few large benefits (orphan drugs vs. PPIs) June 8, 2009

WHY SOCIAL VALUES ARE OFTEN IGNORED Reimbursement committees fail to engage in “values-based” discussions Membership usually includes health economists Members usually receive expert “economic” assessment as well as medical assessment Members receive training in health economics and pharmacoeconomic assessments Members have background in scientific methodology Reimbursement committees not skilled in values deliberations Committees do not include ethicists, social scientists or those who can speak to values Members receive no training in concepts of justice, ethics, or values Members not trained in dialectic process or deliberative dialogue to work through competing values or ethical dilemmas NICE: Values considered “after the fact” and application not systematic June 8, 2009

WHY PATIENT REAL-LIFE IMPACT NOT REPRESENTED Committees may include patients or public members but there is no framework for deliberating on “patient impact” or values “ I don’t know what I’m expected to contribute. Most of the time, I’m not an expert in the disease or drug being discussed.” “ Sometimes, when I put forth an opinion, others listen politely but it doesn’t change anything.” “ Rules and criteria are such that it really doesn’t matter who is at the table; decision would be the same.” Affected Patients and patient impact are excluded from the discussions Testimony from patients or clinicians not allowed at committee “ I can’t talk to you about this because it would put me in a conflict of interest and I would have to excuse myself from decision.” Patients have been forced to take their stories to the media to reintroduce the “real-life” impact June 8, 2009

KEY PROBLEM: LACK OF TRANSPARENCY Committee meetings are closed No record or minutes of discussions Record of decision tends to be sparse Criteria and decision making process are not known No process for patients and clinicians to challenge the decisions June 8, 2009

REIMBURSEMENT COMMITTEES: RATIONAL COST CONTROL Canada’s Common Drug Review provides “centralized” HTA-based recommendation for reimbursement of drugs by public drug plans In 4 years, almost all “me-too” submissions have been recommended for reimbursemen Less than 25% of “first-in-line” or “novel” therapies have been recommended (too expensive, lack of evidence, lack of added value) Almost no drugs for rare disorders (orphan drugs) have been recommended for funding Provincial/territorial/federal drug plans follow similar cost-based decision making In Ontario, drugs that can demonstrate significant cost savings can receive “priority reviews” Priority reviews for drugs that are “life-saving” or treat conditions for which there are no other therapies have generally resulted in negative decisions June 8, 2009

PATIENT “SUFFRAGE” = TURN OF 20TH CENTURY Manitoba gave women right to vote in 1916; Canada in 1919; USA in 1920; and Quebec in 1940 In Quebec, at least one hospital has a patient on P&T Committees (there may be more) In 2006, 2 “public” members were appointed to Canadian Expert Drug Advisory Committee, which recommends drugs for reimbursement on public drug plans In 2007, 2 “patients” were appointed to Ontario Committee to Evaluate Drugs, which recommends drugs for Ontario Public Drug Plan Australia, UK, France, EMEA, Netherlands have patients on drug advisory committees for evaluation and/or reimbursement June 8, 2009

TOUGH CHALLENGES FOR PATIENTS ON REIMBURSEMENT COMMITTEES “ 2 are better than 1” but “2 divided are worse than 1” Expertise in being a patient is not the same as being a patient expert You gotta be able to handle the math No matter how many initials you have after your name, the minute you’re identified as a patient, you lose 50 IQ points If you are paid less, you are valued less June 8, 2009

HOW TO INCORPORATE PATIENT VALUE Continue to challenge the “cost-effectiveness” and “cost-utility” analyses against “real-life” impact and long-term outcomes Train patients and public to better engage in health economic and evidence-based deliberations Develop a values-based decision making framework and train members and public to engage in values deliberations Bring into the committees the patients and clinicians who represent the “real-life” perspective Hold meetings in public and make the process publicly accountable June 8, 2009

CHICKEN AND PIG WHO DECIDE TO OPEN A BREAFAST RESTAURANT 5/2/08 ISPOR Chicken was motivated Pig was totally committed

Value Of Biotech To Patients General

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