Transcript Document
How Canadians Access Drugs for Rare Disorders Health Canada and Provincial Drug Plans
Durhane Wong-Rieger, PhD President Canadian Organization for Rare Disorders
Key Steps to Drug Access
• • • •
Health Canada: Approval to Market Common Drug Review: Cost-Effectiveness Provincial Drug Plans: Impact on Drug Budgets and Comparative Costs What about Orphan Drugs?
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Health Canada: Must Approve Sale
Manufacturer Must Make Application
Company decides to “sell” drug in Canada; applies for specific indications; pays application fee If “breakthrough”, can be given “priority review”
Company submits evidence from clinical trials
Is Drug Safe: cause serious harm? Side effect?
Does drug work: reduce symptoms, improve outcomes, cure?
Do benefits outweigh harms: life-threatening disease, no other treatments, evidence?
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Common Drug Review: Is it Worth the Cost?
Pharmacoeconomics uses economics and clinical science to answer question: Is drug cost-effective?
Effectiveness: what are the benefits of the drugs Cure the disease Reduce major symptoms, fewer side effects, significantly easier to use Gives more years of life Cost per additional years of life Are the additional benefits worth the additional costs?
Is it as cost-effective as previous drugs Drug Approval Process and Orphan Drugs 27 May 2006 4
33 CDR Recommendations as of December 31, 2005
58%
42%
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Avodart Axert Combigan Reyataz Humira Myfortic Neulasta Pegasys RBV Teveten Plus Telzir Vfend Yasmin Tarceva Kivexa List/List with Restrictions
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Do Not List
Evra Iressa (Noc/c) Viread (Noc/c) *** Remodulin *** Fabrazyme Adderall XR Replagal (Noc/c) Zavesca Forteo Ciprodex Gynazole.1
Relpax *** Sensipar Amevive Aldurazyme Lantus *** Norprolac *** Strattera *** Ebixa ***
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*** All listed by Quebec
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Drug Plan Listing Status of CDR Recommendations
30 100% Provincial Drug Plan listing status of the 33 New Drug recommendations from CDR May 2004 – December 31 2005 25 20 15 58% No 10 5 0 To ta l 42% Yes BC 24% 27% 30% 15% 15% 15% 30% 0% AB SK MB ON NB NS PE 27% 27% NL NI H B QC 55%
* Quebec does not participate iMAM® Brogan Inc. December 2005 27 May 2006 Drug Approval Process and Orphan Drugs 6
CDR Decisions to Date (Aug. 24, 2005)
Decision
List in a similar manner as drug plans list other drugs of the same class
No. submissons
8 List with criteria/conditions 4 Not to list Total decisions 16 28 Drug Approval Process and Orphan Drugs 27 May 2006 7
Provincial Response to CDR Decisions
BC AL SK MB ON NB NS PEI NL
No. listing decisions No. listings 1 5 10 2 2 0 13 0 1 1 5 6 2 2 0 5 0 1 Average time to listing (days) Historical time to listing (days) Concordance with CDR recommendation 27 May 2006 284 450 1/1 349 406 5/5 351 346 310 551 10/10 2/2 328 494 2/2 Drug Approval Process and Orphan Drugs - - 302 592 428 744 11/13 - - 8 300 352 1/1
Access to Orphan Drugs in Canada
Unlike USA, UK, Japan, Australia and European Union, Canada does not have an Orphan Drug Policy In USA, provides incentives for companies to research drugs for orphan conditions, negotiated conditions for priority review for FDA approval In most other countries, priority reviews and specific criteria for approval (surrogate markers, small patient populations, collapsed Phase 2/3 trials) Canada can provide priority review and will consider surrogate markers Drug Approval Process and Orphan Drugs 27 May 2006 9
CDR process not suited to orphan disorders
CRD applies standard cost-effectivness review even to orphan indications Typically, drugs for orphan indications are new treatments based on surrogate markers with no long-term studies Typically, drugs for orphan indications will be more costly No specific criteria for treatments that are life-saving or have no other drugs available Evaluation of cost relative to benefits (cost versus savings over other drugs) Cost-effectiveness ($ for Quality-adjusted life year gained) approved if less than $50,000 Off-label (use not approved by Health Canada) will not be assessed by CDR (no clinical data) Drug Approval Process and Orphan Drugs 27 May 2006 10
National Pharmaceutical Strategy
Proposed for a national program for access to drugs (funding and post-market surveillance) approved by F/P/T Ministers in 2004 Could include catastrophic drug coverage (those whose drug costs are exorbitant relative to income) Could include national drug formulary (list and conditions for funding drugs through public drug plans) Could include “expensive drugs for rare disorders” 10-year NPS with first progress report: June 2006 27 May 2006 Drug Approval Process and Orphan Drugs 11
Why Do Advocacy?
Solve an individual problem (specialist appointment, access to treatment & homecare) Address problem that affects group of consumers {disease-specific & community} (hospital parking, clinic hours, specialist care & emergency; insurance coverage) Influence a policy or regulation or law (Drug licensing or formulary listing, hospital closures; disease-specific programs, disability assistance, care in rural areas) Drug Approval Process and Orphan Drugs 27 May 2006 12
What is Advocacy?
Appeal for support by writing letters, making phone calls, visiting decision-makers or those with influence.
Engage public support by publicize stories through media, tell stories at meetings, conferences, gatherings & hold press conferences; conduct polls, surveys & publicize findings.
Engage support of influential others or decision-makers; be present at appropriate events, such as committee meetings, conferences and legislative sessions.
Demonstrate at appropriate events in order to make issues public.
Disrupt service delivery, meetings, conferences or legislative sessions to force attention to the problem or issue.
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Reformer (Member ) Tactics
Legitimize Issue
Influence
Evidence, Trade-offs
Success factors
Expertise, Compromise
Best Outcomes
Internal debate, temporary resolution, policy change
Broker (Independent) Tactics
Public debate, negotiate
Influence
Informed public, expert support
Success factors
Diplomacy, public commitment
Best Outcomes
Win-win solution, commitment to change 27 May 2006
Activist (Group) Individual (Radical) Tactics Tactics
Demonstrations, Class Action Testimonials, Lawsuit
Influence
Sympathy, Guilt
Influence
Confrontation, Media
Success Factors Success factors
Public support, Credible spokespeople Personality, Integrity
Best Outcomes
Win Case, Raise awareness
Best Outcomes
Support for Issue Drug Approval Process and
FEW Credibility Among Consumers (Public) PUBLIC
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Provide A Solution, Not Just the Problem
Start with the need Increased burden of not preventing or not treating could escalate healthcare costs Despite high cost of treatment, cost effective Give them a solution Long-term: Co-ordinated strategy of treatment and prevention reduces impact and future infections Short-term: Investment in diagnosis and early treatment to reduce burden of disease 27 May 2006 Drug Approval Process and Orphan Drugs 15
Don’t Forget the Sizzle
Use language to appeal to emotions: compassion, fear, justice Create a headline: “tainted” blood, “miracle” drug, “silent” killer, “hidden” epidemic, nation of “guinea pigs” Appeal to the audience’s sense of justice or compassion: “the right thing to do” Appeal to the audience’s self-interest: “what’s in it for me” KISS rule still works
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You Gotta Have Friends
Build A Coalition Creates stronger case; Optimizes scarce resources Bring together disparate views Talk to One Another Coordinate activities Share intelligence Keep others posted on your actions Row Together (Our Worse Enemies are Ourselves) Enemies: Different Interests, No Trust Bed Fellows: Common Interest, No Trust Competitors: Different Interests, Trust Allies: Common Interest, Trust Drug Approval Process and Orphan Drugs 27 May 2006 17
Engage the Media
Make media aware of, care about, your cause and/or group Engage media to promote cause and group to public [Patients with private drug insurance get anemia treatment but those on BC public drug plan do not] Use media to press decision makers (embarrass, support, threaten, reward) [BC is the only major province in Canada that does not fund treatment for anemia for cancer patients undergoing chemotherapy] Use media to bring in high profile supporters 27 May 2006 Drug Approval Process and Orphan Drugs 18
8 Simple Rules for Meeting Your Legislator
Rule 1: Don’t make your first ask at a fundraiser unless you are a major contributor. You may use the occasion to reinforce a previous request.
Rule 2: Approach the legislator as a human being. Make yourself likeable. You are here to build a relationship Rule 3: Define the issue simply. Have an answer to “What Can I Do?” Make sure it is do-able.
Rule 4: Listen actively. Confirm points of agreement. Clarify. Stay on topic. It’s your agenda.
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8 Simple Rules for Meeting Your Legislator (2)
Rule 5: Be prepared with facts and figures but you don’t have to be the expert. Admit if you don’t know. Offer to follow up.
Rule 6: Be early. Don’t stay late.
Rule 7: Leave something behind. Rule 8: Follow up with a letter of thanks and ask to meet again.
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You Can Sell Them Anything...If You Know What They Want
Tie in with the government’s agenda Population health and wellness Health quality and patient care Sustainable healthcare system Programs that are evidence-based are harder to step away from Listen for political (pre election) platform and fit into the “context” Identify the overlapping risk factors (other chronic diseases or issues) Find ways to sell issues to other sectors Public Health; Community and Social Services Crime and Safety Relate to high-profile health issues that affect the public Make investments upstream to reduce downstream costs Give positive feedback 27 May 2006 Drug Approval Process and Orphan Drugs 21