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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Drug Approval Process and
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
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Orphan Drugs
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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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 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 ***
Do Not List
Drug Approval Process and
Orphan Drugs
*** All listed by Quebec 5
Drug Plan Listing Status of CDR
Recommendations
100%
Provincial Drug Plan listing status of the
33 New Drug recommendations from CDR
May 2004 – December 31 2005
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No. of Products
25
58%
No
20
15
42%
Yes
10
55%
24%
30%
27%
5
30%
27% 27%
15% 15% 15%
0%
Drug Approval Process and
iMAM®
Brogan
Inc.
December
2005
27 May 2006
Orphan Drugs
Q
C
B
N
IH
N
L
PE
N
S
N
B
O
N
M
B
SK
B
A
BC
To
ta
l
0
* Quebec does
not participate
6
CDR Decisions to Date (Aug.
24, 2005)
Decision
No.
submissons
List in a similar manner as drug plans list other
drugs of the same class
8
List with criteria/conditions
4
Not to list
16
Total decisions
28
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Provincial Response to CDR
Decisions
BC
AL
SK
MB
ON
NB
NS
PEI
NL
No. listing
decisions
1
5
10
2
2
0
13
0
1
No. listings
1
5
6
2
2
0
5
0
1
Average time to
listing (days)
284
349
351
310
328
--
302
--
300
Historical time to
listing (days)
450
406
346
551
494
592
428
744
352
Concordance
with CDR
recommendation
1/1
5/5
10/10
2/2
2/2
--
11/13
--
1/1
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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
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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)
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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
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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)
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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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Legitimacy with Decision Makers
Reformer (Member)
Broker (Independent)
Tactics
Legitimize Issue
Tactics
Influence
Evidence, Trade-offs
Success factors
Expertise, Compromise
Influence
Best Outcomes
Internal debate, temporary resolution,
policy change
Best Outcomes
Public debate, negotiate
Individual (Radical)
Tactics
Testimonials, Lawsuit
Influence
Sympathy, Guilt
Success factors
Personality, Integrity
Best Outcomes
Win Case, Raise awareness
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FEW
Informed public, expert support
Success factors
Diplomacy, public commitment
Win-win solution, commitment to change
Activist (Group)
Tactics
Demonstrations, Class Action
Influence
Confrontation, Media
Success Factors
Public support, Credible spokespeople
Best Outcomes
Support for Issue
Drug Approval Process and
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Drugs
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
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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
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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
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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
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 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
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