How Canadians Access Drugs for Rare Disorders Health Canada and Provincial Drug Plans Durhane Wong-Rieger, PhD President Canadian Organization for Rare.

How Canadians Access Drugs for Rare Disorders

Health Canada and Provincial Drug PlansDurhane Wong-Rieger, PhD

PresidentCanadian Organization for Rare Disorders

27 May 2006 Drug Approval Process and Orphan Drugs

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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 ApplicationCompany decides to “sell” drug in Canada; applies

for specific indications; pays application feeIf “breakthrough”, can be given “priority review”

Company submits evidence from clinical trialsIs 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 diseaseReduce major symptoms, fewer side effects,

significantly easier to useGives more years of life

Cost per additional years of lifeAre the additional benefits worth the additional costs? Is it as cost-effective as previous drugs


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List/List with Restrictions Do Not List

Avodart Axert Combigan Reyataz Humira Myfortic Neulasta Pegasys RBV Teveten Plus Telzir Vfend Yasmin Tarceva Kivexa

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 ***

42%

58%

*** All listed by Quebec

1419

33 CDR Recommendations as of December 31, 2005


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0

5

10

15

20

25

30

No. of Products

TotalBC AB SK MB ON NB NS PE NL

NIHBQC

Provincial Drug Plan listing status of the 33 New Drug

recommendations from CDR May 2004 – December 31 2005

24%

27%30%

15% 15% 15%

30%

0%27% 27%

100%

55% 42%Yes

58%No

* Quebec does not participate

iMAM® Brogan Inc. December 2005

Drug Plan Listing Status of CDR Recommendations


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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

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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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FEW PUBLICGROUP

Reformer (Member)

TacticsLegitimize Issue

InfluenceEvidence, Trade-offs

Success factorsExpertise, Compromise

Best OutcomesInternal debate, temporary resolution,policy change

Individual (Radical)

TacticsTestimonials, Lawsuit

InfluenceSympathy, Guilt

Success factorsPersonality, Integrity

Best OutcomesWin Case, Raise awareness

Activist (Group)

TacticsDemonstrations, Class Action

InfluenceConfrontation, Media

Success FactorsPublic support, Credible spokespeople

Best OutcomesSupport for Issue

Broker (Independent)

TacticsPublic debate, negotiate

InfluenceInformed public, expert support

Success factorsDiplomacy, public commitment

Best OutcomesWin-win solution, commitment to change

Legitimacy with Decision Makers

Credibility Among Consumers (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 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

How Canadians Access Drugs for Rare Disorders Health Canada and Provincial Drug Plans Durhane Wong-Rieger, PhD President Canadian Organization for Rare.

Documents

drug approval process

drug budgets

orphan drug policy

drug safe

drug work

new drug recommendations

drug access health canada

orphan conditions