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Canada’s Rare Disease Strategy: Pathway to Timely Sustainable Access CADTH Conference Durhane Wong-Rieger, PhD Canadian Organization for Rare Disorders President & CEO

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Canada’s Rare Disease Strategy: Pathway to Timely Sustainable Access

CADTH Conference

Durhane Wong-Rieger, PhDCanadian Organization for Rare Disorders

President & CEO

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Getting to Sustainable Access What is current status of access to drugs for rare

diseases in Canada? What is desirable for patients, prescribers, payers, and

producers? What are emerging international best practices? What are the opportunities with Health Canada, CADTH,

pCPA, private and public payers? What are the obstacles? What is a sustainable and workable way forward for


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Rare Diseases: Unaddressed Public Health Issue

But Canada’s federation makes national approach a challenge!

2 in 3 are Children80% Genetic

BUT 50% No

Family History

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# Canadians with Common vs. Rare Diseases







3Milions Affected


2.4 M

1.4 M

2.8 M

1.8 M

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5 Key Goals of Canada’s Rare Disease Strategy

1. Improving early detection and prevention

2. Providing timely, equitable and evidence-informed care

3. Enhancing community support

4. Providing sustainable access to promising therapies

5. Promoting innovative research

Feb 2016Canada Rare Disease Strategy Opportunities for Access

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Rare Disease Strategy Supports Transforming Therapies into Improved Care

Studies designed to promote understanding of effectiveness of interventions in real-world systems of care

Range of therapies and health services as co-interventions, such as diet therapy, physiotherapy, family support and counseling, telehealth, and interdisciplinary team care, specialized treatment centers, and care coordination

Identify long-term impacts of care, including potential adverse effects

Determine how real patients and disease characteristics influences treatment effectiveness; account for clinical heterogeneity

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Traditional HTA Limits Orphan Drug Access

• HTA OFTEN same as for common drugs• RCTs = small samples, short timeframes, surrogate

markers• High $ development, small population = High $/patient• Cost-utility: $/QALY below theoretical $50k threshold• Small # = Low budget impact

• HTA recommends “no” to most drugs for rare disorders• Private drugs plans usually cover (but some do not)• Public drug plans usually adopt HTA recommendations

• HTA limits access in some European countries• Half of all orphan drugs evaluated have been rejected

by one or more HTA bodies in the UK.  • Where Cost-Effectiveness NOT norm, reimbursement

varies; best = 80% to 100% funding


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Canadian (Limited) Access to Rare Disease Drugs

Drug Indication recommend Access

Replagal Fabry’s DNL 3 Yr ResearchZavesca Gaucher’s DNL DNR/CBC

Fabrazyme Fabry’s DNL 3 Yr ResearchAldurazyme MPS I DNL DNR/ON SP/CBCSomavert Acromegaly DNL DNR/CBC:ON

Exjade Iron Overload LWC DNR/LWC AB BC/CBC ON)

Nexavar Kidney Cancer DNL CBCSutent (Renal) Kidney Cancer DNL CBC

Sutent (GSIT) GIST LWC CBCMyozyme Pompe’s LWC DNR/CBCElaprase MPS II DNL DNR/CBCXyrem Narcolepsy DNL DNR

Revlimid Myelodysplastic Syndome JODR - DNL CBCDNL = Do not List; LWC/C = List with Criteria/Conditions; LSM – List Similar MannerDNR = Do not Reimburse; CBC = ICase-by-case; SP = Special Program; UR = Under Review

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Canadian (Limited) Access to Rare Disease Drugs Drug Indication recommend AccessSoliris Paroxysmal Nocturnal

Hemoglobinuria DNL DNR/LWC:ON/CBC:AB MB

Naglazyme MPS VI Not Submitted CBC:ON

Cayston Cystic Fibrosis LWC URIlaris Cryopyrin Associated Syndrome DNL DNR/CBC:ON

Kuvan Phenyketonuria DNL DNR/SP:ONVolibris Pulmonary arterial hypertension LWC CBC/LWC:QC PEIVimizim Morquio (MPS IV) DNL DNR

Revolade Idiopathic pulmonary fibrosis DNL DNR (LWC:QC)Afinitor Tubersclerosis Complex - Renal DNL DNR/UR/SP:ONAfinitor Tubersclerosis Complex SEGA DNL DNR/URFirazyr Hereditary Angeiodema LWC/C UR/LWC:QC

Somatuline Acromegaly LSM LSMJuxtapid Homozygous Familial

HypercholesterolemiaDNL DNR/UR

DNL = Do not List; LWC/C = List with Criteria/Conditions; LSM – List Similar MannerDNR = Do not Reimburse; CBC = ICase-by-case; SP = Special Program; UR = Under Review

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Canadian Limited Access to Rare Disease Drugs Drug Indication recommend Access

Opsumit Pulmonary arterial hypertension LWCC UR

Mozobil Hematopoietic stem cell mobilizer


Zaxine Hepatic encephalopathy LWC/C LWC:QC/URAdempas Thromboembolic pulmonary

hypertsnsion LWC LWCSoliris Hemolytic uremic syndrome DNL DNR/UROfev Idiopathic pulmonary fibrosis LWC UR

Signifor Cushing’s Disease DNL DNR/URAdcirca Pulmonary arterial hypertension LSM DNR/URElelyso Gaucher’s Disease DNL UR

Actemra Polyarticular juvenile idiopathic arthritis LWC LWR/UR

Remodulin Pulmonary arterial hypertension DNL DNR/CBC:ONVPRIV Gaucher’s Disease LWC DNR/SP:ON BC/UR

DNL = Do not List; LWC/C = List with Criteria/Conditions; LSM – List Similar MannerDNR = Do not Reimburse; CBC = Case-by-case; SP = Special Program; UR = Under Review

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Int’l HTA Limits Equity of AccessAustralia (PBAC)

Canada (CADTH)

England (NICE/AGNSS)

France (HAS)

Scotland (SMC)

Kalydeco: cystic fibrosis with G551D mutation

Recommend (March 2014)

Restricted (March 2013)

Funded NHS; review Clinical Priorities Advisory Group (2012)

Recommed (Nov. 2012)

Not recommend (Jan 2013); via Orphan Drug Fund

Soliris: paroxysmal nocturnal hemoglobinuria

Not recommend (Mar 2009); eligible Life Savings Drug Program (2010)

Not recommend (Feb. 2010)

Not recommend (Feb. 2010)

Recommend (Oct. 2007)

Not recommend (July 2011)

Elaprase: Hunter syndrome (MPS II)

Not recommend (Nov. 2007); eligible Life Savings Drug Program (2012)

Not recommend (Dec. 2007)

(Not reviewed by NICE)

Recommend (March 2007)

Not recommend (July 2007)

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ut d



We need timely access to drugs for unmet & urgent needs

We want certainty about safety and benefits of drugs

Balancing Timely Access and Certainty of Benefits/Risks

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Adaptive pathways• Aim to provide timely access to new medicines by balancing

medical need with evolving information on the benefit risk



ng N


r of P




Intensive monitoring of patients

Additional indication(s)

Intensive monitoring of patientsInitial approval of niche indication


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Key Obstacles• MAPPs: perception that earlier access for (some) patients

with limited data could lead to safety problems and undermine public trust

• More drugs for smaller patient subpopulations => not sustainable; unwillingness to pay for early access with limited data

• Need tools to limit access to approved subset (avoid off- label use)

• Practicalities and cost of implementing flexible or outcome based reimbursement strategies

• Extra work for regulators, HTA bodies, and payer due to repeat cycles of assessment and negotiations with sponsors

• Need capacity building and support for all stakeholders in order to fully integrate contribution of patients and patient organizations across drug life cycle

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The real challenges seem to be more associated with efficacy than with safety

Major reasons for late-stage failures (n = 73)

• Efficacy is the most frequent reason for failures in late-stage development

• This is not related to limited

as 22 examples of top-10 pharmacos reveal

• Efficacy failures are largely influenced/ enhanced by– Novelty of mechanism

of action(MoA)

– Objectivity of clinical trial endpoint

– Modality• Novel MoA bears double


• Less objective endpoints (e.g. PROs) on average lead to ~10% increased failure risk

Efficacy(vs. placebo)

Safety(vs. placebo)

Lack of differentiation



Confirmation of early safety


Non classifiable










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Lifecycle Approach with Patient Input & Post-Market/Real World Data

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

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Thank You!


Durhane Wong-Rieger, PhD


Canadian Organization for Rare Disorders



[email protected]