Transcript Summit on Access to Drugs for Rare Diseases

Canada’s Rare Disease Strategy: Pathway
to Timely Sustainable Access
CADTH Conference
Durhane Wong-Rieger, PhD
Canadian Organization for Rare Disorders
President & CEO
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
Canada?
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Rare Diseases: Unaddressed Public Health Issue
But Canada’s federation makes
national approach a challenge!
# Canadians with Common vs. Rare Diseases
Milions Affected
3.5
3
2.8 M
2.5
2
1.5
1
0.5
0
Cancer
2.4 M
Diabetes
Heart disease
1.8 M
1.4 M
Rare Diseases
5 Key Goals of
Canada’s Rare Disease Strategy
Canada Rare Disease Strategy
Opportunities for Access
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 2016
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
Replagal
Indication
Fabry’s
recommend
DNL
Access
3 Yr Research
Zavesca
Gaucher’s
DNL
DNR/CBC
Fabrazyme
Fabry’s
DNL
3 Yr Research
Aldurazyme
MPS I
DNL
DNR/ON SP/CBC
Somavert
Acromegaly
DNL
DNR/CBC:ON
Exjade
Iron Overload
LWC
DNR/LWC AB BC/CBC ON)
Nexavar
Kidney Cancer
DNL
CBC
Sutent (Renal)
Kidney Cancer
DNL
CBC
Sutent (GSIT)
GIST
LWC
CBC
Myozyme
Pompe’s
LWC
DNR/CBC
Elaprase
MPS II
DNL
DNR/CBC
Xyrem
Narcolepsy
DNL
DNR
Revlimid
Myelodysplastic Syndome
JODR - DNL
CBC
DNL = Do not List; LWC/C = List with Criteria/Conditions; LSM – List Similar Manner
DNR = Do not Reimburse; CBC = ICase-by-case; SP = Special Program; UR = Under Review
Canadian (Limited) Access to Rare Disease Drugs
Drug
Indication
recommend
Access
Soliris
Paroxysmal Nocturnal Hemoglobinuria
DNL
DNR/LWC:ON/CBC:AB MB
Naglazyme
MPS VI
Not Submitted
CBC:ON
Cayston
Cystic Fibrosis
LWC
UR
Ilaris
Cryopyrin Associated Syndrome
DNL
DNR/CBC:ON
Kuvan
Phenyketonuria
DNL
DNR/SP:ON
Volibris
Pulmonary arterial hypertension
LWC
CBC/LWC:QC PEI
Vimizim
Morquio (MPS IV)
DNL
DNR
Revolade
Idiopathic pulmonary fibrosis
DNL
DNR (LWC:QC)
Afinitor
Tubersclerosis Complex - Renal
DNL
DNR/UR/SP:ON
Afinitor
Tubersclerosis Complex SEGA
DNL
DNR/UR
Firazyr
Hereditary Angeiodema
LWC/C
UR/LWC:QC
Somatuline
Acromegaly
LSM
LSM
Juxtapid
Homozygous Familial
Hypercholesterolemia
DNL
DNR/UR
DNL = Do not List; LWC/C = List with Criteria/Conditions; LSM – List Similar Manner
DNR = Do not Reimburse; CBC = ICase-by-case; SP = Special Program; UR = Under Review
Canadian Limited Access to Rare Disease Drugs
Drug
Indication
recommend
Access
Opsumit
Pulmonary arterial hypertension
LWCC
UR
Mozobil
Hematopoietic stem cell mobilizer
DNL
DNR/SP/LWC
Zaxine
Hepatic encephalopathy
LWC/C
LWC:QC/UR
Adempas
LWC
LWC
Soliris
Thromboembolic pulmonary
hypertsnsion
Hemolytic uremic syndrome
DNL
DNR/UR
Ofev
Idiopathic pulmonary fibrosis
LWC
UR
Signifor
Cushing’s Disease
DNL
DNR/UR
Adcirca
Pulmonary arterial hypertension
LSM
DNR/UR
Elelyso
Gaucher’s Disease
DNL
UR
Actemra
Polyarticular juvenile idiopathic
arthritis
LWC
LWR/UR
Remodulin
Pulmonary arterial hypertension
DNL
DNR/CBC:ON
VPRIV
Gaucher’s Disease
LWC
DNR/SP:ON BC/UR
DNL = Do not List; LWC/C = List with Criteria/Conditions; LSM – List Similar Manner
DNR = Do not Reimburse; CBC = Case-by-case; SP = Special Program; UR = Under Review
Int’l HTA Limits Equity of Access
Australia
(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
hemoglobinu
ria
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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Balancing Timely Access and Certainty of Benefits/Risks
Evidence about drug
We want certainty
about safety and
benefits of drugs
We need timely access
to drugs for unmet &
urgent needs
Time
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Adaptive pathways
Increasing Number of Patients
• Aim to provide timely access to new medicines by balancing
medical need with evolving information on the benefit risk
FULL APPROVAL
Intensive monitoring of patients
Additional indication(s)
Intensive monitoring of
patients
Initial approval
of niche
indication
Time
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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
The real challenges seem to be more
associated with efficacy than with safety
Major reasons for late-stage failures (n = 73)
50%
Efficacy
(vs. placebo)
Comment
50%
• Efficacy is the most frequent
reason for failures in late-stage
development
• This is not related to limited
Safety
(vs. placebo)
Confirmation of
early safety
concerns
8%
31%
23%
Non classifiable
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
16%
Efficacy
Lack of
differentiation
19%
Safety
3%
– Modality
• Novel MoA bears double failure
risk
• Less objective endpoints (e.g.
PROs) on average lead to ~10%
increased failure risk
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Lifecycle Approach with Patient Input
& Post-Market/Real World Data
Researcher/Clini
cian: Disease
Knowledge;
Drug Discovery;
Treatment
Guidelines
Payer: Budget
Impact; Access
Criteria; R-W
Data Collection
Patient Input
Company:
Clinical Trials &
Outcome
Measures;
biomedical,
clinical, PROs,
Real-World
Impact
HTA:
Comparison
Benefits, Risks,
Cost
w/Alternatives;
Place in Therapy
Regulator:
Approval on
Benefits-RisksUncertainties;
Use & RealWorld
Monitoring
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Thank You!
Durhane Wong-Rieger, PhD
President
Canadian Organization for Rare Disorders
www.raredisorders.ca
416-969-7435
[email protected]
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