rare disease access issues 2015 v2
TRANSCRIPT
Durhane Wong-Rieger, PhD President,
Canadian Organizat ion for Rare Disorders
N o v e m b e r 2 0 1 5
Canada’s Rare Disease Strategy: Making It Work for Patients
November 2015 Rare Disease Drug Access
Rare Disease/Personalized Drug
Development
Win-Lose Drug Funding and Access
November 2015
Innovative Trial Designs
Pinnock, H. Pills, People, and Preferences: Evaluating Real Life Practice in Pragmatic Trials Rare Disease Drug Access
November 2015 Rare Disease Drug Access
View from HTA and Payers
November 2015 Rare Disease Drug Access
Promise of New Medicines
Reality of Access to New
Medicines
Rare Diseases: Unaddressed Public Health Issue
But Canada’s federation makes national approach a challenge!
2 in 3 are Children
80% Genetic
BUT
50% No Family
History
November 2015 Rare Disease Drug Access
# Canadians with Common vs. Rare Diseases
0
0.5
1
1.5
2
2.5
3
3.5 Milions Affected
Cancer Diabetes Heart disease Rare Diseases
2.4 M
1.4 M
2.8 M
1.8 M
November 2015 Rare Disease Drug Access
USA Orphan Drug Act: A Mother, a Congressman, an Actor
November 2015 Rare Disease Drug Access
Challenge of NO Orphan Drug Policy in Canada
� In 2002, US FDA approved a breakthrough drug, also called the “miracle drug” or “silver bullet” for rare cancer, chronic myeloid leukemia because a “complete hematological response” was achieved in 98% of patients. Imatinib was approved in Canada in 2007.
� In 2010, US FDA approved the first medicine that not only manages but prevents “painful swelling” in patients with hereditary angioedema. The drug was submitted to the EU but the small biotech firm had no intention of coming to Canada until a chance conversation on a plane from Poland convinced the VP to at least consider us. It was approved by Health Canada in 2013.
November 2015 Rare Disease Drug Access
Canadian Patients Do Not Access Many Orphan Drugs
� No definition, no orphan drug policy; few orphan drugs. ¡ Prior to 1983, only 10 new drugs approved worldwide for rare
diseases � Many Orphan Drugs in USA and EU
¡ In USA: about 500 products in 30 years since Orphan Drug Act ¡ In EU: about 100 new orphan drugs in 13 years ¡ Benefit: estimates of up to 15 million people
� Canadian patients have NO access to half of these drugs ¡ Canada has approved 47% of orphan drugs licensed in USA ¡ Canada has approved 51% of orphan drugs approved in Europe ¡ Almost half of approved drugs denied reimbursement in Public Drug
Plans
November 2015 Rare Disease Drug Access
Negotiations with Provincial Decision Maker on Managed Access/Risk Sharing
Rare Disease Drug Access
Health Canada Issues NOC & DIN
Manufacturer submits
NOC: Notice of Compliance –!DIN: Drug Identification Number!PMPRB: Patented Med Pricing Review Board!CDR: Common Drug Review!CDEC: Canadian Drug Expert Committee!pCODR: pan-CA Oncology Drug Review!PERC = pCODR Expert Review Committee!INESSS: Institut national d'excellence en santé et en services sociaux!PCPA = panCanadian Pricing Alliance!
Provincial Drug Committee Reviews Health Canada status, CDR/pCODR recommendation, Province-specific assessment
Provincial process recommends to reimburse (or not) through publicly funded program
Common Drug Review products (NCE / new combination product / new indication) CDEC recommendation to drug plans
Manufacturer submits
pCODR Products (NCE / new combination product / new indication) pERC recommendation to drug plans specific to oncology drugs
Up to 2 years Non-transparent
Up to 1 year Transparent
panCanadian Pharmaceutical Alliance (Pricing Negotiations)
Up to 2 mths Transparent
~ varied; not transparent
Open ended
PMPRB Reference-Based Ceiling Price Private Drug Plans
Provincial Exceptional Access Programs Ontario Drugs for Rare Diseases Alberta Special Access/Off-Label Drugs BC Rare Disease Drugs Committee New Brunswick Rare Disease Drugs Fund
INESSS for Quebec drug plan
Final Decision by Executive Officer/DOH/Minister November 2015
November 2015 Rare Disease Drug Access
HTA Related Challenges
Translate clinical controlled trials data into estimates of clinical effectiveness in a real-world setting
Limited evidence:
No control arm Small study population No validated outcome Disease heterogeneity
No long-term data
Uncertainty
HTA Related Challenges
Quantify healthcare costs, utilization and possible savings over the lifetime of the disease or treatment
Uncertainty
Poor data on economic burden and current management
Rapidly growing expenditures on OD
Poor cost- effectiveness of OD
Affordability Equity
HTA RELATED CHALLENGES
VALUE AND WHAT SOCIETY WANTS
NO TAILORED METHOD FOR ODs ¥ Most countries do not have HTA and/or pricing and
reimbursement-specific orphan drug (OD) decision framework
¥ Under usual circumstances ODs are not cost-effective
¥ Two approaches can be used to make it possible for orphan drugs to be considered cost-effective: ¤ Set higher ICER for ODs ¤ Apply weighted ICER criteria
¥ There is a variation in terms of the criteria that are considered by HTA agencies
¥ Evidence requirements differ between HTA agencies
HTA RELATED CHALLENGES
November 2015 Rare Disease Drug Access
Rare Disease Drugs: Challenges for Reimbursement
¥ Incremental Value Added (effectiveness, side effects, tolerability, improved quality of life) may not equal incremental costs
¥ Pricing criteria may not be established, and willingness to pay may have little impact on pricing
¥ Medicines for rare and unmet needs tend to have high R&D, high uncertainty, high cost
¥ Reimbursement strategies may be directed toward reducing uncertainty in safety, effectiveness, appropriate use, and budget impact.
¥ Managed access schemes include registries, CED, prior authorization, limited use, $ capitation.
November 2015 Rare Disease Drug Access
2005: CDR Denies Fabry & MPS-1 Drugs
Fabry and MPS patients protest at FPT Health Ministers’ Annual Meeting; demand access to life-saving drugs
Solution: Fabry’s Disease and MPS-1, “time-limited research program meeting treatment guidelines” on a risk-sharing basis.
Pilot for EXPENSIVE DRUGS FOR RARE DISORDERS? November 2015 Rare Disease Drug Access
2014: CDR “Yes” but Not at This Price = Delayed Access = Patient Protest
November 2015 Rare Disease Drug Access
November 2015 Rare Disease Drug Access
“...Saskatchewan decision sets a precedent in Canada because of the External Panel Review that reversed the province’s decision.”
Specialized Programs for Rare Disease Drugs
� 2010: Ontario Drugs for Rare Diseases Program evaluation framework using Markov Model to set access criteria and outcome benchmarks
� 2010-11: BC Rare Disease Committee review case-by-case applications based on evidence and physician submission
� 2011: Alberta Special Authorization Process for some rare disease drugs; Start-Stop criteria based on submitted evidence; additional $3 million fund for “off-label” or not Special Authorization drugs
� 2011-2015: Interprovincial joint action from panCanadian Purchasing Alliance to panCanadian Pricing Alliance to pC Pharmaceutical Alliance
� Quebec: Individual approval through “mesure du patient d’exception”; now joining pCPA
� 2012: Private drug plans (CLHIA) implements “pooled” insurance fund (full coverage plans only) for all drugs > $25k/year
November 2015 Rare Disease Drug Access
Idealized Life-Cycle Approach to Drug Development, Access and Continuous Review
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
Industry Data
November 2015 Rare Disease Drug Access
November 2015
Engaging Patients throughout Life-Cycle Improves Access and Appropriate Use
Pre-‐approval: Unmet need,
care pathways, resource impact (for
HTA)
During CTs: Advance/Special,
Crossover, Post-trial
access
HTA: Experience, values, A/E tolerance, start/stop
criteria
RW Use: Monitor
outcomes; adherence, feedback;
adjust therapy
Patient partners to improve
access criteria and support optimal use
Rare Disease Drug Access
Lifecycle Approach Sets Up Managed Access Programs
� Propose “coverage with evidence development” for orphan drugs ¡ Early approval based on life-threatening or severely debilitating
condition with no other effective treatments ¡ Regulatory approval for ODs require on-going data collection and
resubmission of outcomes data (5 years) ¡ Patient registries to collect post-market safety and effectiveness ¡ On-going studies with expanded patient population
� Challenge of “no funding” to expanded data collection ¡ Limits access to patients beyond clinical trials ¡ Limits “real world” data collection
November 2015 Rare Disease Drug Access
performance based schemes, managed entry agreements
outcome based
condi5onal coverage
performance linked reimbursement /
outcomes guarantee
financial based
pa5ent level
popula5on level condi5onal
treatment start or
con5nua5on
coverage with evidence
development
only in research
only with research
market share
price volume
u5liza5on or price cap
manufacturer funded
treatment ini5a5on
Ref: Sullivan S, ISPOR Paris, 2009 November 2015 Rare Disease Drug Access
November 2015 Rare Disease Drug Access
Thank You!
November 2015 Rare Disease Drug Access
Durhane Wong-Rieger, PhD President
Canadian Organization for Rare Disorders
www.raredisorders.ca 416-969-7435